Inside the Clinic: Acute Graft-versus-Host Disease - Episode 8
Yi-Bin A. Chen, MD: I think it would be fair to say that many of us were excited when these results [REACH1] were presented. We’re all eagerly awaiting the publication of the full trial and the details, but when this was presented a few months ago, I think many of us were really excited. It was really compelling data and ultimately led to the approval. It was a rather large study—71 patients—and the data that is presented was comparable, if not better than most trials that have been published, or most series, I should say, for steroid-refractory graft-versus-host disease [GVHD]. Zack, do you think that these data are practice changing?
Zachariah M. DeFilipp, MD: I do, Yi-Bin. As you mentioned before, the general shift in graft-versus-host disease therapy has been away from more immune suppressive medications and more towards targeted medications such as ruxolitinib, although major strides have been made toward finding targeted agents that could benefit our patient population.
So when thinking about the results of this study in the context of the other agents that we have available for our patients, I do believe that ruxolitinib should, at this point, be considered the standard of care for most patients who had steroid-refractory acute graft-versus-host disease.
Yi-Bin A. Chen, MD: Certainly the approval helps for access, and the data certainly provides confidence. I think there is some hesitation in the community for a certain subset of patients, and it may be patients with significant lower GI [gastrointestinal] involvement who might have trouble taking pills or might have trouble absorbing this. It’s hard to know. I think the correlative data presented in the trial would suggest that patients, even with lower GI disease, did respond and had therapeutic levels of drug. So I think we’re all waiting…. A lot more follow up and a lot more use by us and our colleagues to really establish if this is truly the standard of care. But it certainly is a standard of care at the moment.
Colleen, you’ve managed and treated a lot of patients with ruxolitinib right now for graft-versus-host disease. Can you discuss the initial dosing and tolerability of the agent with us?
Colleen M. Danielson, NP: We initially dose the patient at 5 mg twice a day to start, and then, after a period of time—usually about 3 to 5 days—we can increase the patient to 10 mg twice a day.
However, we need to keep in mind that a lot of our patients are on antifungal therapy. For those patients who are on azoles, we actually will leave them at that 5 mg twice-a-day dosing. That’s pretty much where we will start and then escalate if we can.
Yi-Bin A. Chen, MD: Zack, in your experience treating patients with ruxolitinib for graft-versus-host disease, what has been your experience with any adverse events? Have you had to manage patients differently because of side effects from the drug?
Zachariah M. DeFilipp, MD: In my experience, ruxolitinib has been very well tolerated by patients. One of the things that we have seen in our transplant patients receiving ruxolitinib is that they can develop cytopenias. Sometimes you see a low white blood [cell] count, a low red blood [cell] count or hemoglobin, and also low platelets. This is usually 1 of the more common things that has been seen with the use of ruxolitinib in this population, but it has very rarely led to us holding ruxolitinib therapy from patients. Sometimes we may need to dose reduce the ruxolitinib to a lower dose in order for it to be more tolerable on the patient’s blood counts.
Outside of hematologic adverse events, we’ve also seen that there is a risk for infection. But all patients receiving a bone marrow transplant, and especially those developing graft-versus-host disease have a risk for infection, and some patients also develop edema. Depending on the severity of edema, once again, sometimes the dose of the ruxolitinib might need to be adjusted and other supportive medications like a diuretic might be used in order to help deal with the patient’s edema.
Yi-Bin A. Chen, MD: I think that mirrors my experience as well, as well as colleagues that I’ve spoken with across the country. I think we have found ruxolitinib to be very well tolerated in our population.
Transcript Edited for Clarity