Prospective longitudinal study on quality of life in relapsed/refractory multiple myeloma patients receiving second- or third-line lenalidomide or bortezomib treatment.

Treatment advances for multiple myeloma (MM) that have prolonged survival emphasise the importance of measuring patients' health-related quality of life (HRQoL) in clinical studies. HRQoL/functioning and symptoms of patients with relapsed/refractory MM (RRMM) receiving second- or third-line lenalidomide or bortezomib treatment were measured in a prospective European multicentre, observational study at different time points. At baseline, patients in the lenalidomide cohort were frailer than in the bortezomib cohort with more rapid disease progression at study entry (more patients with Eastern Cooperative Oncology Group performance status >2, shorter time from diagnosis, more chronic heart failure, higher serum creatinine levels, more patients with dialysis required). About 40% of the patients receiving lenalidomide discontinued the study in <6 months while 55% in the bortezomib cohort discontinued. No substantial HRQoL deterioration was observed for the first 6 months in patients with RRMM receiving one or the other treatment. For patients still on treatment at study completion (month 6), only the European Organization for Research and Treatment of Cancer Quality-of-Life Core domains of Diarrhoea and Global Health Status/QoL had worsened in the lenalidomide and bortezomib cohorts, respectively. A clinically meaningful deterioration in HRQoL was more often observed for patients who discontinued the study prior to 6 months in the bortezomib cohort than in the lenalidomide cohort.

Development and psychometric validation of the REFlective evaLuation of psoriasis Efficacy of Treatment and Severity (REFLETS) questionnaire: a common measure of plaque-type psoriasis severity and treatment efficacy for patients and clinicians.

BACKGROUND: To date, there is no global consensus on the definition of the severity of psoriasis. The REFlective evaLuation of psoriasis Efficacy of Treatment and Severity (REFLETS) questionnaire has recently been developed to provide a better understanding of plaque-type psoriasis severity and treatment efficacy from both patient and clinician perspectives. OBJECTIVE: This study aimed to develop and psychometrically validate the new REFLETS questionnaire to evaluate patient and clinician perceptions of plaque-type psoriasis severity and treatment efficacy. METHODS: Two similar versions of the REFLETS questionnaire were developed following a rigorous methodology for clinicians and patients, referring to 'the psoriasis of your patient' or to 'your psoriasis', respectively. An observational, longitudinal, multicentre study was conducted in France with 34 dermatologists and 430 mild to severe plaque-type psoriasis patients to finalize the questionnaire and evaluate its psychometric properties. RESULTS: Two dimensions were defined--severity and treatment efficacy--with three subdimensions within severity (impact of psoriasis, symptoms and disease course), and two individual items on joint pain. The questionnaire was well accepted by clinicians and patients. Excellent internal consistency (Cronbach's alpha = 0.66-0.98) and test-retest reliability (intraclass correlation coefficients = 0.83-0.94) were demonstrated. REFLETS scores were moderately to highly correlated to Psoriasis Area and Severity Index (r = 0.35-0.70), Skindex-29 (r = 0.46-0.82) and DLQI scores (r = 0.36-0.82). Patients with decreased psoriasis severity and those with increased treatment efficacy, according to patient global evaluations, had lower severity and higher treatment efficacy REFLETS scores, respectively. CONCLUSION: REFlective evaLuation of psoriasis Efficacy of Treatment and Severity is a promising tool for assessing plaque-type psoriasis severity and treatment efficacy from patient and clinician perspectives. It may help to improve patient and clinician communication in treatment decision making.

Deterioration in quality of life (QoL) in patients with malignant ascites: results from a phase II/III study comparing paracentesis plus catumaxomab with paracentesis alone.

BACKGROUND: Malignant ascites (MA) is associated with poor prognosis and limited palliative therapeutic options. Therefore, quality of life (QoL) assessment is of particular importance to demonstrate new treatment value. Following the demonstration of the superiority of catumaxomab and paracentesis over paracentesis on puncture-free survival, this analysis aimed at comparing deterioration in QoL between both the treatment options. PATIENTS AND METHODS: In a randomised, multicentre, phase II/III study of patients with MA due to epithelial cell adhesion molecule (EpCAM) positive cancer, the QoL was evaluated using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 items (EORTC QLQ-C30) questionnaire at screening, 1, 3 and 7 months after treatment and in the case of re-puncture on the day of paracentesis. Time to first deterioration in QoL was defined as a decrease in the QoL score of at least five points and compared between the catumaxomab (n=160) and control (n=85) groups using the log-rank test and Cox proportional hazards models adjusted for baseline score, country and primary tumour type. RESULTS: Deterioration in QoL scores appeared more rapidly in the control than in the catumaxomab group (median 19-26 days versus 47-49 days). The difference in time to deterioration in QoL between the groups was statistically significant for all scores (P<0.01). The hazard ratios ranged from 0.08 to 0.24 (P<0.01). CONCLUSIONS: Treatment with catumaxomab delayed deterioration in QoL in patients with MA. Compared with paracentesis alone, catumaxomab enabled patients to benefit from better QoL for a prolonged survival period.

Psychometric performance and clinical meaningfulness of the Patient Assessment of Constipation-Quality of Life questionnaire in prucalopride (RESOLOR) trials for chronic constipation.

BACKGROUND: The Patient Assessment of Constipation-Quality of Life (PAC-QOL) is a self-reported questionnaire measuring health-related quality of life (HRQL) of constipated patients and was used as secondary endpoint in three identical double-blind, randomized, placebo-controlled Phase III clinical trials. These 12-week trials in subjects with severe chronic constipation evaluated the effects of prucalopride, a selective 5-HT(4) agonist given orally once daily. METHODS: To consolidate the main treatment effect results observed in the prucalopride trial populations, analyses were undertaken on the pooled data of the three trials to confirm the psychometric properties of the PAC-QOL and to provide guidance for the interpretation of the clinical significance of its scores. KEY RESULTS: The evaluation of the psychometric properties confirmed the PAC-QOL reliability, validity and responsiveness to measure the impact of chronic constipation symptoms on HRQL in the prucalopride trials. The 1-point improvement in PAC-QOL scores used as target response level for the main treatment effect analyses was validated as a relevant definition of response for treatment group comparisons. Cumulative distribution curves, drawn for each treatment group to provide more complete information on treatment effects than single minimal important difference point estimates, demonstrated consistent superior effects of prucalopride over placebo on all PAC-QOL scores. CONCLUSIONS & INFERENCES: The PAC-QOL questionnaire is a useful measurement tool to assess, from a patient perspective, the potential therapeutic value of chronic constipation treatments in clinical trials and, by directly reflecting the patient's own perspective on constipation and its treatment, eventually also for informing daily medical practice.

Scoring and psychometric validation of the Perception of Anticoagulant Treatment Questionnaire (PACT-Q).

BACKGROUND: The 'Perception of Anti-Coagulant Treatment Questionnaire' (PACT-Q) was developed to assess patients' expectations of, and satisfaction with their anticoagulant treatment. This questionnaire needs to be finalised and psychometrically validated. METHODS: The PACT-Q was included in the United States, The Netherlands and France into three phase III multinational clinical trials conducted to evaluate efficacy and safety of a new long-acting anticoagulant drug (idraparinux) compared to vitamin K antagonist (VKA). PACT-Q was administered to patients with deep venous thrombosis (DVT), atrial fibrillation (AF) or pulmonary embolism (PE) at Day 1, to assess patients' expectations, and at 3 and 6 months to assess patients' satisfaction and treatment convenience and burden. The final structure of the PACT-Q (Principal Component Analysis--PCA--with Varimax Rotation) was first determined and its psychometric properties were then measured with validity of the structure (Multitrait analysis), internal consistency reliability (Cronbach's alpha coefficients) and known-group validity. RESULTS: PCA and multitrait analyses showed the multidimensionality of the "Treatment Expectations" dimension, comprising 7 items that had to be scored independently. The "Convenience" and "Burden of Disease and Treatment" dimensions of the hypothesised original structure of the questionnaire were combined, thus resulting in 13 items grouped into the single dimension "Convenience". The "Anticoagulant Treatment Satisfaction" dimension remained unchanged and included 7 items. All items of the "Convenience" and "Anticoagulant Treatment Satisfaction" dimensions displayed good convergent and discriminant validity. The internal consistency reliability was good, with a Cronbach's alpha of 0.84 for the "Convenience" dimension, and 0.76 for the "Anticoagulant Treatment Satisfaction" dimension. Known-group validity was good, especially with regard to occurrence of thromboembolic events within 3 months from randomisation. CONCLUSION: The PACT-Q is a valid and reliable instrument that allows the assessment of patients' expectations and satisfaction regarding anticoagulant treatment, as well as their opinion about treatment convenience of use. Its two-part structure--assessment of expectations at baseline in the first part, and of convenience, burden and treatment satisfaction in the second--was validated and displays good and stable psychometric properties. These results are not sufficient to recommend the use of satisfaction as primary endpoint in clinical trials; further validation work is needed to support the interpretation of PACT-Q dimension scores. However, this first validation makes the PACT-Q an appropriate measure for use in clinical and pharmacoepidemiological research, as well as in real-life studies. TRIAL REGISTRATION: (ClinicalTrials.gov numbers, NCT00067093, NCT00062803 and NCT00070655).