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

Abstract

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.

Keywords: Mexico; clinical features; conditioning regime; haematopoietic stem-cell transplantation; outcome; paediatric patients; post-transplant cyclophosphamide; primary immune deficiencies; single centre experience; survival.