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Hemophilia: Current Trends and Future Directions
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Hemophilia: Current Trends and Future Directions

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".

Deadline for manuscript submissions: 25 March 2026 | Viewed by 1367

Special Issue Editors

Dr. Jonathan C. Roberts

E-Mail Website
Guest Editor
1. Bleeding & Clotting Disorders Institute, Dills Family Foundation Center for Research at BCDI, Peoria, IL, USA
2. Departments of Pediatrics and Medicine, University of Illinois College of Medicine at Peoria, Peoria, IL, USA
Interests: hemophilia; Von Willebrand disease; coagulation laboratory testing; hemostasis; thrombosis; platelet disorders; pa-tient-centered outcome measures; anticoagulation
Dr. Michael B. Nichol

E-Mail Website
Guest Editor
Sol Price School of Public Policy, University of Southern California, Los Angeles, CA 90089, USA
Interests: hemophilia; prophylaxis; gene therapy; burden of illness; quality of life; patient-reported outcomes
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Hemophilia diagnosis and treatment have evolved significantly in the recent twenty years. Increasing attention is being paid to the burden of illness, quality of life, and treatment preferences for individuals with this disorder. While principal outcomes frequently measure bleeds, joint arthropathy, and pain, other quality-of-life considerations may be important to patients. Most importantly, there is recognition that this disorder affects individuals across the globe, including women who have been under-recognized with hemophilia. Moreover, international efforts have advocated for broader treatment and treatment options.

With the development of a variety of new treatments, it is essential to develop and assess research on treatment outcomes. In addition to clinical trials, observational studies can provide insights into treatment effects and predictors of success. The focus of this Special Issue is to report on new areas of treatment, new measurements being used to identify treatment effects, and treatment outcomes. This Issue will also consider the potential impacts of treatments presently under development and how clinicians may adopt them.

Dr. Jonathan C. Roberts
Dr. Michael B. Nichol
Guest Editors

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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. Journal of Clinical Medicine is an international peer-reviewed open access semimonthly 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 2600 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
  • prophylaxis
  • gene therapy
  • burden of illness
  • quality of life
  • patient reported outcomes
  • hemarthrosis
  • women and hemophilia

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Published Papers (2 papers)

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Research

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9 pages, 220 KB  
Article
The VincerEmo Pilot Study: Prospective Analysis of Controlled Physical Activity in People with Severe Hemophilia
by Federica Valeri, Cristina Dainese, Piera Merli, Mariella Galizia, Samuel Agostino, Nicolas Cunsolo, Carola Sella, Alessandra Valpreda, Mariagiulia Bailon, Marco Miniotti, Annamaria Porreca, Giuseppe Massazza, Benedetto Bruno and Alessandra Borchiellini
J. Clin. Med. 2025, 14(18), 6652; https://doi.org/10.3390/jcm14186652 (registering DOI) - 21 Sep 2025
Abstract
Background/Objectives: The approach to physical activity in people with hemophilia (PwH) is still conditioned by many difficulties. Thus, a prospective observational pilot study has been carried out aiming to evaluate how an adequate and controlled training program can slow down the onset [...] Read more.
Background/Objectives: The approach to physical activity in people with hemophilia (PwH) is still conditioned by many difficulties. Thus, a prospective observational pilot study has been carried out aiming to evaluate how an adequate and controlled training program can slow down the onset or evolution of arthropathy and improve musculoskeletal health and quality of life. Methods: Performed from April 2022 to April 2023, this study involved nine severe hemophilic A and B patients, aged > 18 years old, on regular prophylaxis with replacement products. Participants, without changing the usual prophylaxis schedule and maintaining a trough level of at least 20% FVIII/FIX before training, were involved in physical activity twice a week. Results: After 12 months, no increase in annual bleeding ratio (ABR) was observed, and baseline joint status (as assessable by HEAD US score, HJHS, and NRS) was maintained. Even if not statistically significant, a trend toward improvement in mean HEAD US score (15.55 vs. 13.11) and HJHS (14.4 vs. 11) from baseline was observed. Some of the physical tests performed showed a significant improvement at 6 months and 12 months from baseline (5 Rep Sit to Stand, Sit and Reach, and 6-minute Walking Test), meaning an improvement in leg strength, dorsal flexibility, and aerobic resistance. Conclusions: This is the first pilot study evaluating at 360 degrees the safety and impact of a controlled physical activity in PwH. No participant experienced bleedings or a worsening in joint status, but they experienced an improvement in articular functionality. Without changing the usual prophylaxis, scheduling training sessions according to individual pharmacokinetics turned out to be a safe and a cost-effective approach. Full article
(This article belongs to the Special Issue Hemophilia: Current Trends and Future Directions)

Review

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17 pages, 825 KB  
Review
Patient-Centred Management of Well-Controlled Haemophilia: Obtaining Opinions and Definitions Through a Delphi Consensus
by Rubén Berrueco, Inmaculada Soto, José María Bastida, José Manuel Calvo Villas, Carmen de Cos, Saturnino Haya, Francisco Sierra García and José Mateo Arranz
J. Clin. Med. 2025, 14(10), 3300; https://doi.org/10.3390/jcm14103300 - 9 May 2025
Viewed by 762
Abstract
Background/Objectives: For people with haemophilia, health-related quality of life mainly depends on the arthropathy caused by repeated joint bleeding. Prophylaxis is the standard of care in patients with severe bleeding phenotypes, but globally, none of the measures used to assess patients’ outcomes [...] Read more.
Background/Objectives: For people with haemophilia, health-related quality of life mainly depends on the arthropathy caused by repeated joint bleeding. Prophylaxis is the standard of care in patients with severe bleeding phenotypes, but globally, none of the measures used to assess patients’ outcomes consider their desires and life expectations. We propose the concept of the “patient-centred management of well-controlled haemophilia” to define individual responses to prophylaxis. The aims of this work are (1) to achieve agreement about the definition of the “patient-centred management of well-controlled haemophilia” by a steering committee of experts, and (2) to share a series of statements that should define the “patient-centred management of well-controlled haemophilia” with other haemophilia clinicians looking for a consensus in this scenario. Methods: An eight-expert group was established to define the concept of the “patient-centred management of well-controlled haemophilia”. Seven major aspects were identified, and a final version of 42 statements was established and distributed to a 75-expert panel for consensus gathering using the Delphi methodology. Results: Forty-eight experts participated in the first round (participation rate: 64%); two sentences from domain 3 were split, leading to a total of 44 statements across the seven domains. Consensus was achieved in 92.85% of cases. Five items and three statements advanced to the second round. Eleven statements were reconsidered in the second round (response rate: 100%). The questionnaire presented high internal consistency. Conclusions: New treatments offer promising solutions for patients, but there is a paucity of models to measure global outcomes. Patient-centred medicine requires multidimensional assessment, and the “patient-centred management of well-controlled haemophilia” concept is intended to enable this. Full article
(This article belongs to the Special Issue Hemophilia: Current Trends and Future Directions)
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