How are patient-reported outcomes and symptoms being measured in adults with relapsed/refractory multiple myeloma? A systematic review

LeBlanc, Matthew Roger; Hirschey, Rachel; Bryant, Ashley Leak; LeBlanc, Thomas W.; Smith, Sophia K.

doi:10.1007/s11136-019-02392-6

Cited by 35 publications

(42 citation statements)

References 70 publications

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“…PROs are important for evaluating patients' HRQoL, but are often suboptimally reported, with deficits including failure to account for missing data from analyses, potential measurement bias, and missed reporting criteria that can potentially improve future PRO studies 2 . The impact of treatment with Rd‐based regimens on PROs in patients with RRMM has been studied previously; overall, and similar to POLLUX, these studies demonstrated maintenance of HRQoL and few differences in HRQoL outcomes between experimental and control arms when on treatment.…”

Section: Discussionmentioning

confidence: 99%

Health‐related quality of life in patients with relapsed or refractory multiple myeloma: treatment with daratumumab, lenalidomide, and dexamethasone in the phase 3 POLLUX trial

et al. 2021

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Summary In the phase 3 POLLUX trial, daratumumab in combination with lenalidomide and dexamethasone (D‐Rd) significantly improved progression‐free survival in patients with relapsed/refractory multiple myeloma (RRMM) compared with lenalidomide and dexamethasone (Rd) alone. Here, we present patient‐reported outcomes (PROs) from POLLUX, assessed using the validated European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30‐item (EORTC QLQ‐C30) and the EuroQol 5‐dimensional descriptive system (EQ‐5D‐5L) questionnaires. Changes from baseline are presented as least‐squares mean changes with 95% confidence intervals (CIs) derived from a mixed‐effects model. PRO assessment compliance rates were high and similar in both D‐Rd and Rd groups through cycle 40 (week 156). In this on‐treatment analysis, mean changes from baseline were significantly greater in EORTC QLQ‐C30 global health status, physical functioning, and pain scores in the D‐Rd group versus the Rd group at multiple time points; however, magnitude of changes was low, suggesting no meaningful impact on health‐related quality of life (HRQoL). Subgroup results were similar to those in the overall population. In the POLLUX study, baseline HRQoL was maintained with prolonged D‐Rd treatment. These findings complement the sustained and significant improvement in progression‐free survival observed with D‐Rd and supports its use in patients with RRMM. Clinical trial registration: NCT02076009.

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Section: Discussionmentioning

confidence: 99%

Health‐related quality of life in patients with relapsed or refractory multiple myeloma: treatment with daratumumab, lenalidomide, and dexamethasone in the phase 3 POLLUX trial

et al. 2021

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“…PROs are an increasingly important metric for evaluating MM treatment, but are inconsistently and sub-optimally reported; drawing conclusions is often impossible because of the limited information reported. 13 There are few published studies regarding the impact of Vd-based regimens on HRQoL in patients with RRMM. In general, rather than improvement, these studies have found no change from baseline HRQoL.…”

Section: Discussionmentioning

confidence: 99%

“…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.…”

Section: Introductionmentioning

confidence: 99%

“…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. 1,3,11,13 Daratumumab is an anti-CD38 monoclonal antibody with a direct on-tumour [15][16][17][18] and immunomodulatory [19][20][21] mechanism of action; daratumumab is approved by the US Food and Drug Administration in patients with relapsed or refractory MM (RRMM) as monotherapy or in combination with other agents. 22 The phase III CASTOR study (NCT02136134) compared daratumumab in combination with bortezomib and dexamethasone (D-Vd) with bortezomib and dexamethasone (Vd) alone in patients who had a median of two prior lines of therapy.…”

Section: Introductionmentioning

confidence: 99%

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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

Hungria¹,

Beksaç

Weisel

et al. 2021

Br J Haematol

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In the phase III CASTOR trial, daratumumab, bortezomib and dexamethasone (D-Vd) significantly extended progression-free survival compared with bortezomib and dexamethasone (Vd) alone in patients with relapsed/refractory multiple myeloma (RRMM). Here, we present patient-reported outcomes (PROs) from the CASTOR trial. PROs were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30-item (EORTC QLQ-C30) and the EuroQol 5dimensional descriptive system questionnaire. Treatment effects through Cycle 8 were measured by a repeated measures mixed-effects model. After Cycle 8, PROs were only collected for patients in the D-Vd group who continued on daratumumab monotherapy. Compliance rates for PRO assessments were high and similar between treatment groups. Mean changes from baseline were generally similar between treatment groups for EORTC QLQ-C30 global health status (GHS), functioning and symptoms, and did not exceed 10 points for either treatment group. Subgroup analyses were consistent with the results observed in the overall population. There was no change in patients' health-related quality of life for the first eight cycles of therapy; thereafter, patients treated with daratumumab over the long-term reported improvements in GHS and pain. These results complement the significant clinical benefits observed with D-Vd in patients with RRMM and support its use in this patient population.

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“…This may indicate that the POM/DEX treatment regimen does not adversely affect patients QoL, which was also suggested from the MM‐003 trial, in which POM/DEX even led to improved QoL compared to DEX alone, 31 although the analyzed questionnaire domains and the survey periods in the MM‐003 differed slightly from those analyzed in the present study. Control and maintenance of treatment‐related impacts on QoL have been rated as particularly important in patients with R/RMM who have received many lines of therapies increasing the risk of cumulative toxicities and side effects 32 …”

Section: Discussionmentioning

confidence: 99%

Pomalidomide plus dexamethasone for patients with relapsed or refractory multiple myeloma: Final results of the non‐interventional study POSEIDON and comparison with the pivotal phase 3 clinical trials

Dechow

Aldaoud

Behlendorf

et al. 2021

European J of Haematology

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Background Prognosis of patients with multiple myeloma (MM) who have relapsed on or become refractory to immunomodulators and bortezomib is poor, and treatment options are limited. While pomalidomide plus low‐dose dexamethasone (POM/DEX) has demonstrated efficacy in clinical trials, real‐world evidence is scarce. Patients and Methods POSEIDON was a prospective non‐interventional study designed to evaluate effectiveness, safety and quality of life (QoL) of POM/DEX in patients with relapsed or refractory MM (R/RMM) pretreated with at least two prior therapy lines including both lenalidomide and bortezomib in real world in Germany. Patients received POM/DEX according to physicians’ choice. Data were analyzed descriptively. Results Between 2014 and 2017, 151 patients were enrolled, 144 patients with a median of three prior therapy lines qualified for effectiveness analysis. Median age was 73.2 years. Median progression‐free and overall survival were 6.3 months [95% confidence interval (CI) 5.2, 8.6] and 12.9 months [95% CI 10.6, 15.1]. Most frequent grade 3/4 adverse events were leukopenia (8.2%), pneumonia (7.5%) and anemia (5.5%). QoL was maintained after start of POM/DEX. Conclusion The results of POSEIDON support the effectiveness and safety of POM/DEX in R/RMM patients pretreated with lenalidomide and bortezomib and highlight the clinical value of the POM/DEX regimen in the real‐world setting. Registered at clinicaltrials.gov (NCT02075996).

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How are patient-reported outcomes and symptoms being measured in adults with relapsed/refractory multiple myeloma? A systematic review

Cited by 35 publications

References 70 publications

Health‐related quality of life in patients with relapsed or refractory multiple myeloma: treatment with daratumumab, lenalidomide, and dexamethasone in the phase 3 POLLUX trial

Health‐related quality of life in patients with relapsed or refractory multiple myeloma: treatment with daratumumab, lenalidomide, and dexamethasone in the phase 3 POLLUX trial

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

Pomalidomide plus dexamethasone for patients with relapsed or refractory multiple myeloma: Final results of the non‐interventional study POSEIDON and comparison with the pivotal phase 3 clinical trials

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