Institute for Cancer Outcomes and Survivorship, University of Alabama at Birmingham, Birmingham, Alabama; Division of Pediatric Hematology and Oncology, University of Alabama at Birmingham, Birmingham, Alabama.
Institute for Cancer Outcomes and Survivorship, University of Alabama at Birmingham, Birmingham, Alabama.
Transplant Cell Ther. 2022 Mar;28(3):157.e1-157.e9. doi: 10.1016/j.jtct.2021.11.023. Epub 2021 Dec 5.
Survival after blood or marrow transplantation (BMT) for inborn errors of metabolism (IEM) is excellent; however, the burden of morbidity in long-term survivors of BMT for IEM remains understudied. This study examined the risk of chronic health conditions (CHC) in ≥2-year survivors of allogeneic BMT for IEM performed between 1974 and 2014 using the BMT Survivor Study. In this retrospective cohort study, participants (or their parents; n = 154) reported demographic data and CHCs (graded using Common Terminology Criteria for Adverse Events version 5), and transplantation characteristics were obtained from institutional databases. Unaffected siblings (n = 494) served as a comparison group. Logistic regression was used to estimated the odds of severe/life-threatening CHCs compared with siblings. Cox proportional hazards regression was used to estimate factors associated with severe/life-threatening/fatal CHCs in survivors of BMT for IEM. Survivors of allogeneic BMT for IEM (leukodystrophies, 43.5%; mucopolysaccharidoses, 41.0%) were at 12.5-fold higher odds of severe/life-threatening CHCs (95% confidence interval [CI], 5.4 to 28.9) compared with their siblings. The mean 10-year post-BMT cumulative incidence of grade 3-5 CHCs was 47.5 ± 4.0%. Reduced-intensity conditioning (RIC) was associated with a 2.7-fold higher risk (95% CI, 1.2 to 6.2; P = .02) of any grade 3-5 CHC, a 6.7-fold higher risk of grade 3-5 cardiopulmonary conditions (95% CI, 1.3 to 35.4), and a 3.0-fold higher risk of severe hearing/vision deficits (95% CI, 1.4 to 6.6). Older (age >26 years) BMT survivors were significantly less likely to graduate from college (odds ratio [OR], 0.3; 95% CI, 0.1 to 0.7) or marry (OR, 0.01; 95% CI, 0.004 to 0.07) compared with their siblings. Survivors of BMT for IEM carry a significant burden of morbidities, which affects their ability to attain adult milestones. Efforts to reduce chronic health conditions in this population are needed.
在接受血液或骨髓移植（BMT）治疗先天性代谢缺陷（IEM）后，患者的生存率很高；然而，IEM 患者 BMT 后长期生存者的发病率负担仍研究不足。本研究使用 BMT 生存者研究，调查了 1974 年至 2014 年间接受异基因 BMT 治疗的 IEM 患者中≥2 年的幸存者发生慢性健康状况（CHC）的风险。在这项回顾性队列研究中，参与者（或其父母；n=154）报告了人口统计学数据和 CHC（使用不良事件通用术语标准 5 进行分级），并从机构数据库中获取了移植特征。未受影响的兄弟姐妹（n=494）作为对照组。使用逻辑回归估计与兄弟姐妹相比，严重/危及生命的 CHC 的几率。使用 Cox 比例风险回归估计 IEM 患者 BMT 后发生严重/危及生命/致命性 CHC 的相关因素。与兄弟姐妹相比，接受异基因 BMT 治疗 IEM（白质营养不良症，43.5%；黏多糖贮积症，41.0%）的患者发生严重/危及生命的 CHC 的几率高 12.5 倍（95%置信区间 [CI]，5.4 至 28.9）。BMT 后 10 年的平均累积发生率为 3-5 级 CHC 为 47.5±4.0%。减强度预处理（RIC）与任何 3-5 级 CHC 的风险增加 2.7 倍（95%CI，1.2 至 6.2；P=0.02）、3-5 级心肺疾病的风险增加 6.7 倍（95%CI，1.3 至 35.4）和严重听力/视力缺陷的风险增加 3.0 倍（95%CI，1.4 至 6.6）相关。与兄弟姐妹相比，年龄较大（年龄>26 岁）的 BMT 幸存者从大学毕业的可能性显著降低（优势比[OR]，0.3；95%CI，0.1 至 0.7）或结婚（OR，0.01；95%CI，0.004 至 0.07）。接受 IEM BMT 的患者有显著的发病率负担，这影响了他们获得成年里程碑的能力。需要努力减少这一人群的慢性健康状况。
