Understanding the patient experience and treatment benefits in patients with non-small-cell lung cancer with brain metastasis.

BACKGROUND: Despite the high prevalence of brain metastases (BM) secondary to non-small-cell lung cancer (NSCLC) (NSCLC/BM), patients' experiences (symptoms and impacts) are not fully understood. This study sought to understand the patient experience with NSCLC/BM and identify a patient-reported outcome (PRO) measure fit to capture the most important NSCLC/BM symptoms and impacts. METHODS: A targeted literature review was completed; the National Comprehensive Cancer Network (NCCN)/Functional Assessment of Cancer Therapy-Brain Symptom Index, 24-item version (NFBrSI-24) was identified as a relevant measure that assessed the core symptoms and impacts associated with NSCLC/BM. Qualitative interviews composed of concept elicitation and cognitive debriefing with oncologists (n = 3) and adult patients (n = 16) with NSCLC/BM were conducted to confirm the content validity and evaluate the relevance and appropriateness of the NFBrSI-24 for this condition. RESULTS: The NSCLC/BM symptoms and impacts identified in the literature and reported by oncologists and patients were consistent and captured in the NFBrSI-24. Study participants reported significant burden associated with the symptoms (commonly fatigue, headache) and impacts of NSCLC/BM. Participants indicated that the NFBrSI-24 captured their most salient experiences with NSCLC/BM and that symptom improvement or a delay in progression, as measured by the NFBrSI-24, would be meaningful. During the cognitive debriefing, participants generally indicated that the NFBrSI-24 was comprehensive and easy to understand/answer and that it assessed symptoms they considered most important to treat. CONCLUSIONS: These results suggest that the NFBrSI-24 adequately captures an appropriate measure of NSCLC/BM symptoms and impact.

Health-related quality of life in women with breast cancer: a review of measures.

BACKGROUND: To identify and describe the breast cancer-specific health-related quality of life (HRQoL) instruments with evidence of validation in the breast cancer population for potential use in patients treated for breast cancer (excluding surgery). METHODS: We conducted a systematic literature review using PubMed, Embase, and PsycINFO databases to identify articles that contain psychometric properties of HRQoL instruments used in patients with breast cancer. Relevant literature from January 1, 2009, to August 19, 2019, was searched. Articles published in English that reported psychometric properties (reliability, validity) of HRQoL instruments were identified. RESULTS: The database search yielded 613 unique records; 131 full-text articles were reviewed; 80 articles presented psychometric data for instruments used in breast cancer (including generic measures). This article reviews the 33 full articles describing psychometric properties of breast cancer-specific HRQoL instruments: EORTC QLQ-C30, EORTC QLQ-BR23, FACT-B, FBSI, NFBSI-16, YW-BCI36, BCSS, QuEST-Br, QLICP-BR, INA-BCHRQoL, and two newly developed unnamed measures, one by Deshpande and colleagues (for use in India) and one by Vanlemmens and colleagues (for use among young women and their partners). The articles that described the EORTC QLQ-C30, QLQ-BR23, and FACT-B centered on validating translations, providing additional support for content validity, and demonstrating acceptability of electronic patient-reported outcome administration. Psychometric properties of the measures were acceptable. Several new measures have been developed in Asia with an emphasis on development on cultural relevance/sensitivity. Others focused on specific populations (i.e., young women with breast cancer). CONCLUSIONS: Historically, there have been limited options for validated measures to assess HRQoL of patients with breast cancer. A number of new measures have been developed and validated, offering promising options for assessing HRQoL in this patient population. This review supports the reliability and validity of the EORTC QLQ-C30 and FACT-B; new translations and electronic versions of these measures further support their use for this population.

Secukinumab sustains early patient-reported outcome benefits through 1 year: Results from 2 phase III randomized placebo-controlled clinical trials comparing secukinumab with etanercept.

BACKGROUND: Psoriasis is a chronic condition with negative impact on patients' quality of life that most often requires lifelong effective and safe treatment. OBJECTIVE: This analysis focused on the effect of secukinumab treatment on patient-reported health-related quality of life as assessed by the Dermatology Life Quality Index (DLQI) in patients with moderate to severe psoriasis. METHODS: The proportion of subjects achieving DLQI score 0/1 response at week 24, time to DLQI score 0/1 response, and sustained DLQI score 0/1 response up to week 52 were compared between secukinumab and etanercept. RESULTS: Of 1470 subjects, 1144 received secukinumab and 326 received etanercept. DLQI score 0/1 response rates were significantly higher for secukinumab than for etanercept at week 24. The median time to DLQI score 0/1 response was significantly shorter for secukinumab versus etanercept (12 vs 24 weeks; P < .01). The majority of secukinumab-treated subjects achieved DLQI score 0/1 response at week 24 and sustained it through week 52 along with a 90% to 100% reduction in the Psoriasis Area and Severity Index total score response. LIMITATIONS: Placebo comparisons are limited during the maintenance period because of rerandomization at week 12. CONCLUSION: Secukinumab treatment provided faster and greater sustained improvements in quality of life than etanercept over 52 weeks, consistent with greater clinical response.

Evidence-based medicine, appropriate-use criteria, and sports medicine: how best to develop meaningful treatment guidelines.

We propose using appropriate-use criteria (AUC) as the methodology of choice for formulating and disseminating evidence-based medicine guidelines in sports medicine and arthroscopy. AUC provide a structured process for integrating findings from the scientific literature with clinical judgment to produce explicit criteria for determining the appropriateness of specific treatments. The use of AUC will enable surgeons to treat patients in a more consistent manner based on expert clinical consensus and evidence-based medicine. This methodology also will ensure that guidelines represent all stakeholders and available evidence.

Validation of the Knee Injury and Osteoarthritis Outcome Score subscales for patients with articular cartilage lesions of the knee.

BACKGROUND: The Knee Injury and Osteoarthritis Outcome Score (KOOS) assesses acute and chronic knee injuries or early-onset osteoarthritis in young, active patients. The United States Food and Drug Administration guidelines recommend that patient-reported outcome instruments used to support clinical trial label claims should demonstrate content validity using patient input and have acceptable psychometric properties in the target population. To use the KOOS subscales in safety and efficacy trials assessing new treatments for patients with articular cartilage lesions, additional validation work, using input from patients with articular cartilage lesions, was necessary. PURPOSE: Qualitative and quantitative evaluations of the KOOS subscales' validity among patients with articular cartilage lesions were conducted to support their use as clinically meaningful end points in clinical trials. STUDY DESIGN: Cohort study (diagnosis); Level of evidence, 2. METHODS: For qualitative analysis, cognitive interviews involving concept elicitation and cognitive debriefing with the KOOS items were conducted with 15 participants aged 25 to 52 years. Participants either were candidates for cartilage repair or had undergone cartilage repair 6 months or more before the study. For the quantitative analysis, a psychometric evaluation of the KOOS was conducted with clinical trial data from 54 patients, aged 18 to 55 years, evaluating the Cartilage Autograft Implantation System in the United States (n = 29) and the European Union (n = 25). Data were collected before surgery and at 7 postsurgical visits up to 12 months. Internal consistency and test-retest reliability, construct validity, responsiveness, and estimates of the minimal detectable change (MDC) were assessed. Test-retest reliability was assessed using data from months 2 and 3 on a subset of stable patients. RESULTS: Qualitative research confirmed that concepts measured on the KOOS are important to patients with articular cartilage lesions. Most participants reported the KOOS was comprehensive and appropriate. In the quantitative research, KOOS subscales showed excellent internal consistency reliability (range, .74-.97 at baseline) and test-retest reliability (range, .78-.82). Construct validity results supported hypothesized relationships, with significant correlations (r ≥ .50) in the expected directions. Responsiveness analyses demonstrated excellent sensitivity to change; standardized response means ranged from 0.8 to 1.2, and MDC estimates ranged from 7.4 to 12.1. CONCLUSION: The study results support the use of the KOOS subscales among patients with articular cartilage lesions.

Clinical staff resource use with intravenous patient-controlled analgesia in acute postoperative pain management: results from a multicenter, prospective, observational study.

The purpose of this study was to evaluate hospital resource utilization associated with intravenous patient-controlled analgesia (IV-PCA), with a focus on nursing, pharmacy, and central supply/engineering time spent from a hospital perspective. Data were collected during a multicenter (29 sites), prospective observational study in the United States of subjects who underwent total knee replacement (TKR), total hip replacement (THR), or abdominal hysterectomy (AH) and were administered analgesia through IV PCA for the management of acute postoperative pain. Nursing staff recorded the IV PCA-related tasks they performed for a subject and the duration of time required to perform each task from initial IV PCA set-up to discontinuation. Hospital administrators, nursing managers, central supply/engineering staff, and pharmacy directors were interviewed to obtain data regarding other IV PCA labor resource use. The distribution of surgery type among the 457 subjects was 31.1% THR, 35.9% TKR, and 33.0% AH. The average duration of IV PCA use was 32.6 hours. Nurses reported having to perform an average of 39.6 IV PCA-related tasks, which required an average of 67.4 minutes. The most common IV PCA-related tasks were evaluating pump use and settings, assessing the IV site, evaluating and addressing analgesia side effects, instructing/reinstructing the subject on use, administering supplemental pain medications, assisting with self-care or moving the subject, and assisting the subject with use of the button. Pharmacists reported that they spend approximately 7.9 minutes and pharmacy technicians spend approximately 9.8 minutes, per subject daily course of IV PCA therapy, on the following tasks: checking and verifying the order, doing inventory of the analgesia, preparing the analgesia (ie, filling reservoirs), checking the analgesia, and delivering the analgesia to the nursing units. In addition, pharmacists and RNs spend an average of 47.3 and 40.7 minutes per year in IV PCA-related training. Intravenous patient-controlled analgesia postoperative care requires coordination and involvement of numerous hospital departments. It is labor intensive and involves numerous time-consuming tasks, oversight of IV PCA, and ongoing training. Alternative methods of patient-controlled pain management with similar efficacy that reduces labor resource utilization may be warranted.