Development and content validation of a self-completed, electronic Pediatric Asthma Symptom Diary.

BACKGROUND: Childhood asthma is an important unmet need. To date, patient-reported outcome measures (PROMs) for children with asthma have used a combination of caregiver or proxy-reported and self-reported measures. No comprehensive measure is available to assess the severity and impact of daytime and nighttime asthma symptoms and rescue medication use for self-completion by children aged 6-11 years. This study aimed to develop a novel, interactive, electronic Pediatric Asthma Symptom Diary (ePASD) measuring self-reported key symptom severity and proximal impacts of asthma in young children with varying reading ability and disease severity, consistent with US Food and Drug Administration (FDA) PRO guidance and the International Society for Health Economics and Outcomes Research (ISPOR) good research practices. METHODS: A targeted literature review and clinician interviews were undertaken to characterize symptoms and impacts experienced by children with mild-to-severe asthma. Concept elicitation interviews (CEIs) were conducted with 44 children and their caregivers (30 US; 14 UK). Following item and digital application development, the ePASD was assessed for relevance, understanding, and interpretability through cognitive debriefing interviews (CDIs) with 21 US children. Face validity/translatability assessments were also performed. RESULTS: Key measurement concepts included cough, wheeze, difficulty breathing, chest tightness/discomfort, nighttime awakening, and daytime activity limitations. Concept saturation was reached during CEIs for primary asthma-related daytime and nighttime symptoms and core impacts. Most CDI participants found the ePASD items clear, understandable, and comprehensive. Standardized training is anticipated to facilitate reliable child self-report. CONCLUSION: The ePASD, a novel PROM for children aged 6-11 years with asthma, uses an innovative multimedia approach and has been developed in accordance with FDA PRO guidance and ISPOR good research practices, directly capturing the child's self-reported asthma symptoms, impacts on daily activities and nighttime awakening, and rescue medication use.

Real world effectiveness and satisfaction with secukinumab in the treatment of patients with psoriatic arthritis: a population survey in five European countries.

OBJECTIVES: To describe the effectiveness of secukinumab in the treatment of psoriatic arthritis (PsA) and associated physician satisfaction with secukinumab treatment, in routine clinical practice across five European countries. METHODS: A retrospective analysis of PsA patients receiving secukinumab for ≥4 months in France, Germany, Italy, Spain and the UK from March to December 2018. Data based on physician-completed questionnaires at initiation of treatment and at the data collection consultation were collected and used to assess effectiveness. RESULTS: 572 PsA patients with a mean age of 47.9 years, 57.0% were male, with 5.6% of patients with mild, 55.2% with moderate and 38.1% severe PsA prior to treatment initiation were included. 33.0% of patients received a dosage of 150 mg and 67.0% a dosage of 300 mg secukinumab. Around 84% of patients received secukinumab for 6 months or longer. Symptoms seen at current assessment in over 20% of patients were tender or swollen joints or psoriatic skin lesions. Between initiation of treatment and the current consultation, improvements in skin, joint and overall severity were reported. Physician satisfaction with secukinumab's ability to control disease was very high during the study period, greater than 90%, and was seen irrespective of disease severity at initiation, prior biologic use, treatment duration, time since diagnosis or onset of symptoms, treatment history, and BMI. CONCLUSION: Physicians were satisfied with the ability of secukinumab to control disease and it was effective in the treatment of PsA patients in routine clinical settings.

Identifying the associated risks of pneumonia in COPD patients: ARCTIC an observational study.

BACKGROUND: Inhaled corticosteroids (ICS) are associated with an increased risk of pneumonia in patients with chronic obstructive pulmonary disease (COPD). Other factors such as severity of airflow limitation and concurrent asthma may further raise the possibility of developing pneumonia. This study assessed the risk of pneumonia associated with ICS in patients with COPD. METHODS: Electronic Medical Record data linked to National Health Registries were collected from COPD patients and matched reference controls in 52 Swedish primary care centers (2000-2014). Levels of ICS treatment (high, low, no ICS) and associated comorbidities were assessed. Patients were categorized by airflow limitation severity. RESULTS: A total of 6623 patients with COPD and 48,566 controls were analyzed. Patients with COPD had a more than 4-fold increase in pneumonia versus reference controls (hazard ratio [HR] 4.76, 95% confidence interval [CI]: 4.48-5.06). ICS use increased the risk of pneumonia by 20-30% in patients with COPD with forced expiratory volume in 1 s ≥ 50% versus patients not using ICS. Asthma was an independent risk factor for pneumonia in the COPD population. Multivariate analysis identified independent predictors of pneumonia in the overall population. The highest risk of pneumonia was associated with high dose ICS (HR 1.41, 95% CI: 1.23-1.62). CONCLUSIONS: Patients with COPD have a greater risk of pneumonia versus reference controls; ICS use and concurrent asthma increased the risk of pneumonia further.

Factors associated with appropriate inhaler use in patients with COPD - lessons from the REAL survey.

Background: Nonadherence to medication and incorrect use of inhalers represent significant barriers to optimal disease management of patients with chronic obstructive pulmonary disease (COPD). Thus, health care professionals (HCPs) play a critical role in educating their patients on appropriate inhaler use and in ensuring medication adherence. However, many patients do not receive appropriate inhaler training or have not had their inhaler technique checked. Methods: The Real-life Experience and Accuracy of inhaLer use (REAL) survey was a computer-assisted, telephonic survey consisting of 23 questions gathering real-world information on correct inhaler use, inhalation technique, device attributes, adherence, dosing accuracy, training, correct device use, ease of use, and factors that influence patient adherence in commercially available inhalers delivering COPD maintenance therapy. All results are based on patient-reported data. Results: The survey was conducted between January 4, 2016 and February 2, 2016. A total of 764 patients using various inhalers (Breezhaler® =186; Ellipta® =191; Genuair® =194; Respimat® =201) with mild to very severe COPD, with a mean ± SD age 56±9.8 years, completed the survey. Patient self-reported adherence was significantly lower in younger patients compared to older patients (p=0.020). Eighty-three percent of patients indicated that a demonstration (in-person) was "very helpful" versus 58% for video. Patient preferences for training methods were as follows: demonstration of inhaler use (83%), video (58%), instructions for use (51%), and leaflet (34%). Twenty-nine percent of patients had not been checked to see if they were using their device correctly by a HCP within the last two years. Patients who were checked were significantly more adherent than unchecked patients (p=0.020). The majority of the patients using Breezhaler reported either being very confident or confident of having taken a full dose, which was higher than those using Genuair, Ellipta (α=0.05), and Respimat (α=0.05). Treatment adherence in the last 30 days was highest with Breezhaler followed by Respimat, Ellipta, and Genuair. Conclusion: The REAL survey identified attributes that influenced patient adherence and optimal inhaler use. Predictive attributes that influence patient adherence which HCPs should be aware of include age and disease severity. Modifiable attributes which the HCP can influence include correct inhaler use training, choice of training methods, checking patient inhaler technique at subsequent visits, and device selection. Inhalers are integral in the effective management of patients with COPD; it is therefore important that patients use the inhaler correctly and have full confidence in the dosage.

Indacaterol/glycopyrronium is cost-effective compared to salmeterol/fluticasone in COPD: FLAME-based modelling in a Swedish population.

BACKGROUND: This study assessed the cost-effectiveness of indacaterol/glycopyrronium (IND/GLY) versus salmeterol/fluticasone (SFC) in chronic obstructive pulmonary disease (COPD) patients with moderate to very severe airflow limitation and ≥1 exacerbation in the preceding year. METHODS: A previously published and validated patient-level simulation model was adapted using clinical data from the FLAME trial and real-world cost data from the ARCTIC study. Costs (total monetary costs comprising drug, maintenance, exacerbation, and pneumonia costs) and health outcomes (life-years (LYs), quality-adjusted life-years (QALYs)) were projected over various time horizons (1, 5, 10 years, and lifetime) from the Swedish payer's perspective and were discounted at 3% annually. Uncertainty in model input values was studied through one-way and probabilistic sensitivity analyses. Subgroup analyses were also performed. RESULTS: IND/GLY was associated with lower costs and better outcomes compared with SFC over all the analysed time horizons. Use of IND/GLY resulted in additional 0.192 LYs and 0.134 QALYs with cost savings of €1211 compared with SFC over lifetime. The net monetary benefit (NMB) was estimated to be €8560 based on a willingness-to-pay threshold of €55,000/QALY. The NMB was higher in the following subgroups: severe (GOLD 3), high risk and more symptoms (GOLD D), females, and current smokers. CONCLUSION: IND/GLY is a cost-effective treatment compared with SFC in COPD patients with mMRC dyspnea grade ≥ 2, moderate to very severe airflow limitation, and ≥1 exacerbation in the preceding year.

Erratum: Changes in initial COPD treatment choice over time and factors influencing prescribing decisions in UK primary care: a real-world study.

This corrects the article DOI: 10.1038/npjpcrm.2016.2.

Greater dyspnea is associated with lower health-related quality of life among European patients with COPD.

OBJECTIVE: Dyspnea is a defining symptom in the classification and treatment of chronic obstructive pulmonary disease (COPD). However, the degree of variation in burden among symptomatic COPD patients and the possible correlates of burden remain unclear. This study was conducted to characterize patients in Europe currently being treated for COPD according to the level of dyspnea in terms of sociodemographics, health-related quality of life, work productivity impairment, and health care resource use assessed by patient reports. METHODS: Data were derived from the 5-EU 2013 National Health and Wellness Survey (N=62,000). Respondents aged ≥40 years who reported currently using a prescription for COPD were grouped according to their level of dyspnea as per the Global Initiative for Chronic Obstructive Lung Disease guidelines and compared on health status (revised Short Form 36 [SF-36]v2), work impairment (Work Productivity and Activity Impairment questionnaire), and number of health care visits in the past 6 months using generalized linear models with appropriate distributions and link functions. RESULTS: Of the 768 respondents who met the inclusion criteria, 245 (32%) were considered to have higher dyspnea (equivalent to modified Medical Research Council score ≥2). Higher dyspnea was associated with decrements ranging from 3.9 to 8.2 points in all eight domains of the SF-36 health profile after adjustment for sociodemographics, general health characteristics, and length of COPD diagnosis; mental component summary scores and Short Form-6D health utility scores were lower by 3.5 and 0.06 points, respectively. Adjusted mean activity impairment (55% vs 37%, P<0.001) and number of emergency room visits (0.61 vs 0.40, P=0.030) were higher in patients with greater dyspnea. CONCLUSION: Many European patients with COPD continue to experience dyspnea despite treatment and at levels associated with notable impairments in the patients' ability to function across a multitude of domains. These patients may benefit from more intense treatment of their symptoms.

Comparative efficacy of long-acting ß2-agonists as monotherapy for chronic obstructive pulmonary disease: a network meta-analysis.

PURPOSE: Long-acting ß2-agonists (LABAs) have demonstrated efficacy in patients with COPD in clinical trials. The purpose of this study was to assess the comparative efficacy of all available dosages of all LABA monotherapies using a network meta-analysis. METHODS: A systematic literature review identified 33 randomized controlled trials of LABA monotherapies (salmeterol 50 µg twice daily [BID]; formoterol 12 µg BID; indacaterol 75, 150, and 300 µg once daily [OD]; olodaterol 5 and 10 µg OD, and vilanterol 25 µg OD). Clinical efficacy was evaluated at 12 and 24 weeks in terms of trough forced expiratory volume in 1 second (FEV1), transition dyspnea index focal score, St George's Respiratory Questionnaire total score, and rate of COPD exacerbations. The relative effectiveness of all LABA monotherapies was estimated by Bayesian network meta-analysis. RESULTS: At 12 and 24 weeks, indacaterol 300 and 150 µg OD were associated with statistically significant improvement in trough FEV1 compared to all other LABA monotherapies; vilanterol 25 µg OD was superior to formoterol 12 µg BID. At 12 weeks, indacaterol 75 µg OD was associated with significant improvement in trough FEV1 compared to formoterol 12 µg BID and olodaterol (5 and 10 µg OD); salmeterol 50 µg BID was superior to formoterol 12 µg BID and olodaterol 5 µg OD. Indacaterol 300 µg OD was also associated with significant improvement in transition dyspnea index focal score compared to all other LABAs at 12 or 24 weeks. Indacaterol 150 µg OD had significantly better results in exacerbation rates than olodaterol 5 µg and olodaterol 10 µg OD. CONCLUSION: Indacaterol 300 µg, followed by 150 and 75 µg, were the most effective LABA monotherapies for moderate to severe COPD.

Cost Effectiveness of the Long-Acting ß2-Adrenergic Agonist (LABA)/Long-Acting Muscarinic Antagonist Dual Bronchodilator Indacaterol/Glycopyrronium Versus the LABA/Inhaled Corticosteroid Combination Salmeterol/Fluticasone in Patients with Chronic Obstructive Pulmonary Disease: Analyses Conducted for Canada, France, Italy, and Portugal.

OBJECTIVE: The objective of this study was to assess the cost effectiveness of the dual bronchodilator indacaterol/glycopyrronium (IND/GLY) compared with salmeterol/fluticasone combination (SFC) in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD) who had a history of one or no exacerbations in the previous year, in Canada, France, Italy, and Portugal. METHODS: A patient-level simulation was developed to compare the costs and outcomes of IND/GLY versus SFC based on data from the LANTERN trial (NCT01709903). Monte-Carlo simulation methods were employed to follow individual patients over various time horizons. Population and efficacy inputs were derived from the LANTERN trial. Considering the payers' perspective, only direct costs were included. Costs and health outcomes were discounted annually at 3.0 % for all countries. Unit costs were taken from publically available sources with all costs converted to euros (€). The cost base year was 2015. Deterministic and probabilistic sensitivity analyses were undertaken to test the robustness of the model results. RESULTS: IND/GLY was found to be the dominant (more effective and less costly) treatment option compared with SFC in all four countries. The use of IND/GLY was associated with mean total cost savings per patient over a lifetime of €6202, €1974, €1611, and €220 in Canada, France, Italy, and Portugal, respectively. Sensitivity analysis showed that exacerbation rates had the largest impact on incremental costs and quality-adjusted life-years (QALYs). The probability of IND/GLY being cost effective was estimated to be >95 % for thresholds above €5000/QALY. CONCLUSION: In patients with moderate to severe COPD, IND/GLY is likely to be a cost-effective treatment alternative compared with SFC.

Changes in initial COPD treatment choice over time and factors influencing prescribing decisions in UK primary care: in UK primary care: a real-world, retrospective, observational.

Prescribing patterns in chronic obstructive pulmonary disease (COPD) are often inconsistent with published guidelines. This retrospective, observational study utilised data from the Optimum Patient Care Research Database to examine the changes in COPD prescribing patterns over time and to identify predictors of physician treatment choice for patients newly diagnosed with COPD. Initial therapy was defined as the treatment(s) prescribed at or within 1 year before COPD diagnosis. Changes over time were assessed in three cohorts based on the date of diagnosis: (1) 1997-2001; (2) 2002-2006; and (3) 2007-2010. Factors affecting the odds of being prescribed any initial therapy or any initial maintenance therapy were identified by univariable and multivariable logistic regression. The analysis included 20,154 patients, 45% of whom were prescribed an initial regimen containing an inhaled corticosteroid (ICS), whereas 28% received no initial pharmacological treatment. Prescribing of ICS monotherapy decreased over time, as did the proportion of patients receiving no therapy at or within 1 year before diagnosis. Comorbid asthma, a high exacerbation rate, increased symptoms and poor lung function each increased the likelihood of being prescribed any initial therapy or initial maintenance therapy; comorbid asthma and an annual rate of ⩾3 exacerbations were the strongest predictors. In conclusion, our analyses revealed major differences between actual prescribing behaviour and guideline recommendations for patients with newly diagnosed COPD, with many patients receiving no treatment and large numbers of patients receiving ICS-containing regimens. Predictors of initial therapy were identified.

Efficacy of Indacaterol/Glycopyrronium in Patients with COPD Who Have Increased Dyspnea with Daily Activities.

Introduction: The Global initiative for chronic Obstructive Lung Disease (GOLD) recommends treating patients with chronic obstructive pulmonary disease (COPD) based on a combined assessment of symptom severity and airflow limitation and/or exacerbation risk. According to GOLD, patients with mild-to-moderate airflow limitation and distressing symptoms such as dyspnea should be treated with a long-acting beta2-agonist (LABA) or a long-acting muscarinic antagonist (LAMA). If symptoms persist on monotherapy, GOLD recommends a combination of bronchodilators (LABA/LAMA). Methods: We performed a post-hoc analysis of data from two 26-week, prospective clinical trials to investigate the effect of treating patients with moderate-to-severe dyspnea with the once-daily LABA/LAMA combination indacaterol/glycopyrronium (IND/GLY) 110/50 µg compared with placebo, once-daily tiotropium 18 µg, and twice-daily salmeterol/fluticasone propionate (SFC) 50/500 µg. In this analysis, a Baseline Dyspnea Index (BDI) score ≤7 was used to identify dyspneic patients. Results: In dyspneic patients, IND/GLY significantly improved Transition Dyspnea Index (TDI) total scores compared with tiotropium (0.59 units; p<0.05) and SFC (0.97 units; p<0.05), and significantly increased the likelihood of a patient achieving a 1-unit improvement in TDI compared with tiotropium (odds ratio [OR] 1.87; p<0.05). IND/GLY also significantly improved trough forced expiratory volume in 1 second (FEV1) compared with tiotropium and SFC (p<0.001 and p<0.0001, respectively), and significantly reduced rescue medication use compared with tiotropium (p<0.001). Conclusions: Our analysis indicates that IND/GLY provides additional improvements in dyspnea and lung function compared with tiotropium and SFC in dyspneic patients.

The inevitable drift to triple therapy in COPD: an analysis of prescribing pathways in the UK.

BACKGROUND: Real-world prescription pathways leading to triple therapy (TT) (inhaled corticosteroid [ICS] plus long-acting ß2-agonist bronchodilator [LABA] plus long-acting muscarinic antagonist) differ from Global initiative for chronic Obstructive Lung Disease [GOLD] and National Institute for Health and Care Excellence treatment recommendations. This study sets out to identify COPD patients without asthma receiving TT, and determine the pathways taken from diagnosis to the first prescription of TT. METHODS: This was a historical analysis of COPD patients without asthma from the Optimum Patient Care Research Database (387 primary-care practices across the UK) from 2002 to 2010. Patient disease severity was classified using GOLD 2013 criteria. Data were analyzed to determine prescribing of TT before, at, and after COPD diagnosis; the average time taken to receive TT; and the impact of lung function grade, modified Medical Research Council dyspnea score, and exacerbation history on the pathway to TT. RESULTS: During the study period, 32% of patients received TT. Of these, 19%, 28%, 37%, and 46% of patients classified as GOLD A, B, C, and D, respectively, progressed to TT after diagnosis (P<0.001). Of all patients prescribed TT, 25% were prescribed TT within 1 year of diagnosis, irrespective of GOLD classification (P=0.065). The most common prescription pathway to TT was LABA plus ICS. It was observed that exacerbation history did influence the pathway of LABA plus ICS to TT. CONCLUSION: Real life UK prescription data demonstrates the inappropriate prescribing of TT and confirms that starting patients on ICS plus LABA results in the inevitable drift to overuse of TT. This study highlights the need for dissemination and implementation of COPD guidelines to physicians, ensuring that patients receive the recommended therapy.

Symptoms and impact of COPD assessed by an electronic diary in patients with moderate-to-severe COPD: psychometric results from the SHINE study.

BACKGROUND: Symptoms, particularly dyspnea, and activity limitation, have an impact on the health status and the ability to function normally in patients with chronic obstructive pulmonary disease (COPD). METHODS: To develop an electronic patient diary (eDiary), qualitative patient interviews were conducted from 2009 to 2010 to identify relevant symptoms and degree of bother due to symptoms. The eDiary was completed by a subset of 209 patients with moderate-to-severe COPD in the 26-week QVA149 SHINE study. Two morning assessments (since awakening and since the last assessment) and one evening assessment were made each day. Assessments covered five symptoms ("shortness of breath," "phlegm/mucus," "chest tightness," "wheezing," and "coughing") and two impact items ("bothered by COPD" and "difficulty with activities") and were scored on a 10-point numeric scale. RESULTS: Patient compliance with the eDiary was 90.4% at baseline and 81.3% at week 26. Correlations between shortness of breath and impact items were >0.95. Regression analysis showed that shortness of breath was a highly significant (P<0.0001) predictor of impact items. Exploratory factor analysis gave a single factor comprising all eDiary items, including both symptoms and impact items. Shortness of breath, the total score (including five symptoms and two impact items), and the five-item symptom score from the eDiary performed well, with good consistency and reliability. The eDiary showed good sensitivity to change, with a 0.6 points reduction in the symptoms scores (on a 0-10 point scale) representing a meaningful change. CONCLUSION: The eDiary was found to be valid, reliable, and responsive. The high correlations obtained between "shortness of breath" and the ratings of "bother" and "difficulty with activities" confirmed the relevance of this symptom in patients with COPD. Future studies will be required to explore further psychometric properties and their ability to differentiate between COPD treatments.

Perceived benefits of sharing health data between people with epilepsy on an online platform.

An epilepsy community was developed on PatientsLikeMe.com to share data between patients to improve their outcomes by finding other patients like them. In a 14-day response period, 221 patients with epilepsy (mean age: 40 years, SD: 12, range: 17-72, 66% female) completed a survey about benefits they perceived. Prior to using the site, a third of respondents (30%) did not know anyone else with epilepsy with whom they could talk; of these, 63% now had at least one other patient with whom they could connect. Perceived benefits included: finding another patient experiencing the same symptoms (59%), gaining a better understanding of seizures (58%), and learning more about symptoms or treatments (55%). Number of benefits was associated with number of relationships with other patients, F(4,216)=8.173, P<0.001). Patients with epilepsy reported an array of perceived benefits similar to those reported by populations with other diseases. Controlled sharing of health data may have the potential to improve disease self-management of people with epilepsy.

Clinically meaningful improvement in health-related quality of life in a randomized controlled trial of certolizumab pegol maintenance therapy for Crohn's disease.

OBJECTIVES: Moderate-to-severe Crohn's disease (CD) is associated with important impairment in health-related quality of life (HRQOL). The aim of this study was to assess the effects of certolizumab pegol (CZP) maintenance therapy on HRQOL. METHODS: During an open-label induction phase, study participants with moderate-to-severe CD were treated with 400 mg CZP every other week. Responders were randomized to monthly maintenance therapy with CZP or placebo. Clinically meaningful improvement in HRQOL was evaluated with three patient-reported outcome (PRO) instruments. Quality-adjusted life-years (QALYs) were calculated from utility scores derived from the EuroQoL-5 dimensions (EQ-5D). Normal life rating was measured by combining clinical disease activity, HRQOL, and measures of professional work productivity and daily activity. RESULTS: A total of 425 responders to induction therapy were randomized to CZP maintenance (n=215) or placebo (n=210). Participants assigned to CZP maintenance reported clinically meaningful improvements in HRQOL relative to baseline and to placebo-treated participants. More participants receiving treatment with CZP reported clinically meaningful improvement in Inflammatory Bowel Disease Questionnaire (IBDQ) score (60 vs. 43%, P<0.001) and in Short-Form 36-Item Health Survey (SF-36) physical (51 vs. 34%, P<0.001) and mental component summary responses (44 vs. 32%, P=0.016) than did those receiving placebo. The proportion of participants who achieved clinically meaningful improvement in the EQ-5D plus health status visual analogue scale (VAS) was significantly greater in those assigned to CZP maintenance than in those assigned placebo (57 vs. 38%, P<0.001). There was also a significantly greater gain in QALYs for the CZP group as compared with the placebo group (mean+/-s.d. 0.25+/-0.10 and 0.21+/-0.11; P=0.001). Significantly more participants receiving CZP maintenance reported living a normal life (21.4%) than did those receiving placebo (12.9%, P=0.019). CONCLUSIONS: Maintenance therapy with CZP resulted in statistically significant and clinically meaningful improvements in HRQOL, as assessed by multiple PRO instruments. CZP improved and maintained the quality and quantity of the remission and response, as measured by QALYs. Furthermore, a significant proportion of study participants who received CZP returned to a normal life compared with those who received placebo.

Certolizumab pegol, a monthly subcutaneously administered Fc-free anti-TNFalpha, improves health-related quality of life in patients with moderate to severe Crohn's disease.

BACKGROUND AND AIMS: Certolizumab pegol, a polyethylene glycolated Fc-free Fab' was efficacious and well tolerated in patients with moderate-to-severe Crohn's disease in a previously reported randomized, placebo-controlled study. In this paper, we report the effect of certolizumab pegol on health-related quality of life (HRQoL). MATERIALS AND METHODS: Patients with moderate-to-severe active Crohn's disease (n = 292) received subcutaneous certolizumab pegol 100, 200, or 400 mg or placebo at weeks 0, 4, and 8. A post hoc analysis of the intent-to-treat population (290 patients with HRQoL data) assessed HRQoL by evaluating patients' responses to the self-administered inflammatory bowel disease questionnaire (IBDQ) at baseline and weeks 2, 4, 6, 8, 10, and 12. RESULTS: Patients receiving certolizumab pegol 400 mg at weeks 0, 4, and 8 demonstrated, via their IBDQ total score, significantly (P

Validation of the PAC-SYM questionnaire for opioid-induced constipation in patients with chronic low back pain.

BACKGROUND: The Patient Assessment of Constipation Symptoms (PAC-SYM) questionnaire is a 12-item self-report instrument divided into abdominal, rectal and stool domains. AIMS: This study aimed to (1) evaluate the psychometric properties of PAC-SYM in assessing the symptoms and severity of opioid-induced constipation; (2) test for differences in opioid-induced constipation between Durogesic fentanyl transdermal reservoir (TDF) and oral sustained-release morphine (SRM) in patients with chronic low back pain (CLBP). METHODS: In a 13-month, open-label, parallel-group study, 680 patients were randomised to receive either TDF (n=338) or SRM (n=342) for CLBP. Assessments were recorded at Visit 1 (baseline), Visit 5 (Day 29) and Visit 17 (Month 13). Concurrent validity, clinical validity and responsiveness of PAC-SYM were determined based on patients' confirmation of constipation (CC) scores. Differences in PAC-SYM scores between treatment groups were also evaluated. RESULTS: The study included 677 patients, of whom 638 were opioid-naïve. Mean PAC-SYM scores for constipated patients were substantially higher than for non-constipated patients, demonstrating good clinical validity for PAC-SYM. The PAC-SYM could detect changes in bowel function over the treatment period, indicating responsiveness. Homogeneity of each symptom domain exceeded Cronbach's alpha coefficient of 0.70, suggesting good internal consistency and reliability. Changes in mean PAC-SYM scores from baseline to Visit 5 and Visit 17 were significantly lower for the TDF group than for the SRM group, indicating that the TDF group experienced less severe constipation. CONCLUSION: PAC-SYM is a reliable, valid and responsive measure of the presence and severity of opioid-induced constipation symptoms.

Development of the cancer therapy satisfaction questionnaire: item generation and content validity testing.

OBJECTIVES: This study was undertaken to develop a new questionnaire, the Cancer Therapy Satisfaction Questionnaire (CTSQ), to focus on the issues oncology patients consider when evaluating chemotherapy in terms of expectations and satisfaction. METHODS: Items of the CTSQ were generated through the review of responses from interviews with oncology patients, physicians, and nurses. Analysis of the data was stratified by disease stage, disease type, and country to explore potential differences between these groups. Two rounds of face and content validity testing were then conducted. RESULTS: Patients explained their hopes for efficacy and factors related to treatment satisfaction. Content validity testing in 30 patients, followed by additional testing in 10 patients on oral therapy, suggested that patients felt the questionnaire was clear, comprehensive, relevant, and easy to complete. Minor revisions were implemented to improve clarity, resulting in deletion of 12 items, modification of 17 items and the rewording of "chemotherapy" to "cancer therapy" to ensure patients on oral therapy were able to respond. The CTSQ contains 21 items and assesses seven domains: Expectations of cancer therapy, Feelings about side effects, Oral cancer therapy adherence, Convenience, Satisfaction with cancer therapy, Stopping cancer therapy, and Reasons for nonadherence. CONCLUSIONS: The CTSQ was designed for adults with a wide range of cancer types and stages, receiving a variety of cancer treatment formulations. A validation study is currently underway to examine the psychometric properties, further refine the questionnaire and develop scoring methods for the CTSQ.

Simultaneous development of the Pediatric GERD Caregiver Impact Questionnaire (PGCIQ) in American English and American Spanish.

BACKGROUND: The objective of this study was to develop simultaneously a new questionnaire, the Pediatric GERD Caregiver Impact Questionnaire (PGCIQ), in American English and American Spanish in order to elucidate the impact of caring for a child with GERD. METHODS: Two focus group discussions were conducted in American English and American Spanish to develop a relevant conceptual model. Focus group participants were the primary caregivers of children with GERD (newborn through 12 years of age). Participant responses were qualitatively analyzed to identify potential differences in caregiver perspectives by the caregiver's language, socio-economic status and demographic profile as well as the child's age and disease severity level. Items in the PGCIQ were generated simultaneously in English and Spanish by reviewing results of qualitative analysis from focus groups in each language. The PGCIQ was finalized in both languages after testing content validity and conducting an in-depth translatability assessment. RESULTS: Analysis of focus group comments resulted in the development of a first draft questionnaire consisting of 58 items in 10 domains. Content validity testing and an in-depth translatability assessment resulted in wording modification of 37 items, deletion of 14 items and the addition of a domain with five items. Feedback from the content validity testing interviews indicated that the instrument is conceptually relevant in both American English and American Spanish, clear, comprehensive and easy to complete within 10 minutes. The final version of the PGCIQ contains 49 items assessing ten domains. An optional module with nine items is available for investigative research purposes and for use only at baseline. CONCLUSION: The PGCIQ was developed using simultaneous item generation, a process that allows for consideration of concept relevance in all stages of development and in all languages being developed. The PGCIQ is the first questionnaire to document the multidimensional impact of caring for an infant or young child with GERD. Linguistic adaptation of the PGCIQ in multiple languages is ongoing. A validation study of the PGCIQ is needed to examine its psychometric properties, further refine the items and develop an appropriate scoring model.