Clinical Characteristics, Treatment Response, and Outcomes of Bronchiolitis Obliterans Syndrome in Children with Hematologic Malignancies Undergoing Haploidentical Hematopoietic Cell Transplantation with Granulocyte Colony-Stimulating Factor/Antithymocyte Globulin-Based Protocol: A Single-Center Study.
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Limited data are available on bronchiolitis obliterans syndrome (BOS) in children undergoing haploidentical hematopoietic cell transplantation (haplo-HCT). To describe the clinical characteristics, treatment response, and factors influencing first-line treatment response in BOS patients undergoing haplo-HCT using a granulocyte colony-stimulating factor/antithymocyte globulin-based protocol. We conducted a single-center retrospective study including 60 pediatric patients with BOS at Peking University People's Hospital between January 2016 and December 2022. BOS was diagnosed at a median of 12.85 (interquartile range: 10.52 to 16.82) mo after haplo-HCT, and patients were followed for a median of 28.4 (95% confidence interval [CI]: 22.9 to 37.8) mo from BOS diagnosis. Among the 52 evaluable patients with long-term follow-up, the overall response rate (ORR) for first-line treatment was 33%. For 15 evaluable patients receiving second-line treatment, the ORR was 13%, resulting in a total ORR of 46% across first-line and second-line therapy. Three significant independent predictors of first-line treatment response were identified: NIH lung score 3 at baseline (odds ratio [OR], 0.112; 95% CI, 0.014 to 0.866; P = .036), disease status of greater than or equal to second complete remission at haplo-HCT (OR, 0.034; 95% CI, 0.002 to 0.488; P = .013), and a duration of ≥12.85 mo from haplo-HCT to BOS diagnosis (OR, 9.890; 95% CI, 1.725 to 56.692; P = .010). Pulmonary function trajectories showed that patients with baseline NIH lung scores of 2 or 3 experienced a more pronounced decline in percent of predicted FEV1 after BOS diagnosis compared to those with a score of 1. These findings highlight the importance of early diagnosis of BOS and initiating treatment for patients with early stages of the disease.