Personalized Prophylaxis in Hemophilia a: A Pediatric Center Experience in Mexico

Abstract

Summary. Prophylaxis is the current standard of care for Hemophilia patients. However, the regimen should be individualized as much as possible based on age, bleeding phenotype and physical activity. PK based regimens have been proposed as an effective way to achieve more successful outcomes. According to 2016 WFH data, Mexico has 1.425 IU/capita mean use, which barely overcomes the limit of 1IU/ minimum target to achieve survival of the hemophilia population (WFH Report on the Annual Global Survey). Objectives. To examine a Mexican Center experience obtaining limited sampling PK profiles in pediatric patients. To determine the difference in frequency dosification in standard prophylaxis vs PK guided. Methods. Limited sampling was done in 14 pediatric patients with Hemophilia A without inhibitors, moderate to severe, in prophylaxis aged 2 to 14 years old using population PK tool myPKFiT. PK profiles based on the same data points plus patient characteristics were generated by a Bayesian algorithm with myPKFiT. Results. The mean age was 5.5 years (range 1-14 years) with a mean standard dose of 35 IU/k 2 times a week. The PK based mean dose was 27IU/kg 4 times a week. The mean half-life was 9.5 hours (range 6.8-11.6 hours. Conclusions. Shorter half life should be expected in pediatric patients, and be taken into account with other variables to achieve a personalized prophylaxis. Tailored prophylaxis facilitate patient education and adherence. Pediatric PK suggest shorter intervals or higher dosing are needed to achieve a desired target level. Use of myPKFiT and clinical, radiological and adherence scores to improve outcomes, in particular in the pediatric population. Integrating PK data into routine hemophilia care could provide promising results in developing countries. DisclosuresNo relevant conflicts of interest to declare.