Deeg H Joachim, Guardiola Philippe
Fred Hutchinson Cancer Research Center, and University of Washington, Seattle, USA.
Int J Hematol. 2002 Aug;76 Suppl 2:29-34. doi: 10.1007/BF03165082.
Myelodysplastic syndrome (MDS) and myeloproliferative disorders associated with myelofibrosis (MF) are stem cell disorders, and hemopoietic stem cell transplantation (HSCT) is currently the only therapy with curative potential. Among patients with less advanced MDS, 3 year survivals of 65% to 70% are achievable with HLA-identical related and HLA-matched unrelated donors. The probability of relapse is < 5%. Among patients with advanced disease (> or = 5% marrow blasts), about 35 to approximately 45% and 25 to approximately 30%, respectively, are surviving in remission after transplantation from related or unrelated donors. The incidence of post-transplant relapse is 1035%. Criteria of the International Prognostic Scoring System (IPSS), originally developed for nontransplanted patients, also predict survival following transplantation. Patients with MF, either idiopathic or on the basis of pre-existing disorders, are also transplanted successfully with stem cells from related or unrelated donors. Transplants early in the disease, before leukemic transformation, are successful in 60 to approximately 80% of patients. Success rates are lower in patients who have developed MDS or leukemia. New conditioning regimens have permitted successful HSCT even in patients in the seventh decade of life. Results with a regimen using a combination of busulfan (targeted to predetermined plasma levels) and cyclophosphamide are particularly encouraging. Improved survival with transplants from unrelated volunteer donors may, in part, reflect selection of donors on the basis of high resolution (allele-level) HLA typing. Nevertheless, transplant-related morbidity and mortality, including graft- vs. -host disease, remain challenges that need to be addressed with innovative approaches.
骨髓增生异常综合征（MDS）以及与骨髓纤维化（MF）相关的骨髓增殖性疾病均为干细胞疾病，而造血干细胞移植（HSCT）是目前唯一具有治愈潜力的疗法。在病情不太严重的MDS患者中，使用人类白细胞抗原（HLA）匹配的相关供体和HLA匹配的无关供体，3年生存率可达65%至70%。复发概率小于5%。在病情较重（骨髓原始细胞≥5%）的患者中，分别接受相关或无关供体移植后，约35%至45%和约25%至30%的患者可存活于缓解期。移植后复发率为10%至35%。最初为未接受移植的患者制定的国际预后评分系统（IPSS）标准，也可预测移植后的生存率。原发性或基于既往疾病的MF患者，也可成功接受来自相关或无关供体的干细胞移植。在疾病早期、白血病转化之前进行移植，60%至约80%的患者可获得成功。已发展为MDS或白血病的患者成功率较低。新的预处理方案甚至使70岁患者也能成功进行HSCT。使用白消安（靶向预定血浆水平）和环磷酰胺联合方案的结果尤其令人鼓舞。来自无关志愿供体的移植提高了生存率，这可能部分反映了基于高分辨率（等位基因水平）HLA分型选择供体。然而，包括移植物抗宿主病在内的移植相关发病率和死亡率，仍然是需要通过创新方法加以解决的挑战。
