Steroid-refractory acute graft vs host disease (SR-aGVHD) is associated with high mortality rates in pediatric patients, despite second-line therapy, according to results of a retrospective study presented at the Tandem Meetings 2022.
Morbidity and mortality are known to be associated with pediatric SR-aGVHD. The aim of this study was to evaluate outcomes of pediatric patients who develop SR-aGVHD over a 10-year time period.
The retrospective, nationwide cohort study included data from 786 pediatric patients who had received an allogeneic hematopoietic stem cell transplant (allo-HSCT) between 2010 and 2020 at 2 centers in the Netherlands. Of these patients, 20% developed grade II-IV aGVHD.
Among the patients with aGVHD, 49% had SR-aGVHD, with 16% having grade II, 56% grade III, and 28% grade IV SR-aGVHD.
Second-line treatment was initiated within a median of 8 days after aGVHD diagnosis. The most common therapies used were mesenchymal stem cells (48%), infliximab (31%), and a combination of therapies (14%).
Resolution of SR-aGVHD occurred within 100 days or 1 year for 30% and 40% of patients, respectively, who received second-line therapy. The rate of mortality by day 100 was 26%, followed by 41% by 1 year and 44% after 2 years. Nearly one-third of deaths were related to unexplained or lung-GVHD-related respiratory insufficiency.
In a multivariate analysis, older age was associated with an increased risk of death in patients with SR-aGVHD (hazard ratio [HR], 1.07; 95% CI, 1.01-1.13; P =.021). The type of underlying disease or the use of cord blood vs bone marrow as a source were not associated with survival.
Better SR-aGVHD outcomes were significantly associated with younger age (a median of less than 8.9 years). Younger patients were more likely to experience remission at 1 year, at 55%, compared with 23% for older patients. Mortality rates among younger patients were almost half that of older patients, with a rate of 28% compared with 54%, respectively.
“This cohort will be the reference for the next 10 years [when] newer monoclonal and small-molecule therapies will have entered the guidelines,” the study authors concluded.
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Jansen SA, Verbeek AB, von Asmuth EGJ, et al. Age is a risk factor for mortality in pediatric steroid-refractory acute graft-versus-host disease: a multicenter study. Tandem Meetings 2022; April 23-26, 2022. Abstract 373.
This article originally appeared on Hematology Advisor