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Challenges in the Management of Hemophilia
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Challenges in the Management of Hemophilia

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

Deadline for manuscript submissions: 20 May 2024 | Viewed by 1541

Special Issue Editor

Dr. Maria Elisa Mancuso

E-Mail Website
Guest Editor
IRCCS Humanitas Research Hospital, Rozzano, 20089 Milan, Italy
Interests: hemophilia; bleeding disorder
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

This Special Issue is the second edition of “Diagnosis and Management of Hemophilia: Old and New Challenges” (https://www.mdpi.com/journal/jcm/special_issues/Hemophilia).

In the field of congenital bleeding disorders, hemophilia A and B represent the forms with the major clinical impact. In the recent past, a tremendous advance in the treatment landscape, along with the optimization of diagnostic and monitoring tools, has allowed changing the natural history of the disease with consequent improvement in survival and disease-related morbidity. Prophylaxis is the cornerstone of treatment aiming at zero bleeds and healthy joints, and thanks to EHL FVIII/FIX products and nonreplacement therapies, this is feasible in all patients irrespective of the presence of inhibitory antibodies. Moreover, gene therapy is rapidly developing. Despite this exciting progress, treating physicians still face old and new challenges for the diagnosis and the management of hemophilia and its complications. Preimplant/prenatal diagnosis and genetic counseling are not available worldwide as an adequate supply of concentrates to allow for the widespread use of prophylaxis. The early detection of joint damage remains challenging and the possibility to prevent it through new nonreplacement therapies still unknown. Management of inhibitor patients requires high-level expertise, and the need for inhibitor eradication is a matter of debate in the new treatment era. Finally, the advent of many different new molecules represents a challenge for the laboratory where the most reliable monitoring assays should be identified.

Dr. Maria Elisa Mancuso
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. 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

  • prenatal diagnosis
  • prophylaxis
  • EHL clotting factor concentrates
  • nonreplacement therapies
  • inhibitors
  • immune tolerance induction
  • hemophilic arthropathy
  • laboratory assays
  • outcome measures

Related Special Issue

Published Papers (2 papers)

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Research

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10 pages, 210 KiB  
Article
Better Communication for Better Management of Persons with Hemophilia: Results from a Patients’-Clinicians’ Project to Address the New Therapeutic Landscape
by Laura Banov, Silvia Linari, Luigi Ambroso, Enrico Ferri Grazzi, Samanta Gallo, Patrizio Pasqualetti and Maria Elisa Mancuso
J. Clin. Med. 2024, 13(2), 568; https://doi.org/10.3390/jcm13020568 - 19 Jan 2024
Viewed by 871
Abstract
Applying the Delphi method, this study aims at characterizing the perceptions and needs of physicians, individuals with hemophilia, and their caregivers in relation to the management of routine visits during regular follow-ups. A single structured questionnaire, prepared by an advisory board, was administered [...] Read more.
Applying the Delphi method, this study aims at characterizing the perceptions and needs of physicians, individuals with hemophilia, and their caregivers in relation to the management of routine visits during regular follow-ups. A single structured questionnaire, prepared by an advisory board, was administered to 139 participants, comprising hemophilia treaters, patients and caregivers, during the period from May to June 2023. Agreement (defined following the Delphi method as developed by RAND Corporation) was reached on several topics. The Principal Component Analysis methods identified the four most relevant areas where consensus was reached among the interviewees, underscoring the necessity for in-depth discussions during routine visits: (1) medical aspects related to symptoms, life-limitations, pain, etc.; (2) non-medical related aspects (ambitions, lifestyle, network, etc.); (3) logistical-organizational aspects (home–hospital distance, alternative modalities of communication); and (4) visit duration and telemedicine integration. The results of both the Delphi and Principal Component Analysis underline that the care of individuals with hemophilia extends beyond merely prescribing drugs or treatment regimens. Instead, it necessitates consideration of numerous variables from both therapeutic and non-therapeutic domains, all of which are deemed important for the holistic management of the individuals. As a result, these aspects are routinely discussed and addressed during visits. Full article
(This article belongs to the Special Issue Challenges in the Management of Hemophilia)

Review

Jump to: Research

14 pages, 572 KiB  
Review
Total Knee Arthroplasty in People with Hemophilia: Higher Incidence of Periprosthetic Joint Infection and 1-Year Revision/Re-Operation than the General Population and Lower Prosthetic Survival When Early Postoperative Bleeding Complications Occurred: Current Literature Review
by Emerito Carlos Rodriguez-Merchan, Mario Mosconi, Hortensia De la Corte-Rodriguez, Eugenio Jannelli and Gianluigi Pasta
J. Clin. Med. 2024, 13(8), 2447; https://doi.org/10.3390/jcm13082447 - 22 Apr 2024
Viewed by 426
Abstract
The purpose of this narrative review of the recent literature is to analyze the outcomes, complications, and implant survival of total knee arthroplasty (TKA) carried out on people with hemophilia (PWH). It has been shown that TKA substantially alleviates preoperative pain and improves [...] Read more.
The purpose of this narrative review of the recent literature is to analyze the outcomes, complications, and implant survival of total knee arthroplasty (TKA) carried out on people with hemophilia (PWH). It has been shown that TKA substantially alleviates preoperative pain and improves knee function and the patient’s quality of life. However, the complication rates of TKA range between 8.5% and 28.7, with postoperative hemarthrosis being the most frequent (7.6%). Besides, when comparing if the TKA was implanted before or after the year 2000, a reduction was found in the rates of periprosthetic joint infection—PJI (6.2% to 3.9%) and aseptic loosening (3.8% to 2.1%). Comparing prosthesis survival between PWH who had suffered early postoperative bleeding complications (EPBC) and patients who did not suffer EBPC, the mean survival duration was 17 years for the EPBC group and 22.1 years for the non-EPBC group. Survival rates were 80% for the EPBC group and 96.4% for the non-EPBC group. Compared to patients without hemophilia, PWH had a substantially higher incidence of PJI (Odds Ratio—OR 1.6) and 1-year revision/re-operation (OR 1.4). In short, although TKA substantially improves the quality of life of PWH, it is an intervention that has a non-negligible percentage of complications. TKA in PWH should preferably be performed only in highly specialized centers for the orthopedic treatment of hemophilia. Full article
(This article belongs to the Special Issue Challenges in the Management of Hemophilia)
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