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Dr Franco Locatelli Discusses the Treatment of Patients with cGvHD


Franco Locatelli, MD, PhD, head of the Department of Pediatric Hematology and Oncology at IRCCS Bambino Gesu Children’s Hospital in Rome, discusses the treatment of both adult and pediatric patients with chronic graft versus host disease (cGvHD).

Monitoring the safety and efficacy of janus kinase (JAK) inhibitors is key when treating patients with chronic graft versus host disease (cGvHD), says Franco Locatelli, MD, PhD, head of the Department of Pediatric Hematology and Oncology, IRCCS Bambino Gesu Children’s Hospital, Rome, and professor of pediatrics at the Sapienza, University of Rome, Italy.


What are some important considerations researchers should take into account when testing drugs in pediatric populations with cGvHD?

It's important that the studies on drugs used to treat different complications be performed in the pediatric population. Number 1, to identify the recommended dose, and number 2, to validate the data that has been obtained in adults, and also to draw important information about the predictive prognostic factors in terms of both response and overall survival rate.

Are there any specific monitoring or follow-up recommendations for pediatric patients receiving treatment with JAK inhibitors?

No, at the moment, the safety profile, as I mentioned before, is comparable to that reported in the adult population. Infections occured in around three-quarters of the patients, and in particular, viral infections were diagnosed in 40% of the patients, including SARS-CoV-2 [COVID-19] infection. So, a careful viral monitoring is highly recommended in patients with cGvHD treated with immune suppressive drugs.

What should providers to keep in mind when making decisions on whether to prescribe a pediatric patient with JAK inhibitors?

It's important to make very careful selection of the patients and to monitor the safety profile and the efficacy of the drug. But certainly, in my personal view, ruxolitinib is a highly valuable therapeutic option in what can be defined as an unmet medical need. Now, pediatricians have a very important therapeutic options for treating these severe complications.

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