Delays in Physiotherapy Don’t Limit Spinraza’s Potential, Study Suggests
Treatment with Spinraza (nusinersen) stabilized or improved motor abilities in children and adults with spinal muscular atrophy (SMA) regardless of interruptions in physiotherapy due to the COVID-19 pandemic, a small and single-site study suggests.
These findings add to the large body of evidence supporting Spinraza’s benefits in SMA patients, and they indicate that interrupting or delaying treatment is likely to be the more harmful action in terms of patient health and motor function, the researchers noted.
The findings, “Effect of Nusinersen treatment on motor functions in children and adolescents with spinal muscular atrophy who gave a break to physiotherapy during COVID-19 pandemic,” were published in the form of a letter in the Turkish Journal of Physical Medicine and Rehabilitation.
SMA is caused by low to no levels of the SMN protein, leading to the progressive loss of motor neurons, the specialized nerve cells that control voluntary movement, and resulting in muscle weakness and wasting.
As such, physiotherapy, a movement-based therapy, is typically recommended for this patient population to improve posture, prevent joint immobility, and slow muscle weakness and atrophy.
Biogen’s Spinraza — which works by increasing SMN levels — was the first approved disease-modifying therapy for children and adults with all SMA types. It is administered directly into the spinal canal at a recommended regimen of four initial loading doses, followed by maintenance treatment every four months.
Spinraza’s benefits on motor function independent of physiotherapy, however, remain unclear.
A team of researchers in Istanbul, Turkey, evaluated whether physiotherapy delays or interruptions due to the COVID-19 pandemic affected Spinraza’s ability to aid motor function in 48 children and adults with SMA followed at their neuromuscular center.
Use of Spinraza began at that center in 2017, and “physiotherapy given by a physiotherapist in a medical center … has always been included in the treatment protocol,” the researchers wrote.
While SMA patients were considered to be at higher risk of severe COVID-19 infection, the center maintained Spinraza treatment throughout the pandemic “due to its proven positive effects on prognosis and the devastating effects of lack of treatment,” they added.
But many of its patients or caregivers preferred physiotherapy to be given at the home, “either with solely parental involvement or in combination with healthcare professional support,” they wrote.
Notably, all these patients — 22 with SMA type 1, 14 with type 2, and 12 with type 3 — received home-based physiotherapy without professional support between March 2020 and May 2021. Among these SMA types, type 1 is the most severe and type 3 the least severe.
Patients’ mean age was nearly 5.5 years (65 months), ranging from newborns to 18.5 years old. Prior to March 2020, type 1 patients had received a mean of five (range, 0 to 11) doses of Spinraza, while those with types 2 or 3 had been treated with a mean of two doses (range, two to 13).
Fifteen patients (31.2%) started on Spinraza treatment during the pandemic.
Motor function was assessed with the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders Scale for type 1 patients and with the Hammersmith Functional Motor Scale for type 2 and 3 patients. In both measures, higher scores indicate better motor function.
Researchers compared each patient’s score taken closest to the pandemic’s March 2020 start (collected between December 2019 and August 2020) with scores taken just before May 2021.
Results showed that motor function scores for all patients rose or stabilized during the months when effective and professionally given physiotherapy was interrupted, but Spinraza treatment continued as scheduled.
SMA type 1 patients showed a mean score increase of six points, while scores for types 2s and 3s rose by a mean of four points. This difference may be related to the higher number of Spinraza doses given to type 1 patients before the pandemic, the researchers noted.
Importantly, no patient had a decline in motor function scores that would make them ineligible for Spinraza treatment under local government guidelines.
These findings suggest that “timely administered, Spinraza treatment has noticeable positive effects on motor functions independent of physiotherapy,” the researchers wrote.
“Avoidance of [Spinraza] treatment due to pandemic concerns is likely to have more devastating effects on the patients’ motor functions,” they added.