Paul Richardson1, Anuja Roy2, Suddhasatta Acharyya3, Ashok Panneerselvam3, Estella Mendelson3, Andreas Günther4, Sagar Lonial5, Hermann Einsele6. 1. a Department of Hematologic Oncology , Dana Farber Cancer Institute , Boston , MA , USA. 2. b Department of Global Value & Access , Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA. 3. c Department of Biostatistics , Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA. 4. d Division of Stem Cell Transplantation and Immunotherapy, 2nd Department of Medicine , University Hospital Schleswig-Holstein and University of Kiel , Kiel , Germany. 5. e Winship Cancer Institute , Emory University , Atlanta , GA , USA. 6. f Medizinische Klinik und Poliklinik II , Julius Maximilians Universitaet , Wuerzburg , Germany.
Abstract
BACKGROUND: Patients with relapsed or relapsed/refractory multiple myeloma (RRMM) face poor treatment options by the time third-line therapy is required, despite advances in overall survival in recent years. Treatment free interval (TFI) and opportunities to maintain quality of life (QoL) have been cited as additional measures of efficacy that can be utilized in personalized treatment decisions. METHODS: The clinical health outcomes data from PANORAMA-1, the pivotal phase-3 trial comparing panobinostat-bortezomib-dexamethasone (PAN-BTZ-DEX) with placebo (PBO)-BTZ-DEX in RRMM patients treated with 1 to 3 prior regimens, retrospectively assessed TFI as a health outcome measure and metric of patient treatment experience relevant to the RRMM population. RESULTS: PAN-BTZ-DEX shows promise for prolonged TFI (mean TFI, 7.49 months; 95% CI, 6.02 to 8.71) compared to PBO-BTZ-DEX (mean TFI, 3.86 months; 95% CI, 3.08 to 4.60) for heavily pre-treated advanced RRMM patients), due to the short duration of therapy and extended progression free-survival. Further, QoL during the TFI was similar to baseline. CONCLUSIONS: PAN-BTZ-DEX provides a treatment regimen with prolonged TFI benefits previously not available for RRMM patients. TFI has not been traditionally measured in clinical trials, but should be assessed in prospective data collection given its value to payers, providers, and patients.
BACKGROUND:Patients with relapsed or relapsed/refractory multiple myeloma (RRMM) face poor treatment options by the time third-line therapy is required, despite advances in overall survival in recent years. Treatment free interval (TFI) and opportunities to maintain quality of life (QoL) have been cited as additional measures of efficacy that can be utilized in personalized treatment decisions. METHODS: The clinical health outcomes data from PANORAMA-1, the pivotal phase-3 trial comparing panobinostat-bortezomib-dexamethasone (PAN-BTZ-DEX) with placebo (PBO)-BTZ-DEX in RRMM patients treated with 1 to 3 prior regimens, retrospectively assessed TFI as a health outcome measure and metric of patient treatment experience relevant to the RRMM population. RESULTS:PAN-BTZ-DEX shows promise for prolonged TFI (mean TFI, 7.49 months; 95% CI, 6.02 to 8.71) compared to PBO-BTZ-DEX (mean TFI, 3.86 months; 95% CI, 3.08 to 4.60) for heavily pre-treated advanced RRMM patients), due to the short duration of therapy and extended progression free-survival. Further, QoL during the TFI was similar to baseline. CONCLUSIONS:PAN-BTZ-DEX provides a treatment regimen with prolonged TFI benefits previously not available for RRMM patients. TFI has not been traditionally measured in clinical trials, but should be assessed in prospective data collection given its value to payers, providers, and patients.
Entities:
Keywords:
Multiple myeloma; cancer drug value; disease progression; panobinostat; quality of life; treatment-free interval; value assessment
Authors: Matthew R LeBlanc; Rachel Hirschey; Ashley Leak Bryant; Thomas W LeBlanc; Sophia K Smith Journal: Qual Life Res Date: 2019-12-17 Impact factor: 4.147
Authors: Meredith M Regan; Lillian Werner; Sumati Rao; Komal Gupte-Singh; F Stephen Hodi; John M Kirkwood; Harriet M Kluger; James Larkin; Michael A Postow; Corey Ritchings; Mario Sznol; Ahmad A Tarhini; Jedd D Wolchok; Michael B Atkins; David F McDermott Journal: J Clin Oncol Date: 2019-09-09 Impact factor: 44.544