title: Changes in health-related quality of life with treatment of longer-acting clotting factors: results in the A-LONG and B-LONG clinical studies.
abstract: BACKGROUND
  In haemophilia, prophylactic infusion of replacement factor can result in improvements in health-related quality of life (HRQoL) when compared with episodic treatment. The Haemophilia-specific Quality of Life (Haem-A-QoL) questionnaire assessed HRQoL in adults with severe haemophilia A or B who received prophylactic or episodic treatment with recombinant factor VIII or IX Fc fusion protein (rFVIIIFc or rFIXFc) in the A-LONG or B-LONG clinical studies.
  
  
  OBJECTIVE
  Understand changes in HRQoL during the A-LONG and B-LONG trials.
  
  
  METHODS
  Group-level and individual-level changes over time for the Haem-A-QoL key domains of 'Physical Health' and 'Sports & Leisure,' and 'Total Score' were evaluated in adults through baseline and 6-month HRQoL assessments. Previously determined responder definitions (RDs) were used for evaluating meaningful subject-level HRQoL improvements.
  
  
  RESULTS
  The analysis included 67 A-LONG and 51 B-LONG subjects who completed the Haem-A-QoL (baseline and 6 months). While HRQoL improvements were observed among all treatment groups, greater improvements in HRQoL were observed among subjects who received episodic treatment pre-study (and prophylaxis on-study) compared to those who received hyphenate prophylaxis. Applying the RDs for interpreting 6-month changes, 47.4%/33.3% ('Physical Health'), 35.9%/50.0% ('Sports & Leisure') and 23.9%/33.3% ('Total Score') of A-LONG subjects who received individualized or weekly prophylaxis were classified as HRQoL responders. In B-LONG, 69.2%/57.9% ('Physical Health'), 44.4%/56.7% ('Sports & Leisure') and 41.7%/44.1% ('Total Score') of subjects who received individualized or weekly prophylaxis were classified as HRQoL responders.
  
  
  CONCLUSIONS
  Changes in Haem-A-QoL key domains and 'Total Score' suggest that prophylaxis with long-acting rFVIIIFc or rFIXFc resulted in meaningful HRQoL improvements.
authors.affiliation: Evidera, Bethesda, MD, USA.
authors.forename: K W
authors.initials: KW
authors.lastname: Wyrwich
authors.affiliation: Biogen, Inc., Cambridge, MA, USA.
authors.forename: S
authors.initials: S
authors.lastname: Krishnan
authors.affiliation: Evidera, Bethesda, MD, USA.
authors.forename: P
authors.initials: P
authors.lastname: Auguste
authors.affiliation: Evidera, Bethesda, MD, USA.
authors.forename: J-L
authors.initials: JL
authors.lastname: Poon
authors.affiliation: Evidera, Bethesda, MD, USA.
authors.forename: R
authors.initials: R
authors.lastname: von Maltzahn
authors.affiliation: Evidera, Bethesda, MD, USA.
authors.forename: R
authors.initials: R
authors.lastname: Yu
authors.affiliation: Biogen, Inc., Cambridge, MA, USA.
authors.forename: G F
authors.initials: GF
authors.lastname: Pierce
authors.affiliation: Biogen, Inc., Cambridge, MA, USA.
authors.forename: B
authors.initials: B
authors.lastname: Mei
authors.affiliation: Adult Hemophilia Comprehensive Care Unit at University of the Witwatersrand, Faculty of Health Sciences Charlotte Maxeke Johannesburg Hospital, Johannesburg, South Africa.
authors.forename: J
authors.initials: J
authors.lastname: Mahlangu
authors.affiliation: Institute of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.
authors.forename: S
authors.initials: S
authors.lastname: von Mackensen
mesh_terms: D000328:Adult; D000368:Aged; D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D005260:Female; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D011795:Surveys and Questionnaires; D055815:Young Adult
chemical_list: D001779:Blood Coagulation Factors
keywords: haemophilia; prophylactic treatment; quality of life; recombinant factor IX replacement; recombinant factor VIII replacement; responder definition