Podcasts about fviii

Hemophilia A (HA) is an X-linked bleeding disorder caused by a mutation in the factor 8 (F8) gene that codes for FVIII coagulation protein. FVIII is naturally synthesized in the liver and the mutation results in the loss of function of FVIII protein which is critical in the blood clotting cascades. HA occurs is 1 to 5000 male births and it is estimated that there are a total of 3000 patients with HA in Canada. Currently, HA patients are treated with replacement therapy of the deficient factor. However, this approach is transient because of the short half-life of recombinant or plasma derived FVIII and is insufficient due to the formation of anti-drug antibodies. The alternative approach which is curative and promises a long-lasting expression of FVIII protein is gene therapy. For upcoming interviews check out the Grad Chat webpage on Queen’s University School of Graduate Studies & Postdoctoral Affairs website .

On this episode of the Global Hemophilia Report, Senior Advisor Dr. Donna DiMichele leads a comprehensive discussion on FVIII inhibitors—a critical complication in Hemophilia A. The episode features experts such as Dr. Kathleen Pratt, Dr. David Lillicrap, Dr. Bhavya Doshi, Dr. Carol Miao, and Dr. Radek Kazmarek who explore the intricacies of immune responses, gene therapy, and novel therapies like emicizumab. They delve into factors like antigen-presenting cells, B cells, T cells, microbiome effects, and glycans that influence inhibitor development and tolerance. Despite advancements in treatment, the experts underscore the enduring importance of ongoing research in understanding and preventing FVIII inhibitors.   Contributors: Glaivy Batsuli, MD Bhavya Doshi, MD David Lillicrap, MD Carol Miao, PhD Kathleen Pratt, PhD Radek Kaczmarek, PhD   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Listen to the Global Hemophilia Report Episode 1: Inhibitors: Prevention, Eradication, and Lived Experiences   Conflicts of Interest: Dr. Kathleen Pratt is an inventor on patents related to factor VIII immunogenicity, and she has received research funding in the past from pharmaceutical companies that manufacture human FVIII. She has no other relevant conflicts to report.   Disclaimer: The contents of Dr. Pratt's presentation are the sole responsibility of the speaker and do not necessarily reflect the views, opinions or policies of Uniformed Services University of the Health Sciences (USUHS), The Henry M. Jackson Foundation for the Advancement of Military Medicine, Inc., the Department of Defense (DoD) or the Departments of the Army, Navy, or Air Force. Mention of trade names, commercial products, or organizations does not imply endorsement by the U.S. Government.    Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter  

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The topic of discussion for this episode leans more toward the scientific side but has huge clinical relevance. For today, we will be asking our expert panel to lend their experience and expertise to the topic of FVIII and FIX, which we will be discussing from several related perspectives.    Contributors: Radek Kaczmarek, PhD, MSc; University of Indiana, USA Peter Lenting, PhD; INSERM, Paris, France Ben Samuelson -Jones, M.D., PhD; CHOP Research Institute, USA Courtney Thornburg, M.D., MS; Rady Children's Hospital, UCSD, USA   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/SFD860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this new PeerView activity based on a recorded symposium at the 75th Annual Bleeding Disorders Conference, an expert panel engages in thought-provoking, interactive case-based discussions and offers guidance on how healthcare professionals can address real-world challenges of hemophilia A management with novel EHL FVIII prophylaxis. Our experts provide guidance on critically evaluating the latest clinical evidence, tailoring treatment plans to individual patient needs, and navigating the challenges associated with patient adherence, access to therapy, and transitioning patients from conventional options (eg, SHL FVIII replacement therapy) to innovative EHL FVIII prophylaxis. Access this activity now to see if you are prepared to lead the way to better outcomes for hemophilia A! Upon completion of this activity, participants should be better able to: Summarize the mechanistic differences and latest clinical evidence with novel EHL FVIII replacement agents for hemophilia A; Identify unmet medical needs and barriers to optimal prophylaxis of hemophilia A; Integrate novel EHL FVIII replacement therapies into personalized treatment regimens for patients with hemophilia A based on clinically-relevant factors, current evidence, and guideline recommendations, among other considerations; and Develop strategies to address practical aspects of care, such as dosing, safety considerations, monitoring, patient education, and adherence counseling, when using novel EHL FVIII agents

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Go online to PeerView.com/WEM860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Are you aware of the evidence supporting the use of innovative EHL FVIII therapies for the prophylactic management of hemophilia A? Find out in this activity based on a “MasterClass” event recorded at the 2023 Congress of the International Society on Thrombosis and Haemostasis. Hear what hemophilia experts are saying about the latest clinical evidence that supports the use of innovative EHL FVIII therapy in hemophilia as they link the data directly to case-based decisions in real-world settings—with a goal of clearly illustrating the practicalities of safely using novel and emerging factor products. The experts also provide guidance on implementing evidence-based protocols and preventing and managing complications in the context of EHL FVIII therapy. Upon completion of this activity, participants should be better able to: Assess the latest safety, efficacy, and pharmacokinetic data among currently approved and emerging EHL FVIII products for prophylaxis of hemophilia A; Develop personalized prophylactic regimens with novel and emerging EHL FVIII products based on patient- and disease-specific factors; and Manage practical aspects of care, such as patient education, adherence counseling, monitoring, and AE management, when using novel and emerging EHL FVIII products for prophylaxis of hemophilia A

Neste episódio teremos o bate-papo entre Dra. Ieda Pinto, Dra. Claudia Lorenzato e Dra Christiane Pinto, com a moderação da Dra. Ana Clara Kneese falando sobre o contexto médico da disponibilidade das novas tecnologias de FVIII recombinante de longa ação, expiração do prazo de disponibilidade das tecnologias ao SUS após 180 dias, implicações, impactos, expectativas, gerenciamento das expectativas dos tratadores, equipe multidisciplinar e necessidade dos pacientes.

Prophylaxis in hemophilia has led to extraordinary progress, however, recent studies suggest that although the overall musculoskeletal health of persons with hemophilia has significantly improved, the goal of attaining a level of musculoskeletal health comparable to that of the general population without hemophilia has still not been achieved. Furthermore, the impact of prophylaxis has been uneven across hemophilia populations and the promise of greater physical activity free of hemorrhage has not been entirely fulfilled. This episode explores the history, evolution, and current state of prophylaxis, and highlights the current and future areas of further investigation.   Contributors: Episode Advisors: Dr. Marilyn Manco Johnson Dr. Kathelijn Fischer   Contributors: Dr. Robert Sidonio Dr. Manuel Carcao  Dr. Maria Elisa Mancuso Susan Lynch   Senior Advisor: Dr. Donna DiMichele   Links to learn more: A history of prophylaxis in haemophilia Hemophilia A and B: Routine management including prophylaxis Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens Optimising prophylaxis in haemophilia A: The ups and downs of treatment Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Inhibitor development has long been considered the most significant complication of severe hemophilia, but with the licensure of the first non-factor therapy and a robust pipeline of novel and gene therapeutics being investigated, is it still of critical importance that we better understand inhibitors? If so, what research is currently taking place, and what can he hope to learn from it? This episode of the Global Hemophilia Report from BloodStream Media features contributions and citations from investigators, clinicians, and research funders dedicated to this topic, as well as from patients and caregivers who can speak to their lived experience of inhibitors.   For more on inhibitor research: Inhibitors and Hemophilia - CDC ATHN 8: PUPs Matter Study - ATHN The Hemophilia Inhibitor Eradication Trial - ClinicalTrials.gov A New Plan for Tackling Inhibitors - NHF ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders - ASH The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development - ASH   Show Notes: Featured Advertiser: Sanofi Genzyme Subscribe to BloodStream Media's Global Hemophilia Report Senior Adviser: Dr. Donna DiMichele   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

The prospect of a clinical strategy using an adeno-associated virus (AAV) vector for expression of therapeutic levels of factor VIII (FVIII) has been highly desirable. This was initially anticipated by promising data from clinical studies on AAV5-FVIII in men with severe hemophilia A. However, long-term follow-up showed a unique efficacy concern on the sustainability and durability derived from a continuous decline in the FVIII transgene levels starting 1 year after vector injection through year 5. Additional follow-up of early-phase studies and outcomes of an ongoing phase 3 study will likely provide evidence on the feasibility of this approach. Here, the potential underlying mechanisms of the FVIII declining levels, together with the revision of several unique early and late onset findings, are discussed. The lack of long-term preclinical studies in large animal models prevents the firm conclusion that FVIII levels decline was unexpected. It is possible that the combination of vector manufacturing platform and dose, accompanied with ectopic expression of supraphysiologic levels of FVIII at short-term follow-up, may all contribute to the sustainability and durability of the transgene levels. Notably, vector readministration to further improve the FVIII levels is not feasible at this time. Thus, the need of a one-and-done AAV strategy to achieve sustain FVIII levels of expression is sine qua non to impact favorably the disease phenotype.

In this episode, listen to Guy A. Young, MD, as he counsels a teenage patient with severe hemophilia A and the patient's mother on various available treatment options and strategies for optimizing hemophilia A prophylaxis and adherence. Topics include:Overcoming challenges to adherenceChoice of standard vs extended half-life factor VIII vs emicizumabSafety and efficacy data with emicizumabPresenter:Guy A. Young, MDProfessor of PediatricsDirector, Hemostasis and Thrombosis CenterChildren's Hospital Los AngelesUniversity of Southern California Keck School of MedicineLos Angeles, CaliforniaLink to full program:https://bit.ly/3FxrVk2

CME credits: 0.50 Valid until: 25-02-2022 Claim your CME credit at https://reachmd.com/programs/cme/personalizing-prophylaxis-hemophilia-and-b-new-era-treatment-and-management/12152/ Hemophilia A and hemophilia B are congenital X-linked bleeding disorders caused by deficiency of coagulation factor VIII (FVIII) and IX (FIX). While the life expectancy of patients with hemophilia has dramatically increased over the past several decades, the hemophilia population still faces unique challenges that require appropriate use of effective strategies. The preferred treatment option for patients with hemophilia is replacement therapy. Prophylaxis with standard recombinant factor requires regular intravenous infusion at least two (FIX) to three (FVIII) times a week. Recombinant FVIII and FIX products with an extended half-life are currently available and newer strategies are on the horizon. With reported mean half-life extensions of 1.5–1.8 times that of standard products for FVIII and 3–5 times that of standard products for FIX, these therapies have the potential to address and individualize the needs of patients. Click here to access patient information on hemophilia from the CDC.

CME credits: 0.50 Valid until: 25-02-2022 Claim your CME credit at https://reachmd.com/programs/cme/personalizing-prophylaxis-hemophilia-and-b-new-era-treatment-and-management/12152/ Hemophilia A and hemophilia B are congenital X-linked bleeding disorders caused by deficiency of coagulation factor VIII (FVIII) and IX (FIX). While the life expectancy of patients with hemophilia has dramatically increased over the past several decades, the hemophilia population still faces unique challenges that require appropriate use of effective strategies. The preferred treatment option for patients with hemophilia is replacement therapy. Prophylaxis with standard recombinant factor requires regular intravenous infusion at least two (FIX) to three (FVIII) times a week. Recombinant FVIII and FIX products with an extended half-life are currently available and newer strategies are on the horizon. With reported mean half-life extensions of 1.5–1.8 times that of standard products for FVIII and 3–5 times that of standard products for FIX, these therapies have the potential to address and individualize the needs of patients. Click here to access patient information on hemophilia from the CDC.

In this episode, Michael Callaghan, MD; Miguel A. Escobar, MD; and Rebecca Kruse-Jarres, MD, MPH, answer questions from a clinician audience on topics related to hemophilia management including:Maintaining long-term musculoskeletal healthIdentifying patients most likely to benefit from pharmacokinetics-guided prophylaxisManaging combination therapies in older patients with comorbiditiesYoungest age at which patients can be considered for emicizumabDevelopment of resistance to emicizumabSwitching recombinant factor therapiesUsing bypassing agents with emicizumab for breakthrough bleeds in patients with inhibitorsPotential clinical role of extending FVIII half-life with BIVV001Considerations when dosing emicizumab monthlyPresenters:Michael Callaghan, MDAssociate ProfessorDepartment of HematologyWayne State UniversityHematologistDivision of Hematology/OncologyChildren’s Hospital of MichiganDetroit, MichiganMiguel A. Escobar, MDProfessor of Medicine and PediatricsDepartment of HematologyUniversity of Texas Health Science CenterMcGovern Medical SchoolDirectorGulf States Hemophilia and Thrombophilia CenterHouston, TexasRebecca Kruse-Jarres, MD, MPHProfessorDepartment of MedicineUniversity of WashingtonExecutive/Medical DirectorWashington Institute for CoagulationSeattle, WashingtonContent supported by educational grants from Genentech, a member of the Roche Group; Sanofi Genzyme Corporation; and Takeda Pharmaceutical Company Ltd.Link to full program, including downloadable slidesets, expert commentaries, and on-demand Webcast: http://bit.ly/2MW5uhi

Experts discuss the use of PK studies to optimize prophylaxis regimens in children with hemophilia A. Earn Credit / Learning Objectives & Disclosures: https://www.medscape.org/viewarticle/940993?src=mkm_podcast_addon_940993

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Go online to PeerView.com/CEV860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. In this activity, experts in hemophilia discuss the use of extended half-life replacement factor VIII products for the treatment of patients with hemophilia A. Upon completion of this activity, participants will be able to: Describe the benefits and limitations of conventional replacement factor VIII regimens for the management of hemophilia A, Assess the latest clinical data regarding efficacy, safety, and tolerability of extended half-life replacement factor VIII products for the treatment of hemophilia A, Incorporate extended half-life replacement FVIII regimens into the management of patients with hemophilia A, Discuss the importance of a collaborative approach among physicians, pharmacists, and other clinicians in the management of patients with hemophilia A.

Presenting a NEW BloodLine podcast, “FVIII Inhibitors: The Next Chapter.” In our third episode, we speak to community members and advocates about their hopes for the future of FVIII inhibitor treatment as well as local and nationwide resources available to FVIII inhibitor patients. “FVIII Inhibitors: The Next Chapter” is a three-part podcast series looking at the current and future state of living with hemophilia A with factor VIII inhibitors. Special, huge thanks to our guests: Justin Levesque Dr. Guy Young Sue Geraghty Liz Purvis Eric Lowe Janet Brewer and Benjamin Denman BloodLine—FVIII Inhibitors: The Next Chapter is produced by me, Patrick James Lynch, and Ryan Gielen. Written by Ryan Gielen. Edited by Josh Davis. Produced at BloodStream Media's Los Angeles studio, and mixed by Michael Capuano at Icemen Sound. For more information on the podcast, visit our website at www.BloodLinePod.com. This series was produced by Believe Limited in collaboration with Genentech, the makers of HEMLIBRA® (emicizumab-kxwh). What is HEMLIBRA? HEMLIBRA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors. What is the most important information I should know about HEMLIBRA? HEMLIBRA increases the potential for your blood to clot. Carefully follow your healthcare provider’s instructions regarding when to use an on-demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. HEMLIBRA may cause serious side effects when used with activated prothrombin complex concentrate (aPCC; FEIBA®), including thrombotic microangiopathy (TMA), and blood clots (thrombotic events). If aPCC (FEIBA®) is needed, talk to your healthcare provider in case you feel you need more than 100 U/kg of aPCC (FEIBA®) total. How should I use HEMLIBRA? • Stop (discontinue) prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. • You may continue prophylactic use of factor VIII for the first week of HEMLIBRA prophylaxis. What should I know about lab monitoring? HEMLIBRA may interfere with laboratory tests that measure how well your blood is clotting and may cause a false reading. Talk to your healthcare provider about how this may affect your care. The most common side effects of HEMLIBRA include: redness, tenderness, warmth, or itching at the site of injection; headache; and joint pain. These are not all of the possible side effects of HEMLIBRA. Speak to your healthcare provider for medical advice about side effects. You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at (888) 835-2555. Please see the HEMLIBRA full Prescribing Information and Medication Guide for Important Safety Information, including Serious Side Effects, here: bit.ly/2kp959s HEMLIBRA® is a registered trademark of Chugai Pharmaceutical Co., Ltd., Tokyo, Japan. The Genentech logo is a registered trademark of Genentech, Inc. © 2019 Genentech USA, Inc. All rights reserved. EMI/053019/0107e 07/19

Presenting a NEW BloodLine podcast, “FVIII Inhibitors: The Next Chapter.” In our second episode, we learn about the ten-year journey to bring HEMLIBRA® (emicizumab-kxwh) to market and take a deep look at the clinical trials of HEMLIBRA. “FVIII Inhibitors: The Next Chapter” is a three-part podcast series looking at the current and future state of living with hemophilia A with factor VIII inhibitors. Special, huge thanks to our guests: Justin Levesque Dr. Guy Young Sue Geraghty Liz Purvis Eric Lowe Janet Brewer and Benjamin Denman BloodLine—FVIII Inhibitors: The Next Chapter is produced by me, Patrick James Lynch, and Ryan Gielen. Written by Ryan Gielen. Edited by Josh Davis. Produced at BloodStream Media's Los Angeles studio, and mixed by Michael Capuano at Icemen Sound. For more information on the podcast, visit our website at www.BloodLinePod.com. This series was produced by Believe Limited in collaboration with Genentech, the makers of HEMLIBRA® (emicizumab-kxwh). What is HEMLIBRA? HEMLIBRA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors. What is the most important information I should know about HEMLIBRA? HEMLIBRA increases the potential for your blood to clot. Carefully follow your healthcare provider’s instructions regarding when to use an on-demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. HEMLIBRA may cause serious side effects when used with activated prothrombin complex concentrate (aPCC; FEIBA®), including thrombotic microangiopathy (TMA), and blood clots (thrombotic events). If aPCC (FEIBA®) is needed, talk to your healthcare provider in case you feel you need more than 100 U/kg of aPCC (FEIBA®) total. How should I use HEMLIBRA? • Stop (discontinue) prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. • You may continue prophylactic use of factor VIII for the first week of HEMLIBRA prophylaxis. What should I know about lab monitoring? HEMLIBRA may interfere with laboratory tests that measure how well your blood is clotting and may cause a false reading. Talk to your healthcare provider about how this may affect your care. The most common side effects of HEMLIBRA include: redness, tenderness, warmth, or itching at the site of injection; headache; and joint pain. These are not all of the possible side effects of HEMLIBRA. Speak to your healthcare provider for medical advice about side effects. You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at (888) 835-2555. Please see the HEMLIBRA full Prescribing Information and Medication Guide for Important Safety Information, including Serious Side Effects, here: bit.ly/2kp959s HEMLIBRA® is a registered trademark of Chugai Pharmaceutical Co., Ltd., Tokyo, Japan. The Genentech logo is a registered trademark of Genentech, Inc. © 2019 Genentech USA, Inc. All rights reserved. EMI/053019/0107e 07/19

Presenting a NEW BloodLine podcast, “FVIII Inhibitors: The Next Chapter.” In our first episode, we meet patients from around the country who live with hemophilia A with factor VIII inhibitors and hear about their journeys first hand. “FVIII Inhibitors: The Next Chapter” is a three-part podcast series looking at the current and future state of living with hemophilia A with factor VIII inhibitors. Special, huge thanks to our guests: Justin Levesque Dr. Guy Young Sue Geraghty Liz Purvis Eric Lowe Janet Brewer and Benjamin Denman BloodLine—FVIII Inhibitors: The Next Chapter is produced by me, Patrick James Lynch, and Ryan Gielen. Written by Ryan Gielen. Edited by Josh Davis. Produced at BloodStream Media's Los Angeles studio, and mixed by Michael Capuano at Icemen Sound. For more information on the podcast, visit our website at www.BloodLinePod.com. This series was produced by Believe Limited in collaboration with Genentech, the makers of HEMLIBRA® (emicizumab-kxwh). What is HEMLIBRA? HEMLIBRA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors. What is the most important information I should know about HEMLIBRA? HEMLIBRA increases the potential for your blood to clot. Carefully follow your healthcare provider’s instructions regarding when to use an on-demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. HEMLIBRA may cause serious side effects when used with activated prothrombin complex concentrate (aPCC; FEIBA®), including thrombotic microangiopathy (TMA), and blood clots (thrombotic events). If aPCC (FEIBA®) is needed, talk to your healthcare provider in case you feel you need more than 100 U/kg of aPCC (FEIBA®) total. How should I use HEMLIBRA? • Stop (discontinue) prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. • You may continue prophylactic use of factor VIII for the first week of HEMLIBRA prophylaxis. What should I know about lab monitoring? HEMLIBRA may interfere with laboratory tests that measure how well your blood is clotting and may cause a false reading. Talk to your healthcare provider about how this may affect your care. The most common side effects of HEMLIBRA include: redness, tenderness, warmth, or itching at the site of injection; headache; and joint pain. These are not all of the possible side effects of HEMLIBRA. Speak to your healthcare provider for medical advice about side effects. You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at (888) 835-2555. Please see the HEMLIBRA full Prescribing Information and Medication Guide for Important Safety Information, including Serious Side Effects, here: bit.ly/2kp959s HEMLIBRA® is a registered trademark of Chugai Pharmaceutical Co., Ltd., Tokyo, Japan. The Genentech logo is a registered trademark of Genentech, Inc. © 2019 Genentech USA, Inc. All rights reserved. EMI/053019/0107e 07/19

Star Wars Celebration is in less than a week, Episode VIII: The Last Jedi is eight months away, the Han Solo anthology film is in nearly a year... and all that's just the visible half of this veritable Star Wars sarlaac pit. Lucasfilm has a plan for us all; and that's to be blissfully digesting space stories for over a thousand years. Or... not? We've got some hot new intel about what's to come - intel that could very well be confirmed in this coming week - and not only does that involve some exciting, long-rumored film projects but also a shockingly tactful return to Star Wars scarcity before the next trilogy. There's lots of provocative details to discuss. Plus: the latest casting surprises and details behind VIII and Han Solo, fascinating things happening in comics and novels, and Willow Watch wherein we learn of a new Willow ally and put out a call to celebrate the film's 30th in a big way. For links and more info, head to the main episode page: http://nerdyshow.com/2017/04/state-of-the-empire-31-the-fviii-of-the-forceLearn more about your ad choices. Visit megaphone.fm/adchoices See acast.com/privacy for privacy and opt-out information.

In this episode, we take a look back at a historic summer that saw the National Hemophilia Foundation's (NHF) Annual Meeting and the World Federation of America's (WFH) World Congress convene in Orlando for the largest gathering ever of people with bleeding disorders. In addition to featuring some conference related content, our Like Share Comment segment highlight a story on Elijah Warren, a high school swimming star with hemophilia as well as a recent article from John Hopkins University on the power of humor in healthcare. Finally, we close this month's episode with a remembrance of Matt Stinger, a community member and advocate who passed unexpectedly this month at 33 years old. Headlines Novo Nordisk’s B-HERO-S study has found, to the surprise of some, that many patients with mild or moderate hemophilia B in the United States experience their condition negatively impacting their career, education, and recreational activities- something that can be overlooked in those affected by mild or moderate hemophilia as opposed to severe. Link. High schooler Federico Parres is one of five team members on the VEX Robotics World Championship Team F, responsible for creating Caerus-7, a ball-flinging robot. Federico lives with severe hemophilia A and plans on becoming an engineer one day. Link. Bayers’ Hemophilia Awards Program recently announced 16 physicians and researchers from 11 countries who will receives grant money totaling about two million dollars, including 6 individuals from the United States. This is the fourteenth year Bayer has run this program. Link A study published in the European journal Haematologica examines a new potential model for how to treatment patients with hemophilia A who undergo surgery. Something I personally find interesting given our ongoing need to learn more about aging with hemophilia. Link The FDA has approved a new needleless reconstitution device called Baxject III for Shire’s long-lasting factor FVIII therapy Adynovate. For those familiar, it is a very similar device to that which is found in Advate. Link Two medical advisory updates have been announced by the National Hemophilia Foundation regarding existing products: NHF Medical Advisory 419 (four-one-nine) states that Bayer has announced a voluntary recall of two lots of Kogenate FS for a loss of potency. The lot numbers are 270TN1C and 270R978. For more information or to arrange for an exchange please call the Recall Processing Center at 855-838-5782. Link And NHF Medical Advisory 420 (four-two-zero) states that CSL Behring has announced a voluntary recall of two lots of Helixate FS for a loss of potency. Those lot numbers are 270TN1G and 270R979. For more information or to arrange an exchange for these products, call CSL Behring’s Customer Service at 1800-683-1288. Link Product Related Headlines (not mentioned in the show) Genentech/Roche has released encouraging Phase 1/2 extension results for their investigational subcutaneous medicine, emicizumab-previously referred to as ACE910. Link The Netherlands-based uniQure, a company self-described as a leader in human gene therapy, announced encouraging clinical data from their ongoing Phase 1/2 trail of AMT-060, an investigational gene therapy for patients with hemophilia B. Link​ BioMarin has presented positive interim data from a Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for people severe hemophilia A. The company has announced intent to begin another study with an eye toward regulatory approval in both the US and Europe. Link CSL Behring’s hemophilia B therapy Idelvion shows promise after encouraging Phase 3 trial results. Link The company also posted positive results from a Phase III study from its FDA approved, hemophilia A treatment AFSTYLA, unique for being the first and only single-chain recombinant product available in the US. Spark Therapeutics and Pfizer received Breakthrough Therapy Status from the FDA for expedited development of a hemophilia B gene therapy treatment SPK-9001. The treatment is currently also undergoing a Phase 1/2 clinical trial. Link. Alnylam Pharmaceuticals announced positive results of a Phase 1 trial for their subcutaneous therapy fitusiran, designed to treat patients with both hemophilia A and B, with or without inhibitors. Link. Like Share Comment Segment NHF Daily, a digital recap of Annual Meeting. Link WFH The Congress Daily. Link High school swimming star Elijah Warren. Link John Hopkins University article on therapeutic humor. Link BloodStream on Social BloodStream Facebook Page BloodStream Twitter Account BloodStream Host Patrick James Lynch on Twitter and Instagram.

Among the discussed risk factors for high-titre inhibitor (HRI) development in patients with hemophilia A(HA) are high dose FVIII replacement therapy and use of recombinant FVIII concentrates (rFVIII). The aim of this study was to evaluate the aforementioned risk factors for HRI development in children with hemophilia A

Fluorescence Correlation Spectroscopy (FCS) allows one to measure protein-membrane binding, self-assembly and other molecular reactions and parameters quantitatively in buffer as well as in complex media. Subject of this thesis was to investigate protein-membrane interactions within blood coagulation in buffer as well as in their biological environment with FCS. Binding of Factor VIII (FVIII) to phosphatidylserine (PS)-expressing platelets is a key process in the intravascular pathway of the blood coagulation cascade. Representing a complex component of the highly regulated network of the coagulation cascade, this protein-membrane interaction is influrenced by many cofactors, such as annexin, which binds to PS-containing membranes as well. Since defects in coagulation, particularly in FVIII binding to membranes lead to severe bleeding disorders, a better understanding of the underlying biophysical and biochemical mechanisms and regulatory influences of this interaction could boost diagnosis and therapy of such diseases, especially when used in combination with an improved systems biology description of the cascade. This thesis investigates the mechanism of FVIII binding to PS-containing model membranes and its regulation by annexin using FCS. Activated FVIII, in contrast to inactivated FVIII, was found to exhibit a striking binding anomaly, consisting in a sharply peaked dependence of the binding constant K(PS) as a function of the PS content. It exceeds the binding of inactivated FVIII in a regime around 12% PS, including physiological concentrations. Furthermore, the regulatory influence of annexin, which can both, increase as well as decrease the binding of activated FVIII, was explained based on this binding anomaly. A quantitative model of this regulatory mechanism assuming efficient shielding of charges by annexin was developed, which allows for the reconstruction of the full three-dimensional phase diagram of FVIII binding to membranes as a function of their PS-content and the concentration of annexin. In order to prove the relevance of these results for coagulation, the experiments were repeated in plasma. Since plasma is a scattering medium, which is crowded by macromolecules and hence strongly affects FCS experiments, a procedure to analyze measurements performed in such complex media was developed. To this end, the influences of scattering and crowding on FCS were investigated using a model system of GFP in highly concentrated vesicle solutions. Scattering was found to enhance and distort the focal volume, whereas crowding slows down diffusion. Taking both effects into account, corrections could be applied, which were demonstrated to allow for artifact-free analysis of binding measurements in complex soft matter systems. To further improve the performance of FCS in complex media and, particularly, in cells, a two-photon FCS microscope was set up. Based on the results of the investigations on scattering and crowding, FCS experiments on living cells were performed. The effective viscosity in dictyostelium discoideum cells was probed and compared to values obtained in lysate. The enhancement of viscosity in the cytoplasm was found to be due to crowding by polydisperse macromolecules, whereas the viscosity of the actin cortex was determined by actin polymerization. Drug treatment allowed for regulation of the polymerization level in the cytoplasm and for detection and determination of the viscosity of actin waves. A project in close colaboration with the groups of Prof. Bein and Prof. Bräuchle succeded in the design, characterization and testing of a drug delivery system employing colloidal mesoporous silica nanoparticles efficiently coated by lipids with a solvent exchange method. Using cross-correlation spectroscopy the lipids were shown to form a close and dense bilayer around the nanoparticles. In-vitro drug delivery experiments gave evidence of the capping-mechanism of the lipids and in-vivo studies proved the effcient delivery and release of drugs by the lipid-coated nanoparticles.