Evaluating the serial use of the myelofibrosis symptom assessment form for measuring symptomatic improvement: Performance in 87 myelofibrosis patients on a JAK1 and JAK2 inhibitor (INCB018424) clinical trial

Ruben A. Mesa, Hagop Kantarjian, Ayalew Tefferi, Amylou Dueck, Richard Levy, Kris Vaddi, Susan Erickson-Viitanen, Deborah A. Thomas, Jorge Cortes, Gautam Borthakur, Animesh D. Pardanani, Zeev Estrov, Srdan Verstovsek

Research output: Contribution to journalArticle

39 Scopus citations


BACKGROUND: Symptomatic burden from constitutional symptoms, anemia, and splenomegaly-related symptoms are common and morbidity inducing in patients with myelofibrosis (MF). The authors previously developed a MF-specific instrument for capturing the burden of MF-associated disease-related symptoms, the Myelofibrosis Symptom Assessment Form. METHODS: The authors evaluated the usefulness of serial administration of the Myelofibrosis Symptom Assessment Form as an instrument for the assessment of symptomatic burden and improvement in conjunction with the therapeutic clinical trial of the open label phase 2 trial of the JAK1 and JAK2 inhibitor INCB018424 in patients with MF. RESULTS: The analysis cohort of 87 patients treated in this trial demonstrated that the instrument was comprehensive and sensitive to symptoms present at trial enrollment. In addition, baseline Myelofibrosis Symptom Assessment Form symptom scores correlated well with objective parameters such as splenomegaly and impaired performance status assessed by the 6-minute walk test. Serial administration while on therapy with INCB018424 demonstrated the instrument to be sensitive to symptomatic change, and that improvements in symptoms correlated well with objective improvements in both weight loss and performance status (6-minute walk test). CONCLUSIONS: The use of the Myelofibrosis Symptom Assessment Form in this phase 2 trial helped characterize the symptomatic improvements observed with use of INCB018424 in MF patients. In an era of many targeted therapies undergoing testing for MF with potential symptomatic benefit, the Myelofibrosis Symptom Assessment Form may provide a useful tool for objective symptomatic assessment and potentially allow some nonrandomized comparison between therapeutic agents.

Original languageEnglish (US)
Pages (from-to)4869-4877
Number of pages9
Issue number21
Publication statusPublished - Nov 1 2011



  • JAK2 inhibitor
  • Myelofibrosis Symptom Assessment Form
  • myelofibrosis
  • myeloproliferative neoplasms
  • symptoms

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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