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Systematic Review of Psychometric Properties of The EQ-5D Questionnaires In Patients with Hematologic Malignant Neoplasms
A388
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 3 4 7 – A 7 6 6
to other regions. 6 articles compared or developed further TTO techniques. Of the 65 comparisons between TTO and EQ-5D, 6 articles were excluded due to not measureable results. Results from the remaining 59 articles showed a broad spectrum of results. In general, TTO methods were seen as more sensitive or suitable than an EQ-5D questionnaire, while some others argue the EQ-5D might be easier in its application with the general population or patients than TTO. Conclusions: The analysis showed differences in the assessment of the two methods TTO and EQ-5D with a more sensitive and disease specific coverage of the TTO method. PRM168 Qualitative Research Approaches In Rare Diseases: Acid Sphingomyelinase Deficiency (ASMD) Symptoms and Impact as Reported by Patients and Caregivers Avetisyan R1, Hareendran A2, Stringer S3, Tan S1, Sanson B1, Hass S1 Genzyme, Cambridge, MA, USA, 2Evidera, London, UK, 3Evidera, Bethesda, MD, USA
1Sanofi
Objectives: Conducting large studies in rare diseases is challenging. However, much valuable information can be obtained using qualitative and mixed-method approaches, such as in the case of acid sphingomyelinase deficiency (ASMD). ASMD, also called Niemann-Pick Disease Types A and B, is a rare, genetic, life-threatening condition with an estimated incidence of 0.4-0.6 per 100,000. There are significant unmet needs associated with this condition, and currently disease management is limited to symptomatic/palliative care only. The aim of this research was to address the lack of evidence about ASMD and specifically patients’ experiences of the condition. Methods: A qualitative research study was conducted with 22 people with ASMD and/or their caregivers, to obtain information about the disease, its symptoms and impact. Participants were interviewed using open-ended questions and probes developed based on literature review and clinical expert interviews. Participants also completed symptom importance/bothersomeness ratings and questionnaires to characterize the sample. Results were also used to develop a conceptual disease model (CDM). Results: The mean (range) age was 37(18-63) years for adults and 10 (2-17) years for pediatric patients; 64% of adult and 45% of children were female. The most commonly reported symptoms were enlarged abdomen (due to hepatosplenomegaly), breathing difficulties, frequent respiratory infections, abdominal pain, bleeding/bruising, back and limb pain, and fatigue. Specific symptoms reported as being most bothersome included: fatigue, shortness of breath, bruising/bleeding, lower limb/joint pain, sleep disturbances, headaches, and diarrhea. ASMD was found to substantially impact physical, social, and emotional functioning in both adults and children. A CDM was developed and used to identify concepts most common and relevant to patients. Conclusions: This study describes experiences of patients with ASMD and its significant impact on patients’ lives. The results can be used to support the development of ASMD disease-specific measures for the evaluation of patient-focused health outcomes in the condition. PRM169 Fore! Sweeping Generalisations do not Bowl Over all Cultures… Cultural Adaptation in the Kidney Disease Quality of Life Questionnaire Richards A, Mead H, Towns L ICON Plc, Abingdon, UK
Objectives: The aim of this study is to assess how the moderate activities in the Kidney Disease Quality of Life Questionnaire (KDQoL), namely, ‘vacuuming, bowling and golf’ are adapted to suit various cultures. Methods: Linguistic Validation was carried out on a total 60 language pairs spanning 41 countries (covering 11 language families) and went through cognitive debriefing whereby adaptations were confirmed or discussed in more detail. We assessed if changes were made for each language pair and calculated what percentage of languages necessitated an adaptation based on the cultural norms. Based on this data, we then evaluated the activities that were considered more culturally appropriate for the target culture. Results: An overview of the language families that deemed an adaptation necessary, the types of activities that were substituted, and how this has enhanced the conceptual equivalency of the translation was created. The source activities were culturally adapted into a range of equivalent moderate activities judged relevant to the target population. Golf was a particularly troublesome term as many cultures considered this a niche sport and was therefore not fully understood as a moderate activity by the general public. Out of the 60 translations, changes were necessary in 60% of cases for the activities to relate to their culture. 5% used ‘gathering berries and mushrooms’ as an equivalent, 17% adapted golf as variants of walking such as ‘forest walking’, 5% changed ‘bowling’ to ‘Tai-chi’ and ‘vacuuming’ was changed to ‘sweeping’ for 11% of languages. Conclusions: To enable understanding and relatability to the moderate activities given in the questionnaire, it was essential to adapt the activity to one that would be familiar to the target culture. The range of alternative activities that replaced the original source terms demonstrates that in order to achieve conceptual equivalence, cultural adaptation is often vital to the translation process. PRM170 Systematic Review of Psychometric Properties of The EQ-5D Questionnaires In Patients with Hematologic Malignant Neoplasms Mlynczak K1, Wojcik A1, Golicki D2 1HealthQuest, Warsaw, Poland, 2Department of Experimental & Clinical Pharmacology, Medical University of Warsaw, Warsaw, Poland
Objectives: The aim of this study was to systematically review the published literature to assess the psychometric properties (validity, reliability and responsiveness) of the EQ-5D questionnaires (EQ-5D-3L, EQ-5D-5L, EQ-5D-Y) reported in patients with hematologic malignant neoplasms (ICD-10: C81 - C96 and D46). Methods: Nine databases were systematically searched (MEDLINE (PubMed), EMBASE, Cochrane Library, EuroQol website, CEA Registry, LILACS (VHL), ProQuest, CRD and Web of Science) up to February 2016. Additional references were obtained from reviewed papers. No study design restrictions were implemented. The search strategy
combined MESH, EMTREE and free-text terms respecting the population and the EQ-5D instruments. Data from studies meeting the inclusion criteria were extracted using a pre-determined extraction form, specified for the psychometric properties. The review data were grouped into three categories: validity, reliability and responsiveness. Results: Three hundred eighty eight abstracts were initially identified and screened and 101 records were screened in full text. Finally, 21 full text papers were included into the review of Hodgkin and non-Hodgkin lymphomas, leukemias, myelodysplastic syndrome. Psychometric properties, validity, reliability and responsiveness were described in 14 (n= 2884), 4 (n= 379) and 11 (n= 4416) papers, respectively. Known group analysis was the most common method for validity assessment (10 papers; n= 2007 participants). In terms of responsiveness effect size, SRM and ceiling and floor effects were presented in 3 (n= 773), 2 (n= 913) and 2 (n= 913) papers, respectively. The reliability of the EQ-5D questionnaire was measured by retesting, assessing inter-rater comparison and internal consistency in 2 (n= 182), 2 (n= 197), and 1 (n= 148) trials, respectively. Conclusions: The psychometric properties of EQ-5D questionnaire were described in a large number of clinical trials in patients with hematologic malignant neoplasms. However, differences in populations of studies make difficult the aggregation of results. PRM171 Content Validation of the Pulmonary Arterial Hypertension Symptoms and Impact (PAH-SYMPACT®) Questionnaire Gomberg-Maitland M1, Channick R2, Chin K3, Fischer A4, Frantz R5, Roberts L6, Miller C7, Hunsche E8, Zamanian R9, Badesch D4 1University of Chicago, Chicago, IL, USA, 2Massachusetts General Hospital, Boston, MA, USA, 3University of Texas Southwestern, Dallas, TX, USA, 4University of Colorado Denver, Aurora, CO, USA, 5Mayo Clinic Rochester, Rochester, MN, USA, 6Evidera, Bethesda, MD, USA, 7Piedmont Healthcare, Atlanta, GA, USA, 8Actelion Pharmaceuticals Ltd, Allschwil, Switzerland, 9Stanford University, Stanford, CA, USA
Objectives: PAH-SYMPACT® is a patient reported outcome (PRO) instrument; the draft consists of 16 symptom and 25 impact items. The PRO was developed based on interviews with patients with pulmonary arterial hypertension (PAH) following the process outlined in the FDA’s PRO guidance. Its psychometric characteristics are being evaluated using data from SYMPHONY - a phase IIIb PAH study with macitentan. The goal of the analyses was the finalization of item content and domain structure while ensuring the instrument is clinically and patient relevant. Methods: In SYMPHONY, patients completed generic and disease-specific questionnaires while clinicians provided assessment of severity and changes in symptomatology. Item performance, Rasch and Factor analyses of baseline data were used together with expert input to select the final items and define the domain structure. Results: Data from 278 patients (79% female, mean age 60 years) were included in the analysis. 40% and 59% of patients were in WHO functional class II and III, respectively, and 41% were receiving background therapy with a phosphodiesterase type-5 inhibitor. As expected for a moderately severe population, some items had high (> 50%) floor effects. Following removal of redundant or mis-fitting items, the final instrument has 11 symptom items (shortness of breath, fatigue, lack of energy, swelling in ankles/legs, swelling in stomach area, cough, heart palpitations, rapid heartbeat, chest pain, chest tightness, lightheadedness) across 2 domains (cardiopulmonary and cardiovascular symptoms), and 11 impact items (walk slowly/quickly on flat surface, walk uphill, carry things, light chores, need help, wash/dress self, think clearly, sad, worried, frustrated) across 2 domains (physical impacts and cognitive/emotional impacts). Compliance in completing the questionnaire was 83%. Conclusions: PAH-SYMPACT® is a clinically and PAH-patient relevant instrument. This PRO has the potential to become an important outcome measure for PAH in both clinical practice and clinical trials.
PRM172 A Study of the Successful Adaptation of the Quality of Life Questionnaire - Bronchiectasis From Paper To Electronic Format Simpson H1, Ong SH2, Quittner A3, McEvoy K4, Dhariwal M5 Hartford, CT, USA, 2Novartis Pharma AG, Basel, Switzerland, 3University of Miami, Miami, FL, USA, 4CRF Health, London, UK, 5Novartis Ireland Ltd, Dublin, Ireland
1Corporate Translations, Inc., East
Objectives: ISPOR ePRO Task-Force recommends that when an ePRO measure is adapted from a paper, evidence should be generated demonstrating that the measurement properties of the ePRO are comparable to the original paper PRO format (Coons et al, 2009). The objective of this testing was to migrate the paperbased source US English Quality of Life-Bronchiectasis (QOL-B) questionnaire v3.1 (Quittner et al. 2010) to an electronic platform. This study evaluated whether the electronic version was user-friendly and whether patient understanding was impacted. Cognitive debriefing and usability testing were performed using the ISPOR recommendations. Methods: Initially, ePRO screens using the proprietary software (CRF Health Inc.) were created and checked to ensure the wording had been migrated successfully. The tablet was then tested to ensure user-friendliness and functionality. Following this, the eQOL-B was tested with 10 bronchiectasis patients in the USA. Patients were asked questions about the usability of the device and whether they had any recommendations based on their experience. Five (5) of these patients participated in additional full cognitive debriefing interviews, ensuring that the migration process did not influence their understanding of the questions or rating scales. Results: Minor changes to the format of questions and instructions were made in the electronic version. Respondent understanding and device functionality were tested during cognitive debriefing and usability testing, with no issues were identified. All patients found the device straightforward and easy to use, even when it was their first time completing a PRO on an electronic device. Conclusions: The review of the draft eQOL-B ensured that the electronic version of the QOL-B was faithful to the source paper version. Intensive testing of the measure concluded that the electronic version was user-friendly, intuitive and maintained the integrity of the instrument. Thus, the electronic version of the QOL-B is considered ready for use in clinical trials or clinical practice.
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 3 4 7 – A 7 6 6
to other regions. 6 articles compared or developed further TTO techniques. Of the 65 comparisons between TTO and EQ-5D, 6 articles were excluded due to not measureable results. Results from the remaining 59 articles showed a broad spectrum of results. In general, TTO methods were seen as more sensitive or suitable than an EQ-5D questionnaire, while some others argue the EQ-5D might be easier in its application with the general population or patients than TTO. Conclusions: The analysis showed differences in the assessment of the two methods TTO and EQ-5D with a more sensitive and disease specific coverage of the TTO method. PRM168 Qualitative Research Approaches In Rare Diseases: Acid Sphingomyelinase Deficiency (ASMD) Symptoms and Impact as Reported by Patients and Caregivers Avetisyan R1, Hareendran A2, Stringer S3, Tan S1, Sanson B1, Hass S1 Genzyme, Cambridge, MA, USA, 2Evidera, London, UK, 3Evidera, Bethesda, MD, USA
1Sanofi
Objectives: Conducting large studies in rare diseases is challenging. However, much valuable information can be obtained using qualitative and mixed-method approaches, such as in the case of acid sphingomyelinase deficiency (ASMD). ASMD, also called Niemann-Pick Disease Types A and B, is a rare, genetic, life-threatening condition with an estimated incidence of 0.4-0.6 per 100,000. There are significant unmet needs associated with this condition, and currently disease management is limited to symptomatic/palliative care only. The aim of this research was to address the lack of evidence about ASMD and specifically patients’ experiences of the condition. Methods: A qualitative research study was conducted with 22 people with ASMD and/or their caregivers, to obtain information about the disease, its symptoms and impact. Participants were interviewed using open-ended questions and probes developed based on literature review and clinical expert interviews. Participants also completed symptom importance/bothersomeness ratings and questionnaires to characterize the sample. Results were also used to develop a conceptual disease model (CDM). Results: The mean (range) age was 37(18-63) years for adults and 10 (2-17) years for pediatric patients; 64% of adult and 45% of children were female. The most commonly reported symptoms were enlarged abdomen (due to hepatosplenomegaly), breathing difficulties, frequent respiratory infections, abdominal pain, bleeding/bruising, back and limb pain, and fatigue. Specific symptoms reported as being most bothersome included: fatigue, shortness of breath, bruising/bleeding, lower limb/joint pain, sleep disturbances, headaches, and diarrhea. ASMD was found to substantially impact physical, social, and emotional functioning in both adults and children. A CDM was developed and used to identify concepts most common and relevant to patients. Conclusions: This study describes experiences of patients with ASMD and its significant impact on patients’ lives. The results can be used to support the development of ASMD disease-specific measures for the evaluation of patient-focused health outcomes in the condition. PRM169 Fore! Sweeping Generalisations do not Bowl Over all Cultures… Cultural Adaptation in the Kidney Disease Quality of Life Questionnaire Richards A, Mead H, Towns L ICON Plc, Abingdon, UK
Objectives: The aim of this study is to assess how the moderate activities in the Kidney Disease Quality of Life Questionnaire (KDQoL), namely, ‘vacuuming, bowling and golf’ are adapted to suit various cultures. Methods: Linguistic Validation was carried out on a total 60 language pairs spanning 41 countries (covering 11 language families) and went through cognitive debriefing whereby adaptations were confirmed or discussed in more detail. We assessed if changes were made for each language pair and calculated what percentage of languages necessitated an adaptation based on the cultural norms. Based on this data, we then evaluated the activities that were considered more culturally appropriate for the target culture. Results: An overview of the language families that deemed an adaptation necessary, the types of activities that were substituted, and how this has enhanced the conceptual equivalency of the translation was created. The source activities were culturally adapted into a range of equivalent moderate activities judged relevant to the target population. Golf was a particularly troublesome term as many cultures considered this a niche sport and was therefore not fully understood as a moderate activity by the general public. Out of the 60 translations, changes were necessary in 60% of cases for the activities to relate to their culture. 5% used ‘gathering berries and mushrooms’ as an equivalent, 17% adapted golf as variants of walking such as ‘forest walking’, 5% changed ‘bowling’ to ‘Tai-chi’ and ‘vacuuming’ was changed to ‘sweeping’ for 11% of languages. Conclusions: To enable understanding and relatability to the moderate activities given in the questionnaire, it was essential to adapt the activity to one that would be familiar to the target culture. The range of alternative activities that replaced the original source terms demonstrates that in order to achieve conceptual equivalence, cultural adaptation is often vital to the translation process. PRM170 Systematic Review of Psychometric Properties of The EQ-5D Questionnaires In Patients with Hematologic Malignant Neoplasms Mlynczak K1, Wojcik A1, Golicki D2 1HealthQuest, Warsaw, Poland, 2Department of Experimental & Clinical Pharmacology, Medical University of Warsaw, Warsaw, Poland
Objectives: The aim of this study was to systematically review the published literature to assess the psychometric properties (validity, reliability and responsiveness) of the EQ-5D questionnaires (EQ-5D-3L, EQ-5D-5L, EQ-5D-Y) reported in patients with hematologic malignant neoplasms (ICD-10: C81 - C96 and D46). Methods: Nine databases were systematically searched (MEDLINE (PubMed), EMBASE, Cochrane Library, EuroQol website, CEA Registry, LILACS (VHL), ProQuest, CRD and Web of Science) up to February 2016. Additional references were obtained from reviewed papers. No study design restrictions were implemented. The search strategy
combined MESH, EMTREE and free-text terms respecting the population and the EQ-5D instruments. Data from studies meeting the inclusion criteria were extracted using a pre-determined extraction form, specified for the psychometric properties. The review data were grouped into three categories: validity, reliability and responsiveness. Results: Three hundred eighty eight abstracts were initially identified and screened and 101 records were screened in full text. Finally, 21 full text papers were included into the review of Hodgkin and non-Hodgkin lymphomas, leukemias, myelodysplastic syndrome. Psychometric properties, validity, reliability and responsiveness were described in 14 (n= 2884), 4 (n= 379) and 11 (n= 4416) papers, respectively. Known group analysis was the most common method for validity assessment (10 papers; n= 2007 participants). In terms of responsiveness effect size, SRM and ceiling and floor effects were presented in 3 (n= 773), 2 (n= 913) and 2 (n= 913) papers, respectively. The reliability of the EQ-5D questionnaire was measured by retesting, assessing inter-rater comparison and internal consistency in 2 (n= 182), 2 (n= 197), and 1 (n= 148) trials, respectively. Conclusions: The psychometric properties of EQ-5D questionnaire were described in a large number of clinical trials in patients with hematologic malignant neoplasms. However, differences in populations of studies make difficult the aggregation of results. PRM171 Content Validation of the Pulmonary Arterial Hypertension Symptoms and Impact (PAH-SYMPACT®) Questionnaire Gomberg-Maitland M1, Channick R2, Chin K3, Fischer A4, Frantz R5, Roberts L6, Miller C7, Hunsche E8, Zamanian R9, Badesch D4 1University of Chicago, Chicago, IL, USA, 2Massachusetts General Hospital, Boston, MA, USA, 3University of Texas Southwestern, Dallas, TX, USA, 4University of Colorado Denver, Aurora, CO, USA, 5Mayo Clinic Rochester, Rochester, MN, USA, 6Evidera, Bethesda, MD, USA, 7Piedmont Healthcare, Atlanta, GA, USA, 8Actelion Pharmaceuticals Ltd, Allschwil, Switzerland, 9Stanford University, Stanford, CA, USA
Objectives: PAH-SYMPACT® is a patient reported outcome (PRO) instrument; the draft consists of 16 symptom and 25 impact items. The PRO was developed based on interviews with patients with pulmonary arterial hypertension (PAH) following the process outlined in the FDA’s PRO guidance. Its psychometric characteristics are being evaluated using data from SYMPHONY - a phase IIIb PAH study with macitentan. The goal of the analyses was the finalization of item content and domain structure while ensuring the instrument is clinically and patient relevant. Methods: In SYMPHONY, patients completed generic and disease-specific questionnaires while clinicians provided assessment of severity and changes in symptomatology. Item performance, Rasch and Factor analyses of baseline data were used together with expert input to select the final items and define the domain structure. Results: Data from 278 patients (79% female, mean age 60 years) were included in the analysis. 40% and 59% of patients were in WHO functional class II and III, respectively, and 41% were receiving background therapy with a phosphodiesterase type-5 inhibitor. As expected for a moderately severe population, some items had high (> 50%) floor effects. Following removal of redundant or mis-fitting items, the final instrument has 11 symptom items (shortness of breath, fatigue, lack of energy, swelling in ankles/legs, swelling in stomach area, cough, heart palpitations, rapid heartbeat, chest pain, chest tightness, lightheadedness) across 2 domains (cardiopulmonary and cardiovascular symptoms), and 11 impact items (walk slowly/quickly on flat surface, walk uphill, carry things, light chores, need help, wash/dress self, think clearly, sad, worried, frustrated) across 2 domains (physical impacts and cognitive/emotional impacts). Compliance in completing the questionnaire was 83%. Conclusions: PAH-SYMPACT® is a clinically and PAH-patient relevant instrument. This PRO has the potential to become an important outcome measure for PAH in both clinical practice and clinical trials.
PRM172 A Study of the Successful Adaptation of the Quality of Life Questionnaire - Bronchiectasis From Paper To Electronic Format Simpson H1, Ong SH2, Quittner A3, McEvoy K4, Dhariwal M5 Hartford, CT, USA, 2Novartis Pharma AG, Basel, Switzerland, 3University of Miami, Miami, FL, USA, 4CRF Health, London, UK, 5Novartis Ireland Ltd, Dublin, Ireland
1Corporate Translations, Inc., East
Objectives: ISPOR ePRO Task-Force recommends that when an ePRO measure is adapted from a paper, evidence should be generated demonstrating that the measurement properties of the ePRO are comparable to the original paper PRO format (Coons et al, 2009). The objective of this testing was to migrate the paperbased source US English Quality of Life-Bronchiectasis (QOL-B) questionnaire v3.1 (Quittner et al. 2010) to an electronic platform. This study evaluated whether the electronic version was user-friendly and whether patient understanding was impacted. Cognitive debriefing and usability testing were performed using the ISPOR recommendations. Methods: Initially, ePRO screens using the proprietary software (CRF Health Inc.) were created and checked to ensure the wording had been migrated successfully. The tablet was then tested to ensure user-friendliness and functionality. Following this, the eQOL-B was tested with 10 bronchiectasis patients in the USA. Patients were asked questions about the usability of the device and whether they had any recommendations based on their experience. Five (5) of these patients participated in additional full cognitive debriefing interviews, ensuring that the migration process did not influence their understanding of the questions or rating scales. Results: Minor changes to the format of questions and instructions were made in the electronic version. Respondent understanding and device functionality were tested during cognitive debriefing and usability testing, with no issues were identified. All patients found the device straightforward and easy to use, even when it was their first time completing a PRO on an electronic device. Conclusions: The review of the draft eQOL-B ensured that the electronic version of the QOL-B was faithful to the source paper version. Intensive testing of the measure concluded that the electronic version was user-friendly, intuitive and maintained the integrity of the instrument. Thus, the electronic version of the QOL-B is considered ready for use in clinical trials or clinical practice.