Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation for Children with High-Risk Neuroblastoma Using a Reduced-Intensity Conditioning Regimen: Results from the AIEOP Trial.

Autor: Prete A; Pediatric Hematology and Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy., Lanino E; Hematopoietic Stem Cell Transplantation Unit, IRCSS Istituto Giannina Gaslini, Genoa, Italy., Saglio F; Pediatric Oncohematology, Stem Cell Transplantation and Cell Therapy Division, AOU Città della Salute e della Scienza-Regina Margherita Children's Hospital, Turin, Italy., Biffi A; Pediatric Hematology, Oncology and Stem Cell Transplant Division, University-Hospital of Padua, Padua, Italy., Calore E; Pediatric Hematology, Oncology and Stem Cell Transplant Division, University-Hospital of Padua, Padua, Italy., Faraci M; Hematopoietic Stem Cell Transplantation Unit, IRCSS Istituto Giannina Gaslini, Genoa, Italy., Rondelli R; Pediatric Hematology and Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy., Favre C; Department of Pediatric Hematology/Oncology and Hematopoietic Stem Cell Transplantation, Meyer Children's University Hospital, Florence, Italy., Zecca M; Department of Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy., Casazza G; Pediatric Oncohematology, Azienda Ospedaliero Universitaria Pisana, Pisa, Italy., Porta F; Pediatric Oncohematology and Bone Marrow Transplant Unit, Children's Hospital, ASST Spedali Civili of Brescia, Brescia, Italy., Luksch R; Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy., Cesaro S; Pediatric Hematology Oncology Unit, Department of Mother and Child, Azienda Ospedaliera Universitaria Integrata, Verona, Italy., Rabusin M; Department of Pediatrics, Institute of Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy., Parasole R; Department of Pediatric Hemato-Oncology and Cellular Therapy, Azienda Sanitaria di Rilievo Nazionale Santobono-Pausilipon, Napoli, Italy., Mura RM; Pediatric Oncology Unit, Azienda Ospedaliera Brotzu, Cagliari, Italy., Lo Nigro L; Regional Reference Center for Pediatric Hematology and Oncology, Azienda Policlinico 'G. Rodolico-San Marco', Catania, Italy., Leardini D; Pediatric Hematology and Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy. Electronic address: davide.leardini3@studio.unibo.it., Pagliara D; Department of Pediatric Hematology and Oncology, Bambino Gesù Children's Hospital, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, Italy., Locatelli F; Department of Pediatric Hematology and Oncology, Bambino Gesù Children's Hospital, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, Italy; Catholic University of the Sacred Heart, Rome, Italy., Fagioli F; Pediatric Oncohematology, Stem Cell Transplantation and Cell Therapy Division, AOU Città della Salute e della Scienza-Regina Margherita Children's Hospital, Turin, Italy; University of Turin, Turin, Italy.
Jazyk: angličtina
Zdroj: Transplantation and cellular therapy [Transplant Cell Ther] 2024 May; Vol. 30 (5), pp. 530.e1-530.e8. Date of Electronic Publication: 2024 Mar 07.
DOI: 10.1016/j.jtct.2024.03.002
Abstrakt: Despite aggressive multimodal treatment, the outcomes of pediatric patients with high-risk (HR) neuroblastoma (NB) remain poor. The rationale for allogeneic hematopoietic stem cell transplantation (allo-HCT) to treat NB was based on the possible graft-versus-tumor effect; however, toxicity limits its efficacy. We sought to prospectively assess the feasibility and efficacy of allo-HCT using a reduced-intensity conditioning regimen in pediatric patients with HR NB in a multicenter phase II trial. Primary endpoints were the rate of neutrophil and platelet engraftment, 5-year transplantation-related mortality (TRM), and disease-free survival (DFS). Secondary endpoint measures included the incidence of acute graft-versus-host disease (aGVHD) and chronic GVHD. Fifty-one patients were enrolled in the study. The 5-year cumulative incidence (CuI) of TRM was 29.4 ± 6.4%, and that of DFS was 11.8 ± 4.5%. Patients undergoing allo-HCT within 1 year of diagnosis or with bone marrow as their stem cell source had a higher DFS probability. The CuI of neutrophil engraftment, platelet engraftment, and grade II-IV aGVHD was 97.9 ± 2.1%, 93.8 ± 3.5%, and 47.1 ± 7.0%, respectively. The development of new therapeutic strategies could further improve disease control.
(Copyright © 2024. Published by Elsevier Inc.)
Databáze: MEDLINE
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