Patient‐reported outcomes of multiple myeloma patients treated with panobinostat after ≥2 lines of therapy based on the international phase 3, randomized, double‐blind, placebo‐controlled PANORAMA‐1 trial

Abstract

The phase 3 PANORAMA-1 trial led to regulatory approvals of panobinostat (PAN) in combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of multiple myeloma after ≥2 prior regimens, including BTZ and an immunomodulatory drug. Patient-reported outcomes (PROs) were assessed in PANORAMA-1, with data available for 73 patients in the PAN + BTZ + DEX arm and 74 patients in the placebo (PBO) + BTZ + DEX arm. Per the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), global health status/quality of life (QoL) scores initially declined with PAN + BTZ + DEX during the first 24 weeks before approaching baseline scores and remaining steady during the next 24 weeks, with no difference between arms at Week 48. The EORTC QLQ-Myeloma module (EORTC QLQ-MY20) demonstrated initial improvements and subsequent stabilization of disease symptom scores in both arms and initial worsening and subsequent improvement of side effects of treatment scores, with the initial worsening more pronounced and recovery less pronounced with PAN + BTZ + DEX. Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity scores remained relatively stable and similar between the arms. Overall, these PRO findings support the addition of PAN to the BTZ+DEX regimen as an efficacious treatment option, with limited symptomatology and impact on patients' QoL. The reported results are based on a descriptive analysis of the data. No formal statistical tests have been performed.

Patient Perceptions Regarding Multiple Myeloma and Its Treatment: Qualitative Evidence from Interviews with Patients in the United Kingdom, France, and Germany

“…In agreement with previous studies on treatment preferences in patients with MM [3,10], participants prioritized increased life expectancy as a treatment benefit of utmost importance. As in previous studies, patients reported high symptom burden, including symptoms associated with HRQoL [1,2,4,5,7,9]. In agreement with another qualitative study on treatment preferences in MM [3], symptoms such as fatigue and bone pain, and side effects such as gastrointestinal symptoms and peripheral neuropathy, were the most frequently discussed by patients as significantly disruptive to HRQoL.…”

“…Multiple myeloma (MM) is the second most common hematological cancer and is characterized by high levels of mortality [1][2][3][4]. Despite recent treatment advances, MM remains incurable [1][2][3][5][6][7][8] and becomes harder to treat over time [2,3,8]. Development of new treatment options, particularly those that can be tolerated over the long term, is an ongoing priority.…”

Health-related quality of life of carfilzomib- and daratumumab-based therapies in patients with relapsed/refractory multiple myeloma, based on German benefit assessment data

“…The OS benefit in the subgroup of patients with ≥ 2 prior treatments was 5 months. In the PANORAMA-1 study, HRQoL scores after 48 weeks of treatment with bortezomib/dexamethasone, with or without panobinostat, showed no benefit, and no significant difference in OS was observed between treatments [43]. Interestingly, it has been reported that there may be a relationship between improvement in HRQoL and treatment response in patients with cancer [36,44].…”

Health‐related quality of life maintained over time in patients with relapsed or refractory multiple myeloma treated with daratumumab in combination with bortezomib and dexamethasone: results from the phase III CASTOR trial

“…Despite recent treatment advances, multiple myeloma (MM) remains an incurable disease with a substantial negative impact on patients' health-related quality of life (HRQoL). [1][2][3][4][5][6][7][8][9] MM treatment is associated with demanding administration and monitoring schedules 2 as well as adverse events. 1,2,5 Since treatment until disease progression (rather than a fixed-duration regimen) is recommended, 6,10 and because treatment with novel agents has extended progression-free survival (PFS), 11 assessment of the impact of treatment on patients' long-term HRQoL is warranted.…”

“…1,2,5 Since treatment until disease progression (rather than a fixed-duration regimen) is recommended, 6,10 and because treatment with novel agents has extended progression-free survival (PFS), 11 assessment of the impact of treatment on patients' long-term HRQoL is warranted. 3,7,9,12,13 Moreover, MM is primarily a disease of older persons 1 for whom treatment selection may be complicated by co-morbidities, such as diabetes or cardiovascular disease. 14 Patient-reported outcomes (PROs), including evaluation of the impact of treatment on HRQoL, offer valuable insights to supplement clinical endpoints and aid in therapeutic decision-making.…”