MARIA FERNANDA ABALEM DE SA CARRICONDO
Projetos de Pesquisa
Unidades Organizacionais
Instituto Central, Hospital das Clínicas, Faculdade de Medicina - Médico
LIM/33 - Laboratório de Oftalmologia, Hospital das Clínicas, Faculdade de Medicina
LIM/33 - Laboratório de Oftalmologia, Hospital das Clínicas, Faculdade de Medicina
6 resultados
Resultados de Busca
Agora exibindo 1 - 6 de 6
- The Michigan Vision-Related Anxiety Questionnaire: A Psychosocial Outcomes Measure for Inherited Retinal Degenerations(2021) LACY, Gabrielle D.; ABALEM, Maria Fernanda; ANDREWS, Chris A.; ABUZAITOUN, Rebhi; POPOVA, Lilia T.; SANTOS, Erin P.; YU, Gina; RAKINE, Hanan Y.; BAIG, Natasha; EHRLICH, Joshua R.; FAHIM, Abigail T.; BRANHAM, Kari H.; SWENOR, Bonnielin K.; LICHTER, Paul R.; DAGNELIE, Gislin; STELMACK, Joan A.; MUSCH, David C.; JAYASUNDERA, K. Thiranpatient-reported outcome measure for screening and monitoring vision-related anxiety in patients with inherited retinal degenerations. center dot DESIGN: Item-response theory and graded response modeling to quantitatively validate questionnaire items generated from qualitative interviews and patient feedback. center dot METHODS: Patients at the Kellogg Eye Center (University of Michigan, Ann Arbor, Michigan, USA) with a clinical diagnosis of an inherited retinal degeneration (n = 128) participated in an interviewer-administered questionnaire. The questionnaire consisted of 166 items, 26 of which pertained to concepts of ""worry""and ""anxiety.""The subset of vision-related anxiety questions was analyzed by a graded response model using the Cai Metropolis-Hastings Robbins-Monro algorithm in the R software mirt package. Item reduction was performed based on item fit, item information, and item discriminability. To assess test-retest variability, 25 participants completed the questionnaire a second time 4 to 16 days later. center dot RESULTS: The final questionnaire consisted of 14 items divided into 2 unidimensional domains: rod function anxiety and cone function anxiety. The questionnaire exhibited convergent validity with the Patient Health Questionnaire for symptoms of depression and anxiety. This vision-related anxiety questionnaire has high marginal reliability (0.81 for rod-function anxiety, 0.83 for cone-function anxiety) and exhibits minimal test-retest
- Positive feedback loop between vision-related anxiety and self-reported visual difficulty(2023) POPOVA, Lilia T.; ABUZAITOUN, Rebhi O.; FRESCO, David M.; ABALEM, Maria Fernanda; ANDREWS, Chris A.; MUSCH, David C.; EHRLICH, Joshua R.; JAYASUNDERA, K. ThiranBackgroundPatients with Inherited Retinal Diseases typically experience progressive, irreversible vision loss resulting in low vision and blindness. As a result, these patients are at high risk for vision-related disability and psychological distress, including depression and anxiety. Historically, the relationship between self-reported visual difficulty (encompassing metrics of vision-related disability and quality of life, among others) and vision-related anxiety has been regarded as an association and not a causal relationship. As a result, there are limited interventions available that address vision-related anxiety and the psychological and behavioral components of self-reported visual difficulty.Materials and MethodsWe applied the Bradford Hill criteria to evaluate the case for a bidirectional causal relationship between vision-related anxiety and self-reported visual difficulty.ResultsThere is sufficient evidence to satisfy all nine of the Bradford Hill criteria of causality (strength of association, consistency, biological gradient, temporality, experimental evidence, analogy, specificity, plausibility, and coherence) for the relationship between vision-related anxiety and self-reported visual difficulty.ConclusionsThe evidence suggests that there is a direct positive feedback loop-a bidirectional causal relationship-between vision-related anxiety and self-reported visual difficulty. More longitudinal research on the relationship between objectively-measured vision impairment, self-reported visual difficulty, and vision-related psychological distress is needed. Additionally, more investigation of potential interventions for vision-related anxiety and visual difficulty is needed.
- Content generation for patient-reported outcome measures for retinal degeneration therapeutic trials(2020) LACY, Gabrielle D.; ABALEM, Maria Fernanda; POPOVA, Lilia T.; SANTOS, Erin P.; YU, Gina; RAKINE, Hanan Y.; ROSENTHAL, Julie M.; EHRLICH, Joshua R.; MUSCH, David C.; JAYASUNDERA, K. ThiranPurpose Generate content for a patient-reported outcome (PRO) measure for use in future clinical trials for inherited retinal degenerations. Methods Patients at the University of Michigan Kellogg Eye Center with a clinical diagnosis of inherited retinal degeneration with varying phenotypes were recruited for interviews. First, in-depth interviews were performed to solicit a wide range of patient experiences pertaining to visual function. Coders qualitatively analyzed the transcripts from these interviews using Atlas.ti software (Version 8.1.3 (522)) to draft questionnaire items. Next, the questionnaire was tested and refined based on participant feedback in cognitive interviews and administrator feedback in the pilot survey administration (pilot interviews). Results A total of 55 participants with a clinical diagnosis of inherited retinal degeneration were interviewed throughout the three study phases: in-depth interviews (n = 26), cognitive interviews (n = 16), and pilot interviews (n = 13). Coded items were analyzed for frequency of occurrence and related themes, then organized into common domains. Within each domain, PRO items were drafted to address the functional limitations or adaptations experienced by patients. Conclusions Items for a PRO measure have been drafted and evaluated for interpretability in the target inherited retinal degeneration patient population. Content validity for the items was established through a process of in-depth interviews, cognitive interviews, and pilot interviews.
- The validation of inherited retinal disease-specific patient-reported outcome measures in adolescent patients(2023) SELVAN, Kavin; ABUZAITOUN, Rebhi; ABALEM, Maria Fernanda; VINCENT, Ajoy; ANDREWS, Chris A. A.; LACY, Gabrielle D. D.; FARJO, Rafid; KAO, Karissa; KAO, Krystal; DAGNELIE, Gislin; MUSCH, David C. C.; JAYASUNDERA, K. Thiran; HEON, ElisePurpose: To determine the validity of the validate the adult patient-reported outcome measure tools, the Michigan Retinal Degeneration Questionnaire (MRDQ) and Michigan Vision-Related Anxiety Questionnaire (MVAQ), in adolescent patients with inherited retinal diseases (IRDs).Methods: Ninety-one adolescent patients diagnosed with IRDs were recruited at the Hospital for Sick Children (University of Toronto) and the Kellogg Eye Center (University of Michigan). The patients were administered the MRDQ, MVAQ, and Patient Health Questionnaire-4 (PHQ-4). Test-retest variability was assessed in eighteen patients within 14 days of the initial administration. Adolescent responses were analyzed for validity and reliability. As a further validation step, comparisons were made to adult data from the original MRDQ and MVAQ studies to ensure consistency in response ranges.Results: The existing MRDQ and MVAQ content and format could accurately detect the impact of IRD on activities of daily living in adolescents with IRDs. No floor/ceiling effects were identified, test-retest reliability was established (r = 0.73-0.86), and no items were excluded after differential item functioning analysis. Domain and trait associations with visual acuity and IRD phenotypes were similar between adolescents and adults.Conclusions: The MRDQ and MVAQ are psychometrically validated questionnaires for which we have shown validity for use in adolescent patients with IRDs.
- Challenges of Cost-Effectiveness Analyses of Novel Therapeutics for Inherited Retinal Diseases(2022) JAYASUNDERA, K. Thiran; ABUZAITOUN, Rebhi O.; LACY, Gabrielle D.; ABALEM, Maria Fernanda; SALTZMAN, Gregory M.; CIULLA, Thomas A.; JOHNSON, Mark W.center dot PURPOSE: To investigate the challenges and potential improvement strategies of cost-effectiveness analyses performed for therapeutics targeting inherited retinal diseases (IRDs). center dot DESIGN: Perspective. center dot METHODS: A literature review was conducted with discussion of current limitations and improvement recommendations. center dot RESULTS: Cost-effectiveness analysis (CEA) performed for IRD therapeutics has multiple limitations. First, the available methods used to measure health-related quality of life and health utilities can be inaccurate when used in IRDs. Second, the financial burden to patients and society from vision impairment associated with IRDs has been inadequately studied and includes a variety of expenditures ranging from direct costs of IRD specialty health care to indirect expenses associated with daily living activities. Third, our collective understanding is limited in the areas of IRD natural history and health benefits gained from new IRD treatments (eg, gene therapies). In addition, the therapeutic effect from a patient perspective and its duration of action are not fully understood. Due to the scarcity of data, CEA for newly approved therapies has relied on assumptions and creations of predictive models for both costs and health benefits for these new therapeutics in order to calculate the incremental cost-effectiveness ratio. center dot CONCLUSIONS: CEA studies performed for IRD therapeutics have been limited by the established health utilities in ophthalmology and the lack of disease-specific information. The assumptions and extrapolations in these studies create substantial uncertainty in incremental costeffectiveness ratio results. An improved framework is required for CEA of IRD therapeutics in order to determine the cost-effectiveness of each therapy brought from clinical trials to clinical practice. (Am J Ophthalmol 2022;235: 90-97.
- The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease(2022) SELVAN, Kavin; ABALEM, Maria F.; LACY, Gabrielle D.; VINCENT, Ajoy; HEON, ElisePatient-reported outcome measures (PROMs) are questionnaires that assess health outcomes meaningful to the patient. PROMs have multiple applications, such as supporting clinicians' decision-making for patient care, understanding the impact of disease on patient functioning, and evaluating the efficacy of therapeutics. Though PROMs were developed for various eye conditions, no PROM was tailored to pediatric patients with inherited retinal disease (IRD). Hence, a literature search was conducted using MEDLINE and Embase to identify PROMs potentially relevant to this patient population. This review evaluated selected pediatric PROMs against the US Food and Drug Administration (FDA) guidelines and found restricted use in the context of IRD. As there is a need for PROMs tailored to pediatric patients with IRD, we provide a perspective on applying the International Society for Pharmacoeconomics and Outcomes Research and FDA standards on the development of PROMs specific to IRD. Plain Language Summary Inherited retinal diseases refer to a group of genetic conditions that affect the eye's light-sensing cells and lead to vision loss. When a patient undergoes an eye assessment, the measures used are technical (e.g., visual acuity, visual field) and do not routinely address the patient's experience. It is increasingly evident that the technical tools used do not really reflect how patients' vision affects their daily lives. Questionnaires designed to assess how a condition impacts a daily activity are referred to as patient-reported outcome measures. The perspective of the impact of a condition on daily activities differs between adults and children. These tools are being created to evaluate health outcomes important to the patient on the basis of their condition and age. This is especially important when determining the value of therapies from the patient perspective. To date, no such questionnaire has been designed for pediatric patients with inherited retinal disease, an important cause of blindness. We explored the literature to evaluate existing pediatric vision tools and found that those could not be used to fill this gap. Given that we found a need to develop questionnaires tailored to pediatric patients with IRD, we also provide insight into how such a tool can be created for this population.