The challenges of interventional trials in fetal therapy

Morris, RK; Daniels, Jane; Deeks, Jonathan J; Field, David; Kilby, Mark D.

doi:10.1136/archdischild-2013-305624

Cited by 4 publications

(3 citation statements)

References 24 publications

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“…There was also a higher number of parents who opted for termination of pregnancy than was anticipated (as documented in the voluntary subregister). Impediments to recruitment and influences on women's decision‐making are discussed further in other publications. This study gave a unique opportunity to look at how the characteristics of those entering the RCT reflected the characteristics of those with LUTO in general and of those not opting for treatment randomization, and what factors influenced parents' or clinicians' choice of management.…”

Section: Discussionmentioning

confidence: 98%

“…Finally, the PLUTO RCT demonstrated that, in the context of an RCT for intervention in a rare fetal disease, relatively few women (or their families) were willing to consider randomization of treatment and either opted for entry onto the registry (where they or their healthcare professional could choose treatment) or, more commonly, opted for termination of pregnancy. Further research in this area should be directed at improving recruitment to interventional trials in fetal therapy, investigating new techniques for the assessment of fetal urine analytes such as proteonomics, and further investigate the long‐term outcome after VAS compared with fetal cystoscopy.…”

Section: Discussionmentioning

confidence: 99%

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Outcome in fetal lower urinary tract obstruction: a prospective registry study

Morris

Middleton

Malin

et al. 2015

Ultrasound Obstet Gynecol

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Section: Discussionmentioning

confidence: 98%

Section: Discussionmentioning

confidence: 99%

Outcome in fetal lower urinary tract obstruction: a prospective registry study

Morris

Middleton

Malin

et al. 2015

Ultrasound Obstet Gynecol

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“…Some studies declare "normal renal function" without providing any definition (24, 26). Some rely only on SCr (27,28), and others have omitted to show the data despite using a specific definition (29). In neonates with congenital anomalies of the kidney and urinary tract (CAKUT), including cLUTO, peak SCr is associated with discharge SCr (30).…”

Section: Discussionmentioning

confidence: 99%

Predictors of advanced chronic kidney disease in infancy after definitive vesicoamniotic shunting for congenital lower urinary tract obstruction

et al. 2022

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BackgroundSevere congenital lower urinary tract obstruction (cLUTO) is associated with poor postnatal outcomes, including chronic and end stage kidney disease, and high mortality. Studies of the impact of fetal intervention through vesicoamniotic shunting are marred by a device malfunction rate of up to 60%. In this study, we delineate the postnatal course and infant kidney function following definitive urinary diversion in utero.Materials and MethodsThis is a retrospective, single-center cohort study of 16 male infants who survived the fetal intervention to birth, from 2010 to 2014 at a single center. All had patent shunts in place at birth. Perinatal and biochemical characteristics were collected with patients followed for one year, or until demise, with serial measures of serum creatinine (SCr) and serum cystatin C (CysC).ResultsOf the 16 males, 81% were non-white (38% black, 43% Hispanic). Shunts were placed at a median of 20 weeks (IQR 19,23) gestation, with median fetal bladder volume of 39 cm3 (IQR 9.9,65). All neonates were born preterm [median 34 weeks (IQR 31,35)] and the majority with low birth weight [median 2340 grams (1,895, 2,600)]. 63% required positive pressure ventilation. Advanced chronic kidney disease stage 4–5 at 1 year of age was predicted by neonatal characteristics: peak SCr ≥2 mg/dl, time to peak SCr > 6 days, discharge SCr ≥1.0 mg/dl, CysC ≥2.5 mg/l, urine protein:creatinine ≥4.8 mg/mg, urine microalbumin:creatinine ≥2.2 mg/mg. In infancy, a nadir SCr ≥0.5 mg/dl occurring before 160 days (5.3 months) of age was also predictive of advanced chronic kidney disease stage 4–5 at 1 year. Three patients died in the neonatal period, with 1 receiving kidney replacement therapy (KRT). Three additional patients required KRT before 12 months of age.ConclusionsEven with definitive vesicoamniotic shunting for cLUTO, postnatal morbidity and mortality remain high, emphasizing the role of renal dysplasia, in spite of urinary diversion, in postnatal kidney dysfunction. Neonatal and infant biochemical parameters exhibit distinct trends that offer families and physicians a better understanding of the prognosis of childhood kidney function.

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Current State of Fetal Intervention for Lower Urinary Tract Obstruction

Clayton

Brock

2018

Curr Urol Rep

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The challenges of interventional trials in fetal therapy

Cited by 4 publications

References 24 publications

Outcome in fetal lower urinary tract obstruction: a prospective registry study

Outcome in fetal lower urinary tract obstruction: a prospective registry study

Predictors of advanced chronic kidney disease in infancy after definitive vesicoamniotic shunting for congenital lower urinary tract obstruction

Current State of Fetal Intervention for Lower Urinary Tract Obstruction

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