T-replete HLA-matched grafts vs T-depleted HLA-mismatched grafts in inborn errors of immunity.


Journal

Blood advances
ISSN: 2473-9537
Titre abrégé: Blood Adv
Pays: United States
ID NLM: 101698425

Informations de publication

Date de publication:
22 02 2022
Historique:
received: 18 12 2020
accepted: 07 11 2021
pubmed: 1 1 2022
medline: 27 4 2022
entrez: 31 12 2021
Statut: ppublish

Résumé

Hematopoietic cell transplantation (HCT) has become standard-of-care for an increasing number of inborn errors of immunity (IEI). This report is the first to compare transplant outcomes according to T-cell-replete (ie, T-replete) HLA-matched grafts using alemtuzumab (n = 117) and T-cell-depleted (ie, T-depleted) HLA-mismatched grafts using T-cell receptor-αβ (TCRαβ)/CD19 depletion (n = 47) in children with IEI who underwent first HCT between 2014 and 2019. All patients received treosulfan-based conditioning except patients with DNA repair disorders. For T-replete grafts, the stem cell source was marrow in 25 (21%) patients, peripheral blood stem cell (PBSC) in 85 (73%), and cord blood in 7 (6%). TCRαβ/CD19 depletion was performed on PBSCs from 45 haploidentical parental donors and 2 mismatched unrelated donors. The 3-year overall survival (OS) and event-free survival for the entire cohort were 85% (77%-90%) and 79% (69%-86%), respectively. Analysis according to age at transplant revealed a comparable 3-year OS between T-replete grafts (88%; 76%-94%) and T-depleted grafts (87%; 64%-96%) in younger patients (aged <5 years at HCT). For older patients (aged >5 years), the OS was significantly lower in T-depleted grafts (55%; 23%-78%) compared with T-replete grafts (87%; 68%-95%) (P = .03). Grade III to IV acute graft-versus-host disease was observed in 8% of T-replete marrow, 7% of T-replete PBSC, 14% of T-replete cord blood, and 2% of T-depleted PBSC (P = .73). Higher incidence of viremia (P < .001) and delayed CD3 reconstitution (P = .003) were observed after T-depleted graft HCT. These data indicate that mismatched donor transplant after TCRαβ/CD19 depletion represents an excellent alternative for younger children with IEI in need of an allograft.

Identifiants

pubmed: 34972212
pii: 483293
doi: 10.1182/bloodadvances.2020004072
pmc: PMC8864655
doi:

Substances chimiques

Antigens, CD19 0
Receptors, Antigen, T-Cell, alpha-beta 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

1319-1328

Subventions

Organisme : Wellcome Trust
ID : 204721/Z/16/Z
Pays : United Kingdom

Informations de copyright

© 2022 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.

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Auteurs

Su Han Lum (SH)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Sinéad Greener (S)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Inigo Perez-Heras (I)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Daniel Drozdov (D)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Rebecca P Payne (RP)

Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Helen Watson (H)

Blood Sciences.

Kay Carruthers (K)

Newcastle Advanced Therapies, and.

Robert January (R)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Zohreh Nademi (Z)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Stephen Owens (S)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Eleri Williams (E)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Sheila Waugh (S)

Microbiology and Virology Department, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom; and.

Shirelle Burton-Fanning (S)

Microbiology and Virology Department, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom; and.

Timmothy Ronan Leahy (TR)

Department of Paediatric Immunology, CHI at Crumlin, Dublin, Ireland.

Andrew Cant (A)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Mario Abinun (M)

Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Terry Flood (T)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Sophie Hambleton (S)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Andrew R Gennery (AR)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Mary Slatter (M)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

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