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Annals of translational medicine
Jul, 2021;9 (13): 1091.

Current therapeutic approaches in the management of hemophilia-a consensus view by the Romanian Society of Hematology.

Ionut Hotea, Melen Brinza, Cristina Blag, Alina-Andreea Zimta, Noemi Dirzu, Corina Burzo, Ioana Rus, Dragos Apostu, Horea Benea, Mirela Marian, Alexandru Mester, Sergiu Pasca, Sabina Iluta, Patric Teodorescu, Ciprian Jitaru, Mihnea Zdrenghea, Anca Bojan, Tunde Torok-Vistai, Radu Niculescu, Cristina Tarniceriu, Delia Dima, Cristina Truica, Margit Serban, Ciprian Tomuleasa, Daniel Coriu
Author Information
  1. Ionut Hotea: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  2. Melen Brinza: Department of Hematology, Fundeni Clinical Institute, Bucharest, Romania.
  3. Cristina Blag: Department of Pediatrics, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  4. Alina-Andreea Zimta: Medfuture Research Center for Advanced Medicine, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  5. Noemi Dirzu: Medfuture Research Center for Advanced Medicine, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  6. Corina Burzo: Department of Hematology, Ion Chiricuta Clinical Cancer Center, Cluj Napoca, Romania.
  7. Ioana Rus: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  8. Dragos Apostu: Department of Orthopedics, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  9. Horea Benea: Department of Orthopedics, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  10. Mirela Marian: Department of Hematology, Ion Chiricuta Clinical Cancer Center, Cluj Napoca, Romania.
  11. Alexandru Mester: Department of Oral Health, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  12. Sergiu Pasca: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  13. Sabina Iluta: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  14. Patric Teodorescu: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  15. Ciprian Jitaru: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  16. Mihnea Zdrenghea: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  17. Anca Bojan: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  18. Tunde Torok-Vistai: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  19. Radu Niculescu: Department of Hematology, Fundeni Clinical Institute, Bucharest, Romania.
  20. Cristina Tarniceriu: Department of Anatomy, Grigore T. Popa University of Medicine and Pharmacy, Iasi, Romania.
  21. Delia Dima: Department of Hematology, Ion Chiricuta Clinical Cancer Center, Cluj Napoca, Romania.
  22. Cristina Truica: Department of Hematology, Constantin Opris Emergency Hospital, Baia Mare, Romania.
  23. Margit Serban: Department of Hematology, Victor Babes University of Medicine and Pharmacy, Timisoara, Romania.
  24. Ciprian Tomuleasa: Department of Hematology, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj Napoca, Romania.
  25. Daniel Coriu: Department of Hematology, Fundeni Clinical Institute, Bucharest, Romania.
PMID: 34423003 DOI: 10.21037/atm-21-747

Abstract

Hemophilia A (HA) and hemophilia B (HB) are rare disorders, being caused by the total lack or under-expression of two factors from the coagulation cascade coded by genes of the X chromosome. Thus, in hemophilic patients, the blood does not clot properly. This results in spontaneous bleeding episodes after an injury or surgical intervention. A patient-centered regimen is considered optimal. Age, pharmacokinetics, bleeding phenotype, joint status, adherence, physical activity, personal goals are all factors that should be considered when individualizing therapy. In the past 10 years, many innovations in the diagnostic and treatment options were presented as being either approved or in development, thus helping clinicians to improve the standard-of-care for patients with hemophilia. Recombinant factors still remain the standard of care in hemophilia, however they pose a challenge to treatment adherence because they have short half-life, which where the extended half-life (EHL) factors come with the solution, increasing the half-life to 96 hours. Gene therapies have a promising future with proven beneficial effects in clinical trials. We present and critically analyze in the current manuscript the pros and cons of all the major discoveries in the diagnosis and treatment of HA and HB, as well as identify key areas of hemophilia research where improvements are needed.

Keywords

Hemophilia Romanian Society of Hematology consensus view diagnosis and management

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