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Treatment Option Overview

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Allogeneic bone marrow transplantation (BMT) for young patients with MDS offers the potential for long-term DFS.[39] In two large studies, 45% to 60% of patients with de novo MDS were projected to be long-term disease-free survivors.[48,49] Outcome tends to be better in younger patients with fewer bone marrow blasts, but long-term benefit has been noted in all FAB classification types, and in patients with marrow fibrosis, a variety of karyotypic findings, and different preparative regimens.[48,49,50] A retrospective review of outcomes of allogeneic BMT according to pretransplant IPSS score showed that the IPSS score predicted relapse rate and DFS. The 5-year DFS rates were 60% for the low-risk and intermediate-1 risk group, 36% for the intermediate-2 risk group, and 28% for the high-risk group.[51][Level of evidence: 3iiDii] A review of 118 young MDS patients (median age 24, age range 0.3-53 years) who received allogeneic BMT from matched unrelated donors reported an actuarial survival of 28% at 2 years. Transplant-related mortality was influenced by the age of the patient (18 years or younger, 40%; age 18 to 35 years, 61%; 35 years or older, 81%). Relapse rate was influenced by FAB classification. This study included patients who received transplants as early as 1986, which may have influenced the patient survival data.[52][Level of evidence: 3iiiA] Outcomes may not be as good for patients with treatment-related MDS (5-year dfs of 8% to 30%).[53]

Allogeneic stem cell transplantation with nonmyeloablative conditioning is under clinical evaluation for treatment of MDS. A retrospective analysis of 836 allogeneic transplants for MDS using HLA-matched sibling donors was performed and included 215 patients who received nonmyeloablative conditioning regimens. The 3-year probabilities of progression-free survival and OS were similar in both groups (39% after myeloablative condition vs. 33% in reduced intensity conditioning RIC and 45% vs. 41%, respectively; these differences were not significant). Relapses were more common in the reduced intensity group, but nonrelapse mortality was decreased.[54][Level of evidence: 3iiiA]

The farnesyl transferase inhibitor tipifarnib, which is under clinical evaluation, has been examined in 82 patients: 32% of patients responded, and 15% had complete responses. Median response duration was 11 months.[55][Level of evidence: 3iiiDiv]. Arsenic trioxide induced major hematologic improvement in approximately 20% of 185 MDS patients treated in two multicenter phase II trials.[56,57]

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