Health-related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors-Results from the HAVEN 2 study.

Content Provider	Europe PMC
Author	Mancuso, Maria Elisa Mahlangu, Johnny Sidonio, Robert Trask, Peter Uguen, Marianne Chang, Tiffany Shima, Midori Young, Guy Oldenburg, Johannes von Mackensen, Sylvia
Abstract	AbstractIntroductionPersons with haemophilia A (PwHA) with factor (F)VIII inhibitors, including children, have impaired health‐related quality of life (HRQoL). The HAVEN 2 study (NCT027955767) of paediatric PwHA with FVIII inhibitors demonstrated that subcutaneous emicizumab prophylaxis resulted in low annualizedbleed rates.AimWe assessed the impact of emicizumab prophylaxis on the HRQoL of children and their caregivers participating in HAVEN 2.MethodsChildren aged 8‐11 years self‐reported HRQoL using the Haemophilia‐Specific Quality of Life Assessment Instrument for Children and Adolescents Short Form (Haemo‐QoL SF II). Caregivers of children aged 0‐11 years completed the Adapted Inhibitor‐Specific Quality of Life Assessment with Aspects of Caregiver Burden. All scores were transformed to a 0‐100 scale, where lower scores reflect a better HRQoL. The number of missed days from school/day care and hospitalizations was also recorded.ResultsIn HAVEN 2 (n = 88), the median age was 6.5 years (range: 1‐15 years); 85 participants were aged < 12 years and included in this analysis, and 34 participants were aged 8‐11 years, thereby eligible to complete the Haemo‐QoL SF II questionnaire. The mean (standard deviation, n) baseline Haemo‐QoL SF II ‘Total’ score was 30.2 (14.9, 30), indicating moderate impairment; with emicizumab, mean score decreased by −9.62 (7.73, 17) points to 23.0 (13.93, 20) by Week 49. The most improved domains were ‘Sports & School’ and ‘Physical Health’. Caregivers reported similar improvements.ConclusionProphylactic emicizumab is accompanied by substantial and sustained improvements in HRQoL of paediatric PwHA with FVIII inhibitors and their caregivers.
Related Links	https://europepmc.org/backend/ptpmcrender.fcgi?accid=PMC7821108&blobtype=pdf
Page Count	10
ISSN	13518216
Journal	Haemophilia
Volume Number	26
DOI	10.1111/hae.14183
PubMed Central reference number	PMC7821108
Issue Number	6
PubMed reference number	33084175
e-ISSN	13652516
Language	English
Publisher	John Wiley and Sons Inc.
Publisher Date	2020-10-21
Publisher Place	Hoboken
Access Restriction	Open
Rights License	This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. © 2020 The Authors. Haemophilia published by John Wiley & Sons Ltd
Subject Keyword	caregiver burden children emicizumab haemophilia health‐related quality of life inhibitors
Content Type	Text
Resource Type	Article
Subject	Hematology Genetics (clinical)