| Summary: | INTRODUCTION AND STUDY DESIGN: We conducted a retrospective analysis of consecutive referrals of patients under 18 years of age undergoing immunohematopoietic stem cell transplantation to assess the influence of age, diagnosis, graft type and gender on survival. We also contrasted program activity and outcome to that reported from a state hospital in the same geographical area over a comparable period. METHODS: Conditioning employed either a sequential combination of fractionated 12Gy whole body and 6Gy total nodal irradiation separated by 120 mg/kg of cyclophosphamide in patients over 15 years of age. Alternatively, the latter agent was combined initially with oral busulphan and later the intravenous equivalent. Neuroblastoma cases were prepared using a different regimen. In allografts the harvested product underwent ex vivo T-cell depletion with the humanized version of anti-CD 52 monoclonal antibody designated Campath 1H. No additional immunosuppression was given except where matched unrelated volunteer donors were employed. RESULTS: Sixty-eight procedures were carried out in 61 patients over a 6-year period. Of 11 with acute myeloid leukemia, 8 are alive and well whereas 8 of the 14 with the lymphoblastic variant have died. Of the remaining 12 with hematologic malignancy, all but 2 are alive. Ten of the 17 with aplasia are alive as are all with thalassemia or sickle cell disease. None of the four variables tested affected survival. CONCLUSION: Our analysis indicates that the standardized preparative regimen, coupled with a now well-established immunosuppressive regimen, is as effective in patients under 18 years of age as in adults. Our analysis also indicates that in a resource-scarce or developing country, it is mandatory to limit high-risk and relatively expensive procedures to active teams that enjoy international accreditation, whether these be in the state or private sector.
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