Trial reportBlood2016
Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A.
Trial report in Blood, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01486927 (A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII), which is not on this map. Cited by 35 papers, 1 of them a synthesis 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 Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to Recombinant Human Antihaemophilic Factor VIII (rFVIII; INN: Octocog Alfa) in Subjects With Hemophilia A, and a Repeat PK, Safety and Efficacy Study
Who cites it
35 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A.Advances in therapy · 2021Pooled it
- Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Efficacy, safety and bioequivalence of the human-derived B-domain-deleted recombinant factor VIII TQG202 for prophylaxis in severe haemophilia A patients.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Trial
- New therapies in hemophilia: extend the half-life, mimic, or rebalance?Hematology. American Society of Hematology. Education Program · 2025Review
- Efficacy and Safety of rVIII-SingleChain in Surgical Prophylaxis.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Switching From Standard to Extended Half-Life Coagulation Factor Replacement in Haemophilia: Clinical Outcomes and Costs of Care in Finland.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Observational
- Relationship between factor VIII levels and bleeding for rFVIII-SingleChain in severe hemophilia A: A repeated time-to-event analysis.CPT: pharmacometrics & systems pharmacology · 2023Article
- A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis.Thrombosis and haemostasis · 2023Article
- Immunogenicity of Current and New Therapies for Hemophilia A.Pharmaceuticals (Basel, Switzerland) · 2022Review
- Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies.Research and practice in thrombosis and haemostasis · 2022Article
- Hemophilia A: Strategies for Improving Long-Term Holistic Management, Adherence, and Quality of Life.Journal of the advanced practitioner in oncology · 2022Review
- Italian experience with rVIII-single chain: a survey of patients with haemophilia A and their physicians.Journal of thrombosis and thrombolysis · 2022Article
- Recombinant single-chain factor VIII in severe hemophilia: Long-term safety and efficacy in previously treated patients in the AFFINITY extension study.Research and practice in thrombosis and haemostasis · 2022Article
- Application of Multicriteria Decision Analysis to Determine the Value of Prophylaxis Relative to On-Demand Treatment in Hemophilia A and Emicizumab versus Replacement Therapy in the Greek Healthcare Setting.Clinical drug investigation · 2022Review
- How do we optimally utilize factor concentrates in persons with hemophilia?Hematology. American Society of Hematology. Education Program · 2021Review
- Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device.Molecular therapy. Methods & clinical development · 2021Article
- Advances in the management of haemophilia: emerging treatments and their mechanisms.Journal of biomedical science · 2021Review
- Development of a novel fully functional coagulation factor VIII with reduced immunogenicity utilizing an in silico prediction and deimmunization approach.Journal of thrombosis and haemostasis : JTH · 2021Article
- Efficacy of NuwiqClinical medicine insights. Blood disorders · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
27 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recombinant VIII (rVIII)-SingleChain is a novel B-domain-truncated recombinant factor VIII (rFVIII), comprised of covalently bonded factor VIII (FVIII) heavy and light chains. It was designed to have a higher binding affinity for von Willebrand factor (VWF). This phase 1/3 study investigated the efficacy and safety of rVIII-SingleChain in the treatment of bleeding episodes, routine prophylaxis, and surgical prophylaxis. Participants were ≥12 years of age, with severe hemophilia A (endogenous FVIII <1%). The participants were allocated by the investigator to receive rVIII-SingleChain in either an on-demand or prophylaxis regimen. Of the 175 patients meeting study eligibility criteria, 173 were treated with rVIII-SingleChain, prophylactically (N = 146) or on-demand (N = 27). The total cumulative exposure was 14 306 exposure days (EDs), with 120 participants reaching ≥50 EDs and 52 participants having ≥100 EDs. Hemostatic efficacy was rated by the investigator as excellent or good in 93.8% of the 835 bleeds treated and assessed. Across all prophylaxis regimens, the median annualized spontaneous bleeding rate was 0.00 (Q1, Q3: 0.0, 2.4) and the median overall annualized bleeding rate (ABR) was 1.14 (Q1, Q3: 0.0, 4.2). Surgical hemostasis was rated as excellent/good in 100% of major surgeries by the investigator. No participant developed FVIII inhibitors. In conclusion, rVIII-SingleChain is a novel rFVIII molecule showing excellent hemostatic efficacy in surgery and in the control of bleeding events, low ABR in patients on prophylaxis, and a favorable safety profile in this large clinical study. This trial was registered at www.clinicaltrials.gov as #NCT01486927.
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