Impact of bone marrow transplantation for symptomatic sickle cell disease:an interim report

Fifty children who had symptomatic sickle cell disease received matched sib ling marrow allografts between September 1991 and March 1999, with Kaplan-M eier probabilities of survival and event-free survival of 94% and 84%, resp ectively. Twenty-six patients (16 male, 10 female) had at least 2 years of follow-up after transplantation and were evaluated for late effects of tran splantation and for its impact on sickle cell-related central nervous syste m (CNS) and pulmonary disease. Patients ranged between 3.3 and 14.0 (median , 9.4) years of age and had a median follow-up of 57.9 (range 38-95) months after transplantation. Among 22 of 26 patients who had stable donor engraf tment, complications related to sickle cell disease resolved, and none expe rienced further episodes of pain, stroke, or acute chest syndrome. All 10 e ngrafted patients with a prior history of stroke had stable or improved cer ebral magnetic resonance imaging results. Pulmonary function tests were sta ble in 22 of the 26 patients, worse in two, and not studied in two. Seven o f eight patients transplanted for recurrent acute chest syndrome had stable pulmonary function. Linear growth measured by median height standard devia tion score improved from -0.7 before transplantation to -0.2 after transpla ntation. An adverse effect of busulfan conditioning on ovarian function was demonstrated In five of seven evaluable females who are currently at least 13 years of age. None of the four males tested had elevated serum gonadotr opin levels. These data confirm that allogenic bone marrow transplantation establishes normal erythropoiesis and is associated with improved growth an d stable CNS imaging and pulmonary function in most patients. (C) 2000 by T he American Society of Hematology.