Scientific Poster Session III - Original Research

Original Research
  Monday, November 13, 2023
  01:00 PM–02:30 PM

Abstract

Introduction: Nusinersen (NUS), an antisense oligonucleotide approved for treatment of spinal muscular atrophy (SMA) across all ages, is administered intrathecally at a dose of 12 mg. Limited evidence exists on real-world adherence for NUS stratified by age bands.

Research Question or Hypothesis: Real-world NUS adherence does not vary by age bands.

Study Design: Retrospective database study of patients on NUS stratified by age.

Methods: Patients on NUS were identified using anonymized administrative claims from Komodo’s Healthcare Map™ 1/1/2017-9/30/2022. Included were patients likely to have complete information on date of NUS initiation and continuous enrollment 12 months prior to first NUS record (index date). Using number (%) of on-time doses and distribution of inter-dose intervals, adherence was measured for loading and maintenance phases among those with =2 doses. Adherence was evaluated for the overall population and by age bands (in years): Infants (0 - <2); Pediatrics (Peds) (2 - <6; 6 - <18); Adults (18 - <25; 25 - <50; =50).

Results: Overall, 428 individuals receiving NUS were identified; 52% were female with a median age of 16 years. Two percent were infants, 51% pediatric, and 47% adult. Payor mix was Medicaid (49%), Commercial (42%) and Medicare (9%). The majority of NUS doses were received on-time (%): Overall (86); Infants (74); Peds (88;86); Adults (86;85;81). Similar results were seen for loading and maintenance phases separately. Calculated inter-dose intervals by age aligned with the expected dosing schedule of NUS, such as 4 months for maintenance doses. Median [quartile (Q)1, Q3] days from previous dose for the maintenance phase were: Overall (125 [118, 133]); Infant (114 [111, 125]); Peds (125 [119, 132]; 124 [118, 132]); Adults (124 [113, 132] ;126 [119, 135];126 [121,151]).

Conclusion: NUS doses, regardless of age, were consistent with the recommended dosing schedule.

Presenting Author

Paula Alvarez RPh, MBA, MS
Biogen

Authors

Kori Hack PharmD
Queenie Ip PharmD, PhD
Nicole Johnson BS
Joseph Neenan BS
Bridget Neville MPH
Tanner Odom PharmD
Andi Chin BS
Zoe Wu MA
Bora Youn PhD