Patient Reported Outcome (PRO) in Patients Receiving High and Moderate Emetogenic Chemotherapy (Ctx) - Results of a German Prospective Non-Interventional Study with Netupitant/Palonosetron Fixed Dose Combination (NEPA)

Introduction:NEPA is a fixed dose combination of the NK₁-receptor antagonist (RA) netupitant and 5-HT₃-RA palonosetron approved for prevention of chemotherapy-induced nausea and vomiting (CINV) in pts receiving highly emetogenic (HEC) on cisplatin-basis or moderately emetogenic Ctx (MEC). NEPA demonstrated safety and efficacy in platinum, anthracycline and cyclophosphamid based Ctx, These drugs are frequently used in regimens for hematology pts. A German non-interventional study investigated NEPA's efficacy and impact on quality of life in adult cancer pts by patient-related outcomes (PRO) and physicians' personal assessment under real life conditions. Primary objective was the evaluation of quality of life (QoL) in adults receiving NEPA for CINV prevention. Secondary endpoints were efficacy and safety of NEPA.

Methods: Open label, non-interventional, prospective, national, multicenter study that evaluates CINV prevention and patients' QoL receiving NEPA in pts with either HEC or MEC on up to 2 consecutive Ctx days for at least consecutive 3 cycles. QoL was evaluated by the validated FLIE questionnaires at the end of each Ctx cycle. Efficacy was documented by the treating physicians and via patient diaries for 3 Ctx cycles within 24 hrs and on 4 additional d after Ctx. Safety, additional medication and physicians' overall satisfaction was reported via eCRF.

Results: Between June 2015 and Sept 2017 a total of 2429 pts were enrolled with 1997 pts being eligible for the 2nd interim analysis. 1901 pts were included in the efficacy analysis in the 1st, 1808 pts in the 2nd and 1734 pts in the 3rd cycle. Median age was 58 (range of 28-89). A total of 630 evaluable pts received MEC (53% carboplatin based) and 1185 pts received HEC (88% anthracycline/ cyclophosphamide based), PRO with complete response (CR=no nausea, no vomiting, no rescue medication) was analyzed based on patient diaries. Diaries for PRO in MEC-pts (n=401) reported CR in 94% in cycle 1, 85% in cycle 2 and 86% in cycle 3. Efficacy, assessed by physicians (n=630 pts) on a 4-point scale, was rated very good/good for 91%, 93%, and 94% in cycle 1, 2 and 3, respectively. PRO of 896 pts with HEC reported 81% CR in cycle 1, 82% in cycle 2 and 83% in cycle 3. Efficacy, assessed by physicians (1185 HEC pts), was rated very good/good for 88%, 89%, and 90% in cycle 1, 2 and 3, respectively. A high percentage of patients receiving HEC or MEC did not suffer from any emesis (93%, 93% and 94% in cycle 1-3, respectively). Over 85%of pts reported no impact on daily QoL due to vomiting in all 3 cycles with HEC or MEC.The most common treatment emergent adverse events were constipation and insomnia of mild-moderate intensity.

Conclusions: NEPA prevented CINV highly effective in the acute and delayed phase of HEC and MEC with no impact on daily life due to vomiting in >85% of pts. Physicians evaluation was concordant to PRO.Safety profile was good.