The Cape Town experience with haematopoietic stem cell transplantation: the paediatric programme
Roux, P.; Novitzky, N.
South African Medical Journal 90(8): 804-811
2000
ISSN/ISBN: 0256-9574 PMID: 11022631 Document Number: 521149
Objective: To determine the outcome of children with blood malignancies and bone marrow failure syndromes treated by paediatricians in the context of an adult haematopoietic transplantation programme. Design: Retrospective chart review. Setting: Hospital wards in a provincial tertiary institution in the Western Cape (Department of Haematology, Groote Schuur Hospital). Subjects: Twenty-eight hospitalised children with haematological malignancies (acute lymphoblastic leukaemia (ALL) N=4, acute myeloblastic leukaemia (AML) N = 13), or bone marrow failure syndromes (N = 11), who consecutively received autologous or allogeneic marrow grafts from HLA-identical siblings. Outcome measures: Children (younger than 18 years) received allogeneic or autologous stem cell transplants. In the former group, two forms of graft-versus-host disease (GVHD) prophylaxis were used. Conditioning with radiation-containing regimens was followed by stem cell product infusion after T-cell depletion (CAMPATH 1, ex vivo immunoglobulin G (IgG); rat anti CD52). Children with malignancies who received unfractionated grafts were myeloablated, mainly with busulfan 16 mg/kg and cyclophosphamide 120 mg/kg. Those affected by marrow failure were prepared with cyclophosphamide and antilymphocyte globulin. Median age at time of transplantation was 116 months (range 18 - 212 months). The main cause of death was disease recurrence (N = 5) and GVHD (N = 3). Twenty-one children survived, 11 of 16 in complete remission (CR) from malignancy. Nine of the eleven patients presenting with marrow failure and 1 patient with severe combined immunodeficiency (SCID) remained disease free at a median follow-up of 934 days (range 70 - 2 330 days). Significantly longer disease-free (P = 0.03) and overall survival (P = 0.05, Cox Mantel test) was experienced by those who received T-cell-depleted stem cell grafts. Conclusions: The strategy of T-cell depletion of bone marrow/blood stem cells from HLA-matched siblings for transplantation into children with blood disorders has been successful and cost effective. These favourable results are the consequence of rational co-operation between adult and paediatric transplant physicians.