Risk and promise: an 11-year, single-center retrospective study of severe acute GVHD in pediatric patients undergoing allogeneic HSCT for nonmalignant diseases.

graft-vs.-host disease hematopoietic stem cell transplantation outcome pediatric nonmalignant diseases survival

Journal

Frontiers in pediatrics
ISSN: 2296-2360
Titre abrégé: Front Pediatr
Pays: Switzerland
ID NLM: 101615492

Informations de publication

Date de publication:
2023
Historique:
received: 27 03 2023
accepted: 19 04 2023
medline: 12 6 2023
pubmed: 12 6 2023
entrez: 12 6 2023
Statut: epublish

Résumé

Hematopoietic stem cell transplantation (HSCT) is the only curative option for many nonmalignant hematopoietic-derived diseases in pediatric patients. Survival after HSCT has improved in recent years and resulted in a 90% survival rate and cure in some nonmalignant diseases. Graft-vs.-host disease (GVHD) remains a frequent and major complication of HSCT, and a leading cause of morbidity and mortality. Prognosis of patients with high-grade GVHD is dismal, with survival rates varying from 25% in the adult population to 55% in pediatric patients. The main aim of this study is to evaluate the incidence, risk factors, and outcome of severe acute GVHD (AGVHD) in pediatric patients with nonmalignant diseases, following allogeneic HSCT. Clinical and transplant data were retrospectively collected for all pediatric patients who underwent allogeneic HSCT for nonmalignant diseases at the Hadassah Medical Center between 2008 and 2019. Patients who developed severe AGVHD were compared with those who did not. A total of 247 children with nonmalignant diseases underwent 266 allogeneic HSCTs at Hadassah University Hospital over an 11-year period. Seventy-two patients (29.1%) developed AGVHD, 35 of them (14.1%) severe AGVHD (grade 3-4). Significant risk factors for developing severe AGVHD were unrelated donor ( These results demonstrate a high survival rate in pediatric patients with nonmalignant diseases despite severe GVHD. Significant mortality risk factors found in these patients were the source of donor PBSC (

Sections du résumé

Background UNASSIGNED
Hematopoietic stem cell transplantation (HSCT) is the only curative option for many nonmalignant hematopoietic-derived diseases in pediatric patients. Survival after HSCT has improved in recent years and resulted in a 90% survival rate and cure in some nonmalignant diseases. Graft-vs.-host disease (GVHD) remains a frequent and major complication of HSCT, and a leading cause of morbidity and mortality. Prognosis of patients with high-grade GVHD is dismal, with survival rates varying from 25% in the adult population to 55% in pediatric patients.
Methods UNASSIGNED
The main aim of this study is to evaluate the incidence, risk factors, and outcome of severe acute GVHD (AGVHD) in pediatric patients with nonmalignant diseases, following allogeneic HSCT. Clinical and transplant data were retrospectively collected for all pediatric patients who underwent allogeneic HSCT for nonmalignant diseases at the Hadassah Medical Center between 2008 and 2019. Patients who developed severe AGVHD were compared with those who did not.
Results UNASSIGNED
A total of 247 children with nonmalignant diseases underwent 266 allogeneic HSCTs at Hadassah University Hospital over an 11-year period. Seventy-two patients (29.1%) developed AGVHD, 35 of them (14.1%) severe AGVHD (grade 3-4). Significant risk factors for developing severe AGVHD were unrelated donor (
Conclusions UNASSIGNED
These results demonstrate a high survival rate in pediatric patients with nonmalignant diseases despite severe GVHD. Significant mortality risk factors found in these patients were the source of donor PBSC (

Identifiants

pubmed: 37303759
doi: 10.3389/fped.2023.1194891
pmc: PMC10250657
doi:

Types de publication

Journal Article

Langues

eng

Pagination

1194891

Informations de copyright

© 2023 Zaidman, Even-Or, Aharoni, Averbuch, Dinur-Schejter, NaserEddin, Slae, Shadur and Stepensky.

Déclaration de conflit d'intérêts

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Références

N Engl J Med. 2017 Nov 30;377(22):2167-2179
pubmed: 29171820
Bone Marrow Transplant. 2013 Oct;48(10):1359-61
pubmed: 23604011
Biol Blood Marrow Transplant. 2009 May;15(5):639-42
pubmed: 19361757
Br J Haematol. 2011 Jul;154(1):143-6
pubmed: 21477160
Biol Blood Marrow Transplant. 2016 May;22(5):910-8
pubmed: 26748160
J Adv Res. 2015 May;6(3):449-58
pubmed: 26257943
Biol Blood Marrow Transplant. 2017 Mar;23(3):522-528
pubmed: 28017733
Blood. 1990 Feb 15;75(4):1024-30
pubmed: 2302454
Medicine (Baltimore). 2016 Nov;95(44):e5242
pubmed: 27858879
Front Immunol. 2017 Mar 21;8:328
pubmed: 28377772
Haematologica. 2020 Sep 17;105(11):2550-2560
pubmed: 33054103
Blood. 1990 Oct 15;76(8):1464-72
pubmed: 2207321
Biol Blood Marrow Transplant. 2020 Aug;26(8):e177-e182
pubmed: 32438042
Stem Cells Transl Med. 2013 Jan;2(1):25-32
pubmed: 23283494
Biol Blood Marrow Transplant. 2012 Aug;18(8):1150-63
pubmed: 22510384
Bone Marrow Transplant. 1995 Jun;15(6):825-8
pubmed: 7581076
Anticancer Res. 2017 Apr;37(4):1547-1555
pubmed: 28373413
Biol Blood Marrow Transplant. 2003 Apr;9(4):215-33
pubmed: 12720215
Bone Marrow Transplant. 2014 Oct;49(10):1251-8
pubmed: 24887389
Int J Hematol. 2020 Jun;111(6):869-876
pubmed: 32052319
Bone Marrow Transplant. 2011 Jan;46(1):143-7
pubmed: 20348971
Front Immunol. 2017 Mar 20;8:79
pubmed: 28373870
Med Clin (Barc). 2019 Jan 4;152(1):22-28
pubmed: 30309668

Auteurs

Irina Zaidman (I)

Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Ehud Even-Or (E)

Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Elroee Aharoni (E)

Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.

Dina Averbuch (D)

Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Department of Pediatrics, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Yael Dinur-Schejter (Y)

Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Adeeb NaserEddin (A)

Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Mordechai Slae (M)

Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Department of Pediatrics, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Bella Shadur (B)

Immunology Division, Garvan Institute of Medical Research, Sydney, NSW, Australia.
St Vincent's Clinical School, University of New South Wales, Sydney, NSW, Australia.

Polina Stepensky (P)

Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Classifications MeSH