Permanent impairment (PI) of vital organs is one of the transplantation-related health problems affecting the quality of life and morbidity even in patients who do not develop graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (allo-HCT), but no data are available on PI of multiple organs. This retrospective study aimed to estimate a novel composite endpoint of PI-free, relapse-free survival (PIRFS) in 164 allo-HCT recipients. We defined PI as >26% to 30% impairment of the whole person in 6 vital organs using the whole person impairment rating. Conventional GVHD-free/relapse-free survival (GRFS) and PIRFS at 5 years were 33.8% (95% confidence interval [CI], 26.5% to 41.3%) and 40.6% (95% CI, 32.6% to 48.4%), respectively. In the whole cohort, PIRFS was higher than GRFS at any time after allo-HCT. However, PIRFS was lower than GRFS after day 397 post-transplantation in patients who underwent umbilical cord blood transplantation (UCBT). In UCBT recipients, 5-year GRFS and PIRFS were 47.6% (95% CI, 34.3% to 59.7%) and 39.2% (95% CI, 26.6% to 51.5%), respectively. The cumulative incidence of PI after 5 years was 20.9% (95% CI, 13.7% to 29.0%) in patients surviving for ≥6 months without relapse. The multivariate analysis revealed that high disease risk (hazard ratio [HR], 1.91; 95% CI, 1.26 to 2.88; P < .01) and Karnofsky Performance Status score ≤90% at transplantation (HR, 1.73; 95% CI, 1.14 to 2.63; P = .01) were correlated with the lower PIRFS, whereas UCBT (HR, 2.35; 95% CI, 1.11 to 4.99; P = .03), grade III-IV acute GVHD by day 180 (HR, 3.59; 95% CI, 1.04 to 12.4; P = .04), and thrombotic microangiopathy by day 180 (HR, 2.74; 95% CI, 1.10 to 6.87; P = .03) were significantly correlated with a higher incidence of PI. More than 20% of long-term survivors had PI. Our data suggest that PIRFS is a useful endpoint for assessing long-term transplantation success from a different perspective than has been established previously.

中文翻译：

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永久无损伤，无复发生存：评估异基因移植长期成功的新型复合终点。

甚至在异基因造血干细胞移植（allo-HCT）后未发生移植物抗宿主病（GVHD）的患者中，重要器官的永久性损伤（PI）是影响生活质量和发病率的与移植相关的健康问题之一），但没有多个器官的PI数据。这项回顾性研究旨在评估164名All-HCT接受者的无PI，无复发生存（PIRFS）的新型复合终点。我们使用全人损伤等级将PI定义为6个重要器官中全人的损伤> 26％至30％。常规的5年无GVHD /无复发生存率（GRFS）和PIRFS分别为33.8％（95％置信区间[CI]，26.5％至41.3％）和40.6％（95％CI，32.6％至48.4％），分别。在整个队列中，异基因HCT后任何时间的PIRFS均高于GRFS。然而，脐带血移植（UCBT）患者在移植后397天后的PIRFS低于GRFS。在UCBT接受者中，五年期GRFS和PIRFS分别为47.6％（95％CI，34.3％至59.7％）和39.2％（95％CI，26.6％至51.5％）。存活≥6个月而未复发的患者，5年后PI的累积发生率为20.9％（95％CI，13.7％至29.0％）。多因素分析显示，移植时疾病风险高（危险比[HR]，1.91； 95％CI，1.26至2.88； P <.01），Karnofsky行为状态评分≤90％（HR，1.73； 95％CI，1.14）至2.63; P = .01）与较低的PIRFS相关，而UCBT（HR，2.35; 95％CI，1.11至4.99; P = .03），第180天的III-IV级急性GVHD（HR，3.59; 95％CI，1.04至12.4; P = .04），以及第180天血栓性微血管病（HR，2.74; 95％CI，1.10至6）。87; P = .03）与更高的PI发生率显着相关。超过20％的长期幸存者患有PI。我们的数据表明，PIRFS是从以前不同的角度评估长期移植成功的有用终点。