Background Steroid-refractory graft-versus-host disease (GVHD) is a significant complication of allogeneic haematopoietic stem cell transplantation (HSCT) and a leading cause of morbidity and non-relapse mortality.
Adult clinical trials with ruxolitinib have demonstrated benefit in this population, but there are no paediatric reports describing this effectiveness.
Purpose Analyse effectiveness and safety of ruxolitinib in paediatric patients, with steroid-refractory GVHD.
Material and methods Retrospective study including patients diagnosed with GVHD treated with ruxolitinib from January 2017 to October 2018. Demographic and clinical data were collected from electronic medical records and pharmacy software: sex, age, weight, type, location and severity of GVHD, previous treatments, dosing, duration of treatment, response and toxicities.
Results Seven patients were included, 5 boys and 2 girls, with a median age of 11 years (5–18); and a median weight of 40 kg (15–63). One patient developed severe acute intestinal GVHD (aGVHD) and six chronic GVHD (cGVHD), moderate (n=1) and severe (n=5). The median number of affected organs per patient was three (1–4): skin (n=6), gastrointestinal tract (n=4), joints (n=2), lungs (n=2) and liver (n=1).
Median number of treatments used before ruxolitinib was four (2–5), always including corticosteroids as the first option. Treatments in the second or third line were: extracorporeal-photoapheresis, mesenchymal stem cells, immunosuppressants and infliximab.
Four patients started with 5 mg/12 hour increasing to 10 mg/12 hour if they weighed >25 kg. One started at 1.25 mg/12 hour because they were in treatment with posaconazol increasing to 2.5 mg/12 hour, and two started directly at 10 mg/12 hour. The median treatment’s duration was 10 months (3–19). All cGVHD were still in treatment at the end of the study.
All patients responded to ruxolotinib: the only patient with aGVHD and one patient with cGVHD had complete response, and the remainder had partial response.
Digestive, cutaneous and joints symptoms showed improvement, while GVHD affecting the lungs and liver did not.
No patient died during the study. Only two patients presented with leukopaenia and two suffered reactivations of cytomegalovirus, but there was no dose reduction due to toxicity.
Conclusion In our patients ruxolitinib has proven to be an effective and safe treatment option, but well-designed clinical trials are necessary to know its real benefit in paediatric patients with steroid-refractory GVHD.
References and/or acknowledgements Thank you to all authors for their involvement.
No conflict of interest.
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