Pulmonary function and long-term survival in patients with PERDS after autologous hematopoietic stem cell transplantation.
Pulmonary function and long-term survival in patients with PERDS after autologous hematopoietic stem cell transplantation.
复制标题
自体造血干细胞移植后 PERDS 患者的肺功能和长期生存。
DOI：
10.1038/s41409-023-02101-5
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发表时间：
2023
影响因子：
4.8
通讯作者：
Yadav,Hemang
中科院分区：
文献类型：
--
作者：
Zhang,Zhenmei;Wieruszewski,PatrickM;HefaziTorghabeh,Mehrdad;Hogan,WilliamJ;Yadav,Hemang
植皮周围呼吸窘迫综合征（PERDS）是一种肺部并发症，发生在约5%的自体造血细胞移植（HCT）受者中。它是累及肺部的移植物综合征的一个亚型[1,2]。先前的研究表明，女性、更快的植入时间、更频繁的血小板输注和POEMS（多神经病变、器官肿大、内分泌病变、单克隆γ病和皮肤异常）综合征都与PERDS的发生有关，且PERDS患者的短期生存率低于无PERDS患者。肺功能受损，如15秒用力呼气量（FEV1）低、用力肺活量（FVC）低和一氧化碳弥散能力（DLCO）低，与HCT后肺部并发症的风险增加有关[4,5]。没有数据评估肺功能受损是否会影响PERDS的风险。此外，没有PERDS幸存者的长期随访报告。因此，我们进行了这项研究，a)评估移植前肺功能和PERDS风险，b)确定发生PERDS患者的长期预后。这是一项对2005年1月1日至2016年12月31日期间在明尼苏达州罗切斯特市梅奥诊所接受自体HCT的成人患者队列的二次分析。本研究由梅奥诊所机构审查委员会批准，机构审查放弃书面知情同意书。PERDS定义为影像学上新发肺浸润、缺氧伴新需氧量、非感染或心力衰竭所致临床综合征、中性粒细胞移植后5天内（移植后中性粒细胞绝对计数连续3天超过500细胞/μl的第1天）发生[3]。患者进行肺功能测试（PFT）作为移植前评估的一部分，我们收集他们在HCT之前最近的FEV1、FVC和DLCO作为基线。肺功能评分（LFS）由FEV1和DLCO百分比预测评分之和计算，> 80%= 1,70 - 80%= 2,60 - 70%= 3,< 60%= 4 >。在PERDS患者中，我们通过医疗记录确定了那些在PERDS发作后出现PFT的患者，并回顾了那些没有重复PFT的患者肺部症状的临床文献。收集最后一次随访、疾病复发和死亡日期进行生存分析。当病人失去随访或5年后还活着时，他们就会被审查。汇总统计包括连续变量的均值和标准差，分类变量的频率，并在适当时使用双侧Student 's t检验，配对Student 's t检验或chisqusquared检验进行比较。多变量logistic模型纳入了先前已知的PERDS和PFT测量的风险因素[3]。死亡率分析采用Kaplan-Meier估计和Cox比例风险模型。进行竞争风险分析以评估疾病复发对总生存期的影响。p值< 0.05认为有统计学意义。使用BlueSky Statistics （Chicago， IL， USA）和R Studio集成开发环境（Boston， MA， USA）进行统计分析。2005年至2016年，3473例患者接受了自体HCT并有植入数据。其中发生PERDS 167例（4.8%），需机械通气33例（20%），100 d死亡9例（5.7%）。移植前肺活量测定3457例（99.5%），DLCO 3442例（99.1%）。将患有PERDS的患者与未患PERDS的患者进行比较，
Peri-engraftment respiratory distress syndrome (PERDS) is a pulmonary complication occurring in around 5% of autologous hematopoietic cell transplant (HCT) recipients. It is a subset of engraftment syndrome with pulmonary involvement [1, 2]. Prior studies showed that female sex, faster time-to-engraftment, more frequent platelet transfusions and POEMS (Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal gammopathy and Skin abnormalities) syndrome are all associated with PERDS development and short-term survival of PERDS patients is lower than those without PERDS [3]. Impaired pulmonary function such as low forced expiratory volume in 1s (FEV1), low forced vital capacity (FVC) and low diffusing capacity for carbon monoxide (DLCO) is associated with increased risk of pulmonary complications after HCT [4, 5]. No data have evaluated whether impaired pulmonary function affects the risk of PERDS. Also, no long-term follow-up of PERDS survivors has been reported. Therefore, we conducted this study to a) evaluate pre-transplant pulmonary function and PERDS risk, and b) determine longer-term outcomes in patients who developed PERDS. This was a secondary analysis of a previously defined cohort of adult patients who underwent autologous HCT at Mayo Clinic, Rochester, Minnesota between January 1, 2005 and December 31, 2016 [3]. This study was approved by the Mayo Clinic Institutional Review Board and written informed consent was waived by institutional review. PERDS was defined as new pulmonary infiltrates on imaging, hypoxia with new oxygen requirement, clinical syndrome not due to infection or heart failure, and occurring within 5 days of either side of neutrophil engraftment (the first of three consecutive days when the absolute neutrophil count exceeded 500 cells/μl after transplantation)[3]. Patients had pulmonary function testing (PFT) as part of pretransplant evaluation, and we collected their most recent FEV1, FVC and DLCO prior to HCT as baseline. Lung Function Score (LFS) was calculated from the sum of FEV1 and DLCO percent predicted scores where> 80%= 1, 70–80%= 2, 60–70%= 3 and< 60%= 4 [6]. In patients with PERDS, we identified those with PFT after their PERDS episodes through medical records and reviewed clinical documentation for pulmonary symptoms in those without repeat PFT. Dates of last follow-up, disease relapse and death were collected for survival analysis. Patients were censored when they were lost to follow up or were alive after 5 years. Summary statistics included mean and standard deviation for continuous variables and frequency for categorical variables, and compared using two-sided Student’s t test, paired Student’s t test or chisquared test when appropriate. Multivariable logistic model incorporated previously known risk factors for PERDS and PFT measurements [3]. Kaplan–Meier estimate and Cox proportional hazard model were used to analyze mortality. Competing risk analysis was performed to evaluate the impact of disease relapse on overall survival. A p-value< 0.05 was considered statistically significant. Statistical analysis was performed using BlueSky Statistics (Chicago, IL, USA) and R Studio Integrated Development Environment (Boston, MA, USA). Between 2005 and 2016, 3473 patients underwent autologous HCT and had engraftment data. Among them, 167 developed PERDS (4.8%) of which 33 (20%) required mechanical ventilation and 9 died at 100 days (5.7%)[3]. Pre-transplant spirometry was available for 3457 patients (99.5%) and DLCO for 3442 patients (99.1%). Comparing patients who developed PERDS to those who did not, patients with …