ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

Study Summary

In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5 In 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7 With this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8

Primary Outcome

Safety will be measured by those events in the European Safety Surveillance (EUHASS)1: 1. Allergic or other acute events 2. Treatment-emergent side effects of therapy 3. Transfusion transmitted infections 4. Inhibitor development 5. Thrombosis 6. Cardiovascular events 7. Malignancies 8. Neurological events 9. Death In addition to the modified EUHASS endpoints, the following events will be collected as adverse events of special interest (AESI): 1. The occurrence of thrombotic microangiopathies

Conditions Studied

Study Locations (20)