Emicizumab as preventive treatment of bleeding in a patient with severe hemophilia A. A case report
Keywords:
HEMOPHILIA A, INHIBITORS, EMICIZUMAB.Abstract
Introduction: hemophilia is an inherited disease that prevents proper blood clotting and constitutes 80 to 85% of all hereditary bleeding diathesis. It is caused by decreased activity of factor VIII (hemophilia A) or factor IX (hemophilia B).
Case report: 18-year-old adolescent with white skin, attended at the Hematology Department of the Pepe Portilla Pediatric Teaching Hospital in Pinar del Río, who in the neonatal stage presented a hematoma on the right thigh after the administration of the hepatitis B vaccine associated to bleeding from the puncture sites. At 3 months of age he presented his first hemarthrosis in the right knee, an unusual event at this stage of life due to poor mobility. At 11 years of age, physiotherapy treatment was started with hydrotherapy with encouraging results in the recovery of muscle tone. At the beginning of 2015, when she was 12 years old, she began to use the prophylaxis regimen with F VIII three times a week, which together with the rehabilitation sessions, made possible the decrease of bleeding, as well as deambulation, incorporating in person and without the support of wheelchairs to the teaching scenario.
Conclusions: it is concluded that the use of emicizumab in a patient with hemophilia A without inhibitors, presented a high safety profile, as well as a high efficiency in the prevention of bleeding tendency.
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1. Rodrigo Valero AM. Coagulopatías congénitas y adquiridas. NPunto [Internet]. Marzo 2020 [Citado 20 de junio de 2021]; III (24): 4-22. Disponible en: https://www.npunto.es/content/src/pdf-articulo/5e79d8dd5a9c8NPvolumen24-4-22.pdf
2. Hemophilia News Today. What Is Hemophilia? [Internet]. BioNews; 2022 [Citado 20 de junio de 2021]. Disponible en: https://hemophilianewstoday.com/what-is-hemophilia/?cn-reloaded=1
3. Castillo González D, Lardoeyt Ferrer R, Almagro Vázquez D, Lam Díaz R, Lavaut Sánchez K, et al. Prevalence of hemophilia in six cuban provinces. Revista Cubana de Hematología, Inmunología y Hemoterapia [Internet]. 2014 [Citado 20 de junio de 2021]; 30(2). Disponible en: http://www.revhematologia.sld.cu/index.php/hih/article/view/145
4. Bladen M, Carroll L, Dodd C, Drechsler W, Hashem F, Patel V, et al(2020). Results of feasibility and safety of randomised controlled trial of a musculoskeletal exercise intervention versus usual care for children with haemophilia. Haemophilia [Internet]. 2020 Sep [Citado 20 de junio de 2021]; 26(5):e223-e225. Disponible en: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7780159/
5. Osorio GM, Chávez SL, Silva GM, Parrello S, Iorio I. Partners of patients with a chronic disease. Narrative enquiry of hemophilia. Rev Electrónica Psicol Iztacala [Internet]. 2017 [Citado 09/08/2019]; 20(2): 548-569. Disponible en: http://www.iztacala.unam.mx/carreras/psicologia/psiclin
65. Osorio GM, Puente R MdC, Bazán R GE. Repercusiones físicas y sociales en la salud de portadoras que son a la vez cuidadoras primarias de pacientes con hemofilia. Psicol y salud [Internet]. 2019 [Citado 17/08/2019]; 29(1): 33–40. Disponible en:
https://psicologiaysalud.uv.mx/index.php/psicysalud/article/download/2566/4461
7. Oladapo AO, Lu M, Walsh S, et al. Inhibitor clinical burden of disease: a comparative analysis of the CHESS data. Orphanet J Rare Dis [Internet]. 2018 [Citado 20 de junio de 2021]; 13(198). Disponible en: https://doi.org/10.1186/s13023-018-0929-9
8. Halimeh S, Bidlingmaier Ch, Heller Ch, Gutsche S, Holzhauer S, Kenet G, et al. Clinical Study Risk Factors for High-Titer Inhibitor Development in Children with Hemophilia A: Results of a Cohort Study. BioMed Research International [Internet]. 2013 [Citado 20 de junio de 2021]; 2013(901975): 2-7. Disponible en: http://www.hindawi.com/journals/bmri/2013/901975/
9. Castillo-González D. Hemofilia: aspectos históricos y genéticos. Rev. Cubana Hematol. Inmunol [Internet]. 2012 [Citado 20 de junio de 2021]; 28(1):22-33. Disponible en: https://www.medigraphic.com/pdfs/revcubheminmhem/rch-2012/rch121c.pdf
10. Boulassel MR, Al-Ghonimi M, Al-Balushi B, Al-Naamani A, Al- Qarni Z, Wali Y, et al. Regulatory B cells are functionally impaired in patients having hemophilia A with inhibitors. Clin Appl Thromb Hemost [Internet]. 2018 [Citado 20 de junio de 2021]; 24(4): 618-24. Disponible en: https://journals.sagepub.com/doi/full/10.1177/1076029617702244
11. Franchini M, Mannucci PM. Non-factor replacement therapy for haemophilia: A current update. Blood Transfus [Internet]. 2018 [Citado 20 de junio de 2021]; 16(5): 457-461. Disponible en: https://pubmed.ncbi.nlm.nih.gov/29517971/
12. Raut S, Heath A. South Mimms Inhibitor Assay (SMIA): An affordable and improved method form measurement of FVIII inhibitors. Presented at WFH congress [Internet] 2016. [Citado 15/01/2020]. Disponible en: https://www.postersessiononline.eu/173580348_eu/congresos/WFH2016/aula/-PP-W_78_WFH2016.pdF
13. D Fantl. Guides 2019. Hematología [Internet]. 2019 [Citado 15/01/2020]; 22(No Extra). Disponible en: https://redib.org/Record/oai_articulo2531430-gu%C3%ADas-de-diagn%C3%B3stico-y-tratamiento-de-la-sociedad-argentina-de-hematolog%C3%ADa-edici%C3%B3n-2019
14. Lai JD, Lillicrap D. Factor VIII inhibitors: Advances in basic and translational science. Int J Lab Hematol [Internet]. 2017 May [Citado 15/01/2020]; 39 (Suppl 1): 6-13. Disponible en: https://pubmed.ncbi.nlm.nih.gov/28447409/
15. Franchini M, Favaloro EJ, Lippi G. Mild hemophilia A. J Thromb Haemost [Internet]. 2010 [citado mayo de 2021]; 8(3): 421-32. Disponible en: https://pubmed.ncbi.nlm.nih.gov/19995408/
16. Kitazawa T, Shima M. Emicizumab, a humanized bispecific antibody to coagulation factors IXa and X with a factor VIIIa-cofactor activity. Int J Hematol [Internet]. 2020 [Citado 20 de junio de 2021]; 111(1): 20-30. Disponible en: https://link.springer.com/article/10.1007/s12185-018-2545-9
17. Knight T, Callaghan MU. The role of emicizumab, a bispecific factor IXa- and factor X-directed antibody, for the prevention of bleeding episodes in patients with hemophilia A. Ther Adv Hematol [Internet]. 2018 [Citado 20 de junio de 2021]; 9(10): 319-334. Disponible en: https://doi.org/10.1177/2040620718799997
18. Mahlangu J. Emicizumab for the prevention of bleeds in hemophilia A. Expert Opin Biol Ther [Internet]. 2019 [Citado 20 de junio de 2021]; 19(8): 753-761. Disponible en: https://doi.org/10.1080/14712598.2019.1626370
19. Franchini M, Marano G, Pati I, et al. Emicizumab for the treatment of haemophilia A: a narrative review. Blood Transfus [Internet]. 2019 [Citado 20 de junio de 2021]; 17(3): 223-228. Disponible en: https://doi.org/10.2450/2019.0026-19
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