Natural history of familial cerebral cavernous malformations: the CCM_Italia cohort study.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Lanfranconi Silvia, Scola Elisa, Novelli Deborah, Poggesi Anna, Pescini Francesca, Pavanello Marco, Romano Ferruccio, D'Alessandris Quintino Giorgio, Marani Walter, Signorelli Francesco, Iaconetta Giorgio, Torelli Giovanni, Fainardi Enrico, Severino Mariasavina, Remore Luigi Gianmaria, Bertani Giulio Andrea, Conte Giorgio, Capra Valeria, Vasamì Antonella, Nicolis Enrico, Contino Giorgia, Ronchi Dario, Palmieri Maria Chiara, Previtali Alessandra, Mattogno Pier Paolo, Sturiale Carmelo Lucio, Solarino Maria Elena, Caliulo Rita, Bozzi Maria Teresa, Fratini Filippo, Zanier Elisa R, Latini Roberto, Meessen Jennifer Marie Theresia Anna, Locatelli Marco, and the CCM_Italia investigators
Аннотация
METHODS: Pediatric and adult fCCM patients, whether symptomatic or asymptomatic, will be enrolled and followed annually over a 2-year period. Participants will undergo clinical assessments, blood sampling, and 3 T brain MRI scans at baseline, 12 months, and 24 months. The primary outcome is the new occurrence of symptomatic ICH or FNDs attributable to CCMs over 24 months. Patient characteristics will be assessed for the primary and secondary endpoints and illustrated using Kaplan-Meier curves and Cox proportional hazard regressions. This trial is registered with ClinicalTrials.gov, NCT06983132 and is currently recruiting participants.
DISCUSSION/CONCLUSION: Despite increasing efforts in basic and clinical research and an improved understanding of the pathogenic mechanisms underlying fCCM, tools to predict disease progression, identify at-risk individuals, and pinpoint effective therapeutic targets are still lacking. This study aims to create the largest Italian cohort of fCCM patients, who will be monitored closely over time to collect data that may help identify risk factors and disease trajectories. The collection of standardized information on clinical and radiological evolution, along with results from circulating biomarkers, will help address the complexities of the disease and may suggest potential reliable markers of disease progression.
CLINICAL TRIAL REGISTRATION: ClinicalTrials.gov, identifier NCT06983132.