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Review
. 2010 May;30(2):179-94.
doi: 10.1016/j.iac.2010.02.001.

Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment

Sung-Yun Pai  1 , Luigi D Notarangelo
Affiliations
Review

Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment

Sung-Yun Pai et al. Immunol Allergy Clin North Am. 2010 May.

Abstract

The Wiskott-Aldrich syndrome (WAS) is an X-linked disorder characterized by a triad of diagnostic clinical elements: immunodeficiency, eczema, and hemorrhage caused by thrombocytopenia with small-sized platelets. The formal proof that hematopoietic cell transplantation (HCT) could be used to cure WAS revealed a requirement for both immunosuppression and myelosuppression that still underlies the standard approach to curative therapy today. The current short- and long-term toxicities of HCT are the main stumbling block for the ability to cure every patient with WAS and X-linked thrombocytopenia, and much remains to be done.

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Figures

Figure 1
Figure 1
Hematopoietic cell transplants for Wiskott-Aldrich syndrome in the SCETIDE Registry The absolute number (left) and percentages (right) of HCT performed for WAS using different donor types (genotypically identical matched sibling, white; phenotypically identical matched family member, speckled; unrelated donor, black; mismatched haploidentical family member, grey striped) as reported to the SCETIDE Registry for the indicated time periods is shown. (Data courtesy of Andrew Gennery et. al. on behalf of the SCETIDE Registry.)
See this image and copyright information in PMC

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