scholarly journals Patients With Becker Muscular Dystrophy Have Severe Paraspinal Muscle Involvement

2021 ◽  
Vol 12 ◽  
Author(s):  
Aisha M. Sheikh ◽  
Karen Rudolf ◽  
Josefine de Stricker Borch ◽  
Tahmina Khawajazada ◽  
Nanna Witting ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Becker Muscular Dystrophy ◽  
Erector Spinae ◽  
Paraspinal Muscle ◽  
Paraspinal Muscles ◽  
Muscle Mri ◽  
Cross Sectional ◽  
Muscle Involvement ◽  
Fat Replacement ◽  
Fat Fraction

Introduction: Paraspinal muscles are important for gross motor functions. Impairment of these muscles can lead to poor postural control and ambulation difficulty. Little knowledge exists about the involvement of paraspinal muscles in Becker muscular dystrophy.Objective: In this cross-sectional study, we investigated the involvement of paraspinal muscles with quantitative trunk strength measure and quantitative muscle MRI.Methods and Materials: Eighteen patients with Becker muscular dystrophy underwent trunk, hip, and thigh strength assessment using a Biodex dynamometer and an MRI Dixon scan. Fourteen age- and body mass index-matched healthy men were included for comparison.Results: Muscle fat fraction (FF) of the paraspinal muscles (multifidus and erector spinae) was higher in participants with Becker muscular dystrophy vs. healthy controls at all three examined spinal levels (C6, Th12, and L4/L5) (p < 0.05). There was a strong and inverse correlation between paraspinal muscle FF and trunk extension strength (ρ = −0.829, p < 0.001), gluteus maximus FF and hip extension strength (ρ = −0.701, p = 0.005), FF of the knee extensor muscles (quadriceps and sartorius) and knee extension strength (ρ = −0.842, p < 0.001), and FF of the knee flexor muscles (hamstring muscles) and knee flexion strength (ρ = −0.864, p < 0.001). Fat fraction of the paraspinal muscles also correlated with muscle FF of the thigh muscles and lower leg muscles.Conclusion: In conclusion, patients with Becker muscular dystrophy demonstrate severe paraspinal muscular involvement indicated by low back extension strength and high levels of fat replacement, which parallel involvement of lower limb muscles. Assessment of paraspinal muscle strength and fat replacement may serve as a possible biomarker for both the clinical management and further study of the disease.

scholarly journals Selection Approach to Identify the Optimal Biomarker Using Quantitative Muscle MRI and Functional Assessments in Becker Muscular Dystrophy

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012233
Author(s):  
Nienke M. van de Velde ◽  
Melissa T. Hooijmans ◽  
Aashley S.D. Sardjoe Mishre ◽  
Kevin R. Keene ◽  
Zaida Koeks ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Disease Progression ◽  
Intraclass Correlation ◽  
Becker Muscular Dystrophy ◽  
Knee Extension ◽  
Muscle Mri ◽  
Cross Sectional ◽  
Functional Assessments ◽  
The Difference ◽  
Muscle Groups

Objective:To identify the best quantitative fat-water MRI biomarker for disease progression of leg muscles in Becker muscular dystrophy (BMD) by applying a stepwise approach based on standardized response mean (SRM) over 24 months, correlations with baseline ambulatory tests and reproducibility.Methods:Dixon fat-water imaging was performed at baseline (n=24) and 24 months (n=20). Fat fractions (FF) were calculated for three center slices and the whole muscles for 19 muscles and six muscle groups. Contractile cross sectional area (cCSA) was obtained from the center slice. Functional assessments included knee extension and flexion force, and three ambulatory tests (North Star Ambulatory Assessment (NSAA), 10-meter run, six-minute walking test). MR parameters were selected using SRM (≥0.8) and correlation with all ambulatory tests (rho≤-0.8). Parameters were evaluated based on intraclass correlation coefficient (ICC) and standard deviation (SD) of the difference. Sample sizes (SS) were calculated assuming 50% reduction in disease progression over 24 months in a clinical trial with 1:1 randomization.Results:Median whole muscle FF increased between 0.2-2.6% without consistent cCSA changes. High SRMs and strong functional correlations were found for eight FF but no cCSA parameters. All parameters showed excellent ICC (≥0.999) and similar SD of the inter-rater difference. Whole thigh three center slices FF was the best biomarker (SRM=1.04, correlations rho≤-0.81, ICC=1.00, SD=0.23%, SS=59) based on low SD and acquisition and analysis time.Conclusion:In BMD, median FF of all muscles increased over 24 months. Whole thigh three center slices FF reduced the SS by approximately 40% compared to NSAA.

scholarly journals Decision-Making And Selection Bias in Four Observational Studies on Duchenne and Becker Muscular Dystrophy

2020 ◽  
Vol 7 (4) ◽  
pp. 433-442
Author(s):  
Karin J. Naarding ◽  
Nathalie Doorenweerd ◽  
Zaïda Koeks ◽  
Ruben G.F. Hendriksen ◽  
Kinita A. Chotkan ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Travel Time ◽  
Selection Bias ◽  
Observational Studies ◽  
Brain Mri ◽  
Becker Muscular Dystrophy ◽  
Nonparametric Tests ◽  
Observational Research ◽  
Muscle Mri ◽  
Cross Sectional

Background: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscular dystrophy (BMD), but recruitment for observational studies can be challenging. Objective: We reviewed reasons why patients or caregivers declined participation, and compared characteristics of participants and non-participants to assess possible selection bias in four observational studies, three on DMD and one on BMD. Methods: Three pediatric DMD studies focused on cross-sectional cognitive function and brain MRI (DMDbrain, n = 35 and DMDperfusion, n = 12), and on longitudinal upper extremity function and muscle MRI (DMDarm, n = 22). One adult BMD study assessed longitudinal functioning (n = 36). Considerations for non-participation were retrospectively reviewed from screening logs. Age, travel-time, DMD gene mutations and age at loss of ambulation (DMDarm and BMD study only), of participants and non-participants were derived from the Dutch Dystrophinopathy Database and compared using nonparametric tests (p < 0.05). Results: The perceived burden of the protocol (38.2%), use of MRI (30.4%), and travel-time to the study site (19.1%) were the most frequently reported considerations for non-participation. Only few patients reported lack of personal gain (0.0– 5.9%). Overall, participating patients were representative for the studied sub-populations, except for a younger age of DMDarm study participants and a complete lack of participants with a mutation beyond exon 63. Conclusion: Optimizing patient involvement in protocol design, improving MRI experiences, and integrating research into clinics are important factors to decrease burden and facilitate participation. Nationwide registries are essential to compare participants and non-participants and ensure representative observational research. Specific effort is needed to include patients with distal mutations in cognitive studies.

scholarly journals Quantitative muscle MRI biomarkers in Duchenne muscular dystrophy: cross-sectional correlations with age and functional tests

2021 ◽  
Author(s):  
Sarah P Sherlock ◽  
Yao Zhang ◽  
Michael Binks ◽  
Shannon Marraffino
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
T2 Mapping ◽  
Quantitative Mri ◽  
Thigh Muscle ◽  
Ambulatory Assessment ◽  
Functional Tests ◽  
Muscle Mri ◽  
Cross Sectional ◽  
Fat Fraction

Aim: Using baseline data from a clinical trial of domagrozumab in Duchenne muscular dystrophy, we evaluated the correlation between functional measures and quantitative MRI assessments of thigh muscle. Patients & methods: Analysis included timed functional tests, knee extension/strength and North Star Ambulatory Assessment. Patients (n = 120) underwent examinations of one thigh, with MRI sequences to enable measurements of muscle volume (MV), MV index, mean T2 relaxation time via T2-mapping and fat fraction. Results: MV was moderately correlated with strength assessments. MV index, fat fraction and T2-mapping measures had moderate correlations ( r ∼ 0.5) to all functional tests, North Star Ambulatory Assessment and age. Conclusion: The moderate correlation between functional tests, age and baseline MRI measures supports MRI as a biomarker in Duchenne muscular dystrophy clinical trials. Trial registration: ClinicalTrials.gov , NCT02310763 ; registered 4 November 2014.

scholarly journals Quantitative Muscle MRI as Outcome Measure in Patients With Becker Muscular Dystrophy—A 1-Year Follow-Up Study

2021 ◽  
Vol 11 ◽  
Author(s):  
Aisha M. Sheikh ◽  
Karen Rudolf ◽  
Nanna Witting ◽  
John Vissing
Keyword(s):  
Muscular Dystrophy ◽  
Muscle Strength ◽  
Lower Limb ◽  
Becker Muscular Dystrophy ◽  
Quantitative Mri ◽  
Erector Spinae ◽  
Muscle Mri ◽  
Follow Up Study ◽  
Lower Limb Muscles

Introduction: With the advent of emerging molecular therapies for muscular dystrophies, the need for knowledge about natural history course of such diseases is of utmost importance in the preparation for future trials. However, for Becker muscular dystrophy such knowledge is scarce.Objective: In this 1-year follow-up study, we examined disease progression in Becker muscular dystrophy by monitoring changes in MRI-assessed muscle fat fraction (FF) in axial and lower limb muscles and quantitative muscle strength of axial muscles.Methods and Materials: Sixteen patients with Becker muscular dystrophy were investigated by (1) muscle strength of the trunk using a Biodex dynamometer and (2) Dixon muscle MRI of paraspinal and lower limb muscles. Quantitative MRI data was analyzed in two parts: The first part consisted of all participants (N = 16). The second analysis assessed two separate groups comprising lesser affected participants (N = 5) and more severely affected patients (n = 11).Results: Trunk extension and flexion strength remained stable from baseline to follow-up. MRI did not show any significant increase in muscle FF % from baseline to follow-up in all patients, except for multifidus at the spinal level T12 (p = 0.01). However, when we analyzed the two subgroups, according to disease severity, FF% increased in the lesser severely affected group at L4/L5 erector spinae (p = 0.047), sartorius (p = 0.028), gracilis (p = 0.009), tibialis anterior (p = 0.047), peroneals (p = 0.028), and gastrocnemius medialis (p = 0.009), while the severely affected group only increased significantly at T12 multifidus (p = 0.028) and T12 erector spinae (p = 0.011). No difference in muscle strength was observed in the two subgroups.Conclusion: Our results add to the existing knowledge about the natural rate of disease progression in BMD. As quantitative MRI was able to identify changes where strength assessment was not, MRI could be a strong biomarker for change in BMD. However, our findings show that it is important to stratify patients with BMD according to phenotype for future clinical trials.

scholarly journals Radiological Assessment of Postoperative Paraspinal Muscle Changes After Lumbar Interbody Fusion With or Without Minimally Invasive Techniques

2021 ◽  
pp. 219256822199479
Author(s):  
Keigo Kameyama ◽  
Tetsuro Ohba ◽  
Tomoka Endo ◽  
Marina Katsu ◽  
Fujita Koji ◽  
...  
Keyword(s):  
Minimally Invasive ◽  
Interbody Fusion ◽  
Cross Sectional Area ◽  
Paraspinal Muscle ◽  
Lumbar Interbody Fusion ◽  
Sectional Area ◽  
Paraspinal Muscles ◽  
Cross Sectional ◽  
Muscle Density ◽  
Muscle Changes

Study Design: Retrospective cohort study. Background: Percutaneous pedicle screws (PPS) have the advantage of being able to better preserve the paraspinal muscles when compared with a traditional open approach. However, the nature of changes in postoperative paraspinal muscle after damage by lumbar fusion surgery has remained largely unknown. It is clinically important to clarify and compare changes in paraspinal muscles after the various surgeries. Objective: (1) To determine postoperative changes of muscle density and cross-sectional area using computed tomography (CT), and (2) to compare paraspinal muscle changes after posterior lumbar interbody fusion (PLIF) with traditional open approaches and minimally invasive lateral lumbar interbody fusions (LLIF) with PPS. Methods: We included data from 39 consecutive female patients who underwent open PLIF and 23 consecutive patients who underwent single-staged treatment with LLIF followed by posterior PPS fixation at a single level (L4-5). All patients underwent preoperative, 6 months postoperative, and 1-year postoperative CT imaging. Measurements of the cross-sectional area (CSA) and muscle densities of paraspinal muscles were obtained using regions of interest defined by manual tracing. Results: We did not find any decrease of CSA in any paraspinal muscles. We did find a decrease of muscle density in the multifidus at 1 year after surgery in patients in the PILF group, but not in those in LLIF/PPS group. Conclusions: One year after surgery, a significant postoperative decrease of muscle density of the multifidi was observed only in patients who underwent open PLIF, but not in those who underwent LLIF/PPS.

Association Between Deep Posterior Cervical Paraspinal Muscle Morphology and Clinical Features in Patients With Cervical Ossification of the Posterior Longitudinal Ligament

2021 ◽  
pp. 219256822198965
Author(s):  
Toru Doi ◽  
Nozomu Ohtomo ◽  
Fumihiko Oguchi ◽  
Keiichiro Tozawa ◽  
Hiroyuki Nakarai ◽  
...  
Keyword(s):  
Clinical Features ◽  
Fatty Infiltration ◽  
Neck Disability Index ◽  
Posterior Longitudinal Ligament ◽  
Paraspinal Muscle ◽  
Paraspinal Muscles ◽  
Muscle Area ◽  
Cross Sectional ◽  
Study Results ◽  
Neck Disability

Study Design: A retrospective observational study. Objective: To clarify the association of the paraspinal muscle area and composition with clinical features in patients with cervical ossification of the posterior longitudinal ligament (OPLL). Methods: Consecutive patients with cervical OPLL who underwent cervical magnetic resonance imaging (MRI) before surgery were reviewed. The cross-sectional area (CSA) and fatty infiltration ratio (FI%) of deep posterior cervical paraspinal muscles (multifidus [MF] and semispinalis cervicis [SCer]) were examined. We assessed the association of paraspinal muscle measurements with the clinical characteristics and clinical outcomes, such as Neck Disability Index (NDI) score. Moreover, we divided the patients into 2 groups according to the extent of the ossified lesion (segmental and localized [OPLL-SL] and continuous and mixed [OPLL-CM] groups) and compared these variables between the 2 groups. Results: 49 patients with cervical OPLL were enrolled in this study. The FI% of the paraspinal muscles was significantly associated with the number of vertebrae ( ρ = 0.283, p = 0.049) or maximum occupancy ratio of OPLL ( ρ = 0.397, p = 0.005). The comparative study results indicated that the NDI score was significantly worse (OPLL-SL, 22.9 ± 13.7 vs. OPLL-CM, 34.4 ± 13.7) and FI% of SCer higher (OPLL-SL, 9.1 ± 1.7% vs. OPLL-CM, 11.1 ± 3.7%) in the OPLL-CM group than those in the OPLL-SL group. Conclusions: Our results suggest that OPLL severity may be associated with fatty infiltration of deep posterior cervical paraspinal muscles, which could affect neck disability in patients with cervical OPLL.

Comparison of Paraspinal Muscle Degeneration and Decompression Effect Between Conventional Open and Minimal Invasive Approaches for Posterior Lumbar Spine Surgery: a prospective cohort study.

2020 ◽  
Author(s):  
CHEN-JU FU ◽  
Wen-Chien Chen ◽  
Meng-Ling Lu ◽  
Chih-Hsiu Cheng ◽  
Chi-Chien Niu
Keyword(s):  
Transforaminal Lumbar Interbody Fusion ◽  
Fatty Degeneration ◽  
Minimal Invasive ◽  
Erector Spinae ◽  
Lumbar Spine Surgery ◽  
Paraspinal Muscles ◽  
Adjacent Level ◽  
Cross Sectional ◽  
Erector Spinae Muscle ◽  
Dural Sac

Abstract Background Posterior Instrumented Transforaminal Lumbar Interbody Fusion (TLIF) is used to treat spinal stenosis. Minimally invasive surgery (MIS) can cause less muscle injury than conventional open surgery (COS) The purpose of this study was to compare the degree of postoperative fatty degeneration in the paraspinal muscles and the spinal decompression between COS and MIS based on magnetic resonance imaging (MRI). Methods Forty-six patients received TLIF (21 COS, 25 MIS) from February 2016 to January 2017. Lumbar MRI was performed within 3 months before surgery and 1 year after surgery. The postoperative muscle-fat-index (MFI) change of the paraspinal muscles (multifidus and erector spinae) and the dural sac cross-sectional area (DSCAS) change were compared between the 2 groups. Results The average MFI change at L2-S1 erector spinae muscle was significantly greater in the COS group (27.37 ± 21.37% vs. 14.13 ± 19.19%, P = 0.044). A significant MFI change difference between the COS and MIS group was also found in the erector spinae muscle at the caudal adjacent level (54.47 ± 37.95% vs. 23.60 ± 31.59%, P = 0.016). No significant differences at the operated and cranial adjacent level were found. DSCSA improvement after surgery was significantly greater in the COS group (128.15 ± 39.83 mm2 vs. 78.15 ± 38.5 mm2, P = 0.0005) Conclusion COS is associated with more prominent fatty degeneration of the paraspinal muscles. Statically significant post-operative MFI change was only noted in erector spinae muscle at caudal adjacent level and L2-S1 mean global level. COS produces a greater decompressive effect than MIS.

scholarly journals A Cross-Sectional Study of School Experiences of Boys with Duchenne and Becker Muscular Dystrophy

2016 ◽  
Vol 35 (2) ◽  
pp. 1-22 ◽  
Author(s):  
Aida Soim ◽  
Molly Lamb ◽  
Kimberly Campbell ◽  
Shree Pandya ◽  
Holly Peay ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Speech Therapy ◽  
Becker Muscular Dystrophy ◽  
Educational Interventions ◽  
Cross Sectional Study ◽  
Research Network ◽  
Sectional Study ◽  
Cross Sectional ◽  
Resource Room ◽  
Instructional Assistant

The objectives of this study were to investigate types of supportive school services received and factors related to provision of these services. We conducted a cross-sectional study to describe the school experience of males with Duchenne and Becker muscular dystrophies. Study subjects were identified through the Muscular Dystrophy Surveillance, Tracking, and Research Network. Non-ambulatory males with Duchenne muscular dystrophy (DMD) were significantly more likely to use an instructional assistant and resource room support when compared to ambulant males with DMD at the time of the caregiver interview. Males with DMD who received occupational therapy were more likely to use an instructional assistant, while those who received speech therapy were more likely to repeat a grade, use an instructional assistant, and receive resource room support. Males with DMD whose primary caregivers had less than 12 years of education were more likely to use an instructional assistant and resource room support. Non-ambulatory males with DMD should continue receiving appropriate school accommodations so they can keep pace with their physical demands. Males with DMD with speech deficits should be considered for various educational interventions.

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