Prophylaxis is the new standard of care in patients with haemophilia

dc.contributor.authorMahlangu, J.
dc.contributor.authorBassa, F.
dc.contributor.authorBassingthwaighte, M.
dc.contributor.authorCruickshank, A-L.
dc.contributor.authorDu Plessis, J.
dc.contributor.authorGoga, Y.
dc.contributor.authorJoubert, J.
dc.contributor.authorLouw, V.
dc.contributor.authorMahlachana, N.
dc.contributor.authorMathew, R.
dc.contributor.authorNeethling, B.
dc.contributor.authorPotgieter, J.
dc.contributor.authorRapiti, N.
dc.contributor.authorRayner, B.
dc.contributor.authorReynders, David
dc.contributor.authorSutton, C.
dc.contributor.authorVan Zyl, A.
dc.date.accessioned2022-11-28T13:31:59Z
dc.date.available2022-11-28T13:31:59Z
dc.date.issued2022-06
dc.description.abstractRandomised controlled clinical trial evidence on prophylaxis as optimal care for patients with haemophilia was generated more than a decade ago. However, this knowledge has not translated into clinical practice in South Africa (SA) owing to many barriers to prophylaxis. These include the high treatment burden imposed by prophylaxis (frequent injections two to four times a week), the need for intravenous access to administer replacement clotting factor therapies, and the higher volume of clotting factor required compared with episodic treatment. The recently introduced non-factor therapies in haemophilia care have addressed many of these barriers. For example, emicizumab, which is currently the only globally approved non-factor therapy, can be administered subcutaneously less frequently (weekly, fortnightly or every 4 weeks) and has led to global adoption of prophylaxis as the standard of care in haemophilia by the bleeding disorders community. Haemophilia A is the most prevalent clotting factor deficiency in SA, with >2 000 people diagnosed to date. However, only a few of these patients are currently on prophylaxis. In this ‘In Practice’ article, we review the rationale for prophylaxis, outline its goals and benefits, and provide evidence-based guidance on which haemophilia patients should be prioritised for emicizumab prophylaxis. This consensus guidance facilitates the adoption of prophylaxis as a national policy and the new standard of care in haemophilia in SA.en_US
dc.description.departmentHaematologyen_US
dc.description.departmentPaediatrics and Child Healthen_US
dc.description.librariandm2022en_US
dc.description.urihttp://www.samj.org.zaen_US
dc.identifier.citationMahlangu, J., Bassa, F., Bassingthwaighte, M., et al. Prophylaxis is the new standard of care in patients with haemophilia. South African Medical Journal 2022, vol. 112, no. 6, pp.405-408. http://dx.doi.org/10.7196/SAMJ.2022.v112i6.16362.en_US
dc.identifier.issn2078-5135 (online)
dc.identifier.issn0256-9574 (print)
dc.identifier.other10.7196/SAMJ.2022.v112i6.16362
dc.identifier.urihttps://repository.up.ac.za/handle/2263/88503
dc.language.isoenen_US
dc.publisherHealth and Medical Publishing Groupen_US
dc.rightsThis open-access article is distributed under Creative Commons licence CC-BY-NC 4.0.en_US
dc.subjectProphylaxisen_US
dc.subjectPatientsen_US
dc.subjectHaemophiliaen_US
dc.titleProphylaxis is the new standard of care in patients with haemophiliaen_US
dc.typeArticleen_US

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