Hematopoietic Stem Cell Transplantation in Children with Inborn Errors of Immunity: a Multi-center Experience in Colombia

Abstract

Purpose

To characterize the pediatric population with inborn errors of immunity (IEI) that was treated with hematopoietic stem cell transplantation (HSCT) in three reference centers in Colombia. What have been the characteristics and outcomes of hematopoietic stem cell transplantation in pediatric patients with inborn errors of immunity in three reference care centers in Colombia between 2007 and 2018?

Methods

We conducted an observational, retrospective cohort study in children with a diagnosis of IEI who underwent HSCT between 2007 and 2018.

Results

Forty-seven patients were identified, and 5 were re-transplanted. Sixty-eight percent were male. The median age at diagnosis was 0.6 years, and for HSCT was 1.4 years. The most common diseases were chronic granulomatous disease (38%) followed by severe combined immune deficiencies (19%) and hemophagocytic lymphohistiocytosis (15%). Cord blood donors were the most used source of HSCT (44%). T cell–replete grafts from haploidentical donors using post-transplantation cyclophosphamide represent 37% of the cohort. All patients received conditioning, 62% with a non-myeloablative regimen. Calcineurin inhibitors were the main graft-versus-host disease prophylaxis (63.8%). Acute graft-versus-host disease developed in 35% of the total patients. The most frequent post-transplant infections were viral and fungal infections. The 1-year overall survival rates for the patients who received HSCT from identical, haploidentical, and cord sources were 80%, 72%, and 63%, respectively. The 5-year overall survival was 63%.

Conclusions

HSCT is a curative treatment option for some IEI and can be performed with any donor type. Early and timely treatment in referral centers can improve survival.

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Notes

  1. 1.

    Fundación Valle del Lili (Cali), Hospital Pablo Tobón Uribe (Medellín) and HOMI Fundación Hospital Pediátrico la Misericordia (Bogotá).

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Authors

Contributions

All authors contributed to the study conception and design. Material preparation, critical revision, data collection, and analysis were performed by Manuela Olaya, Alexis Franco, Mauricio Chaparro, Natalia Builes Restrepo, Jose L Franco, Marcela Estupiñan, David Aristizabal, Andrés F Zea-Vera, Jaime Patiño, Harry Pachajoa, Paola Perez, and Diego Medina Valencia. The first draft of the manuscript, quality control, and statistical analysis of data for the work were performed by Mayra Estacio, Eliana Manzi, and Estefania Beltran and all authors commented on previous versions of the manuscript. All authors listed in the title page have contributed significantly to the work. All authors read and approved the final manuscript version to be submitted and agreed to be accountable for all aspects of the work and to ensure that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

Corresponding author

Correspondence to Diego Medina-Valencia.

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The Institutional Review Board Ethics Committee of each center approved the study protocol

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The authors declare that they have no conflict of interest.

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Olaya, M., Franco, A., Chaparro, M. et al. Hematopoietic Stem Cell Transplantation in Children with Inborn Errors of Immunity: a Multi-center Experience in Colombia. J Clin Immunol (2020). https://doi.org/10.1007/s10875-020-00856-w

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Keywords

  • Hematopoietic stem cell transplantation
  • transplant recipients
  • primary immunodeficiency diseases
  • pediatrics
  • haploidentical transplantations
  • child