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Pharmaceuticals
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Recent Developments of Coagulation Factor Products in Hemophilia Treatment
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Recent Developments of Coagulation Factor Products in Hemophilia Treatment

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".

Deadline for manuscript submissions: closed (20 August 2023) | Viewed by 2238

Special Issue Editor

Dr. Samantha Pasca

E-Mail Website
Guest Editor
Department of Biomedical Sciences (DSB), University Hospital of Padua, 35138 Padua, Italy
Interests: hemophilia; congenital rare bleeding diseases; coagulation; acquired hemorrhagic disorders; venous thromboembolism

Special Issue Information

Dear Colleagues,

In recent years, hemophilia therapy has made significant progress. Several new drugs have been added to the list of available treatments, significantly improving patients’ quality of life. The extended half-life factor concentrates have enabled a reduced number of infusions, maintaining (and indeed, often increasing) the efficacy of treatments. These new drugs have also led to a reduction in bleeding, bringing us one step closer to the coveted “zero bleeds”, which is the ultimate goal of clinicians and patients. Another advantage of these concentrates is their versatility; thanks to the use of pharmacokinetics and an accurate anamnesis that considers the needs and wishes of the patients, optimal personalized therapy can now be defined.

This Special Issue aims to highlight every aspect of these new coagulation factor concentrates, their efficacy, their safety, and their impact on patients' lives. Original articles, brief reports, communications, research articles, and reviews are welcome.

Dr. Samantha Pasca
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceuticals is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • hemophilia
  • clotting factor concentrates
  • extended half-life concentrates
  • pharmacokinetics
  • new hemophilia treatments
  • prophylaxis

Published Papers (2 papers)

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Research

10 pages, 274 KiB  
Article
Changes in Quality of Life, Adherence, and Kinesiophobia in Patients with Hemophilia Treated with Extended Half-Life Treatment: Final Results of the LongHest Project
by Roberto Ucero-Lozano, Raúl Pérez-Llanes, Rubén Cuesta-Barriuso and Elena Donoso-Úbeda
Pharmaceuticals 2024, 17(7), 835; https://doi.org/10.3390/ph17070835 - 25 Jun 2024
Viewed by 657
Abstract
(1) Background: Hemophilia is a bleeding disorder characterized by hemarthrosis. Prophylaxis is the gold standard for bleeding prevention. Extended half-life (EHL) recombinant FVIII replacement products have shown to be associated with low bleeding rates. The aim was to evaluate the efficacy of EHL [...] Read more.
(1) Background: Hemophilia is a bleeding disorder characterized by hemarthrosis. Prophylaxis is the gold standard for bleeding prevention. Extended half-life (EHL) recombinant FVIII replacement products have shown to be associated with low bleeding rates. The aim was to evaluate the efficacy of EHL prophylaxis in improving perceived quality of life, adherence to treatment, and kinesiophobia in patients with hemophilia. (2) Methods: This was a prospective cohort study. Forty-six patients from different regions, who had started EHL FVIII concentrate prophylactic treatment, were evaluated at baseline and at 12-month follow-up. The study variables were as follows: perceived quality of life (36-Item Short Form Health Survey), adherence to treatment (Validated Hemophilia Regimen Treatment Adherence Scale—Prophylaxis), and kinesiophobia (Tampa Scale of Kinesiophobia). (3) Results: There were statistically significant differences in the domains Role-Physical (p < 0.001), Bodily Pain (p < 0.001), Role-Emotional (p < 0.001), Vitality (p = 0.04), and Social Functioning (p = 0.01) and the total scores, Physical Health (p < 0.001) and Mental Health (p < 0.001) on perceived quality of life. There were significant differences in the domains Skipping (p < 0.01), Communicating (p < 0.001), and the total score (p = 0.01) in terms of adherence. There were also significant differences in kinesiophobia (p = 0.02) after the study period. (4) Conclusions: EHL prophylaxis can improve the perceived quality of life of people with hemophilia. This prophylactic regimen, which requires fewer infusions, may improve adherence to treatment in adult patients with hemophilia over a 12-month period. The administration of extended half-life factor VIII concentrates can reduce kinesiophobia in adult patients with hemophilic arthropathy. Full article
(This article belongs to the Special Issue Recent Developments of Coagulation Factor Products in Hemophilia Treatment)
10 pages, 666 KiB  
Article
Damoctocog Alfa Pegol for Hemophilia A Prophylaxis: An Italian Multicenter Survey
by Ezio Zanon
Pharmaceuticals 2023, 16(9), 1195; https://doi.org/10.3390/ph16091195 - 22 Aug 2023
Cited by 1 | Viewed by 1220
Abstract
Hemophilia A is characterized by a deficiency of clotting factor VIII (FVIII) requiring lifelong prophylactic treatment, typically with recombinant FVIII. In recent years, drugs with extended half-lives have become available, including damoctocog alfa pegol (Bayer S.p.A.). The clinical efficacy and safety of damoctocog [...] Read more.
Hemophilia A is characterized by a deficiency of clotting factor VIII (FVIII) requiring lifelong prophylactic treatment, typically with recombinant FVIII. In recent years, drugs with extended half-lives have become available, including damoctocog alfa pegol (Bayer S.p.A.). The clinical efficacy and safety of damoctocog alfa pegol were demonstrated in the PROTECT VIII phase II/III development program. To assess the physicians’ experience and to collect data on prophylactic treatment with damoctocog alfa pegol, a monitoring survey was carried out among 15 Italian hemophilia centers. A total of 149 patients on treatment with damoctocog alfa pegol for at least 6 months were considered. Zero bleeds were reported in 75% of patients treated with damoctocog alfa pegol in the last 6 months; zero hemarthroses were reported in 82% of the same patients. Overall, 86% of patients with damoctocog alfa pegol reduced their monthly infusions. The estimated average reduction in IU/kg during prophylaxis with damoctocog alfa pegol, both monthly and annually, was around 17.5%. All involved clinicians were satisfied with damoctocog alfa pegol. Survey results suggest that damoctocog alfa pegol reduced the number of weekly infusions, annual bleeding, and joint bleeding rate in the majority of patients, improving joint health and patients’ quality of life. Full article
(This article belongs to the Special Issue Recent Developments of Coagulation Factor Products in Hemophilia Treatment)
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