title: Bone marrow transplantation in beta-thalassemia major. The Israeli experience.
abstract: The present report summarizes our experience in applying a new approach in bone marrow transplantation for the treatment of beta-thalassemia major. Ex-vivo pretransplant T-lymphocyte depletion with CAMPATH-1 was used for prevention of acute and chronic graft versus host disease and total lymphoid irradiation was added for the conditioning regimen for abrogation of potential rejection of T-cell depleted marrow allografts. Ten patients with homozygous beta-thalassemia major were 9-48 months of age (median 18.5 months) and received HLA-identical allogeneic T-cell depleted marrow after treatment with total lymphoid irradiation, busulfan and cyclophosphamide. Seven patients are alive and free of disease, 3-46 months post-transplantation. The actuarial probability of survival and of disease-free survival at two years was 70%. Three patients died: one of intracranial hemorrhage post-transplantation, one from busulfan interstitial pneumonitis, and one who rejected the first graft and developed fatal chronic graft versus host disease after a second transplant. Seven patients are alive and well with follow-up of 3-45 months, with no signs of acute or chronic GVHD. We conclude that T-cell depleted bone marrow transplantation is indicated for homozygous transfusion dependent young patients with beta-thalassemia who are minimally transfused, particularly in areas where optimal conventional therapy is not feasible.
authors.affiliation: Department of Bone Marrow Transplantation, Jerusalem, Israel.
authors.forename: R
authors.initials: R
authors.lastname: Or
authors.forename: E
authors.initials: E
authors.lastname: Naparstek
authors.forename: G
authors.initials: G
authors.lastname: Cividalli
authors.forename: M
authors.initials: M
authors.lastname: Aker
authors.forename: D
authors.initials: D
authors.lastname: Engelhard
authors.forename: S
authors.initials: S
authors.lastname: Slavin
authors.forename: E A
authors.initials: EA
authors.lastname: Rachmilewitz
mesh_terms: D016026:Bone Marrow Transplantation; D006084:Graft Rejection; D006801:Humans; D007557:Israel; D015182:Lymphatic Irradiation; D013601:T-Lymphocytes; D013789:Thalassemia / Q000628:therapy*