Jada Bascom Foundation
All conditions

Inherited immune disorders

Wiskott-Aldrich syndrome

Also called: WAS · WAS gene deficiency · primary immunodeficiency · PID · Wiskott-Aldrich immunodeficiency syndrome · Classic WAS

What a donor has to do with this

An approved gene or cell therapy now exists for this condition and can be an alternative to a donor transplant. Which route fits a person depends on their situation, and that decision belongs to them and their treating team.

This is our reading of published transplant guidelines for this condition, not a measurement of how many people need a donor. Where a source actually counted donors, the figure and the people it counted are shown further down. Where none did, we say so rather than estimate.

What the evidence says

Who it affects
Typical onset/diagnosis: this X-linked disorder affects boys and usually begins in infancy or early childhood. Evidence: U.S. NIH GARD synthesis (updated 2026); no markedly enriched geographic population was identified.
Treatments other than a transplant
Waskyra (etuvetidigene autotemcel) — approved — United States, 2025 — For patients aged at least 6 months for whom HSCT is appropriate and no suitable HLA-matched related donor is available.; Waskyra (etuvetidigene autotemcel) — approved — European Union, 2026 — For patients aged at least 6 months for whom HSCT is appropriate and no suitable HLA-matched related donor is available.
If a transplant is used, the cells come from
bone marrow: used in opened IEI transplant guidance; disease-specific share was not reported; mobilized peripheral blood stem cells: used in opened IEI transplant guidance; disease-specific share was not reported; umbilical cord blood: used as an alternative in opened IEI transplant guidance; disease-specific share was not reported; dominance: no dominant graft source was reported in the opened disease-specific sources
How often the donor was unrelated
Not reported. No source we could read states this for this condition, so we do not give a number. An estimate here would be a guess dressed as evidence.

Where this gets complicated

Waskyra was approved after the 2021 EBMT guideline and directly changes the donor-search pathway when no matched related donor is available.; X-linked thrombocytopenia and X-linked neutropenia are allelic WAS-gene disorders but are not automatically equivalent transplant indications.

Written for transplant clinicians, not for patients. We quote it so you can see what the guidance actually says:
“for whom hematopoietic stem cell transplantation (HSCT) is appropriate, but a suitable human leukocyte antigen (HLA)-matched related stem cell donor is not available”

It describes what teams consider in general. It cannot say what applies to any one person. Read the source.

We are not asking you to register on this page

An unrelated donor is not a usual part of treating this condition, so it would be dishonest to use this page to ask you to register. Other conditions in the library are a different story.

Related conditions

Others in inherited immune disorders. They are genuinely different diseases with different treatments — the group name is not a diagnosis.

Where this came from