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.”

Read more of Hematology Advisor’s coverage of the 2022 Tandem Meetings by visiting the conference page.

Reference

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.