ZAPSMA (Zolgensma Access Program Spinal Muscular Atrophy): A Single Survey Analysis of the AveXis IND/MAP Program Patient Group (COMIRB # 19-0846)


Gene targeted therapies

Poster Number: 76


Kyle Kusmik, CCRC, BS, Jennifer M. Kwon, MD, Cory Sieburg, MSN RN, CPNP-AC APNP, Rebecca Scharf, MD, Emma Ciafaloni, MD, Megan Waldrop, MD, Elizabeth A. Kichula, MD, PhD, Chamindra Konersman, MD, Fawn Leigh, MD, Arpita Lakhotia, MD, Cristian Ionita, MD, Nancy Bass, MD, Aravindhan Veerapandiyan, MD, Crystal Proud, MD, Gyula Acsadi, MD PhD, Randal Richardson, MD, Matt Harmelink, Peter Karachunski, MD, Sharon Kim, MD, Julie Parsons, MD


1. Children's Hospital Colorado, 2. University of Wisconsin School of Medicine and Public Health, 3. University of Wisconsin, 4. University of Virginia Department of Pediatrics, 5. University of Rochester, 6. Nationwide Children's Hospital, 7. Children's Hospital of Philadelphia, Division of Neurology, 8. UCSD and Rady Children's Hospital, 9. Seattle Children's Hospital, 10. Norton Children's Hospital/ University of Louisville, 11. Yale School of Medicine, 12. Rainbow Babies and Children's Hospital/ University Hospitals of Cleveland, 13. Arkansas Children's Hospital, 14. Children's Hospital of The King's Daughters, 15. Connecticut Children's Medical Center, 16. Gillette Children's Hospita, 17. Medical College of Wisconsin, 18. University of Minnesota Medical Center/ University of Minnesota Masonic Children's Hospital, 19. Children's Hospital of Orange County, 20. Children's Hospital of Colorado, University of Colorado School of Medicine

Background –
Spinal Muscular Atrophy (SMA) is a progressive neurodegenerative disorder caused by a deletion/mutation of the SMN1 gene. Nusinersen and onasemnogene abeparvovec-xioi (zolgensma) are FDA approved therapies.

Objective –
We describe the use of dual therapies in a group of patients treated under AveXis Inc.’s managed access program.

Approach –
Data from 28 infants at 18 sites were obtained by surveys completed by treating physicians. Outcomes analyzed were treatment timelines, delivery strategies, motor milestones achieved, and adverse events.

Results –
The median age at survey was 9 (IQR= 8-12) months. Median age at zolgensma treatment was 7 (IQR= 4.4-9) months. Time from zolgensma treatment to data collection was 3.1 (IQR= 3-4.6) months. 57% of treated babies achieved head control, 43% rolled, and 32% were able to sit independently. These milestones were met 2 months later than WHO guidelines for unaffected babies. 14.3% of enrolled patients meet CDC/WHO age appropriate standards for head control, 17.9% meet these standards for rolling and 21.5% meet age appropriate standards for sitting. Adverse events were reported in 93%. Most common AEs included fever (64% n=18), respiratory events (43% n=12), and elevated transaminases (36% n=10) with 36% requiring hospitalization. Zolgensma administration occurred at a median of 34.5 (IQR= 8.5-84) days post nusinersen dose. 50% of zolgensma infusions were done in an inpatient unit with an observation period of 16 (IQR= 4.5-23.5) hours.

Conclusion –
Dual therapy in babies with SMA was well tolerated. Additional studies to determine if outcomes are improved in those treated with both nusinersen and zolgensma compared to mono therapy would help inform clinical decision making.