Outcome of Non-hematological Autoimmunity After Hematopoietic Cell Transplantation in Children with Primary Immunodeficiency.


Journal

Journal of clinical immunology
ISSN: 1573-2592
Titre abrégé: J Clin Immunol
Pays: Netherlands
ID NLM: 8102137

Informations de publication

Date de publication:
01 2021
Historique:
received: 23 08 2020
accepted: 12 10 2020
pubmed: 4 11 2020
medline: 15 1 2022
entrez: 3 11 2020
Statut: ppublish

Résumé

Knowledge of post-hematopoietic cell transplantation (HCT) non-hematological autoimmune disease (AD) is far from satisfactory. This multicenter retrospective study focuses on incidence, risk factors, and outcomes of post-HCT AD in 596 children with primary immunodeficiency (PID) who were transplanted from 2009 to 2018. The indications of HCT were severe combined immunodeficiency (SCID, n = 158, 27%) and non-SCID PID (n = 438, 73%). The median age at HCT was 2.3 years (range, 0.04 to 18.3 years). The 5-year overall survival for the entire cohort was 79% (95% cumulative incidence (CIN), 74-83%). The median follow-up of surviving patients was 4.3 years (0.08 to 14.7 years). The CIN of post-HCT AD was 3% (2-5%) at 1 year post-HCT, 7% (5-11%) at 5 years post-HCT, and 11% (7-17%) at 8 years post-HCT. The median onset of post-HCT AD was 2.2 years (0.12 to 9.6 years). Autoimmune thyroid disorder (n = 19, 62%) was the most common post-HCT AD, followed by neuromuscular disorders (n = 7, 22%) and rheumatological manifestations (n = 5, 16%). All patients but one required treatment for post-HCT AD. After multivariate analysis, age at transplant (p = 0.01) and T cell-depleted graft (p < 0.001) were significant predictors of post-HCT AD. None of the T cell-depleted graft recipients developed post-HCT AD. Patients with a lower CD3+ count at 6 months post-HCT had a significant higher incidence of post-HCT AD compared to disease controls. Graft-versus-host disease, viral infection, and donor chimerism had no association with post-HCT AD. Post-HCT AD occurred in 11% at 8 years post-HCT and its occurrence was associated with older age at HCT and unmanipulated graft.

Identifiants

pubmed: 33141919
doi: 10.1007/s10875-020-00895-3
pii: 10.1007/s10875-020-00895-3
doi:

Types de publication

Journal Article Multicenter Study

Langues

eng

Sous-ensembles de citation

IM

Pagination

171-184

Subventions

Organisme : Department of Health
ID : RP-2014-05-007
Pays : United Kingdom

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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, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK. s.lum@nhs.net.
Willem-Alexander Children's Hospital, Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands. s.lum@nhs.net.

Reem Elfeky (R)

Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Federica R Achini (FR)

Willem-Alexander Children's Hospital, Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.
Division of Pediatric Stem Cell Transplantation and Children's Research Center (CRC), University Children's Hospital of Zurich, Zurich, Switzerland.

Adriana Margarit-Soler (A)

Department of Blood and Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Bianca Cinicola (B)

Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Inigo Perez-Heras (I)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.

Zohreh Nademi (Z)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.

Terry Flood (T)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.

Tim Cheetham (T)

Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Department of Paediatric Endocrinology, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, UK.

Austen Worth (A)

Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Waseem Qasim (W)

Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Rakesh Amin (R)

Department of Paediatric Endocrinology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Kanchan Rao (K)

Department of Blood and Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Robert Chiesa (R)

Department of Blood and Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Robbert G M Bredius (RGM)

Willem-Alexander Children's Hospital, Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.

Persis Amrolia (P)

Department of Blood and Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Mario Abinun (M)

Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.

Sophie Hambleton (S)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.

Paul Veys (P)

Department of Blood and Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Andrew R Gennery (AR)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.

Arjan Lankester (A)

Willem-Alexander Children's Hospital, Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.

Mary Slatter (M)

Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.
Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.

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