Gratwohl A, Pfirrmann M, Zander A, Kröger N, Beelen D, Novotny J, Nerl C, Scheid C, Spiekermann K, Mayer J, Sayer H G, Falge C, Bunjes D, Döhner H, Ganser A, Schmidt-Wolf I, Schwerdtfeger R, Baurmann H, Kuse R, Schmitz N, Wehmeier A, Fischer J Th, Ho A D, Wilhelm M, Goebeler M-E, Lindemann H W, Bormann M, Hertenstein B, Schlimok G, Baerlocher G M, Aul C, Pfreundschuh M, Fabian M, Staib P, Edinger M, Schatz M, Fauser A, Arnold R, Kindler T, Wulf G, Rosselet A, Hellmann A, Schäfer E, Prümmer O, Schenk M, Hasford J, Heimpel H, Hossfeld D K, Kolb H-J, Büsche G, Haferlach C, Schnittger S, Müller M C, Reiter A, Berger U, Saußele S, Hochhaus A, Hehlmann R
Klinik für Hämatologie, Universitätsspital Basel, Basel, Switzerland.
Institut für Medizinische Informationsverarbeitung, Biometrie und Epidemiologie (IBE), Ludwig-Maximilians-Universität München, München, Germany.
Leukemia. 2016 Mar;30(3):562-9. doi: 10.1038/leu.2015.281. Epub 2015 Oct 14.
Tyrosine kinase inhibitors represent today's treatment of choice in chronic myeloid leukemia (CML). Allogeneic hematopoietic stem cell transplantation (HSCT) is regarded as salvage therapy. This prospective randomized CML-study IIIA recruited 669 patients with newly diagnosed CML between July 1997 and January 2004 from 143 centers. Of these, 427 patients were considered eligible for HSCT and were randomized by availability of a matched family donor between primary HSCT (group A; N=166 patients) and best available drug treatment (group B; N=261). Primary end point was long-term survival. Survival probabilities were not different between groups A and B (10-year survival: 0.76 (95% confidence interval (CI): 0.69-0.82) vs 0.69 (95% CI: 0.61-0.76)), but influenced by disease and transplant risk. Patients with a low transplant risk showed superior survival compared with patients with high- (P<0.001) and non-high-risk disease (P=0.047) in group B; after entering blast crisis, survival was not different with or without HSCT. Significantly more patients in group A were in molecular remission (56% vs 39%; P=0.005) and free of drug treatment (56% vs 6%; P<0.001). Differences in symptoms and Karnofsky score were not significant. In the era of tyrosine kinase inhibitors, HSCT remains a valid option when both disease and transplant risk are considered.
酪氨酸激酶抑制剂是目前慢性髓性白血病（CML）的首选治疗方法。异基因造血干细胞移植（HSCT）被视为挽救疗法。这项前瞻性随机CML-III A研究在1997年7月至2004年1月期间从143个中心招募了669例新诊断的CML患者。其中，427例患者被认为符合HSCT条件，并根据是否有匹配的家族供体随机分为初次HSCT组（A组；N = 166例患者）和最佳可用药物治疗组（B组；N = 261例）。主要终点是长期生存。A组和B组的生存概率没有差异（10年生存率：0.76（95%置信区间（CI）：0.69 - 0.82）对0.69（95%CI：0.61 - 0.76）），但受疾病和移植风险影响。移植风险低的患者与B组中高风险（P < 0.001）和非高风险疾病（P = 0.047）的患者相比，生存率更高；进入急变期后，接受或未接受HSCT的患者生存率没有差异。A组分子缓解的患者明显更多（56%对39%；P = 0.005），且无需药物治疗的患者更多（56%对6%；P < 0.001）。症状和卡诺夫斯基评分的差异不显著。在酪氨酸激酶抑制剂时代，当同时考虑疾病和移植风险时，HSCT仍然是一个有效的选择。
