The World Federation of Hemophilia (WFH) has launched a gene therapy registry to monitor the safety and effectiveness of gene therapies for hemophilia. The registry is called the WFH Gene Therapy Registry and aims to collect data from people with hemophilia who have undergone or are planning to undergo gene therapy. The registry is expected to provide valuable information to researchers, clinicians, and patients about the long-term safety and efficacy of gene therapy for hemophilia. WFH Vice President, Medical Glenn Pierce comments “Patient safety is all of our responsibility. Collecting data in one global registry—the WFH GTR—is essential to ensure that rare adverse events, in a small patient population over a large geographical area, will be detected.”  Patient participation is voluntary.  

Hemophilia is a rare genetic disorder that affects the blood’s ability to clot, leading to prolonged bleeding and other complications. Gene therapy is a promising new treatment for hemophilia that involves replacing or correcting the faulty genes that cause the disorder.However, gene therapy is still relatively new, and its long-term safety and effectiveness are not yet fully understood.

The WFH Gene Therapy Registry will help address this knowledge gap by collecting data from people with hemophilia who have undergone gene therapy. The registry will track information such as the type of gene therapy used, the patient’s bleeding episodes, and any adverse events that occur. This information will be used to monitor the safety and effectiveness of gene therapy and to inform future research and clinical practice. HTCs, where most gene therapy treatments will be administered, will invite patients to participate, once they have made the decision to receive gene therapy.  Patient data will be de-identified for privacy.  The registry is supported by a number of hemophilia organizations and product manufacturers worldwide.

The registry is an important step forward for the hemophilia community and for gene therapy research. It will provide valuable insights into the long-term safety and efficacy of gene therapy for hemophilia and help to ensure that patients receive the best possible care.

For more information: https://wfh.org/research-and-data-collection/gene-therapy-registry/