Department of Pediatrics, Taipei Veterans General Hospital and National Yang-Ming University School of Medicine, Taipei, Taiwan, R.O.C.
Pediatric Department, Children's Hospital Colorado, Aurora, CO.
Blood. 2020 May 21;135(21):1847-1858. doi: 10.1182/blood.2019003858.
Patient-reported outcomes among survivors of pediatric hematopoietic stem cell transplant (HSCT) are understudied. We compared symptom prevalence, health-related quality of life (HRQOL), and risk factors in adult survivors of childhood hematologic malignancies treated with HSCT to those treated with conventional therapy and noncancer controls. Survivors of childhood hematologic malignancies (HSCT N = 112 [70% allogeneic, 30% autologous]; conventionally treated N = 1106) and noncancer controls (N = 242) from the St. Jude Lifetime Cohort Study completed surveys assessing 10 symptom domains and SF-36 HRQOL summary scores. Chronic health conditions (CHCs) were validated by clinical assessment. Multivariable logistic regression reveals that compared with noncancer controls, HSCT survivors endorsed a significantly higher symptom prevalence in sensation (OR = 4.7, 95% confidence interval [CI], 2.6-8.4), motor/movement (OR = 4.3, 95% CI, 1.6-11.0), pulmonary (OR = 4.6, 95% CI, 1.8-11.8), and memory domains (OR = 4.8, 95% CI, 2.5-9.2), and poorer physical HRQOL (OR = 6.9, 95% CI, 2.8-17.0). HSCT and conventionally treated survivors had a similar prevalence of all symptom domains and HRQOL (all P > .05); however, HSCT survivors had a significantly higher cumulative prevalence for specific symptoms: double vision (P = .04), very dry eyes (P < .0001), and trouble seeing when wearing glasses (P < .0001). Occurrence of organ-specific CHCs, instead of transplant receipt, was significantly associated with a higher prevalence of all symptom domains (all P < .05) in adult survivors of childhood cancer, except for pain and anxiety domains. This study found that patient-reported outcomes were equally impaired between HSCT and conventionally treated survivors, but poorer in both groups compared with noncancer controls. Poor patient-reported outcomes in all survivors of childhood hematologic malignancies correlated with the presence of CHCs, whether treated with conventional therapy or HSCT.
在接受造血干细胞移植 (HSCT) 的儿科血液病幸存者中，患者报告的结局研究较少。我们比较了接受 HSCT 治疗的儿童血液恶性肿瘤幸存者、接受常规治疗的幸存者和非癌症对照者的症状流行率、健康相关生活质量 (HRQOL) 和危险因素。来自圣裘德终身队列研究的儿童血液恶性肿瘤幸存者 (HSCT N=112[70%异基因，30%自体];常规治疗 N=1106) 和非癌症对照者 (N=242) 完成了评估 10 个症状域和 SF-36 HRQOL 综合评分的调查。慢性健康状况 (CHC) 通过临床评估得到验证。多变量逻辑回归显示，与非癌症对照者相比，HSCT 幸存者在感觉 (OR=4.7，95%置信区间 [CI]，2.6-8.4)、运动/运动 (OR=4.3，95%CI，1.6-11.0)、肺 (OR=4.6，95%CI，1.8-11.8) 和记忆域 (OR=4.8，95%CI，2.5-9.2) 中报告的症状流行率显著更高，且物理 HRQOL 更差 (OR=6.9，95%CI，2.8-17.0)。HSCT 和常规治疗幸存者的所有症状域和 HRQOL 患病率相似 (均 P>.05);然而，HSCT 幸存者有更高的特定症状的累积患病率：复视 (P=.04)、眼睛非常干燥 (P<.0001) 和戴眼镜时视力困难 (P<.0001)。与器官特异性 CHC 的发生而非移植接受情况显著相关，除疼痛和焦虑域外，与儿童癌症幸存者的所有症状域患病率较高有关 (均 P<.05)。本研究发现，HSCT 和常规治疗幸存者的患者报告结局同样受损，但与非癌症对照者相比，两组患者的报告结局更差。所有儿童血液恶性肿瘤幸存者的患者报告结局较差与 CHC 的存在相关，无论是否接受常规治疗还是 HSCT 治疗。
