The German version of the KOOS-Child questionnaire (Knee injury and Osteoarthritis Outcome Score for children) shows a good to excellent internal consistency and a high test-retest reliability in children with knee problems.

PURPOSE: The Knee Injury Osteoarthritis Outcome Score for children (KOOS-Child) is a self-administered, valid and reliable questionnaire for children and adolescents with knee disorders such as Osgood Schlatter disease, anterior knee pain, and patella dislocation. This study aimed to cross-culturally adapt the German version of the KOOS-Child questionnaire and test the reliability in two groups of children, one treated conservatively and the other surgically. METHODS: A forward-backward translation of the original questionnaire into the German language was conducted. Children and adolescents between 10 and 18 years of age with knee disorders were included. Two groups were compared: sample one consisted of 24 participants with knee pain [20.8% boys; mean age = 13.4 (1.8) years treated conservatively. These participants completed the KOOS-Child questionnaire twice within two weeks to assess test-retest reliability. The second sample included 23 subjects (21.7% boys; mean age = 15.3 (1.9) years] treated surgically due to a knee disorder. They completed the questionnaire before surgery and six months postoperatively. Test-retest reliability and internal consistency were assessed using Spearman's rank correlation and Cronbach's alpha. RESULTS: All subscales showed a good to excellent internal consistency at both measurement points in both groups (conservatively treated group: a = 0.88-0.95; surgery group a = 0.80-0.91), with the exception of the subscale knee problems (conservatively treated: a = 0.60 and 0.52; surgery: α = 0.77 and 0.66). Test-retest reliability was between r = 0.85 and 0.94. CONCLUSION: The predominantly good to excellent internal consistency and the high test-retest reliability justifies the use of the German adaptation of the KOOS-Child questionnaire as a reliable multidimensional instrument for measuring health status and therapeutic effects in adolescents' knee disorders.

Effect of a physical activity intervention on lower body bone health in childhood cancer survivors: A randomized controlled trial (SURfit).

It remains controversial whether physical activity promotes bone health in childhood cancer survivors (CCS). We aimed to assess the effect of a one-year general exercise intervention on lower body bone parameters of CCS. CCS ≥16 years at enrollment, <16 years at diagnosis and ≥5 years in remission were identified from the national Childhood Cancer Registry. Participants randomized to the intervention group were asked to perform an additional ≥2.5 hours of intense physical activity/week, controls continued exercise as usual. Bone health was assessed as a secondary trial endpoint at baseline and after 12-months. We measured tibia bone mineral density (BMD) and morphology by peripheral quantitative computed tomography and lumbar spine, hip and femoral neck BMD by dual-energy x-ray absorptiometry. We performed intention-to-treat, per protocol, and an explorative subgroup analyses looking at low BMD using multiple linear regressions. One hundred fifty-one survivors (44% females, 7.5 ± 4.9 years at diagnosis, 30.4 ± 8.6 years at baseline) were included. Intention-to-treat analysis revealed no differences in changes between the intervention and control group. Per protocol analyses showed evidence for an improvement in femoral neck and trabecular BMD between 1.5% and 1.8% more in participants being compliant with the exercise program. Trabecular BMD increased 2.8% more in survivors of the intervention group with BMD z-score ≤-1 compared to those starting at z-score >-1. A nonstandardized personalized exercise programs might not be specific enough to promote bone health in CCS, although those compliant and those most in need may benefit. Future trials should include bone stimulating exercise programs targeting risk groups with reduced bone health and motivational features to maximize compliance.

A systematic review on conservative treatment options for OSGOOD-Schlatter disease.

OBJECTIVES: Osgood-Schlatter disease (OSD) is a sport- and growth-associated knee pathology with locally painful alterations around the tibial tuberosity apophysis. Up to 10% of adolescents are affected by OSD. Treatment is predominantly conservative. The aims of this systematic review are to comprehensively identify conservative treatment options for OSD, compare their effectiveness in selected outcomes, and describe potential research gaps. METHODS: A systematic literature search was conducted using CENTRAL, CINAHL, EMBASE, MEDLINE, and PEDro databases. In addition, ongoing and unpublished clinical studies, dissertations, and other grey literature on OSD were searched. We also systematically retrieved review articles for extraction of treatment recommendations. RESULTS: Of 767 identified studies, thirteen were included, comprising only two randomised controlled trials (RCTs). The included studies were published from 1948 to 2019 and included 747 patients with 937 affected knees. Study quality was poor to moderate. In addition to the studies, 15 review articles were included, among which the most prevalent treatment recommendations were compiled. CONCLUSION: Certain therapeutic approaches, such as stretching, have apparent efficacy, but no RCT comparing specific exercises with sham or usual-care treatment exists. Carefully controlled studies on well-described treatment approaches are needed to establish which conservative treatment options are most effective for patients with OSD.

The 6-minute walk test, motor function measure and quantitative thigh muscle MRI in Becker muscular dystrophy: A cross-sectional study.

Becker muscular dystrophy (BMD) has an incidence of 1 in 16 000 male births. This cross-sectional study investigated the relation between validated functional scores and quantitative MRI (qMRI) of thigh muscles in 20 ambulatory BMD patients, aged 18.3-60 years (mean 31.2; SD 11.1). Clinical assessments included the motor function measure (MFM) and its subscales, as well as timed function tests such as the 6-minute walk test (6MWT) and the timed 10-m run/walk test. Quantitative MRI of the thigh muscles included the mean fat fraction (MFF) using a 2-point Dixon (2-PD) technique, and transverse relaxation time (T2) measurements. The mean MFM value was 80.4%, SD 9.44 and the D1 subscore 54.5%, SD 19.9. The median 6MWT was 195m, IQR 160-330.2. The median 10-m run/walk test was 7.4 seconds, IQR 6.1-9.3. The mean fat fraction of the thigh muscles was 55.6%, SD 17.4%, mean T2 relaxation times of all muscles: 69.9 ms, SD 14.4. The flexors had the highest MFF and T2 relaxation times, followed by the extensors and the adductors. MFF and global T2 relaxation times were highly negatively correlated with the MFM total, D1-subscore and 6MWT, and positively correlated with the 10 m run/walk test time (p < 0.01). Age was not correlated with MFF, global T2 relaxation time or clinical assessments. Both MFF and T2 measures in the thigh muscle were well correlated with clinical function in BMD and may serve as a surrogate outcome measure in clinical trials.

Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study.

UNLABELLED: Altered neuronal nitric oxide synthase function in Duchenne muscular dystrophy leads to impaired mitochondrial function which is thought to be one cause of muscle damage in this disease. The study tested if increased intramuscular nitric oxide concentration can improve mitochondrial energy metabolism in Duchenne muscular dystrophy using a novel therapeutic approach through the combination of L-arginine with metformin. Five ambulatory, genetically confirmed Duchenne muscular dystrophy patients aged between 7­10 years were treated with L-arginine (3 x 2.5 g/d) and metformin (2 x 250 mg/d) for 16 weeks. Treatment effects were assessed using mitochondrial protein expression analysis in muscular biopsies, indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, and clinical scores of muscle performance. There were no serious side effects and no patient dropped out. Muscle biopsy results showed pre-treatment a significantly reduced mitochondrial protein expression and increased oxidative stress in Duchenne muscular dystrophy patients compared to controls. Post-treatment a significant elevation of proteins of the mitochondrial electron transport chain was observed as well as a reduction in oxidative stress. Treatment also decreased resting energy expenditure rates and energy substrate use shifted from carbohydrates to fatty acids. These changes were associated with improved clinical scores. In conclusion pharmacological stimulation of the nitric oxide pathway leads to improved mitochondria function and clinically a slowing of disease progression in Duchenne muscular dystrophy. This study shall lead to further development of this novel therapeutic approach into a real alternative for Duchenne muscular dystrophy patients. TRIAL REGISTRATION: ClinicalTrials.gov NCT02516085.

Quantitative muscle MRI: A powerful surrogate outcome measure in Duchenne muscular dystrophy.

In muscular dystrophies quantitative muscle MRI (qMRI) detects disease progression more sensitively than clinical scores. This prospective one year observational study compared qMRI with clinical scores in Duchenne muscular dystrophy (DMD) to investigate if qMRI can serve as a surrogate outcome measure in clinical trials. In 20 DMD patients the motor function measure (MFM) total and subscores (D1-D3) were done for physical examination, and the fat fraction (MFF) of thigh muscle qMRI was obtained using the two-point Dixon method. Effect sizes (ES) were calculated for all measures. Sample size estimation (SS) was done modelling assumed treatment effects. Ambulant patients <7 years at inclusion improved in the MFM total and D1 score (ES 1.1 and 1.0). Ambulant patients >7 years (highest ES in the MFM D1 subscore (1.2)), and non-ambulant patients (highest ES in the total MFM score (0.7)) worsened. In comparison the ES of QMRI was much larger, e.g. SS estimations for qMRI data were up to 17 fold smaller compared to the MFM total score and up to 7 fold to the D1 subscore, respectively. QMRI shows pathophysiological changes in DMD and might serve as a surrogate outcome measure in clinical trials.