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Clinical profile and outcome of children with haemophilia A: The Royal Hospital, Oman’s experience Cover

Clinical profile and outcome of children with haemophilia A: The Royal Hospital, Oman’s experience

Open Access
|Mar 2025

References

  1. Kaneda M, Kawasaki R, Matsumoto N, et al. Detailed analysis of anti-emicizumab antibody decreasing drug efficacy, using plasma samples from a patient with hemophilia A. J Thromb Haemost 2021; 19: 2938–46. doi: 10.1111/jth.15506.
  2. Bolton-Maggs PHB, Pasi KJ. Haemophilias A and B. Lancet 2003; 361: 1801–9. doi: 10.1016/S0140-6736(03)13405-8.
  3. Franchini M, Mannucci PM. Hemophilia A in the third millennium. Blood Rev 2013; 27:179–84. doi: 10.1016/j.blre.2013.06.002.
  4. Kulkarni R, Soucie JM. Pediatric hemophilia: a review. Semin Thromb Hemost 2011; 37: 737–44. doi: 10.1055/s-0031-1297164.
  5. Sidonio RF. “A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH”: Reply to comment. J Thromb Haemost 2022; 20: 1745–6. doi: 10.1111/jth.15726.
  6. 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; 171:540–6. doi: 10.7326/M19-1208.
  7. Kulkarni R, Soucie JM, Lusher J, et al. Sites of initial bleeding episodes, mode of delivery and age of diagnosis in babies with haemophilia diagnosed before the age of 2 years: a report from The Centers for Disease Control and Prevention’s (CDC) Universal Data Collection (UDC) project. Haemophilia 2009; 15: 1281–90. doi: 10.1111/j.1365-2516.2009.02074.x.
  8. Tagliaferri A, Di Perna C, Riccardi F, et al. The natural history of mild haemophilia: a 30-year single centre experience. Haemophilia 2012; 18: 166–74. doi: 10.1111/j.1365-2516.2011.02617.x.
  9. Balak DMW, Gouw SC, Plug I, et al. Prenatal diagnosis for haemophilia: a nationwide survey among female carriers in the Netherlands. Haemophilia 2012; 18: 584–92. doi: 10.1111/j.1365-2516.2011.02742.x.
  10. Valentino LA. Blood-induced joint disease: the pathophysiology of hemophilic arthropathy. J Thromb Haemost 2010; 8: 1895–902. doi: 10.1111/j.1538-7836.2010.03962.x.
  11. Zimmerman B, Valentino LA. Hemophilia: in review. Pediatr Rev 2013; 34: 289–94. doi: 10.1542/pir.34-7-289.
  12. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535–44. doi: 10.1056/NEJMoa067659.
  13. Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011; 9: 700–10. doi: 10.1111/j.1538-7836.2011.04214.x.
  14. Gouw SC, van den Berg HM, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood 2013;121:4046–55. https://doi.org/10.1182/blood-2012-09-457036.
  15. Regling K, Callaghan MU, Sidonio R. Managing severe hemophilia A in children: pharmacotherapeutic options. Pediatr Health Med Ther 2022; 13: 27–35. doi: 10.2147/PHMT.S293246.
  16. Negrier C, Young G, Abdul Karim F, et al. Recombinant long-acting glycoPEGylated factor IX (nonacog beta pegol) in haemophilia B: assessment of target joints in multinational phase 3 clinical trials. Haemophilia 2016; 22: 507–13. doi: 10.1111/hae.12902.
  17. Oudat R, Al-Maharmeh M, Al-Ghrayeb R, Ogeilat T, Mustafa MK. Prevalence of FVIII inhibitors among children with hemophilia A: Experience at the Jordanian Royal Medical Services. Med Arch Sarajevo Bosnia Herzeg 2020; 74: 187–90. doi: 10.5455/medarh.2020.74.187-190.
  18. Trimble SR, Parker CS, Grant AM, Soucie JM, Reyes N. Assessing emerging infectious threats to blood safety for the blood disorders community. Am J Prev Med 2010; 38: S468–474. doi: 10.1016/j.amepre.2009.12.019.
  19. Oldenburg J. Mutation profiling in haemophilia A. Thromb Haemost 2001; 85: 577–9. doi: 10.1055/s-0037-1615636.
  20. 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: 535–44. doi: 10.2450/2017.0150-17.
  21. Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet 2016; 388: 187–97. doi: 10.1016/S0140-6736(15)01123-X.
  22. Pezeshkpoor B, Oldenburg J, Pavlova A. Insights into the molecular genetic of hemophilia A and hemophilia B: The relevance of genetic testing in routine clinical practice. Hamostaseologie 2022; 42: 390–9. doi: 10.1055/a-1945-9429.
  23. Owaidah T, Momen AA, Alzahrani H, et al. The prevalence of factor VIII and IX inhibitors among Saudi patients with hemophilia: Results from the Saudi national hemophilia screening program. Medicine (Baltimore) 2017; 96: e5456. doi: 10.1097/MD.0000000000005456.
  24. Kadhim KAR, Al-Lami FH, Baldawi KH. Epidemiological profile of hemophilia in Baghdad-Iraq. Inquiry 2019; 56: 46958019845280. doi: 10.1177/0046958019845280.
  25. Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood 2015; 125: 2038–44. doi: 10.1182/blood-2015-01-528414.
  26. Rehill AM, McCluskey S, O’Donnell JS, et al. Heterogeneity in bleeding tendency and arthropathy development in individuals with hemophilia. Semin Thromb Hemost 2021; 47: 183–91. doi: 10.1055/s-0041-1723769.
  27. Gringeri A, von Mackensen S, Auerswald G, et al. Health status and health-related quality of life of children with haemophilia from six West European countries. Haemophilia 2004; 10 Suppl 1: 26–33. doi: 10.1111/j.1355-0691.2004.00876.x.
  28. Plug I, van der Bom JG, Peters M, et al. Thirty years of hemophilia treatment in the Netherlands, 1972–2001. Blood 2004; 104: 3494–500. doi: 10.1182/blood-2004-05-2008.
  29. Di Minno MND, Ambrosino P, Franchini M, Coppola A, Di Minno G. Arthropathy in patients with moderate hemophilia a: a systematic review of the literature. Semin Thromb Hemost 2013; 39: 723–31. doi: 10.1055/s-0033-1354422.
  30. Gualtierotti R, Solimeno LP, Peyvandi F. Hemophilic arthropathy: Current knowledge and future perspectives. J Thromb Haemost 2021; 19: 2112–21. doi: 10.1111/jth.15444.
  31. Mannucci PM. Hemophilia therapy: the future has begun. Haematologica 2020; 105: 545–53. doi: 10.3324/haematol.2019.232132.
  32. Nazir H, khanbashi L, Wali Y, et al. Screening for inhibitor development and its risk factors in patients with severe haemophilia a in Oman. Hematol Transfus Int J 2018; 6: 87–90. doi: 10.15406/htij.2018.06.00159.
  33. Morfini M, Haya S, Tagariello G, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 2007; 13: 606–12. doi: 10.1111/j.1365-2516.2007.01518.x.
Language: English
Page range: 23 - 28
Published on: Mar 19, 2025
Published by: Haemnet Ltd
In partnership with: Paradigm Publishing Services
Publication frequency: 1 times per year

© 2025 Anood AlRawahi, Ibrahim AlGaithi, Fatma Al Riyami, Maather Al Abri, Hajer Al Shukaili, Abdulhakim Al Rawahi, published by Haemnet Ltd
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.