Ruxolitinib Is a Safe and Effective Therapeutic Option for Management of Chronic Gvhd - a Single Center Experience

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Chronic GVHD can have a significant impact on the quality of life and is an important consideration while deciding for allogeneic stem cell transplant. Beyond the 1st line use of corticosteroids, there is no established order of treatment options. Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor that was approved for management of acute GVHD. Here, we present our experience using ruxolitinib for chronic GVHD. We identified 30 patients at our center who have been treated with ruxolitinib for chronic GVHD. A retrospective chart review was conducted to gather data. Chronic GVHD was graded in individual organ systems according to the NIH severity scoring. NIH criteria for global severity and response assessment were not used. While valuable for clinical trials, these are challenging to use in daily practice. We considered each involved organ in a patient as an ‘event' (total 143 ‘events' in 30 patients.) Severity score before and after starting ruxolitinib for each event was compared. Outcomes were classified in to 4 groups – Improvement, Stable, Worsening and New Onset (after start of ruxolitinib.) Using this system, 53 (37%) events showed an improvement, 70 (49%) were stable, 6 (4%) had worsening while 14 (10%) new onset events occurred after starting ruxolitinib. Majority of the patients had been transplanted for MDS/AML (23/30) from MRD (14/30) or MUD (13/30) using PBSC (28/30). Dose reduction was needed in 8/30 patients; 6 due to cytopenias and 1 each for drug-interaction and rectal bleeding. We also reviewed prior, concomitant and additional therapies used. Effects of chronic GVHD on lung are often irreversible. Interestingly, two of our lung GVHD patients were able to come off continuous oxygen. This observation suggests ruxolitinib as a safe and effective option for treatment of chronic GVHD even in heavily pretreated patients.