Professor and Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology, University of Michigan and CS Mott Children’s Hospital, Ann Arbor, Michigan

Steven W. Pipe, MD

pipe (1).jpg

Pipe discussed a symposium at ASH 2024 about practical implementation of gene therapy in the field.

Steven W. Pipe, MD: Supporting Gene Therapy Implementation for Hemophilia

As gene therapies grow in prominence in the hemophilia field, discussions are needed around the practical implementation of gene therapies, whether concerning the differences in gene therapies for hemophilia A and hemophilia B, accurately identifying eligible candidates for gene therapy, monitoring patients with hemophilia who receive gene therapies, and counseling patients and colleagues about gene therapies.

 66th American Society of Hematology (ASH) Annual Meeting and Exposition

, held December 7-10, 2024, in San Diego, California. Participants included Steven W. Pipe, MD, Professor and Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology, University of Michigan and CS Mott Children’s Hospital, Ann Arbor, Michigan; Doris V. Quon, MD, PhD, Associate Medical Director at Sarcoma Oncology Center; and Amy L. Dunn, MD, Director of Pediatric Hematology, Hemophilia Treatment Center, Nationwide Children's Hospital.

“I was surprised, even in my own clinical setting, that some of the institutional hurdles we had to go through are different than what we had to set up the clinical trial program. And so, I believe a lot of centers are now just sort of working through those processes,” Pipe told 

spoke with Pipe to learn more about the discussion and its main takeaways. He emphasized the importance of helping to guide patients and colleagues through determining eligibility for gene therapy and achieving clinical readiness for administering gene therapy. He noted that payers are beginning to understand the cost-effectiveness of gene therapy and thus, he thinks that education and hesitancy for clinicians and patients have overcome cost as the primary deterrent for gene therapy.

Pipe SW, Quon DV, Dunn A. Clinical assessment of gene therapies for hemophilia A and hemophilia B: Key takeaways for your practice. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Satellite symposium.

Videos

Pipe discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

Steven W. Pipe, MD: Fitusiran With Anti-Thrombin Modulation Yields Effective Bleed Control, Reduces Infusions

Fitusiran prophylaxis, in combination with appropriate bleed management guidelines, allowed fewer infusions and lower doses of clotting factor concentrates (CFC) or bypassing agents (BPAs) to treat breakthrough bleeding in people with hemophilia A or B.

These findings, from the ATLAS OLE study, were presented at the

, held December 7-10, 2024, in San Diego, California, by Steven W. Pipe, MD, Professor and Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology, University of Michigan and CS Mott Children’s Hospital, Ann Arbor, Michigan.

“Fitusiran is a highly effective prophylactic agent. The modulation of thrombin generation allows us to achieve effective bleed control with substantially lower doses of factor and bypassing agents, as well as fewer infusions. And this is just an overall better patient experience,” Pipe told 

Pipe and colleagues found that doses per bleed for participants without inhibitors and with inhibitors was reduced during fitusiran prophylaxis as compared to the CFC/BPA prophylaxis period, and overall, participants required fewer infusions for the management of breakthrough bleeds with fitusiran antithrombin-based dosing regimen (with inhibitors, 57; without inhibitors, 148) versus those who received CFC (n = 189) or BPA (n = 465) prophylaxis.

Pipe SW, Kavakli K, Matsushita T, et al. Reduced Doses of Factor Concentrates and Bypassing Agents to Treat Breakthrough Bleeds in Patients with Hemophilia A and B on Fitusiran Antithrombin-Based Dosing Regimen: ATLAS-OLE. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 128.

Steven W. Pipe, MD

Articles