Cultural and Linguistic Adaptation for the Romanian Language Version of the Cardiff Acne Disability Index: A Pilot Study on the Web-Based Experience of Cognitive Debriefing

Background The Cardiff Acne Disability Index (CADI) is a validated measurement instrument for quality of life evaluation in young patients with acne. The original version was designed in English, and it has been translated to other languages. An adaptation for the Romanian language was lacking. Objective The main objective of this study was to evaluate the comprehensibility of the Romanian language adaptation of the CADI in a small sample of patients with acne. Methods Guided by the team at Cardiff University, we conducted the stages of the standardized translation process—forward translation, the reconciliation of translated versions, back translation, and cognitive debriefing. The cognitive debriefing stage involved applying the CADI to a small sample of patients. This was followed by individual interviews in which each question was discussed. Ethical approval was obtained for the cognitive debriefing stage. We administered this measure as a web-based form. The completion times for each individual question and for the entire survey were automatically recorded. The interviews for assessing comprehensibility and suitability for the Romanian language and culture were also held as live, web-based meetings. Results A total of 7 patients with acne—4 females and 3 males—aged between 19 and 34 years were included. All subjects were native speakers of the Romanian language. They had mild or moderate acne. The mean completion time for the survey was 3.28 minutes. The mean score for the CADI was 5.4286. All participants agreed that the language used in this quality of life measurement instrument was simple, clear, and adequate for their native language. Conclusions Despite the epidemiologic restrictions against COVID-19, through teledermatology, we achieved cultural adaptation for the CADI in a language that previously lacked a specific tool for assessing quality of life impairment in patients with acne. Conflicts of Interest CMS receives royalties from Springer Nature, consulting fees from Vichy International, and support for attending meetings from Leo Pharma. SC has no conflicts of interest.

Cultural and Linguistic Adaptation for the Romanian Language Version of the Cardiff Acne Disability Index: A Pilot Study on the Web-Based Experience of Cognitive Debriefing (Preprint)

BACKGROUND The Cardiff Acne Disability Index (CADI) is a validated measurement instrument for quality of life evaluation in young patients with acne. The original version was designed in English, and it has been translated to other languages. An adaptation for the Romanian language was lacking. OBJECTIVE The main objective of this study was to evaluate the comprehensibility of the Romanian language adaptation of the CADI in a small sample of patients with acne. METHODS Guided by the team at Cardiff University, we conducted the stages of the standardized translation process—forward translation, the reconciliation of translated versions, back translation, and cognitive debriefing. The cognitive debriefing stage involved applying the CADI to a small sample of patients. This was followed by individual interviews in which each question was discussed. Ethical approval was obtained for the cognitive debriefing stage. We administered this measure as a web-based form. The completion times for each individual question and for the entire survey were automatically recorded. The interviews for assessing comprehensibility and suitability for the Romanian language and culture were also held as live, web-based meetings. RESULTS A total of 7 patients with acne—4 females and 3 males—aged between 19 and 34 years were included. All subjects were native speakers of the Romanian language. They had mild or moderate acne. The mean completion time for the survey was 3.28 minutes. The mean score for the CADI was 5.4286. All participants agreed that the language used in this quality of life measurement instrument was simple, clear, and adequate for their native language. CONCLUSIONS Despite the epidemiologic restrictions against COVID-19, through teledermatology, we achieved cultural adaptation for the CADI in a language that previously lacked a specific tool for assessing quality of life impairment in patients with acne.

Development, psychometric evaluation and cognitive debriefing of the rheumatoid arthritis symptom and impact questionnaire (RASIQ)

Background Rheumatoid arthritis (RA) is a chronic inflammatory disease often associated with persistent pain. There is a need for a patient-reported outcome measure (PROM) that is rooted in the patient experience and psychometrically validated. We describe the development of the Rheumatoid Arthritis Symptom and Impact Questionnaire (RASIQ), a novel PROM with potential to record key symptoms and impacts of RA with a 24-h recall period. Results A literature review identified RA concepts that patients considered most important to their disease experience, including pain, fatigue, joint swelling and stiffness. From this, an initial item pool (33 items; 27 related to symptoms, 6 related to impacts) was developed with a recall period of 24 h. Two rheumatologists evaluated each item’s relevance, and the second version of the RASIQ was refined (29 items; 21 related to symptoms, 8 related to impacts). Next, three rounds of cognitive debriefing interviews were conducted with patients with RA (n = 15 overall). The RASIQ was revised to remove items deemed irrelevant or redundant, leaving 16 items measuring symptoms (joint pain, energy/tiredness, joint stiffness) and impacts (rest, sleep). A parallel series of semi-structured concept elicitation interviews (n = 30) facilitated the design of a conceptual model of RA symptoms, impacts and treatment experiences. Post-hoc comparison of the model with RASIQ revealed that all items selected were among the most important and relevant symptoms and impacts for patients. A final round of cognitive debriefing interviews (n = 12) confirmed that the final 16-item RASIQ was relevant and easy to understand, with no further changes recommended. Psychometric evaluation using data from two Phase II RA clinical trials confirmed a 3-factor structure, as well as the reliability and validity of the scale scores, and the ability of RASIQ to detect changes in symptoms and impacts when administered at specific study timepoints, using a 24-h recall period. Conclusions RASIQ is a novel, 16-item PROM developed with substantial patient input. Results from concept elicitation, cognitive debriefing, and psychometric evaluation confirmed the validity of the instrument, which has the potential to measure symptoms and impacts through a 24-h recall period and complement existing disease activity instruments with longer recall periods.

Linguistic Validation of Indonesian Version of the Audit of Diabetes-Dependent Quality of Life Questionnaire

Background: One of the impacts experienced by diabetes mellitus patients is a decrease in their quality of life. The Audit of Diabetes-Dependent Quality of Life (ADDQoL) is a widely used individualized diabetes-specific quality of life measure. However, there was no version available in the Indonesian language. Objective: This study is aimed to undertake linguistic validation, including a cultural adaptation of the ADDQoL questionnaire into the Indonesian language. Method: The original developer granted permission to use and modify the questionnaire. The international linguistic validation procedure developed by the Mapi Research Institute was used. There were six steps involved: forward translation, reconciliation, back translation, expert panel review by a psychologist and clinician, cognitive debriefing with diabetes patients, and proofreading. Result: Problems that arose during the linguistic validation process were resolved by finding conceptually equivalent alternatives and changing sentence structures to achieve equivalence in language, concept, and culture with the original version of the ADDQoL. The developer's team reviewed and discussed all actions taken. Cognitive debriefing interviews with five respondents showed that the ADDQoL questionnaire was simple to understand. Conclusion: The Indonesian version of the ADDQoL is linguistically and culturally validated. Further studies are needed to confirm the structure and reliability of the Indonesian ADDQoL.

Development and content validation of a symptom assessment for eosinophilic gastritis and eosinophilic gastroenteritis in adults and adolescents

Background A patient reported outcome (PRO) instrument with evidence of validity and reliability for assessing symptoms of eosinophilic gastritis (EG) and eosinophilic gastroenteritis (EGE) is needed to measure treatment benefit in clinical trials. The aim of this research is to develop an EG/EGE symptom PRO instrument for patients aged 12 and above. Methods The Symptom Assessment for Gastrointestinal Eosinophilic Diseases (SAGED) was developed through a literature review, discussions with expert clinicians, and concept elicitation and cognitive debriefing interviews with patients. Patients (n = 28) were recruited based on confirmed diagnosis and self-reported symptoms. The final instrument was translated and linguistically validated with additional cognitive debriefing interviews (n = 105). Results SAGED is a 24-h recall questionnaire consisting of eight items evaluating the core symptoms of EG and EGE (abdominal pain, nausea, bloating, early satiety, loss of appetite, vomiting, and diarrhea). Seven of the eight items are evaluated on an 11-point numerical rating scale ranging from ‘none’ to ‘worst imaginable’. Cognitive debriefing interviews showed that adults and adolescents understand the content and are able to select a response that reflects their experience. The linguistic validation process produced 21 translations that are understandable to patients and conceptually equivalent to the source version. Conclusions SAGED is suitable for measuring symptom improvement in adult and adolescent patients with EG and/or EGE. The content validity of SAGED has been established through best practices in qualitative research for PRO instrument development. The psychometric properties of SAGED will be evaluated in a future study.

Symptoms, impacts, and suitability of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT™) questionnaire in patients with sarcoidosis-associated pulmonary hypertension (SAPH): a qualitative interview study

Background Sarcoidosis-associated pulmonary hypertension (SAPH) is a prevalent and serious complication of sarcoidosis. No SAPH-specific self-report instruments for assessing SAPH symptoms and their impact on patients are available to date. This study sought to determine whether the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT™) questionnaire is suitable for use in patients with SAPH. Methods Patients diagnosed with SAPH participated in qualitative one-on-one telephone interviews to better understand SAPH symptoms and their impacts on patients’ lives and to determine the appropriateness of the PAH-SYMPACT™ for use in patients with SAPH. The interviews comprised concept elicitation, completion of the PAH-SYMPACT™, and cognitive debriefing. Interview transcripts were analyzed by content analysis. Results Eleven patients with SAPH were interviewed between August 2019 and June 2020. In the concept elicitation, all 11 participants endorsed shortness of breath and nine participants (82%) rated it as their “most bothersome or severe” symptom. Impacts endorsed by all 11 participants were difficulty walking uphill or up stairs and difficulty in performing daily activities. Cognitive debriefing indicated that the PAH-SYMPACT™ items were relevant and understandable to most participants and reflected their experiences of SAPH. Participants indicated that no key symptoms or impacts of SAPH were missing. They also reported that the PAH-SYMPACT™ instructions and response options were clear, and that it would be feasible to complete the 11 symptom items and one oxygen use item as part of their daily schedule. Conclusions This study suggests the PAH-SYMPACT™ is suitable for assessing symptoms and their impact in patients with SAPH. However, larger longitudinal studies are needed to confirm that it is fit for use in this patient population and that it can be used to reliably detect temporal changes in patients’ symptom status. Trial registration Not applicable.

Development of a Cold Agglutinin Disease-Specific Patient-Reported Outcome Symptom Measure

Introduction Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia in which red blood cells are bound by cold agglutinin autoantibodies. In addition to cold-induced symptoms, patients with CAD experience symptoms of chronic anemia and hemolysis including fatigue, weakness, and shortness of breath (Swiecicki PL et al. Blood 2013;122(7):1114-21). The objectives of this study were to identify disease-related symptoms and impacts most important to patients with CAD, and to evaluate whether existing patient-reported outcome (PRO) measures are appropriate to assess relevant CAD concepts. Methods As part of a larger study, qualitative concept elicitation (CE) interviews were conducted to better understand the disease burden of CAD from the patient perspective. The results from the first set of interviews were reported by Su J et al. Blood 2020;136(1):29-30. Based on the identified symptoms and impacts, standard survey methodological principles were used to develop a draft item pool to address concepts of interest. Items were refined and evaluated during the second set of interviews, which included 3 rounds of 60-minute phone interviews with patients with CAD using a semi-structured interview guide that included a CE section and cognitive debriefing. Pooled results from both sets of interviews were used to develop a conceptual model of the symptoms and impacts of CAD. Physician advisors were also invited to review the concepts identified in the interviews and to select those that they believed were most relevant as part of an advisory board. A PubMed review of literature published between 2010-2020 was also conducted to identify existing PRO measures used in the assessment of symptoms, impacts, and/or quality of life and patient experiences in adults with CAD or similar disease areas. Identified PRO measures were assessed to determine the extent to which they captured CAD-specific concepts identified in the CE interviews. Results In total, 37 participants diagnosed with primary or secondary CAD took part in the interviews (Set 1, n=16; Set 2, n=21). Overall, the mean age of participants was 67.2 years (range: 35-87), and the majority were female (73%). The most frequently reported symptom of CAD was reactions to cold environments (n=36; 97%), e.g., cold or numb feet/hands, and skin discoloration. Other reported symptoms included fatigue (n=35; 95%), shortness of breath (n=28; 76%), and trouble with thinking/concentration (n=21; 57%). The most frequently reported unfavorable impact of CAD was on day-to-day activities (n=32; 87%). Other negative impacts included effects on enjoyable activities (n=29; 78%) e.g., gardening, physical health/activities (n=28; 76%), social/leisure life (n=26; 70%); and mood/emotions (n=24; 65%). Most participants (n=30; 81%) reported they had made lifestyle/behavioral changes to help limit their CAD symptoms, such as wearing extra clothing in places that were likely to be cold. A conceptual model of the symptoms and impacts of CAD was developed based on these data (Figure 1). Physician advisors (n=7) agreed that the patient-reported symptoms and impacts identified during the interviews were relevant concepts to CAD. Following a literature review, no existing PRO measure was found to adequately address concepts deemed critical to CAD from the patient perspective. Owing to the lack of an existing fit-for-purpose measure, 14 concepts were identified from the interviews to develop an initial draft item pool. During the cognitive debriefing in the second set of interviews, participants (n=21: Round 1 n=8; Round 2 n=7; Round 3 n=6) evaluated these concepts. After 3 rounds of interviews, the new item set yielded 11 items relating to: fatigue; cold sensitivity; dyspnea; wearing extra clothing; limited physical, social, and enjoyable activities; difficulty with usual activities; mood; frustration; and anxiety/stress. Participants reported these items were comprehensive of their experiences with CAD, easy to understand, and would be relevant to gaining a better understanding of individuals' experiences with CAD. Conclusion These results support the need for a novel PRO measure(s) that adequately addresses concepts critical to the measurement of CAD symptoms and impacts from the patient perspective. Figure 1 Figure 1. Disclosures Joly: Sanofi: Current Employment. Dasmahapatra: Sanofi: Current Employment, Current equity holder in publicly-traded company. Su: Sanofi: Current equity holder in publicly-traded company, Ended employment in the past 24 months; Astellas US LLC: Current Employment. DiBenedetti: RTI Health Solutions: Current Employment, Other: Employee of RTI Health Solutions, funded by Sanofi Genzyme to conduct the original work which this abstract is based on. Kosa: RTI Health Solutions: Current Employment. Hill: Novartis: Consultancy, Honoraria; Grifols: Consultancy, Honoraria; Sanofi: Consultancy; ReAlta: Consultancy; Alexion: Honoraria; Amgen: Honoraria; Argenx: Consultancy; Apellis: Consultancy, Honoraria.

Advanced translation and cultural adaption of the LYMPH-Q Upper Extremity Module from English to Danish

Background To better quantify the impact that breast cancer-related arm lymphedema (BCRL) has on health-related quality of life (HR-QOL), a disease-specific patient-reported outcome measure (PROM) is needed. The LYMPH-Q Upper Extremity Module was recently developed for patients with BCRL. The aim of this study was to perform an advanced translation and culturally adapt the LYMPH-Q Upper Extremity Module for use in Denmark. Methods The LYMPH-Q Upper Extremity Module was translated into Danish according to the guidelines of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the World Health Organization (WHO). The process included two forward and one back translation, an expert panel meeting, and cognitive debriefing interviews with patients. The focus of the translation was to develop a Danish version that used appropriate patient-friendly language while maintaining the meaning of the items, instructions and response options. Results The two forward translations resulted in minor differences in terminology. These discrepancies were discussed among the translators and a harmonized Danish version 1 was achieved. Comparison of the back translation to the original English version identified 14 items/instructions/response options that required re-translation. Subsequently, experts helped to identify and resolve the language for 10 items/instructions/response options that did not maintain the same meaning as the English version. Participants in the cognitive debriefing interviews did not report any difficulties with understanding the items/instructions/response options. Conclusions The translation and cultural adaption process led to the development of a conceptually equivalent Danish version of the LYMPH-Q Upper Extremity Module. Level of Evidence: Not gradable

Development of symptom-focused outcome measures for advanced and indolent systemic mastocytosis: the AdvSM-SAF and ISM-SAF©

Background Advanced systemic mastocytosis (AdvSM), indolent systemic mastocytosis (ISM), and smoldering systemic mastocytosis (SSM) are rare diseases characterized by neoplastic mast cell infiltration of more than one organ. A content-valid patient-reported outcome (PRO) questionnaire that assesses relevant signs and symptoms that are important and understandable to individuals with a condition is critical for assessing new treatment benefit as well as supporting product labeling claims. Notably, no such PRO questionnaire has been developed in accordance with regulatory and scientific guidelines for use in AdvSM, ISM, and SSM patient populations. To fill that gap, this study documents the development and content validity of instruments evaluating signs and symptoms of systemic mastocytosis. Methods A review of peer-reviewed literature, advice meetings with clinical therapeutic area experts, patient concept elicitation interviews, concept selection and questionnaire construction meetings, and patient cognitive debriefing interviews were conducted, and regulatory feedback was incorporated. Results For AdvSM, 26 sign- and symptom-level concepts were identified in literature, 39 by clinicians, and 33 by patients. For ISM/SSM, 38 sign- and symptom-level concepts were identified in the literature, 39 by clinicians, and 57 by patients. Two patient-reported instruments, the Advanced Systemic Mastocytosis Symptom Assessment Form (AdvSM-SAF) and Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF)(©Blueprint Medicines Corporation), were developed based on consolidated findings. Cognitive debriefing interviews with AdvSM and ISM patients showed the AdvSM-SAF and ISM-SAF were understood and interpreted as intended by the majority of patients. Conclusion The AdvSM-SAF and ISM-SAF are content-valid tools measuring symptoms from AdvSM and ISM patients’ perspective.

Italian Version of the YQOL-DHH Questionnaire: Translation and Cross-cultural Adaptation

Objective To translate and cross-culturally adapt into Italian the YQOL-DHH (Youth Quality of Life Instrument–Deaf and Hard of Hearing Module), an instrument to evaluate the health-related quality of life in young deaf people. It could be useful for professionals, teachers, and parents to take care of deaf adolescents’ needs. Study Design Forward-backward translation, cross-cultural adaptation, and cognitive debriefing. Setting The cognitive debriefing was performed online with professionals and during clinical practice with deaf adolescents. Methods A methodological study was conducted according to the guidelines provided by the development team. The study consisted of a forward-backward translation and a cross-cultural adaptation. After the original authors’ confirmation, a cognitive debriefing was conducted with 30 professionals who work with deaf young people and with 10 deaf adolescents aged 11 to 18 years. Results For the linguistic translation and cross-cultural adaptation, some variations to the original instrument were made to obtain equivalence, such as the expression “deaf or hard of hearing” translated only with the Italian word “sordo.” During the cognitive debriefing, the clarity and comprehensibility of the items were reported by professionals and deaf adolescents. Eventually, the authors approved the final version. Conclusion The YQOL-DHH was translated and culturally adapted into Italian. The translated items were pertinent to the Italian culture and equivalent to the original ones. A validation study is suggested to make the instrument feasible for use in different clinical or educational contexts. In addition, to guarantee accessibility and autonomy for young deaf signers, Italian Sign Language translation of the questionnaire is suggested.