In Practice

Prophylaxis is the new standard of care in patients with haemophilia

J Mahlangu, F Bassa, M Bassingthwaighte, A-L Cruickshank, J du Plessis, Y Goga, J Joubert, V Louw, N Mahlachana, R Mathew, B Neethling, J Potgieter, N Rapiti, B Rayner, D Reynders, C Sutton, A van Zyl

Abstract


Randomised 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.


Authors' affiliations

J Mahlangu, Department of Molecular Medicine and Haematology, National Health Laboratory Service, Charlotte Maxeke Johannesburg Academic Hospital and School of Pathology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa

F Bassa, Division of Clinical Haematology, Department of Medicine, Faculty of Medicine and Health Sciences, Stellenbosch University and Tygerberg Academic Hospital, Cape Town, South Africa

M Bassingthwaighte, Department of Paediatrics and Child Health, Faculty of Health Sciences, University of the Witwatersrand and Chris Hani Baragwanath Hospital, Johannesburg, South Africa

A-L Cruickshank, Division of Clinical Haematology, Department of Medicine, Faculty of Health Sciences, University of Cape Town and Groote Schuur Hospital, Cape Town, South Africa

J du Plessis, Department of Paediatrics and Child Health, Faculty of Health Sciences, University of the Free State, Bloemfontein, South Africa

Y Goga, Department of Paediatrics and Child Health, School of Clinical Medicine, College of Health Sciences, University of KwaZulu-Natal and Inkosi Albert Luthuli Central Hospital, Durban, South Africa

J Joubert, Department of Haematology and Cell Biology, Faculty of Health Sciences, University of the Free State, Bloemfontein, South Africa

V Louw, Division of Clinical Haematology, Department of Medicine, Faculty of Health Sciences, University of Cape Town and Groote Schuur Hospital, Cape Town, South Africa

N Mahlachana, Department of Paediatrics and Child Health, Faculty of Health Sciences, University of the Witwatersrand and Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa

R Mathew, Department of Medicine, Frere Hospital, East London, South Africa

B Neethling, Department of Paediatrics and Child Health, School of Clinical Medicine, College of Health Sciences, University of KwaZulu-Natal, Durban, South Africa

J Potgieter, Department of Haematology, National Health Laboratory Service, Steve Biko Academic Hospital and School of Medicine, Faculty of Health Sciences, University of Pretoria, South Africa

N Rapiti, Department of Haematology, National Health Laboratory Service, King Edward Vlll Hospital and School of Clinical Medicine, College of Health Sciences, University of KwaZulu-Natal, Durban, South Africa

B Rayner, South African Haemophilia Foundation

D Reynders, Department of Paediatrics and Child Health, School of Medicine, Faculty of Health Sciences, University of Pretoria, South Africa

C Sutton, Department of Paediatrics and Child Health, Faculty of Health Sciences, University of Limpopo and Pietersburg and Mankweng hospitals, Limpopo, South Africa

A van Zyl, Department of Paediatrics and Child Health, Faculty of Health Sciences, Stellenbosch University and Tygerberg Academic Hospital, Cape Town, South Africa

Full Text

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Keywords

Prophylaxis; Haemophilia; Clotting factor deficiency; Novel therapies; Emicizumab; Evidence-based consensus; Policy

Cite this article

South African Medical Journal 2022;112(6):405-408.

Article History

Date submitted: 2022-05-31
Date published: 2022-05-31

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