Wiskott-Aldrich Syndrome Progression and Outcomes Review

Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990

Protocol Description

This multi-center cohort study by the Primary Immune Deficiency Treatment Consortium (PIDTC) collects medical record information about male patients with Wiskott-Aldrich syndrome, a rare disease that affects the immune system and that can lead to easy bruising, bleeding, and infections. Through data collection and collaboration, researchers hope to determine a more complete picture of the full range of causes, natural history, and the best methods of treatment of Wiskott-Aldrich syndrome.

Eligibility Criteria

Subject to certain exclusion criteria, this study collects and reviews medical record and biological information of male patients of all ages who were seen for Wiscott-Aldrich syndrome at PIDTC centers since 1990 and who received or will receive hematopoietic cell transplant (bone marrow transplant) during the study period.
Males: All ages


Participation entails collection of blood and tissue samples from participants and completion of several quality of life questionnaires, which can be accomplished in a single study visit arranged in connection with a regularly scheduled clinic appointment. Information collected is maintained in a non-identifiable manner in the study database.
Visits: 1
Duration: Not applicable

Status: Active, Not Recruiting

Source(s) of Support

National Institute of Allergy and Infectious Diseases
Primary Immune Deficiency Treatment Consortium
Rare Diseases Clinical Research Network

Primary Investigator

Hey Jin Chong, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Clinical Research Coordinators
Linette Milkovich, RN