Immunotherapy With Ex Vivo–Expanded Donor-Derived Nk Cells After Haploidentical Hsct in Pediatric Patients With Aml: A Phase I Pilot Study Publisher



T Rostami TAHEREH ; M Ahmadvand MOHAMMAD ; B Chahardoli BAHRAM ; Mr Rostami Mohammadreza REZA ; M Azari MOJTABA ; M Azari MORTEZA ; A Kiumarsi AZADEH ; G Janbabaei GHASEM
Source: Current Research in Translational Medicine Published:2025

Abstract

Background: Post-transplant relapse remains a considerable challenge for achieving successful outcomes in pediatric patients with acute myeloid leukemia (AML) receiving haploidentical hematopoietic stem cell transplantation (HSCT). Adoptive immune cell therapy strategies utilizing highly purified donor-derived natural killer (NK) cells have been extensively explored in various transplantation settings, demonstrating promise in preventing disease recurrence, especially in pediatric AML patients. Methods: Five pediatric and adolescent patients with high-risk AML were included in this pilot study and received haploidentical HSCT. On day 7 post-HSCT, all the patients received a single infusion of interleukin (IL)-2 stimulated ex vivo-expanded haploidentical NK cells (1 × 106/kg CD56+ cells of patient body weight). Results: All the patients tolerated the administration of NK cells without any adverse events during or after the infusion. Relapse occurred in two patients, both within the first 100 days post-transplantation, while three patients remained alive and disease-free one year post-transplantation. Conclusion: This pilot study demonstrated that the activation, expansion, and infusion of NK cells from readily available haploidentical donors in pediatric and adolescent patients with high-risk AML after HSCT is safe and feasible. © 2025 Elsevier B.V., All rights reserved.