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Hosahalli Vasanna S, Pereda MA, Dalal J. Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott-Aldrich Syndrome. J Multidiscip Healthc. 2021;14:3497-3512doi: 10.2147/JMDH.S295386.
Hosahalli Vasanna, S., Pereda, M. A., & Dalal, J. (2021). Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott-Aldrich Syndrome. Journal of multidisciplinary healthcare, 143497-3512. https://doi.org/10.2147/JMDH.S295386
Hosahalli Vasanna, Smitha, et al. "Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott-Aldrich Syndrome." Journal of multidisciplinary healthcare vol. 14 (2021): 3497-3512. doi: https://doi.org/10.2147/JMDH.S295386
Hosahalli Vasanna S, Pereda MA, Dalal J. Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott-Aldrich Syndrome. J Multidiscip Healthc. 2021 Dec 23;14:3497-3512. doi: 10.2147/JMDH.S295386. eCollection 2021. PMID: 34992377; PMCID: PMC8711845.
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J Multidiscip Healthc. 2021 Dec 23;14:3497-3512. doi: 10.2147/JMDH.S295386. eCollection 2021.

Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott-Aldrich Syndrome.

Journal of multidisciplinary healthcare

Smitha Hosahalli Vasanna, Maria A Pereda, Jignesh Dalal

Affiliations

  1. Department of Pediatrics, Division of Pediatric Hematology Oncology, Rainbow Babies and Children's Hospital, University Hospitals, Cleveland, OH, USA.

PMID: 34992377 PMCID: PMC8711845 DOI: 10.2147/JMDH.S295386

Abstract

Wiskott-Aldrich syndrome (WAS) is a rare X-linked recessive inborn error of immunity (IEI) first described in 1937. Classic WAS is characterized by the triad of thrombocytopenia with small platelets, recurrent infections due to combined immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) was the only curative option available for five decades, with excellent outcomes reported for matched sibling donors (MSD) and matched unrelated donors (MUD). More recently, alternative donor transplants such as umbilical cord blood (UCB) and haploidentical transplant have emerged as viable options due to improvements in better graft selection, cell dosing, and effective allograft manipulation measures. Gene therapy is another potential curative option with promising results, yet currently is offered only as part of a clinical trial.

© 2021 Hosahalli Vasanna et al.

Keywords: Wiskott–Aldrich syndrome; X-linked thrombocytopenia; gene therapy; hematopoietic stem cell transplantation; supportive care

Conflict of interest statement

The authors report no conflicts of interest in this work.

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