Research and practice in thrombosis and haemostasis · 2026
Outcomes of hemophilia A in children under primary and secondary prophylaxis: results from the HEMFIL Cohort Study.
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Cardoso Patrícia Santos Resende, Santana Márcio Antônio Portugal, Vaz Dos Santos Eder Luciano, da Silva Camila Beatriz Pereira Gomes, Renó Brisa Yuan, de Carvalho Leonardo Fontenelle, Rezende Suely Meireles
Аннотация
BACKGROUND: Early prophylaxis reduces the risk of joint damage in children with hemophilia A (CwHA). Long-term comparative data on bleeding outcomes and joint health in CwHA under primary prophylaxis (PP) and secondary prophylaxis (SP) remain limited.
METHODS: This prospective cohort study enrolled previously untreated or minimally treated (<5 exposure days) CwHA (factor VIII < 0.02 IU/mL). Patients were followed until 75 exposure days or inhibitor development. Annual bleeding rate (ABR) and annual joint bleeding rate (AjBR) were evaluated before and after prophylaxis initiation. Joint status was assessed once, at a median patient age of 8.4 years, using the Haemophilia Early Arthropathy Detection with Ultrasound and the Haemophilia Joint Health Score.
RESULTS: A total of 43 children were included (PP, n = 23; SP, n = 20). Children receiving PP were younger at diagnosis and prophylaxis initiation. Before prophylaxis initiation, PP was associated with higher median ABR (8.5 vs 5.3; P = 0.014) and lower AjBR (0.6 vs 2.0; P = .006) vs SP, respectively. After prophylaxis initiation, ABR declined in both groups, with a greater reduction in SP (P = .016), while AjBR was comparable between the groups. Multivariable analysis showed that earlier diagnosis of hemophilia A and younger age at first joint bleeding were associated with a more severe bleeding phenotype. Ultrasound of 252 joints showed mild synovial hypertrophy in 35.7%; bone damage was uncommon and observed exclusively in SP.
CONCLUSION: In this study, CwHA receiving PP appeared to have a more severe bleeding phenotype compared with those on SP. However, this requires confirmation in future studies. Long-term joint outcomes were generally mild.
METHODS: This prospective cohort study enrolled previously untreated or minimally treated (<5 exposure days) CwHA (factor VIII < 0.02 IU/mL). Patients were followed until 75 exposure days or inhibitor development. Annual bleeding rate (ABR) and annual joint bleeding rate (AjBR) were evaluated before and after prophylaxis initiation. Joint status was assessed once, at a median patient age of 8.4 years, using the Haemophilia Early Arthropathy Detection with Ultrasound and the Haemophilia Joint Health Score.
RESULTS: A total of 43 children were included (PP, n = 23; SP, n = 20). Children receiving PP were younger at diagnosis and prophylaxis initiation. Before prophylaxis initiation, PP was associated with higher median ABR (8.5 vs 5.3; P = 0.014) and lower AjBR (0.6 vs 2.0; P = .006) vs SP, respectively. After prophylaxis initiation, ABR declined in both groups, with a greater reduction in SP (P = .016), while AjBR was comparable between the groups. Multivariable analysis showed that earlier diagnosis of hemophilia A and younger age at first joint bleeding were associated with a more severe bleeding phenotype. Ultrasound of 252 joints showed mild synovial hypertrophy in 35.7%; bone damage was uncommon and observed exclusively in SP.
CONCLUSION: In this study, CwHA receiving PP appeared to have a more severe bleeding phenotype compared with those on SP. However, this requires confirmation in future studies. Long-term joint outcomes were generally mild.