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...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
Marika Pane [verfasserIn] Beatrice Berti [verfasserIn] Anna Capasso [verfasserIn] Giorgia Coratti [verfasserIn] Antonio Varone [verfasserIn] Adele D’Amico [verfasserIn] Sonia Messina [verfasserIn] Riccardo Masson [verfasserIn] Valeria Ada Sansone [verfasserIn] Maria Alice Donati [verfasserIn] Caterina Agosto [verfasserIn] Claudio Bruno [verfasserIn] Federica Ricci [verfasserIn] Antonella Pini [verfasserIn] Delio Gagliardi [verfasserIn] Massimiliano Filosto [verfasserIn] Stefania Corti [verfasserIn] Daniela Leone [verfasserIn] Concetta Palermo [verfasserIn] Roberta Onesimo [verfasserIn] Roberto De Sanctis [verfasserIn] Martina Ricci [verfasserIn] Ilaria Bitetti [verfasserIn] Maria Sframeli [verfasserIn] Claudia Dosi [verfasserIn] Emilio Albamonte [verfasserIn] Chiara Ticci [verfasserIn] Noemi Brolatti [verfasserIn] Enrico Bertini [verfasserIn] Richard Finkel [verfasserIn] Eugenio Mercuri [verfasserIn] Maria Carmela Pera [verfasserIn] Chiara Bravetti [verfasserIn] Marco Piastra [verfasserIn] Orazio Genovese [verfasserIn] Gianpaolo Cicala [verfasserIn] Nicola Forcina [verfasserIn] Sara Carnicella [verfasserIn] Giulia Stanca [verfasserIn] Michele Sacchini [verfasserIn] Michela Catteruccia [verfasserIn] Michele Tosi [verfasserIn] Renato Cutrera [verfasserIn] Claudio Chierchi [verfasserIn] Maria Beatrice Chiarini [verfasserIn] Francesca Salmin [verfasserIn] Marina Pedemonte [verfasserIn] Alessandra Govoni [verfasserIn] Irene Mizzoni [verfasserIn] Simone Morando [verfasserIn] Riccardo Zanin [verfasserIn] Enrica Rolle [verfasserIn] Eleonora Salomon [verfasserIn] Melania Giannotta [verfasserIn] Gaia Scarpini [verfasserIn] Antonio Toscano [verfasserIn] Eloisa Gitto [verfasserIn] Roberto Materia [verfasserIn] Rossella D’Alessandro [verfasserIn] |
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| Format: |
E-Artikel |
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| Sprache: |
Englisch |
| Erschienen: |
2023 |
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| Schlagwörter: |
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| Übergeordnetes Werk: |
In: EClinicalMedicine - Elsevier, 2018, 59(2023), Seite 101997- |
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| Übergeordnetes Werk: |
volume:59 ; year:2023 ; pages:101997- |
| Links: |
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| DOI / URN: |
10.1016/j.eclinm.2023.101997 |
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| Katalog-ID: |
DOAJ090090624 |
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| 245 | 1 | 0 | |a 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 |
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| 520 | |a 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. | ||
| 650 | 4 | |a Spinal muscular atrophy | |
| 650 | 4 | |a Gene therapy | |
| 650 | 4 | |a Follow-up | |
| 650 | 4 | |a Longitudinal | |
| 650 | 4 | |a Safety | |
| 653 | 0 | |a Medicine (General) | |
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10.1016/j.eclinm.2023.101997 doi (DE-627)DOAJ090090624 (DE-599)DOAJ40165d9821824625a9a6285a88f2fdbc DE-627 ger DE-627 rakwb eng R5-920 Marika Pane verfasserin aut 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 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier 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. Spinal muscular atrophy Gene therapy Follow-up Longitudinal Safety Medicine (General) Beatrice Berti verfasserin aut Anna Capasso verfasserin aut Giorgia Coratti verfasserin aut Antonio Varone verfasserin aut Adele D’Amico verfasserin aut Sonia Messina verfasserin aut Riccardo Masson verfasserin aut Valeria Ada Sansone verfasserin aut Maria Alice Donati verfasserin aut Caterina Agosto verfasserin aut Claudio Bruno verfasserin aut Federica Ricci verfasserin aut Antonella Pini verfasserin aut Delio Gagliardi verfasserin aut Massimiliano Filosto verfasserin aut Stefania Corti verfasserin aut Daniela Leone verfasserin aut Concetta Palermo verfasserin aut Roberta Onesimo verfasserin aut Roberto De Sanctis verfasserin aut Martina Ricci verfasserin aut Ilaria Bitetti verfasserin aut Maria Sframeli verfasserin aut Claudia Dosi verfasserin aut Emilio Albamonte verfasserin aut Chiara Ticci verfasserin aut Noemi Brolatti verfasserin aut Enrico Bertini verfasserin aut Richard Finkel verfasserin aut Eugenio Mercuri verfasserin aut Maria Carmela Pera verfasserin aut Chiara Bravetti verfasserin aut Marco Piastra verfasserin aut Orazio Genovese verfasserin aut Gianpaolo Cicala verfasserin aut Nicola Forcina verfasserin aut Sara Carnicella verfasserin aut Giulia Stanca verfasserin aut Michele Sacchini verfasserin aut Michela Catteruccia verfasserin aut Michele Tosi verfasserin aut Renato Cutrera verfasserin aut Claudio Chierchi verfasserin aut Maria Beatrice Chiarini verfasserin aut Francesca Salmin verfasserin aut Marina Pedemonte verfasserin aut Alessandra Govoni verfasserin aut Irene Mizzoni verfasserin aut Simone Morando verfasserin aut Riccardo Zanin verfasserin aut Enrica Rolle verfasserin aut Eleonora Salomon verfasserin aut Melania Giannotta verfasserin aut Gaia Scarpini verfasserin aut Antonio Toscano verfasserin aut Eloisa Gitto verfasserin aut Roberto Materia verfasserin aut Rossella D’Alessandro verfasserin aut In EClinicalMedicine Elsevier, 2018 59(2023), Seite 101997- (DE-627)1035271834 25895370 nnns volume:59 year:2023 pages:101997- https://doi.org/10.1016/j.eclinm.2023.101997 kostenfrei https://doaj.org/article/40165d9821824625a9a6285a88f2fdbc kostenfrei http://www.sciencedirect.com/science/article/pii/S2589537023001748 kostenfrei https://doaj.org/toc/2589-5370 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2110 GBV_ILN_2112 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4333 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 59 2023 101997- |
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10.1016/j.eclinm.2023.101997 doi (DE-627)DOAJ090090624 (DE-599)DOAJ40165d9821824625a9a6285a88f2fdbc DE-627 ger DE-627 rakwb eng R5-920 Marika Pane verfasserin aut 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 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier 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. Spinal muscular atrophy Gene therapy Follow-up Longitudinal Safety Medicine (General) Beatrice Berti verfasserin aut Anna Capasso verfasserin aut Giorgia Coratti verfasserin aut Antonio Varone verfasserin aut Adele D’Amico verfasserin aut Sonia Messina verfasserin aut Riccardo Masson verfasserin aut Valeria Ada Sansone verfasserin aut Maria Alice Donati verfasserin aut Caterina Agosto verfasserin aut Claudio Bruno verfasserin aut Federica Ricci verfasserin aut Antonella Pini verfasserin aut Delio Gagliardi verfasserin aut Massimiliano Filosto verfasserin aut Stefania Corti verfasserin aut Daniela Leone verfasserin aut Concetta Palermo verfasserin aut Roberta Onesimo verfasserin aut Roberto De Sanctis verfasserin aut Martina Ricci verfasserin aut Ilaria Bitetti verfasserin aut Maria Sframeli verfasserin aut Claudia Dosi verfasserin aut Emilio Albamonte verfasserin aut Chiara Ticci verfasserin aut Noemi Brolatti verfasserin aut Enrico Bertini verfasserin aut Richard Finkel verfasserin aut Eugenio Mercuri verfasserin aut Maria Carmela Pera verfasserin aut Chiara Bravetti verfasserin aut Marco Piastra verfasserin aut Orazio Genovese verfasserin aut Gianpaolo Cicala verfasserin aut Nicola Forcina verfasserin aut Sara Carnicella verfasserin aut Giulia Stanca verfasserin aut Michele Sacchini verfasserin aut Michela Catteruccia verfasserin aut Michele Tosi verfasserin aut Renato Cutrera verfasserin aut Claudio Chierchi verfasserin aut Maria Beatrice Chiarini verfasserin aut Francesca Salmin verfasserin aut Marina Pedemonte verfasserin aut Alessandra Govoni verfasserin aut Irene Mizzoni verfasserin aut Simone Morando verfasserin aut Riccardo Zanin verfasserin aut Enrica Rolle verfasserin aut Eleonora Salomon verfasserin aut Melania Giannotta verfasserin aut Gaia Scarpini verfasserin aut Antonio Toscano verfasserin aut Eloisa Gitto verfasserin aut Roberto Materia verfasserin aut Rossella D’Alessandro verfasserin aut In EClinicalMedicine Elsevier, 2018 59(2023), Seite 101997- (DE-627)1035271834 25895370 nnns volume:59 year:2023 pages:101997- https://doi.org/10.1016/j.eclinm.2023.101997 kostenfrei https://doaj.org/article/40165d9821824625a9a6285a88f2fdbc kostenfrei http://www.sciencedirect.com/science/article/pii/S2589537023001748 kostenfrei https://doaj.org/toc/2589-5370 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2110 GBV_ILN_2112 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4333 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 59 2023 101997- |
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10.1016/j.eclinm.2023.101997 doi (DE-627)DOAJ090090624 (DE-599)DOAJ40165d9821824625a9a6285a88f2fdbc DE-627 ger DE-627 rakwb eng R5-920 Marika Pane verfasserin aut 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 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier 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. Spinal muscular atrophy Gene therapy Follow-up Longitudinal Safety Medicine (General) Beatrice Berti verfasserin aut Anna Capasso verfasserin aut Giorgia Coratti verfasserin aut Antonio Varone verfasserin aut Adele D’Amico verfasserin aut Sonia Messina verfasserin aut Riccardo Masson verfasserin aut Valeria Ada Sansone verfasserin aut Maria Alice Donati verfasserin aut Caterina Agosto verfasserin aut Claudio Bruno verfasserin aut Federica Ricci verfasserin aut Antonella Pini verfasserin aut Delio Gagliardi verfasserin aut Massimiliano Filosto verfasserin aut Stefania Corti verfasserin aut Daniela Leone verfasserin aut Concetta Palermo verfasserin aut Roberta Onesimo verfasserin aut Roberto De Sanctis verfasserin aut Martina Ricci verfasserin aut Ilaria Bitetti verfasserin aut Maria Sframeli verfasserin aut Claudia Dosi verfasserin aut Emilio Albamonte verfasserin aut Chiara Ticci verfasserin aut Noemi Brolatti verfasserin aut Enrico Bertini verfasserin aut Richard Finkel verfasserin aut Eugenio Mercuri verfasserin aut Maria Carmela Pera verfasserin aut Chiara Bravetti verfasserin aut Marco Piastra verfasserin aut Orazio Genovese verfasserin aut Gianpaolo Cicala verfasserin aut Nicola Forcina verfasserin aut Sara Carnicella verfasserin aut Giulia Stanca verfasserin aut Michele Sacchini verfasserin aut Michela Catteruccia verfasserin aut Michele Tosi verfasserin aut Renato Cutrera verfasserin aut Claudio Chierchi verfasserin aut Maria Beatrice Chiarini verfasserin aut Francesca Salmin verfasserin aut Marina Pedemonte verfasserin aut Alessandra Govoni verfasserin aut Irene Mizzoni verfasserin aut Simone Morando verfasserin aut Riccardo Zanin verfasserin aut Enrica Rolle verfasserin aut Eleonora Salomon verfasserin aut Melania Giannotta verfasserin aut Gaia Scarpini verfasserin aut Antonio Toscano verfasserin aut Eloisa Gitto verfasserin aut Roberto Materia verfasserin aut Rossella D’Alessandro verfasserin aut In EClinicalMedicine Elsevier, 2018 59(2023), Seite 101997- (DE-627)1035271834 25895370 nnns volume:59 year:2023 pages:101997- https://doi.org/10.1016/j.eclinm.2023.101997 kostenfrei https://doaj.org/article/40165d9821824625a9a6285a88f2fdbc kostenfrei http://www.sciencedirect.com/science/article/pii/S2589537023001748 kostenfrei https://doaj.org/toc/2589-5370 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2110 GBV_ILN_2112 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4333 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 59 2023 101997- |
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10.1016/j.eclinm.2023.101997 doi (DE-627)DOAJ090090624 (DE-599)DOAJ40165d9821824625a9a6285a88f2fdbc DE-627 ger DE-627 rakwb eng R5-920 Marika Pane verfasserin aut 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 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier 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. Spinal muscular atrophy Gene therapy Follow-up Longitudinal Safety Medicine (General) Beatrice Berti verfasserin aut Anna Capasso verfasserin aut Giorgia Coratti verfasserin aut Antonio Varone verfasserin aut Adele D’Amico verfasserin aut Sonia Messina verfasserin aut Riccardo Masson verfasserin aut Valeria Ada Sansone verfasserin aut Maria Alice Donati verfasserin aut Caterina Agosto verfasserin aut Claudio Bruno verfasserin aut Federica Ricci verfasserin aut Antonella Pini verfasserin aut Delio Gagliardi verfasserin aut Massimiliano Filosto verfasserin aut Stefania Corti verfasserin aut Daniela Leone verfasserin aut Concetta Palermo verfasserin aut Roberta Onesimo verfasserin aut Roberto De Sanctis verfasserin aut Martina Ricci verfasserin aut Ilaria Bitetti verfasserin aut Maria Sframeli verfasserin aut Claudia Dosi verfasserin aut Emilio Albamonte verfasserin aut Chiara Ticci verfasserin aut Noemi Brolatti verfasserin aut Enrico Bertini verfasserin aut Richard Finkel verfasserin aut Eugenio Mercuri verfasserin aut Maria Carmela Pera verfasserin aut Chiara Bravetti verfasserin aut Marco Piastra verfasserin aut Orazio Genovese verfasserin aut Gianpaolo Cicala verfasserin aut Nicola Forcina verfasserin aut Sara Carnicella verfasserin aut Giulia Stanca verfasserin aut Michele Sacchini verfasserin aut Michela Catteruccia verfasserin aut Michele Tosi verfasserin aut Renato Cutrera verfasserin aut Claudio Chierchi verfasserin aut Maria Beatrice Chiarini verfasserin aut Francesca Salmin verfasserin aut Marina Pedemonte verfasserin aut Alessandra Govoni verfasserin aut Irene Mizzoni verfasserin aut Simone Morando verfasserin aut Riccardo Zanin verfasserin aut Enrica Rolle verfasserin aut Eleonora Salomon verfasserin aut Melania Giannotta verfasserin aut Gaia Scarpini verfasserin aut Antonio Toscano verfasserin aut Eloisa Gitto verfasserin aut Roberto Materia verfasserin aut Rossella D’Alessandro verfasserin aut In EClinicalMedicine Elsevier, 2018 59(2023), Seite 101997- (DE-627)1035271834 25895370 nnns volume:59 year:2023 pages:101997- https://doi.org/10.1016/j.eclinm.2023.101997 kostenfrei https://doaj.org/article/40165d9821824625a9a6285a88f2fdbc kostenfrei http://www.sciencedirect.com/science/article/pii/S2589537023001748 kostenfrei https://doaj.org/toc/2589-5370 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2110 GBV_ILN_2112 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4333 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 59 2023 101997- |
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10.1016/j.eclinm.2023.101997 doi (DE-627)DOAJ090090624 (DE-599)DOAJ40165d9821824625a9a6285a88f2fdbc DE-627 ger DE-627 rakwb eng R5-920 Marika Pane verfasserin aut 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 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier 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. Spinal muscular atrophy Gene therapy Follow-up Longitudinal Safety Medicine (General) Beatrice Berti verfasserin aut Anna Capasso verfasserin aut Giorgia Coratti verfasserin aut Antonio Varone verfasserin aut Adele D’Amico verfasserin aut Sonia Messina verfasserin aut Riccardo Masson verfasserin aut Valeria Ada Sansone verfasserin aut Maria Alice Donati verfasserin aut Caterina Agosto verfasserin aut Claudio Bruno verfasserin aut Federica Ricci verfasserin aut Antonella Pini verfasserin aut Delio Gagliardi verfasserin aut Massimiliano Filosto verfasserin aut Stefania Corti verfasserin aut Daniela Leone verfasserin aut Concetta Palermo verfasserin aut Roberta Onesimo verfasserin aut Roberto De Sanctis verfasserin aut Martina Ricci verfasserin aut Ilaria Bitetti verfasserin aut Maria Sframeli verfasserin aut Claudia Dosi verfasserin aut Emilio Albamonte verfasserin aut Chiara Ticci verfasserin aut Noemi Brolatti verfasserin aut Enrico Bertini verfasserin aut Richard Finkel verfasserin aut Eugenio Mercuri verfasserin aut Maria Carmela Pera verfasserin aut Chiara Bravetti verfasserin aut Marco Piastra verfasserin aut Orazio Genovese verfasserin aut Gianpaolo Cicala verfasserin aut Nicola Forcina verfasserin aut Sara Carnicella verfasserin aut Giulia Stanca verfasserin aut Michele Sacchini verfasserin aut Michela Catteruccia verfasserin aut Michele Tosi verfasserin aut Renato Cutrera verfasserin aut Claudio Chierchi verfasserin aut Maria Beatrice Chiarini verfasserin aut Francesca Salmin verfasserin aut Marina Pedemonte verfasserin aut Alessandra Govoni verfasserin aut Irene Mizzoni verfasserin aut Simone Morando verfasserin aut Riccardo Zanin verfasserin aut Enrica Rolle verfasserin aut Eleonora Salomon verfasserin aut Melania Giannotta verfasserin aut Gaia Scarpini verfasserin aut Antonio Toscano verfasserin aut Eloisa Gitto verfasserin aut Roberto Materia verfasserin aut Rossella D’Alessandro verfasserin aut In EClinicalMedicine Elsevier, 2018 59(2023), Seite 101997- (DE-627)1035271834 25895370 nnns volume:59 year:2023 pages:101997- https://doi.org/10.1016/j.eclinm.2023.101997 kostenfrei https://doaj.org/article/40165d9821824625a9a6285a88f2fdbc kostenfrei http://www.sciencedirect.com/science/article/pii/S2589537023001748 kostenfrei https://doaj.org/toc/2589-5370 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2110 GBV_ILN_2112 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4333 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 59 2023 101997- |
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Marika Pane @@aut@@ Beatrice Berti @@aut@@ Anna Capasso @@aut@@ Giorgia Coratti @@aut@@ Antonio Varone @@aut@@ Adele D’Amico @@aut@@ Sonia Messina @@aut@@ Riccardo Masson @@aut@@ Valeria Ada Sansone @@aut@@ Maria Alice Donati @@aut@@ Caterina Agosto @@aut@@ Claudio Bruno @@aut@@ Federica Ricci @@aut@@ Antonella Pini @@aut@@ Delio Gagliardi @@aut@@ Massimiliano Filosto @@aut@@ Stefania Corti @@aut@@ Daniela Leone @@aut@@ Concetta Palermo @@aut@@ Roberta Onesimo @@aut@@ Roberto De Sanctis @@aut@@ Martina Ricci @@aut@@ Ilaria Bitetti @@aut@@ Maria Sframeli @@aut@@ Claudia Dosi @@aut@@ Emilio Albamonte @@aut@@ Chiara Ticci @@aut@@ Noemi Brolatti @@aut@@ Enrico Bertini @@aut@@ Richard Finkel @@aut@@ Eugenio Mercuri @@aut@@ Maria Carmela Pera @@aut@@ Chiara Bravetti @@aut@@ Marco Piastra @@aut@@ Orazio Genovese @@aut@@ Gianpaolo Cicala @@aut@@ Nicola Forcina @@aut@@ Sara Carnicella @@aut@@ Giulia Stanca @@aut@@ Michele Sacchini @@aut@@ Michela Catteruccia @@aut@@ Michele Tosi @@aut@@ Renato Cutrera @@aut@@ Claudio Chierchi @@aut@@ Maria Beatrice Chiarini @@aut@@ Francesca Salmin @@aut@@ Marina Pedemonte @@aut@@ Alessandra Govoni @@aut@@ Irene Mizzoni @@aut@@ Simone Morando @@aut@@ Riccardo Zanin @@aut@@ Enrica Rolle @@aut@@ Eleonora Salomon @@aut@@ Melania Giannotta @@aut@@ Gaia Scarpini @@aut@@ Antonio Toscano @@aut@@ Eloisa Gitto @@aut@@ Roberto Materia @@aut@@ Rossella D’Alessandro @@aut@@ |
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<?xml version="1.0" encoding="UTF-8"?><collection xmlns="http://www.loc.gov/MARC21/slim"><record><leader>01000naa a22002652 4500</leader><controlfield tag="001">DOAJ090090624</controlfield><controlfield tag="003">DE-627</controlfield><controlfield tag="005">20230526104247.0</controlfield><controlfield tag="007">cr uuu---uuuuu</controlfield><controlfield tag="008">230526s2023 xx |||||o 00| ||eng c</controlfield><datafield tag="024" ind1="7" ind2=" "><subfield code="a">10.1016/j.eclinm.2023.101997</subfield><subfield code="2">doi</subfield></datafield><datafield tag="035" ind1=" " ind2=" "><subfield code="a">(DE-627)DOAJ090090624</subfield></datafield><datafield tag="035" ind1=" " ind2=" "><subfield code="a">(DE-599)DOAJ40165d9821824625a9a6285a88f2fdbc</subfield></datafield><datafield tag="040" ind1=" " ind2=" "><subfield code="a">DE-627</subfield><subfield code="b">ger</subfield><subfield code="c">DE-627</subfield><subfield code="e">rakwb</subfield></datafield><datafield tag="041" ind1=" " ind2=" "><subfield code="a">eng</subfield></datafield><datafield tag="050" ind1=" " ind2="0"><subfield code="a">R5-920</subfield></datafield><datafield tag="100" ind1="0" ind2=" "><subfield code="a">Marika Pane</subfield><subfield code="e">verfasserin</subfield><subfield code="4">aut</subfield></datafield><datafield tag="245" ind1="1" ind2="0"><subfield code="a">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</subfield></datafield><datafield tag="264" ind1=" " ind2="1"><subfield code="c">2023</subfield></datafield><datafield tag="336" ind1=" " ind2=" "><subfield code="a">Text</subfield><subfield code="b">txt</subfield><subfield code="2">rdacontent</subfield></datafield><datafield tag="337" ind1=" " ind2=" "><subfield code="a">Computermedien</subfield><subfield code="b">c</subfield><subfield code="2">rdamedia</subfield></datafield><datafield tag="338" ind1=" " ind2=" "><subfield code="a">Online-Ressource</subfield><subfield code="b">cr</subfield><subfield code="2">rdacarrier</subfield></datafield><datafield tag="520" ind1=" " ind2=" "><subfield code="a">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. 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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 |
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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 |
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R5-920 |
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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 |
| abstract |
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. |
| abstractGer |
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. |
| abstract_unstemmed |
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. |
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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 |
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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 |
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