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
None
Any age
Having a congenital or acquired blood disorder; or
Having a bleeding phenotype as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; or
Connective tissue disorder with bleeding tendency as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score.
This is a real-world study in which participants with congenital or acquired blood disorders will be enrolled.
Cohort
Prospective
Not listed
7
Trial groupsSee each trial group below.
To determine the safety of therapies used in the treatment of participants with congenital or acquired non-neoplastic, bleeding and clotting disorders and connective tissue disorders with bleeding tendency (blood disorders).
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, injection site reactions and cases of potential drug-induced liver injury 2. The development of anti-drug antibodies, to be measured and confirmed, if feasible 3. Severe, unanticipated bleeding 4. Hospitalizations 5. Glomerulonephritis 6. Any arm or module-specific AESI as stated in their corresponding Safety Assessment section Additional safety events of interest may be collected.
To establish a platform to support study arms and modules for participants with blood disorders.
For each Arm, a brief set of data elements of interests will be developed and reported for study participants.
To describe medication dosing regimens in participants with blood disorders.
This objective will be evaluated by: 1. Determining the number of participants who initiate and/or switch treatment with non-factor products and participants' reasons for initiating and/or switching treatment with non-factor products 2. Determining the number of participants who do not initiate treatment with non-factor products 3. Determining the number of participants who switch between different non-factor products and the participants' reasons for switching non-factor products 4. Determining the number of participants who discontinue treatment with non-factor products and participants' reasons for discontinuing treatment with non-factor products
To describe real-world effectiveness of therapies used for participants with blood disorders.
1. Health care utilization as measured by number and type of visits and hospitalizations per year 2. Patient reported outcomes (PROs) as measured by the Patient Reported Outcomes Measurement Information System (PROMIS®) Profile 29/25/Parent Proxy, Global Adherence Rating (GAR) (ages 7 and older), CATCH, and EQ-5D5L
To grow and evolve the ATHN Transcends Biorepository for current and future research through the collection of biospecimens from participants enrolled on this protocol
All participants have the option to provide consent to have specimens stored in the ATHN Transcends Biorepository. Samples will be collected as needed, including at the Baseline Visit, Annual Visit, and at Study Exit Visit (if not collected at your Annual Visit), as funding allows.
Click on the lead sponsor to view all of their trials.
American Thrombosis and Hemostasis Network
Lead sponsor
Pfizer
Collaborator
Hemophilia of Georgia, Inc.
Collaborator
Genentech, Inc.
Collaborator
CSL Behring
Collaborator
Sanofi
Collaborator
Novo Nordisk A/S
Collaborator
Hemab ApS
Collaborator
