Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: Prospective International Assessment of an Abbreviated Symptom Burden Scoring System Among Patients With MPNs
Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: Prospective International Assessment of an Abbreviated Symptom Burden Scoring System Among Patients With MPNs
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DOI：
10.1200/jco.2012.42.3863
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发表时间：
2012-11-20
影响因子：
45.3
通讯作者：
Mesa, Ruben A.
中科院分区：
文献类型：
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作者：
Emanuel, Robyn M.;Dueck, Amylou C.;Mesa, Ruben A.
目的骨髓增生性肿瘤(MPN)的症状是困扰患者的问题，减轻这一负担是发展MPN导向治疗的首要治疗目标。我们的目的是评估最相关和最具代表性的MPN症状的简明症状评分在随后的治疗反应评估中的应用。患者和方法骨髓增生性肿瘤症状评估表总症状评分(MPN-SAF TSS)计算来自两个先前验证的评分系统的10个项目的平均分数。问题集中在疲劳、注意力集中、早饱、不活动、盗汗、瘙痒、骨痛、腹部不适、体重减轻和发烧。结果1,433例MPNS患者中有1,408例可以计算MPN-SAF TSS，平均得分为21.2(标准差[SD]，16.3)。MPN-SAF TSS结果在MPN疾病亚型之间有显著差异(P<；.001)，在原发性血小板增多症、真性红细胞增多症和骨髓纤维化患者中，平均值分别为18.7(SD，15.3)、21.8(SD，16.3)和25.3(SD，17.2)。MPN-SAF TSS与总体生活质量(QOL；r=0.59；P<；.001)和欧洲癌症研究与治疗组织生活质量问卷C30(EORTC QLQ-C30)功能量表(除社会功能r=0.48外，所有P<；.001和绝对值r&>=0.50)显著相关。当比较治疗亚组时，没有出现明显的趋势。MPN-SAF TSS具有良好的内部一致性(Cronbach‘sα=.83)。结论MPN-SAF TSS是一种简明、有效、准确的MPN症状负担评估方法，在迄今为止最大规模的MPN前瞻性症状研究中显示出临床实用价值。这种新的前瞻性评分方法可用于在临床实践和试验环境中评估MPN症状负担。
PurposeMyeloproliferative neoplasm (MPN) symptoms are troublesome to patients, and alleviation of this burden represents a paramount treatment objective in the development of MPN-directed therapies. We aimed to assess the utility of an abbreviated symptom score for the most pertinent and representative MPN symptoms for subsequent serial use in assessing response to therapy.Patients and MethodsThe Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (MPN-SAF TSS) was calculated as the mean score for 10 items from two previously validated scoring systems. Questions focus on fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers.ResultsMPN-SAF TSS was calculable for 1,408 of 1,433 patients with MPNs who had a mean score of 21.2 (standard deviation [SD], 16.3). MPN-SAF TSS results significantly differed among MPN disease subtypes (P < .001), with a mean of 18.7 (SD, 15.3), 21.8 (SD, 16.3), and 25.3 (SD, 17.2) for patients with essential thrombocythemia, polycythemia vera, and myelofibrosis, respectively. The MPN-SAF TSS strongly correlated with overall quality of life (QOL; r = 0.59; P < .001) and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) functional scales (all P < .001 and absolute r >= 0.50 except social functioning r = 0.48). No significant trends were present when comparing therapy subgroups. The MPN-SAF TSS had excellent internal consistency (Cronbach's alpha = .83). Factor analysis identified a single underlying construct, indicating that the MPN-SAF TSS is an appropriate, unified scoring method.ConclusionThe MPN-SAF TSS is a concise, valid, and accurate assessment of MPN symptom burden with demonstrated clinical utility in the largest prospective MPN symptom study to date. This new prospective scoring method may be used to assess MPN symptom burden in both clinical practice and trial settings.