Post-transplant cyclophosphamide prevents graft-versus-host disease in
Haploidentical stem-cell transplanted children with inborn errors of
immunity: a single-center experience
INTRODUCTION: For many patients with Primary immune deficiency (PID),
stem-cell transplantation (SCT) may be lifesaving. 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, 31.2 months. Seven
grafts were umbilical cord and 12 haploidentical. The conditioning
regime was myeloablative, with seven 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.