title: Changes in quality of life and disease-related symptoms in patients with polycythemia vera receiving ruxolitinib or standard therapy.
abstract: OBJECTIVE
  Polycythemia vera (PV)-related symptoms may not be adequately controlled with conventional therapy. This current analysis of the RESPONSE trial evaluated the effects of ruxolitinib compared with standard therapy on quality of life (QoL) and symptoms in patients with PV who were hydroxyurea resistant/intolerant.
  
  
  METHODS
  In the previously reported primary analysis, ruxolitinib achieved the primary composite endpoint of hematocrit control and ≥35% reduction in spleen volume at Week 32. The current analysis evaluated patient-reported outcomes using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF), the Pruritus Symptom Impact Scale (PSIS), and the Patient Global Impression of Change (PGIC).
  
  
  RESULTS
  Compared with standard therapy, ruxolitinib was associated with greater improvements in global health status/QoL, functional subscales, and individual symptom scores of the EORTC QLQ-C30. At Week 32, more patients in the ruxolitinib arm (44%) achieved a ≥10-point improvement in global health status/QoL vs. standard therapy (9%). Improvements in MPN-SAF symptom scores were consistent with improvements in EORTC QLQ-C30, PSIS, and PGIC scores.
  
  
  CONCLUSIONS
  Ruxolitinib provides clinically relevant improvements in QoL and ameliorates symptom burden in patients with PV who are hydroxyurea resistant/intolerant.
authors.affiliation: Mayo Clinic Cancer Center, Scottsdale, AZ, USA.
authors.forename: Ruben
authors.initials: R
authors.lastname: Mesa
authors.affiliation: The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
authors.affiliation: Hôpital Saint-Louis et Université Paris Diderot, Paris, France.
authors.forename: Jean-Jacques
authors.initials: JJ
authors.lastname: Kiladjian
authors.affiliation: Johannes Wesling Clinic, Minden, Germany.
authors.forename: Martin
authors.initials: M
authors.lastname: Griesshammer
authors.affiliation: St. István and St. László Hospital, Budapest, Hungary.|Semmelweis University, Budapest, Hungary.
authors.forename: Tamas
authors.initials: T
authors.lastname: Masszi
authors.affiliation: Royal Brisbane & Women's Hospital, Brisbane, Qld, Australia.
authors.forename: Simon
authors.initials: S
authors.lastname: Durrant
authors.affiliation: Ospedale di Circolo e Fondazione Macchi, Varese, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Passamonti
authors.affiliation: Guy's and St. Thomas' NHS Foundation Trust, London, UK.
authors.forename: Claire N
authors.initials: CN
authors.lastname: Harrison
authors.affiliation: University of Naples Federico II, Naples, Italy.
authors.forename: Fabrizio
authors.initials: F
authors.lastname: Pane
authors.affiliation: ZNA Stuivenberg, Antwerp, Belgium.
authors.forename: Pierre
authors.initials: P
authors.lastname: Zachee
authors.affiliation: Incyte Corporation, Wilmington, DE, USA.
authors.forename: Huiling
authors.initials: H
authors.lastname: Zhen
authors.affiliation: Incyte Corporation, Wilmington, DE, USA.
authors.forename: Mark M
authors.initials: MM
authors.lastname: Jones
authors.affiliation: Incyte Corporation, Wilmington, DE, USA.
authors.forename: Shreekant
authors.initials: S
authors.lastname: Parasuraman
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
authors.forename: Jingjin
authors.initials: J
authors.lastname: Li
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Côté
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.affiliation: University of Florence, Florence, Italy.
authors.forename: Alessandro M
authors.initials: AM
authors.lastname: Vannucchi
mesh_terms: D000328:Adult; D000368:Aged; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009570:Nitriles; D000071066:Patient Reported Outcome Measures; D011087:Polycythemia Vera / Q000175:diagnosis / Q000453:epidemiology* / Q000628:therapy*; D047428:Protein Kinase Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D011720:Pyrazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D011743:Pyrimidines; D011788:Quality of Life; D059039:Standard of Care; D016896:Treatment Outcome
chemical_list: D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib
keywords: polycythemia vera; quality of life; signs and symptoms