Podcasts about rare and orphan diseases

Guest: John Cho, MD, PhD, FRCPC While early findings using the SMART protocol for mesothelioma have been encouraging, replication has proven challenging due to steep surgical learning curves, complexities in planning, and skepticism. Dr. John Cho explores why adoption has been limited and what's next for this approach, which he discussed at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Guest: John Cho, MD, PhD, FRCPC Surgery for mesothelioma after radiation therapy (SMART) offers new hope for patients with resectable epithelioid mesothelioma by reversing the traditional treatment sequence. Hear from Dr. John Cho as he discusses the rationale behind this decade-long clinical advancement, which he spoke about at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Sairah Ahmed, MD CAR T-cell therapies have helped transform the treatment of aggressive lymphomas, but could they also change the game for slower-growing, harder-to-treat diseases like marginal zone lymphoma? Based on new data from the TRANSCEND FL study, liso-cel achieved a 95 percent overall response rate and sustained 24-month progression-free survival in relapsed/refractory marginal zone lymphoma. Here with Dr. Charles Turck to share the key efficacy and safety findings is Dr. Sairah Ahmed, Professor in the Department of Lymphoma and Myeloma and CAR T Program Director at MD Anderson Cancer Center.

Guest: John Cho, MD, PhD, FRCPC While early findings using the SMART protocol for mesothelioma have been encouraging, replication has proven challenging due to steep surgical learning curves, complexities in planning, and skepticism. Dr. John Cho explores why adoption has been limited and what's next for this approach, which he discussed at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Guest: John Cho, MD, PhD, FRCPC Surgery for mesothelioma after radiation therapy (SMART) offers new hope for patients with resectable epithelioid mesothelioma by reversing the traditional treatment sequence. Hear from Dr. John Cho as he discusses the rationale behind this decade-long clinical advancement, which he spoke about at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Fibrodysplasia ossificans progressiva (FOP) is a severely disabling myopathy in which extraskeletal bone forms and accumulates over time within muscles and soft tissues.1,2 Here's what we know so far about the mechanisms of heterotopic ossification in patients with FOP. References: Kaplan FS et al. Best Pract Res Clin Rheumatol 2008;22:191–205. Pignolo RJ et al. Orphanet J Rare Dis 2011;6:80. © 2025 Ipsen Biopharmaceuticals Inc. All rights reserved. ALLSC-US-001927 | August 2025

Guest: Mateo Mejia Saldarriaga, M.D. Mateo Mejia Saldarriaga, M.D., a hematologist/oncologist at NewYork-Presbyterian and Weill Cornell Medicine, explains how he and his team conducted a retrospective study that identified a biomarker to enhance treatment planning for BCMA CAR T-cell therapy in multiple myeloma. By measuring absolute lymphocyte count (ALC) through a routine CBC 15 days after a CAR T-cell therapy injection, they found patients with an ALC > 1,000 had a median progression-free survival (PFS) of 30 months whereas patients with an ALC ≤ 500 had a median PFS of 6 months. This new biomarker is now being leveraged to help doctors predict whether a patient is benefiting from treatment in as early as 15 days. © 2025 NewYork-Presbyterian

Guest: Abiodun Ologunowa Pediatric sickle cell disease treatment, particularly the use of hydroxyurea, NSAIDs, and opioids, has evolved in response to clinical guidelines and regulatory shifts, but gaps still remain in how children receive essential medications. Joining Dr. Charles Turck to discuss these national prescribing trends, disparities in care, and the implications of evolving treatment guidelines for this population is Dr. Abiodun Ologunowa. Dr. Ologunowa is a doctoral candidate and research assistant in the Department of Pharmacy Practice and Clinical Research at the University of Rhode Island College of Pharmacy.

Guest: Abiodun Ologunowa Pediatric sickle cell disease treatment, particularly the use of hydroxyurea, NSAIDs, and opioids, has evolved in response to clinical guidelines and regulatory shifts, but gaps still remain in how children receive essential medications. Joining Dr. Charles Turck to discuss these national prescribing trends, disparities in care, and the implications of evolving treatment guidelines for this population is Dr. Abiodun Ologunowa. Dr. Ologunowa is a doctoral candidate and research assistant in the Department of Pharmacy Practice and Clinical Research at the University of Rhode Island College of Pharmacy.

Guest: Abiodun Ologunowa Pediatric sickle cell disease treatment, particularly the use of hydroxyurea, NSAIDs, and opioids, has evolved in response to clinical guidelines and regulatory shifts, but gaps still remain in how children receive essential medications. Joining Dr. Charles Turck to discuss these national prescribing trends, disparities in care, and the implications of evolving treatment guidelines for this population is Dr. Abiodun Ologunowa. Dr. Ologunowa is a doctoral candidate and research assistant in the Department of Pharmacy Practice and Clinical Research at the University of Rhode Island College of Pharmacy.

Guest: Yogindra Persuad Despite the promise of curative therapies for sickle cell disease, access and cost barriers highlight the ongoing need for disease-modifying treatments like hydroxyurea. Tune in to hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, discuss a multimodal approach to care, emphasizing research on oral agents and broader symptom management beyond pain.

Guest: Yogindra Persuad Despite the promise of curative therapies for sickle cell disease, access and cost barriers highlight the ongoing need for disease-modifying treatments like hydroxyurea. Tune in to hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, discuss a multimodal approach to care, emphasizing research on oral agents and broader symptom management beyond pain.

Guest: Parul Rai Cardiopulmonary complications are a leading cause of early mortality in adults with sickle cell disease, with evidence showing that cardiac injury may begin as early as childhood. However, despite the severity of these issues, there are currently no uniform guidelines for asymptomatic cardiac screening in this population. Join Dr. Parul Rai, a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee, to learn about current research, early detection strategies, and the need for more sensitive diagnostic markers to prevent severe cardiac outcomes in patients with sickle cell disease.

Guest: Parul Rai Cardiopulmonary complications are a leading cause of early mortality in adults with sickle cell disease, with evidence showing that cardiac injury may begin as early as childhood. However, despite the severity of these issues, there are currently no uniform guidelines for asymptomatic cardiac screening in this population. Join Dr. Parul Rai, a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee, to learn about current research, early detection strategies, and the need for more sensitive diagnostic markers to prevent severe cardiac outcomes in patients with sickle cell disease.

Guest: Nidhi Bhatt Effective transition from pediatric to adult care should begin as early as age twelve for patients with sickle cell disease, incorporating factors like education, self-advocacy skills, and support from transition care coordinators. Tune in to hear Dr. Nidhi Bhatt, who works in the Department of Hematology at St. Jude Children's Hospital in Memphis, Tennessee, discuss how involving patients and families early on helps identify barriers and improve long-term outcomes.

Guest: Parul Rai Hydroxyurea and chronic transfusions remain cornerstone therapies in managing sickle cell disease, with proven benefits in reducing stroke risk and improving anemia. However, considerations remain, including their impact on cardiac health. Learn about these long-standing therapies and best practices for using them to manage sickle cell disease with Dr. Parul Rai. Dr. Rai is a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Guest: Parul Rai Hydroxyurea and chronic transfusions remain cornerstone therapies in managing sickle cell disease, with proven benefits in reducing stroke risk and improving anemia. However, considerations remain, including their impact on cardiac health. Learn about these long-standing therapies and best practices for using them to manage sickle cell disease with Dr. Parul Rai. Dr. Rai is a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Guest: Yogindra Persuad For decades, hydroxyurea has been the cornerstone therapy for sickle cell disease, but new disease-modifying therapies, curative strategies, and gene therapies are expanding options for patients. While these advancing approaches come with some risks, they can help offer symptom relief, reduce complications, and give patients more choices when it comes to managing their sickle cell disease. Hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, walk through the history of sickle cell disease treatment and discuss the benefits and risks of these developing therapies.

Guest: Yogindra Persuad For decades, hydroxyurea has been the cornerstone therapy for sickle cell disease, but new disease-modifying therapies, curative strategies, and gene therapies are expanding options for patients. While these advancing approaches come with some risks, they can help offer symptom relief, reduce complications, and give patients more choices when it comes to managing their sickle cell disease. Hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, walk through the history of sickle cell disease treatment and discuss the benefits and risks of these developing therapies.

Guest: Nidhi Bhatt Effective transition from pediatric to adult care should begin as early as age twelve for patients with sickle cell disease, incorporating factors like education, self-advocacy skills, and support from transition care coordinators. Tune in to hear Dr. Nidhi Bhatt, who works in the Department of Hematology at St. Jude Children's Hospital in Memphis, Tennessee, discuss how involving patients and families early on helps identify barriers and improve long-term outcomes.

CME credits: 1.00 Valid until: 14-07-2026 Claim your CME credit at https://reachmd.com/programs/cme/treatment-of-a-broader-population-of-patients-with-dmd/33167/ By completing this activity, learners will gain the skills and confidence to determine therapeutic candidacy and conduct appropriate assessments for patients with Duchenne Muscular Dystrophy (DMD) across the age spectrum, including older and non-ambulatory individuals. They will improve their ability to interpret and apply the latest clinical data to real-world scenarios and make informed decisions about treatment and monitoring. Through case-based education, learners will enhance their competence in aligning patients to appropriate therapies and managing long-term care in a rapidly evolving treatment landscape.=

Guest: John A. Morren, MD, FAAN, FAANEM The symptom variability, prolonged asymptomatic periods, and inconsistent presentations of generalized myasthenia gravis (gMG) often lead to significant diagnostic delays and misdiagnoses. Based on recent studies, these delays then correlate with increased disease severity, longer time to symptom control, and worsened quality of life. Here to help uncover the hidden toll of diagnostic delays on patients with gMG is Dr. John Morren. He's the Program Director of the Neuromuscular Medicine Fellowship at Cleveland Clinic and an Associate Professor of Neurology at Cleveland Clinic Lerner College of Medicine of Case Western Reserve University.

Guest: John A. Morren, MD, FAAN, FAANEM The symptom variability, prolonged asymptomatic periods, and inconsistent presentations of generalized myasthenia gravis (gMG) often lead to significant diagnostic delays and misdiagnoses. Based on recent studies, these delays then correlate with increased disease severity, longer time to symptom control, and worsened quality of life. Here to help uncover the hidden toll of diagnostic delays on patients with gMG is Dr. John Morren. He's the Program Director of the Neuromuscular Medicine Fellowship at Cleveland Clinic and an Associate Professor of Neurology at Cleveland Clinic Lerner College of Medicine of Case Western Reserve University.

Host: Gerard A. Silvestri MD, MS, Master FCCP Guest: Anurag Singh, MD Guest: Adam H. Fox, MD, MSc Guest: Mariam Alexander, MD, PhD Despite the typically poor prognosis of extensive-stage small cell lung cancer, recent advancements are reshaping clinical perspectives on treatment. New and emerging options show promise for prolonged survival and improved quality of life. Join Drs. Gerard Silvestri, Adam Fox, Mariam Alexander, and Anurag Singh as they discuss how the therapeutic landscape is evolving for these patients. Silvestri is a pulmonologist and the Hillenbrand Professor of Thoracic Oncology at the Medical University of South Carolina. Dr. Fox is a pulmonologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Alexander is a medical oncologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Singh is a Professor of Radiation Oncology and the Director of Radiation Research at the Roswell Park Cancer Center in New York. This program is produced in partnership with the American College of Chest Physicians and is sponsored by AstraZeneca.

Host: Gerard A. Silvestri MD, MS, Master FCCP Guest: Anurag Singh, MD Guest: Adam H. Fox, MD, MSc Guest: Mariam Alexander, MD, PhD Recent therapeutic advances are reshaping our approach to limited-stage small cell lung cancer. In this multidisciplinary discussion, Dr. Gerard Silvestri sits down with Drs. Adam Fox, Mariam Alexander, and Anurag Singh to explore the evolving standard of care and practical considerations for timely and effective care. Dr. Silvestri is a pulmonologist and the Hillenbrand Professor of Thoracic Oncology at the Medical University of South Carolina. Dr. Fox is a pulmonologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Alexander is a medical oncologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Singh is a Professor of Radiation Oncology and the Director of Radiation Research at the Roswell Park Cancer Center in New York. This program is produced in partnership with the American College of Chest Physicians and is sponsored by AstraZeneca.

Guest: Divya Shankar, MD A recent study investigated whether pulse-dose corticosteroids offer a benefit over lower doses in managing acute exacerbations of idiopathic pulmonary fibrosis (IPF). Join Dr. Divya Shankar as she explains the real-world data, variability in prescribing patterns, and outcomes observed in different levels of care. Divya Shankar is an Assistant Professor of Medicine at Boston University Chobanian and Avedisian School of Medicine as well as a Pulmonary and Critical Care Physician at Boston Medical Center, and she spoke about this topic at the 2025 American Thoracic Society International Conference.

CME credits: 1.00 Valid until: 09-05-2026 Claim your CME credit at https://reachmd.com/programs/cme/new-frontiers-in-the-treatment-of-dmd-across-the-age-spectrum/32740/ Explore the latest treatment options, age-specific management strategies, and clinical trial data for patients with Duchenne muscular dystrophy (DMD). Through expert-led discussions and real-world patient cases, you'll gain practical insights to help you confidently navigate today's ever-evolving treatment landscape and deliver personalized, informed care.=

Guest: Rosaline Quinlivan From sustained therapy and emergency planning to psychological support, coordinated care is essential as patients with Duchenne muscular dystrophy (DMD) transition into adulthood. Explore multidisciplinary approaches to optimize function, independence, and quality of life in adults with DMD with Dr. Rosaline Quinlivan, Professor of Neuromuscular Disease at University College London.

Guest: Rosaline Quinlivan As patients with Duchenne muscular dystrophy (DMD) transition from pediatric to adult care, they may encounter healthcare professionals—from primary care to emergency medicine—without prior experience managing their condition. Explore critical gaps in adult services for DMD and gain practical insights to help deliver timely, informed, and compassionate care with Dr. Rosaline Quinlivan, Professor of Neuromuscular Disease at University College London.

Guest: Rosaline Quinlivan As patients with Duchenne muscular dystrophy (DMD) transition from pediatric to adult care, they may encounter healthcare professionals—from primary care to emergency medicine—without prior experience managing their condition. Explore critical gaps in adult services for DMD and gain practical insights to help deliver timely, informed, and compassionate care with Dr. Rosaline Quinlivan, Professor of Neuromuscular Disease at University College London.

Guest: Rosaline Quinlivan From sustained therapy and emergency planning to psychological support, coordinated care is essential as patients with Duchenne muscular dystrophy (DMD) transition into adulthood. Explore multidisciplinary approaches to optimize function, independence, and quality of life in adults with DMD with Dr. Rosaline Quinlivan, Professor of Neuromuscular Disease at University College London.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Jason Ackrivo, MD, MSCE Guest: Bethany Lussier, MD Respiratory decline in patients with neuromuscular diseases and mitochondrial myopathies can be challenging to identify, especially because its early signs may be subtle and vary from person to person. However, knowing how to evaluate a patient's respiratory function is key to improving outcomes and quality of life. Joining Dr. Charles Turck to discuss this importance and provide recommendations for optimizing respiratory care are Drs. Jason Ackrivo and Bethany Lussier. Dr. Ackrivo is an Assistant Professor of Medicine in Pulmonary, Allergy, and Critical Care at the Hospital of the University of Pennsylvania, and Dr. Lussier is an Associate Professor of Internal Medicine at UT Southwestern Medical Center and a member of its Division of Pulmonary and Critical Care Medicine.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Jason Ackrivo, MD, MSCE Guest: Bethany Lussier, MD Respiratory decline in patients with neuromuscular diseases and mitochondrial myopathies can be challenging to identify, especially because its early signs may be subtle and vary from person to person. However, knowing how to evaluate a patient's respiratory function is key to improving outcomes and quality of life. Joining Dr. Charles Turck to discuss this importance and provide recommendations for optimizing respiratory care are Drs. Jason Ackrivo and Bethany Lussier. Dr. Ackrivo is an Assistant Professor of Medicine in Pulmonary, Allergy, and Critical Care at the Hospital of the University of Pennsylvania, and Dr. Lussier is an Associate Professor of Internal Medicine at UT Southwestern Medical Center and a member of its Division of Pulmonary and Critical Care Medicine.

Guest: Jordan Orange, MD, PhD Guest: Joshua Milner, MD Guest: Eric Silver, MD Guest: Steven Lobritto, MD On this episode of Advances in Care, host Erin Welsh explores the story behind the GUARDIAN study, where thousands of newborn babies have been screened against rare disease by sequencing their genes, and looking for more conditions than any of the current standard screening panels. First, she hears from Dr. Jordan Orange, Physician-in-Chief at Morgan Stanley Children's Hospital at NewYork-Presbyterian and Columbia, about why genetic testing is a promising way of not only catching treatable rare diseases in infants, but also expanding health equity and medical resources to marginalized populations. Erin also hears from Dr. Josh Milner, a pediatric immunologist who treated a patient with a rare form of SCID, or severe combined immune deficiency, also known as bubble boy disease that was detected in the GUARDIAN screening panel. SCID is a disease that typically occurs in 1 of 50,000 babies. But GUARDIAN caught two cases within the first 10,000 babies involved in the program, indicating that the rate of the disease might be higher than expected, and that the most accurate way to detect is through genetic screening. Dr. Steven Lobritto, a pediatric gastroenterologist, also weighs in on how genetic screening can help identify Wilson's disease, a copper storage …

Host: Jennifer Caudle, DO Guest: Naval Daver, MD Not only is CD123 one of the most common antigens expressed on the surface of acute myeloid leukemia (AML) tumors, but it's also associated with more proliferative disease that's resistant to standard therapies. Given its prevalence and potential implications, a number of different CD123-targeting approaches are under investigation, including antibody-drug conjugates, bi-specific antibodies, fusion protein, and CAR T-cell therapy. In light of these new approaches, Dr. Jennifer Caudle and Dr. Naval Daver discuss the importance of targeting CD123 in AML. Dr. Daver is a Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center in Houston.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Aaron Logan, MD, PhD Although acute lymphoblastic leukemia (ALL) is a relatively rare disease with fewer than 7,000 people diagnosed each year in the US,1,2 there's been a lot of progress in the management of B-cell ALL over the years. One key development comes from the E1910 Phase III trial, which explored the addition of blinatumomab to frontline consolidation chemotherapy for patients with Philadelphia chromosome-negative B-lineage ALL (B-ALL).3 Joining Dr. Charles Turck to discuss the impacts of this data on B-ALL clinical guidelines and practice is Dr. Aaron Logan. Dr. Logan is a Professor of Clinical Medicine in the Division of Hematology/Oncology and Director of the Hematologic Malignancies Tissue Bank at UCSF. References: National Cancer Institute. SEER Cancer Stat Facts: Acute Lymphocytic Leukemia (ALL). Accessed at https://seer.cancer.gov/statfacts/html/alyl.html on March 31, 2025 Dana-Farber Cancer Institute. Acute Lymphoblastic Leukemia (ALL). Accessed September 17, 2024. https://www.dana-farber.org/cancer-care/types/acute-lymphoblastic-leukemia Litzow MR, et al. Blood. 2022;140(suppl 2):LBA-1

Guest: Jennie Taylor, MD The FDA approval of vorasidenib marks a new era for mutant isocitrate dehydrogenase (mIDH) gliomas. Approved in 2024 for grade 2 tumors after surgery, it doubled progression-free survival in the INDIGO trial. Dr. Jennie Taylor, Associate Professor of Neurology and Neurological Surgery at the University of California San Francisco, dives into the data and explains what questions remain about long-term use and broader applications.

Guest: Lauren Schaff, MD Glioblastomas are fast, aggressive, and resistant to many standard therapies. Dr. Lauren Schaff, a neuro-oncologist at Memorial Sloan Kettering Cancer Center, explains how new molecular understandings and treatment avenues are paving the way for a more personalized, hopeful approach to care.

Guest: Jennie Taylor, MD Defined by the production of 2-hydroxyglutarate, mutant isocitrate dehydrogenase (mIDH) gliomas are diffuse, slow-growing tumors. Managing these tumors requires personalized strategies that consider resectability, histology, and long-term treatment impacts. Dr. Jennie Taylor, Assistant Professor of Neurology and Neurological Surgery at the University of California San Francisco, explains the complexities behind this type of tumor and implications for patient care. Dr. Taylor also spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Guest: Hans Katzberg, MD Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a complex immune-mediated condition affecting the peripheral nervous system. Join Dr. Hans Katzberg, Professor of Medicine at the University of Toronto, as he explains the pathophysiology behind CIDP, risk factors in disease development, and diagnostic strategies.

Guest: Sami Khella, MD With personalized approaches, alternatives to steroids, and novel therapies like efgartigimod, the treatment landscape for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is continuing to evolve. Join Dr. Sami Khella as he shares expert insights on our available options. Dr. Khella is the Director of Clinical Electrophysiology and a Professor of Clinical Neurology at the University of Pennsylvania, and he spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Host: Michelle Kittleson, MD, PhD Transthyretin cardiac amyloidosis (ATTR-CM) is an underrecognized disorder, leading to a variety of unmet needs for patients. Dr. Michelle Kittleson, Director of Postgraduate Education in Heart Failure and Transplantation and Professor of Medicine at the Cedars-Smidt Heart Institute, explores these challenges and discusses how early recognition, genetic screening, and emerging therapies can help improve patient outcomes.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Anthony Stein, MD Could a novel triplet combination therapy offer a new path forward in acute myeloid leukemia (AML) treatment? A recent Phase 1B trial explored the potential of tagraxofusp, azacitidine, and venetoclax for AML patients. To discuss the findings on this triplet combination therapy, Dr. Charles Turck speaks with Dr. Anthony Stein, Professor in the Department of Hematology and Hematopoietic Cell Transplantation at City of Hope in Duarte, California.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Anthony Stein, MD Could a novel triplet combination therapy offer a new path forward in acute myeloid leukemia (AML) treatment? A recent Phase 1B trial explored the potential of tagraxofusp, azacitidine, and venetoclax for AML patients. To discuss the findings on this triplet combination therapy, Dr. Charles Turck speaks with Dr. Anthony Stein, Professor in the Department of Hematology and Hematopoietic Cell Transplantation at City of Hope in Duarte, California.

Host: Jennifer Caudle, DO Guest: Naval Daver, MD Not only is CD123 one of the most common antigens expressed on the surface of acute myeloid leukemia (AML) tumors, but it's also associated with more proliferative disease that's resistant to standard therapies. Given its prevalence and potential implications, a number of different CD123-targeting approaches are under investigation, including antibody-drug conjugates, bi-specific antibodies, fusion protein, and CAR T-cell therapy. In light of these new approaches, Dr. Jennifer Caudle and Dr. Naval Daver discuss the importance of targeting CD123 in AML. Dr. Daver is a Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center in Houston.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Yi Bin Chen, MD Guest: Olaf Penack, MD For the past several decades, standard treatment for acute graft-versus-host disease (aGVHD) has been systemic high-dose steroids.1 While essential in some ways to initially control aGVHD, steroids are not beneficial in the long run due to the immunosuppression and toxicities associated with high cumulative doses.1,2 So what can be done moving forward to improve first-line steroid response and minimize cumulative steroid exposure? Joining Dr Charles Turck to discuss the potential of nonimmunosuppressive steroid-sparing agents for the first-line treatment of aGVHD are Drs Yi Bin Chen and Olaf Penack. Dr Chen is the Director of the Hematopoietic Cell Transplant and Cell Therapy Program at Massachusetts General Hospital, and Dr Penack is a senior physician and principal investigator within Hematology and Oncology at Charité Berlin. References: Bell EJ, Yu J, Bhatt V, et al. Healthcare resource utilization and costs of steroid-associated complications in patients with graft-versus-host disease. Transplant and Cell Ther. 2022;28(10):707.e1-707.e7. Martin PJ, Rizzo JD, Wingard JR, et al. First- and second-line systemic treatment of acute graft-versus-host disease: recommendations of the American Society of Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2012;18(8):1150-1163. © 2025 CSL BehringCMD-964-0016-JAN25

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Miguel-Angel Perales, MD Guest: Anna Sureda, MD, PhD Unfortunately, the treatment paradigm for acute graft-versus-host disease (aGVHD) has not changed for a couple of decades as steroids remain the first-line treatment.1 However, steroids are not a targeted therapy, and they have many side effects that are not well tolerated by patients, including life-threatening infections.1-3 Given these risks, it is important to know how to manage infections related to immunosuppression and optimize the care of patients with aGVHD. Joining Dr Charles Turck to discuss the current landscape of aGVHD management are Dr Miguel-Angel Perales and Dr Anna Sureda. Dr Miguel-Angel Perales is the Chief of the Adult Bone Marrow Transplantation Service at Memorial Sloan Kettering Cancer Center in New York, and Dr Sureda is the Head of Clinical Hematology at the University of Barcelona in Spain. References: Bell EJ, Yu J, Bhatt V, et al. Healthcare resource utilization and costs of steroid-associated complications in patients with graft-versus-host disease. Transplant Cell Ther. 2022;28(10):707.e1-707.e7. doi:10.1016/j.jtct.2022.04.014 Sullivan PW, Ghushchyan VH, Globe G, Sucher B. Health-related quality of life associated with systemic steroids. Qual Life Res. 2017;26(4):1037-1058. García-Cadenas I, Rivera I, Martino R, et al. Patterns of infection and infection-related mortality in patients with steroid-refractory acute graft versus host disease. Bone Marrow …

CME credits: 1.00 Valid until: 31-12-2025 Claim your CME credit at https://reachmd.com/programs/cme/recent-guidelines-updates-implications-to-practice/26659/ This series of bite-sized episodes will take you on the winding journey that patients with idiopathic multicentric Castleman disease (iMCD) often undergo to achieve an accurate diagnosis. Once the condition is finally identified, even the most astute specialists can find themselves challenged by the treatment, management, and monitoring of these patients. Join Drs. Corey Casper and Sudipto Mukherjee as they explain the ups and downs of this rare disease.

Guest: Mateo Bustamante Idiopathic multicentric Castleman disease (iMCD) is a rare, life-threatening condition. Early diagnosis through excisional biopsies is key for speeding up treatment and improve patient outcomes. Learn more with Mateo Bustamante, a Senior Clinical Data Analyst at the Center for Cytokine Storm Treatment and Laboratory at University of Pennsylvania's Perelman School of Medicine.

Guest: Mateo Bustamante Idiopathic multicentric Castleman disease (iMCD) is a rare, life-threatening condition. Early diagnosis through excisional biopsies is key for speeding up treatment and improve patient outcomes. Learn more with Mateo Bustamante, a Senior Clinical Data Analyst at the Center for Cytokine Storm Treatment and Laboratory at University of Pennsylvania's Perelman School of Medicine.