title: Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study.
abstract: Patients with haemophilia A and inhibitors are at high risk for severe bleeding, progression of joint disease and deterioration of health-related quality of life (HRQoL). To determine the impact of prophylaxis with an activated prothrombin complex concentrate (aPCC) on HRQoL, HRQoL was assessed using the Short-Form (SF)-36 Health Survey and the EQ-5D questionnaire in subjects ≥ 14 years participating in a prospective, randomized, crossover study comparing 6 months of aPCC prophylaxis with 6 months of on-demand therapy. Eighteen of 19 patients completed the survey or questionnaire before and after the on-demand therapy and prophylaxis periods. A general trend towards improved HRQoL after prophylaxis was observed for the 18 evaluable patients in all SF-36 dimensions except for vitality/energy and physical functioning. After prophylaxis, 'good responders,' defined as patients experiencing ≥ 50% reduction in bleeding, exhibited statistically and clinically significant differences in the physical component score (P = 0.021), role - physical (P = 0.042), bodily pain (P = 0.015), and social functioning (P = 0.036). Similarly, the EQ-5D health profile showed a trend towards improvement after prophylaxis in all evaluable patients. Among the good responders, improvements did not differ from those observed after on-demand treatment. EQ visual analogue scale values were slightly improved following prophylaxis for all evaluable patients and the EQ-5D utility index improved in the good responders only. During prophylaxis, patients missed significantly fewer days from school or work because of bleeding than during on-demand treatment (P = 0.01). In conclusion, by significantly reducing bleeding frequency in good responders, aPCC prophylaxis improved HRQoL compared with on-demand treatment.
authors.affiliation: Department of Clinical Sciences and Community Health, Università degli Studi di Milano, Milan, Italy. alessandro.gringeri@gmail.com
authors.forename: A
authors.initials: A
authors.lastname: Gringeri
authors.forename: C
authors.initials: C
authors.lastname: Leissinger
authors.forename: P A
authors.initials: PA
authors.lastname: Cortesi
authors.forename: H
authors.initials: H
authors.lastname: Jo
authors.forename: F
authors.initials: F
authors.lastname: Fusco
authors.forename: S
authors.initials: S
authors.lastname: Riva
authors.forename: B
authors.initials: B
authors.lastname: Antmen
authors.forename: E
authors.initials: E
authors.lastname: Berntorp
authors.forename: C
authors.initials: C
authors.lastname: Biasoli
authors.forename: S
authors.initials: S
authors.lastname: Carpenter
authors.forename: K
authors.initials: K
authors.lastname: Kavakli
authors.forename: M
authors.initials: M
authors.lastname: Morfini
authors.forename: C
authors.initials: C
authors.lastname: Négrier
authors.forename: A
authors.initials: A
authors.lastname: Rocino
authors.forename: W
authors.initials: W
authors.lastname: Schramm
authors.forename: J
authors.initials: J
authors.lastname: Windyga
authors.forename: B
authors.initials: B
authors.lastname: Zülfikar
authors.forename: L G
authors.initials: LG
authors.lastname: Mantovani
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002675:Child, Preschool; D018592:Cross-Over Studies; D005169:Factor VIII / Q000276:immunology*; D005260:Female; D006467:Hemophilia A / Q000188:drug therapy* / Q000276:immunology / Q000523:psychology*; D006801:Humans; D007518:Isoantibodies / Q000096:biosynthesis / Q000276:immunology*; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D011516:Prothrombin / Q000008:administration & dosage*; D011788:Quality of Life; D011795:Surveys and Questionnaires; D055815:Young Adult
chemical_list: D007518:Isoantibodies; D011516:Prothrombin; D005169:Factor VIII
keywords: activated prothrombin complex concentrate; haemophilia; health-related quality of life; inhibitors; prophylaxis