ReviewHaematologica2020
Hemophilia therapy: the future has begun.
Review in Haematologica, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07736976 (Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers), which is not on this map. Cited by 112 papers.
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.
Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers: A Cross-Sectional Observational Study
Who cites it
112 citing papers in PubMed.
- Trial
- Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.Blood advances · 2025Trial
- Trial
- Efficacy and safety of subcutaneous prophylaxis with dalcinonacog alfa in adults with haemophilia B.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Trial
- Trial
- Emicizumab and shared medical decision in patients living with hemophilia A.Hematology, transfusion and cell therapy · 2026Article
- Psychometric properties and outcomes of the PROMIS Sexual Function and Satisfaction measures in individuals with congenital bleeding disorders.Research and practice in thrombosis and haemostasis · 2026Article
- Focus on Women with Hemophilia and Carriers: How to Address All the Existing Gaps in Diagnosis and Care.Journal of clinical medicine · 2026Review
- Managing massive gastrointestinal and abdominal haemorrhage in inherited bleeding disorders: experience from a pediatric cohort.International journal of hematology · 2026Article
- Association of Patient-Reported Outcomes with Hemophilia A Inhibitor Status and Treatment Product Type.Journal of clinical medicine · 2026Article
- The Epigenetic Landscape of Hemophilia.Current molecular medicine · 2026Review
- Prophylaxis for von Willebrand disease: Is it time for parity with established practice in hemophilia A?Therapeutic advances in hematology · 2026Review
- Perceived barriers and facilitators for physical activity in youth and young adults living with hemophilia. A focus group study.Frontiers in sports and active living · 2026Article
- In memory of Ryan White, the boy with haemophilia who showed the world the other side of AIDS Lest We Forget….Journal of preventive medicine and hygiene · 2025Article
- Integrating CRISPR/Cas technology with clinical trials: Principles, progress and challenges.Asian journal of pharmaceutical sciences · 2025Review
- Haemophilia B: an illustrative review of current challenges and opportunities.Research and practice in thrombosis and haemostasis · 2025Article
- A qualitative study on the impact of hemophilia on sexual functioning and well-being in Dutch men.Journal of patient-reported outcomes · 2025Article
- Transamniotic Delivery of Coagulation Factor VIII mRNA: A Step Toward a Potential Novel Strategy for the Perinatal Management of Hemophilia A.FASEB bioAdvances · 2025Article
- Burden of Haemophilia A in South Korea: A Serial Cross-Sectional Study From 2008 to 2021.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Review
52 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The success story of hemophilia care first began in the 1970s, when the availability of plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided efficacious treatment of bleeding in patients with hemophilia A and B. This positive scenario was consolidated in terms of greater safety and availability in the 1990s, when the first recombinant coagulation factors were produced. This meant that, instead of only treating episodic bleeding events, prophylaxis regimens could be implemented as a preventive measure. Following the demonstration of its superiority in the frame of two randomized clinical trials, prophylaxis became evidence-based standard of care. In high-income countries, these achievements have led to a patients' life expectancy being extended to close to that of the general male population. Alongside this, the last decade has witnessed further spectacular therapeutic progress, such as the availability of coagulation factors with a longer plasma half-life that allow for wider intervals between treatment. Moreover, new therapeutic products based on new mechanisms other than the replacement of the deficient factor, have become available (emicizumab) or are at an advanced stage of development. This review celebrates the success story of hemophilia care, while also discussing current limitations, issues and as yet unmet needs. The prospects of cure by means of gene therapy are also outlined.
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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.