Ruxolitinib in refractory acute and chronic graft-versus-host disease: a multicenter survey study

Abstract

Graft-versus-host disease is the main cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation. First-line treatment is based on the use of high doses of corticosteroids. Unfortunately, second-line treatment for both acute and chronic graft-versus-host disease, remains a challenge. Ruxolitinib has been shown as an effective and safe treatment option for these patients. Seventy-nine patients received ruxolitinib and were evaluated in this retrospective and multicenter study. Twenty-three patients received ruxolitinib for refractory acute graft-versus-host disease after a median of 3 (range 1-5) previous lines of therapy. Overall response rate was 69.5% (16/23) which was obtained after a median of 2 weeks of treatment, and 21.7% (5/23) reached complete remission. Fifty-six patients were evaluated for refractory chronic graft-versus-host disease. The median number of previous lines of therapy was 3 (range 1-10). Overall response rate was 57.1% (32/56) with 3.5% (2/56) obtaining complete remission after a median of 4 weeks. Tapering of corticosteroids was possible in both acute (17/23, 73%) and chronic graft-versus-host disease (32/56, 57.1%) groups. Overall survival was 47% (CI: 23-67%) at 6 months for patients with aGVHD (62 vs 28% in responders vs non-responders) and 81% (CI: 63-89%) at 1 year for patients with cGVHD (83 vs 76% in responders vs non-responders). Ruxolitinib in the real life setting is an effective and safe treatment option for GVHD, with an ORR of 69.5% and 57.1% for refractory acute and chronic graftversus-host disease, respectively, in heavily pretreated patients.

Acute Graft‐versus‐Host Disease: Emerging Insights and Updates into Detection, Prevention, and Treatment

“…61 Additional reports continue to support the use of ruxolitinib in this setting of steroid-refractory acute GVHD in adult and pediatric populations. 62,63 The most recent data published includes REACH2 (clinicaltrials.gov; NCT02913261), a phase III randomized, openlabel trial evaluating the efficacy of ruxolitinib 10 mg twice/day compared with best available therapy (BAT) for steroid-refractory acute GVHD. Day 28 overall responses were improved in the ruxolitinib group (62%) compared with the BAT group (39%, p < 0.001).…”

Treatment of refractory acute myeloid leukaemia during pregnancy with venetoclax, high‐dose cytarabine and mitoxantrone

“…Cytogenetic analysis showed a normal female karyotype. The patient developed a steroid‐refractory gastrointestinal graft‐ versus ‐host disease (Grade 2), which was treated with ruxolitinib and extra‐corporeal photopheresis and controlled 12 …”

Adverse events in second- and third-line treatments for acute and chronic graft-versus-host disease: systematic review

“…16 Whilst ruxolitinib was approved by the United States (US) Food and Drug Administration (FDA) for steroid-refractory aGvHD in May 2019, 17 standardised recommendations on how to best implement such a diverse array of treatments remain lacking. 18 The objective of this study was to review and describe reported harm outcomes associated with second- and third-line therapies for GvHD following allo-HSCT.…”