Haemophilia B in Algeria: Realities and therapeutic perspectives.


Journal

Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916

Informations de publication

Date de publication:
Sep 2023
Historique:
revised: 19 06 2023
received: 22 02 2023
accepted: 04 07 2023
medline: 19 9 2023
pubmed: 19 7 2023
entrez: 19 7 2023
Statut: ppublish

Résumé

Haemophilia B is a debilitating hereditary coagulation disorder characterized by prolonged or spontaneous episodes of bleeding caused by a deficiency of endogenous factor IX. In Algeria, even though many studies are being carried out to evaluate the prevalence and management of haemophilia B, there is a paucity of locally published literature that can be used to understand the most recent information on the disease's epidemiology, diagnostic techniques and treatment options. The aim of this manuscript is to raise awareness among patients and family clinicians about current practices, recent developments and unmet needs related to haemophilia B in Algeria. A comprehensive literature search was conducted through online scientific databases to review publications regarding haemophilia B in Algeria. Exclusions of the review include case studies, interregional comparisons, abstract-only papers and studies outside the range of 2012-2022. The findings discussed relate to the epidemiology of haemophilia B in Algeria, the clinical diagnostic process, disease symptoms, the benefits of molecular and genetic testing, advancements in prophylactic care, as well as unmet needs hindering the progression of optimal haemophilia B management. These findings are crucial to encourage the maintenance of national registries with updated epidemiological data, facilitate early and timely detection of disease symptoms, improve the provision of diagnostic facilities and enhance the overall treatment landscape for better patient outcomes.

Identifiants

pubmed: 37467156
doi: 10.1111/hae.14827
doi:

Substances chimiques

Factor IX 9001-28-9

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

1176-1183

Subventions

Organisme : Pfizer

Informations de copyright

© 2023 John Wiley & Sons Ltd.

Références

Shen G, Gao M, Cao Q, Li W. The molecular basis of FIX deficiency in hemophilia B. Int J Mol Sci. 2022;23(5):2762.
Benson G, Auerswald G, Dolan G, et al. Diagnosis and care of patients with mild haemophilia: practical recommendations for clinical management. Blood Transfus. 2018;16(6):535-544.
Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia. Haemophilia. 2020;26(6):1-158. 3rd edition. Suppl.
Iorio A, Stonebraker JS, Chambost H, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med. 2019;171(8):540-546.
World Federation of Hemophilia. Annual Global Survey 2020. 2020.
Castaman G. The benefits of prophylaxis in patients with hemophilia B. Expert Rev Hematol. 2018;11(8):673-683.
Association Nationale Des Hemophiles Algeriens. 2016. Accessed September 26, 2022. https://anha-dz.com/ressources/
Santagostino E, Fasulo MR. Hemophilia A and hemophilia B: different types of diseases? Semin Thromb Hemost. 2013;39(7):697-701.
Quick AJ, Hussey CV. Hemophilia B (PTC deficiency, or Christmas disease). AMA Arch Intern Med. 1959;103(5):762-775.
Franchini M, Mannucci PM. Non-factor replacement therapy for haemophilia: a current update. Blood Transfus. 2018;16(5):457-461.
Berkouk-Redjimi Y, Ouarlent Y, Hamdi S, et al. Orthopaedic status and surgical needs of patients with haemophilia in Algeria. Open J Hematol. 2013;4:1.
Franchini M, Mannucci PM. Inhibitors of propagation of coagulation (factors VIII, IX and XI): a review of current therapeutic practice. Br J Clin Pharmacol. 2011;72(4):553-562.
Gomez K, Klamroth R, Mahlangu J, Mancuso ME, Mingot ME, Ozelo MC. Key issues in inhibitor management in patients with haemophilia. Blood Transfus. 2014;12(suppl 1):s319-329.
Goodeve AC. Hemophilia B: molecular pathogenesis and mutation analysis. J Thromb Haemost. 2015;13(7):1184-1195.
Taylor JA, Kruse-Jarres R. A new era for hemophilia B treatment. Blood. 2016;127(14):1734-1736.
Hemophiliacs TNAo. National guidelines on prophylaxis in children with hemophilia.
Bencheikh EL, Fegoun S. Epidemiology of hemophilia and its complications in Algeria in 2006. In: 3rd Congress of the Algerian Society of Hematology and Blood Transfusion, Algeria. 2006.
Berkouk-Redjimi Y. Hierarchy of orthopedic surgery gestures in hemophiliacs in Algeria. In: 5th Maghreb Congress of Hematology, Marrakesh, Morocco; 2008.
Touhami H. Essay to evaluate the care of hemophiliacs in Algeria in relation to the ten current European principles. In: IX National Congress of SAHTS, Tlemcen, 10-12 May 2012. 2012.
Bensadok MBM.Hemophilia: current situation in Algeria. Training day on hemophilia. May 3, 2014.
Nekkal S, Ndir D, Bouzbid S, et al. First real-world evidence study of patients with hemophilia in Algeria. Value in Health. 2022;25(12):463-464.
Konkle BA, Huston H, Nakaya Fletcher S, et al. Hemophilia B. In: Adam MP, Everman DB, Mirzaa GM, et al., eds. GeneReviews(®). University of Washington. Copyright © 1993-2022, University of Washington, Seattle. GeneReviews is a registered trademark of the University of Washington. Seattle. All rights reserved. 1993.
Motlagh H, Pezeshkpoor B, Dorgalaleh A. Hemophilia B. In: Congenital Bleeding Disorders. Springer International Publishing; 2018:139-160.
Abla ZMY, Hejer EM, et al. Molecular genetic study of hemophilia B in an Algerian population. African J Biotech. 2016;15(51):2784-2788.
Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388(10040):187-197.
Mehta P, Reddivari AKR. Hemophilia. In: StatPearls. StatPearls Publishing LLC; 2022.
Boender J, Kruip MJ, Leebeek FW. A diagnostic approach to mild bleeding disorders. J Thromb Haemost. 2016;14(8):1507-1516
Goodeve AC, Rosen S, Verbruggen B. Haemophilia A and von Willebrand's disease. Haemophilia. 2010;16(suppl 5):79-84.
Kizilocak H, Young G. Diagnosis and treatment of hemophilia. Clin Adv Hematol Oncol. 2019;17(6):344-351.
Abla Z, Mouloud Y, Hejer EM, et al. Mutations causing hemophilia B in Algeria: Identification of two novel mutations of the factor 9 gene. Biodiversitas J Biol Diversity. 2018;19(1):52-58.
Décision n17 du 16 Mai 2022 Fixant le Reseau National de Prophylaxie des Hemophiles. [press release]. Ministre de la Santé, Republique Algérienne Démocratique et Populaire; 2022.
Coppola A, Tagliaferri A, Di Capua M, Franchini M. Prophylaxis in children with hemophilia: evidence-based achievements, old and new challenges. Semin Thromb Hemost. 2012;38(1):79-94.
Nazeef M, Sheehan JP. New developments in the management of moderate-to-severe hemophilia B. J Blood Med. 2016;7:27-38.
Arruda VR, Doshi BS, Samelson-Jones BJ. Novel approaches to hemophilia therapy: successes and challenges. Blood. 2017;130(21):2251-2256.
Von Drygalski A, Giermasz A, Castaman G, et al. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Adv. 2019;3(21):3241-3247.
Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1-47.
Torres-Ortuño A. Adherence to prophylactic treatment. Blood Coagul Fibrinolysis. 2019;30(suppl 1):S19-S21.
Berkouk-Redjimi H. Experience in primary prophylaxis in an emerging country «Algeria» and the preliminary results of a low dose protocol. In: WFH Congress; 2014.
Monahan PE. Gene therapy in an era of emerging treatment options for hemophilia B. J Thromb Haemost. 2015;13(suppl 1):S151-160.
Rodríguez-Merchán EC, De Pablo-Moreno JA, Liras A. Gene therapy in hemophilia: recent advances. Int J Mol Sci. 2021;22(14):7647.
Tomeo F, Mariz S, Brunetta AL, Stoyanova-Beninska V, Penttila K, Magrelli A. Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective. Br J Clin Pharmacol. 2021;87(11):4183-4196.
Santoro C, Quintavalle G, Castaman G, et al. Inhibitors in hemophilia B. Semin Thromb Hemost. 2018;44(6):578-589.
Ling G, Nathwani AC, Tuddenham EGD. Recent advances in developing specific therapies for haemophilia. Br J Haematol. 2018;181(2):161-172.
Zhao Y, Weyand AC, Shavit JA. Novel treatments for hemophilia through rebalancing of the coagulation cascade. Pediatr Blood Cancer. 2021;68(5):e28934.
Ellsworth P, Ma A. Factor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates? Hematol Am Soc Hematol Educ Program. 2021;2021(1):219-225.
Jimenez-Yuste V, Auerswald G, Benson G, et al. Practical considerations for nonfactor-replacement therapies in the treatment of haemophilia with inhibitors. Haemophilia. 2021;27(3):340-350.
Miesbach W, Schwäble J, Müller MM, Seifried E. Treatment options in hemophilia. Dtsch Arztebl Int. 2018;116(47):791-798.
Trinchero A, Sholzberg M, Matino D. The evolution of hemophilia care: clinical and laboratory advances, opportunities, and challenges. Hamostaseologie. 2020;40(3):311-321.
Ramaswamy S, Tonnu N, Menon T, et al. Autologous and heterologous cell therapy for hemophilia B toward functional restoration of factor IX. Cell Rep. 2018;23(5):1565-1580.
Ghosh K, Ghosh K. Management of haemophilia in developing countries: challenges and options. Indian J Hematol Blood Transfus. 2016;32(3):347-355.
Ghosh K, Ghosh K. Overcoming the challenges of treating hemophilia in resource-limited nations: a focus on medication access and adherence. Expert Rev Hematol. 2021;14(8):721-730.
Mbanya DN, Diop S, Ndoumba Mintya AN, El Kiaby M. Hemophilia care in Africa: status and challenges. Transfus Clin Biol. 2021;28(2):158-162.
Lambert T, Benson G, Dolan G, et al. Practical aspects of extended half-life products for the treatment of haemophilia. Ther Adv Hematol. 2018;9(9):295-308.
Ar MC, Balkan C, Kavakli K. Extended half-life coagulation factors: a new era in the management of hemophilia patients. Turk J Haematol. 2019;36(3):141-154.
Gouider E, Jouini L, Achour M, et al. Low dose prophylaxis in Tunisian children with haemophilia. Haemophilia. 2017;23(1):77-81.
Bensadok M, Almomen A, Alzoebie A, et al. Home treatment of haemarthrosis with recombinant activated factor VII in patients with haemophilia A or B and inhibitors: experience from developing countries. Blood Coagul Fibrinolysis. 2017;28(2):145-151.
Goga Y, Boukari R, Bensadok M, et al. REACH: a programme for improving care in haemophilia. Haemophilia. 2019;25(3):e211-e215.

Auteurs

Mohamed Salim Nekkal (MS)

Issad Hassani Hospital, Algiers, Algeria.

Naima Mesli (N)

Dr Tidjani Damerdji University Hospital, Tlemcen, Algeria.

Fatiha Grifi (F)

Annaba University Hospital, Annaba, Algeria.

Nacira Cherif (N)

Issad Hassani Hospital, Algiers, Algeria.

Zohra Ouchenane (Z)

Constantine Dr Benbadis University Hospital, Constantine, Algeria.

Mohamed Said Bettayeb (MS)

Hospital Business and Rare Diseases, Pfizer Inc. Ltd, Algiers, Algeria.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH