Trusted Resources: Education
Scientific literature and patient education texts
Muscle Impairment in MRI Affect Variability in Treatment Response to Nusinersen in Patients With Spinal Muscular Atrophy Type 2 and 3: A Retrospective Cohort Study
source: Brain & Development
year: 2022
authors: Yuko Shimizu-Motohashi, Emiko Chiba, Katsuhiro Mizuno, Hiroyuki Yajima, Akihiko Ishiyama, Eri Takeshita, Noriko Sato, Mari Oba, Masayuki Sasaki, Shuichi Ito, Hirofumi Komaki
summary/abstract:Background:
Real-world data have shown variability in treatment responses to nusinersen in spinal muscular atrophy (SMA). We investigated whether the magnitude of muscle impairment assessed by magnetic resonance imaging (MRI) at baseline can predict the treatment response.
Methods:
We retrospectively assessed the clinical data in relevance to the thigh and pelvic MRI taken before the nusinersen treatment. A total of 16 patients with SMA types 2 and 3 (age = mean [SD]; 9.2 [4.6] year) receiving nusinersen treatment were enrolled. The T1-weighted MRI images of the pelvis and thigh were scored for muscle fatty infiltration and atrophy. The minimally clinically important difference (MCID) was considered as gaining at least 3 points of Hammersmith Functional Motor Scale-Expanded (HFMSE) from baseline.
Results:
Of these 16 individuals, 14 had been treated for at least 15 months with baseline data. At 15 months, seven individuals obtained MCID in HFMSE. Baseline muscle MRI score could not differentiate the two groups; however, individuals who obtained MCID had significantly less severe scoliosis. In addition, there was a significant and negative relationship between baseline MRI score and the change of score in HFMSE after 15 months of treatment. Further, baseline Cobb angle along with MRI score also indicated the correlation to the degree of change in motor function.
Conclusion:
The degree of muscle damage may confer the variability in response to nusinersen in SMA types 2 and 3. Muscle MRI score along with the severity of scoliosis assessed at baseline may help to predict the motor function change.
DOI: 10.1016/j.braindev.2022.11.002
read more full text
Related Content
-
Organotypic Spinal Cord Cultures: An in Vitro 3D Model to Preliminary Screen Treatments for Spinal Muscular AtrophySpinal muscular atrophy (SMA) is a sever...
-
Intrathecal Administration of Nusinersen Using the Ommaya Reservoir in an Adult With 5q-Related Spinal Muscular Atro...Spinal muscular atrophy (SMA) is a hered...
-
DNA From Saliva Has Potential for Detecting SMAA noninvasive method using dried saliva ...
-
Let’s Start From the BeginningScarlet was born at 40 weeks and 1 day. ...
-
Jessica Nance, MD, MSDr. Jessica Nance, treats pediatric pati...
-
Managing Pregnancy in a Spinal Muscular Atrophy Type III Patient in Indonesia: A Case ReportSpinal muscular atrophy (SMA) is a genet...
-
Looking After Yourself if you’re an Adult Who Has Had a Recent Diagnosis of SMASMA Type 4, the more common form of adul...