Effectiveness of pharmacological treatments in Duchenne muscular dystrophy: a protocol for a systematic review and meta-analysis

Pascual‐Morena, Carlos; Martínez‐Vizcaíno, Vicente; Álvarez‐Bueno, Celia; Fernández-Rodríguez, Rubén; Jiménez‐López, Estela; Torres‐Costoso, Ana; Cavero‐Redondo, Iván

doi:10.1136/bmjopen-2019-029341

Cited by 7 publications

(4 citation statements)

References 33 publications

(32 reference statements)

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“…Later, it occurs loss of muscular strength and ability to ambulate. Other common DMD complications include scoliosis, heart failure, respiratory insufficiency, fractures of long bones, and vertebrae due to osteoporosis [ 3 ]. Currently, glucocorticoids are the only disease-modifying therapeutic agent shown to improve short-term muscle strength [ 4 ].…”

Section: Introductionmentioning

confidence: 99%

Validity of bioelectrical impedance to estimate fat-free mass in boys with Duchenne muscular dystrophy

et al. 2020

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The evaluation of fat-free mass (FFM) in patients with Duchenne muscular dystrophy (DMD) is useful to investigate disease progression and therapeutic efficacy. This study aimed to validate the Bioelectrical impedance (BIA) method compared with the dual-energy X-ray absorptiometry (DXA) for estimating the %FFM in boys with DMD. This is a cross-sectional study performed with children and adolescents diagnosed with DMD. Resistance and reactance were measured with a BIA analyzer, from which eight predictive equations estimated the %FFM. The %FFM was also determined by DXA and its used as a reference method. Pearson correlation test, coefficient of determination, the root-mean-square error, the interclass correlation coefficient, and linear regression analysis were performed between %FFM values obtained by BIA and DXA. The agreement between these values was verified with the Bland-Altman plot analysis. Forty-six boys aged from 5 to 20 years were enrolled in the study. All the equations showed a correlation between the %FFM estimated by BIA and determined by DXA (p < 0.05). The Bland-Altman method indicated that two equations have a significant bias (p < 0.05) and six equations showed no significant bias of %FFM (p > 0.05). However, one of them has high variation and wide limits of agreement. Five of eight %FFM predictive equations tested in DMD were accurate when compared with the DXA. It can be concluded that BIA is a validity method to evaluate patients with DMD.

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Section: Introductionmentioning

confidence: 99%

Validity of bioelectrical impedance to estimate fat-free mass in boys with Duchenne muscular dystrophy

et al. 2020

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“…Third, some studies may lack methodological description like the methods of randomization and lead to a poor methodological quality. [ 24 ]…”

Section: Discussionmentioning

confidence: 99%

Efficacy of Chinese herbal injections combined with fluoropyrimidine and oxaliplatin-based chemotherapy for advanced colorectal cancer

Wang

Zhang

et al. 2020

Medicine

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Background: Fluoropyrimidine combined with oxaliplatin-based chemotherapy have become the first-line treatment for advanced colorectal cancer (CRC). Chinese herbal injections (CHIs), as an important part of TCM, have been widely applied as adjunctive treatments to chemotherapy in patients with advanced CRC. However, the efficacy of this combination therapy has not been evaluated comprehensively. Methods: We will conduct this systematic review and meta-analysis in accordance with the Preferred Reported Items for Systematic Review and Meta-analysis (PRISMA) guidelines. 7 databases will be searched for related randomized controlled trials (RCTs) from their inception to August 31, 2020: PubMed, Cochrane Library, EMBASE, China National Knowledge Infrastructure (CNKI), VIP Database for Chinese Technical Periodicals (VIP), SinoMED and Wanfang Database. Two researchers will perform study selection, data extraction, and assessment of risk of bias independently. The primary outcomes are the disease control rate (DCR) and the objective response rate (ORR), the secondary outcomes are progression-free survival (PFS), survival rate, quality of life (QoL) and adverse effects. Cochrane Review Manager (RevMan 5.3) software will be used to analyze the outcomes. Results and Conclusion: This systematic review will evaluate the efficacy of CHIs and fluoropyrimidine and oxaliplatin-based chemotherapy for advanced CRC so as to provide valuable evidence to the application of CHIs in advanced CRC. Systematic review registration: INPLASY registration number: INPLASY2020100050.

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“…Eventual accumulation of inflammation and fibrosis lead to progressive muscle weakening and loss of muscle mass and function [ 37 ]. The efficiency of regeneration appears to be low [ 38 ]. This progressive muscle wasting condition leads to severe disability and follows with premature death in affected individuals due to respiratory and/or cardiac failure, typically by or before the age of 30.…”

Section: Duchenne Muscular Dystrophy: Is There a Role For Satellitmentioning

confidence: 99%

“The Social Network” and Muscular Dystrophies: The Lesson Learnt about the Niche Environment as a Target for Therapeutic Strategies

Cappellari

Mantuano

2020

Cells

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The muscle stem cells niche is essential in neuromuscular disorders. Muscle injury and myofiber death are the main triggers of muscle regeneration via satellite cell activation. However, in degenerative diseases such as muscular dystrophy, regeneration still keep elusive. In these pathologies, stem cell loss occurs over time, and missing signals limiting damaged tissue from activating the regenerative process can be envisaged. It is unclear what comes first: the lack of regeneration due to satellite cell defects, their pool exhaustion for degeneration/regeneration cycles, or the inhibitory mechanisms caused by muscle damage and fibrosis mediators. Herein, Duchenne muscular dystrophy has been taken as a paradigm, as several drugs have been tested at the preclinical and clinical levels, targeting secondary events in the complex pathogenesis derived from lack of dystrophin. We focused on the crucial roles that pro-inflammatory and pro-fibrotic cytokines play in triggering muscle necrosis after damage and stimulating satellite cell activation and self-renewal, along with growth and mechanical factors. These processes contribute to regeneration and niche maintenance. We review the main effects of drugs on regeneration biomarkers to assess whether targeting pathogenic events can help to protect niche homeostasis and enhance regeneration efficiency other than protecting newly formed fibers from further damage.

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Effectiveness of pharmacological treatments in Duchenne muscular dystrophy: a protocol for a systematic review and meta-analysis

Cited by 7 publications

References 33 publications

Validity of bioelectrical impedance to estimate fat-free mass in boys with Duchenne muscular dystrophy

Validity of bioelectrical impedance to estimate fat-free mass in boys with Duchenne muscular dystrophy

Efficacy of Chinese herbal injections combined with fluoropyrimidine and oxaliplatin-based chemotherapy for advanced colorectal cancer

“The Social Network” and Muscular Dystrophies: The Lesson Learnt about the Niche Environment as a Target for Therapeutic Strategies

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