{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T23:51:36Z","timestamp":1777679496494,"version":"3.51.4"},"reference-count":13,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,4,17]],"date-time":"2026-04-17T00:00:00Z","timestamp":1776384000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,4,17]],"date-time":"2026-04-17T00:00:00Z","timestamp":1776384000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>The aim of our study was to establish the prevalence of adverse events in a real\u2010world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>The cohort included 90 ambulant boys, with age when treatment started between 6 and 23\u2009years (mean 10.1\u2009years, SD: 3.2\u2009years) and with a follow up between 6.0 and 14.6\u2009months (mean 10.6\u2009months; SD 2.6\u2009months).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      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                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200934), diarrhea (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20092), and hypertriglyceridemia (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20094), 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.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>Our results confirm the safety profile observed in the pivotal study providing further evidence of the management of the drug in a real\u2010world 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.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/acn3.70397","type":"journal-article","created":{"date-parts":[[2026,4,18]],"date-time":"2026-04-18T02:00:43Z","timestamp":1776477643000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Safety and Tolerability of Givinostat: Evidence From Real\u2010World and Clinical Practice"],"prefix":"10.1002","author":[{"given":"Marika","family":"Pane","sequence":"first","affiliation":[{"name":"Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS  Rome Italy"},{"name":"Pediatric Neurology Universit\u00e0 Cattolica del Sacro Cuore  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Capasso","sequence":"additional","affiliation":[{"name":"Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS  Rome Italy"},{"name":"Pediatric Neurology Universit\u00e0 Cattolica del Sacro Cuore  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chiara","family":"Arpaia","sequence":"additional","affiliation":[{"name":"Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS  Rome Italy"},{"name":"Pediatric Neurology Universit\u00e0 Cattolica del Sacro Cuore  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2438-2624","authenticated-orcid":false,"given":"Adele","family":"D\u2019Amico","sequence":"additional","affiliation":[{"name":"Department of Neurosciences Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Ges\u00f9 Children's Hospital, IRCCS  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emilio","family":"Albamonte","sequence":"additional","affiliation":[{"name":"The NEMO Center in Milan, Neurorehabilitation Unit University of Milan, ASST Niguarda Hospital  Milan Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Federica","family":"Trucco","sequence":"additional","affiliation":[{"name":"Center of Translational and Experimental Myology IRCCS Istituto Giannina Gaslini  Genoa Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria","family":"Sframeli","sequence":"additional","affiliation":[{"name":"Department of Clinical and Experimental Medicine University of Messina  Messina Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Riccardo","family":"Masson","sequence":"additional","affiliation":[{"name":"Developmental Neurology Unit Fondazione IRCCS Istituto Neurologico Carlo Besta  Milan Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francesca","family":"Magri","sequence":"additional","affiliation":[{"name":"Neurology Unit Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico  Milan Italy"},{"name":"Department of Pathophysiology and Transplantation Dino Ferrari Center University of Milan  Milan Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3075-6525","authenticated-orcid":false,"given":"Luca","family":"Bello","sequence":"additional","affiliation":[{"name":"Dipartimenti di Neuroscienze, DNS Universit\u00e0 Degli Studi di Padova  Padova Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Romina","family":"Venditti","sequence":"additional","affiliation":[{"name":"Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS  Rome Italy"},{"name":"Pediatric Neurology Universit\u00e0 Cattolica del Sacro Cuore  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudia","family":"Dosi","sequence":"additional","affiliation":[{"name":"Developmental Neurology Unit Fondazione IRCCS Istituto Neurologico Carlo Besta  Milan Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michela","family":"Catteruccia","sequence":"additional","affiliation":[{"name":"Department of Neurosciences Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Ges\u00f9 Children's Hospital, IRCCS  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9083-7837","authenticated-orcid":false,"given":"Michele","family":"Tosi","sequence":"additional","affiliation":[{"name":"Department of Neurosciences Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Ges\u00f9 Children's Hospital, IRCCS  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudio","family":"Bruno","sequence":"additional","affiliation":[{"name":"Center of Translational and Experimental Myology IRCCS Istituto Giannina Gaslini  Genoa Italy"},{"name":"Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health University of Genova  Genova Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sonia","family":"Messina","sequence":"additional","affiliation":[{"name":"Department of Clinical and Experimental Medicine University of Messina  Messina Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giacomo","family":"Comi","sequence":"additional","affiliation":[{"name":"Neurology Unit Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico  Milan Italy"},{"name":"Department of Pathophysiology and Transplantation Dino Ferrari Center University of Milan  Milan Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7740-4156","authenticated-orcid":false,"given":"Elena","family":"Pegoraro","sequence":"additional","affiliation":[{"name":"Dipartimenti di Neuroscienze, DNS Universit\u00e0 Degli Studi di Padova  Padova Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Valeria A.","family":"Sansone","sequence":"additional","affiliation":[{"name":"The NEMO Center in Milan, Neurorehabilitation Unit University of Milan, ASST Niguarda Hospital  Milan Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS  Rome Italy"},{"name":"Pediatric Neurology Universit\u00e0 Cattolica del Sacro Cuore  Rome Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2026,4,17]]},"reference":[{"issue":"9869","key":"e_1_2_10_2_1","doi-asserted-by":"crossref","first-page":"845","DOI":"10.1016\/S0140-6736(12)61897-2","article-title":"Muscular Dystrophies","volume":"381","author":"Mercuri 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J.","year":"2018","journal-title":"Lancet Neurology"},{"issue":"3","key":"e_1_2_10_4_1","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","article-title":"Diagnosis and Management of Duchenne Muscular Dystrophy, Part 1: Diagnosis, and Neuromuscular, Rehabilitation, Endocrine, and Gastrointestinal and Nutritional Management","volume":"17","author":"Birnkrant D. J.","year":"2018","journal-title":"Lancet Neurology"},{"issue":"1","key":"e_1_2_10_5_1","doi-asserted-by":"crossref","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne Muscular Dystrophy","volume":"18","author":"Duan D.","year":"2021","journal-title":"Nature Reviews Disease Primers"},{"issue":"7","key":"e_1_2_10_6_1","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1038\/s41582-019-0203-3","article-title":"Therapeutic Developments for Duchenne Muscular Dystrophy","volume":"15","author":"Verhaart I. E. C.","year":"2019","journal-title":"Nature Reviews. Neurology"},{"issue":"10","key":"e_1_2_10_7_1","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1016\/j.nmd.2016.07.002","article-title":"Histological Effects of Givinostat in Boys With Duchenne Muscular Dystrophy","volume":"26","author":"Bettica P.","year":"2016","journal-title":"Neuromuscular Disorders"},{"issue":"1","key":"e_1_2_10_8_1","doi-asserted-by":"crossref","first-page":"79","DOI":"10.2119\/molmed.2013.00011","article-title":"Preclinical Studies in the mdx Mouse Model of Duchenne Muscular Dystrophy With the Histone Deacetylase Inhibitor Givinostat","volume":"19","author":"Consalvi S.","year":"2013","journal-title":"Molecular Medicine"},{"key":"e_1_2_10_9_1","doi-asserted-by":"crossref","DOI":"10.1016\/j.phrs.2021.105751","article-title":"Givinostat as Metabolic Enhancer Reverting Mitochondrial Biogenesis Deficit in Duchenne Muscular Dystrophy","volume":"170","author":"Giovarelli M.","year":"2021","journal-title":"Pharmacological Research"},{"issue":"1","key":"e_1_2_10_10_1","doi-asserted-by":"crossref","DOI":"10.1186\/s13395-021-00273-6","article-title":"The Pan HDAC Inhibitor Givinostat Improves Muscle Function and Histological Parameters in Two Duchenne Muscular Dystrophy Murine Models Expressing Different Haplotypes of the LTBP4 Gene","volume":"11","author":"Licandro S. A.","year":"2021","journal-title":"Skeletal Muscle"},{"issue":"4","key":"e_1_2_10_11_1","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1016\/S1474-4422(24)00036-X","article-title":"Safety and Efficacy of Givinostat in Boys With Duchenne Muscular Dystrophy (EPIDYS): A Multicentre, Randomised, Double\u2010Blind, Placebo\u2010Controlled, Phase 3 Trial","volume":"23","author":"Mercuri E.","year":"2024","journal-title":"Lancet Neurology"},{"issue":"11","key":"e_1_2_10_12_1","doi-asserted-by":"crossref","first-page":"2335","DOI":"10.1002\/acn3.70165","article-title":"Long\u2010Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons","volume":"12","author":"McDonald C. M.","year":"2025","journal-title":"Annals of Clinical and Translational Neurology"},{"issue":"1718","key":"e_1_2_10_13_1","doi-asserted-by":"crossref","first-page":"204","DOI":"10.58347\/tml.2024.1718c","article-title":"Givinostat (Duvyzat) for Duchenne Muscular Dystrophy","volume":"66","year":"2024","journal-title":"Medical Letter on Drugs and Therapeutics"},{"issue":"4","key":"e_1_2_10_14_1","first-page":"135","article-title":"Cardiac Surveillance in the Era of Duvyzat: Do We Need to Do More?","volume":"44","author":"Pane M.","year":"2025","journal-title":"Acta Myologica"}],"container-title":["Annals of Clinical and Translational Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/acn3.70397","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/acn3.70397","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/acn3.70397","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,18]],"date-time":"2026-04-18T02:00:45Z","timestamp":1776477645000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/acn3.70397"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4,17]]},"references-count":13,"alternative-id":["10.1002\/acn3.70397"],"URL":"https:\/\/doi.org\/10.1002\/acn3.70397","archive":["Portico"],"relation":{},"ISSN":["2328-9503","2328-9503"],"issn-type":[{"value":"2328-9503","type":"print"},{"value":"2328-9503","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4,17]]},"assertion":[{"value":"2026-01-27","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-03-23","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-04-17","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"acn3.70397"}}