Sandra Nolte

Charité Universitätsmedizin Berlin | Charité · Psychometrics & Health Outcomes Group

The use of then-test (retrospective pre-test) scores has frequently been proposed as a solution to potential confounding of change scores because of response shift, as it is assumed that then-test and post-test responses are provided from the same perspective. However, this assumption has not been formally tested using robust quantitative methods....

Program evaluations are frequently based on 'then-test' data, i.e., pre-test collected in retrospect. While the application of the then-test has practical advantages, little is known about the validity of then-test data. Because of the collection of then-test in close proximity to post-test questions, this study was aimed at exploring whether the p...

Objectives: To provide an in-depth analysis of outcome measures used in the evaluation of chronic disease self-management programs consistent with the Stanford curricula. Methods: Based on a systematic review on self-management programs, effect sizes derived from reported outcome measures are categorized according to the quality of life appraisa...

The Health Literacy Questionnaire (HLQ), developed in Australia in 2012 using a ‘validity-driven’ approach, has been rapidly adopted and is being applied in many countries and languages. It is a multidimensional measure comprising nine distinct domains that may be used for surveys, needs assessment, evaluation and outcomes assessment as well as for...

Objective The European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 health-related quality of life questionnaire is one of the most widely used cancer-specific health-related quality of life questionnaires worldwide. General population norm data can facilitate the interpretation of QLQ-C30 data obtained from cancer patients. Th...

Purpose The European Organisation for Research and Treatment of Cancer (EORTC) quality of life core questionnaire (QLQ-C30) is a validated and widely-used Patient-Reported Outcome Measure for measuring the health-related quality of life (HRQoL) of cancer patients. To facilitate interpretation of results obtained in studies using the EORTC QLQ-C30,...

Background The Alzheimer's Disease Cooperative Study – Activities of Daily Living Scale for use in Mild Cognitive Impairment (MCI), the ADCS-ADL-MCI, is an evaluation scale with information provided by an informant/caregiver to describe the functional impairment of patients with MCI. As the ADCS-ADL-MCI has yet to undergo a full psychometric evalua...

Background: As a local treatment for prostate cancer (PCa), robot-assisted radical prostatectomy (RARP) may have a quality of life (QoL) benefit over open surgery. Recent analyses revealed substantial between-country differences in the function and symptom scale scores for the European Organisation for Research and Treatment of Cancer Quality of L...

Objectives: The EORTC QLU-C10D is a cancer-specific preference-based measure, providing health utilit ies for use in economic evaluations derived from the widely used health-related quality of life measure, EORTC QLQ-C30. Several EORTC QLU-C10D country-specific value sets are available. This article aims to provide EORTC QLU-C10D general populatio...

Objectives Self-report measures of health literacy frequently encompass a group of conceptually distinct but related domains scored as either a single scale or separately for each domain. Psychometric studies of the 44-item Health Literacy Questionnaire, scored as nine separate scales, typically report the results of correlated factors confirmatory...

Definitions of health literacy have evolved from notions of health-related literacy to a multidimensional concept that incorporates the importance of social and cultural knowledge, practices and contexts. This evolution is evident in the development of instruments that seek to measure health literacy in different ways. Health literacy measurement i...

Objectives This study aimed to determine the relationship between frequently used patient-reported outcome (PRO) measures and a multi-task performance outcome (PerfO) measure of general physical function (PF) and to examine the association of these measures with depressive mood, pain, and age. Methods Frequently used PRO measures of general PF (Pa...

Background General population normative values for the widely used health-related quality of life (HRQoL) measure, European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire – Core 30 (EORTC QLQ-C30), are available for a range of countries. These are mostly countries in northern Europe. However, there is still a lack o...

Background Metastatic pancreatic cancer (mPC) and its treatments significantly impact health-related quality of life (HRQoL). POLO, a randomized, double-blind, placebo-controlled phase 3 trial evaluated the efficacy of olaparib as maintenance therapy in germline BRCA mutated mPC patients who had not progressed during ≥16 weeks of first-line platinu...

Purpose Results of patient-reported outcome measures (PROMs) are increasingly used to inform healthcare decision-making. Research has shown that response shift can impact PROM results. As part of an international collaboration, our goal is to provide a framework regarding the implications of response shift at the level of patient care (micro), heal...

Aims Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening intravascular hematologic disorder with significant morbidity and premature mortality. Clinical trials (NCT02946463 and NCT03056040) comparing ravulizumab with eculizumab for PNH have supported the non-inferiority of the former and similar safety and tolerability. This se...

Purpose Distress screening has become mandatory and essential in comprehensive cancer care. We evaluated an electronic psycho-oncological adaptive screening (EPAS) which assesses objective indicators of care needs and subjectively perceived care needs and subsequently provides patient feedback with individualized recommendations about psychosocial...

Background The European Organization for research and Treatment of Cancer (EORTC) Core Quality of Life Questionnaire (QLQ-C30) scales are scored on a 4-point response scale, ranging from not at all to very much. Previous studies have shown that the German translation of the response option quite a bit as mäßig violates interval scale assumptions, a...

Purpose General population normative data for the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 questionnaire facilitates interpretation of data assessed from cancer patients. This study aims to present normative data of the general Spanish population. Methods/patients Data were obtained from a prior larger study colle...

Background Contrary to common usage in the health sciences, the term “valid” refers not to the properties of a measurement instrument but to the extent to which data-derived inferences are appropriate, meaningful, and useful for intended decision making. The aim of this study was to determine how validity testing theory (the Standards for Education...

Objectives: To explore the existence and magnitude of the potential impact of response shift on patient-reported outcomes (PRO) change over time in randomized controlled trials (RCTs). Methods: We included RCTs in which the measurement of response shift in at least one PRO for any two time points was reported. PubMed, CINAHL, EMBASE, and PsycINFO d...

Introduction Haemophilia is an inherited bleeding disorder characterised by spontaneous bleeding, often leading to impaired health-related quality of life (HRQoL). Commonly used treatments include episodic and prophylactic treatment regimens. Gene therapies could soon become available, potentially creating a paradigm shift in patient management. A...

Background: The European Organization for research and Treatment of Cancer (EORTC) Core Quality of Life Questionnaire (QLQ-C30) scales are scored on a 4-point response scale, ranging from not at all to very much. Previous studies have shown that the German translation of the response option quite a bit as mäßig violates interval scale assumptions,...

PurposeGeneral population normative data for the European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30 questionnaire facilitates interpretation of data assessed from cancer patients. This study aimed to present normative data of the Spanish general population.Methods/Patients Data were obtained from a prior larger study collect...

Objective To explore the moderating effects of sociodemographic variables on treatment benefits received from participating in an internet intervention for depression. Design Randomised, assessor-blind, controlled trial. Setting Online intervention, with participant recruitment using multiple settings, including inpatient and outpatient medical a...

393 Background: Metastatic pancreatic cancer (mPC) and its treatments significantly impact patients’ (pts) health-related quality of life (HRQoL). POLO, a randomized, double-blind, placebo-controlled phase 3 trial evaluated the efficacy of olaparib as maintenance therapy in mPC pts who had not progressed during ≥16 weeks of first-line platinum-base...

Objectives: For valid and reliable assessment of patients' Health-Related Quality of Life (HRQoL), it is crucial to use psychometrically robust instruments. In the context of rare diseases such as Merkel cell carcinoma (MCC), validated disease-specific instruments are often not available. The Functional Assessment of Cancer Therapy - Melanoma (FAC...

Objective: The PROMIS® physical function (PF) item bank has been developed to standardize patient-reported PF across medical fields. However, evidence of scoring equivalence across cardiology and rheumatology patients is still missing. Therefore, this study aims to investigate both 1) the extent of disease-related differential item functioning (DI...

Aim: To evaluate changes in health-related quality of life (HRQoL) in a Phase II trial (NCT02155647) of treatment-naive patients with metastatic Merkel cell carcinoma treated with avelumab (15-month follow-up). Materials & methods: Mixed-effect Models for Repeated Measures were applied to HRQoL data (FACT-M; EQ-5D-5L) to assess changes over time. C...

Background: The eHealth literacy Questionnaire (eHLQ) is designed to capture users’ perspective on digital health engagements, such as feeling safe and in control while using digital health applications. The eHLQ was developed in Danish and English, and is available in several languages. We report here on the translation and cultural adaption into...

Objective The European Organisation for Research and Treatment of Cancer (EORTC) core questionnaire, QLQ-C30, is a frequently used patient-reported outcome (PRO) instrument to assess health-related quality of life of patients with cancer. To enhance the understanding and interpretation of PRO data, it is important to obtain norm data from the gener...

Background The European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 is a widely used cancer-specific questionnaire assessing 15 domains of health-related quality of life (HRQoL). Our aim was to facilitate the interpretation of scores on this questionnaire by providing Austrian normative data based on a general population sampl...

Background The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group (QLG) has developed computerised adaptive tests (CATs) for the 14 functional and symptom domains of the EORTC QLQ-C30 quality of life questionnaire. This is expected to optimise measurement precision, relevance to patients and flexibility. Here,...

Introduction: To achieve optimized blood glucose concentrations (assessed by HbA1c) and high health-related quality of life (HRQL), children and adolescents with diabetes mellitus type 1 (T1DM) must follow strict disease management strategies. This study aims to investigate HRQL of children and adolescents with T1DM and its association with HbA1c v...

Background: Psychometric theory offers a range of tests that can be used as supportive evidence of both validity and reliability of instruments aimed at measuring patient-reported outcomes (PRO). The aim of this paper is to illustrate psychometric tests within the Classical Test Theory (CTT) framework, comprising indices that are frequently applie...

Background In the single-arm, open-label, multicentre, international phase II trial (NCT02155647 Part A), metastatic Merkel cell carcinoma (mMCC) patients received second-line or later avelumab 10 mg/kg every two weeks. Health-related quality of life (HRQoL) was assessed using the skin cancer-specific FACT-M and the generic EQ-5D. We report HRQoL r...

This trial evaluated quality of life (QoL) using the EORTC QLQ-C30 and the EORTC QLQ-MY20 instruments in 90 patients with relapsed/refractory multiple myeloma during induction and maintenance therapy with eight cycles of ixazomib-thalidomide-dexamethasone, followed by 12 months of ixazomib maintenance therapy. When patient’s baseline QoL was compar...

Introduction: The improvement—or at least maintenance—of health-related quality of life (HRQoL) in children and adolescents is one of the main aims of chronic disease care. This study examines HRQoL of children and adolescents with three different chronic conditions (i.e., diabetes mellitus, asthma, juvenile arthritis) using the computer-adaptive t...

Objective The computer-adaptive test (CAT) of the European Organisation for Research and Treatment of Cancer (EORTC), the EORTC CAT Core, assesses the same 15 domains as the EORTC QLQ-C30 health-related quality of life questionnaire but with increased precision, efficiency, measurement range and flexibility. CAT parameters for estimating scores hav...

Health-related quality of life (HRQoL) acquires increased importance as a target parameter for different stakeholders in healthcare, e. g. to assess treatment outcome in chronically ill patients. In this educational article, we explain the levels of the health-related quality of life construct and associated main dimensions including physical, ment...

Background: Persistent somatic symptoms are associated with psychological distress, impaired function, and medical help-seeking behavior. The Patient Health Questionnaire (PHQ)-15 is used as a screening instrument for somatization and as a monitoring instrument for somatic symptom severity. A bifactorial model has been described, with one general f...

Backround: The number of cancer survivors is growing steadily and increasingly, clinical trials are being designed to include long-term follow-up to assess not only survival, but also late effects and health-related quality of life (HRQOL). Therefore it is is essential to develop patient-reported outcome measures (PROMs) that capture the full rang...

Purpose: Health-related quality of life (HRQoL) is an important patient-reported outcome in clinical and health research. The EQ-5D-Y assesses child and adolescent HRQoL by five items on mobility, self-care, usual activities, pain/discomfort, and anxiety/depression as well as a visual analogue scale (VAS) on the current health state. This study in...

Objectives: To develop the first item bank to measure Stress Resilience (SR) in clinical populations. Study design and setting: Qualitative item development resulted in an initial pool of 131 items covering a broad theoretical SR concept. These items were tested in n=521 patients at a psychosomatic outpatient clinic. Exploratory and Confirmatory...

Objective: To investigate the relationship between regular use of physiotherapy treatment, health locus of control and self-management competency in people with musculoskeletal disorders. Methods: This cross-sectional study included 507 patients between 18 and 70 years of age with musculoskeletal disorders who attended a physiotherapy clinic. Th...

Objective: This study aims at identifying predictors of generic health-related quality of life (HRQoL) in chronically ill children and adolescents over time. The newly developed computer-adaptive test Kids-CAT was used to assess five dimensions of HRQoL. Methods: Longitudinal data from the Kids-CAT study on children and adolescents with asthma,...

ZIELSTELLUNG Die Patient-Reported Outcomes Measurement Information System (PROMIS®) Initiative hat Itembanken zur standardisierten Erfassung verschiedener Konstrukte der gesundheitsbezogenen Lebensqualität auf Basis der Item-Response Theorie (IRT) entwickelt (Cella et al., 2010). Die statistische Beziehung zwischen den einzelnen Items und dem gemes...

Background Web-based interventions for individuals with depressive disorders have been a recent focus of research and may be an effective adjunct to face-to-face psychotherapy or pharmacological treatment. Objective The aim of our study was to examine the early change patterns in Web-based interventions to identify differential effects. Methods W...

PurposeNational initiatives, such as the UK Improving Access to Psychological Therapies program (IAPT), demonstrate the feasibility of conducting empirical mental health assessments on a large scale, and similar initiatives exist in other countries. However, there is a lack of international consensus on which outcome domains are most salient to mon...

The PROMIS® Physical Function (PF) item bank has been calibrated as a unidimensional scale, enabling computerized adaptive testing (CAT). Consequently, several PF subdomains (e.g., upper extremity, mobility) are represented by a composite score. However, different diseases may be associated with condition-specific PF profiles, potentially leading t...

Aims: The new computer-adaptive test (CAT) version of the European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire, the QLQ-CAT, was developed to assess health-related quality of life in cancer patients with increased precision and flexibility compared to the static QLQ-C30. Using item-response theory methods...

Several PROMIS Physical Function measures, including a generic short form (SF-20a) and the PROMIS Mobility bank, are highly significant predictors of general physical performance in different clinical settings. An exception was the PROMIS Upper Extremity bank that appeared to be strongly associated with overall physical performance in rheumatology...

Background: As mortality rates decline, life expectancy increases, and populations age, non-fatal outcomes of diseases and injuries are becoming a larger component of the global burden of disease. The Global Burden of Diseases, Injuries, and Risk Factors Study 2016 (GBD 2016) provides a comprehensive assessment of prevalence, incidence, and years l...