Idebenone treatment in paediatric and adult patients with Friedreich ataxia: Long-term follow-up

Pineda, Merçè; Arpa, Javier; Montero, Raquel; Aracil, A.; Domı́nguez, Francisco; Galván, Marta Ferrer; Mas, Anna; Martorell, Loreto; Sierra, Cristina; Brandi, Nuria; García‐Arumí, Elena; Rissech, Miquel; Velasco, Daniel; Costa, Juan Antonio Orts; Artuch, Rafael

doi:10.1016/j.ejpn.2007.11.006

Cited by 91 publications

(61 citation statements)

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“…13,14 Our results showed no statistically significant differences in ICARS scores or patient age between Groups 1 and 2 at the start of the therapy, suggesting that these variables did not explain the different clinical outcomes. Nor did treatment compliance differ between the two groups, according to plasma CoQ 10 values.…”

Section: Discussionmentioning

confidence: 57%

Coenzyme Q₁₀‐responsive ataxia: 2‐year‐treatment follow‐up

et al. 2010

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We assessed the clinical outcome after coenzyme Q(10) (CoQ(10)) therapy in 14 patients presenting ataxia classified into two groups according to CoQ(10) values in muscle (deficient or not). We performed an open-label prospective study: patients were evaluated clinically (international cooperative ataxia rating scale [ICARS] scale, MRI, and videotape registration) at baseline and every 6 months during a period of 2 years after CoQ(10) treatment (30 mg/kg/day). Patients with CoQ(10) deficiency showed a statistically significant reduction of ICARS scores (Wilcoxon test: P = 0.018) after 2 years of CoQ(10) treatment when compared with baseline conditions. In patients without CoQ(10) deficiency, no statistically significant differences were observed in total ICARS scores after therapy, although 1 patient from this group showed a remarkable clinical amelioration. Biochemical diagnosis of CoQ(10) deficiency was a useful tool for the selection of patients who are good candidates for treatment as all of them responded to therapy. However, the remarkable clinical response in 1 case without CoQ(10) deficiency highlights the importance of treatment trials for identification of patients with CoQ(10)-responsive ataxia.

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Section: Discussionmentioning

confidence: 57%

Coenzyme Q₁₀‐responsive ataxia: 2‐year‐treatment follow‐up

et al. 2010

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“…In these studies, idebenone consistently improved cardiac hypertrophy, which is an important feature of FRDA. Neurological symptoms have not been improved as much as cardiac problems, however higher doses of idebenone especially in younger patients seem to be much more promising for neurological efficacy [76,[85][86][87].…”

Section: Idebenonementioning

confidence: 99%

Clinical and pharmacological aspects of bath salt use: A review of the literature and case reports

Miotto¹,

Striebel²,

Cho

et al. 2013

Drug and Alcohol Dependence

121

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Abstract:Reactive oxygen species (ROS) are widely believed to cause or aggravate several human pathologies such as neurodegenerative diseases, cancer, stroke and many other ailments. Antioxidants are assumed to counteract the harmful effects of ROS and therefore prevent or treat oxidative stress-related diseases. In this report, recent human studies exploring the efficiency of antioxidants in prevention and treatment of various diseases are reviewed. Few antioxidants including edaravone (for ischemic stroke in Japan), Nacetylcysteine (for acetaminophen toxicity), alfa-lipoic acid (for diabetic neuropathy) and some flavonoids (polyphenolic compounds present in dietary plants), such as micronized purified flavonoid fraction (diosmin and hesperidin) and oxerutins (for chronic venous insufficiency) as well as baicalein and catechins (for osteoarthritis) have found accepted clinical use. However, despite much enthusiasm in the 1980s and 1990s, many well-known agents such as antioxidant vitamins and also more recently developed compounds such as nitrones have not successfully passed the scrutiny of clinical trials for prevention and treatment of various diseases. This has given rise to a pessimistic view of antioxidant therapy, however, the evidence from human epidemiological studies about the beneficial effects of dietary antioxidants and preclinical in vitro and animal data are compelling. We have probably wasted too much time on agents like antioxidant vitamins instead of focusing on more disease specific, target-directed, highly bioavailable antioxidants. We here discuss possible reasons for the lack of success in some clinical trials and seek to provide some suggestions to be considered if antioxidant therapy is to succeed as an effective therapeutic strategy.

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“…ICARS scores showed significant improvement from baseline after 3, 6, and 12 months of treatment (P = 0.017, P = 0.012, and P = 0.007, respectively), indicating the possibility of a rapid onset of efficacy in young patients. A recent study described 10 children (aged 8 to 18 years) and 14 adults (aged 18 to 46 years) with FRDA who were treated for up to 5 years with idebenone [18]. In children, dosing started at 5 mg/kg/day for 18 months with a subsequent increase to 10 mg/kg/day; in adults, the same starting dose was given for 1 year, with subsequent increases to 10 mg/kg/day and then to 20 mg/kg/day.…”

Section: ■ Efficacy Of Idebenone On Neurological Symptoms Of Frdamentioning

confidence: 99%

“…To date, efficacy data have been reported from 11 clinical studies of idebenone in the treatment of FRDA. Table 1 provides an overview of clinical trials published from 1999 to 2008 [11][12][13][14][15][16][17][18][19][20][21][22]. Most of these studies were open label, but three were randomised, double-blind, placebo-controlled trials.…”

Section: Evidence For Clinical Efficacy Of Idebenone In Frdamentioning

confidence: 99%

“…An open-label study investigated 10 children and 14 adults with FRDA treated with idebenone for up to 5 years, with doses starting at 5 mg/kg/day and increasing to 10 mg/kg/day (children) or 20 mg/kg/day (adults) [18]. Among paediatric patients, measures of cardiac hypertrophy (SWT and PWT) remained stable over the entire study period, and after therapy, only 2 of 10 patients showed impaired heart function.…”

Section: ■ Evidence For Efficacy Of Idebenone On Hypertrophic Cardiommentioning

confidence: 99%

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Idebenone: An emerging therapy for Friedreich ataxia

Meier

Buyse

2009

J Neurol

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This paper reviews the history and pre-clinical development of idebenone and summarises the results of clinical studies, published from 1999 to 2008, on the use of idebenone in the treatment of patients with Friedreich ataxia (FRDA). As a benzoquinone that can undergo reversible redox reactions, idebenone influences the electron balance in mitochondria. In vitro studies have shown that it acts both as an anti-oxidant, preventing damage to the mitochondrial membrane, and, more importantly, as an electron carrier, supporting mitochondrial function and adenosine triphosphate (ATP) production. In clinical studies, idebenone has been well tolerated by patients with various pathological conditions. The most common adverse events have been gastrointestinal effects of mild to moderate severity. No neurotoxic or adverse cardiac reactions have been reported in pre-clinical or clinical studies. The good safety profile of idebenone is supported by large clinical trials in Alzheimer's disease and by post-marketing surveillance. Phase 1 studies demonstrated the safety and tolerability of idebenone at relatively high doses (up to 60 mg/kg/day). Results from 11 clinical studies (randomised, controlled, and open-label trials), involving a total of about 200 patients, provide evidence of improvement in both cardiac hypertrophy and neurological symptoms among patients with FRDA treated with idebenone.

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Idebenone treatment in paediatric and adult patients with Friedreich ataxia: Long-term follow-up

Cited by 91 publications

References 16 publications

Coenzyme Q₁₀‐responsive ataxia: 2‐year‐treatment follow‐up

Coenzyme Q₁₀‐responsive ataxia: 2‐year‐treatment follow‐up

Clinical and pharmacological aspects of bath salt use: A review of the literature and case reports

Idebenone: An emerging therapy for Friedreich ataxia

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Idebenone treatment in paediatric and adult patients with Friedreich ataxia: Long-term follow-up

Cited by 91 publications

References 16 publications

Coenzyme Q10‐responsive ataxia: 2‐year‐treatment follow‐up

Coenzyme Q10‐responsive ataxia: 2‐year‐treatment follow‐up

Clinical and pharmacological aspects of bath salt use: A review of the literature and case reports

Idebenone: An emerging therapy for Friedreich ataxia

Contact Info

Product

Resources

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Coenzyme Q₁₀‐responsive ataxia: 2‐year‐treatment follow‐up

Coenzyme Q₁₀‐responsive ataxia: 2‐year‐treatment follow‐up