Steroid-refractory acute graft vs host disease (SR-aGVHD) is associated with high mortality rates among pediatric patients who had undergone an allogeneic hematopoietic stem cell transplant (allo-HSCT), despite second-line treatment, according to results of a retrospective study presented at the 2022 Tandem Meetings.

Morbidity and mortality are known to be associated with pediatric SR-aGVHD. The aim of this study was to evaluate the outcomes of pediatric patients who develop SR-aGVHD during a 10-year time period.

The retrospective, nationwide cohort study evaluated the data from 786 pediatric patients who had undergone an allo-HSCT between 2010 and 2020 at 2 centers in the Netherlands. Of these patients, 20% developed grade II-IV aGVHD.

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There were 49% of patients who developed steroid-refractory disease of the patients with aGVHD, with 16% considered grade II, 56% grade III, and 28% grade IV. Second-line treatment was initiated within a median of 8 days after diagnosis of aGVHD, with the most common therapies used including 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.

An increased risk of death among patients with SR-aGVHD was associated with older age in a multivariate analysis (hazard ratio [HR], 1.07; 95% CI, 1.01-1.13; P =.021). The type of underlying disease or the use of cord blood versus bone marrow as a source were not associated with survival.

Better SR-aGVHD outcomes was significantly associated with younger age at a median of less than 8.9 years. Younger patients were more likely to experience remission at 1 year at 55% compared with 23% of older patients. Mortality rates among younger patients were almost half that of older patients, with a rate of 28% compared with 54%, respectively.

The authors concluded that “this cohort twill be the reference for the next 10 years in which newer monoclonal and small-molecule therapies will have entered the guidelines.”

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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. Presented at 2022 Tandem Meetings; April 23-26, 2022. Abstract 373.