Safety and Tolerability of Givinostat: Evidence From Real‐World and Clinical Practice
Year: 2026
Type: article
Study design: Observational Study
Abstract: OBJECTIVE: The aim of our study was to establish the prevalence of adverse events in a real-world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy. METHODS: The cohort included 90 ambulant boys, with age when treatment started between 6 and 23 years (mean 10.1 years, SD: 3.2 years) and with a follow up between 6.0 and 14.6 months (mean 10.6 months; SD 2.6 months). RESULTS: Platelets count decrease and triglyceride levels increase were the most common adverse events, followed by diarrhea. A dose reduction was needed in 38 of the 90 boys following thrombocytopenia (n = 34), diarrhea (n = 2), and hypertriglyceridemia (n = 4), with two of the boys presenting both thrombocytopenia and hypertriglyceridemia. Eleven of the 38 boys with initial dose reduction (10 with thrombocytopenia and 1 with hypertriglyceridemia) required an additional dose reduction for persistence of values outside the threshold, with one of them discontinuing treatment because of persistent hypertriglyceridemia even with the lowest dose. INTERPRETATION: Our results confirm the safety profile observed in the pivotal study providing further evidence of the management of the drug in a real-world setting. In our experience, treatment with givinostat was well managed by maintaining a strict monitoring. This was facilitated by allowing families to perform blood tests in local labs, reducing the stress and burden of frequent visits to the hospital.
DOI: 10.1002/acn3.70397
Authors: Marika Pane, Anna Capasso, Chiara Arpaia, Adele D’Amico, Emilio Albamonte, Federica Trucco, Maria Sframeli, Riccardo Masson, Francesca Magri, Luca Bello, Romina Venditti, Claudia Dosi, Michela Catteruccia, Michele Tosi, Claudio Bruno, Sonia Messina, Giacomo Comi, Elena Pegoraro, Valeria A. Sansone, Eugenio Mercuri
Institutions: Università Cattolica del Sacro Cuore, Agostino Gemelli University Polyclinic, Bambino Gesù Children's Hospital, Istituti di Ricovero e Cura a Carattere Scientifico, University of Milan, Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda, Istituto Giannina Gaslini, University of Messina, Fondazione IRCCS Istituto Neurologico Carlo Besta, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Padua, University of Genoa
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $4,100
OpenAlex ID: W7154705240