title: 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.
abstract: 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.
authors.affiliation: Department of Hematology, Mayo Clinic, Scottsdale, Arizona.
authors.forename: Ruben A
authors.initials: RA
authors.lastname: Mesa
authors.affiliation: Department of Hematology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Hagop
authors.initials: H
authors.lastname: Kantarjian
authors.affiliation: Department of Hematology, Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Ayalew
authors.initials: A
authors.lastname: Tefferi
authors.affiliation: Department of Biostatistics, Mayo Clinic, Scottsdale, Arizona.
authors.forename: Amylou
authors.initials: A
authors.lastname: Dueck
authors.affiliation: Department of Hematology, Incyte, Wilmington, Delaware.
authors.forename: Richard
authors.initials: R
authors.lastname: Levy
authors.affiliation: Department of Hematology, Incyte, Wilmington, Delaware.
authors.forename: Kris
authors.initials: K
authors.lastname: Vaddi
authors.affiliation: Department of Hematology, Incyte, Wilmington, Delaware.
authors.forename: Susan
authors.initials: S
authors.lastname: Erickson-Viitanen
authors.affiliation: Department of Hematology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Deborah A
authors.initials: DA
authors.lastname: Thomas
authors.affiliation: Department of Hematology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Jorge
authors.initials: J
authors.lastname: Cortes
authors.affiliation: Department of Hematology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Gautam
authors.initials: G
authors.lastname: Borthakur
authors.affiliation: Department of Hematology, Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Animesh D
authors.initials: AD
authors.lastname: Pardanani
authors.affiliation: Department of Hematology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Zeev
authors.initials: Z
authors.lastname: Estrov
authors.affiliation: Department of Hematology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
mesh_terms: D017322:Clinical Trials, Phase II as Topic; D005260:Female; D006801:Humans; D053613:Janus Kinase 1 / Q000037:antagonists & inhibitors*; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors*; D008297:Male; D009570:Nitriles; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000188:drug therapy*; D011720:Pyrazoles / Q000627:therapeutic use*; D011743:Pyrimidines; D013163:Splenomegaly / Q000175:diagnosis; D011795:Surveys and Questionnaires
chemical_list: D009570:Nitriles; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib; D053613:Janus Kinase 1; D053614:Janus Kinase 2