CINRG pilot trial of coenzyme Q10 in steroid‐treated duchenne muscular dystrophy

Spurney, Christopher F.; Rocha, Carolina Tesi; Henricson, E.; Florence, Julaine; Mayhew, J.; Gorni, Ksenija; Pasquali, Livia; Pestronk, Alan; Martin, Gerard R.; Hu, Fengming; Connolly, Anne M.; Escolar, Diana M.

doi:10.1002/mus.22047

Cited by 46 publications

(37 citation statements)

References 21 publications

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“…We observed an independent association of Vitamin D usage with higher Age WC , which appeared to be unrelated to bone fracture risk. Coenzyme Q10 is associated with prolonged ambulation in those DMD patients also taking steroids, which supports a recent smaller scale six month clinical trial of Coenzyme Q10 in DMD [34]. It is unclear if there is any relationship between the potential therapeutic benefit of Vitamin D and the reported observation of a relatively high frequency of Vitamin D deficiency in boys with DMD [35], but the data from DuchenneConnect suggest that broader usage of Vitamin D supplementation might be worth exploring as a therapeutic adjunct.…”

Section: Cc-by-ndmentioning

confidence: 52%

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Wang

Fadlon

Ulm

et al. 2014

Preprint

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To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Data collected were compared to prior natural history data in regard to age at diagnosis, mutation spectrum, and age at loss of ambulation. Because registrants reported differences in steroid and other medication usage, as well as age and ambulation status, we could explore these data for correlations with age at loss of ambulation. Using multivariate analysis, current steroid usage was the most significant and largest independent predictor of improved wheelchair-free survival. Thus, these online self-report data were sufficient to retrospectively observe that current steroid use by patients with DMD is associated with a delay in loss of ambulation. Comparing commonly used steroid drugs, deflazacort prolonged ambulation longer than prednisone (median 14 years and 13 years, respectively). Further, use of Vitamin D and Coenzyme Q10, insurance status, and age at diagnosis after 4 years were also significant, but smaller, independent predictors of longer wheelchair-free survival. Nine other common supplements were also individually tested but had lower study power. This study demonstrates the utility of DuchenneConnect data to observe therapeutic differences, and highlights needs for improvement in quality and quantity of patient-report data, which may allow exploration of drug/therapeutic practice combinations impractical to study in clinical trial settings.

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Section: Cc-by-ndmentioning

confidence: 52%

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Wang

Fadlon

Ulm

et al. 2014

Preprint

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show abstract

“…Interestingly, even controlling for the large effect of steroids, boys using Coenzyme Q10 or Vitamin D were observed to have a delay in age at loss of ambulation suggesting some small therapeutic effect. Coenzyme Q10 benefit was noted by a recent CINRG study [26], but the effect of Vitamin D is novel and the benefit appeared unrelated to a decrease in bone fractures [11]. This illustrates the benefits of aggregating individual data for seeding ideas for future clinical trials.…”

Section: Effect Of Supplementsmentioning

confidence: 95%

What can Duchenne Connect teach us about treating Duchenne muscular dystrophy?

Wang

Nelson²

2015

Current Opinion in Neurology

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Purpose of Review This review aims to describe the benefits and limitations of using the DuchenneConnect patient registry to provide information particularly in regard to active treatment choices in Duchenne muscular dystrophy and their impact on disease progression. Recent findings Clinical trials and natural history studies are difficult for rare diseases like Duchenne muscular dystrophy. Using an online patient self-report survey model, DuchenneConnect provides relevant data that are difficult to gather in other ways. Validation of the overall dataset is supported by comparable mutational spectrum relative to other cohorts and demonstrated beneficial effect of corticosteroid use in prolonging ambulation. These types of analyses are provocative and allow multivariate analyses across the breadth of patient and physician medication and supplement practices. Because the data is self-reported and online, the barrier to participation is low and great potential exists for novel directions of further research in a highly participatory forum. Summary Patient registries for Duchenne and Becker muscular dystrophy are powerful tools for monitoring patient outcomes, comparing treatments options, and relating information between patients, researchers and clinicians. DuchenneConnect is an online patient self-report registry for individuals with DBMD that facilitates aggregation of treatment modalities, outcomes and genotype data and has played a vital role in furthering DBMD research, particularly in the US, in a highly participatory and low cost manner.

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“…In support, generalized ROS scavenging has been shown to be deleterious to acute exercise adaptation in healthy muscle (Petersen et al, 2012; Strobel et al, 2011) showing that a basal level of ROS signaling is important for positive adaptation. This same general ROS reduction strategies are also ineffective as therapeutic strategies in neuromuscular diseases (i.e., idebenone (Buyse et al, 2011), Coenzyme Q 10 (Spurney et al, 2011), tocopherol (Arthur, Austin, & Roberts, 1988)) where excess ROS is known to be a disease contributor. In these cases, the source and the species of ROS are likely disease specific and in this case, a targeted approach for therapy is likely needed.…”

Section: Resultsmentioning

confidence: 99%

ROS and RNS Signaling in Skeletal Muscle: Critical Signals and Therapeutic Targets

Michaelson¹,

Iler²,

Ward³

2013

Annu Rev Nurs Res

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The health of skeletal muscle is promoted by optimal nutrition and activity/exercise through the activation of molecular signaling pathways. Reactive oxygen species (ROS) or reactive nitrogen species (RNS) have been shown to modulate numerous biochemical processes including glucose uptake, gene expression, calcium signaling and contractility. In pathological conditions, ROS/RNS signaling excess or dysfunction contributes to contractile dysfunction and myopathy in skeletal muscle. Here we provide a brief review of ROS/RNS chemistry and discuss concepts of ROS/RNS signaling and its role in physiological and pathophysiological processes within striated muscle.

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CINRG pilot trial of coenzyme Q10 in steroid‐treated duchenne muscular dystrophy

Abstract: Introduction-Corticosteroid treatment slows disease progression and is the standard of care for Duchenne muscular dystrophy (DMD). Coenzyme Q10 (CoQ10) is a potent antioxidant that may improve function in dystrophin deficient muscle.

Cited by 46 publications

References 21 publications

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

What can Duchenne Connect teach us about treating Duchenne muscular dystrophy?

ROS and RNS Signaling in Skeletal Muscle: Critical Signals and Therapeutic Targets

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