Department of Internal Medicine III, University Hospital Regensburg, Regensburg, Germany.
Klinik für Innere Medizin II, Abteilung für Hämatologie und Internistische Onkologie, Universitätsklinikum Jena, Jena, Germany.
Biol Blood Marrow Transplant. 2019 Jul;25(7):1450-1455. doi: 10.1016/j.bbmt.2019.03.003. Epub 2019 Mar 13.
Chronic graft-versus-host disease (cGVHD) remains the leading cause of late morbidity and mortality. Despite the growing number of treatment options in cGVHD, evidence remains sparse. The German-Austrian-Swiss GVHD Consortium performed a survey on clinical practice in treatment of cGVHD among transplant centers in Germany, Austria, and Switzerland in 2009 and 2018 and compared the results. The survey performed in 2009 contained 20 questions on first-line treatment and related issues and 4 questions on second-line scenarios followed by a survey on all systemic and topic treatment options known and applied, with 31 of 36 transplant centers (86%) responding. The survey in 2018 repeated 7 questions on first-line treatment and 3 questions on second-line scenarios followed by an updated survey on all current systemic treatment options known and applied, with 29 of 66 centers (43%) responding. In summary, the results show a large overlap of first-line treatment practice between centers and the 2 surveys because of a lack of new data that changes practice, except significant heterogeneity of treatment of cGVHD progressive onset type, which can be explained by the lack of trials focusing on this high-risk entity. In contrast, treatment options applied to second-line therapy vary considerably, with new agents like ibrutinib and ruxolitinib entering clinical practice. Moreover, treatment of bronchiolitis obliterans syndrome demonstrates heterogeneity in applied therapeutic options and sequence because of a lack of controlled data and different conclusions from already existing evidence. In summary, the survey results demonstrate an increasing number of treatment options applied to cGVHD accompanied by a significant heterogeneity in second-line treatment and underline the urgent need for clinical trials and registry analyses on rare entities with high mortality like progressive onset type and lung involvement of cGVHD.
慢性移植物抗宿主病（cGVHD）仍然是导致晚期发病率和死亡率的主要原因。尽管 cGVHD 的治疗选择越来越多，但证据仍然很少。德国-奥地利-瑞士 GVHD 联合会在 2009 年和 2018 年对德国、奥地利和瑞士的移植中心治疗 cGVHD 的临床实践进行了调查，并比较了结果。2009 年进行的调查包含了 20 个关于一线治疗和相关问题的问题，以及 4 个关于二线方案的问题，随后是一个关于所有已知和应用的系统性和专题治疗方案的调查，36 个移植中心中的 31 个（86%）做出了回应。2018 年的调查重复了 7 个关于一线治疗的问题和 3 个关于二线方案的问题，随后是一个关于所有已知和应用的当前系统性治疗方案的更新调查，66 个中心中的 29 个（43%）做出了回应。总的来说，结果表明中心之间的一线治疗实践存在很大的重叠，并且由于缺乏改变实践的新数据，两次调查之间存在很大的重叠，除了 cGVHD 进行性发病类型的治疗存在显著异质性，这可以解释为缺乏专注于这一高风险实体的试验。相比之下，二线治疗中应用的治疗方案差异很大，新的药物如伊布替尼和鲁索替尼已进入临床实践。此外，由于缺乏对照数据和现有证据的不同结论，闭塞性细支气管炎综合征的治疗方案在应用的治疗方案和顺序方面存在异质性。总的来说，调查结果表明，应用于 cGVHD 的治疗方案越来越多，二线治疗存在显著异质性，并强调了对进展性发病类型和 cGVHD 肺部受累等死亡率高的罕见实体进行临床试验和登记分析的迫切需要。
