Treatment of Ph1-positive chronic myelogenous leukemia in children: comparison between allogeneic bone marrow transplantation and conventional chemotherapy

Background and Objective. To compare the estimated survival and disease-fre e survival between children with Ph1-positive chronic myeloid leukemia trea ted with allogeneic bone marrow transplantation or conventional chemotherap y. Design and Methods. In this retrospective study we compared the results obt ained in a group of 14 children who received allogeneic bone marrow transpl antation (BMT) between 1983 and 1993, and another group of 27 children trea ted with busulfan, hydroxyurea or alpha-interferon during the same time per iod. Patients were transplanted at a median of 7 months from diagnosis and all except one were in their first chronic phase. Conditioning consisted in total body irradiation and cyclophosphamide in 12 cases, and busulfan was added in two. Results. Of the 14 patients treated with BMT, two died of transplant-relate d complications and two relapsed 18 and 48 months after the BMT. Ten childr en remain alive and disease free at a median follow up of 60 months. The pr obability of DFS at 5 years is 70%. Of the 27 patients treated with chemoth erapy, 22 have died at a median of 36 months from diagnosis. The probabilit y of survival at 5 years is 5% versus 83% for the BMT group (p = 0.001). Interpretation and Conclusions. Allogeneic BMT is a safe and very effective treatment for Ph-positive CML in children. Patients who have an HLA-identi cal sibling donor must receive a transplant as soon as possible after being diagnosed. (C)1998, Ferrata Storti Foundation.