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Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA).
Am Heart J. 2011 Mar; 161(3):639-645.e1.AH

Abstract

BACKGROUND

Friedreich ataxia (FRDA) is commonly associated with hypertrophic cardiomyopathy, but little is known about its frequency, severity, or treatment. In this 6-month randomized, double-blind, controlled study, we sought to determine whether idebenone improves cardiac measures in FRDA.

METHODS

Seventy pediatric subjects were treated either with idebenone (450/900 mg/d or 1,350/2,250 mg/d) or with placebo. Electrocardiograms (ECGs) were assessed at each visit, and echocardiograms, at baseline and week 24.

RESULTS

We found ECG abnormalities in 90% of the subjects. On echocardiogram, 81.4% of the total cohort had left ventricular (LV) hypertrophy, as measured by increased LV mass index-Dubois, and the mean ejection fraction (EF) was 56.9%. In linear regression models, longer PR intervals at baseline were marginally associated with longer GAA repeat length (P = .011). Similarly, GAA repeat length did not clearly predict baseline EF (P = .086) and LV mass by M-mode (P = .045). Left ventricular mass index, posterior wall thickness, EF, and ECG parameters were not significantly improved by treatment with idebenone. Some changes in echocardiographic parameters during the treatment phase correlated with baseline status but not with treatment group.

CONCLUSIONS

Idebenone did not decrease LV hypertrophy or improve cardiac function in subjects with FRDA. The present study does not provide evidence of benefit in this cohort over a 6-month treatment period.

Authors+Show Affiliations

Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, USA.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Randomized Controlled Trial

Language

eng

PubMed ID

21392622

Citation

Lagedrost, Sarah J., et al. "Idebenone in Friedreich Ataxia Cardiomyopathy-results From a 6-month Phase III Study (IONIA)." American Heart Journal, vol. 161, no. 3, 2011, pp. 639-645.e1.
Lagedrost SJ, Sutton MS, Cohen MS, et al. Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J. 2011;161(3):639-645.e1.
Lagedrost, S. J., Sutton, M. S., Cohen, M. S., Satou, G. M., Kaufman, B. D., Perlman, S. L., Rummey, C., Meier, T., & Lynch, D. R. (2011). Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). American Heart Journal, 161(3), 639-e1. https://doi.org/10.1016/j.ahj.2010.10.038
Lagedrost SJ, et al. Idebenone in Friedreich Ataxia Cardiomyopathy-results From a 6-month Phase III Study (IONIA). Am Heart J. 2011;161(3):639-645.e1. PubMed PMID: 21392622.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). AU - Lagedrost,Sarah J, AU - Sutton,Martin St John, AU - Cohen,Meryl S, AU - Satou,Gary M, AU - Kaufman,Beth D, AU - Perlman,Susan L, AU - Rummey,Christian, AU - Meier,Thomas, AU - Lynch,David R, Y1 - 2011/01/31/ PY - 2010/09/02/received PY - 2010/10/29/accepted PY - 2011/3/12/entrez PY - 2011/3/12/pubmed PY - 2011/5/27/medline SP - 639 EP - 645.e1 JF - American heart journal JO - Am Heart J VL - 161 IS - 3 N2 - BACKGROUND: Friedreich ataxia (FRDA) is commonly associated with hypertrophic cardiomyopathy, but little is known about its frequency, severity, or treatment. In this 6-month randomized, double-blind, controlled study, we sought to determine whether idebenone improves cardiac measures in FRDA. METHODS: Seventy pediatric subjects were treated either with idebenone (450/900 mg/d or 1,350/2,250 mg/d) or with placebo. Electrocardiograms (ECGs) were assessed at each visit, and echocardiograms, at baseline and week 24. RESULTS: We found ECG abnormalities in 90% of the subjects. On echocardiogram, 81.4% of the total cohort had left ventricular (LV) hypertrophy, as measured by increased LV mass index-Dubois, and the mean ejection fraction (EF) was 56.9%. In linear regression models, longer PR intervals at baseline were marginally associated with longer GAA repeat length (P = .011). Similarly, GAA repeat length did not clearly predict baseline EF (P = .086) and LV mass by M-mode (P = .045). Left ventricular mass index, posterior wall thickness, EF, and ECG parameters were not significantly improved by treatment with idebenone. Some changes in echocardiographic parameters during the treatment phase correlated with baseline status but not with treatment group. CONCLUSIONS: Idebenone did not decrease LV hypertrophy or improve cardiac function in subjects with FRDA. The present study does not provide evidence of benefit in this cohort over a 6-month treatment period. SN - 1097-6744 UR - https://wwww.unboundmedicine.com/medline/citation/21392622/Idebenone_in_Friedreich_ataxia_cardiomyopathy_results_from_a_6_month_phase_III_study__IONIA__ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0002-8703(10)01006-9 DB - PRIME DP - Unbound Medicine ER -