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Primary prophylaxis was associated with lower arthropathy in Colombian men with haemophilia B: A longitudinal
Juliana Alexandra Hernández Vargas1, Adriana Linares2,3, María Helena Solano4
1Cuenta de Alto Costo, Fondo Colombiano de Enfermedades de Alto Costo, Bogotá, Colombia.
Insights
Primary prophylaxis (PPr) significantly reduced chronic haemophilic arthropathy (CHA) by 89.70% in Colombian males with haemophilia B (HB). This supports PPr as a key strategy for managing HB and preventing joint damage.
Area of Science:
- Hematology
- Orthopedics
- Public Health
Background:
- Chronic haemophilic arthropathy (CHA) is a significant complication of haemophilia B (HB), impacting quality of life.
- The severity of HB is linked to the risk of developing CHA.
- Primary prophylaxis (PPr) is a potential strategy to mitigate CHA incidence and its effects.
Purpose of the Study:
- To assess the association between PPr and CHA in Colombian males diagnosed with haemophilia B.
- To analyze data from 2015 to 2019 to understand the impact of prophylaxis strategies on CHA.
Main Methods:
- A nationwide open cohort study of individuals with congenital coagulopathies was analyzed.
- Panel-time analysis and logistic random-effect regression models (LRERM) were employed.
- Adjustments were made for age at diagnosis, prophylaxis dose/frequency, severity, haemarthrosis, and high-titre inhibitors.
Main Results:
- A cohort of 362 men with HB was analyzed; 36.84% had baseline CHA.
- PPr was administered to 37.85% of patients.
- PPr was associated with an 89.70% reduction in the odds of CHA (aOR = 0.103, P < .001) compared to secondary/tertiary prophylaxis (STPr).
Conclusions:
- Primary prophylaxis significantly reduces the odds of developing chronic haemophilic arthropathy in males with haemophilia B.
- These findings support the implementation of PPr as a standard treatment strategy for haemophilia B patients in Colombia.
- The study reinforces the efficacy of PPr in preventing joint disease progression in haemophilia B.
Introduction:
The risk of chronic haemophilic arthropathy (CHA) is related to severity. Evidence suggests that primary prophylaxis (PPr) could reduce CHA incidence and its impact on quality of life.
Aim:
To evaluate the association between PPr and CHA in Colombian males with haemophilia B (HB) during 2015 to 2019.
Methods:
A panel-time analysis was performed with data provided by the National Health System to update a nationwide open cohort of people with congenital coagulopathies. The association was evaluated in a logistic random-effect regression model (LRERM), adjusted by age at diagnosis, prophylaxis dose and frequency, severity, haemarthrosis and high-titre inhibitors.
Results:
During 2015-2019, a total of 362 men with HB and treated with either, primary, secondary or tertiary prophylaxis were identified. At baseline, CHA prevalence in the cohort was 36.84% (n = 133), median age was 19.0 years (IQR: 10.0-27.0), and median age at diagnosis was 1.0 year (IQR: 0.0-4.0). PPr was prescribed in 37.85% (n = 137), and median dose (IU/Kg/dose) was almost the same for primary vs. secondary/tertiary prophylaxis. Patients in PPr had a lower frequency of severe HB, CHA, haemarthrosis, infectious complications and high-titre inhibitors than those in secondary or tertiary prophylaxis (STPr). In the LRERM, PPr was associated with a significant reduction of 89.70% in the odds of CHA (aOR = 0.103, IC 95%: 0.040, 0.270; P < .001), compared with STPr.
Conclusions:
PPr decreased the odds of CHA by 89.70% in males with HB in Colombia. Our findings are consistent with previous studies and support the strategy to prescribe PPr to our patients.
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