Many transplanters will now offer ibrutinib to their patients because this agent is available, approved by the U.S. Food and Drug Administration for other closely related indications, and also serves an important unmet medical need.

— Kanti R. Rai, MD

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Kanti R. Rai, MD, Professor of Medicine, Hofstra Northwell School of Medicine, New Hyde Park, New York, commented on these study findings. “I congratulate the investigators for a work done with discipline, excellent collaboration, and speed.”

“All of us who have become familiar with ibrutinib (Ibruvica), which is now well known for effective and dramatic salvage of patients with relapsed and refractory chronic lymphocytic leukemia, have been expecting it might have an important role in the treatment of chronic graft-vs-host disease occurring after allogeneic stem cell transplantation,” mentioned Dr. Rai. He thinks the reason for this expectation is the underlying mechanism by which ibrutinib inhibits Bruton’s tyrosine kinase, a critically important enzyme controlling the B-cell receptor (and in turn, B-cell proliferation, survival, and trafficking) as well as the functions of T cells.

‘Truly Welcome News’

“The report presented by Dr. ­David Miklos and colleagues is truly welcome news,” Dr. Rai announced. “Finally, we might be witnessing a very important landmark in finding an effective agent that controls chronic graft-vs-host disease.” According to Dr. Rai, this is a respectable sample size, and these investigators clearly demonstrate that more than 65% patients have a response, with a 21% complete response rate. And, these were lasting responses, added Dr. Rai, who considered the severe adverse effects to be within a tolerable range.

Dr. Rai concluded: “These results clearly are very promising and compare favorably with alternative therapies, all of which unfortunately depend upon drugs that are more immunosuppressive, with the expected adverse activities. In my opinion, many transplanters will now offer ibrutinib to their patients because this agent is available, approved by the U.S. Food and Drug Administration for other closely related indications, and also serves an important unmet medical need.” ■

Disclosure: Dr. Rai has served on the medical advisory boards of Pharmacyclics, Gilead, Celgene, Incyte, and Roche/Genentech.