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REVIEW ARTICLE

Hematopoietic stem cell transplantation in Wiskott–Aldrich syndrome: A systematic review and meta-analysis

Esam Abdul Wahab1 ,  Yaqoub Nael Hamdan Abu Hamdan1 ,  Hussein Omari Sombi1 ,  Omar Essa1 ,  Malik Mossa Jafar1 ,  Ahmad Alamassi1 ,  Idris Sula2 ,  Muhammad Candragupta Jihwaprani3,4 ,  Khawaja Husnain Haider1*
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1 Department of Basic Sciences, College of Medicine, Sulaiman Al Rajhi University, Al Bukayriyah, Al-Qassim , Saudi Arabia
2 College of Medical Applied Sciences, Al Bukayriyah, Al-Qassim , Saudi Arabia
3 Research Unit Department, College of Medicine, Sulaiman Al Rajhi University, Al Bukayriyah, Al-Qassim , Saudi Arabia
4 Department of Medical Education, Sidra Medicine, Doha , Qatar
JCTR, 026340091 https://doi.org/10.36922/JCTR026340091
Received: 19 August 2026 | Revised: 15 September 2026 | Accepted: 21 September 2026 | Published online: 16 October 2026
© 2026 by the Author(s). This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution -Noncommercial 4.0 International License (CC-by the license) ( https://creativecommons.org/licenses/by-nc/4.0/ )
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Abstract

Background: Wiskott–Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency that typically presents early in life with recurrent infections, eczema, and microthrombocytopenia. Patients are also at increased risk of autoimmune complications and malignancies. In recent years, hematopoietic stem cell transplantation (HSCT) has emerged as a potentially curative treatment option.  Aim: To systematically review and meta-analyze the available evidence on the safety and effectiveness of HSCT in patients with WAS.  Methods: This systematic review and meta-analysis followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines (PROSPERO: CRD42025640272). We searched four databases (Cochrane CENTRAL, ClinicalTrials.gov, PubMed/MEDLINE, and ScienceDirect) for relevant studies from inception through May 2024. We included observational studies and clinical trials, including those involving WAS patients undergoing HSCT. Outcomes of interest included overall mortality and survival, event-free survival, graft-versus-host disease (GVHD), autoimmunity, symptomatic recovery, engraftment, and chimerism.  Results: We analyzed 24 studies, pooling 884 HSCT-recipient WAS patients. Most studies were of low methodological quality (83%). The pooled overall mortality was 13% (95% confidence interval = 9.5%–16.5%), with infections being the most common cause of death (63.6%), followed by GVHD (24.1%). Event-free survival and symptomatic recovery were 84.6% and 72%, respectively. Mean neutrophil and platelet engraftment occurred at 18.75 and 31.74 days post-HSCT, respectively. Full and mixed chimerism was achieved in 72.5% and 18.9% of cases, respectively. Selected complications included acute GVHD (35.9%), chronic GVHD (11.9%), and autoimmune manifestations (19%).  Conclusion: HSCT is associated with favorable survival and clinical outcomes in patients with WAS; however, treatment-related mortality remains substantial, with infections and GVHD reported as the leading causes of mortality among HSCT recipients. Relevance for patients: This study helps patients and families weigh the favorable survival and symptom recovery associated with HSCT against potential risks such as infections, graft-versus-host disease, and autoimmune complications.

Keywords
Heart
Hematopoietic stem cells
Stem cells
Wiskott–Aldrich syndrome
Funding
None.
Conflict of interest
The authors declare they have no conflicts of interest.
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Journal of Clinical and Translational Research, Electronic ISSN: 2424-810X Print ISSN: 2382-6533, Published by AccScience Publishing
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