R. Arbuckle

The reliability, validity and responsiveness of the Restless Legs Syndrome Quality of Life questionnaire (RLSQoL) in a trial population

BackgroundThe aim of this study was to determine the reliability, validity and responsiveness of the Restless Legs Syndrome Quality of Life questionnaire (RLSQoL) in a clinical trial setting.MethodsTwo matching, placebo-controlled, multinational studies assessing the effectiveness and safety of ropinirole for treating moderate-to-severe Restless Legs Syndrome (RLS) formed the basis of this psychometric assessment. Validity and reliability were assessed using baseline data. Responsiveness was determined using longitudinal data collected at baseline and 12 weeks.ResultsA total of 547 subjects formed the baseline validation population; 519 were used for assessing responsiveness (n = 284/263 and 271/248 for both studies, respectively). Construct validity assessment confirmed that an overall life impact score could be calculated. All item-scale correlations were = 0.4, except items 1 (r = 0.36) and 5 (r = 0.35) in one study. Floor and ceiling effects were minimal. Cronbachs alpha values were 0.82 and 0.87, respectively, confirming internal consistency reliability. Correlations with the International Restless Legs Syndrome Study Groups severity rating scale (International Restless Legs Scale; IRLS) were moderate (r = -0.68 and -0.67, respectively; p < 0.0001). The RLSQoL was able to discriminate between levels of sleep problems (p < 0.0001) and between levels of global health status determined by a Clinical Global Impression of severity (CGI-S) (p < 0.0001). Responsiveness was demonstrated by significant differences in overall life impact change scores between CGI improvement levels after 12 weeks (p < 0.0001).ConclusionThe RLSQoL is a valid, reliable and responsive measure of quality of life for patients with RLS, in a clinical trial setting where group comparisons are anticipated.

Development of the Pediatric Restless Legs Syndrome Severity Scale (P-RLS-SS)©: A patient-reported outcome measure of pediatric RLS symptoms and impact

OBJECTIVE To develop a questionnaire to measure Pediatric Restless Legs Syndrome (P-RLS) symptoms and impact for use in clinical research. METHODS Questionnaire items were developed based on open-ended, qualitative interviews of 33 children and adolescents diagnosed with definite RLS (ages 6-17 years) and their parents. The draft questionnaire was then tested through cognitive debriefing interviews with 21 of the same children/adolescents and 15 of their parents. This involved the children and parents answering the draft items and then interviewing them about the childs ability to understand and interpret the questionnaire. Expert clinicians provided clinical guidance throughout. RESULTS Draft severity questions were generated to measure the four-symptom and four-impact domains identified from the concept elicitation interviews: RLS sensations, move/rub due to RLS, relief from move/rub, pain, and impact of RLS on sleep, awake activities, emotions, and tiredness. RLS descriptions, symptoms, and impact were compared between those who had comorbid attention-deficit/hyperactivity disorder and those who did not. Revisions to several questions were made based on the cognitive debriefing interviews and expert clinician review, resulting in a severity scale with 17 morning and 24 evening items. Caution regarding self-administration in children ages 6-8 years is recommended. To complement the child/adolescent measures, a separate parent questionnaire was also developed. CONCLUSIONS The P-RLS-SS was constructed based on detailed input from children and adolescents with RLS, their parents, and clinical experts, thus providing a scale with strong content validity that is intended to be comprehensive, clinically relevant, and important to patients. Validation of this scale is recommended.

Pediatric Restless Legs Syndrome: Analysis of Symptom Descriptions and Drawings

The specific aims of this study were to collect and analyze detailed symptom descriptions from patients with pediatric restless legs syndrome, ages 6 to 17 years, as well as assess symptom impact and the usefulness of drawings. Trained qualitative interviewers conducted face-to-face audio-recorded interviews of children and adolescents who met criteria for definite restless legs syndrome. Thirty-three patients in 3 age groups used 16 different categories of descriptors for restless legs sensations, with a mean of 3 or more categories used per patient in each age group. “Need to move/kick,” “pain/hurts,” “uncomfortable/cannot get comfortable,” and “like bugs or ants/crawling” were the most common descriptors. Two-thirds reported daytime sensations, and nearly half had arm involvement. They described impact on sleep, cognitive function, and affect. Drawings provided useful diagnostic information. These detailed empirical data will be useful in clinical practice, as well as in the development of formal diagnostic tools and severity measures.

Patient experiences with oily skin: The qualitative development of content for two new patient reported outcome questionnaires

ObjectiveTo develop the content for two new patient reported outcome (PRO) measures to: a) assess the severity of symptoms; and b) the impact of facial skin oiliness on emotional wellbeing using qualitative data from face to face, and internet focus groups in Germany and the US.MethodsUsing input from initial treatment satisfaction focus groups (n = 42), a review of relevant literature and expert clinicians (n = 3), a discussion guide was developed to guide qualitative inquiry using Internet focus groups (IFGs). IFGs were conducted with German (n = 26) and US (n = 28) sufferers of oily skin. Questionnaire items were generated using coded transcript data from the focus groups. Cognitive debriefing was conducted online with 42 participants and face to face with an additional five participants to assess the comprehension of the items.ResultsThere were equal numbers of male and female participants; mean age was 35.4 (SD 9.3) years. On average, participants had had oily skin for 15.2 years, and 74% (n = 40) reported having mild-moderate acne. Participants reported using visual, tactile and sensory (feel without touching their face) methods to evaluate the severity of facial oiliness. Oily facial skin had both an emotional and social impact, and was associated with feelings of unattractiveness, self-consciousness, embarrassment, irritation and frustration. Items were generated for a measure of oily skin severity (Oily Skin Self-Assessment Scale) and a measure of the impact of oily skin on emotional well-being (Oily Skin Impact Scale). Cognitive debriefing resulted in minor changes to the draft items and confirmed their face and content validity.ConclusionThe research provides insight into the experience of having oily skin and illustrates significant difficulties associated with the condition. Item content was developed for early versions of two PRO measures of the symptoms and emotional impact of oily facial skin. The psychometric validation of these measures reported elsewhere.

Item Reduction and Psychometric Validation of the Oily Skin Self Assessment Scale (OSSAS) and the Oily Skin Impact Scale (OSIS)

INTRODUCTION Developed using focus groups, the Oily Skin Self Assessment Scale (OSSAS) and Oily Skin Impact Scale (OSIS) are patient-reported outcome measures of oily facial skin. OBJECTIVE The aim of this study was to finalize the item-scale structure of the instruments and perform psychometric validation in adults with self-reported oily facial skin. METHODS The OSSAS and OSIS were administered to 202 adult subjects with oily facial skin in the United States. A subgroup of 152 subjects returned, 4 to 10 days later, for test–retest reliability evaluation. RESULTS Of the 202 participants, 72.8% were female; 64.4% had self-reported nonsevere acne. Item reduction resulted in a 14-item OSSAS with Sensation (five items), Tactile (four items) and Visual (four items) domains, a single blotting item, and an overall oiliness item. The OSIS was reduced to two three-item domains assessing Annoyance and Self-Image. Confirmatory factor analysis supported the construct validity of the final item-scale structures. The OSSAS and OSIS scales had acceptable item convergent validity (item-scale correlations >0.40) and floor and ceiling effects (<20%). Cronbachs alpha coefficients ranged from 0.83 to 0.89 for the OSSAS and 0.82 to 0.87 for the OSIS, demonstrating excellent internal consistency. The a priori test–retest reliability criterion (intraclass correlation [ICC] ≥0.7) was met for one of the three OSSAS domains and one of the two OSIS domains. OSSAS and OSIS domains distinguished among groups that differed in patient-reported facial oily skin severity (P < 0.0001), and bother associated with oily skin (P < 0.0001). CONCLUSIONS The OSSAS and OSIS versions tested in this study have been found to have strong psychometric properties in this patient sample (adults with self-reported oily facial skin), as assessments of self-reported oily facial skin severity and its emotional impact, respectively.

Measuring the Symptoms of Pediatric Constipation and Irritable Bowel Syndrome with Constipation: Expert Commentary and Literature Review

BackgroundSymptom measurement in pediatric chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C) trials requires appropriately developed clinical outcome assessments (COAs).MethodsLiterature was reviewed to identify symptom COAs meeting regulatory standards. Searches were conducted in Pubmed/Medline, EMBASE, and PsychINFO. Title/abstracts were reviewed to identify qualitative studies and those using COAs to measure pediatric CIC/IBS symptoms. Pediatric functional gastrointestinal experts provided input on relevant symptom-concepts to measure.ResultsReview of 1,105 abstracts identified 1 relevant qualitative article and 113 articles including COAs. Symptoms most frequently measured in CIC studies were frequency of bowel movements, fecal incontinence/encopresis, abdominal pain, stool consistency, and painful defecation. Symptoms most frequently measured in IBS were abdominal pain, abdominal distention/bloating, stool consistency, frequency of bowel movements, and gas. Evidence of development/validity of COAs was limited. Expert feedback was broadly consistent with the literature.ConclusionFindings demonstrate consistency in the literature on key CIC/IBS symptoms to measure in pediatric trials, but existing COAs do not meet regulatory standards.

PNL19 VALIDATING THE RESTLESS LEGS SYNDROME QUALITY OF LIFE QUESTIONNAIRE (RLSQOL) IN A TRIAL PATIENT POPULATION

Patients previously diagnosed, suffering from a psychotic disorder or unable to follow-up the study were excluded. Three visits were conducted per protocol (baseline, 6 and 12 months). RESULTS: A total of 921 patients were included in the analysis. Mean (SD) age was 74.3 (6.8) years and 66.9% were women. First suspected diagnosis were: age related memory impairment (17.5% of patients), cognitive impairment (21.9%), dementia (29.2%), and psychopathological disorders (14.9%). Diagnostic methods used were: anamnesis (96,2% of patients); physical and neurological examination (87,5% and 85.6%), screening and laboratory test (85.9% and 89%). In all, 630 patients attended to at least one visit “out of protocol” to their PCP during the follow-up period. Mean (SD) number of visits was 4.28 (3.1). Percentile 25, 50, and 75 were 2, 3, and 6 visits respectively. The visits were previously appointed by physicians (54.7%) or were patients spontaneous consultations (39.7%). Main reason for “out of protocol” visits were to evaluate patient evolution. 45.1% of patients were never derived, 36.9% and 18% were derived to specialist once, or more than once, respectively. Main reason was diagnostic confirmation. CONCLUSION: Initial visit of patients with memory complaints or cognitive impairment to PCP, originate a careful follow-up of the patient. Different number of diagnostic methods and high time consumer procedures (as anamnesis, or screening test) have been applied. Derivations to specialist seem to be low.

Qualitative Interviews To Provide In-Depth Understanding Of The Impact Of Non-Small Cell Lung Cancer (Nsclc) And Its Treatment On Of The Lives Of Patients And Their Families/Caregivers.

>> Lung cancer is one of the leading causes of cancer-related deaths worldwide. Non-small cell lung cancer (NSCLC) accounts for approximately 85% of all lung cancers and has a 5 year survival rate of approximately 8%1. >> NSCLC is associated with the symptoms pain, breathlessness, hoarseness and coughing, which can be present prior to diagnosis and continue throughout the course of the disease, adversely affecting patients’ functional status and quality of life.3-22 >> Treatment is associated with severe side effects5,8,10 and can be logistically burdensome in terms of the time required to attend appointments.10,23 In addition, there is considerable burden placed on the informal caregivers and families who provide physical, emotional, financial and logistical support to patients with NSCLC.9,15,16,18,19,23-27 >> In addition to overall and progression free survival; the improvement, maintenance or delayed deterioration of health related quality of life (HRQoL) are desirable outcomes of any lung cancer treatment. When assessing HRQoL in clinical trials it is critical that the instruments being used adequately capture the concepts which are most important to patients and that have the greatest impact on their quality of life, in addition to being valid, reliable and sensitive to changes over time.28,29

Evaluation of a crosswalk between the European Quality of Life Five Dimension Five Level and the Menopause-Specific Quality of Life questionnaire

Abstract Objectives: Postmenopausal (PM) women taking therapies using estrogens plus progestogens (EPTs) can experience side effects (breast pain, vaginal spotting/bleeding). Sensitivity of the European Quality of Life Five Dimension Five Level (EQ-5D-5L) in measuring quality of life of PM women experiencing side effects of EPTs is unknown. A crosswalk between the Menopause-Specific Quality of Life (MENQOL) questionnaire and the EQ-5D-5L was assessed. Methods: The measures were administered to 352 PM women (side effects = 202; control = 75; untreated = 75) in a non-interventional study. MENQOL total scores, treated as continuous and categorical predictors, were mapped onto EQ-5D-5L utilities using regression. Ordinary least-squares regression using averaged scores over time, goodness of fit, and estimated coefficients was also assessed. Results: Mean age was 53.7 years. The first model (MENQOL as a continuous variable) showed a moderate correlation (−0.589) and statistically significant relationship with the EQ-5D-5L (p < 0.001), with an equation of EQ-5D-5L = 0.992 − 0.042 × MENQOL. The EQ-5D-5L mean scores were comparable (side effects = 0.854; control = 0.927; untreated = 0.836) to MENQOL mean scores estimated in the first model (side effects = 0.865 [standard deviation 0.07]; control = 0.909; untreated = 0.833). Linearity assumptions were supported with MENQOL scores as a categorical predictor. Goodness of fit was moderate (R2 = 0.347; root mean squared error = 0.093). Conclusion: The crosswalk supports conversion of MENQOL scores to EQ-5D-5L-derived health utilities for group-level analyses in PM women.

Translatability Assessment and Linguistic Validation of The Patient-Reported Outcome Instrument for Irritable Bowel Syndrome With Diarrhea (Ibs-D Pro)

1Astellas Pharma Global Development, 1 Astellas Way, Northbrook, IL, USA; 2Pharmerit International, 275 Grove Street, Newton, MA, USA (formerly of Adelphi Values, Boston, MA, USA); 3TransPerfect, 100 High Street, Boston, MA, USA; 4Astellas Pharma Inc., 2-5-1, Nihonbashi-Honcho, Chuo-ku, Tokyo 103-8411, Japan; 5Adelphi Values, Adelphi Mill, Grimshaw Lane, Bollington, Cheshire, UK, 6Adelphi Values, 290 Congress Street, Boston, MA, USA.