Patient journey in cystinosis: focus on non-adherence and disease management

Gema Ariceta, Simón Lalanza, Catalina Peña, Marta Martínez Montero, Carlos Bezos Daleske, Laura Acuña Álvarez, Elisa Giner

Article Type

Original Research

Published

This study assesses the impact of cystinosis and treatmentassociated challenges on therapeutic adherence to detect potential leverages of change and propose lines of actions based on patient-reported experience measures.

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Adherence to risk evaluation and mitigation strategies (REMS) requirements for monthly testing of liver function

Christopher M Blanchette, Anthony P Nunes, Nancy D Lin, Kathleen M Mortimer, Joshua Noone, Krishna Tangirala, Stephen Johnston, Benjamin Gutierrez

Article Type

Original Research

Published

Risk evaluation and mitigation strategies (REMS), as mandated by the FDA for medications with the potential for harm, are increasingly incorporating rigid protocols for patient evaluation, but little is known about compliance with these programs. This study assessed adherence to liver function test (LFT) requirements included in the REMS program for bosentan and the results suggest that the program may not have adequately guaranteed adherence to monthly monitoring of LFTs.

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