The study authors conclude that in this randomized, placebo-controlled trial of ibrutinib in combination with corticosteroids for NIH-defined moderate/severe cGVHD, the primary endpoint did not meet statistical significance. However, numerical trends of improved clinical outcomes in the ibrutinib-pred arm were noted, including longer duration of response and event-free survival and improved patient-reported outcomes. Safety was consistent with the known profiles of ibrutinib and pred and was similar between treatment arms. According to the authors, the positive trends observed in other important clinical endpoints along with no additional safety trends and concerns suggest that ibrutinib may have value in some previously untreated pts with cGVHD.

The study authors conclude that ruxolitinib led to high response rates in pts who crossed over from best available therapy to ruxolitinib. ORR and durable ORR were consistent with those seen with ruxolitinib during the randomized period. No new safety signals were observed in the crossover patients. These findings support the use of ruxolitinib in patients with steroid-refractory aGVHD who failed treatment with other systemic therapies.

The study authors conclude that low doses of glasdegib demonstrate high rates of clinical responses in heavily pretreated patients with sclerotic chronic GVHD, resulting in durable responses in one-third of the patients. Treatment schedule adjustments may be required in order to optimize tolerability.

The study authors conclude that the safety and efficacy observed to date and benefit-risk profile support continued study and evaluation in future randomized controlled trials.