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Post-transplant cyclophosphamide prevents graft-versus-host disease in Haploidentical stem-cell transplanted children with inborn errors of immunity: a single-center experience
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  • Aidé Tamara Staines Boone,
  • Guadalupe Gonzalez-Villareal,
  • Maria Teresa Pompa-Garza,
  • Teodoro Muñiz-Ronquillo,
  • Adriana Carolina Sandoval-Gonzalez,
  • David Muzquiz-Zermeño,
  • Marco Antonio Padilla-Castro,
  • Jorge Alberto García-Campos,
  • Luz Maria Sanchez-Sanchez,
  • Edna Venegas Montoya,
  • Saul Oswaldo Lugo Reyes
Aidé Tamara Staines Boone
Hospital Regional de Especialidades No 25
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Guadalupe Gonzalez-Villareal
Hospital Regional Monterrey
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Maria Teresa Pompa-Garza
Hospital Regional Monterrey
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Teodoro Muñiz-Ronquillo
Hospital Regional Monterrey
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Adriana Carolina Sandoval-Gonzalez
Hospital Regional Monterrey
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David Muzquiz-Zermeño
Hospital Regional Monterrey
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Marco Antonio Padilla-Castro
Hospital Regional Monterrey
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Jorge Alberto García-Campos
Hospital Regional Monterrey
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Luz Maria Sanchez-Sanchez
Hospital Regional Monterrey
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Edna Venegas Montoya
Hospital Regional Monterrey
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Saul Oswaldo Lugo Reyes
Instituto Nacional de Pediatria
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Abstract

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.