The low-dose prophylaxis (preventive treatment) regimen followed for haemophilia patients at the Haemophilia Treatment Centre (HTC), Aluva, will be presented at the 58th annual conference of the American Society of Haematology in December in San Diego.
Prophylaxis for haemophilia is globally accepted as a superior method of treatment, compared with on-demand treatment, under the care of persons with the rare disorder. Prophylaxis is rarely practised in low- and middle-income countries owing to unaffordability of clotting factor concentrates (CFCs), lack of awareness, absence of comprehensive home care infrastructure and misconceptions about the treatment.
The study paper was accepted by the American society to look into the methodology and outcome, even though the Malmo Protocol followed worldwide calls for higher dosage. The low dose, if proved valid, could turn out to be a major boon for patients in developing countries that find it difficult to ensure effective management of haemophilia.
Kerala has been offering CFCs free of cost for on-demand treatment. An initiative in this regard was taken recently at HTC with support from the District Panchayat, Ernakulam, and it was titled ‘Royal Medicine Project’. The objective of the initiative is to provide secondary prophylaxis free of cost to children with severe haemophilia (aged 5-15 years).
A clinical audit was done on 11 children at the centre for a year (six months retrospectively for on-demand treatment and six months prospectively for prophylactic treatment).
The variables taken as outcome measures were changes in bleed rates, haemophilia joint health score (HJHS), functional independence score in haemophilia (FISH), hospitalisation rates, and school absenteeism.
Financial aid
The results showed a marked improvement in ABR (annual bleed rate), joint health, school absenteeism, and performance at school. Last year, the district panchayat had provided Rs.45 lakh under the Royal Medicine Project. However, the panchayat could not finance the spillover project because of resource constraints.
N. Vijayakumar, medical officer in-charge of HTC, said it was found that low dose prophylaxis for severe haemophilia in a developing economy was feasible and had marked clinical benefits with greater well-being when compared with on-demand therapy. He also suggested that the project be extended to other districts.
