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罕见病生存质量:618 例尤文肉瘤长期生存者的临床功能结局和体力活动的观察性队列研究。

Quality of Survivorship in a Rare Disease: Clinicofunctional Outcome and Physical Activity in an Observational Cohort Study of 618 Long-Term Survivors of Ewing Sarcoma.

机构信息

Andreas Ranft, Christiane Hoffmann, Ann-Christin Warby, Claudia Rossig, Uta Dirksen, and Herbert Juergens, University Children's Hospital; Corinna Seidel, and Dieter Rosenbaum, University Hospital Muenster; Michael Paulussen, Witten/Herdecke University, Datteln, Germany; Henk van den Berg, Emma Children's Hospital, Amsterdam, the Netherlands; and Ruth Ladenstein, Children's Cancer Research Institute, Wien, Austria.

出版信息

J Clin Oncol. 2017 May 20;35(15):1704-1712. doi: 10.1200/JCO.2016.70.6226. Epub 2017 Mar 29.

DOI:10.1200/JCO.2016.70.6226
PMID:28355116
Abstract

Purpose Significantly improved survival rates in patients with Ewing sarcoma have raised interest in accessing the quality of long-term survivorship. In this study, subjective and objective measurement tools, preclassified as physical or mental scores, were used to assess clinicofunctional outcome and physical activity after intensive bone tumor treatment. Methods Long-term outcome of 618 survivors from consecutive Ewing sarcoma trials was assessed by the Toronto Extremity Salvage Score, Short-Form Health Survey (SF-36), Brief Symptom Inventory (BSI), and Rosenberg Self-Esteem Scale questionnaires and by the accelerometric StepWatch 3 Activity Monitor. Prospective measurements were correlated retrospectively with standardized primary trial data. Results were compared with 316 nonrandom healthy peers by using effect sizes ( d). Median observation time was 12.9 years from primary diagnosis (range, 3.7 to 31.2 years). Results Absolute subjective scores were moderate to good for survivors. Compared with control subjects, unfavorable outcome was shown on physical Toronto Extremity Salvage Score, SF-36 Physical Component Summary, and BSI-Somatization scales (| d| ≥ 0.50; P < .01), in contrast to SF-36 Mental Component Summary, BSI-Anxiety, BSI-Depression, and Rosenberg Self-Esteem Scale mental scales (| d| ≤ 0.31). Survivors were less active than control subjects, as demonstrated by a step count difference of 1,742 steps per day ( d = -0.43; P < .01); however, on average, the recommended level for an active lifestyle was achieved (≥ 10,000 steps). Location of pelvic tumor was the major inferior disease-specific prognostic factor in physical scores ( P < .01), whereas nondisease-specific inferior factors in questionnaires were older age and female sex ( P < .01). Conclusion Survivors of Ewing sarcoma apparently returned to a normal life with minor limitations. Observed reductions in physical scores should be a focus in future research to optimize treatment strategies to reduce a negative impact on the quality of survivorship.

摘要

目的

尤因肉瘤患者的生存率显著提高,人们对评估长期生存质量的兴趣日益浓厚。本研究使用主观和客观测量工具,分为生理或心理评分,评估强化骨肿瘤治疗后的临床功能结局和身体活动。

方法

通过多伦多肢体挽救评分、健康调查简表(SF-36)、简明症状量表(BSI)和罗森伯格自尊量表对连续接受尤因肉瘤试验的 618 名幸存者进行长期预后评估,并使用加速度计 StepWatch 3 活动监测仪进行前瞻性测量。将结果与标准化的主要试验数据进行回顾性相关分析。使用效应大小(d)将结果与 316 名非随机健康同龄人进行比较。从原发性诊断开始的中位观察时间为 12.9 年(范围为 3.7 至 31.2 年)。

结果

幸存者的绝对主观评分中等至良好。与对照组相比,躯体 Toronto 肢体挽救评分、SF-36 生理成分综合评分和 BSI 躯体化量表的生存者预后不良(|d|≥0.50;P<0.01),而 SF-36 心理成分综合评分、BSI 焦虑量表、BSI 抑郁量表和罗森伯格自尊量表的心理评分则无差异(|d|≤0.31)。与对照组相比,幸存者的活动量较少,每天相差 1742 步(d=-0.43;P<0.01);然而,平均而言,达到了积极生活方式的推荐水平(≥10000 步)。骨盆肿瘤的位置是影响生理评分的主要疾病特异性预后因素(P<0.01),而问卷中影响非疾病特异性的预后因素是年龄较大和女性(P<0.01)。

结论

尤因肉瘤幸存者显然恢复了正常生活,仅有轻微的限制。观察到的生理评分下降应成为未来研究的重点,以优化治疗策略,减少对生存质量的负面影响。

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