Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding. The immune abnormalities cause patients with WAS to be very susceptible to infections. Depending on the specific type of primary immune deficiency diseases, there are effective treatments, including antibiotics, cellular therapy and gene therapy, but studies of large numbers of patients are needed to determine the full range of causes, natural history, or the best methods of treatment for long term success. This multicenter study combines retrospective, prospective and cross-sectional analyses of the transplant experiences for patients with WAS who have already received HCT since 1990, or who will undergo Hematopoietic cell transplant (HCT) during the study period. The retrospective and prospective portions of the study will address the impact of a number of pre and post-transplant factors on post-transplant disease correction and ultimate benefit from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years post-HCT in consenting surviving patients.
Sex
MALE
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Longitudinal Analysis: Overall Survival From Time of HCT/Gene Therapy
Timeframe: an expected average of 5 years
Cross-Sectional Analysis: Proportion of Participants Achieving Full T Cell Reconstitution
Timeframe: an expected average of 5 years
Cross-Sectional Analysis: Proportion of Participants Achieving Full B Cell Reconstitution
Timeframe: an expected average of 5 years
Cross-Sectional Analysis: Proportion of Participants Achieving Resolution of thrombocytopenia
Timeframe: an expected average of 5 years
Cross-Sectional Analysis: Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL
Timeframe: an expected average of 5 years
Cross-sectional Analysis: Day of Recovery of Platelet Count to 20,000 / uL
Timeframe: an expected average of 5 years
Cross-sectional Analysis: Day of Recovery of Platelet Count to 50,000 / uL
Timeframe: an expected average of 5 years