Hemophilia Gene Therapy Registry Launched to Monitor Long-Term Outcomes


The World Federation of Hemophilia’s registry will include long-term safety and efficacy data in people with hemophilia treated with gene therapies.

The World Federation of Hemophilia (WFH) is developing a Gene Therapy Registry (GTR) to collect long-term data on safety and efficacy in people with hemophilia treated with gene therapies to learn more about durability and variability of therapies.1

The registry was introduced at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress by Donna Coffin, MSc, director of research & public policy, World Federation of Hemophilia.

“Gene therapy for hemophilia is an evolving therapeutic modality, with many challenges and unresolved questions that will not be fully answered at the completion of current ongoing clinical trial programs,” Coffin and colleagues wrote.1

The prospective, observational, and longitudinal registry will be overseen by a steering committee made up of health care professionals and patients advocates as well as representatives from regulatory agencies and pharmaceutical industry. The WFH hopes to eventually enroll all eligible people with hemophilia in the registry.

READ NOW: Hemophilia B Gene Therapy AMT-060 Shows Durable Effect

"The WFH GTR will ensure that rare adverse events, in a small patient population over a large geographical area, will be detected. The many unknowns of gene therapy for hemophilia make it critical that the bleeding disorders community ensures all patients are followed over their lifetimes,” Coffin and colleagues said.1 

The registry steering committee developed a standardized set of core data to collect from patients through an iterative process under the guidance of the European Medicines Agency and FDA. These data will be collected every 3 months for up to 24 months after gene therapy infusion and annually after that point. Participating hemophilia treatment centers will collect the data, which will be combined with data from existing hemophilia registries. Outreach to these centers began in Fall 2020. Additionally, in the US, data will be collected from the American Thrombosis and Hemostasis Network. In terms of data security, a scientific advisory board of patient and physician advocates is developing policies regarding data usage. 

“Our current knowledge leaves many unanswered questions about the safety and long-term efficacy of gene therapy. These gaps in evidence dictate that we must all contribute to strengthening the evidence base. This data collection/surveillance effort should be a shared responsibility. Health care providers and patients will need to work together to collect standardized data on patients who receive gene therapy, ensuring that their experiences are captured in a registry, over their entire lifetime,” Coffin and colleagues wrote in an editorial published in Haemophilia.2 

Coffin and colleagues outlined unmet needs, pointing to small patient populations and too short follow-up as pressing current issues that exist within clinical trials investigating gene therapies. Additionally, the use of a global registry overcomes the issue of limited data in rare diseases.

“Integrating the collection of data into the clinical practice of physicians and the daily lives of patients requires a harmonious and uniform data collection methodology that will be accepted and used by all stakeholders. Only through cohesive efforts by all treating physicians, patients, regulatory agencies and manufacturers worldwide, will we be successful in ensuring gene therapy is safe and efficacious for our patients today, and in the future,” Coffin and colleagues wrote.2

1. Konkle BA, Coffin D, Naccache M, et al. The world federation of hemophilia gene therapy registry. Presented at: ISTH 2021 Congress; July 17-21, 2021. Abstract PB0661.
2. Konkle BA, Coffin D, Pierce GF, et al. World federation of hemophilia gene therapy registry. Haemophilia. 2020;26:563-564. doi:10.1111/hae.14015
Recent Videos
Lucas Harrington, PhD, the cofounder and chief scientific officer of Mammoth Biosciences
Stephanie Tagliatela on Researching AAV for Lennox-Gastaut, Alzheimer Disease, SCN9a Pain
Miloš Miljković, MD, on mRNA-CAR-T Descartes-08's Potential for Treating Myasthenia Gravis
Manali Kamdar, MD, on Liso-Cel's Ongoing Benefit in the Treatment Lanscape for LBCL
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
Manali Kamdar, MD, on The Importance of Bringing Liso-Cel to Earlier Lines of Lymphoma Treatment
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
© 2024 MJH Life Sciences

All rights reserved.