Prevention, Identification, Management and Long-Term Complications of Intracranial Haemorrhage in Children With Haemophilia and Other Severe Bleeding Disorders-A Practical Guidance.

INTRODUCTION

Intracranial haemorrhage (ICH) is the most serious complication in infants and children with severe congenital bleeding disorders causing substantial mortality and long-term neurological morbidity. The neonatal period carries the highest risk, particularly in severe haemophilia and rare factor deficiencies. We review the epidemiology, risk factors, prevention, recognition, management, and long-term outcomes of ICH in children with congenital bleeding disorders.

METHODS

Current evidence from cohort studies, literature reviews, and recent clinical data addressing ICH in childhood is summarized, with attention to evolving prophylactic strategies.

RESULTS

Neonatal ICH occurs in 2%-4% of infants with haemophilia and more frequently in severe FXIII and FX deficiency. Unrecognized bleeding disorders and traumatic delivery-especially with forceps or vacuum-substantially increase risk. Immediate factor replacement prior to diagnostic imaging improves survival and reduces neurological sequelae. After the neonatal period, risk remains elevated, particularly in children <1 year not receiving prophylaxis. Early initiation of prophylaxis, historically limited by challenges with intravenous access, markedly reduces ICH incidence. Emerging non-factor therapies (FVIII mimetics and rebalancing agents) enable earlier and more effective protection. Despite advances in care, 30%-40% of survivors experience long-term neurological impairments with outcomes influenced by bleed location, cerebral shift, age at time of ICH, and treatment delays.

CONCLUSIONS

ICH remains a major threat to children with severe bleeding disorders. Prevention requires early identification of at-risk pregnancies, careful delivery planning, and timely diagnosis of bleeding disorders. Advances in prophylactic therapy offer the potential to significantly reduce ICH incidence and improve long-term outcomes.