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Abstract Details

Nusinersen Dosing Patterns in Patients With Spinal Muscular Atrophy Type 1 in the United States: Findings From a Retrospective Claims Database Analysis
Child Neurology and Developmental Neurology
Child Neurology and Developmental Neurology Posters (7:00 AM-5:00 PM)
074
To investigate dosing patterns in patients with spinal muscular atrophy (SMA) type 1 treated with nusinersen in the United States.
SMA type 1 is a devastating neuromuscular disorder caused by deletion or mutation of the survival motor neuron-1 (SMN1) gene; without intervention, motor function deteriorates rapidly, typically resulting in death by 2 years of age. Nusinersen, an antisense oligonucleotide, was approved by the FDA in 2016, and enhances expression of functional SMN protein from the survival motor neuron-2 (SMN2) backup gene. The indicated dosing regimen requires ongoing intrathecal injections every 4 months following the first 4 injections, administered over a 2-month loading phase.

Using Symphony Health’s Integrated Dataverse®, we sought patients diagnosed with SMA type 1 after 23Dec2016 who subsequently initiated nusinersen therapy, and followed until last observed clinical activity or end of data availability (30Nov2019).
We identified 62 patients with SMA type 1 who received a mean of 3.7 doses (median: 3.0) over a mean follow-up of 15.7 months (median: 15.4). Patients received on average 58.4% (median: 60.0%) of the doses expected based on recommended dosing schedule (allowing for a 2-week grace period), and the majority of patients (49/62; 79.0%) had received ≥1 fewer dose than expected. Among patients with ≥2 months of follow-up, the loading phase (≥4 doses) was completed for 27/61 (44.3%). Loading was completed over a mean duration of 6.7 months (median: 5.6), with 11/27 (40.7%) completing it within ≤2 months, and 15/27 (55.6%) requiring ≥5 months. Among patients with ≥1 maintenance dose (n=20), mean interval between maintenance doses was 3.8 months (median: 4.0).
These results indicate frequent deviations from the recommended dosing schedule in patients with SMA type 1 receiving nusinersen. Treatment delays in these patients may result in loss of motor neurons and disease progression.
Authors/Disclosures

PRESENTER 		No disclosure on file
No disclosure on file
Martin Cloutier, MD No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
Mattias Bischof, MD Dr. Bischof has received personal compensation for serving as an employee of Novartis Gene Therapy. Dr. Bischof has received stock or an ownership interest from Novartis Gene Therapy.
No disclosure on file
Lydia Shenouda Lydia Shenouda has received personal compensation for serving as an employee of Novartis Gene Therapies. Lydia Shenouda has received stock or an ownership interest from Novartis Gene Therapies.