Hematology

Paroxysmal Nocturnal Hemoglobinuria

Expert Roundtables Podcast: An Overview of Treatment for Paroxysmal Nocturnal Hemoglobinuria

expert roundtables by Ronald S. Go, MD; David J. Kuter, MD, DPhil; Anem Waheed, MD, MPH

Overview

Over the past 2 decades, treatment advances have transformed paroxysmal nocturnal hemoglobinuria (PNH) from a life-threatening disease associated with chronic hemolysis, thrombosis, and reduced survival into a highly treatable condition. Today’s podcast features an expert discussion on how proximal complement inhibitors, in particular, have reshaped PNH management, with appropriate treatment selection and long-term monitoring enabling hemoglobin optimization, improved quality of life, and durable disease control.

References

Apostolidou E, Georgoulis V, Leonardos D, Kapsali E, Hatzimichael E. Paroxysmal nocturnal hemoglobinuria: unraveling its molecular pathogenesis and advancing targeted therapeutic strategies. Diseases. 2025;13(9):298. doi:10.3390/diseases13090298

Bienz M, Oliver M, Sperlich C, Patriquin CJ. Optimizing care in patients with paroxysmal nocturnal hemoglobinuria: managing suboptimal response and uncontrolled disease. J Blood Med. 2026;17:561117. doi:10.2147/JBM.S561117

Bienz M, Patriquin CJ. The varieties of therapeutic experience: navigating treatment options for patients with PNH. Hematology Am Soc Hematol Educ Program. 2025;2025(1):154-163. doi:10.1182/hematology.2025000701

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Ronald S. Go, MD

Professor of Medicine
Chair, Classical Hematology Group
Division of Hematology, Department of Internal Medicine
Mayo Clinic
Rochester, MN

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David J. Kuter, MD, DPhil

Distinguished Physician
Massachusetts General Hospital
Professor of Medicine
Harvard Medical School
Boston, MA

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Anem Waheed, MD, MPH

Clinical Assistant Professor of Medicine
Weill Cornell Medical College
New York, NY
Leader of Classical Hematology
Astera Cancer Care
East Brunswick and Somerset, NJ