Trial reportBlood2021
Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies.
Trial report in Blood, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to 5 registered trials, which are not on this map. Cited by 142 papers, 3 of them syntheses that pooled it.
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.
A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients With Inhibitors
A Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Administration of Emicizumab in Hemophilia A Pediatric Patients With Inhibitors
A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients Without Inhibitors
A Multicenter, Open-Label, Phase III Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks (Q4W) in Patients With Hemophilia A
Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study
Who cites it
142 citing papers in PubMed, 3 syntheses or guidelines pooled it, 275 citations in OpenAlex.
- Cost-effectiveness of emicizumab for the treatment of hemophilia A: a systematic review.Frontiers in public health · 2025Pooled it
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- Pharmacokinetics and Associated Efficacy of Emicizumab in Humans: A Systematic Review.Clinical pharmacokinetics · 2021Pooled it
- Coagulation potential of concomitant factor VIII administration in people with hemophilia A receiving emicizumab prophylaxis (CAGUYAMA study): a multicenter, open-label, nonrandomized clinical trial.Research and practice in thrombosis and haemostasis · 2026Trial
- Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.Research and practice in thrombosis and haemostasis · 2026Trial
- Trial
- Marstacimab prophylaxis in hemophilia A/B without inhibitors: results from the phase 3 BASIS trial.Blood · 2025Trial
- Trial
- Final Analysis Results from the AGEHA Study: Emicizumab Prophylaxis for Acquired Hemophilia A with or without Immunosuppressive Therapy.Thrombosis and haemostasis · 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
- Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A.Blood advances · 2024Trial
- Trial
- Trial
- Emicizumab dose up-titration in case of suboptimal bleeding control in people with haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2023Trial
- Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Trial
- Trial
- A novel humanized FIX/FX hemophilia A mouse model for preclinical evaluation of FVIIIa-mimetic bispecific antibodies.Blood advances · 2026Article
- A crowded podium in hemophilia A: when a low annualized bleeding rate is no longer enough to tell the contenders apart.Research and practice in thrombosis and haemostasis · 2026Article
- Personalized Treatment of Hemophilia: Matching Therapies to Patient Needs in a Rapidly Evolving Landscape.Drugs · 2026Review
- Long-term outcomes with emicizumab prophylaxis for haemophilia A in China: A multicentre, large-cohort retrospective study.British journal of haematology · 2026Observational
82 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
- Erratum issued
Authors and funding
20 authors at 12 institutions in 9 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Prophylaxis with emicizumab, a subcutaneously administered bispecific humanized monoclonal antibody, promotes effective hemostasis in persons with hemophilia A (PwHAs). The primary efficacy, safety, and pharmacokinetics of emicizumab were reported previously, but long-term data were limited. Here, data from 401 pediatric and adult PwHAs with/without factor VIII (FVIII) inhibitors who were enrolled in the phase 3 HAVEN 1, HAVEN 2, HAVEN 3, and HAVEN 4 studies (NCT02622321, NCT02795767, NCT02847637, NCT03020160) have been pooled to establish a long-term efficacy, safety, and pharmacokinetics profile. Across a median efficacy period of 120.4 weeks (interquartile range, 89.0-164.4) (data cutoff 15 May 2020), the model-based treated annualized bleed rate (ABR) was 1.4 (95% confidence interval [CI], 1.1-1.7). ABRs declined and then stabilized at <1 in an analysis of 24-week treatment intervals; at weeks 121 to 144 (n = 170), the mean treated ABR was 0.7 (95% CI, 0-5.0). During weeks 121 to 144, 82.4% of participants had 0 treated bleeds, 97.6% had ≤3 treated bleeds, and 94.1% reported no treated target joint bleeds. Bleeding into target joints decreased substantially. Emicizumab was well tolerated, and no participant discontinued because of adverse events beyond the 5 previously described. This data cutoff includes the previously reported 3 thrombotic microangiopathies (one in the PwHA with fatal rectal hemorrhage) and 2 thromboembolic events, all associated with activated prothrombin complex concentrate use, as well as a myocardial infarction and a venous device occlusion. With 970.3 patient-years of exposure, emicizumab prophylaxis maintained low bleed rates in PwHAs of all ages with/without FVIII inhibitors and remains well tolerated, with no new safety concerns identified.
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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.