Researchers, led by Dominique Rongvaux-Gaïda, evaluated arsenic trioxide (ATO) plus corticosteroids (CS) as a first-line therapy in patients who developed chronic graft-versus-host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The team reported that the combination was associated with a high response rate and rapid decrease of CS use. The findings were published in Transplantation and Cellular Therapy.
The single-arm trial enrolled 21 patients across five university hospitals in France. ATO was administered within 10 days of initiating CS at 1 mg/kg per day. ATO dosing was set at 11 infusions of 0.15 mg/kg over either 1 or 2 28-day cycles based on treatment response and clinician’s opinion. Patients were assessed at 6 weeks, 14 weeks, 6 months, 9 months, and 12 months after the first ATO infusion. Preliminary efficacy was based on the overall response rate at 6 months.
Chronic GVHD Treatment Results
In total, 11 patients completed 1 cycle, 9 completed 2 cycles, and 1 received an incomplete cycle. Six patients continued cyclosporine treatment after enrollment, and 4 others resumed cyclosporine during the study.
The overall response rate at 6 months was 75.0% (95% confidence interval [CI], 50.9-91.3%), of which 35% were complete responses (CR) and 40% were partial responses (PR). The failure-free survival rate was 90.9% (95% CI, 65.6-97.5%) at 6 months and 65.0% (95% CI, 40.3-81.5%) at 12 months. The rate of progression-free survival was 95.0% (95% CI, 69.5-99.3%) at 6 months and 83.8% (95% CI, 57.7-94.5%) at 12 months.
Notably, CS dosage decreased from baseline by 74.6% ± 32.7% at 6 months and 91.0% ± 14.6% at 12 months. Additionally, 30.0% and 47.4% of patients had completed stopped CS by the 6- and 12-month follow-ups. Two patients passed from causes unrelated to ATO, and one patient withdrew due to transient hepatotoxicity.
Ultimately, Rongvaux-Gaïda and colleagues concluded that “the first-line combination of ATO and CS was associated with a high clinical response rate and rapid CS sparing in cGVHD after previous allo-HSCT.”
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