Long-term survival after bone marrow transplantation: A 15-year follow-up report of a patient with wiskott-aldrich syndrome

Differential contribution of Wiskott-Aldrich syndrome protein to selective advantage in T- and B-cell lineages

“…7 These data and our results support a reduced role for WASP in B-cell differentiation, proliferation, and survival, which is in line with the observations in WASP knock-out mice that show unchanged or only minimal B-cell defects. 8,9 The recognition of differential selective advantage conferred by WASP expression to T and B (and possibly other hematopoietic) lineages will provide important insights into the biology of the disease, help explain the occurrence of mixed chimerism in patients with WAS after allogeneic bone marrow transplantation, [10][11][12] and offer valuable perspectives on the possible outcomes of gene therapy for WAS.…”

Addison disease 10 years after bone marrow transplantation for Wiskott-Aldrich syndrome

“…It encodes a 501 amino acid protein likely to be a key regulator of lymphocyte and platelet function [3]. Bone marrow transplantation (BMT) is the only curative therapy resulting in excellent survival rates [6][7][8][9][10]12]. Few studies report long-term follow up of WAS after BMT.…”

Wiskott-Aldrich Syndrome

“…The ultimate cure for WAS is by bone marrow transplantation [15,[18][19][20][21]. For children who do not have a suitable donor, an alternative treatment such as splenectomy needs to be considered [15,22].…”