A Phase II Trial of Netupitant/Palonosetron for Prevention of Chemotherapy Induced Nausea/Vomiting in Patients Receiving BEAM Prior to Hematopoietic Cell Transplantation

Abstract

Abstract Objective The purpose of this study was to investigate the efficacy and safety of NEPA for the prevention of CINV for HCT patients receiving BEAM therapy. Study Design This phase II, prospective, intention-to-treat, single-center, single arm study involved 43 adult patients who received NEPA and dexamethasone for prevention of CINV due to BEAM conditioning chemotherapy. An interim analysis, performed after 13 patients, determined utility versus futility and supported continuation to full enrollment. Descriptive statistics were used to report complete response (CR), complete protection (CP), incidence of emesis, and administration of rescue agents. A Kaplan-Meier curve depicted time to first emesis and first rescue medication. Patients self-reported levels of daily nausea descriptively via a CINV Questionnaire. Results By study end, 13 of 43 patients achieved a CR with an average of 10.6 emesis free days (SD 0.95) over the 11-day observation period, with no emetic events in any patient during the acute/chemotherapy phase. Nausea was well controlled throughout the acute therapy phase (Days 1–6) and increased during the delayed phase (Day 7–11) with a peak mean level of 2.79/10 at Day 10. Aside from lower grade (≤ 2) headaches, constipation and diarrhea were the most widely reported adverse effects. Conclusion The combination of NEPA and dexamethasone is safe and effective for the prevention of CINV in patients receiving BEAM conditioning therapy prior to HCT. The regimen demonstrated greater effectiveness in the acute phase versus the delayed phase, with low levels of nausea throughout the study period and complete emesis prevention during chemotherapy. NCT03097588 registered March 31, 2017.