The Ottawa Hospital, Ottawa, Ontario, Canada.
CHU de Poitiers, CIC 1402, Poitiers, France.
Arthritis Rheumatol. 2021 Feb;73(2):305-314. doi: 10.1002/art.41519. Epub 2020 Dec 29.
To quantify the magnitude, domains, and duration of change in health-related quality of life (HRQoL) in patients with systemic sclerosis (SSc) who underwent autologous hematopoietic stem cell transplantation (HSCT) as compared to SSc patients with similar characteristics who did not undergo autologous HSCT.
The study was designed as a retrospective study comparing SSc patients who underwent autologous HSCT and SSc patients who met the criteria for transplantation but were treated with conventional care. Outcomes included scores on the 36-item Short Form (SF-36) health survey and the Health Assessment Questionnaire (HAQ) and its disease-specific symptom scales. Differences in scores between the groups were compared using linear models, adjusting for baseline scores and inverse probability of treatment and censoring weights.
In total, 41 SSc patients who underwent autologous HSCT and 65 SSc patients treated with conventional care were compared. In marginal linear weighted models, the SF-36 physical component summary score was a mean ± SEM 7.02 ± 1.94 points higher at the first annual visit (P = 0.001) and 14.40 ± 6.16 points higher at the seventh annual visit (P = 0.03) in patients treated with autologous HSCT compared to the conventional care group. HAQ scores were significantly better in the autologous HSCT group compared to the conventional care group during follow-up (mean ± SEM difference from baseline -0.57 ± 0.13 [P < 0.001] at the first annual visit and -0.94 ± 0.49 [P = 0.07] at the seventh annual visit). There were no differences in the SF-36 mental component summary scores between the 2 groups either at baseline or during follow-up.
This study provides robust complementary HRQoL data, including overall and event-free survival data, to expand on the standard repertoire of biomedical variables, thus potentially supporting the physical benefits of autologous HSCT in patients with SSc.
与未接受自体造血干细胞移植（HSCT）的具有相似特征的 SSc 患者相比，量化接受自体 HSCT 的系统性硬化症（SSc）患者的健康相关生活质量（HRQoL）变化的幅度、域和持续时间。
该研究设计为回顾性研究，比较接受自体 HSCT 的 SSc 患者和符合移植标准但接受常规治疗的 SSc 患者。结果包括 36 项简短健康调查（SF-36）和健康评估问卷（HAQ）及其疾病特异性症状量表的评分。使用线性模型比较两组之间的评分差异，调整基线评分、治疗逆概率和删失权重。
共比较了 41 例接受自体 HSCT 的 SSc 患者和 65 例接受常规治疗的 SSc 患者。在边际线性加权模型中，自体 HSCT 治疗组在首次年度就诊时 SF-36 生理成分综合评分平均±SEM 高 7.02±1.94 分（P=0.001），在第七年就诊时高 14.40±6.16 分（P=0.03），与常规治疗组相比。在随访期间，HAQ 评分在自体 HSCT 组明显优于常规治疗组（与基线相比的平均差异-0.57±0.13[P<0.001]，首次就诊时，-0.94±0.49[P=0.07]，第七年就诊时）。两组在基线或随访期间 SF-36 心理成分综合评分均无差异。
本研究提供了强大的 HRQoL 数据补充，包括总体和无事件生存率数据，以扩展生物医学变量的标准范围，从而可能支持 SSc 患者自体 HSCT 的身体益处。
