title: Effect of acute bleeding on daily quality of life assessments in patients with congenital hemophilia with inhibitors and their families: observations from the dosing observational study in hemophilia.
abstract: BACKGROUND
  Quality-of-life (QOL) assessments in frequently bleeding patients with congenital hemophilia with inhibitors and their families are confounded by preexisting arthropathy and family circumstances. Periodic QOL assessments typically made on nonbleed days may not provide complete reflections of the burden on patients/families.
  
  
  OBJECTIVE
  To evaluate the impact of bleeding episodes on patients/caregivers/families and the association between monthly QOL scores and patients' average diary experiences.
  
  
  METHODS
  Frequently bleeding inhibitor patients (≥four bleeds in 3 months), or their caregivers, provided daily assessment of EuroQol five-dimensional questionnaire and EuroQol five-dimensional questionnaire visual analogue scale, pain (11-point Likert scale), and family anxiety/stress/activity change over 3 to 6 months. QOL scores were stratified by bleed/nonbleed days.
  
  
  RESULTS
  Patient QOL assessments were recorded by 37 of the 39 enrolled patients/caregivers (3771 of 3777 eligible dairy days, 472 bleed/3299 nonbleed days). Median (range) diary duration was 91 (66-180) days, with 8.2% (0%-72.2%) bleed days. Mean health scores were significantly worse on bleed days than on nonbleed days (P < 0.0001 for all): EuroQol five-dimensional questionnaire index, 0.66 versus 0.82; visual analogue scale health, 69.7 versus 77.4; and pain score, 4.1 versus 1.8. Bleed days also had higher (P < 0.001) proportions of days with abnormalities in family anxiety/stress (42% vs. 30%) and family activity changes (34% vs. 21%).
  
  
  CONCLUSIONS
  Assessing the impact of hemophilia with inhibitors on patient/family QOL typically includes periodic (likely nonbleed day) evaluations reflecting baseline abnormalities. Daily assessment, however, indicated that frequent acute bleeds impair QOL beyond patient's nonbleed day baseline. New approaches are required to assess the cumulative impact of frequent acute bleeds on patients and their families.
authors.affiliation: Children's Hospital Boston, Boston, MA 02115, USA. ellis.neufeld@childrens.harvard.edu
authors.forename: Ellis J
authors.initials: EJ
authors.lastname: Neufeld
authors.forename: Michael
authors.initials: M
authors.lastname: Recht
authors.forename: Hernan
authors.initials: H
authors.lastname: Sabio
authors.forename: Kapil
authors.initials: K
authors.lastname: Saxena
authors.forename: Caitlyn T
authors.initials: CT
authors.lastname: Solem
authors.forename: A Simon
authors.initials: AS
authors.lastname: Pickard
authors.forename: Robert Z
authors.initials: RZ
authors.lastname: Gut
authors.forename: David L
authors.initials: DL
authors.lastname: Cooper
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005190:Family / Q000523:psychology*; D006467:Hemophilia A / Q000276:immunology* / Q000503:physiopathology*; D006470:Hemorrhage / Q000523:psychology*; D006801:Humans; D007223:Infant; D007518:Isoantibodies / Q000378:metabolism*; D008297:Male; D008875:Middle Aged; D036301:Qualitative Research; D011788:Quality of Life / Q000523:psychology*; D011795:Surveys and Questionnaires; D014481:United States; D055815:Young Adult
chemical_list: D007518:Isoantibodies