Katy Wortman

Neuro-QOL Brief measures of health-related quality of life for clinical research in neurology

Objective: To address the need for brief, reliable, valid, and standardized quality of life (QOL) assessment applicable across neurologic conditions. Methods: Drawing from larger calibrated item banks, we developed short measures (8–9 items each) of 13 different QOL domains across physical, mental, and social health and evaluated their validity and reliability. Three samples were utilized during short form development: general population (Internet-based, n = 2,113); clinical panel (Internet-based, n = 553); and clinical outpatient (clinic-based, n = 581). All short forms are expressed as T scores with a mean of 50 and SD of 10. Results: Internal consistency (Cronbach α) of the 13 short forms ranged from 0.85 to 0.97. Correlations between short form and full-length item bank scores ranged from 0.88 to 0.99 (0.82–0.96 after removing common items from banks). Online respondents were asked whether they had any of 19 different chronic health conditions, and whether or not those reported conditions interfered with ability to function normally. All short forms, across physical, mental, and social health, were able to separate people who reported no health condition from those who reported 1–2 or 3 or more. In addition, scores on all 13 domains were worse for people who acknowledged being limited by the health conditions they reported, compared to those who reported conditions but were not limited by them. Conclusion: These 13 brief measures of self-reported QOL are reliable and show preliminary evidence of concurrent validity inasmuch as they differentiate people based upon number of reported health conditions and whether those reported conditions impede normal function.

Ambulatory Cancer and US General Population Reference Values and Cutoff Scores for the Functional Assessment of Cancer Therapy

Health‐related quality of life (HRQOL) measures are commonly used in oncology research. Interest in their use for monitoring or screening is increasing. The Functional Assessment of Cancer Therapy (FACT) is one of the most widely used HRQOL instruments. Consequently, oncology researchers and practitioners have an increasing need for reference values for the Functional Assessment of Cancer Therapy–General (FACT‐G) and its 7‐item rapid version, the Functional Assessment of Cancer Therapy–General 7 (FACT‐G7), to compare FACT scores across specific subgroups of patients in research trials and practice. The objectives of this study are to provide 1) reference values from a sample of the general US adult population and a sample of adults diagnosed with cancer and 2) cutoff scores for quality of life.

Phase I/II trial of vorinostat with rituximab, cyclophosphamide, etoposide and prednisone as palliative treatment for elderly patients with relapsed or refractory diffuse large B-cell lymphoma not eligible for autologous stem cell transplantation.

The standard treatment of relapsed/refractory diffuse large B‐cell lymphoma (DLBCL) in frail elderly patients has not been established. A variation was made on rituximab (R), cyclophosphamide (C), etoposide (E), procarbazine and prednisone (P), substituting vorinostat (V) for procarbazine. Patients ≥aged 60 years with relapsed/refractory DLBCL, not candidates for autologous stem cell transplantation, were treated R‐CVEP [R 375 mg/m2 intravenously (IV), day 1; C 600 mg/m2 IV days 1, 8: E 70 mg/m2 IV day 1, 140 mg/m2 days 2, 3 orally (PO); V (300 vs. 400 mg) PO and P 60 mg/m2 PO days 1–10] every 28 d for six cycles. Quality of life (QoL) was assessed in addition to response. Thirty patients (median age 76 years, 69–88) were enrolled (one died before treatment). Maximum tolerated dose (MTD) for V was 300 mg. For 23 patients at MTD (six phase I + 17 phase II), two were discontinued for toxicity, one withdrew consent, eight achieved complete response (35%), five achieved partial response (22%) and seven progressed (25%). Median overall survival was 17·5 months. Median progression‐free survival was 9·2 months. Nine patients are alive. QoL declined during treatment but improved above baseline for patients who completed treatment. In conclusion, R‐CVEP was tolerated at MTD and produced durable responses with improved QoL.

Validating Neuro-QoL short forms and targeted scales with people who have multiple sclerosis

Background: Multiple sclerosis (MS) is a chronic, progressive, and disabling disease of the central nervous system with dramatic variations in the combination and severity of symptoms it can produce. The lack of reliable disease-specific health-related quality of life (HRQL) measures for use in clinical trials prompted the development of the Neurology Quality of Life (Neuro-QOL) instrument, which includes 13 scales that assess physical, emotional, cognitive, and social domains, for use in a variety of neurological illnesses. Objective: The objective of this research paper is to conduct an initial assessment of the reliability and validation of the Neuro-QOL short forms (SFs) in MS. Methods: We assessed reliability, concurrent validity, known groups validity, and responsiveness between cross-sectional and longitudinal data in 161 recruited MS patients. Results: Internal consistency was high for all measures (α = 0.81–0.95) and ICCs were within the acceptable range (0.76–0.91); concurrent and known groups validity were highest with the Global HRQL question. Longitudinal assessment was limited by the lack of disease progression in the group. Conclusions: The Neuro-QOL SFs demonstrate good internal consistency, test-re-test reliability, and concurrent and known groups validity in this MS population, supporting the validity of Neuro-QOL in adults with MS.

Self-Reported Cancer Prevalence among Hispanics in the US: Results from the Hispanic Community Health Study/Study of Latinos

Cancer has surpassed heart disease as the leading cause of death among Hispanics in the U.S., yet data on cancer prevalence and risk factors in Hispanics in regard to ancestry remain scarce. This study sought to describe (a) the prevalence of cancer among Hispanics from four major U.S. metropolitan areas, (b) cancer prevalence across Hispanic ancestry, and (c) identify correlates of self-reported cancer prevalence. Participants were 16,415 individuals from the Hispanic Community Health Study/Study of Latinos (HCHS/SOL), who self-identified as Cuban, Dominican, Mexican, Puerto Rican, Central or South American. All data were collected at a single time point during the HCHS/SOL baseline clinic visit. The overall self-reported prevalence rate of cancer for the population was 4%. The rates varied by Hispanic ancestry group, with individuals of Cuban and Puerto Rican ancestry reporting the highest cancer prevalence. For the entire population, older age (OR = 1.47, p < .001, 95% CI, 1.26–1.71) and having health insurance (OR = 1.93, p < .001, 95% CI, 1.42–2.62) were all significantly associated with greater prevalence, whereas male sex was associated with lower prevalence (OR = 0.56, p < .01, 95% CI, .40-.79). Associations between study covariates and cancer prevalence also varied by Hispanic ancestry. Findings underscore the importance of sociodemographic factors and health insurance in relation to cancer prevalence for Hispanics and highlight variations in cancer prevalence across Hispanic ancestry groups. Characterizing differences in cancer prevalence rates and their correlates is critical to the development and implementation of effective prevention strategies across distinct Hispanic ancestry groups.

Examining Web Equivalence and Risk Factor Sensitivity of the COPD Population Screener

OBJECTIVES The primary aim was to assess the equivalence of an Internet-based chronic obstructive pulmonary disease-population screener (COPD-PS) relative to a validated paper-and-pencil version. A secondary aim was to compare groups based on known COPD risk factors, such as smoking status and gender. METHODS Using an online panel survey organization, participants were randomized to internet or paper-and-pencil assessment where they completed the COPD-PS and other study forms. A subset of respondents also completed a test-retest reliability assessment. Finally, several thousand additional online respondents completed the COPD-PS for risk factor analyses. RESULTS A total of 1006 adults completed the randomized study (N = 504 online, N = 502 by mail). There were no differences between the arms in mean COPD-PS scores (mean difference: 0.12; 95% confidence interval: -0.14-+0.37; P = 0.365). In the web arm, 106/504 (21.0%) exceeded the screening cut-off compared to 101/502 (20.1%) in the paper-administration arm (difference in proportions: 0.9%; 95% confidence interval: -4.1%-+5.9%; P = 0.720). Subgroup analyses on a separate cohort of 3001 adults demonstrated hypothesized differences between groups defined by smoking status, presence of COPD, and shortness of breath. CONCLUSION The methods of administration that were evaluated in this study (internet vs. paper and pencil) resulted in no significant differences in COPD-PS mean scores. Furthermore, the predictive utility of the COPD-PS was not different between methods of administration, even after accounting for age and smoking status.

Brief versions of the FACIT-fatigue and FAACT subscales for patients with non-small cell lung cancer cachexia.

259 Background: Cancer anorexia-cachexia syndrome (CACS) is common in advanced cancer patients and associated with weight loss, fatigue, impaired quality of life (QoL), and poor prognosis. The goal of this project was to identify the most responsive items and evaluate their validity from two QoL measures in the ROMANA 2 (NCT01387282) Phase III global study evaluating anamorelin HCl in the treatment of non-small cell lung cancer (NSCLC) cachexia: the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and the Functional Assessment of Anorexia/ Cachexia Therapy (FAACT). METHODS In the ROMANA 2 trial, 477 patients with unresectable Stage III or IV NSCLC and cachexia were to be enrolled and randomized (2:1) to receive anamorelin HCl or placebo once daily for 12 weeks. All 383 patients who reached the Week 12 visit at the time of this blinded data analysis were included. Co-primary endpoints were change from baseline in lean body mass and handgrip strength. QoL was a key secondary outcome with FACIT-F and FAACT questionnaires administered at baseline and at weeks 3, 6, 9, and 12. RESULTS Two 4-item scales (referred to as the Simplified Evaluation of Fatigue [SEF] & Simplified Evaluation of Appetite [SEA]) from the FACIT-F and FAACT, respectively, demonstrated good internal consistency reliability with Cronbachs alphas >0.70. In analysis of known group validity, scores significantly differed between groups defined by performance status and appetite (with effect sizes ≥0.34). Convergent validity analysis showed significant correlations with general QoL/functioning scales (correlation coefficients 0.41-0.77), further demonstrating validity. In terms of responsiveness, changes from baseline to week 12 in SEF and SEA scores were associated with changes in general QoL/ functioning, appetite, weight, and lean body mass (correlations >0.20). The estimated important difference for each scale was ~1-2 points. CONCLUSIONS These brief scales are valid and responsive measures that provide psychometric properties necessary to promote future research in NSCLC patients with CACS. Additional work should examine clinical utility of these scales and their impact on treatment decision-making.