Complete Correction of the Wiskott-Aldrich Syndrome by Allogeneic Bone-Marrow Transplantation

Abstract

Two patients with the Wiskott-Aldrich syndrome had complete donor lymphoid and hematopoietic engraftment after successful allogeneic bone-marrow transplantation. One patient had had only a temporary donor T-lymphocyte graft after a previous transplantation, for which he had been prepared with cytarabine and cyclophosphamide; the patient's own T lymphocytes returned six months later. A repeat transplant, for which the patient was prepared with anti-human thymocyte serum, total-body irradiation and procarbazine, resulted in complete donor engraftment. The second patient underwent a successful transplantation after similar preparation, except that procarbazine was omitted. At 11 and five months after transplantation both had normal hematopoiesis and no evidence of graft-versus-host disease. This treatment of the Wiskott-Aldrich syndrome may be a model for the correction of other genetically determined immune and hematologic bone-marrow disorders.

Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies

“…9 Some years later in 1977, cure with complete lymphoid and myeloid chimaerism was achieved in a patient with Wiskott-Aldrich syndrome using myeloablative conditioning. 10 In those early years, only patients with an HLAidentical sibling donor (o20%) were able to benefit from this treatment.…”

Unrelated partially matched peripheral blood stem cell transplantation with highly purified CD34+ cells in a child with Wiskott–Aldrich syndrome

“…[7][8][9][10][11][12][13] An HLA-identical sibling graft is the preferred stem cell source, whereas the outcome with HLA-non-identical bone marrow or peripheral blood stem cells with or without T cell depletion is rather poor because of graft rejection and acute/chronic GvHD. 14-16 Therefore Ozsahin et al 11 concluded that transplants from partially incompatible donors should be restricted to patients with severe complications of WAS.…”

“…5,6 The only curative approach is allogeneic stem cell transplantation. 7,8 Most successful transplants utilized HLA-identical sibling donors. [9][10][11][12][13] The results of unrelated matched or mismatched donor transplantations are associated with an increased risk of graft rejection and graft-versus-host disease (GVHD).…”

Allogeneic peripheral blood stem cell transplantation in a Wiskott-Aldrich syndrome patient

“…3 Cytologic changes in different locations varying from a 'polychromatic and polymorphous smear type' 4 to cancer-mimicking alterations 5 have been described in patients after receiving alkylating substances. Changes described were enlargement of cells, marked nuclear enlargement and hyperchromatism.…”