Evaluation of bleeding symptoms and laboratory parameters related to bleeding in sisters of patients with hemophilia A and B

Akdeniz, Nadiye; Karakuş, Abdullah; Yıldız, İsmail; Ayyildiz, Mehmet Orhan

doi:10.1016/j.transci.2020.103044

Cited by 2 publications

(1 citation statement)

References 17 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The spontaneous- and injury-related bleeding are important evaluations for hemophilia, especially in prophylactic treatment use. 27 Without an appropriate prophylactic treatment, patients have a tendency to experience monthly spontaneous bleeding episodes (including spontaneous joint bleeds) and prolonged and excessive bleeding after minor trauma. 28 , 29 Our results showed that the ABRs of rurioctocog alfa pegol were similar for spontaneous- and injury-related bleeding.…”

Section: Discussionmentioning

confidence: 99%

Efficacy, safety, and immunogenicity of rurioctocog alfa pegol for prophylactic treatment in previously treated patients with severe hemophilia A: a systematic review and meta-analysis of clinical trials

et al. 2022

View full text Add to dashboard Cite

Background: Patients with severe hemophilia often present with painful joint and soft tissue bleeding which may restrict them from their daily activities. The current standard of care still relies on a regular prophylactic factor VIII (FVIII), which has a high daily treatment burden. Recently, rurioctocog alfa pegol, a third-generation recombinant FVIII with a modification in its polyethylene glycol (PEG) component, has been developed. Several trials have studied this synthetic drug as bleeding prophylaxis in severe hemophilia A. This study aims to evaluate the efficacy, safety, and immunogenicity of rurioctocog alfa pegol for previously treated patients with severe hemophilia A. Methods: This study was conducted in conformity with the PRISMA guidelines. Data were retrieved from PubMed, Scopus, Cochrane Library, Wiley Online Library, and CINAHL (via EBSCOhost). Study qualities were assessed using the Methodological Index for Non-Randomized Studies (MINORS) and Modified Jadad scales. Results: Four studies involving 517 previously treated severe hemophilia A patients were included in this study. The pooled mean of total annualized bleeding rate (ABR) and hemostatic efficacy was 2.59 (95% CI = 2.04–3.14) and 92% (95% CI = 85%–97%), respectively. Only 30 (2.3%) non-serious and one (1.4%) serious adverse events were considered related to rurioctocog alfa pegol treatment. At the end of the studies, no development of FVIII inhibitory antibodies was observed. None of the developed binding antibodies to FVIII, PEG-FVIII, or PEG was correlated to the treatment efficacy and safety. Conclusions: Despite the limited availability of direct comparison studies, our analyses indicate that rurioctocog alfa pegol could serve as a safe and effective alternative for bleeding prophylaxis in previously treated hemophilia A patients. Moreover, it appears to have low immunogenicity, which further increases the safety profile of the drug in such clinical conditions.

show abstract

Section: Discussionmentioning

confidence: 99%

Efficacy, safety, and immunogenicity of rurioctocog alfa pegol for prophylactic treatment in previously treated patients with severe hemophilia A: a systematic review and meta-analysis of clinical trials

et al. 2022

View full text Add to dashboard Cite

show abstract

Proactive systematic hemophilia carrier screening: a step toward gender equity in hemophilia care

Krumb,

Lambert,

Van Damme

et al. 2024

Blood Advances

View full text Add to dashboard Cite

Despite numerous efforts to raise awareness, many hemophilia carriers and female persons with hemophilia (PWHs) remain undiagnosed. Between May 2021 and April 2023, we identified potential and obligate carriers of hemophilia A (HA) and B (HB) by updating pedigrees of all PWHs followed at the Cliniques universitaires Saint-Luc, Brussels. Retrospective data on previously screened females were collected, including bleeding history, coagulation factor levels, and testing for the proband's pathogenic variant. Additionally, a proactive approach involved sending 125 invitation letters to unscreened or incompletely screened individuals, through related PWHs. In pedigrees of 287 male PWHs (226 HA, 61 HB) and 7 female index patients from 236 families (184 HA, 52 HB), a total of 900 female individuals were identified. Of those, 454 were obligate and/or genetically proven carriers, and 118 were noncarriers. Genetic testing was conducted in 133 obligate, 237 potential, and 4 sporadic carriers, with 190 obligate and 328 potential carriers remaining untested. Among carriers with known factor levels (261/454), 42 (23.0%) HA and 23 (29.5%) HB carriers, had a factor level below 40 IU/dL. Carriers with a factor deficiency were screened on average 6 years earlier than other females (p=0.034). This study represents the first systematic effort to identify potential carriers among families of all PWHs within a single center, emphasizing the challenges in comprehensive screening for female individuals genetically linked to one or more PWHs. Such initiatives are vital for achieving equitable access to hemophilia care for all potentially affected individuals, irrespective of gender. Clinical.Trials.gov identifier: NCT05217992

show abstract

Evaluation of bleeding symptoms and laboratory parameters related to bleeding in sisters of patients with hemophilia A and B

Cited by 2 publications

References 17 publications

Efficacy, safety, and immunogenicity of rurioctocog alfa pegol for prophylactic treatment in previously treated patients with severe hemophilia A: a systematic review and meta-analysis of clinical trials

Efficacy, safety, and immunogenicity of rurioctocog alfa pegol for prophylactic treatment in previously treated patients with severe hemophilia A: a systematic review and meta-analysis of clinical trials

Proactive systematic hemophilia carrier screening: a step toward gender equity in hemophilia care

Contact Info

Product

Resources

About