The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): International Prospective Validation and Reliability Trial in 402 patients

Blood - Tập 118 Số 2 - Trang 401-408 - 2011
Robyn M. Scherber1, Amylou C. Dueck2, Peter Johansson3, Tiziano Barbui4, Giovanni Barosi5, Alessandro M. Vannucchi6, Francesco Passamonti7, Björn Andréasson3, Maria L Ferarri8, Alessandro Rambaldi4, Jan Samuelsson9, Gunnar Birgegård10, Ayalew Tefferi11, Claire Harrison12, Deepti Radia12, Ruben A. Mesa13
1Rosalind Franklin School of Medicine, Chicago, IL;
2Section of Biostatistics, Mayo Clinic, Scottsdale, AZ
3Internal Medicine, NU Hospital Organization, Uddevalla, Sweden
4Unit of Hematology, Ospedali Riuniti di Bergamo, Bergamo, Italy
5Laboratory of Clinical Epidemiology, IRCCS Policlinico S. Matteo Foundation, Pavia, Italy;
6Hematology, Department of Medical and Surgical Care, University of Florence, Florence, Italy;
7Department of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy
8Biolology Science, Ospedali Riuniti di Bergamo, Bergamo, Italy;
9Department of Internal Medicine, Karolinska Institutet, Stockholm South Hospital, Stockholm, Sweden
10Department of Hematology, Uppsala University, Uppsala, Sweden
11Hematology, Mayo Clinic, Rochester, MN
12Department of Haematology, Guy's and St Thomas NHS Foundation Trust, London, United Kingdom; and
13Mayo Clinic, Scottsdale, AZ

Tóm tắt

Abstract 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.

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Blood

Tập 118 Số 2

401-408

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