Work

Haploidentical Stem Cell Transplantation With TCR αβ+/CD19+ Depletion in Children With Nonmalignant Hematologic Disorders: Outcomes From a Referral Center in Peru

Year: 2024

Type: article

Abstract: • Haplo-HSCT has a survival rate of 33.70%. • Almost half of haplo-HSCT recipients (49.80%) experience graft failure. • Acute GvHD is observed in 25.60% of haplo-HSCT recipients. • More than half (59.40%) of haplo-HSCT recipients are affected by viral infections. • The high incidence of graft failure and infections may impact survival in haplo-HSCT. Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) with TCR αβ + /CD19 + cell depletion is a promising therapeutic alternative for children with nonmalignant hematologic disorders, especially in low-income countries where finding a compatible donor is challenging. The use of this transplantation approach for nonmalignant hematologic disorders has not been previously described in the Peruvian pediatric population. We present the outcomes of children under 19 with nonmalignant hematologic disorders who underwent haplo-HSCT with TCR αβ + /CD19 + cell depletion between 2018-2022 at a referral center in Lima, Peru. Survival probabilities and cumulative incidence functions were calculated using the Kaplan-Meier method. A total of 17 children aged between 1 to 18.6 years (median = 9.7 years) were included. The follow-up period ranged from 10 days to 66.20 months, with a median of 4.34 months. The probability of overall survival, event-free survival, and failure-free survival was 33.70%, 31.40%, and 68.8%, respectively. The incidence rate of graft failure was 49.80%, while the mortality rate not associated with graft failure was 18.8%. The incidence rate of acute graft-versus-host disease (GvHD) was 25.60%, and the incidence rate of viral infections was 59.40%. The high incidence rates of graft failure and viral infections suggest that these factors may negatively impact the survival of children with nonmalignant hematologic disorders who undergo haplo-HSCT with TCR αβ+/CD19+ cell depletion. Therefore, optimizing the current conditioning regimens and ensuring timely access to first, second, and third-line antivirals is crucial to improve the survival of these patients.

Source: Transplantation Proceedings

DOI: 10.1016/j.transproceed.2024.08.038

Authors: Jackeline Carol Rodríguez-Torres, Alejandra Pando-Caciano, Pauline E. Mamaní-Cajachagua, Sergio A. Murillo-Vizcarra

Institutions: Instituto Nacional de Salud del Niño-San Borja, Universidad Peruana Cayetano Heredia, Instituto Nacional de Salud del Niño, Hospital Nacional Edgardo Rebagliati Martins

Language: English

Topic: Hematopoietic Stem Cell Transplantation

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,120