Development of a symptom assessment in patients with myelofibrosis: qualitative study findings

Health Qual Life Outcomes. 2019 Apr 11;17(1):61. doi: 10.1186/s12955-019-1121-1.


Background: The goal of the research reported here was to understand the patient experience of living with myelofibrosis (MF) and establish content validity of the Modified Myeloproliferative Neoplasm Symptom Assessment Diary (MPN-SD).

Methods: Qualitative interviews were performed in patients with MF, including both concept elicitation and cognitive debriefing. Patients with MF were asked to spontaneously report on their signs, symptoms, and impacts of MF, as well as their understanding of the MPN-SD content, and use of the tool on an electronic platform. A supplementary literature review and meetings with MF experts were also performed.

Results: Twenty-three patients with MF participated in qualitative interviews. Signs and symptoms most commonly reported by ruxolitinib-experienced patients (n = 16) were: fatigue and/or tiredness (n = 16, 100%), shortness of breath (n = 11, 69%), pain below the ribs on the left side and/or stomach pain and/or abdominal pain (n = 9, 56%), and enlarged spleen (n = 9, 56%) and for ruxolitinib-naïve patients (n = 7) were: fatigue and/or tiredness (n = 6, 86%), pain below the ribs on the left side (n = 6, 86%), enlarged spleen (n = 4, 57%), full quickly/filling up quickly (n = 4, 57%), night sweats and/or general sweats (n = 4, 57%), and itching (n = 4, 57%). Patients demonstrated that they were able to read, understand, and provide meaningful responses to the MPN-SD. The final version of the MPN-SD includes the 10 most commonly reported concepts from the MF patient interviews.

Conclusions: The findings demonstrate the comprehensiveness of the MPN-SD in assessing MF symptoms in both ruxolitinib-experienced and ruxolitinib-naïve patients, while remaining easy for patients to understand and complete.

Keywords: Janus kinase inhibitors; Myeloproliferative disorders; Patient reported outcome measures; Primary myelofibrosis; Product labeling; Symptom assessment.

Publication types

  • Validation Study

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Fatigue / etiology
  • Female
  • Humans
  • Interviews as Topic
  • Male
  • Middle Aged
  • Primary Myelofibrosis / physiopathology
  • Primary Myelofibrosis / psychology*
  • Pyrazoles / therapeutic use
  • Qualitative Research
  • Quality of Life*
  • Severity of Illness Index
  • Symptom Assessment / standards*


  • INCB018424
  • Pyrazoles