Clinical Experience With Gene Therapy in Older Patients With Spinal Muscular Atrophy
| Autor: | Vanessa Battista, Jean Flickinger, Elizabeth Kichula, Susan E. Matesanz, Jennifer N. Jones |
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| Rok vydání: | 2021 |
| Předmět: |
Male
medicine.medical_specialty Recombinant Fusion Proteins Genetic enhancement Oligonucleotides Motor Activity Spinal Muscular Atrophies of Childhood Cohort Studies 03 medical and health sciences 0302 clinical medicine Developmental Neuroscience Older patients 030225 pediatrics Internal medicine medicine Humans Dosing Biological Products business.industry Confounding Age Factors Infant Genetic Therapy Spinal muscular atrophy medicine.disease SMA Clinical trial Treatment Outcome Neurology Pediatrics Perinatology and Child Health Female Neurology (clinical) Intravenous steroids business 030217 neurology & neurosurgery |
| Zdroj: | Pediatric Neurology. 118:1-5 |
| ISSN: | 0887-8994 |
| DOI: | 10.1016/j.pediatrneurol.2021.01.012 |
| Popis: | Background Onasemnogene abeparvovec was recently approved for the treatment of spinal muscular atrophy (SMA) in children younger than two years; however, clinical trials were primarily completed in children younger than seven months, so practical experience dosing older children began in summer 2019. Here, we look at the safety and efficacy of onasemnogene in seven infants older than seven months who were treated at our center. Methods Seven patients were included. Results Acute viral symptoms with emesis and/or fever were seen in six of seven patients two to three days after the infusion. Thrombocytopenia occurred in four of seven patients, and six of seven patients had prolonged steroid courses due to persistently elevated liver enzymes, one of whom required escalation to intravenous steroids. All patients demonstrated motor improvements, which were apparent by three months, although with continued progress in those patients followed for longer periods of time. Conclusions Overall, onasemnogene appears to be efficacious in children older than seven months and well tolerated. Side effects were similar to those previously reported, although more common and in some cases more severe and more prolonged than seen in the original trials. The impact of age, weight, and other confounding factors on development of side effects still needs to be elucidated. |
| Databáze: | OpenAIRE |
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