Inherited immune disorders
Wiskott-Aldrich syndrome
Wiskott-Aldrich syndrome is an X-linked disorder affecting platelets and immune function. It can cause bleeding, eczema, infections and autoimmunity, with increased risk of some cancers.
Other names and abbreviations
WAS · WAS gene deficiency · primary immunodeficiency · PID · Wiskott-Aldrich immunodeficiency syndrome · Classic WAS
Where transplant fits
Allogeneic transplantation can correct the blood and immune defects. In the US, Waskyra is an autologous gene-therapy option for eligible patients when transplant is appropriate but no suitable HLA-matched related donor is available. Gene therapy uses the patient’s own cells.
Treatment depends on the exact diagnosis, disease stage, prior treatment and the person’s health. These categories are not estimates of donor demand.
Treatment at a glance
- Who it affects
- Usually affects boys from infancy or childhood. Related WAS-gene disorders can have milder or different manifestations.
- Other treatment options
- Bleeding management, infection prevention and treatment, immunoglobulin support and eczema care address complications. Definitive-treatment selection follows the individual phenotype and product eligibility.
- Cells used for transplantation
- Suitable donor blood-forming stem cells for allogeneic transplantation, or the patient’s own gene-modified stem cells for eligible autologous gene therapy.
Why the details matter
Classic WAS must not be equated with every case of X-linked thrombocytopenia or X-linked neutropenia. Gene therapy requires conditioning and safety follow-up, and availability differs by country.
Questions to bring to your care team
What is the exact diagnosis or subtype? What is the goal of each treatment option? If transplant is being considered, why does it fit this situation, which cells would be used and what are the alternatives?
Sources and further reading
- WAS-Related Disorders
GeneReviews, University of Washington / NCBI Bookshelf · Accessed 2026-09-05 - WASKYRA: indication and current prescribing information
FDA · Accessed 2026-09-05 - Guidelines for hematopoietic stem cell transplantation for inborn errors of immunity
EBMT / ESID Inborn Errors Working Party · 2021
Understanding can become action.
Some patients need a blood stem cell donor. Others receive different treatment. Wherever your interest began, you can help JBF reach more people who may be able to donate.
Explore the official registry serving where you live. It explains who can join, how registration works and what donation involves.
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