ArticleHaemophilia : the official journal of the World Federation of Hemophilia2019
Health-related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non-interventional study (NIS).
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02476942 (A Multicenter, Non-Interventional Study Evaluating Bleeding Incidence, Health-Related Quality of Life, and Safety in Patients With Hemophilia A Under Standard-of-Care Treatment), which is not on this map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
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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.
A Multicenter, Non-Interventional Study Evaluating Bleeding Incidence, Health-Related Quality of Life, and Safety in Patients With Hemophilia A Under Standard-of-Care Treatment
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 46 citations in OpenAlex.
- Emicizumab prophylaxis in people with hemophilia A and inhibitors: a systematic review and meta-analysis.Sao Paulo medical journal = Revista paulista de medicina · 2024Pooled it
- Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study.Thrombosis and haemostasis · 2021Trial
- Post Hoc Analyses of A-SURE and PREVENT Confirm the Effectiveness of rFVIIIFc Prophylaxis Across All Ages, BMIs, Severities, and Inhibitor Histories.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.Research and practice in thrombosis and haemostasis · 2024Article
- Intraindividual bleeding outcomes in patients with hemophilia A on emicizumab prophylaxis in Australia.Blood vessels, thrombosis & hemostasis · 2024Article
- The Evolution of Hemophilia Therapeutics: An Illustrated Review.Research and practice in thrombosis and haemostasis · 2024Article
- Health-related quality of life and its associated factors among hemophilia patients: experience from Ethiopian Hemophilia Treatment Centre.Journal of pharmaceutical health care and sciences · 2024Article
- Treatment Adherence and Health-Related Quality of Life in Patients with Hemophilia in Hong Kong.International journal of environmental research and public health · 2022Article
- Current and Emerging Approaches for Pain Management in Hemophilic Arthropathy.Pain and therapy · 2022Review
- The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Disease burden and quality of life of in children with sickle cell disease in Italy: time to be considered a priority.Italian journal of pediatrics · 2021Review
- Exposure-Bleeding Count Modeling of Emicizumab for the Prophylaxis of Bleeding in Persons with Hemophilia A with/Without Inhibitors Against Factor VIII.Clinical pharmacokinetics · 2021Article
- Article
- A Delphi Consensus Approach for Difficult-to-Treat Patients with Severe Hemophilia A without Inhibitors.Journal of blood medicine · 2021Article
- Patients' Perception of the Impact of Innovation on Hemophilia Care Management Organization: A Qualitative Study Protocol (INNOVHEMO Study).Patient preference and adherence · 2021Article
- Article
- Clinical Evidence and Safety Profile of Emicizumab for the Management of Children with Hemophilia A.Drug design, development and therapy · 2020Review
- Health-related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non-interventional study (NIS).Haemophilia : the official journal of the World Federation of Hemophilia · 2019Article
Corrections and comments
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Authors and funding
15 authors at 10 institutions in 8 countries.
Funding
Abstract
introductionReal-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice.
aimTo report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS.
methodsThis cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS).
resultsOne hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups).
conclusionsThese RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.