Autologous bone marrow transplantation for childhood acute lymphoblastic leukaemia in second remission - long-term follow-up

From 1984 to 1996, 31 consecutive children without sibling donors, aged 5-1 9 years (median 8) with acute lymphoblastic leukaemia (ALL) in second compl ete remission (CR), received unpurged autologous bone marrow transplantatio n (ABMT) after melphalan and single fraction total body irradiation (TBI), ABMT was performed using fresh unmanipulated marrow harvested after standar d reinduction and consolidation therapy 2-11 months (median 5) after relaps e. With a median survival of 2.9 years the probability of survival for all patients in continuing second CR was 45.1% (95% CI, 24%-62 %) after 5 years . Regimen-related and non-leukaemia mortality was 7% (95% CI, 2%-26%). The longest time to second relapse from ABMT was 3.1 years. Pituitary and gonad al dysfunction requiring hormonal replacement therapy occurred in the major ity of long-term survivors. Twelve patients developed cataracts. ABMT with melphalan/single fraction TBI has proved an effective anti-leukaemia treatm ent with low regimen-related mortality but significant long-term morbidity, The current approach of allogeneic BMT from an unrelated donor when no sib ling donor is available, following conditioning with cyclophosphamide/ frac tionated TBI has resulted in a reduced relapse rate and improved short-term overall survival in the treatment of relapsed childhood ALL. However, long -term results are awaited.