The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): International Prospective Validation and Reliability Trial in 402 patients
The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): International Prospective Validation and Reliability Trial in 402 patients
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DOI：
10.1182/blood-2011-01-328955
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发表时间：
2011-07-14
期刊：
影响因子：
20.3
通讯作者：
Mesa, Ruben A.
中科院分区：
文献类型：
--
作者：
Scherber, Robyn;Dueck, Amylou C.;Mesa, Ruben A.
大多数患者都存在骨髓增生性肿瘤的症状负担，并影响生活质量。我们试图验证一种广泛适用的 18 项工具（骨髓增生性肿瘤症状评估表 [MPN-SAF]，与简短疲劳量表共同使用），以评估美国、瑞典和意大利前瞻性队列中的骨髓纤维化、原发性血小板增多症和真性红细胞增多症的症状。总共对 161 名原发性血小板增多症患者、145 名真性红细胞增多症患者和 96 名骨髓纤维化患者进行了 402 项 MPN-SAF 调查（英语 [25%]、意大利语 [46%] 和瑞典语 [28%]）。在控制 MPN 亚型后，3 种管理语言的反应表现出极大的一致性。 MPN-SAF 和欧洲癌症研究与治疗组织生活质量问卷 C30 中的各个项目与关键症状要素之间存在很强的相关性。入组医生对患者症状的盲目意见（评估的 6 种症状）与相应患者的反应高度相关。对 53 名患者进行英国 MPN-SAF 的连续给药表明，大多数 MPN-SAF 项目具有良好的相关性（r > 0.5，P < .001）并且具有高度可重复性（组内相关系数 > 0.7）。 MPN-SAF 是一款全面、可靠的工具，有多种语言版本，可在全球临床试验中评估与所有类型 MPN 相关的症状。 (血液.2011;118(2):401-408)
Symptomatic burden in myeloproliferative neoplasms is present in most patients and compromises quality of life. We sought to validate a broadly applicable 18-item instrument (Myeloproliferative Neoplasm Symptom Assessment Form [MPN-SAF], coadministered with the Brief Fatigue Inventory) to assess symptoms of myelofibrosis, essential thrombocythemia, and polycythemia vera among prospective cohorts in the United States, Sweden, and Italy. A total of 402 MPN-SAF surveys were administered (English [25%], Italian [46%], and Swedish [28%]) in 161 patients with essential thrombocythemia, 145 patients with polycythemia vera, and 96 patients with myelofibrosis. Responses among the 3 administered languages showed great consistency after controlling for MPN subtype. Strong correlations existed between individual items and key symptomatic elements represented on both the MPN-SAF and the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-C30. Enrolling physicians' blinded opinion of patient symptoms (6 symptoms assessed) were highly correlated with corresponding patients' responses. Serial administration of the English MPN-SAF among 53 patients showed that most MPN-SAF items are well correlated (r > 0.5, P < .001) and highly reproducible (intraclass correlation coefficient > 0.7). The MPN-SAF is a comprehensive and reliable instrument that is available in multiple languages to evaluate symptoms associated with all types of MPNs in clinical trials globally. (Blood.2011;118(2):401-408)