ArticleHaemophilia : the official journal of the World Federation of Hemophilia2020
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.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
12 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- Denecimig (Mim8) prophylaxis in children with hemophilia A with or without factor VIII inhibitors.Research and practice in thrombosis and haemostasis · 2026Trial
- Health-Related Quality of Life, Physical Activity and Joint Health in People With Severe Haemophilia A Receiving Emicizumab: Results From the Phase IV HemiNorth 2 Study.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Pain-Related Quality of Life Outcomes in People With Haemophilia A Receiving Emicizumab: A Post Hoc Analysis of the HAVEN 1, 3 and 4 and STASEY Studies.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Paediatric Subanalysis of TSUBASA, Assessing Physical Activity, Bleeding, Quality of Life and Safety in People with Haemophilia A Receiving Emicizumab.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study.Research and practice in thrombosis and haemostasis · 2025Article
- The Current and Potential Role of Pediatric Clinical Pharmacologists in Italy: A Review with Evidence Synthesis and Literature Analysis.Current medicinal chemistry · 2025Review
- Development and validation of the Child Hemophilia Treatment Experience Measure: A new observer-reported outcome measure.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Emicizumab prophylaxis for people with hemophilia A: Waste estimation and the Brazilian perspective.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2023Article
- Between Scylla and Charybdis: thrombosis in children with hemophilia.Frontiers in pediatrics · 2023Review
- Quality of life in children and adolescents with hemophilia A: A systematic review and meta-analysis.Research and practice in thrombosis and haemostasis · 2023Article
- Emicizumab state-of-the-art update.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
Corrections and comments
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Authors and funding
10 authors at 8 institutions in 6 countries.
Funding
Abstract
introductionPersons 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.
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