Kim D H, Sohn S K, Baek J H, Lee K-H, Lee J-H, Choi S-J, Shin I-H
Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu, Korea.
Bone Marrow Transplant. 2007 Oct;40(8):779-84. doi: 10.1038/sj.bmt.1705806. Epub 2007 Aug 13.
GVHD-specific survival (GSS) has been investigated as a potential study end point to describe the clinical course and outcome of chronic GVHD (cGVHD). However, reaching this end point requires a long observation time. We hypothesized that the time to the first flare-up (FFU) of cGVHD (TTF) can be an alternative statistical end point to GSS. This retrospective study included 96 patients with a diagnosis of cGVHD from a cohort of 119 patients with a prior history of acute GVHD. The median TTF was 73 days after the diagnosis of cGVHD. The 2-year cumulative incidences of first, second and third episodes of flare-up (FU) during courses of cGVHD were estimated as 69.5, 46.4 and 22.1%. Those patients who did not have an episode of FU of cGVHD had 96.0% of 2-years GSS rate, while those with 1 and > or =2 episodes had 50.8 and 46.8%, respectively (P=0.001). Shorter TTF was associated with poor GSS and decreased overall survival. The shorter TTF during the course of cGVHD was significantly associated with extensive cGVHD (P=0.002), Hopkins' risk category (P=0.022) and progressive-type cGVHD (P<0.001) in multivariate analysis. We propose that TTF can be an alternative end point to GSS in cGVHD trials.
移植物抗宿主病特异性生存率（GSS）已被作为一种潜在的研究终点进行调查，以描述慢性移植物抗宿主病（cGVHD）的临床病程和结局。然而，达到这一终点需要较长的观察时间。我们假设cGVHD首次病情突然加重（FFU）的时间（TTF）可作为GSS的替代统计终点。这项回顾性研究纳入了119例有急性移植物抗宿主病病史队列中的96例cGVHD诊断患者。cGVHD诊断后TTF的中位数为73天。cGVHD病程中首次、第二次和第三次病情突然加重（FU）发作的2年累积发生率估计分别为69.5%、46.4%和22.1%。那些没有cGVHD病情突然加重发作的患者2年GSS率为96.0%，而有1次和≥2次发作的患者分别为50.8%和46.8%（P = 0.001）。较短的TTF与较差的GSS和降低的总生存率相关。在多变量分析中，cGVHD病程中较短的TTF与广泛型cGVHD（P = 0.002）、霍普金斯风险类别（P = 0.022）和进行性cGVHD（P < 0.001）显著相关。我们提出TTF可作为cGVHD试验中GSS的替代终点。
