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Dr Franco Locatelli: Assessing Ruxolitinib Treatment in Pediatric Patients With cGvHD


Franco Locatelli, MD, PhD, a professor and hematologist from Italy, discusses results from the the phase 2 REACH 5 studies assessing the use of ruxolitinib in pediatric patients with chronic graft versus host disease (cGvHD).

Ruxolitinib proved to be the best overall treatment option for patients in terms of response and experience, says Franco Locatelli, MD, PhD, head of the Department of Pediatric Hematology and Oncology, IRCCS Bambino Gesu Children’s Hospital, Rome, professor of pediatrics at the Sapienza, University of Rome, Italy.


Can you share some of the main findings from the phase 2 REACH 5 studies assessing the use of ruxolitinib in pediatric patients with chronic graft versus host disease (cGvHD)?

CdGvHD represents a serious complication, sometimes also fatal for patients that are given allogeneic stem cell transplantation, and the impact of the complication may be particularly detrimental in the pediatric population considering their long-life expectancy and considering that we are talking about growing patients. Corticosteroids groups represent the first line of treatment for adult patient with cGvHD, while in the pediatric population there is no standard second line of treatment for patients who do not respond to corticosteroids.

Ruxolitinib is a selective JAK [janus kinase inhibitor] 1 and 2 inhibitor, which demonstrated greater efficacy in adults, and later adolescents, with cGvHD. In particular, in a phase 3 randomized clinical trial, the REACH 3 trial, it was demonstrated that ruxolitinib was better than the best available therapy in terms of overall response in patients experiencing in this complication.

Today, I presented the results of the phase 2 trial, which involved pediatric patients with an age compliance between 2 and 18 years. And in the study, there were 45 patients affected by either treatment-naïve or steroid-refractory cGVHD. And indeed, most of the patient had steroid refractory cGvHD.

The study allowed us to identify the recommended dose to be employed in the pediatric population, which was 10 mg twice a day for patients between 12 and 18 years old, 5 mgs twice a day in patients with an age between 6 and 12 years, and 4 mg/m2 twice a day in patients between 2 and 6 years.

That overall response rate at day 1 of cycle 7 was the main endpoint of the study, and 40% of patients had this overall response rate, with no difference between a treatment-naïve and steroid-refractory cGvHD patients.

We also evaluated that the best overall response up to day 1 of cycle 7, and in this case, [it reached] 82%. Remarkably, there was no difference in terms of the overall response rate and best overall response across the 3 age groups that I mentioned before.

The probability of response was a little bit higher in patients with moderate cGvHD in comparison to those with severe cGvHD and the joints, face, and the upper gastrointestinal tract and bowel, [which] were the organs that responded better to treatment when it hasn’t in other previous studies on age and years for the treatment of cGvHD. Both the lung and the liver continue to be difficult to treat.

There were 10 fatal events, but in different regions, and 45 patients enrolled into the trial. But 9 out of the 10 fatalities occurred in the steroid-refractory population, so the probability of being alive at 1-year after the beginning of treatment was higher in the treatment-naïve [group] compared to the steroid-refractory population. All in all, this data indicated that ruxolitinib is an [acceptable] therapeutical option for the pediatric population experiencing cGvHD. The severity profile is consistent with that reported in the other populations and now pediatricians have other therapeutic options for treating this severe, sometimes life-threatening complication of allogeneic stem cell transplant.

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