Observational studyHaemophilia : the official journal of the World Federation of Hemophilia
Treatment of Haemophilia A Without Inhibitors: Real-World Treatment Patterns and Clinical Outcomes in the US.
Observational study in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Plasma emicizumab concentrations and bleeding rates in children and adults with severe hemophilia A.Research and practice in thrombosis and haemostasis · 2026Observational
Corrections and comments
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Authors and funding
15 authors.
Funding
Abstract
introductionDespite available prophylactic therapies for haemophilia A, breakthrough bleeding and consequent pain and joint damage still occur.
aimTo provide insights into the unmet treatment needs of people with moderate-to-severe haemophilia A in the US.
methodsFor this descriptive, observational cohort study, eligible participants diagnosed with moderate-to-severe haemophilia A were enrolled in the PicnicHealth database on or before 3 October 2022. Three patient groups were established to identify: (1) treatment patterns; (2) clinical outcomes by therapy class (standard half-life [SHL] factor VIII [FVIII]; extended half-life FVIII [EHL]; emicizumab); (3) health-related quality of life (HRQoL) using the Patient-Reported Outcomes Measuring Instruments System (PROMIS)-29 survey and a supplemental pain survey. Patients switching treatment classes were included in multiple treatment groups.
resultsOf 211 participants in the 'treatment patterns population', 123 (58.3%) used SHL FVIII prophylaxis, 81 (38.4%) EHL FVIII prophylaxis, and 83 (39.3%) emicizumab prophylaxis. In the 'clinical outcomes population', 63 participants (70.8%) receiving SHL, 43 (61.4%) EHL and 36 (62.1%) emicizumab had ≥1 bleed at Year 1. Pain-related events within the past year occurred in 18 (12.8%), 16 (18.2%) and 10 (12.2%) participants, respectively. The most frequent patient-reported symptoms were 'sleep disturbance' (84.5%), 'inability to participate in social roles and activities' (69.7%) and 'fatigue' (66.9%). The most impacted PROMIS scores were anxiety and pain interference.
conclusionRegardless of the prophylaxis product class used, most participants experienced ≥1 bleeding event per year and reduced QoL. Further treatment optimisation may be needed to prevent bleeding episodes and improve patient QoL.
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