Immunotherapy with ex vivo–expanded donor-derived NK cells after haploidentical HSCT in pediatric patients with AML: a phase I pilot study - 28/08/25
, Mohammad Ahmadvand b, Bahram Chahardoli b, Mohammad Reza Rostami c, Mojtaba Azari a, Morteza Azari a, Azadeh Kiumarsi d, ⁎
, Ghasem Janbabaei aHighlights |
• | NK cell therapy has shown potential as a relapse-prevention strategy in high-risk patients with AML undergoing haploidentical HSCT. |
• | No infusion-related toxicities were observed in our study, supporting the safety of adoptive NK cell therapy after haploidentical HSCT in pediatric patients with AML. |
• | The results of our study support further trials to optimize timing, dosing, and donor selection (e.g., KIR-HLA mismatch analysis) for NK cell therapy after haploidentical HSCT in high-risk pediatric AML. |
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 × 10 6 /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.
Le texte complet de cet article est disponible en PDF.Keywords : Haploidentical hematopoietic stem cell transplantation, Acute myeloid leukemia, Natural killer cell therapy, Pediatrics, Relapse
Plan
Vol 73 - N° 4
Article 103534- décembre 2025 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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