Validation of NASH-CHECK: a novel patient-reported outcome measure for nonalcoholic steatohepatitis.

BACKGROUND: Standardized measures for evaluating patients' experiences with nonalcoholic steatohepatitis (NASH) and their perceived changes with treatment in clinical trials have been limited. To meet this need, a patient-reported outcome (PRO) measure, NASH-CHECK, was developed to evaluate symptoms and health-related quality of life for patients with NASH. The objective of this study was to conduct a quantitative evaluation of the psychometric properties of NASH-CHECK. METHODS: The study used data from a phase 2, randomized controlled trial of adult patients with NASH (NCT02855164). Analyses were conducted to determine the optimal scoring of NASH-CHECK and to evaluate reliability, construct validity, and ability to detect change in NASH-CHECK scale scores. RESULTS: Data were available for 253 patients with NASH (61% female; mean [standard deviation] age = 53 [12] years). Following initial item-level analyses, including correlations and exploratory factor analysis, three items were removed from the measure. Confirmatory factor analysis supported the formation of four multi-item scales (Cognitive Symptoms, Activity Limitations, Social Impact, and Emotional Impact) and five single-item scales (Abdominal Pain, Abdominal Bloating, Fatigue, Sleep, and Itchy Skin). Psychometric analyses of the final NASH-CHECK scales provided support for their internal reliability, test-retest reliability, construct validity, and ability to detect change. CONCLUSION: The results support NASH-CHECK as a reliable, valid, and responsive measure to assess patients' perspectives of symptoms and the health-related quality of life impact of NASH in clinical trials and in routine practice.

Development of a Patient-Reported Outcome Measure for Non-Alcoholic Steatohepatitis (NASH-CHECK): Results of a Qualitative Study.

BACKGROUND: Non-alcoholic steatohepatitis (NASH) is a progressive form of non-alcoholic fatty liver disease characterized by excessive liver fat accumulation, inflammation, cell injury, and fibrosis. It is viewed as largely asymptomatic in its earlier (non-cirrhotic) stages, and information on the patient-perceived impact of NASH is scarce. OBJECTIVE: This study aimed to develop a NASH-specific patient-reported outcome (PRO) measure (NASH-CHECK) for use as a trial endpoint, using methods compliant with regulatory expectations. METHODS: A NASH conceptual model was developed based on the literature and clinical/patient expert review. The model guided concept elicitation (CE) interviews in patients with non-cirrhotic NASH recruited via a US tertiary care center. NASH-CHECK content was generated via thematic analysis of CE data and review by clinical/patient experts. Cognitive debriefing (CD) interviews with US patients evaluated content validity. RESULTS: The literature review confirmed that NASH impacts on functioning and health-related quality of life (HRQoL). Overall, 23 CE and 20 CD interviews were conducted. Key symptoms reported in CE interviews included pain in the upper-right abdomen (n = 14), fatigue (n = 18), poor sleep quality (n = 12), impaired memory (n = 13), and reduced focus (n = 11); key HRQoL impacts included impaired physical functioning, reduced ability to conduct daily living tasks, reduced quality of relationships, low mood, anxiety, and self-consciousness. The 52-item first-draft NASH-CHECK was reduced to 31 items based on patient feedback on item relevance, acceptability, and comprehension. CONCLUSIONS: The interviews revealed key symptoms and broad HRQoL impacts of NASH. As a disease-specific PRO measure assessing symptoms and HRQoL, the NASH-CHECK is relevant, comprehensive, and acceptable to patients and clinicians.

Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers.

BACKGROUND AND METHODS: Metachromatic leukodystrophy (MLD) is a rare, autosomal recessive lysosomal storage disease caused by deficient activity of arylsulfatase A. Neurological involvement results in severe disability and premature death, but understanding of the natural history of the disease remains limited. In this study, 32 caregivers of patients with MLD in the USA (16 with late-infantile MLD; 16 with juvenile MLD) were interviewed about their experiences of the disease. Qualitative analysis of the interview transcripts was performed to gain insights into symptom onset, the diagnostic process and disease progression, with a focus on the differences between late-infantile and juvenile MLD. RESULTS: The mean ages of patients at interview were 7.6 years and 20.7 years for individuals with late-infantile and juvenile MLD, respectively. Patients with late-infantile MLD had a mean age of 1.5 years at symptom onset and 2.6 years at diagnosis. The most common initial symptoms in this group related to problems with gross motor function (12/16 patients); 11 patients never learned to walk independently. For patients with juvenile MLD, the mean ages at symptom onset and diagnosis were 8.7 years and 11.6 years, respectively. Cognitive or social/behavioural problems were the most common first reported symptoms in this group (9/16 and 7/16 patients, respectively); these were generally followed by deterioration in motor function. The rate of functional decline was more rapid in patients with late-infantile MLD than those with juvenile MLD; the mean time from first symptom to first functional loss was 1 year versus 6.1 years, respectively. Nine patients with juvenile MLD and three with late-infantile MLD had undergone a haematopoietic stem cell transplant; outcomes following transplant were variable. CONCLUSIONS: Our data highlight clear overall differences in symptom profiles and disease progression between late-infantile and juvenile MLD, but also indicate some degree of interindividual variability within each subtype. These findings are broadly consistent with previously published descriptions of MLD and enhance our knowledge of the natural history of the disease, which ultimately should help to improve patient care and aid assessments of the effectiveness of disease-related interventions in the future.

A qualitative study of the impact of Crohn's disease from a patient's perspective.

OBJECTIVE: To understand how the lives of people with Crohn's disease (CD) are affected. Most research in CD has focused on symptoms and functioning rather than on how these outcomes influence quality of life (QoL). DESIGN: As part of a study to develop a CD-specific patient-reported outcome measure, qualitative interviews were conducted with patients from Manchester Royal Infirmary to determine how CD affects QoL. The needs-based model was adopted for the study. The interviews, which took the form of focused conversations covering all aspects of the impact of CD and its treatment, were audio-recorded. Theoretical thematic analysis of the transcripts identified needs affected by CD. RESULTS: Thirty patients (60% female) aged 25-68 years were interviewed. Participants had experienced CD for between 2 and 40 years. Nearly 1300 statements relating to the impact of CD were identified. Thirteen main need themes were identified: nutrition, hygiene, continence, freedom from infection, security, self-esteem, role, attractiveness, relationships, intimacy, clear-mindedness, pleasure and autonomy. CONCLUSIONS: The findings from the interviews indicate that CD has a major impact on need-fulfilment. Such issues should be addressed in CD audit, clinical trials and when evaluating clinical practice.

Portuguese validation of the Cambridge pulmonary hypertension outcome review (CAMPHOR) questionnaire.

BACKGROUND: Patients with pulmonary arterial hypertension (PAH) and other forms of precapillary pulmonary hypertension (PH) have impaired quality of life (QoL). The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) is a PH-specific patient-reported outcome measure that assesses symptoms, activity limitations and QoL. It was originally developed in UK-English. The main objective of this study was to create an adaptation of the CAMPHOR suitable for a Portuguese-speaking population. METHODS: A multi-step approach was followed: bilingual and lay panel translation; cognitive debriefing interviews; and psychometric testing in repeated postal surveys (2 weeks apart) including assessment of internal consistency, reproducibility and validity. The Nottingham Health Profile (NHP) questionnaire was used as a comparator instrument to test convergent validity. RESULTS: The CAMPHOR was translated without difficulty by the two panels. Cognitive debriefing interviews showed the questionnaire was easily understood and considered relevant to patients' experience with their illness. Psychometric evaluation was performed with 50 PAH patients (47 ± 14 years, 37 women). Cronbach's alpha coefficients showed good internal consistency for the three CAMPHOR scales [Symptoms = 0.95; Activities = 0.93 and QoL = 0.94]. Test-retest coefficients showed that all scales had excellent reliability (Symptoms = 0.94; Activities = 0.89 and QoL = 0.93), indicating low levels of random measurement error. The CAMPHOR correlated as expected with the NHP. The magnitude of correlations followed a similar pattern to those in the original development study. The CAMPHOR also exhibited evidence of known group validity in its ability to distinguish between self-reported severity and general health groups. CONCLUSIONS: A valid and reliable version of the CAMPHOR questionnaire for the European Portuguese-speaking population was developed and is recommended for use.

Assessing quality of life in Crohn's disease: development and validation of the Crohn's Life Impact Questionnaire (CLIQ).

PURPOSE: Despite the significant impact of Crohn's disease (CD) on patients' physical and emotional well-being, no CD-specific patient-reported outcome (PRO) measure is available for determining the efficacy of interventions. The objective of the study was to develop and validate the Crohn's Life Impact Scale (CLIQ), the first such measure. METHODS: Questionnaire content was derived from qualitative interviews with CD patients and face and content validity assessed by cognitive debriefing interviews (CDIs) with patients. A postal survey was conducted to identify the final scale, confirm its unidimensionality and determine reproducibility and construct validity. A subset of the respondents was sent a second questionnaire package 2 weeks after the first. The survey included the CLIQ, Nottingham Health Profile (NHP) and Unidimensional Fatigue Impact Scale (U-FIS). RESULTS: Content analysis was conducted on the 30 interview transcripts and a draft scale produced. The CDIs indicated that the draft scale was relevant, clear and easy to use. The questionnaire package was completed by 273 CD patients (65.6 % male; aged 16-79 (mean 43.9; SD 15.1) years). Of these, 104 also completed the second package. Rasch analysis confirmed a 27-item unidimensional QoL scale (p < 0.05). Both internal consistency and test-retest reliability were high (0.91). Scores on the CLIQ were related to both physical and emotional impairments (NHP) and to fatigue (U-FIS). CONCLUSION: The CLIQ, the first CD-specific PRO, is unidimensional and has excellent psychometric properties. It should prove to be a valuable tool for evaluating the impact of CD and its treatment from the patients' perspective.

Further international adaptation and validation of the Rheumatoid Arthritis Quality of Life (RAQoL) questionnaire.

The Rheumatoid Arthritis Quality of Life (RAQoL) questionnaire was developed directly from rheumatoid arthritis (RA) patients in the United Kingdom and the Netherlands to measure quality of life (QoL). Since then, it has become widely used in clinical studies and trials and has been adapted for use in 24 languages. The objective was to develop and validate 11 additional language versions of the RAQoL in US English, Mexican Spanish, Argentinean Spanish, Belgian French, Belgian Flemish, French, Romanian, Czech, Slovakian, Polish and Russian. The language adaptation and validation required three stages: translation, cognitive debriefing interviews and validation survey. The translation process involved a dual-panel methodology (bilingual panel followed by a lay panel). The validation survey tested the psychometric properties of the new scales and included either the Nottingham Health Profile (NHP) or the Health Assessment Questionnaire (HAQ) as comparators. Internal consistency of the new language versions ranged from 0.90 to 0.97 and test-retest reliability from 0.85 to 0.99. RAQoL scores correlated as expected with the HAQ. Correlations with NHP sections were as expected: highest with energy level, pain and physical mobility and lowest with emotional reactions, sleep disturbance, and social isolation. The adaptations exhibited construct validity in their ability to distinguish subgroups of RA patients varying by perceived disease severity and general health. The new language versions of the RAQoL meet the high psychometric standards of the original UK English version. The new adaptations represent valid and reliable tools for measuring QoL in international clinical trials involving RA patients.

Psychometric performance of the CAMPHOR and SF-36 in pulmonary hypertension.

BACKGROUND: The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) and the Medical Outcomes Study Short Form 36 (SF-36) are widely used to assess patient-reported outcome in individuals with pulmonary hypertension (PH). The aim of the study was to compare the psychometric properties of the two measures. METHODS: Participants were recruited from specialist PH centres in Australia and New Zealand. Participants completed the CAMPHOR and SF-36 at two time points two weeks apart. The SF-36 is a generic health status questionnaire consisting of 36 items split into 8 sections. The CAMPHOR is a PH-specific measure consisting of 3 scales; symptoms, activity limitations and needs-based QoL. The questionnaires were assessed for distributional properties (floor and ceiling effects), internal consistency (Cronbach's alpha), test-retest reliability and construct validity (scores by World Health Organisation functional classification). RESULTS: The sample comprised 65 participants (mean (SD) age = 57.2 (14.5) years; n(%) male = 14 (21.5%)). Most of the patients were in WHO class 2 (27.7%) and 3 (61.5%). High ceiling effects were observed for the SF-36 bodily pain, social functioning and role emotional domains. Test-retest reliability was poor for six of the eight SF-36 domains, indicating high levels of random measurement error. Three of the SF-36 domains did not distinguish between WHO classes. In contrast, all CAMPHOR scales exhibited good distributional properties, test retest reliability and distinguished between WHO functional classes. CONCLUSIONS: The CAMPHOR exhibited superior psychometric properties, compared with the SF-36, in the assessment of PH patient-reported outcome.

Development and validation of the Bowel Cleansing Impact Review (BOCLIR).

OBJECTIVE: Acceptability and tolerability of bowel cleansers influence whether patients are able to complete the prescribed dose and, consequently, the quality of the cleansing achieved. No standardised means of assessing patients' experience of using bowel cleansing is currently available. The aim of the study was to develop the Bowel Cleansing Impact Review (BOCLIR) to assess patient response to bowel cleansing products. DESIGN: Content was derived from qualitative interviews. Face and content validity were assessed via cognitive-debriefing interviews. Finally, patients completed the BOCLIR and a demographic questionnaire. Item response theory (Rasch analysis) was employed for item reduction and assessment of unidimensionality. Internal consistency and construct validity were also assessed. RESULTS: Analysis of 40 interviews resulted in the production of three scales; patient satisfaction, symptomatic impact and activity limitations. Scales were designed to be used alone or together. 19 debriefing interviews demonstrated BOCLIR acceptability, relevance and ease of completion. The validation survey involved 166 patients (52% male, mean (SD) age 54.3 (15.2) years). After misfitting and redundant items were removed all scales fit the Rasch model confirming their unidimensionality. Cronbach's α-coefficients were high (0.77-0.94) indicating good internal consistency. Scores on the BOCLIR were related to patients' willingness to use the product in future and ease of drinking the full preparation (p<0.01 for each scale). CONCLUSIONS: The BOCLIR is a new measure consisting of three unidimensional scales (satisfaction, symptoms and activity limitations) with good psychometric and scaling properties. The BOCLIR will allow accurate assessment of patients' response to bowel cleansing preparations.

Adaptation and validation of the Cambridge pulmonary hypertension outcome review for Sweden.

BACKGROUND: The Cambridge pulmonary hypertension outcome review (CAMPHOR) is the first pulmonary hypertension-specific instrument for the assessment of the patient's perceived symptoms, activity limitations and quality of life (QoL). PURPOSE: To produce and validate a Swedish language version of the CAMPHOR. METHODS: Bilingual (n = 5) and lay panels (n = 5) were conducted to translate the CAMPHOR into Swedish. This new questionnaire was then field-tested with 14 patients and finally, it underwent psychometric evaluation by means of a postal validation study involving 38 patients with pulmonary hypertension (PH). RESULTS: Few problems were experienced in translating the CAMPHOR into Swedish. The field-test participants found the scales relevant, comprehensible and easy to complete. Psychometric analyses showed that the Swedish adaptation was successful. The Swedish CAMPHOR scales had good internal consistency. Cronbach's alpha coefficients were 0.92 for the symptoms scale, 0.92 for activity limitations and 0.95 for the quality of life. Predicted correlations with the Nottingham Health Profile provided evidence of the construct validity of the scales. The Swedish scales also indicated known groups validity. CONCLUSIONS: The Swedish version of the CAMPHOR is a reliable and valid measure of the impact of pulmonary hypertension on the lives of affected patients. It is recommended for use in clinical studies and routine practice in pulmonary hypertension patients.

The German adaptation of the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR).

BACKGROUND: Individuals with precapillary pulmonary hypertension (PH) experience severely impaired quality of life. A disease-specific outcome measure for PH, the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) was developed and validated in the UK and subsequently adapted for use in additional countries. The aim of this study was to translate and assess the reliability and validity of the CAMPHOR for German-speaking populations. METHODS: Three main adaptation stages involved; translation (employing bilingual and lay panels), cognitive debriefing interviews with patients and validation (assessment of the adaptation's psychometric properties). The psychometric evaluation included 107 patients with precapillary PH (60 females; age mean (standard deviation) 60 (15) years) from 3 centres in Austria, Germany and Switzerland. RESULTS: No major problems were found with the translation process with most items easily rendered into acceptable German. Participants in the cognitive debriefing interviews found the questionnaires relevant, comprehensive and easy to complete. Psychometric analyses showed that the adaptation was successful. The three CAMPHOR scales (symptoms, activity limitations and quality of life) had excellent test-retest reliability correlations (Symptoms = 0.91; Activity limitations = 0.91; QoL = 0.90) and internal consistency (Symptoms = 0.94; Activity limitations = 0.93; QoL = 0.94). Predicted correlations with the Nottingham Health Profile provided evidence of the construct validity of the CAMPHOR scales. The CAMPHOR adaptation also showed known group validity in its ability to distinguish between participants based on perceived general health, perceived disease severity, oxygen use and NYHA classification. CONCLUSIONS: The CAMPHOR has been shown to be valid and reliable in the German population and is recommend for use in clinical practice.

The living with chronic obstructive pulmonary disease scale was successfully adapted for use in Southern European (Italian and Spanish) and Eastern European (Russian) cultures.

OBJECTIVE: The living with chronic obstructive pulmonary disease (LCOPD) scale was developed in the United Kingdom and the United States to assess the quality of life (QoL) of people with chronic obstructive pulmonary disease. The scale was shown to be well accepted by patients, to be unidimensional, and to have very good psychometric properties. This article reports on the adaptation of the measure for use in representative Southern European (Italian and Spanish) and Eastern European (Russian) languages. STUDY DESIGN AND SETTING: The same methodology was used in each country: translation panels were held to produce new language versions. These were then tested with patients to ensure face and content validity. Finally, test-retest postal surveys were conducted to establish internal consistency, reproducibility, and construct validity. RESULTS: Few problems were found in translating the LCOPD, and patient interviews indicated that the measure was considered relevant and easy to complete. The validation surveys showed that the new versions of the LCOPD were consistent, reproducible, and had good construct validity. The psychometric properties of the new versions were similar to those found for the original U.K. and U.S. versions. CONCLUSION: The study was successful in adapting the LCOPD for use in Southern and Eastern European languages.

Can we rely on the Dermatology Life Quality Index as a measure of the impact of psoriasis or atopic dermatitis?

The Dermatology Life Quality Index (DLQI) is a widely used health-related quality of life measure. However, little research has been conducted on its dimensionality. The objectives of the current study were to apply Rasch analysis to DLQI data to determine whether the scale is unidimensional, to assess its measurement properties, test the response format, and determine whether the measure exhibits differential item functioning (DIF) by disease (atopic dermatitis versus psoriasis), gender, or age group. The results show that there were several problems with the scale, including misfitting items, DIF by disease, age, and gender, disordered response thresholds, and inadequate measurement of patients with mild illness. As the DLQI did not benefit from the application of Rasch analysis in its development, it is argued that a new measure of disability related to dermatological disease is required. Such a measure should use a coherent measurement model and ensure that items are relevant to all potential respondents. The current use of the DLQI as a guide to treatment selection is of concern, given its inadequate measurement properties.

Validation of the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) for the Australian and New Zealand population.

BACKGROUND AND OBJECTIVE: Individuals with pulmonary arterial hypertension (PAH) experience severely impaired quality of life. A disease-specific patient reported outcome measure for PAH (the Cambridge Pulmonary Hypertension Outcome Review--CAMPHOR) has recently been developed and validated in the UK, USA and Canada. It has demonstrated reliability and validity in PAH populations in these countries. The aim of this study was to assess the reliability and validity of the CAMPHOR in an Australian and New Zealand (NZ) PAH population. METHODS: Semistructured interviews were conducted with a cohort of 15 PAH patients (aged 68.9±10.0 years; 11 women) to determine the relevance of the CAMPHOR and ensure the terminology and language used was understandable and appropriate for our PAH population. The test-retest reliability, internal consistency and construct validity of the CAMPHOR were then examined in an Australian and NZ PAH population (n=61, aged 56.9±14.5 years; 48 women). RESULTS: Data from the patient interviews confirmed that the CAMPHOR is appropriate for use in our PAH population. The three CAMPHOR scales (symptoms, activity limitations and quality of life) had excellent test-retest reliability (correlation coefficients (r(s))=0.86-0.94, P<0.01) and internal consistency (Cronbach's alpha coefficients=0.89-0.92). The CAMPHOR also demonstrated the ability to distinguish between individuals with PAH who differed according to World Health Organisation functional class. CONCLUSIONS: We have shown the CAMPHOR to be valid and reliable in an Australian and NZ PAH population and recommend its use in clinical practice.

Adaptation of the QoL-AGHDA scale for adults with growth hormone deficiency in four Slavic languages.

PURPOSE: The Quality of Life in Adult Growth Hormone Deficiency Assessment (QoL-AGHDA) is a disease-specific quality of life measure specific to individuals who are growth hormone deficient. The present study describes the adaptation of the QoL-AGHDA for use in the following four Slavic languages; Czech, Polish, Serbian and Slovakian. METHODS: The study involved three stages in each language; translation, cognitive debriefing and validation. The validation stage assessed internal consistency (Cronbach's alpha), reproducibility (test-retest reliability using Spearman's rank correlations), convergent and divergent validity (Correlations with the NHP) and known group validity. RESULTS: The QoL-AGHDA was successfully translated into the target languages with minimal problems. Cognitive debriefing interviewees (n = 15-18) found the measures easy to complete and identified few problems with the content. Internal consistency (Czech Republic = 0.91, Poland = 0.91, Serbia = 0.91 and Slovakia = 0.89) and reproducibility (Czech Republic = 0.91, Poland = 0.91, Serbia = 0.88 and Slovakia = 0.93) were good in all adaptations. Convergent and divergent validity and known group validity data were not available for Slovakia. The QoL-AGHDA correlated as expected with the NHP scales most relevant to GHD. The QoL-AGHDA was able to distinguish between participants based on a range of variables. CONCLUSIONS: The QoL-AGHDA was successfully adapted for use in the Czech Republic, Poland, Serbia and Slovakia. Further validation of the Slovakian version would be beneficial. The addition of these new language versions will prove valuable to multinational clinical trials and to clinical practice in the respective countries.

Development and validation of the living with chronic obstructive pulmonary disease questionnaire.

PURPOSE: Available patient-reported outcome (PRO) measures for chronic obstructive pulmonary disease (COPD) focus primarily on impairment (symptoms) and activities (functioning). The purpose of the study was to develop a patient-based PRO measure for COPD that captures the overall everyday impact of living with COPD from the patient's perspective. METHODS: LCOPD items (Living with COPD Questionnaire) were generated from qualitative interviews in the U.K. and focus groups in the U.S.A. The draft measure was tested for face and content validity in both countries. Item reduction and testing for reproducibility and construct validity was conducted via Rasch and traditional psychometric analyses. RESULTS: The draft LCOPD was found to be relevant and acceptable to patients in the U.K. (N = 19) and U.S. (N = 16). Application of Rasch analysis to data collected in validation studies (n = 162 in the U.K. and 145 in U.S.) identified a 22-item scale that measured a single construct in both countries. Psychometric analyses indicated that this version was internally consistent and reproducible. Scores on the measure were related as expected to clinician ratings of disease severity and patient ratings of COPD severity and general health. CONCLUSIONS: The LCOPD is a new measure examining the everyday impact of living with COPD. It demonstrates good scaling properties and may prove valuable in understanding treatment benefits.

The brazilian version of the Quality of Life Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDA): Four-stage translation and validation / Versão brasileira do questionário "Quality of Life - Assessment of Growth Hormone Deficiency in Adults" (QoL-AGHDA): Tradução e validação em quatro estágios

OBJECTIVE: This study reports on the Brazilian Portuguese adaptation of the QoL-AGHDA (Quality of Life Assessment of Growth Hormone Deficiency in Adults) for use in adult growth hormone deficient (GHD) patients. MATERIALS AND METHODS: The translation process adopted the dual panel methodology. The questionnaire was tested through field-test interviews (16 GHD patients). In the final stage, data from 120 GHD patients (81 included in a test-retest analysis) were analyzed for internal consistency, test-retest reliability, convergent validity and validity among known groups. RESULTS: The translation panels were successful and the draft version was amended to improve the wording as a result of the field-test interviews. Cronbach's alpha was 0.90 and test-retest reliability 0.88. QoL-AGHDA scores had the expected pattern of association with NHP scale scores and QoL-AGHDA was able to differentiate significantly between patients based on patient-reported general health (p < 0.01) and QoL (p < 0.01). CONCLUSIONS: The adaptation of the QoL-AGHDA for a Brazilian population was successful and the adapted questionnaire was shown to be reliable and valid.

OBJETIVO: Este estudo relata o processo de adaptação da versão brasileira do questionário QoL-AGHDA (Quality of Life - Assessment of Growth Hormone Deficiency in Adults) para pacientes com deficiência do hormônio de crescimento (DGH). MATERIAIS E MÉTODOS: A tradução adotou a metodologia de duplo painel. O questionário foi testado por intermédio de entrevistas direcionadas com 16 pacientes com DGH. No estágio final, dados de 120 pacientes com DGH (81 com teste/reteste) foram analisados para consistência interna, confiabilidade teste/reteste, validade convergente e validade entre grupos conhecidos. RESULTADOS: Os grupos de tradução foram bem-sucedidos e a versão final foi adaptada seguindo sugestões obtidas das entrevistas com os 16 pacientes. O coeficiente alfa de Cronbach foi 0,90, confiabilidade teste/reteste 0,88, escores QoL-AGHDA se correlacionaram com o NHP (p < 0,01) e também com a saúde geral relatada pelos pacientes (p < 0,01). CONCLUSÕES: A adaptação do QoL-AGHDA para a população brasileira foi bem-sucedida, e a nova versão demonstrou ser válida e confiável.

Interpreting scores on multiple sclerosis-specific patient reported outcome measures (the PRIMUS and U-FIS).

BACKGROUND: The PRIMUS is a Multiple Sclerosis (MS)-specific suite of outcome measures including assessments of QoL (PRIMUS QoL, scored 0-22) and activity limitations (PRIMUS Activities, scored 0-30). The U-FIS is a measure of fatigue impact (scored 0-66). These measures have been fully validated previously using an MS sample with mixed diagnoses. The aim of the present study was to validate the measures further in a specifically Relapse Remitting MS (RRMS) sample and to provide preliminary evidence of the responder definitions (RD; also known as minimal important difference) for these instruments. METHODS: Data were derived from a multi-country efficacy trial of MS patients with assessments at baseline and 12 months. Baseline data were used to assess the internal reliability and validity of the measures. Both anchor-based and distribution-based approaches were employed for estimating RD. Anchor-based estimates were based on published RD values for the EQ-5D and were assessed for those improving and deteriorating separately. Distribution-based estimates were based on standard error of measurement (SEM), change score equivalent to 0.30, and change score equivalent to 0.50, effect sizes (ES). RESULTS: The sample included 911 RRMS patients (67.3% female, age mean (SD) 36.2 (8.4) years, duration of MS mean (SD) 4.8 (5.2) years). Results showed that the PRIMUS and U-FIS had good internal consistency. Appropriate correlations were observed with comparator instruments and both measures were able to distinguish between participants based on Expanded Disability Status Scale scores and time since diagnosis. The anchor-based and distribution-based RD estimates were: PRIMUS Activities range = 1.2-2.3, PRIMUS QoL range = 1.0-2.2, and U-FIS range = 2.4-7.0. CONCLUSIONS: The results show that the PRIMUS and U-FIS are valid instruments for use with RRMS patients. The analyses provide preliminary information on how to interpret scores on the scales. These data will be useful for assessing treatment efficacy and for powering clinical studies. TRIAL REFERENCE NUMBER: ClinicalTrials.gov Identifier NCT00340834.

International development of the patient-reported outcome indices for multiple sclerosis (PRIMUS).

BACKGROUND: The Patient-Reported Indices for Multiple Sclerosis (PRIMUS) comprises a suite of three scales for assessing symptoms, activity limitations, and quality of life in multiple sclerosis (MS). It was developed in the UK and has been shown to have excellent psychometric properties. This study describes the adaptation of eight language versions for Canadian English, Canadian French, French, German, Italian, Spanish, Swedish, and US English. METHODS: The PRIMUS was translated using the dual-panel process. Cognitive debriefing interviews conducted with MS patients assessed face and content validity. Psychometric and scaling properties were assessed via a two-administration postal survey conducted in each country involving the PRIMUS, the Nottingham Health Profile (NHP), the Unidimensional Fatigue Impact Scale (U-FIS), and demographic questions. RESULTS: Cognitive debriefing interviews demonstrated the acceptability of the new language versions. Analysis of survey data showed that the new language versions of the three PRIMUS scales were unidimensional (as indicated by fit to the Rasch model) and that they had good internal consistency and reproducibility. PRIMUS scale scores correlated as expected with those on the NHP and the U-FIS. The scales in all countries were able to discriminate between groups of patients on the basis of their self-reported MS severity, general health, and employment status. CONCLUSIONS: The PRIMUS was successfully adapted into eight new languages. Most of the tests showed the PRIMUS to have good unidimensionality and to have good internal consistency, reproducibility, and construct validity. The measure is now available for use in clinical studies and trials involving these countries and the UK. Further work is required to assess the measure's responsiveness.

International development of the Unidimensional Fatigue Impact Scale (U-FIS).

OBJECTIVE: The 22-item Unidimensional Fatigue Impact Scale (U-FIS) provides an index of the impact of fatigue on patients with multiple sclerosis (MS). The objective is to produce eight new language versions of the U-FIS: Canadian-English, Canadian-French, French, German, Italian, Spanish, Swedish, and US-English. METHODS: The U-FIS was translated via two translation panels. Cognitive debriefing interviews conducted with patients in each country assessed face and content validity. Scaling and psychometric properties were assessed via survey data with patients in each country completing the U-FIS, Nottingham Health Profile (NHP), and demographic questions. RESULTS: Cognitive debriefing interviews demonstrated U-FIS acceptability. Analysis of postal survey data showed all new language versions to be unidimensional. Reliability was high, with test-retest correlations and internal-consistency coefficients exceeding 0.85. Initial evidence of validity was provided by moderate to high correlations with NHP scales. The U-FIS was able to discriminate between groups based on employment status, perceived MS severity, and general health. CONCLUSION: The U-FIS is a practical new measure of the impact of fatigue. It was successfully adapted into eight new languages to broaden availability for researchers. Psychometric analyses indicated that the new language versions were unidimensional and reproducible with promising construct validity.