Allogeneic stem cell transplantation for patients with high-risk myelodysplastic syndrome.

Allogeneic stem cell transplantation (allo-SCT) is the only treatment with curative potential for patients with myelodysplastic syndrome (MDS). From June 1986 to April 1997, we treated 12 patients with primary MDS (5 men, 7 women, median age, 36.5 years) by allo-SCT. All patients had one or more of the following poor prognostic factors: intermediate-2 or high-risk categories according to the International Prognostic Scoring System; disease progression during follow-up; heavy transfusion requirements and recurrent infections. The median duration from diagnosis of MDS to allo-SCT was 6 months. The preconditioning regimen included total body irradiation combined with either high-dose cytarabine (n = 6), high-dose cyclophosphamide (n = 4), or other regimens (n = 2). Ten patients received bone marrow transplantations and two patients received peripheral blood stem cell transplantations. Prophylaxis for graft-versus-host disease (GVHD) consisted of standard cyclosporin and short-course methotrexate. Acute GVHD of grade 2 or above occurred in 10 patients, while chronic GVHD occurred in seven of the nine patients who survived longer than 6 months after allo-SCT. With a median follow-up of 50 months, all nine patients with human leukocyte antigen (HLA)-matched sibling donors survived. One patient had a relapse 6 months after transplantation and achieved complete remission again with low-dose cytarabine therapy. The three patients receiving allo-SCT from unrelated or HLA-mismatched donors died of grade 3 to 4 acute GVHD and infection within 5 months after transplantation. The estimated disease-free survival at 4 years was 67% (95% confidence interval, 40-93%), and the overall survival was 75% (95% confidence interval, 50-99%). Our data suggest that allo-SCT should be considered early in the clinical course for young MDS patients with a poor prognosis and a matched sibling donor.