Dataset Information

Predicting long-term disease control in transplant-ineligible patients with multiple myeloma: impact of an MGUS-like signature.

ABSTRACT: Disease control at 5 years would be a desirable endpoint for elderly multiple myeloma (MM) patients, but biomarkers predicting this are not defined. Therefore, to gain further insights in this endpoint, a population of 498 newly diagnosed transplant-ineligible patients enrolled in two Spanish trials (GEM2005MAS65 and GEM2010MAS65), has been analyzed. Among the 435 patients included in this post-hoc study, 18.6% remained alive and progression free after 5 years of treatment initiation. In these patients, overall survival (OS) rate at 10 years was 60.8% as compared with 11.8% for those progressing within the first 5 years. Hemoglobin (Hb)???12?g/dl (OR 2.74, p?=?0.001) and MGUS-like profile (OR 4.18, p?=?0.005) were the two baseline variables associated with long-term disease-free survival. Upon including depth of response (and MRD), Hb???12?g/dl (OR 2.27) and MGUS-like signature (OR 7.48) retained their predictive value along with MRD negativity (OR 5.18). This study shows that despite the use of novel agents, the probability of disease control at 5 years is still restricted to a small fraction (18.6%) of elderly MM patients. Since this endpoint is associated with higher rates of OS, this study provides important information about diagnostic and post-treatment biomarkers helpful in predicting the likelihood of disease control at 5 years.

SUBMITTER: Rodriguez-Otero P

PROVIDER: S-EPMC6423121 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature

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Predicting long-term disease control in transplant-ineligible patients with multiple myeloma: impact of an MGUS-like signature.

Rodríguez-Otero Paula P Mateos María Victoria MV Martínez-López Joaquín J Hernández Miguel-Teodoro MT Ocio Enrique M EM Rosiñol Laura L Martínez Rafael R Teruel Ana-Isabel AI Gutiérrez Norma C NC Bargay Joan J Bengoechea Enrique E González Yolanda Y de Oteyza Jaime Pérez JP Gironella Mercedes M Nuñez-Córdoba Jorge M JM Encinas Cristina C Martín Jesús J Cabrera Carmen C Palomera Luis L de Arriba Felipe F Cedena María Teresa MT Puig Noemí N Oriol Albert A Paiva Bruno B Bladé Joan J Lahuerta Juan José JJ San Miguel Jesús F JF

Blood cancer journal 20190318 4

Disease control at 5 years would be a desirable endpoint for elderly multiple myeloma (MM) patients, but biomarkers predicting this are not defined. Therefore, to gain further insights in this endpoint, a population of 498 newly diagnosed transplant-ineligible patients enrolled in two Spanish trials (GEM2005MAS65 and GEM2010MAS65), has been analyzed. Among the 435 patients included in this post-hoc study, 18.6% remained alive and progression free after 5 years of treatment initiation. In these p ...[more]

PMID: 30886139

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Project description:ImportanceAlthough the number of treatments for elderly patients with non-transplant-eligible (NTE) multiple myeloma (MM) has increased substantially, evidence is lacking on the clinical effectiveness and cost-effectiveness of novel treatment sequences.ObjectiveTo determine the optimal sequence of treatment for patients with NTE MM from the perspective of the patient, physician, and society.Design, setting, and participantsUsing data from a Dutch observational registry, this economic evaluation combined evidence from network meta-analyses in a patient-level simulation model and modeled time-to-event and types of events from a hospital perspective with a lifetime horizon. Data analysis was performed from June 2019 to September 2020.InterventionsThirty treatment sequences, including up to 3 lines of therapy, were compared with bortezomib-melphalan-prednisone (VMP)-lenalidomide-dexamethasone (LenDex)-pomalidomide-dexamethasone (PomDex).Main outcomes and measuresThe primary outcomes of the model were overall survival (OS), quality-adjusted life-years (QALYs), costs, and cost-effectiveness.ResultsSequences starting with daratumumab-VMP (second line: carfilzomib-lenalidomide-dexamethasone or elotuzumab-lenalidomide-dexamethasone) or bortezomib-melphalan-prednisone-thalidomide-maintenance bortezomib-thalidomide (VMPT-VT) (second line: daratumumab-lenalidomide-dexamethasone) had the largest expected OS (7.5 years), which is 3.5 additional life-years compared with VMP-LenDex-PomDex. Total costs per patient for these sequences ranged between $786 024 and $1 085 794. The sequence VMPT-VT-carfilzomib-lenalidomide-dexamethasone-panobinostat-bortezomib-dexamethasone had the most favorable cost-effectiveness ratio ($98 585 per life-year gained and $132 707 per QALY gained vs VMP-LenDex-PomDex).Conclusions and relevanceThese findings suggest that sequences including novel treatments were highly effective, but the cost-effectiveness ratios were above currently accepted willingness-to-pay thresholds. Treating MM with novel agents necessitates either a large increase in budget or a substantial reduction of drug costs by price negotiations, and these findings can support these reimbursement decisions and price negotiations.

Dataset Information

Predicting long-term disease control in transplant-ineligible patients with multiple myeloma: impact of an MGUS-like signature.

Publications

Predicting long-term disease control in transplant-ineligible patients with multiple myeloma: impact of an MGUS-like signature.

Similar Datasets

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