High-dose busulfan is a major risk factor for ovarian dysfunction in girls after stem cell transplantation

We studied gonadal function in 42 girls who underwent stem cell transplantation (SCT) between 1982 and 1997. Their median age at the time of transplantation was 11 years (range: 5 to 16 years), and that at the time of the study was 17 years (range: 13 to 27 years). Diagnoses included acute leukemia (n=25), aplastic anemia (n=12), chronic myelogenous leukemia (n=3) and lymphoma (n=2). They were assigned to one of four groups according to the preconditioning regimen used for SCT; busulfan plus melphalan (BU group; n=17), high-dose chemotherapy plus total body irradiation (TBI group; n=15), cyclophosphamide plus total lymphoid irradiation (TLI group; n=7), and all others (n=3). Gonadal function was assessed by occurrence of menstruation, serum levels of LH, FSH, and estradiol (E₂). Twenty-eight of forty-two patients (67%) had no menstruation after SCT. The percentages of patients with amenorrhea were 100%, 60% and 14% in the BU, TBI and TLI groups, respectively. These findings confirm that a busulfan plus melphalan regimen is a major risk factor for ovarian dysfunction in children after SCT. Copyright