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Abstract Details

Early Clinical Experience with Onasemnogene Abeparvovec in Spinal Muscular Atrophy (SMA) Type 2 Patients at a Single Center
Child Neurology and Developmental Neurology
P15 - Poster Session 15 (12:00 PM-1:00 PM)
5-004

To review early clinical experience with onasemnogene abeparvovec-xioi in SMA type 2 patients, given the lack of data from prior clinical trials
While onasemnogene was approved for children up to two years of age, including all SMA types, we only have clinical information on pre-symptomatic and SMA type 1 under 7 months.  With the wider approval, use has begun in older SMA type 2 patients, where the efficacy and safety are unknown. 
We reviewed onasemnogene treated patients at our site, to select patients with symptomatic SMA type 2.  We had three such patients.  Charts were reviewed for demographics, motor scores and safety concerns. 
Three patients were treated with SMA type 2.  Average age at diagnosis was 12.35 months, with treatment initiation at 12.93 months. Average age and weight at initiation of onasemnogene abeparvovec-xioi was 16.25 months and 10.74 kg.  Two patients were treated initially with nusinersen, receiving 4 doses before transitioning to onasemnogene therapy.  Patients showed a response to all treatment options.  In first month of follow-up after onasemnogene, Hammersmith Functional Motor Scale Expanded (HMFSE) increased by average of 5.3 points (n=3).  At 3 months, average improvement from baseline was 9.5 points (n=2).  All three patients had viral symptoms 3-4 days after infusion (3 with fever, 1 with emesis), and two were hospitalized for 1-2 days for observation. All had thrombocytopenia (average of 42.67 K/uL) at 7 days, with recovery by 14 days.  All required prolonged steroid courses due to elevated liver enzymes, but with normal overall liver function.   

Early clinical experience with onasemnogene in SMA type 2 patients suggests efficacy, although data on those not treated prior with nusinersen is necessary to be able to fully determine effect.  Side effects including acute viral syndrome, thrombocytopenia, and transaminitis may be higher than those reported in younger, smaller patients. 

Authors/Disclosures
Susan Matesanz, MD
PRESENTER 		Dr. Matesanz has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Matesanz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Matesanz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Atamyo . The institution of Dr. Matesanz has received research support from Sarepta . The institution of Dr. Matesanz has received research support from Pfizer. The institution of Dr. Matesanz has received research support from Genentech/Roche.
Elizabeth A. Kichula, MD, PhD (Children'S Hospital of Philadelphia) No disclosure on file