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Developmental and epilepsy outcomes at age 4 years in the UKISS trial comparing hormonal treatments to vigabatrin for infantile spasms: a multi-centre randomised trial
  1. Katrina Darke1,
  2. Stuart W Edwards1,2,
  3. Eleanor Hancock2,3,
  4. Anthony L Johnson4,5,
  5. Colin R Kennedy6,
  6. Andrew L Lux2,7,
  7. Richard W Newton8,
  8. Finbar J K O'Callaghan1,2,7,9,
  9. Christopher M Verity10,
  10. John P Osborne1,2
  11. the trial steering committee on behalf of participating investigators
  1. 1Royal United Hospital Bath NHS Trust, Combe Park, Bath, UK
  2. 2The School for Health, University of Bath, Bath, UK
  3. 3Child and Family Health Services, Woking, UK
  4. 4Medical Research Council Biostatistics Unit, University of Cambridge, Institute of Public Health, Cambridge, UK
  5. 5MRC Clinical Trials Unit, London, UK
  6. 6Paediatric Neurology, Clinical Neurosciences, University of Southampton, Southampton, UK
  7. 7Department of Paediatric Neurology, Frenchay Hospital and the Bristol Royal Hospital for Children, Bristol, UK
  8. 8Department of Paediatric Neurology, Royal Manchester Children's Hospital, Manchester, UK
  9. 9Department of Medicine and Dentistry, University of Bristol, Bristol, UK
  10. 10Department of Paediatrics, Addenbrooke's Hospital, Cambridge, UK
  1. Correspondence to Dr Finbar O’Callaghan, Bristol Royal Hospital for Children, Upper Maudlin Street, Bristol BS2 8BJ, UK; finbar.ocallaghan{at}bristol.ac.uk

Abstract

Background Infantile spasms is the name given to a difficult to treat, severe infantile epilepsy with high morbidity. The United Kingdom Infantile Spasms Study (UKISS) showed that absence of spasms on days 13 and 14 after randomisation was more common in infants allocated hormonal treatments than vigabatrin. At 12–14 months, those with no identified aetiology allocated hormonal treatment had better development. However, epilepsy outcome was not affected by treatment allocated. It is not known if the difference in development persists as the infants grow.

Methods Infants in UKISS were followed up blind to treatment allocation by telephone at a mean age of 4 years using the Vineland Adaptive Behaviour Scales (VABS) and an epilepsy questionnaire.

Findings 9 of 107 enrolled infants had died. 77 were traced and consented to take part. The median (quartile) VABS scores were 60 (42, 97) for the 39 allocated hormonal treatment and 50 (36, 67) for the 38 allocated vigabatrin (Mann–Whitney U=575; p=0.091; median difference (95% CI): 8 (−1 to 19)). For those with no identified aetiology, VABS scores were 96 (52, 102) for the 21 allocated hormonal treatment and 63 (37, 92) for the 16 allocated vigabatrin (U=98.5; p=0.033; median difference (95% CI): 14 (1 to 42)).The proportions in each treatment group with epilepsy were similar.

Interpretation For all 77 infants, development and epilepsy outcomes were not significantly different between the two treatment groups. The better development seen at 14 months in those with no identified aetiology allocated hormonal treatment was seen again at 4 years in this study.

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Footnotes

  • Patient consent Parental/guardian consent obtained.

  • Funding This study was supported by a grant from the Bath Unit for Research in Paediatrics (BURP) that included support from Cow and Gate and Bishopsgate Financial Management. FJKO'C was supported by the Wellcome Trust, ALL and EH by Cow and Gate and KD and JPO by BURP. The funding source had no role in study design, data collection, data analysis, data interpretation or writing of the report.

  • The work was carried out in Royal United Hospital Bath NHS Trust and the School for Health, the University of Bath.

  • Competing interests JPO unsuccessfully approached Aventis for funding of a follow-up study to investigate visual field defects and appeared in a promotional video about vigabatrin for Hoechst-Marion-Roussel. ALL received funding from Hoechst-Marion-Roussel to attend a conference. All other authors have no conflict of interest.

  • Ethics approval This study was conducted with the approval of the South West MREC.

  • Provenance and peer review Not commissioned; externally peer reviewed.

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