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

Autor:	Nava Gutiérrez W; Hematology and Oncology, Universidad de Monterrey, Monterrey, MEX.; Hematology and Oncology, Instituto Mexicano Del Seguro Social, Monterrey, MEX., Garza-Escobar JD; Hematology and Oncology, Instituto Mexicano Del Seguro Social, Monterrey, MEX., Sandoval-González AC; Pediatric Hematology, Instituto Mexicano Del Seguro Social, Monterrey, MEX., Alonso-Tellez CA; Research, Instituto Mexicano Del Seguro Social, Monterrey, MEX.
Jazyk:	angličtina
Zdroj:	Cureus [Cureus] 2023 Sep 19; Vol. 15 (9), pp. e45540. Date of Electronic Publication: 2023 Sep 19 (Print Publication: 2023).
DOI:	10.7759/cureus.45540
Abstrakt:	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. Competing Interests: The authors have declared that no competing interests exist. (Copyright © 2023, Nava Gutiérrez et al.)
Databáze:	MEDLINE
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