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EBMT 2024 | Novel approaches for the treatment of GvHD in children: ruxolitinib and tissue regeneration

Mattia Algeri, MD, Bambino Gesù Children’s Hospital, Rome, Italy, discusses novel approaches for the treatment of graft-versus-host disease (GvHD) in children. For patients who are steroid-refractory, data from the REACH4 trial (NCT03491215) supports the use of ruxolitinib for acute GvHD in pediatric patients. For those who are also refractory to ruxolitinib, new strategies focus on promoting tissue regeneration rather than immunosuppression with the use of mesenchymal stromal cells or fecal microbiota. This interview took place at the 50th Annual Meeting of the EBMT in Glasgow, Scotland.

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Transcript (edited for clarity)

Even here we are facing a sort of revolution because there are many new approaches that are coming into a field, a field that in the past was limited to corticosteroids. And then after corticosteroids, there were a variety of drugs, but no one had clearly demonstrated superiority over the other one. But now we have the results, at least in adults and adolescents of the REACH studies which clearly demonstrate the superiority of ruxolitinib in steroid refractory acute GvHD versus best nine different best available therapies...

Even here we are facing a sort of revolution because there are many new approaches that are coming into a field, a field that in the past was limited to corticosteroids. And then after corticosteroids, there were a variety of drugs, but no one had clearly demonstrated superiority over the other one. But now we have the results, at least in adults and adolescents of the REACH studies which clearly demonstrate the superiority of ruxolitinib in steroid refractory acute GvHD versus best nine different best available therapies. And this drug now has been tested also in children in the REACH-4 studies. And these results are superimposable to that obtained in adults. So they reinforce the role of ruxolitinib as primary treatment for steroid refractory acute GvHD also in children.

But then we are facing also a paradigm shift in the sense that many new drugs are coming and they are not focused on adding immunosuppression, but rather shifting towards a strategy that promotes organ regeneration, tissue regeneration, restoration of the tissue, repairing of the tissue damage. So in case you have a steroid refractory GvHD and then a ruxolitinib refractory GvHD, the new strategy, the new tendency of treatment is not to continue to add immunosuppression which at the end we have detrimental effects on the patients, on the relapse risk, infection risk, but rather combine immunosuppression, broad immunosuppression, immune modulation and strategies that promote tissue regeneration, such as mesenchymal stromal cells, but also fecal microbiota or new strategies such as GLP-2 agonist.

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Disclosures

Vertex: Advisory Board.