Fibrolamellar carcinoma registry: Opportunities and challenges.
Authors: Layal Al Mahmasani, Marinela Capanu, Kurt Losert, Maeve Aine Lowery, Martina Hennessy, Mathew Ray, Suzanne Chambers, Amin Yaqubie, Andrew Zarski, Imane H. El Dika, Joseph S. Light, Michael P. La Quaglia, Eileen M. O'Reilly, Ghassan K. Abou-Alfa
Journal: Journal of Clinical Oncology (2024)

e16384 Background: Fibrolamellar carcinoma (FLC) is a rare form of primary liver cancer which affects young adults and adolescents without underlying liver disease or cirrhosis. The tumors are typically well circumscribed masses characterized pathologically by well differentiated polygonal hepatic cells with eosinophilic and granular cytoplasm surrounded by fibrous bands. DNAJB1 and PRKACA fusion is perceived as a dominant recurrent genetic alteration in FLC. FLC affects patients worldwide and a continued lack of an international database remains a knowledge gap. Further, there remains a poor understanding of FLC etiology, and major unmet need for more effective therapy. Methods: A patient-centered multi time-points, dynamic registry was developed by Memorial Sloan Kettering (MSK) in collaboration with Google and Maven Wave. The effort was supported by the Fibrolamellar Cancer Foundation (FCF). The study was opened worldwide except for the European Union pending the acquisition of the requiredGeneral Data Protection Regulation (GDPR) of data control and processing. The latter were assigned to Trinity College Dublin (TCD); a partner in this study. Participants were required to have a Google account to access the study. Demographic, clinical and response data were collected at baseline and at selected time points (monthly for up to 18 months). The aim of the registry was to provide a longitudinal understanding of the biography and natural history of the disease and outcomes. Results: Between May 2021 and December 2023, the study was broadcasted publicly worldwide through different venues including MSK, FCF, websites and Google engine. Only 14 patients responded. Table 1 summarizes the study sequence and patients’ enrollment. On average, the participants completed 41% of the monthly surveys. Four responders out of 5 patients were female, with a median age of 32 years (range 21-53). Conclusions: The registry provided a unique opportunity to access a broad global community of patients with FLC. Poor participation was key main limiting factor. Barriers include: (1) the presence of competing registries or studies for FLC, (2) technical limitations such as the exclusivity to access the registry to Google account. Follow-up emails may have been received in spam or junk folders, (3) complexities posed by the GDPR in Europe, and (4) patient-related factors including preoccupation with treatments and symptom management of their illness add to the disinterest in or inability to fill the monthly surveys. Unifying efforts and collaborations among caring physicians and scientists, medical centers, foundations, and patient advocacy groups across the world can help overcome the current challenges and accelerate the buildup of resources for research and practice improvement for the rare FLC. [Table: see text]