Background and importance Ruxolitinib has shown efficacy in the treatment of steroid-refractory graft-versus-host disease (GVHD) after haematopoietic stem cell transplantation (HSCT) in adults, but the evidence in children is still scarce.

Aim and objectives To evaluate the effectiveness and safety of ruxolitinib in paediatric patients with steroid-refractory GVHD.

Material and methods A retrospective observational study including all patients treated with ruxolitinib in our paediatric hospital (January 2017–September 2021) was carried out.

Variables collected from electronic medical records and the pharmacy dispensing program were: age, sex, weight, previous treatments for GVHD, length of treatment, dose, treatment response, reasons for discontinuation and adverse events (AEs) related to ruxolitinib. Effectiveness was assessed by the clinical resolution of GVHD.

Results 31 patients (64.5% male, n=20; median age 13.5 (1–19) years; median weight 36.9 (10–85) kg) received treatment during the period of the study in 34 episodes. In 15 episodes (44.1%) the treatment was for acute GVHD (aGVHD) and in 19 (55.9%) for chronic (cGVHD).

The median number of previous lines was 2 (1–4); all patients had previously received steroids. The median length of treatment was 7.4 (1.4–52.3) months. The median initial dose of ruxolitinib was 11.8% 2.5 mg/12 hours (n=4, weight <15 kg); 58.8% 5 mg/12 hours (n=20, weight 15–60 kg) and 29.4% 10 mg/12 hours (n=10, weight 47–85 kg).

4 episodes of cGVHD were not included in the effectiveness analysis: follow-up was continued in another centre (n=2) and 2 patients died while on treatment from other causes. Complete response rate in aGVHD and cGVHD was 86.7% (n=13) and 60.0% (n=9), respectively. 2 (13.3%) patients with cGVHD showed partial response and treatment was switched to other lines. 2 (13.3%) patients with aGVHD and 1 (6.7%) with cGVHD showed treatment failure. 3 (20.0%) patients were receiving ruxolitinib at the time of the analysis for cGVHD showing stable response (n=2) and improvement (n=1).

AEs related to ruxolitinib were: increased serum alanine aminotransferase and aspartate aminotransferase 8.8% (n=3), herpes zoster infection 5.9% (n=2), hypertension 2.9% (n=1) and anaemia 2.9% (n=1). 1 patient required dose reduction due to grade 4 hepatic toxicity, that was resolved.

Conclusion and relevance In our study, ruxolitinib has shown effectiveness for refractory GHVD in most of the patients. The safety profile in our population is consistent with the literature. Further studies in paediatric patients are warranted.

Conflict of interest No conflict of interest