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

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.

Stem-cell transplantation for children with primary immune deficiencies: A retrospective study of 19 patients from one centre in Mexico.

INTRODUCTION: For many patients with primary immune deficiency (PID), stem-cell transplantation (SCT) may be life-saving. OBJECTIVE: To review our experience of 11 years transplanting children with PID in Mexico. METHODS: Chart review of patients who underwent SCT from 2008 to 2018, to describe their diagnoses, time to transplant, conditioning regime, survival rate and outcomes. All patients received post-transplant cyclophosphamide as graft-versus-host-disease (GVHD) prophylaxis. RESULTS: 19 patients with combined, phagocytic or syndromic PID from 5 states. Twelve of them were male (58%) and 14 survive (79%). Mean age at HSCT was 41.9 months; mean time from diagnosis was 31.2 months. Seven grafts were umbilical cord and 12 haploidentical. The conditioning regime was myeloablative, with five primary graft failures. Two patients had partial and 10 full chimerism. Five patients died within 2 months after transplant. Immune reconstitution was complete in 11 of 19 patients. We found a prevalence of 21% GVHD. DISCUSSION: We describe 19 patients from Mexico with 8 PID diagnoses who underwent allogenic HSCT over a period of 11 years. Survival rate and other outcomes compare well with industrialized countries. We recommend the use of post-transplant cyclophosphamide to prevent GVHD in scenarios of resource scarcity and a lack of HLA-identical donors.