Ruxolitinib trial was in adults but from the reading I've done I understand it's being prescribed for children.
Side effects are viral and bacterial infections and although these can be reversible, common side effect is activation of CMV, which can make GvHD difficult to diagnose.
Ruxolitinib is combined with immune suppressants such as Methotrexate or Sirolimus and for skin GvHD combined with ECP, which is done in hospital. Use of broad immune suppressants can adversely affect graft v cancer.
I recently referred to a small study of Ruxolitinib in sclerodermatous skin GvHD. The benefits appeared modest. Sclerodermatous skin GvHD can be particularly difficult to treat and best to try to prevent it, such as lung GvHD. The biggest risk factor for cGvHD is prior acute.
The study by Zeiser et al is referenced a lot as it's said to show very good results for Ruxolitinib; however, Neumann et al (2019) got poor results in intestinal GvHD, which have also been described by Khandelwal et al's 2017 study in children with SR-aGvHD.
Writing about study by Zeiser et al, Khandelwal et al say:
"In the adult report of ruxolitinib as salvage treatment for steroid-refractory acute GVHD [7], the authors did not use the time point of 4 weeks from starting ruxolitinib to assess responses. Instead, this above-mentioned study mandated that a response needed to last for 3 weeks, and patients were scored for their best response at any time after starting treatment with ruxolitinib, with follow-up censored at the onset of any subsequent systemic immunosuppressive therapy."
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