Regular Physical Therapy Linked to Better Results in Spinraza Study
Undergoing physical therapy nearly every day while on Spinraza (nusinersen) leads to significantly greater motor improvements than occasional sessions for children and adolescents with spinal muscular atrophy (SMA), a small study suggests.
These benefits — detected in patients with types 1, 2, and 3 — were visible after six months of treatment.
The findings support the importance of physical therapy (also known as physiotherapy), particularly done routinely, for children and adolescents with all types of SMA to obtain the best outcomes when treated with Spinraza, the researchers noted.
The study, “Physical Therapy and Nusinersen Impact on Spinal Muscular Atrophy Rehabilitative Outcome,” was published in the journal Frontiers in Bioscience-Landmark.
SMA is caused by low to no levels of SMN, a protein essential for the health of motor neurons, the specialized nerve cells that control voluntary movement.
The progressive loss of motor neurons results in muscle weakness and wasting that mostly affects motor function, but in more severe types, can impair respiratory and swallowing abilities.
Three disease-modifying therapies for SMA — all working to increase SMN levels — have become available in the past decade. To achieve best outcomes, treatment must be administered as early as possible, before symptom onset.
In addition, physiotherapy is typically recommended for this patient population to improve posture, maintain range of motion, prevent joint immobility, and slow muscle weakness and atrophy.
Now, a team of researchers in Romania evaluated the effects of regular versus occasional physiotherapy in the motor skills of SMA patients treated with Biogen’s Spinraza, the first approved DMT for children and adults with all SMA types.
Spinraza is administered directly into the spinal canal at a recommended regimen of four initial loading doses, followed by maintenance treatment every four months.
The researchers retrospectively analyzed the motor function of 55 children and adolescents (29 girls and 26 boys), ranging in age from birth to 17 years, before and after six and 12 months of treatment with Spinraza. A total of 20 patients had the severe SMA type 1, 26 had the intermediate type 2, and nine had the milder type 3 disease.
All were treated by a multidisciplinary team at a single rehabilitation center. Since some children lived far from any facility or professional healthcare, not all could undergo daily physiotherapy.
A total of 39 children (18 with type 1, 16 with type 2, and five with type 3) underwent physiotherapy at least five days per week (physiotherapy group). The remaining 16 (two with type 1, 10 with type 2, and four with type 3) received less than one session per week (control group).
The control group also included patients who had spine surgery and needed prolonged bedrest.
Motor function in type 1 children was assessed with the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (typically used from 3 months to 4 years of age). The Hammersmith Functional Motor Scale Expanded (HFMSE) was completed for those with types 2 and 3 (used in those older than 2 years).
Results showed that after six months on Spinraza, patients undergoing daily physiotherapy had greater motor improvements than those in the control group — by nearly four times in type 1 children, by two-fold in those with type 2, and by 19 times in type 3 patients.
Motor gains were higher between six and 12 months of treatment in both groups, but were two to nearly five times greater in the physiotherapy group than the control group across all SMA types.
Contrary to the physiotherapy group, the HFMSE score remained unchanged in some children with types 2 and 3 who underwent physiotherapy less than once a week.
Further analyses showed that motor improvements reached statistical significance both after six and 12 months of Spinraza treatment among patients undergoing daily physiotherapy, and only at one year for those in the control group.
In addition, the overall physiotherapy group experienced nearly four times greater motor gains relative to the control group (12.7% vs. 3.2%), a difference that was statistically significant.
While more studies are needed to understand why type 1 patients showed better motor improvements than those with type 2, the team hypothesized that this may be related to the fact that the type 2 patients started Spinraza long after diagnosis, since the therapy was only available in the country in 2018.
These findings highlight that when combined with Spinraza, “physical therapy has provided superior results for those who receive it on a regular basis,” the researchers wrote.
Such motor improvements “include the correction of posture, reduction in stiffness, expansion of the range of motion and strengthening of muscles, thus allowing patients to do more movements and boosting their ability to perform everyday tasks,” the team wrote.
As such, “we strongly encourage regular [physiotherapy] sessions (at least 5 times a week–depending on the general health status of the patient) for all SMA patients,” the researchers added. Physiotherapy, “as part of the standards of care in SMA, should be recommended as one of the important and essential tools in SMA treatment.”
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