ArticleHaemophilia : the official journal of the World Federation of Hemophilia
Post‑Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients With Congenital Haemophilia A With Inhibitors.
Article in Haemophilia : the official journal of the World Federation of Hemophilia. 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.
- Post‑Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients With Congenital Haemophilia A With Inhibitors.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
Abstract
introductionThe bispecific monoclonal antibody emicizumab was approved for prophylactic treatment of congenital haemophilia A (HA) in Japan in 2018.
aimTo monitor long-term safety and effectiveness of emicizumab, including appropriate concomitant use of bypassing agents (BPAs), in Japanese patients with congenital HA with inhibitors who initiated emicizumab within 1 year of availability.
methodsThis all-case post-marketing surveillance (PMS) study was conducted between May 2018 and January 2023, in patients of all ages and HA severities. Patients were registered retrospectively after emicizumab initiation or prospectively at the time of initiation. Adverse events (AEs) and adverse drug reactions (ADRs), including thromboembolic events (TEs) and thrombotic microangiopathy (TMA), and their association with BPA use, were examined. FVIII inhibitor levels and annualised bleeding rates (ABRs) were evaluated.
resultsIn total, 134 patients were included in the analysis. Mean (standard deviation) duration of emicizumab treatment was 145.5 (34.8) weeks. Overall, 112 AEs occurred in 47 patients (35.1%) and 22 ADRs occurred in 12 patients (9.0%); 8 ADRs (36.4%) were serious. No TEs/TMAs associated with concomitant use of BPAs occurred. FVIII inhibitor levels remained stable or decreased for 34 patients (55.7%). Sixty-eight patients (50.7%) received BPAs; none received activated prothrombin complex concentrate. Mean (95% confidence interval) model-based all bleed ABR and treated bleed ABR were 1.4 (0.9-2.3) and 1.3 (0.8-2.1), respectively.
conclusionThis study comprises 3 years' PMS for emicizumab. No TEs/TMA associated with BPAs occurred. Results further support the safety and effectiveness of emicizumab in Japanese patients with congenital HA with inhibitors.
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.