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The challenges of interventional trials in fetal therapy
  1. R Katie Morris1,2,
  2. J Daniels1,3,
  3. J Deeks3,4,
  4. D Field5,
  5. M D Kilby1,2
  1. 1Centre for Women's & Children Health and the School of Clinical and Experimental Medicine, College of Medical and Dental Sciences, University of Birmingham, Birmingham, UK
  2. 2Fetal Medicine Centre, Birmingham Women's Hospital NHS Foundation Trust, Birmingham, UK
  3. 3Birmingham Clinical Trials Unit, University of Birmingham, Birmingham, UK
  4. 4School of Health and Population Sciences, College of Medical and Dental Sciences, University of Birmingham, Birmingham, UK
  5. 5Department of Health Sciences, University of Leicester, Leicester, UK
  1. Correspondence to Dr R Katie Morris, Birmingham Centre for Women and Children's Health, School of Clinical and Experimental Medicine, College of Medical and Dental Sciences, University of Birmingham, Birmingham Women's Hospital, Edgbaston, Birmingham B15 2TG, UK; r.k.morris{at}bham.ac.uk

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Fetal therapy is an advancing specialty but the assessment of effectiveness for many therapies has been limited to observational data1 ,2 with randomised controlled trials (RCTs) employed with varying success.3 ,4 More recently systematic reviews have been used to assess the quality and summarise the outcomes of this evidence.5 ,6

Our experiences from one such trial (the PLUTO study4) highlight the difficulties of such research. Congenital lower urinary tract obstruction (LUTO) may be identified using prenatal ultrasound and is associated with high mortality and morbidity (perinatal and childhood), due to pulmonary hypoplasia and chronic renal impairment.7–9 Ultrasound-directed, in utero, vesicoamniotic shunting (VAS) bypasses the congenital urethral obstruction with the aim of improving fetal outcome.1 ,2 ,10 Counselling parents faced with the difficult and distressing news that their baby has a significant problem antenatally is complex and compounded by uncertainties around the effectiveness of the often limited number of options. Providing clinicians with high quality evidence on which to base their counselling affords parents the opportunity to make informed choices and can help to remove some of the anxiety. The PLUTO study aimed to determine the effectiveness, cost-effectiveness and patient acceptability of VAS for fetal LUTO, compared with conservative management. It comprised a multicentre, international RCT and a non-randomised cohort of pregnancies with LUTO (not recruited to the RCT due to patient or clinician preference).4 All fetal medicine centres within England, Scotland and The Republic of Ireland agreed to take part in the study along with the Dutch Obstetric Research Consortium. Expert opinions on the relative benefits of VAS and conservative management were elicited from fetal medicine specialists, paediatric nephrologists and paediatric urologists for use in a Bayesian analysis.11 The planned sample size of the trial was 150, …

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