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
2020
unpublished
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
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... ients 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. HIGHLIGHTS We describe the experience of our hospital, UMAE 25 IMSS in Monterrey NL, Mexico, of 11 years performing hematopoietic stem-cell transplantation (HSCT) in pediatric patients with primary immune deficiencies. We report the clinical features, origin, diagnoses, treatment and outcome of 19 patients with 8 PID diagnoses who underwent HSCT, with a 79% survival rate, 21% GVHD prevalence, 47% stable chimerism, and 58% of complete immune reconstitution. Post-transplant cyclophosphamide has been proven effective as anti-GVHD prophylaxis. Despite all our grafts coming from umbilical cord and haploidentical donors, we were able to prevent GVHD by using post-transplant cyclophosphamide. In Mexico and Latin America, we need better local and regional donor registries. We can achieve encouraging positive results despite resource scarcity and other regional limitations. INTRODUCTION:
doi:10.22541/au.160218202.26794163/v1
fatcat:t44vs3tq2bedbfvibuobk7vmvy
