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

Frontiers in Pediatrics

Pediatrics

Adeeb NaserEddin¹  Yael Dinur-Schejter¹  Elroee Aharoni²  Ehud Even-Or³  Irina Zaidman³  Polina Stepensky³  Mordechai Slae⁴  Dina Averbuch⁴  Bella Shadur⁵ 

[1] Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel;Faculty of Medicine, Israel;Faculty of Medicine, Israel;Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel;Faculty of Medicine, Israel;Department of Pediatrics, Hadassah-Hebrew University Medical Center, Jerusalem, Israel;Immunology Division, Garvan Institute of Medical Research, Sydney, NSW, Australia;St Vincent’s Clinical School, University of New South Wales, Sydney, NSW, Australia;

关键词: hematopoietic stem cell transplantation;    survival;    graft-vs.-host disease;    pediatric nonmalignant diseases;    outcome;   

DOI  :  10.3389/fped.2023.1194891

 received in 2023-03-27, accepted in 2023-04-19,  发布年份 2023

来源: Frontiers

【 摘 要 】

BackgroundHematopoietic 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.MethodsThe 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.ResultsA 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 (p < 0.001), mismatch donor (p < 0.001), and the use of peripheral blood stem cells (PBSCs) (p < 0.001). Survival rates of pediatric patients with severe AGVHD was 71.4%, compared with 91.9% among those with mild (grade 1–2) AGVHD and 83.4% among patients without AGVHD (p = 0.067).ConclusionsThese 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 (p = 0.016) and poor response to steroid treatment (p = 0.007).

【 授权许可】

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