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

Chad Gwaltney, Jean Paty, Virginia E. Kwitkowski, Ruben A. Mesa, Amylou Dueck, Elektra J. Papadopoulos, Lixia Wang, Joseph Feliciano, Stephen Joel Coons

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)26-31
Number of pages6
JournalLeukemia Research
Volume59
DOIs
StatePublished - Aug 1 2017

Fingerprint

Primary Myelofibrosis
Clinical Trials
Symptom Assessment
Patient Reported Outcome Measures
Surveys and Questionnaires
Critical Pathways
Sweat
Ribs
Pruritus
Self Report
Abdominal Pain
Uncertainty
Fatigue
Consensus
Spleen
Bone and Bones
Pain

Keywords

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

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

Development of a harmonized patient-reported outcome questionnaire to assess myelofibrosis symptoms in clinical trials. / Gwaltney, Chad; Paty, Jean; Kwitkowski, Virginia E.; Mesa, Ruben A.; Dueck, Amylou; Papadopoulos, Elektra J.; Wang, Lixia; Feliciano, Joseph; Coons, Stephen Joel.

In: Leukemia Research, Vol. 59, 01.08.2017, p. 26-31.

Research output: Contribution to journalArticle

Gwaltney, C, Paty, J, Kwitkowski, VE, Mesa, RA, Dueck, A, Papadopoulos, EJ, Wang, L, Feliciano, J & Coons, SJ 2017, 'Development of a harmonized patient-reported outcome questionnaire to assess myelofibrosis symptoms in clinical trials', Leukemia Research, vol. 59, pp. 26-31. https://doi.org/10.1016/j.leukres.2017.05.012
Gwaltney, Chad ; Paty, Jean ; Kwitkowski, Virginia E. ; Mesa, Ruben A. ; Dueck, Amylou ; Papadopoulos, Elektra J. ; Wang, Lixia ; Feliciano, Joseph ; Coons, Stephen Joel. / Development of a harmonized patient-reported outcome questionnaire to assess myelofibrosis symptoms in clinical trials. In: Leukemia Research. 2017 ; Vol. 59. pp. 26-31.
@article{cea041223b7a47cab585da3efc0d19ce,
title = "Development of a harmonized patient-reported outcome questionnaire to assess myelofibrosis symptoms in clinical trials",
abstract = "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.",
keywords = "Clinical trials, Myelofibrosis, Patient-reported outcome, Symptoms",
author = "Chad Gwaltney and Jean Paty and Kwitkowski, {Virginia E.} and Mesa, {Ruben A.} and Amylou Dueck and Papadopoulos, {Elektra J.} and Lixia Wang and Joseph Feliciano and Coons, {Stephen Joel}",
year = "2017",
month = "8",
day = "1",
doi = "10.1016/j.leukres.2017.05.012",
language = "English (US)",
volume = "59",
pages = "26--31",
journal = "Leukemia Research",
issn = "0145-2126",
publisher = "Elsevier Limited",

}

TY - JOUR

T1 - Development of a harmonized patient-reported outcome questionnaire to assess myelofibrosis symptoms in clinical trials

AU - Gwaltney, Chad

AU - Paty, Jean

AU - Kwitkowski, Virginia E.

AU - Mesa, Ruben A.

AU - Dueck, Amylou

AU - Papadopoulos, Elektra J.

AU - Wang, Lixia

AU - Feliciano, Joseph

AU - Coons, Stephen Joel

PY - 2017/8/1

Y1 - 2017/8/1

N2 - 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.

AB - 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.

KW - Clinical trials

KW - Myelofibrosis

KW - Patient-reported outcome

KW - Symptoms

UR - http://www.scopus.com/inward/record.url?scp=85019875079&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85019875079&partnerID=8YFLogxK

U2 - 10.1016/j.leukres.2017.05.012

DO - 10.1016/j.leukres.2017.05.012

M3 - Article

C2 - 28544906

AN - SCOPUS:85019875079

VL - 59

SP - 26

EP - 31

JO - Leukemia Research

JF - Leukemia Research

SN - 0145-2126

ER -