SMA THERAPIES I: P.176Experience using Spinraza to treat adults with spinal muscular atrophy

Abstract

Evaluate effectiveness of spinraza clinical protocol in adults with SMA and determine post-treatment functional changes. Natural history data of functional and clinical measures prospectively collected from adults in a SMA network, PNCRN, were compared with spinraza treated adults. Visits included medical & genetic history, establishing standards of care, evaluating anatomic and radiologic findings, and assessing insurance. A minimal data set included at least one of the following: modified CHOP-INTEND, RULM, HFMSE, TUG, 6MWT. We also monitored pulmonary function, limb strength, patient reports, safety labs and adverse events. Natural history was collected from 170 evaluations of 57 untreated adults. Assessments included 39 patients wanting spinraza, 27 treated. None were denied treatment due to insurance, but others have not yet received treatment due to lack of access via fluoroscopy (n=5), and patient choice (n=1). Treated individuals were: 18-65 years old, non-ambulatory (n=20), male (n=16), requiring day and night ventilatory support (n=7), and spinal fusion (n=8). Attestations of qualitative improvements were frequent (85%) but were not reflected in the spinal muscular atrophy functional rating scale (SMAFRS). Baseline measures (median and ranges) included for ambulatory (n=7): 6MWT (342.7m, 69.2-466.0m), TUG (9.9s, 8.4-44.2s) and for non-Ambulatory RULM (n=14) (6, 0-37), FVC (n=12) (1.6L, 0.28-3.8L);(%predicted 40%, 9-90%)). Spinraza has been well tolerated and improvement trends are emerging in multiple measures; data continue to be collected. Analyses of clinical outcomes, to be presented, will define Spinraza efficacy for adults with SMA.