PMID- 37738655
OWN - NLM
STAT- MEDLINE
DCOM- 20240119
LR  - 20240324
IS  - 1528-0020 (Electronic)
IS  - 0006-4971 (Linking)
VI  - 143
IP  - 3
DP  - 2024 Jan 18
TI  - TCRalphabeta/CD19 cell-depleted HLA-haploidentical transplantation to treat pediatric 
      acute leukemia: updated final analysis.
PG  - 279-289
LID - 10.1182/blood.2023021336 [doi]
AB  - TCRalphabeta/CD19 cell depletion is a promising graft manipulation technique frequently 
      used in the context of human leukocyte antigen (HLA)-haploidentical hematopoietic 
      stem cell transplantation (HSCT). We previously reported the results of a phase 
      I-II clinical trial (NCT01810120) to assess the safety and the efficacy of this 
      type of exvivo T-cell depletion in 80 children with acute leukemia, showing 
      promising survival outcomes. We now report an updated analysis on a cohort of 213 
      children with a longer follow-up (median, 47.6 months for surviving patients). 
      With a 5-year cumulative incidence of nonrelapse mortality of 5.2% (95% 
      confidence interval [CI], 2.8%-8.8%) and a cumulative incidence of relapse of 
      22.7% (95% CI, 16.9%-29.2%), projected 10-year overall and disease-free survival 
      (DFS) were 75.4% (95% CI, 68.6%-80.9%) and 71.6% (95% CI, 64.4%-77.6%), 
      respectively. Cumulative incidence of both grade II-IV acute and chronic 
      graft-versus-host disease were low (14.7% and 8.1%, respectively). In a 
      multivariable analysis for DFS including type of disease, use of total body 
      irradiation in the conditioning regimen (hazard ratio [HR], 0.5; 95% CI, 
      0.26-0.98; P = .04), disease status at HSCT (complete remission [CR] >/=3 vs CR 
      1/2; HR, 2.23; 95% CI, 1.20-4.16; P = .01), and high levels of pre-HSCT minimal 
      residual disease (HR, 2.09; 95% CI, 1.01-4.33; P = .04) were independently 
      associated with outcome. In summary, besides confirming the good outcome results 
      already reported (which are almost superimposable on those of transplant from 
      HLA-matched donors), this clinical update allows the identification of patients 
      at higher risk of treatment failure for whom personalized approaches, aimed at 
      reducing the risk of relapse, are warranted.
CI  - (c) 2024 American Society of Hematology. Published by Elsevier Inc. All rights are 
      reserved, including those for text and data mining, AI training, and similar 
      technologies.
FAU - Merli, Pietro
AU  - Merli P
AUID- ORCID: 0000-0001-6426-4046
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Algeri, Mattia
AU  - Algeri M
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Galaverna, Federica
AU  - Galaverna F
AUID- ORCID: 0000-0003-0717-2924
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Bertaina, Valentina
AU  - Bertaina V
AUID- ORCID: 0000-0002-9028-7767
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Lucarelli, Barbarella
AU  - Lucarelli B
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Boccieri, Emilia
AU  - Boccieri E
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Becilli, Marco
AU  - Becilli M
AUID- ORCID: 0000-0002-8874-4092
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Quagliarella, Francesco
AU  - Quagliarella F
AUID- ORCID: 0000-0002-7346-2738
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Rosignoli, Chiara
AU  - Rosignoli C
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Biagini, Simone
AU  - Biagini S
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Girolami, Elia
AU  - Girolami E
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Meschini, Antonella
AU  - Meschini A
AD  - Transfusion Unit, Department of Laboratories, Istituto di Ricovero e Cura a 
      Carattere Scientifico Bambino Gesu Children's Hospital, Rome, Italy.
FAU - Del Principe, Giovanna
AU  - Del Principe G
AD  - Transfusion Unit, Department of Laboratories, Istituto di Ricovero e Cura a 
      Carattere Scientifico Bambino Gesu Children's Hospital, Rome, Italy.
FAU - Sborgia, Raffaella
AU  - Sborgia R
AUID- ORCID: 0000-0002-7428-7302
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Catanoso, Maria Luigia
AU  - Catanoso ML
AUID- ORCID: 0000-0003-1321-7498
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Carta, Roberto
AU  - Carta R
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Strocchio, Luisa
AU  - Strocchio L
AUID- ORCID: 0000-0002-5993-0857
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Pinto, Rita Maria
AU  - Pinto RM
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Buldini, Barbara
AU  - Buldini B
AD  - Pediatric Hematology, Oncology and Stem Cell Transplant Division, Maternal and 
      Child Health Department, University of Padua, Padua, Italy.
FAU - Falco, Michela
AU  - Falco M
AD  - Laboratory of Clinical and Experimental Immunology, IRCCS Istituto Giannina 
      Gaslini, Genoa, Italy.
FAU - Meazza, Raffaella
AU  - Meazza R
AUID- ORCID: 0000-0003-0242-3157
AD  - Laboratory of Pathology and Experimental Immunology, IRCCS Ospedale Policlinico 
      San Martino, Genoa, Italy.
FAU - Pende, Daniela
AU  - Pende D
AUID- ORCID: 0000-0003-1565-451X
AD  - Laboratory of Pathology and Experimental Immunology, IRCCS Ospedale Policlinico 
      San Martino, Genoa, Italy.
FAU - Andreani, Marco
AU  - Andreani M
AUID- ORCID: 0000-0003-3451-3624
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Li Pira, Giuseppina
AU  - Li Pira G
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Pagliara, Daria
AU  - Pagliara D
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Locatelli, Franco
AU  - Locatelli F
AUID- ORCID: 0000-0002-7976-3654
AD  - Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, Istituto di 
      Ricovero e Cura a Carattere Scientifico Bambino Gesu Children's Hospital, Rome, 
      Italy.
AD  - Department of Health Science and Public Health, Catholic University of the Sacred 
      Heart, Rome, Italy.
LA  - eng
PT  - Journal Article
PL  - United States
TA  - Blood
JT  - Blood
JID - 7603509
RN  - 0 (Receptors, Antigen, T-Cell, alpha-beta)
RN  - 0 (HLA Antigens)
RN  - 0 (Histocompatibility Antigens Class II)
SB  - IM
CIN - Blood. 2024 Jan 18;143(3):193-195. PMID: 38236615
MH  - Child
MH  - Humans
MH  - Receptors, Antigen, T-Cell, alpha-beta
MH  - Transplantation, Haploidentical/adverse effects
MH  - *Graft vs Host Disease
MH  - *Leukemia, Myeloid, Acute
MH  - HLA Antigens
MH  - *Hematopoietic Stem Cell Transplantation/methods
MH  - Histocompatibility Antigens Class II
MH  - Recurrence
MH  - Transplantation Conditioning/methods
MH  - Retrospective Studies
EDAT- 2023/09/22 18:42
MHDA- 2024/01/19 06:42
CRDT- 2023/09/22 17:13
PHST- 2023/08/31 00:00 [accepted]
PHST- 2023/05/30 00:00 [received]
PHST- 2024/01/19 06:42 [medline]
PHST- 2023/09/22 18:42 [pubmed]
PHST- 2023/09/22 17:13 [entrez]
AID - S0006-4971(23)02324-8 [pii]
AID - 10.1182/blood.2023021336 [doi]
PST - ppublish
SO  - Blood. 2024 Jan 18;143(3):279-289. doi: 10.1182/blood.2023021336.