Viollier Romaine, Passweg Jakob, Gregor Michael, Favre Geneviève, Kühne Thomas, Nissen Catherine, Gratwohl Alois, Tichelli André
Transplantation Unit, University Hospitals of Basel, Petersgraben 4, 4031 Basel, Switzerland.
Ann Hematol. 2005 Jan;84(1):47-55. doi: 10.1007/s00277-004-0930-3. Epub 2004 Aug 31.
Bone marrow transplantation (BMT) and immunosuppression (IS) have improved the prognosis of aplastic anemia; both treatments have specific advantages and drawbacks but similar survival rates. Analysis of additional endpoints may help in treatment decisions. In a single-center study, patients with aplastic anemia treated with IS (n=155) or BMT (n=52) were compared for survival, event-free survival, and quality-adjusted time without symptoms and toxicity (Q-TWiST). Probability of overall and event-free survival at 15 years was similar among both groups (BMT 51+/-15% and 25+/-14%, IS 53+/-10% and 27+/-8%), with more early deaths in the transplant group and more late deaths in the IS group. There were differences in terms of mean duration of seven analyzed health states: time with symptoms from treatment-related toxicity (IS 0.36 years, BMT 0.27), transfusion dependency (IS 0.66 years, BMT 0.1 years), partial remission (IS 3.27 years, BMT 1.42), and secondary clonal disorder (IS 0.68 years, BMT 0.04) was significantly longer for IS compared to BMT (p< or =0.001). Patients treated with BMT spent more time with extensive chronic graft-versus-host disease (GvHD) (IS 0 years, BMT 0.96, p<0.023) and in CR without drugs (IS 1.22 years, BMT 2.43, p=0.056). In conclusion, survival, event-free survival, and Q-TWiST are similar. BMT-treated patients had longer periods free from symptoms, while IS-treated patients needed closer medical care, transfusion support, and medications.
骨髓移植（BMT）和免疫抑制（IS）改善了再生障碍性贫血的预后；这两种治疗方法都有各自的优缺点，但生存率相似。对其他终点指标的分析可能有助于治疗决策。在一项单中心研究中，对接受IS治疗（n = 155）或BMT治疗（n = 52）的再生障碍性贫血患者的生存率、无事件生存率以及无症状和无毒性的质量调整时间（Q-TWiST）进行了比较。两组患者15年的总生存率和无事件生存率相似（BMT组分别为51±15%和25±14%，IS组分别为53±10%和27±8%），移植组早期死亡更多，IS组晚期死亡更多。在分析的七种健康状态的平均持续时间方面存在差异：与治疗相关毒性导致的有症状时间（IS组0.36年，BMT组0.27年）、输血依赖时间（IS组0.66年，BMT组0.1年）、部分缓解时间（IS组3.27年，BMT组1.42年）以及继发性克隆性疾病时间（IS组0.68年，BMT组0.04年），IS组均显著长于BMT组（p≤0.001）。接受BMT治疗的患者出现广泛慢性移植物抗宿主病（GvHD）的时间更长（IS组0年，BMT组0.96年，p<0.023），且在无药物的完全缓解状态下的时间更长（IS组1.22年，BMT组2.43年，p = 0.056）。总之，生存率、无事件生存率和Q-TWiST相似。接受BMT治疗的患者无症状期更长，而接受IS治疗的患者需要更密切的医疗护理、输血支持和药物治疗。
