Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2020
Safety and efficacy of turoctocog alfa in the prevention and treatment of bleeds in previously untreated paediatric patients with severe haemophilia A: Results from the guardian 4 multinational clinical trial.
Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 2 of them syntheses that pooled it.
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.
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Who cites it
15 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously untreated or minimally treated children with hemophilia A or B.The Cochrane database of systematic reviews · 2025Pooled it
- Efficacy and Safety of Recombinant Factor VIII in Previously Untreated and Previously Treated Children with Hemophilia A: A Systematic Review.Advances in therapy · 2025Pooled it
- Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A.Research and practice in thrombosis and haemostasis · 2026Trial
- 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
- A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial.Blood advances · 2024Trial
- Trial
- Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study.Thrombosis and haemostasis · 2021Trial
- Safety and efficacy of turoctocog alfa in the prevention and treatment of bleeds in previously untreated paediatric patients with severe haemophilia A: Results from the guardian 4 multinational clinical trial.Haemophilia : the official journal of the World Federation of Hemophilia · 2020Trial
- Shaping hemophilia care: lessons and legacy of the SIPPET trial after 10 years.Research and practice in thrombosis and haemostasis · 2026Review
- Inhibitor development and clinical characteristics in children with severe hemophilia A in the ATHN 8 US cohort study.Blood vessels, thrombosis & hemostasis · 2025Article
- Simoctocog alfa (NuwiqTherapeutic advances in hematology · 2024Review
- Immunogenicity of Current and New Therapies for Hemophilia A.Pharmaceuticals (Basel, Switzerland) · 2022Review
- Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
- Regional Brain Tissue Displacement and Strain is Elevated in Subjects with Chiari Malformation Type I Compared to Healthy Controls: A Study Using DENSE MRI.Annals of biomedical engineering · 2021Article
- Safety and Efficacy of Turoctocog Alfa in the Prevention and Treatment of Bleeding Episodes in Previously Treated Patients from China with Severe Hemophilia A: Results from the Guardian 7 Trial.Therapeutics and clinical risk management · 2020Article
Corrections and comments
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Authors and funding
10 authors.
Funding
Abstract
introductionTuroctocog alfa is a recombinant, B domain-truncated factor VIII (FVIII) approved for patients with haemophilia A.
aimTo evaluate the safety and efficacy of turoctocog alfa in previously untreated patients (PUPs) with severe haemophilia A.
methodsGuardian 4 was a multicentre, multinational, non-randomized, open-label phase 3 trial comprising a main and extension phase. The former concluded once ≥ 50 patients had received treatment for ≥ 50 exposure days (EDs) or developed inhibitors. Patients received turoctocog alfa intravenously for prevention and treatment of bleeds. The primary endpoint was the incidence rate of FVIII inhibitors (≥0.6 Bethesda Units) reported during the first 50 EDs.
resultsOf the 58 patients who completed the main phase, 25 (43.1%) patients developed inhibitors (detected within 6-24 [mean: 14.2] EDs from treatment start). High-risk mutations were identified in 60% of patients who developed inhibitors in the main phase and were a significant predictor of inhibitor development (P = .003). Of the 21 patients who started immune tolerance induction therapy, 85.7% completed treatment with a negative inhibitor test (note that data on the last 3 patients completing ITI are based on information collated from sites prior to the final database lock). Haemostatic response (including missing values as failure) was rated as 'excellent' or 'good' for 86.1% of bleeds occurring during prophylaxis. The estimated mean annualized bleeding rate for patients on prophylaxis was 4.26 bleeds/patient/year (95% CI: 3.34 - 5.44).
conclusionsTuroctocog alfa was effective at preventing and stopping bleeds and was well tolerated. Inhibitor development was within the expected range for this PUP population.
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