Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context
Summary: Background: Efficacy and safety of onasemnogene abeparvovec (OA) for Spinal Muscular Atrophy infants under 7 months and <8.5 kg has been reported in clinical trials. This study examines efficacy and safety predictors in a wide age (22 days–72 months) and weight (3.2–17 kg) range, also i...
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2023-05-01
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| Series: | EClinicalMedicine |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2589537023001748 |
| _version_ | 1797832687394750464 |
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| author | Marika Pane Beatrice Berti Anna Capasso Giorgia Coratti Antonio Varone Adele D’Amico Sonia Messina Riccardo Masson Valeria Ada Sansone Maria Alice Donati Caterina Agosto Claudio Bruno Federica Ricci Antonella Pini Delio Gagliardi Massimiliano Filosto Stefania Corti Daniela Leone Concetta Palermo Roberta Onesimo Roberto De Sanctis Martina Ricci Ilaria Bitetti Maria Sframeli Claudia Dosi Emilio Albamonte Chiara Ticci Noemi Brolatti Enrico Bertini Richard Finkel Eugenio Mercuri Maria Carmela Pera Chiara Bravetti Marco Piastra Orazio Genovese Gianpaolo Cicala Nicola Forcina Sara Carnicella Giulia Stanca Michele Sacchini Michela Catteruccia Michele Tosi Renato Cutrera Claudio Chierchi Maria Beatrice Chiarini Francesca Salmin Marina Pedemonte Alessandra Govoni Irene Mizzoni Simone Morando Riccardo Zanin Enrica Rolle Eleonora Salomon Melania Giannotta Gaia Scarpini Antonio Toscano Eloisa Gitto Roberto Materia Rossella D’Alessandro |
| author_facet | Marika Pane Beatrice Berti Anna Capasso Giorgia Coratti Antonio Varone Adele D’Amico Sonia Messina Riccardo Masson Valeria Ada Sansone Maria Alice Donati Caterina Agosto Claudio Bruno Federica Ricci Antonella Pini Delio Gagliardi Massimiliano Filosto Stefania Corti Daniela Leone Concetta Palermo Roberta Onesimo Roberto De Sanctis Martina Ricci Ilaria Bitetti Maria Sframeli Claudia Dosi Emilio Albamonte Chiara Ticci Noemi Brolatti Enrico Bertini Richard Finkel Eugenio Mercuri Maria Carmela Pera Chiara Bravetti Marco Piastra Orazio Genovese Gianpaolo Cicala Nicola Forcina Sara Carnicella Giulia Stanca Michele Sacchini Michela Catteruccia Michele Tosi Renato Cutrera Claudio Chierchi Maria Beatrice Chiarini Francesca Salmin Marina Pedemonte Alessandra Govoni Irene Mizzoni Simone Morando Riccardo Zanin Enrica Rolle Eleonora Salomon Melania Giannotta Gaia Scarpini Antonio Toscano Eloisa Gitto Roberto Materia Rossella D’Alessandro |
| author_sort | Marika Pane |
| collection | DOAJ |
| description | Summary: Background: Efficacy and safety of onasemnogene abeparvovec (OA) for Spinal Muscular Atrophy infants under 7 months and <8.5 kg has been reported in clinical trials. This study examines efficacy and safety predictors in a wide age (22 days–72 months) and weight (3.2–17 kg) range, also including patients previously treated with other drugs. Methods: 46 patients were treated for 12 months between January 2020 and March 2022. Safety profile was also available for another 21 patients with at least 6 month follow-up after OA infusion. 19/67 were treatment naïve when treated with OA. Motor function was measured with the CHOP-INTEND. Findings: CHOP-INTEND changes varied among age groups. Baseline score and age at OA treatment best predicted changes. A mixed model post-hoc analysis showed that in patients treated before the age of 24 months the CHOP-INTEND changes were already significant 3 months after OA while in those treated after the age of 24 months the difference was only significant 12 months after OA. Adverse events occurred in 51/67. The risk for elevated transaminases serum levels was higher in older patients. This was also true for weight and for pre-treatment with nusinersen when analysed individually. A binomial negative regression analysis showed that only age at OA treatment had a significant effect on the risk of elevated transaminases. Interpretation: Our paper describes OA 12-month follow-up showing efficacy across various age and weight groups not targeted by clinical trials. The study identifies prognostic factors for safety and efficacy in treatment selection. Funding: None. |
| first_indexed | 2024-04-09T14:11:49Z |
| format | Article |
| id | doaj.art-40165d9821824625a9a6285a88f2fdbc |
| institution | Directory Open Access Journal |
| issn | 2589-5370 |
| language | English |
| last_indexed | 2024-04-09T14:11:49Z |
| publishDate | 2023-05-01 |
| publisher | Elsevier |
| record_format | Article |
| series | EClinicalMedicine |
| spelling | doaj.art-40165d9821824625a9a6285a88f2fdbc2023-05-06T04:38:47ZengElsevierEClinicalMedicine2589-53702023-05-0159101997Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in contextMarika Pane0Beatrice Berti1Anna Capasso2Giorgia Coratti3Antonio Varone4Adele D’Amico5Sonia Messina6Riccardo Masson7Valeria Ada Sansone8Maria Alice Donati9Caterina Agosto10Claudio Bruno11Federica Ricci12Antonella Pini13Delio Gagliardi14Massimiliano Filosto15Stefania Corti16Daniela Leone17Concetta Palermo18Roberta Onesimo19Roberto De Sanctis20Martina Ricci21Ilaria Bitetti22Maria Sframeli23Claudia Dosi24Emilio Albamonte25Chiara Ticci26Noemi Brolatti27Enrico Bertini28Richard Finkel29Eugenio Mercuri30Maria Carmela PeraChiara BravettiMarco PiastraOrazio GenoveseGianpaolo CicalaNicola ForcinaSara CarnicellaGiulia StancaMichele SacchiniMichela CatterucciaMichele TosiRenato CutreraClaudio ChierchiMaria Beatrice ChiariniFrancesca SalminMarina PedemonteAlessandra GovoniIrene MizzoniSimone MorandoRiccardo ZaninEnrica RolleEleonora SalomonMelania GiannottaGaia ScarpiniAntonio ToscanoEloisa GittoRoberto MateriaRossella D’AlessandroPaediatric Neurology, Catholic University, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyCentro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyPaediatric Neurology, Catholic University, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyPaediatric Neurology, Catholic University, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyDepartment of Neurosciences, Paediatric Neurology, Santobono-Pausilipon Children's Hospital, Naples, ItalyUnit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children’s Hospital IRCCS, Rome, ItalyDepartment of Clinical and Experimental Medicine, University of Messina, Messina, ItalyFondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit, Milan, ItalyNeurorehabilitation Unit, Centro Clinico Nemo, Niguarda Hospital, University of Milan, Milano, ItalyMetabolic and Muscular Unit, Meyer Children's Hospital IRCCS, Florence, ItalyDipartimento di Salute della Donna e del Bambino, Università di Padova, Padua, ItalyCenter of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, ItalyAOU Città della Salute e della Scienza di Torino, Presidio OIRM (SC Neuropsichiatria Infantile), Turin, ItalyNeuromuscular Pediatric Unit, IRCCS Istituto delle Scienze Neurologiche di Bologna-UOC Neuropsichiatria dell’Età Pediatrica, Bologna, ItalyPediatric Neurology Unit, Pediatric Hospital ''Giovanni XXIII'', Bari, ItalyDepartment of Clinical and Experimental Sciences, NeMO-Brescia Clinical Center for Neuromuscular Diseases, University of Brescia; Brescia, ItalyFondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico Milan, Milan, ItalyCentro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyCentro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyRare Disease Unit, Pediatric Unit - Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyPaediatric Neurology, Catholic University, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyPaediatric Neurology, Catholic University, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, ItalyDepartment of Neurosciences, Paediatric Neurology, Santobono-Pausilipon Children's Hospital, Naples, ItalyDepartment of Clinical and Experimental Medicine, University of Messina, Messina, ItalyFondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit, Milan, ItalyNeurorehabilitation Unit, Centro Clinico Nemo, Niguarda Hospital, University of Milan, Milano, ItalyMetabolic and Muscular Unit, Meyer Children's Hospital IRCCS, Florence, ItalyCenter of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, ItalyUnit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children’s Hospital IRCCS, Rome, ItalyDepartment of Paediatric Medicine, Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USAPaediatric Neurology, Catholic University, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Corresponding author. Paediatric Neurology Unit, Policlinico Gemelli, Largo Gemelli, Roma, 00168 Italy.Summary: Background: Efficacy and safety of onasemnogene abeparvovec (OA) for Spinal Muscular Atrophy infants under 7 months and <8.5 kg has been reported in clinical trials. This study examines efficacy and safety predictors in a wide age (22 days–72 months) and weight (3.2–17 kg) range, also including patients previously treated with other drugs. Methods: 46 patients were treated for 12 months between January 2020 and March 2022. Safety profile was also available for another 21 patients with at least 6 month follow-up after OA infusion. 19/67 were treatment naïve when treated with OA. Motor function was measured with the CHOP-INTEND. Findings: CHOP-INTEND changes varied among age groups. Baseline score and age at OA treatment best predicted changes. A mixed model post-hoc analysis showed that in patients treated before the age of 24 months the CHOP-INTEND changes were already significant 3 months after OA while in those treated after the age of 24 months the difference was only significant 12 months after OA. Adverse events occurred in 51/67. The risk for elevated transaminases serum levels was higher in older patients. This was also true for weight and for pre-treatment with nusinersen when analysed individually. A binomial negative regression analysis showed that only age at OA treatment had a significant effect on the risk of elevated transaminases. Interpretation: Our paper describes OA 12-month follow-up showing efficacy across various age and weight groups not targeted by clinical trials. The study identifies prognostic factors for safety and efficacy in treatment selection. Funding: None.http://www.sciencedirect.com/science/article/pii/S2589537023001748Spinal muscular atrophyGene therapyFollow-upLongitudinalSafety |
| spellingShingle | Marika Pane Beatrice Berti Anna Capasso Giorgia Coratti Antonio Varone Adele D’Amico Sonia Messina Riccardo Masson Valeria Ada Sansone Maria Alice Donati Caterina Agosto Claudio Bruno Federica Ricci Antonella Pini Delio Gagliardi Massimiliano Filosto Stefania Corti Daniela Leone Concetta Palermo Roberta Onesimo Roberto De Sanctis Martina Ricci Ilaria Bitetti Maria Sframeli Claudia Dosi Emilio Albamonte Chiara Ticci Noemi Brolatti Enrico Bertini Richard Finkel Eugenio Mercuri Maria Carmela Pera Chiara Bravetti Marco Piastra Orazio Genovese Gianpaolo Cicala Nicola Forcina Sara Carnicella Giulia Stanca Michele Sacchini Michela Catteruccia Michele Tosi Renato Cutrera Claudio Chierchi Maria Beatrice Chiarini Francesca Salmin Marina Pedemonte Alessandra Govoni Irene Mizzoni Simone Morando Riccardo Zanin Enrica Rolle Eleonora Salomon Melania Giannotta Gaia Scarpini Antonio Toscano Eloisa Gitto Roberto Materia Rossella D’Alessandro Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context EClinicalMedicine Spinal muscular atrophy Gene therapy Follow-up Longitudinal Safety |
| title | Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context |
| title_full | Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context |
| title_fullStr | Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context |
| title_full_unstemmed | Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context |
| title_short | Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context |
| title_sort | onasemnogene abeparvovec in spinal muscular atrophy predictors of efficacy and safety in naive patients with spinal muscular atrophy and following switch from other therapiesresearch in context |
| topic | Spinal muscular atrophy Gene therapy Follow-up Longitudinal Safety |
| url | http://www.sciencedirect.com/science/article/pii/S2589537023001748 |
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