Outcomes of Pediatric Patients With Steroid-Refractory Acute Graft Vs Host Disease

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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.


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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.

Read more of Cancer Therapy Advisor’s coverage of Tandem Meetings 2022 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. Tandem Meetings 2022; April 23-26, 2022. Abstract 373.

This article originally appeared on Hematology Advisor



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