Individual results may vary based on several factors,
including severity of disease, initiation of treatment, and
duration of therapy.
CHERISH was a 15-month, phase 3, sham-controlled pivotal trial that evaluated changes in motor function as measured by HFMSE. Results showed patients ages 2-9 years old with later-onset SMA treated with SPINRAZA (n=84) experienced a 3.9-point increase in HFMSE score from a baseline of 22.4, compared to a 1.0-point decrease from a baseline of 19.9 in the sham-treated group (n=42).
Study design: An independent, prospective, multicenter, observational cohort study
Study duration: Up to 14 months. Assessments made at 6, 10, and 14 months
Participants: 139 patients with genetically confirmed 5q later-onset SMA aged 16-65
Primary endpoint: Change from baseline in motor function measured by HFMSE at 6, 10, and 14 months. Patients with missing baseline HFMSE scores were excluded from these analyses
Secondary endpoints: Change from baseline in upper limb and walking ability measured by RULM and 6MWT at 6, 10, and 14 months
Study limitations: No control group; observational design. Study powered on primary endpoint only. Statistics for other endpoints are descriptive only
Safety: Adverse events (AEs) were generally consistent with those reported in the SPINRAZA clinical trials
HFMSE=Hammersmith Functional Motor Scale—Expanded.
SPINRAZA significantly increased mean HFMSE scores compared to baseline.3
139 patients completed an assessment at 6 months, 105 at 10 months, and 61 at 14 months. Patients not included at 10-month and 14-month assessments were those who had not reached the assessment time point (30 at month 10, 44 at month 14), were missing baseline or assessment values (15, 13, and 4 at month 6, 10, and 14), withdrew due an adverse reaction or procedure-related event (n=2), or withdrew due to consent (n=2). Greater improvement of motor function was correlated with lower severity of disease at baseline. Fourteen of 124 patients (11%) showed worsening motor function under treatment as measured by HFMSE.
≥3-point increase is considered clinically meaningful for HFMSE. A 1–2-point increase could be considered a positive outcome in a disease where natural history has shown a progressive decline.4,5
RULM=Revised Upper Limb Module.
SPINRAZA improved mean upper limb function and walking distances compared to baseline at every study time point.3
After 6 months of treatment:
6MWT=6-Minute Walk Test.
A 30-meter increase is considered clinically meaningful, and a 45-meter increase has been shown to greatly improve daily life challenges.6
*Number of patients in each group at 6, 10, and 14 months, respectively, was as follows: Type 2: 45, 30, and 20; Type 3: 77, 60, and 37; ambulant: 46, 35, and 23; nonambulant: 78, 57, and 34; baseline HFMSE ≥35: 39, 33, and 22; baseline HFMSE <35: 85, 59, and 35; previous spondylodesis: 28, 18, and 14; no previous spondylodesis: 96, 74, and 43.