Müller Lutz P, Müller-Tidow Carsten
Department of Internal Medicine IV, University Hospital of Halle (Saale).
Dtsch Arztebl Int. 2015 Apr 10;112(15):262-70. doi: 10.3238/arztebl.2015.0262.
The overall incidence of myeloid malignancies is 8.6 per 100 000 persons. Allogeneic stem-cell transplantation (SCT) is a major therapeutic option despite its risks, which include graft-versus-host disease (GvHD) and infection. In Germany, about 1600 patients with myeloid malignancies undergo SCT each year. The indications for SCT have changed since the introduction of tyrosine kinase inhibitors (TKI) and improved methods of SCT.
This article is based on relevant guidelines from Germany and abroad and on a selective review of the literature from 2010 onward.
The individual indication for SCT is based on the risk of disease progression, accompanying illnesses, the probability that SCT will result in cure, and the risk of complications. There is good evidence favoring allogeneic SCT in the following situations affecting 20% to 50% of patients with the respective disease: advanced chronic myeloid leukemia (CML) or CML that does not respond to TKI, Philadelphia chromosome-negative myeloproliferative neoplasm (Ph- MPN) or myelodysplastic syndrome (MDS) with a high risk of progression, and acute myeloid leukemia (AML) that has high-risk cytogenetic features or is recurrent. Good evidence is accumulating in favor of allogeneic SCT in older patients as well.
The prognosis of patients with myeloid neoplasm can now be assessed more accurately than before. This facilitates well-founded clinical decision-making about SCT, which is the only potentially curative treatment for most patients with myeloid neoplasm. Patients up to about age 75 should be referred to a transplantation center for consultation at an early stage of their disease so that the treatment options can be evaluated. A major goal of current research is to reduce toxicity with innovative forms of treatment.
髓系恶性肿瘤的总体发病率为每10万人8.6例。异基因干细胞移植(SCT)是一种主要的治疗选择,尽管存在风险,包括移植物抗宿主病(GvHD)和感染。在德国,每年约有1600例髓系恶性肿瘤患者接受SCT。自酪氨酸激酶抑制剂(TKI)的引入以及SCT方法的改进以来,SCT的适应症已经发生了变化。
本文基于德国和国外的相关指南以及对2010年以来文献的选择性综述。
SCT的个体适应症基于疾病进展风险、伴随疾病、SCT导致治愈的可能性以及并发症风险。有充分证据支持在以下影响相应疾病20%至50%患者的情况下进行异基因SCT:晚期慢性粒细胞白血病(CML)或对TKI无反应的CML、费城染色体阴性骨髓增殖性肿瘤(Ph-MPN)或具有高进展风险的骨髓增生异常综合征(MDS),以及具有高风险细胞遗传学特征或复发的急性髓系白血病(AML)。支持老年患者进行异基因SCT的证据也在不断积累。
现在可以比以前更准确地评估髓系肿瘤患者的预后。这有助于就SCT做出有充分依据的临床决策,SCT是大多数髓系肿瘤患者唯一可能治愈的治疗方法。年龄在75岁左右及以下的患者应在疾病早期转诊至移植中心进行咨询,以便评估治疗选择。当前研究的一个主要目标是通过创新的治疗形式降低毒性。