ArticleOrphanet journal of rare diseases2025
Clinical, humanistic, and economic burden of haemophilia A in China: findings from a real-world survey.
Article in Orphanet journal of rare diseases, 2025. 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
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
- The therapeutic landscape of inherited bleeding disorders in China.Research and practice in thrombosis and haemostasis · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
objectivesWe aimed to describe current treatments for haemophilia A in China, focusing on their associated clinical, humanistic, and economic burdens in a real-world context.
methodsThis was a retrospective cross-sectional study. We investigated the demographics, disease severity, treatment strategies, and clinical outcomes of patients diagnosed with haemophilia A. We also investigated the cost of the treatment of patients with haemophilia A. In the real world, we estimated annual direct medical costs, direct non-medical costs and indirect costs. In addition, we employed the EQ-5D and SF-6D to measure the humanistic burden of patients.
resultsA total of 60 patients were included in the study, comprising 22 children (< 18 years) and 38 adults (≥ 18 years). The mean age of the children and adults was 9.27 and 33.05 years, respectively. Treatment strategies for adults were primarily on-demand. Patients receiving prophylactic treatment experienced fewer bleeds per year compared to those receiving on-demand treatment (mean ABR: adults 42.91 vs. 20.38; children 20.20 vs. 10.10). The mean EQ-5D utility value reported by children and adults were 0.76 (SD 0.24) and 0.51 (SD 0.34), respectively. For adult patients, the SF-6D utility value was 0.38. The mean total annual direct medical costs associated with haemophilia were ¥429,143 (US$58,666) for children and ¥340,238 (US$46,512) for adults, with medication being the primary cost driver.
conclusionsThese data document the enormous burden of haemophilia A that persists in the real world of China. While we emphasize incremental direct healthcare expenditures, we must also consider the long-term clinical and socio-economic benefits of prophylactic treatment. CLINICAL TRIAL NUMBER: Not applicable.
Indexed as
Identifiers
What OpenQuestion holds
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.