HORIZON (OP-106): Melflufen Plus Dexamethasone (dex) in Patients (pts) with Relapsed/Refractory Multiple Myeloma (RRMM) - Health-Related Quality of Life (HRQoL) Analysis

• Published in: Blood 2020-11, Vol.136 (Supplement 1), p.27-29
• Main Authors: Oriol, Albert, Richardson, Paul G., Mateos, María-Victoria, Larocca, Alessandra, Blade Creixenti, Joan, Cavo, Michele, Rodríguez-Otero, Paula, Norkin, Maxim, Nadeem, Omar, Hiemenz, John W., Hassoun, Hani, Touzeau, Cyrille, Amor, Adrián Alegre, Paner, Agne, Maisel, Christopher, Mazumder, Amitabha, Raptis, Anastasios, Puig, Noemí, Strang, Peter, Sandberg, Anna, Jaques, Christian, Thuresson, Marcus, Orre, Marie, Leleu, Xavier
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2020-135995

Background: With advances in therapy, outcomes have generally improved for pts with MM; however, pts with late-stage RRMM have limited treatment options and poor outcomes (Kumar et al. Leukemia. 2017;31:2443; Gandhi et al. Leukemia. 2019;33:2266). Pts with late-stage RRMM, often older and having comorbidities, require efficacious and tolerable therapies to maintain HRQoL (Richardson et al. Blood. 2019;134[suppl 1]:3487). Melphalan flufenamide (melflufen) is a first-in-class peptide-drug conjugate (PDC) that targets aminopeptidases and rapidly releases alkylating agents into tumor cells. In the pivotal, phase 2, HORIZON study (NCT02963493) melflufen plus dex showed an overall response rate of 29%, median progression-free survival of 4.2 months, median overall survival of 11.6 months, and a manageable safety profile (N=157; Richardson et al. EHA 2020. Abs EP945). A previous baseline (BL) HRQoL analysis concluded that the HORIZON population is representative of RRMM populations, with a poor overall HRQoL relative to other populations with advanced cancers (Richardson et al. ASH 2019. Abs 3487). This analysis evaluates HRQoL in pts with RRMM throughout treatment with melflufen plus dex in the HORIZON study. Methods: Eligible pts received melflufen 40 mg on d1 of each 28-day cycle plus dex 40 mg/wk (20 mg in pts aged ≥75 y). HRQoL was added as a secondary endpoint as an amendment to the HORIZON protocol, which allowed collection of data from a subset of pts using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 V.3 (EORTC QLQ-C30) and the EuroQOL 5 Dimension-3 Level (EQ-5D) questionnaires. EORTC QLC-C30 evaluates functional domains and symptoms on a scale from 0-100 (higher functional scores = better function; higher symptom scores = more symptomatology/problems). The EQ-5D index is evaluated on a scale from 0 (death) to 1 (perfect health) and the EQ-5D VAS is evaluated on a scale from 0 (death) to 100 (perfect health). Questionnaires were administered before dosing at BL (cycle [C] 1) and predose at intervals throughout the study. Descriptive data for pts with post-BL assessments at C2, C4, and C6 are presented herein. Pts with only BL questionnaire assessments were excluded from this analysis. Select EORTC QLQ-C30 and EQ-5D results are presented. This analysis is ongoing. Results: At HRQoL data cutoff (May 28, 2020), of the 64 pts with post-BL HRQoL assessments, 19 remained on therapy. Among 64 pts in the HR