Division of Hematology and Oncology, Mayo Clinic, 13400 E. Shea Blvd, Scottsdale, AZ 85259, USA.
J Clin Oncol. 2012 Nov 20;30(33):4098-103. doi: 10.1200/JCO.2012.42.3863. Epub 2012 Oct 15.
Myeloproliferative 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.
The 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.
MPN-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 α=.83). Factor analysis identified a single underlying construct, indicating that the MPN-SAF TSS is an appropriate, unified scoring method.
The 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.
骨髓增殖性肿瘤（MPN）症状给患者带来了困扰，减轻这种负担是开发 MPN 靶向治疗的首要治疗目标。我们旨在评估一种简化的症状评分，用于评估最相关和最具代表性的 MPN 症状，以便随后连续评估治疗反应。
骨髓增殖性肿瘤症状评估表总症状评分（MPN-SAF TSS）是通过两个先前验证的评分系统的 10 个项目的平均评分计算得出。问题集中在疲劳、注意力、早饱、不活动、盗汗、瘙痒、骨痛、腹部不适、体重减轻和发热。
1433 例 MPN 患者中有 1408 例可计算 MPN-SAF TSS，平均得分为 21.2（标准差[SD]，16.3）。MPN-SAF TSS 结果在 MPN 疾病亚型之间存在显著差异（P<.001），分别为特发性血小板增多症、真性红细胞增多症和骨髓纤维化患者的平均 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）功能量表强烈相关（均 P<.001，绝对 r≥0.50，除社交功能 r=0.48 外）。在比较治疗亚组时，没有出现显著趋势。MPN-SAF TSS 具有良好的内部一致性（Cronbach's α=.83）。因子分析确定了一个单一的潜在结构，表明 MPN-SAF TSS 是一种适当的、统一的评分方法。
MPN-SAF TSS 是一种简洁、有效和准确的 MPN 症状负担评估方法，在迄今为止最大的前瞻性 MPN 症状研究中显示出了临床实用性。这种新的前瞻性评分方法可用于评估临床实践和试验环境中的 MPN 症状负担。
