Difelikefalin for CKD-aP: real world evidence of efficacy and safety in Italian patients

Abstract Introduction Chronic Kidney Disease Associated Pruritus (CKD-aP), defined as moderate to severe itching directly related to kidney disease, affects about 1/3 of haemodialysis patients and has been associated with poor quality of life, poor sleep, depression and increased mortality. Difelikefalin (DFK) is the first-in-class therapy approved by FDA and EMA for this condition. The aim of the present study was to evaluate DFK efficacy and safety in a real-world setting in Italy. Methods We retrospectively collected data of 20 patients treated with DFK in a compassionate manner, before the drug was commercialized, after obtaining approval by the relevant Ethics Committee for each individual case. Mean age and dialysis vintage were 72±11 years and 54±44 months, respectively, basal WI-NRS score being 8.2 and 5D-ITCH 18.6. Scratching lesions were detected in 15/20 patients. DFK was administered IV with weight-based dosing at the end of each dialysis session. Results 18/20 patients were treated for 12 weeks, 2 patients stopped treatment for dizziness (n.1) and discomfort (n.1). WI-NRS score improved >3 in 11/18 (61%) at week 4 and in 16/18 (88%) at week 12. 5D-ITCH decreased from basal 18,6 to 10,5 at week 12, while sleep quality at the same time points changed from 2,6 to 5,8; scratching lesions disappeared in 12/15 (80%). Except for the two early therapy breaks, no other side effects were reported. Conclusions In haemodialysis patients affected by CKD-aP, DFK is an effective and safe treatment that might clearly improve this distressing and overlooked condition.