Children and adolescents with severe haemophilia who do not receive prophylactic therapy are 50 times more likely to suffer intracranial hemorrhages than children who receive regular factor VIII or factor IX infusions.
An international cohort study of 1,515 children with severe haemophilia A or B from 33 treatment centres across 20 countries, identified 29 cases of intracranial hemorrhages over 8,038 patient years.
Spontaneous intracranial bleeds were not seen in any children on full prophylaxis.
Co-author Dr Chris Barnes, a consultant haematologist from the Royal Children’s Hospital in Melbourne, told the limbic the study confirmed the value of prophylaxis against a potentially ‘devastating complication’.
“This paper provides significant evidence that while we commence prophylaxis because of joint bleeds, the benefit is there in the reduction of intracranial hemorrhages.”
“Intracranial hemorrhages are the most devastating complication in terms of long- term consequences.”
The incidence of intracranial hemorrhage was 0.0033/100 patient years with full prophylaxis (2-3 times per week) compared to 0.5/100 patient years with partial prophylaxis (1-2 times per week) and 1.7/100 patient years with no prophylaxis (irregular or less than once per week).
Dr Barnes said Australian families affected by haemophilia were fortunate to have access to prophylaxis via a centralised system of treatment centres offering multidisciplinary care.
He said only a few Australian children had circumstances or comorbidities that prevented full prophylaxis.
“The incidence of haemophilia is similar across all cultural groups and we recognise that unfortunately, a significant proportion of children in developing countries receive inadequate health care.”
“If they are not getting treatment, they can expect to have recurrent, crippling joint bleeds and a relatively high incidence of intracranial hemorrhages.”
The study, which excluded haemorrhages during the neonatal period, found 69% of events occurred in the first five years of life in the ‘no prophylaxis’ group.
Long-term sequelae included intellectual and behavioral problems, paresis or other motor problems, and epilepsy.
The study concluded prophylaxis should start at an early age – ‘possibly before the onset of clinical bleeding’.