Practice Point®: Rare Diseases

Practice Point®: Rare Diseases

by Practice Point Communications
Episode 37 - Part 4 - Applying the Evidence: A Case-Based Approach to Adult HLH Management
Using a realistic patient case, this final episode demonstrates how expert clinicians evaluate, diagnose, and manage a patient with suspected adult HLH in real time. Faculty discuss the interpretation of evolving clinical findings, biomarkers, and imaging, while highlighting common diagnostic pitfalls, treatment escalation strategies, and the importance of identifying the underlying trigger. The case illustrates how multidisciplinary decision-making and early intervention can improve outcomes in this complex and rapidly evolving syndrome. Launch/Release Date: July 23, 2026 Activity Expiration Date: June 30, 2027 Faculty Sarah Nikiforow, MD, PhD Assistant Professor, Stem Cell Transplantation Medical Director, DFCI Cell Manipulation Core Facility Technical Director, DFCI Immune Effector Cell Therapy Program Dana-Farber Cancer Institute Daniella Schwartz, MD Assistant Professor Division of Rheumatology and Clinical Immunology Departments of Medicine and Immunology Co-Director, Autoinflammatory Center of Excellence Co-Director, Pittsburgh Immunogenetics Discovery Center University of Pittsburgh Medical Center This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 36 - Part 3 - Managing Adult HLH: Trigger-Based Treatment and Emerging Therapeutic Strategies
Successful management of adult HLH requires simultaneous control of hyperinflammation and identification of the underlying trigger. In this episode, faculty review practical treatment approaches, including corticosteroids, targeted immunomodulatory therapies, and selective use of etoposide-based regimens, while highlighting the growing role of pathway-directed therapies. The discussion emphasizes multidisciplinary care, ongoing reassessment, and individualized treatment decisions based on disease biology and clinical response. Launch/Release Date: July 23, 2026 Activity Expiration Date: June 30, 2027 Faculty Sarah Nikiforow, MD, PhD Assistant Professor, Stem Cell Transplantation Medical Director, DFCI Cell Manipulation Core Facility Technical Director, DFCI Immune Effector Cell Therapy Program Dana-Farber Cancer Institute Daniella Schwartz, MD Assistant Professor Division of Rheumatology and Clinical Immunology Departments of Medicine and Immunology Co-Director, Autoinflammatory Center of Excellence Co-Director, Pittsburgh Immunogenetics Discovery Center University of Pittsburgh Medical Center This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 35 - Part 2 - Recognizing Adult HLH Earlier: Clinical Clues and Biomarkers That Matter
Early recognition is critical to improving outcomes in adult HLH. This episode explores the clinical features, common triggers, and laboratory abnormalities that should raise suspicion for HLH, while reviewing the strengths and limitations of current diagnostic tools, including the HLH-2004 criteria, HScore, and emerging biomarker strategies. Faculty also discuss how trends in ferritin and immune biomarkers can support earlier diagnosis and more informed risk stratification. Launch/Release Date: July 23, 2026 Activity Expiration Date: June 30, 2027 Faculty Sarah Nikiforow, MD, PhD Assistant Professor, Stem Cell Transplantation Medical Director, DFCI Cell Manipulation Core Facility Technical Director, DFCI Immune Effector Cell Therapy Program Dana-Farber Cancer Institute Daniella Schwartz, MD Assistant Professor Division of Rheumatology and Clinical Immunology Departments of Medicine and Immunology Co-Director, Autoinflammatory Center of Excellence Co-Director, Pittsburgh Immunogenetics Discovery Center University of Pittsburgh Medical Center This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 34 - Part 1 - Understanding Adult HLH: Why Early Recognition Matters
Adult hemophagocytic lymphohistiocytosis (HLH) is a rapidly progressive hyperinflammatory syndrome that often presents with nonspecific features, making early diagnosis challenging. In this introductory episode, expert faculty explain the underlying immunobiology of adult HLH, distinguish primary from secondary disease, and explore why HLH should be viewed as a syndrome rather than a single disease entity. The discussion establishes the clinical framework needed to recognize patients at risk and prepares learners for the practical diagnostic and management strategies covered throughout the series. Launch/Release Date: July 23, 2026 Activity Expiration Date: June 30, 2027 Faculty Sarah Nikiforow, MD, PhD Assistant Professor, Stem Cell Transplantation Medical Director, DFCI Cell Manipulation Core Facility Technical Director, DFCI Immune Effector Cell Therapy Program Dana-Farber Cancer Institute Daniella Schwartz, MD Assistant Professor Division of Rheumatology and Clinical Immunology Departments of Medicine and Immunology Co-Director, Autoinflammatory Center of Excellence Co-Director, Pittsburgh Immunogenetics Discovery Center University of Pittsburgh Medical Center This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 33 - Advanced Therapies and the Future of Uncontrolled Gout
Managing uncontrolled gout isn’t always straightforward—especially when flares persist, comorbidities complicate care, or standard therapies fall short. As our understanding of gout expands beyond just elevated uric acid, clinicians are being asked to think differently, integrating immune pathways, real-world evidence, and emerging treatments into everyday practice. From Refractory to Resolved: Rethinking Uncontrolled Gout in 2026, this CME-accredited podcast explores how to approach these complex cases with greater clarity and confidence. Featuring expert insights and the latest clinical evidence, each episode dives into practical strategies across the continuum of gout care. Listeners will hear discussions on advances in uricase-based therapies, selective URAT1 inhibitors, and inflammation-targeted treatments, along with key considerations around safety, sequencing, and collaborative care. Through real-world examples and focused conversations, this series equips clinicians with actionable tools to personalize treatment, address therapeutic challenges, and improve long-term outcomes for patients living with uncontrolled gout. Launch Date/Release: June 12, 2026 Expiration Date: May 31, 2027 FACULTY Robert Terkeltaub, MD Professor Emeritus of Medicine, University of California, San Diego This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 32 - Optimizing Gout Care Across the Disease Continuum
Managing uncontrolled gout isn’t always straightforward—especially when flares persist, comorbidities complicate care, or standard therapies fall short. As our understanding of gout expands beyond just elevated uric acid, clinicians are being asked to think differently, integrating immune pathways, real-world evidence, and emerging treatments into everyday practice. From Refractory to Resolved: Rethinking Uncontrolled Gout in 2026, this CME-accredited podcast explores how to approach these complex cases with greater clarity and confidence. Featuring expert insights and the latest clinical evidence, each episode dives into practical strategies across the continuum of gout care. Listeners will hear discussions on advances in uricase-based therapies, selective URAT1 inhibitors, and inflammation-targeted treatments, along with key considerations around safety, sequencing, and collaborative care. Through real-world examples and focused conversations, this series equips clinicians with actionable tools to personalize treatment, address therapeutic challenges, and improve long-term outcomes for patients living with uncontrolled gout. Launch Date/Release: June 12, 2026 Expiration Date: May 31, 2027 FACULTY Robert Terkeltaub, MD Professor Emeritus of Medicine, University of California, San Diego This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 31 - Beyond the Joint: The Burden and Biology of Uncontrolled Gout
Managing uncontrolled gout isn’t always straightforward—especially when flares persist, comorbidities complicate care, or standard therapies fall short. As our understanding of gout expands beyond just elevated uric acid, clinicians are being asked to think differently, integrating immune pathways, real-world evidence, and emerging treatments into everyday practice. From Refractory to Resolved: Rethinking Uncontrolled Gout in 2026, this CME-accredited podcast explores how to approach these complex cases with greater clarity and confidence. Featuring expert insights and the latest clinical evidence, each episode dives into practical strategies across the continuum of gout care. Listeners will hear discussions on advances in uricase-based therapies, selective URAT1 inhibitors, and inflammation-targeted treatments, along with key considerations around safety, sequencing, and collaborative care. Through real-world examples and focused conversations, this series equips clinicians with actionable tools to personalize treatment, address therapeutic challenges, and improve long-term outcomes for patients living with uncontrolled gout. Launch Date/Release: June 12, 2026 Expiration Date: May 31, 2027 FACULTY Robert Terkeltaub, MD Professor Emeritus of Medicine, University of California, San Diego This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 30 - Days 3 and 4: Key Breakthroughs from EASL 2026
These podcast episodes explore the rapidly evolving treatment landscape of Primary Biliary Cholangitis, highlighting recent therapeutic advances and reviews emerging clinical evidence on promising investigational therapies. Designed for healthcare professionals, these episodes provides expert commentary on the latest cutting-edge data presented at EASL Congress 2026. Through an engaging podcast format, listeners will gain practical insights into how new evidence can inform personalized treatment strategies, support improved patient outcomes, and enhance the overall quality of care for people living with PBC. Launch Date/Release: June 3, 2026 Expiration Date: May 31, 2027 FACULTY Kris V. Kowdley, MD, AGAF, FAASLD, FACG, FACP Liver Institute Northwest Director, Velocity Clinical Research Medical Director and Senior Scientific Advisor Washington State University Elson S. Floyd College of Medicine – Professor This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 29 - Days 1 and 2: Key Breakthroughs from EASL 2026
These podcast episodes explore the rapidly evolving treatment landscape of Primary Biliary Cholangitis, highlighting recent therapeutic advances and reviews emerging clinical evidence on promising investigational therapies. Designed for healthcare professionals, these episodes provides expert commentary on the latest cutting-edge data presented at EASL Congress 2026. Through an engaging podcast format, listeners will gain practical insights into how new evidence can inform personalized treatment strategies, support improved patient outcomes, and enhance the overall quality of care for people living with PBC. Launch Date/Release: June 3, 2026 Expiration Date: May 31, 2027 FACULTY Kris V. Kowdley, MD, AGAF, FAASLD, FACG, FACP Liver Institute Northwest Director, Velocity Clinical Research Medical Director and Senior Scientific Advisor Washington State University Elson S. Floyd College of Medicine – Professor This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
Episode 28 - NF1-PN Podcast: Key Advances from AAN 2026
Leveraging emerging evidence presented at AAN, Dr. Angela Hirbe provides synthesis of key findings, highlight their relevance across pediatric and adult care, and frame practical considerations for patient assessment and longitudinal management. Launch/Release Date: April 24, 2026 Activity Expiration Date: March 31, 2027 FACULTY Angela Hirbe, MD, PhD Associate Professor of Medicine and Pediatrics Director, Adult Neurofibromatosis Clinical Program Division of Oncology, Sarcoma Section Washington University School of Medicine St. Louis, Missouri This podcast provides accredited continuing education credits. To receive your credit, please read the accreditation information provided in this link prior to listening to this podcast.
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