Symptom burden and its functional impact in patients with "symptomatic" relapsed or refractory multiple myeloma.

PURPOSE: Relapsed or refractory multiple myeloma (RRMM) is labeled "symptomatic" based on laboratory values, but not relevant to quantitative measure of patient's perspectives. This study aimed to describe symptom burden, health status, and quality of life in RRMM patients. METHODS: The cross-sectional study included 184 MM patients (141 RRMM cases and 43 MM patients on follow-up without diagnosis/treatment of RRMM disease as controls), while 64 RRMM patients also provided longitudinal patient-reported outcomes (PROs) data. Symptomatic status was based on clinical measures of disease activity. PROs included the MD Anderson Symptom Inventory multiple myeloma module (MDASI-MM), single-item quality of life (SIQOL), and EuroQol-5D (EQ-5D). Wilcoxon rank test and effect size were used for comparisons. Regression models were used to describe symptom trajectory and to identify predictors of high symptom burden during 3 months of RRMM therapy. RESULTS: Most patients were clinically identified as symptomatic (93%). RRMM patients tended to report more severe symptoms, with significantly lower QOL scores and more severe fatigue, poor appetite, and lower enjoyment of life compared with controls (all p < 0.05). In RRMM patients, lower hemoglobin and higher B-2 microglobulin levels significantly correlated with higher burdens of fatigue, pain, and muscle weakness and also with lower QOL and EQ-5D scores (all p < 0.05). During RRMM therapy, being female, with any comorbidity, ≥ 65 years old, and ≥ 5 years MM history, contributed to high symptoms burden and poor QOL status (each p < 0.05). CONCLUSIONS: MDASI-MM modules were sensitive to detect the RRMM-related symptoms burden, which correlated with objective clinical measures. RRMM patients reported a more compromised QOL.

Tumor mutational burden in lung cancer: a systematic literature review.

Purpose: To assess the association of tumor mutational burden (TMB) with clinical outcomes, other biomarkers and patient/disease characteristics in patients receiving therapy for lung cancer. Results: In total, 4,303 publications were identified; 81 publications were included. The majority of publications assessing clinical efficacy of immunotherapy reported an association with high TMB, particularly when assessing progression-free survival and objective response rate. High TMB was consistently associated with TP53 alterations, and negatively associated with EGFR mutations. High TMB was also associated with smoking, squamous cell non-small cell lung carcinoma, and being male. Methods: A systematic literature review based upon an a priori protocol was conducted following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) and Cochrane methodologies. Searches were conducted in EMBASE, SCOPUS, Ovid MEDLINE®, and Emcare (from January 2012 until April 2018) and in two clinical trial registries. Conference abstracts were identified in EMBASE, and in targeted searches of recent major conference proceedings (from January 2016 until April 2018). Publications reporting data in patients receiving therapy for lung cancer that reported TMB and its association with clinical efficacy, or with other biomarkers or patient/disease characteristics, were included. Results are presented descriptively. Conclusion: This systematic literature review identified several clinical outcomes, biomarkers, and patient/disease characteristics associated with high TMB, and highlights the need for standardized definitions and testing practices. Further studies using standardized methodology are required to inform treatment decisions.

Lost Labor Productivity Costs of Prostate Cancer to Patients and Their Spouses: Evidence From US National Survey Data.

OBJECTIVE: The aim of the study is to estimate lost labor productivity costs of prostate cancer (PC) to patients and their spouses. METHODS: This study used a nationally representative database from the United States, the Medical Expenditure Panel Survey, to estimate lost productivity costs attributable to PC for patients and their spouses. We used multivariate methods that controlled for sociodemographic factors and comorbid diseases. Sensitivity analyses were used to mitigate the tendency for prevalence rates to be underreported in surveys. RESULTS: PC patients had an aggregate national annual lost productivity cost of $5.4 billion ($3601 per individual), whereas their spouses had an aggregate annual lost productivity cost of $3.0 billion ($4013 per individual). CONCLUSIONS: These results enhance our knowledge of lost labor productivity costs of PC morbidity and may inform the management and treatment of PC from an employer's perspective.

The Direct Healthcare Expenditures of Prostate Cancer by Disease Severity: Evidence from US National Survey Data.

Background: In the United States, approximately 2.8 million men have a history of prostate cancer (PC). Objective: This study quantified the effects of PC, overall and by disease severity on direct healthcare costs to insurers and patients. Methods: Using 1996-2010 data from the Medical Expenditure Panel Survey (MEPS), a large, nationally representative US database, multivariate analyses were used to assess the relationship between PC and direct annual healthcare costs to insurers and patients, at individual and US aggregate levels. Men aged 40 years and older with International Classification of Diseases, Ninth Revision (ICD-9) diagnosis code 185 were identified. Disease severity was determined with clinical assistance and based, in part, on the data in MEPS. The cohorts were: localized cancer not treated with chemotherapy, localized cancer treated with chemotherapy, and metastatic cancer. Results: The MEPS database included 1297 patients with PC: 811 patients with localized PC not treated with chemotherapy, 426 patients with PC treated with chemotherapy, and 60 patients with metastatic PC. PC had a larger effect on incremental costs for metastatic patients, $20 357, vs $16 709 for localized PC with chemotherapy, and $5238 for localized PC with no chemotherapy. When aggregated to the US population, PC accounted for an incremental annual cost of $15 billion. The largest aggregate annual costs were incurred by patients with localized PC treated with chemotherapy ($8.6 billion), compared to those not treated with chemotherapy ($4.8 billion) and metastatic patients ($1.6 billion). Conclusions: The aggregate annual costs of PC are substantial for all groups examined and greatest for patients with localized cancer treated with chemotherapy. This reflects the relatively high prevalence and high per capita healthcare expenditures associated with this group. With a growing and aging population, the prevalence of PC is expected to rise, increasing the burden on public health.

A review of quality-of-life questionnaires for urinary incontinence and overactive bladder: which ones to use and why?

Overactive bladder (OAB) and stress urinary incontinence have a profound impact on patients' health-related quality of life (HRQL). The purpose of this article is to identify and describe condition-specific HRQL measures validated among patients with symptoms of OAB or stress urinary incontinence. A Medline literature search was performed to identify published articles or abstracts focusing on the development and psychometric validation of relevant instruments. Target populations and psychometric properties (reliability, validity, responsiveness to change) of 16 questionnaires are reviewed. A range of well-validated, condition-specific HRQL measures are available. Recommendations are provided regarding which measures to use in different situations. When choosing among instruments, psychometric evidence and the match of an instrument to the study population should be considered.