Development of a harmonized patient-reported outcome questionnaire to assess myelofibrosis symptoms in clinical trials

作者：Gwaltney Chad^*; Paty Jean; Kwitkowski Virginia E; Mesa Ruben A; Dueck Amylou C; Papadopoulos Elektra J; Wang Lixia; Feliciano Joseph; Coons Stephen Joel

来源：Leukemia Research, 2017, 59: 26-31.

DOI：10.1016/j.leukres.2017.05.012

• Myelofibrosis
• Symptoms
• Patient-reported outcome
• Clinical trials

Along with reducing spleen size, relieving symptom severity is a key objective of the treatment of myelofibrosis (MF). Several questionnaires have been developed for patient self-report of MF symptoms in clinical trials and each includes unique instructions, items, and/or response scales. This variability in questionnaire content increases uncertainty; it is unclear which questionnaire is the most appropriate for assessing MF symptoms and it makes comparisons across trials difficult. The Patient-Reported Outcome (PRO) Consortium's MF Working Group (WG) was established to review existing MF symptom questionnaires and to develop a harmonized, consensus-based PRO questionnaire for use in future MF trials. The WG focused on the seven core symptoms of MF: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. The resulting Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) asks respondents to report symptom severity at its worst for each of the seven items on a 0 (Absent) to 10 (Worst Imaginable) numeric rating scale. The MFSAF v4.0, for which there are 24-h and 7-day recall formats, will be maintained and licensed by the Critical Path Institute and made publicly available for use in future clinical trials.

• 出版日期2017-8