title: Use of the Functional Assessment of Cancer Therapy--anemia in persons with myeloproliferative neoplasm-associated myelofibrosis and anemia.
abstract: BACKGROUND
  Anemia is common in myeloproliferative neoplasm (MPN)-associated myelofibrosis. The Functional Assessment of Cancer Therapy (FACT) measurement system is a patient-reported outcomes instrument that documents symptoms of the diverse aspects of cancer treatment. One FACT version, FACT-Anemia (FACT-An), documents symptoms of anemia related to cancer. The FACT-An has been validated in diverse cancer populations, but not in MPN-associated myelofibrosis.
  
  
  OBJECTIVE
  Our aim was to evaluate the relationship between anemia response to therapy with pomalidomide with or without corticosteroids and patient-reported outcomes using the FACT-An instrument.
  
  
  METHODS
  Data were obtained from a Phase II, randomized, double-blind Bayesian pick-the-winner trial of prednisone and pomalidomide in patients with MPN-associated myelofibrosis and anemia (red blood cell-transfusion dependence). Details of the study, including definitions of anemia, anemia response, red blood cell-transfusion, red blood cell-transfusion dependence, and red blood cell-transfusion independence, are reported. Change in quality of life from randomization to the last cycle of therapy was evaluated using the FACT-An Physical Well Being, Functional Well Being, Trial Outcome Index, and Anemia domains. Clinically important differences were used to determine the smallest difference in scores that patients perceived as beneficial in the FACT-An domains of interest. Patients were classified as meeting clinically important differences for responsiveness if their change score from baseline was >1 SEM, indicating improvement.
  
  
  RESULTS
  Eighty-five patients were studied. Thirty-one patients (37%) were classified as anemia responders by prospectively defined criteria. Across all FACT-An domains, anemia responders showed greater improvement in Physical Well Being, Functional Well Being, and Trial Outcome Index scores than did nonresponders. This improvement began at the second 28-day cycle of therapy and was sustained.
  
  
  CONCLUSIONS
  We show a correlation between anemia response and improved quality of life measured by the FACT-An instrument in patients with MPN-associated myelofibrosis and anemia.
authors.affiliation: Mayo Clinic, Rochester, Minnesota.
authors.forename: Ayalew
authors.initials: A
authors.lastname: Tefferi
authors.affiliation: Adelphi Values, Boston, Massachusetts. Electronic address: Stacie.hudgens@clinoutsolutions.com.
authors.forename: Stacie
authors.initials: S
authors.lastname: Hudgens
authors.affiliation: Mayo Clinic Cancer Center, Arizona.
authors.forename: Ruben
authors.initials: R
authors.lastname: Mesa
authors.affiliation: Celgene Corporation, Summit, New Jersey.
authors.forename: Robert Peter
authors.initials: RP
authors.lastname: Gale
authors.affiliation: The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
authors.affiliation: Department of Internal Medicine, Ospedale di Circolo, Varese, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Passamonti
authors.affiliation: Hematology Department, Hospital Clínic, IDIBAPS, University of Barcelona, Spain.
authors.forename: Francisco
authors.initials: F
authors.lastname: Cervantes
authors.affiliation: Division of Hematology, Department of Medicine, Mayo Clinic, Jacksonville, Florida.
authors.forename: Candido
authors.initials: C
authors.lastname: Rivera
authors.affiliation: Celgene Corporation, Summit, New Jersey.
authors.forename: Tom
authors.initials: T
authors.lastname: Tencer
authors.affiliation: Celgene Corporation, Summit, New Jersey.
authors.forename: Zeba M
authors.initials: ZM
authors.lastname: Khan
mesh_terms: D000740:Anemia / Q000188:drug therapy* / Q000209:etiology; D001499:Bayes Theorem; D004311:Double-Blind Method; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D009196:Myeloproliferative Disorders / Q000150:complications*; D009369:Neoplasms / Q000188:drug therapy; D011241:Prednisone / Q000627:therapeutic use*; D055728:Primary Myelofibrosis / Q000150:complications*; D011788:Quality of Life; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D013792:Thalidomide; C467566:pomalidomide; D011241:Prednisone
keywords: FACT-An; MPN-associated myelofibrosis; anemia; cancer; clinical trial; fatigue; patient-reported outcome; prednisone; quality of life; responsiveness; validation study