INSIGHT MM: a large, global, prospective, non-interventional, real-world study of patients with multiple myeloma

Caitlin Costello, Faith E Davies, Gordon Cook, Jorge Vela-Ojeda, Jim Omel, Robert M Rifkin, Jesus Berdeja, Noemi Puig, Saad Z Usmani, Katja Weisel, Jeffrey A Zonder, Evangelos Terpos, Andrew Spencer, Xavier Leleu, Mario Boccadoro, Michael A Thompson, Dorothy Romanus, Dawn M Stull, Vania Hungria, Caitlin Costello, Faith E Davies, Gordon Cook, Jorge Vela-Ojeda, Jim Omel, Robert M Rifkin, Jesus Berdeja, Noemi Puig, Saad Z Usmani, Katja Weisel, Jeffrey A Zonder, Evangelos Terpos, Andrew Spencer, Xavier Leleu, Mario Boccadoro, Michael A Thompson, Dorothy Romanus, Dawn M Stull, Vania Hungria

Abstract

With the introduction of new drugs with different mechanisms of action, multiple myeloma (MM) patients' outcomes have improved. However, the efficacy seen in clinical trials is often not seen in real-world settings and data on the effectiveness of MM therapies are needed. INSIGHT MM is a prospective, global, non-interventional, observational study that is enrolling approximately 4200 patients with newly diagnosed or relapsed/refractory MM, making it the largest study of its kind to date. The study aims to describe contemporary, real-world patterns of patient characteristics, clinical disease presentation, therapies chosen, clinical outcomes (response, treatment duration, time-to-next-therapy, progression-free and overall survival), safety, healthcare resource utilization and quality of life. One interim analysis has been conducted to date; current accrual is approximately 3094 patients. Trial registration number: NCT02761187.

Keywords: multiple myeloma; observational study; treatment.

Conflict of interest statement

Financial & competing interests disclosure

This study was funded by Millennium Pharmaceuticals, Inc., Cambridge, MA, USA, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. C Costello has acted on an advisory council or committee for Celgene, Janssen and Takeda. FE Davies has acted on an advisory council or committee for Amgen and Takeda, has received honoraria from ASH, MMRF and TRM Oncology, consulting fees from Amgen, AbbVie, Celgene, Janssen and Takeda, and reports grants/funds from Celgene and Janssen. G Cook has acted on an advisory council or committee for Celgene, Janssen and Takeda, has received honoraria from Celgene, Janssen, Sanofi and Takeda, reports consulting fees from Celgene, Janssen, Sanofi, Seattle Genetics and Takeda, and reports grants/funds from Takeda and Celgene. RM Rifkin has acted on an advisory council or committee for AbbVie, Amgen, Celgene and Takeda. J Berdeja reports research funding to his institution from AbbVie, Amgen, Bluebird, BMS, Celgene, Genentech, Glenmark, Jannssen, Novartis, Poseida, Sanofi, Takeda and Teva. N Puig has received honoraria from Janssen, Takeda, Celgene, Amgen, and The Binding Site, reports consulting fees from Janssen, Takeda, Celgene, and Amgen, and reports grants/funds from Janssen and Celgene. SZ Usmani reports consulting fees from Amgen, BMS, Celgene, Janssen, Merck, SkylineDX and Takeda, and reports holding a patent or receiving royalties from Amgen, Array Biopharma, Celgene, Janssen, Pharmacyclis, Sanofi and Takeda. K Weisel has acted on an advisory council or committee for Adaptive Biotec, Amgen, BMS, Celgene, Janssen, Juno, Sanofi and Takeda, has received honoraria from Amgen, BMS, Celgene, Janssen, Novartis and Takeda, reports consulting fees from Adaptive Biotec, Amgen, BMS, Celgene, Janssen, Juno, Sanofi and Takeda, and reports grants/funds from Amgen, Celgene, Sanofi and Janssen. JA Zonder has acted on an advisory council or committee for Alnylam, Amgen, BMS, Caelum, Celgene, Janssen, Oncopeptides and Takeda, reports consulting fees from Alnylam, Amgen, BMS, Caelum, Celgene, Janssen, Oncopeptides and Takeda, and reports grants/funds from BMS and Celgene. E Terpos has received honoraria and consulting fees from Amgen, BMS, Celgene/Genesis, Janssen, Novartis and Takeda, and reports grants/funds from Amgen, Genesis, Janssen and Takeda. A Spencer has acted on an advisory council or committee for AbbVie, Amgen, Celgene, Janssen, Servier and Takeda, has received honoraria from AbbVie, Amgen, Celgene, Janssen, Servier and Takeda, and reports grants/funds from AbbVie, Amgen, Celgene, Janssen and Takeda. X Leleu has received honoraria from Takeda. M Boccadoro has received honoraria from Amgen, Celgene, BMS, Janssen and Sanofi. MA Thompson reports ownership of stock shares from Doximity, has acted on an advisory council or committee for AIM Specialty Health, Celgene, GSK, Strata Oncology, Syapse, Takeda and VIA Oncology, and reports receiving royalties from UpToDate – Myeloma. D Romanus and DM Stull report employment with Takeda. V Hungria has received honoraria from Amgen, BMS, Celgene, Janssen and Takeda. J Vela-Ojeda and J Omel report no competing interests. Global Outcomes Research is a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. Global Medical Affairs is a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Medical writing assistance was provided by H Johns and L Cancian of FireKite, an Ashfield company, part of UDG Healthcare plc, who provided medical writing assistance, which was funded by Millennium Pharmaceuticals, Inc., Cambridge, MA, USA, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, and complied with Good Publication Practice 3 ethical guidelines (Battisti WP et al. Ann Intern Med. 163, 461–464 [2015]), and R Ferrari (Millennium Pharmaceuticals, Inc.), for editorial support. The corresponding author had full access to all the study protocol and had final responsibility for the decision to submit for publication.

Figures

Figure 1. . Timeline of key agents…
Figure 1.. Timeline of key agents and treatment combinations that are approved/recommended or have been investigated in Phase III clinical trials.
In the (A) NDMM and (B) RRMM settings. Not all regimens or agents are approved or available in all countries. †Carfilzomib twice-weekly (20/27 mg/m2) versus once-weekly (20/70 mg/m2). ASCT: Autologous stem cell transplant; CTd: Cyclophosphamide, thalidomide, dexamethasone; d: Dexamethasone; Dara: Daratumumab; Dara-Rd: Daratumumab, lenalidomide, dexamethasone; Dara-Vd: Daratumumab, bortezomib, dexamethasone; Dara-VMP: Daratumumab, bortezomib, melphalan, prednisone; Elo-Rd: Elotuzumab, lenalidomide, dexamethasone; EMA: European Medicines Agency; EMN: European Myeloma Network; ESMO: European Society for Medical Oncology; IRd: Ixazomib, lenalidomide, dexamethasone; K: Carfilzomib; Kd/D, carfilzomib, dexamethasone; KRd: Carfilzomib, lenalidomide, dexamethasone; MPR-R: Melphalan, prednisone, lenalidomide, plus lenalidomide maintenance; MPT: Melphalan, prednisone, thalidomide; NDMM: Newly diagnosed multiple myeloma; PAD: Bortezomib, doxorubicin, dexamethasone; Pano-VD: Panobinostat, bortezomib, dexamethasone; Pom-d: Pomalidomide, dexamethasone; R: Lenalidomide; Rd: Lenalidomide, dexamethasone; RRMM: Relapsed/refractory multiple myeloma; T: Thalidomide; Td: Thalidomide, dexamethasone; TE: Transplant-eligible; TiE: Transplant-ineligible; V: Bortezomib; VCD: Bortezomib, cyclophosphamide, dexamethasone; Vd: Bortezomib, dexamethasone; VMP: Bortezomib, melphalan, prednisone; VMPT: Bortezomib, melphalan, prednisone, thalidomide; VRd: Bortezomib, lenalidomide, dexamethasone; VT: Bortezomib, thalidomide; VTD: Bortezomib, thalidomide, dexamethasone. Figure based on references [9–12,41,59].
Figure 2. . INSIGHT MM: 15 participating…
Figure 2.. INSIGHT MM: 15 participating countries.

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