Abstract
We studied the safety and efficacy of melphalan (L-PAM) and/or total body irradiation administered as the preconditioning for hematopoietic stem cell transplantation (HSCT) in 15 patients with pediatric cancer in an open trial design. Engraftment was achieved in all patients, and the median time of engraftment was 16 days. Of the 5 patients with evaluable disease, 1 was in complete remission (CR), 1 was in very good partial remission (VGPR), 1 was in partial remission (PR), and 2 showed no change (NC); the efficacy rate was 60.0%. Regarding safety, side effects occurred in all 15 patients. The major symptoms : diarrhea (86.7%); stomatitis·mucositis (80.0%); transient increases of GOT and GPT (66.7%); and nausea and vomiting (53.3%). Definite and suspected infections occurred in 13 patients (86.7%). No transplant-related deaths or serious adverse events occurred. In all, 4 patients died. The causes of the death were disseminated intravascular coagulopathy (DIC) as a result of sepsis in 1 patient and recurrent cancer in 3. Of 7 patients who received allogeneic bone marrow transplantation, 3 had acute graft-versus-host disease (GVHD), assessed as grade I. However, no chronic GVHD was observed at the 2-year follow-up. Overall survival at 3 years (median, 1, 135 days) was 71.4%. These findings suggest that L-PAM is effective as a preconditioning for HSCT in pediatric cancer with an acceptable safety profile.
