PMID- 23668357
OWN - NLM
STAT- MEDLINE
DCOM- 20140414
LR  - 20181202
IS  - 1600-0404 (Electronic)
IS  - 0001-6314 (Linking)
VI  - 129
IP  - 1
DP  - 2014 Jan
TI  - Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia 
      - open-label trial.
PG  - 32-40
LID - 10.1111/ane.12141 [doi]
AB  - OBJECTIVES: The objective of the study was to test the efficacy, safety and 
      tolerability of triple therapy with deferiprone, idebenone and riboflavin in 
      Friedreich's ataxia (FRDA) patients in a clinical pilot study. PATIENTS AND 
      METHODS: Patients included in this study were 10 males and three females, 14-61 
      years of age (average 30.2 +/- 12.1), diagnosed with FRDA with normal ventricular 
      function. Patients were treated with triple therapy with deferiprone at 5-25 
      mg/kg/day, idebenone at 10-20 mg/kg/day and riboflavin at 10-15 mg/kg/day for 
      15-45 months. The efficacy of this triple therapy was assessed by change from 
      baseline on the scale for the assessment and rating of ataxia (SARA) and by the 
      change from baseline in echocardiogram parameters. RESULTS: Four patients 
      discontinued due to adverse events (AEs) related with deferiprone. The annual 
      worsening rate (AWR) was estimated in this series as 0.96 (CI 95%: 0.462-1.608) 
      SARA score, whereas AWR for our FRDA cohort was estimated as 2.05 +/- 1.23 SARA 
      score. LVMI only decreased by 6.5 g/m(2) (6.2%) at the end of the first year of 
      therapy. LVEF remained stable, except in case of three patients. CONCLUSION: Our 
      results seem to indicate some uncertain benefit on the neurological and heart 
      functions of this triple therapy in FRDA.
CI  - (c) 2013 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
FAU - Arpa, J
AU  - Arpa J
AD  - Reference Unit of Hereditary Ataxias and Paraplegias, Departments of Neurology, 
      IdiPAZ, Hospital Universitario La Paz, Madrid, Spain.
FAU - Sanz-Gallego, I
AU  - Sanz-Gallego I
FAU - Rodriguez-de-Rivera, F J
AU  - Rodriguez-de-Rivera FJ
FAU - Dominguez-Melcon, F J
AU  - Dominguez-Melcon FJ
FAU - Prefasi, D
AU  - Prefasi D
FAU - Oliva-Navarro, J
AU  - Oliva-Navarro J
FAU - Moreno-Yanguela, M
AU  - Moreno-Yanguela M
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20130514
PL  - Denmark
TA  - Acta Neurol Scand
JT  - Acta neurologica Scandinavica
JID - 0370336
RN  - 0 (Pyridones)
RN  - 1339-63-5 (Ubiquinone)
RN  - 2BTY8KH53L (Deferiprone)
RN  - HB6PN45W4J (idebenone)
RN  - TLM2976OFR (Riboflavin)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Deferiprone
MH  - Female
MH  - Friedreich Ataxia/diagnostic imaging/*drug therapy/physiopathology
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Pilot Projects
MH  - Pyridones/administration & dosage/*therapeutic use
MH  - Riboflavin/administration & dosage/*therapeutic use
MH  - Severity of Illness Index
MH  - Treatment Outcome
MH  - Ubiquinone/administration & dosage/*analogs & derivatives/therapeutic use
MH  - Ultrasonography
MH  - Ventricular Dysfunction, Left/etiology/physiopathology
MH  - Young Adult
OTO - NOTNLM
OT  - Friedreich's ataxia
OT  - cardiomyopathy
OT  - deferiprone
OT  - idebenone
OT  - riboflavin
EDAT- 2013/05/15 06:00
MHDA- 2014/04/15 06:00
CRDT- 2013/05/15 06:00
PHST- 2013/04/09 00:00 [accepted]
PHST- 2013/05/15 06:00 [entrez]
PHST- 2013/05/15 06:00 [pubmed]
PHST- 2014/04/15 06:00 [medline]
AID - 10.1111/ane.12141 [doi]
PST - ppublish
SO  - Acta Neurol Scand. 2014 Jan;129(1):32-40. doi: 10.1111/ane.12141. Epub 2013 May 
      14.