Treatment Challenges of Acquired Thrombotic Thrombocytopenic Purpura in Pediatric Patients From a Low-Income Country

Abstract

This study presents a comprehensive analysis of two cases of acquired thrombotic thrombocytopenic purpura (aTTP) observed in pediatric patients from a low-income country. In the instances described, both patients underwent a treatment regimen involving plasma exchange and immunosuppressive therapy conducted without the use of caplacizumab. Caplacizumab, an approved drug for adults known for its limited availability and high cost, has exhibited efficacy in reducing response time and recurrence frequency in aTTP cases. This approach resulted in significant clinical improvement and eventual remission of symptoms in one of the cases. These cases underscore the urgent necessity for a more inclusive approach in national health programs and international treatment guidelines. Specifically, there is a call to expand the existing comprehensive treatment algorithms to accommodate countries lacking access to caplacizumab. This adaptation aims to ensure the availability of suitable and effective treatment options for aTTP patients in regions facing limited pharmaceutical accessibility.