Health-related quality of life in patients with newly diagnosed multiple myeloma ineligible for stem cell transplantation: results from the randomized phase III ALCYONE trial

Stefan Knop,Mark Cook,Jianping Wang,Philip Campbell,Ganna Usenko,Kenshi Suzuki,Andre H Crepaldi ,Susan Wróblewski ,Tomoaki Fujisaki,Anna Marina Liberati,Tatiana Shelekhova,Andrzej Jakubowiak,Meletios A Dimopoulos,John Fastenau,María Victoria Mateos ,Mamta Garg,Luděk Pour ,Genadi Losava,Paulo Lúcio ,Sung Soo Yoon ,Katharine S Gries,Zsolt Nagy,Chantal Doyen,Jesús F San Miguel ,Sebastian Grosicki,Anupa Kudva,Michèle Cavo

doi:10.1186/s12885-021-08325-2

Abstract

BackgroundIn the phase III ALCYONE trial, daratumumab plus bortezomib/melphalan/prednisone (D-VMP) significantly improved overall response rate and progression-free status compared with VMP alone in transplant-ineligible patients with newly diagnosed multiple myeloma (NDMM). Here, we present patient-reported outcomes (PROs) from ALCYONE.MethodsThe European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30-item (EORTC QLQ-C30) and EuroQol 5-dimensional descriptive system (EQ-5D-5L) questionnaire were administered at baseline, every 3 months (year 1) and every 6 months (until progression). Treatment effects were assessed using a repeated-measures, mixed-effects model.ResultsCompliance with PRO assessments was comparable at baseline (> 90%) and throughout study (> 76%) for both treatment groups. Improvements from baseline were observed in both groups for EORTC QLQ-C30 Global Health Status (GHS), most functional scales, symptom scales and EQ-5D-5L visual analog scale (VAS). Between-group differences were significant for GHS (p = 0.0240) and VAS (p = 0.0160) at month 3. Improvements in pain were clinically meaningful in both groups at all assessment time points. Cognitive function declined in both groups, but the magnitude of the decline was not clinically meaningful.ConclusionsPatients with transplant-ineligible NDMM demonstrated early and continuous improvements in health-related quality of life, including improvements in functioning and symptoms, following treatment with D-VMP or VMP.Trial registrationClinicalTrials.gov identifier NCT02195479, registered September 21, 2014

Highlights

In the phase III ALCYONE trial, daratumumab plus bortezomib/melphalan/prednisone (D-VMP) significantly improved overall response rate and progression-free status compared with VMP alone in transplantineligible patients with newly diagnosed multiple myeloma (NDMM)
Treatment approaches for newly diagnosed multiple myeloma (NDMM) are chosen based on the patient’s fitness; those considered fit usually receive induction, high-dose chemotherapy and autologous stem cell transplant (ASCT) as standard of care [1]
Compliance was calculated at baseline and for each postbaseline Patient-reported outcome (PRO) assessment visit as a percentage, with the number of PRO assessments received as the numerator and the number of PRO assessments expected at that time point as the denominator

Summary

Introduction

In the phase III ALCYONE trial, daratumumab plus bortezomib/melphalan/prednisone (D-VMP) significantly improved overall response rate and progression-free status compared with VMP alone in transplantineligible patients with newly diagnosed multiple myeloma (NDMM). In patients ineligible for ASCT, treatment with bortezomib, melphalan and prednisone (VMP) or lenalidomide plus low-dose dexamethasone is recommended [1]. Older patients and those who are transplant ineligible have significantly shorter relative survival than younger, fitter, transplanteligible patients [2, 3]. Reports on HRQoL in the ASCT-ineligible population (in particular) are limited

Methods

Results

Conclusion