Objective To determine the likely rate of patient randomisation and to facilitate sample size calculation for a full-scale phase III trial of varicella zoster immunoglobulin (VZIG) and aciclovir as postexposure prophylaxis against chickenpox in children with cancer.
Design Multicentre pilot randomised controlled trial of VZIG and oral aciclovir.
Setting England, UK.
Patients Children under 16 years of age with a diagnosis of cancer: currently or within 6 months of receiving cancer treatment and with negative varicella zoster virus (VZV) serostatus at diagnosis or within the last 3 months.
Interventions Study participants who have a significant VZV exposure were randomised to receive PEP in the form of VZIG or aciclovir after the exposure.
Main outcome measures Number of patients registered and randomised within 12 months of the trial opening to recruitment and incidence of breakthrough varicella.
Results The study opened in six sites over a 13-month period. 482 patients were screened for eligibility, 32 patients were registered and 3 patients were randomised following VZV exposure. All three were randomised to receive aciclovir and there were no cases of breakthrough varicella.
Conclusions Given the limited recruitment to the PEPtalk2 pilot, it is unlikely that the necessary sample size would be achievable using this strategy in a full-scale trial. The study identified factors that could be used to modify the design of a definitive trial but other options for defining the best means to protect such children against VZV should be explored.
Trial registration number ISRCTN48257441, EudraCT number: 2013-001332-22, sponsor: University of Birmingham.
- paediatric oncology
- paediatric haematology
Statistics from Altmetric.com
Contributors All authors contributed to study design and approved the content of the final paper. SB was the trial coordinator. AH and KW provided statistical analysis. Sponsor: University of Birmingham.
Funding This study was funded by National Institute of Health Research—Research for Patient Benefit and Programme Grants for Applied Research (PB-PG-0211-24142). JCC was supported by National Health Service funding to the National Institute for Health Research Biomedical Research Center of the Royal Marsden Hospital. MJ was supported by the National Institute for Health Research Health Protection Research Unit (NIHR HPRU) in Immunisation at the London School of Hygiene and Tropical Medicine in partnership with Public Health England (PHE) (grant reference code HPRU-2012-10096).
Disclaimer This paper presents independent research funded by the National Institute for Health Research (NIHR) under its Research for Patient Benefit Programme (Grant Reference Number PB-PG-1207-15250). The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health and Social Care.
Competing interests None declared
Patient consent As PEPtalk2 had a two-stage enrolment process, beginning with registration and then followed (in the event of a chickenpox exposure) by randomisation, written informed consent from the patient’s legal representative was obtained at both stages.
Ethics approval MREC number 13/LP/0551.
Provenance and peer review Not commissioned; externally peer reviewed.
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.