The Cape Town experience with haematopoietic stem cell transplantation: the paediatric programme.

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.