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.