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Dermatologic Therapy Mar 2021
Topics: Diagnosis, Differential; Diaper Rash; Humans; Infant; Plasma Gases
PubMed: 33404181
DOI: 10.1111/dth.14739 -
Molecular Genetics and Metabolism Jan 2021Neonatal onset Urea cycle disorders (UCDs) can be life threatening with severe hyperammonemia and poor neurological outcomes. Glycerol phenylbutyrate (GPB) is safe and...
BACKGROUND/AIMS
Neonatal onset Urea cycle disorders (UCDs) can be life threatening with severe hyperammonemia and poor neurological outcomes. Glycerol phenylbutyrate (GPB) is safe and effective in reducing ammonia levels in patients with UCD above 2 months of age. This study assesses safety, ammonia control and pharmacokinetics (PK) of GPB in UCD patients below 2 months of age.
METHODS
This was an open-label study in UCD patients aged 0 - 2 months, consisting of an initiation/transition period (1 - 4 days) to GPB, followed by a safety extension period (6 months to 2 years). Patients presenting with a hyperammonemic crisis (HAC) did not initiate GPB until blood ammonia levels decreased to below 100 µmol/L while receiving sodium phenylacetate/sodium benzoate and/or hemodialysis. Ammonia levels, PK analytes and safety were evaluated during transition and monthly during the safety extension for 6 months and every 3 months thereafter.
RESULTS
All 16 patients with UCD (median age 0.48 months, range 0.1 to 2.0 months) successfully transitioned to GPB within 3 days. Average plasma ammonia level excluding HAC was 94.3 µmol/L at baseline and 50.4 µmol/L at the end of the transition period (p = 0.21). No patient had a HAC during the transition period. During the safety extension, the majority of patients had controlled ammonia levels, with mean plasma ammonia levels lower during GPB treatment than baseline. Mean glutamine levels remained within normal limits throughout the study. PK analyses indicate that UCD patients <2 months are able to hydrolyze GPB with subsequent absorption of phenylbutyric acid (PBA), metabolism to phenylacetic acid (PAA) and conjugation with glutamine. Plasma concentrations of PBA, PAA, and phenylacetylglutamine (PAGN) were stable during the safety extension phase and mean plasma phenylacetic acid: phenylacetylglutamine ratio remained below 2.5 suggesting no accumulation of GPB. All patients reported at least 1 treatment emergent adverse event with gastroesophageal reflux disease, vomiting, hyperammonemia, diaper dermatitis (37.5% each), diarrhea, upper respiratory tract infection and rash (31.3% each) being the most frequently reported.
CONCLUSIONS
This study supports safety and efficacy of GPB in UCD patients aged 0 -2 months who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. GPB undergoes intestinal hydrolysis with no accumulation in this population.
Topics: Age of Onset; Ammonia; Child, Preschool; Female; Glycerol; Humans; Hyperammonemia; Infant; Infant, Newborn; Male; Pediatrics; Phenylacetates; Phenylbutyrates; Renal Dialysis; Urea Cycle Disorders, Inborn
PubMed: 33388234
DOI: 10.1016/j.ymgme.2020.12.002 -
Pediatrics in Review Jan 2021
Topics: Diaper Rash; Female; Humans; Infant
PubMed: 33386309
DOI: 10.1542/pir.2018-0141 -
Pediatrics in Review Jan 2021
Topics: Diagnosis, Differential; Diaper Rash; Humans; Infant
PubMed: 33386307
DOI: 10.1542/pir.2020-0128 -
Pediatrics and Neonatology Mar 2021Baby wipes have been shown to be safe and effective in maintaining skin integrity when compared to the use of water alone. However, no previous study has compared... (Comparative Study)
Comparative Study
The BaSICS (Baby Skin Integrity Comparison Survey) study: A prospective experimental study using maternal observations to report the effect of baby wipes on the incidence of irritant diaper dermatitis in infants, from birth to eight weeks of age.
BACKGROUND
Baby wipes have been shown to be safe and effective in maintaining skin integrity when compared to the use of water alone. However, no previous study has compared different formulations of wipe. The aim of the BaSICS study was to identify any differences in incidence of irritant diaper dermatitis (IDD) in infants assigned to three different brands of wipe, all marketed as suitable for neonates, but which contained varying numbers of ingredients.
METHODS
Women were recruited during the prenatal period. Participants were randomly assigned to receive one of three brands of wipe for use during the first eight weeks following childbirth. All participants received the same nappies. Participants reported their infant's skin integrity on a scale of 1-5 daily using a bespoke smartphone application. Analysis of effect of brand on clinically significant IDD (score 3 or more) incidence was conducted using a negative binomial generalised linear model, controlling for possible confounders at baseline. Analysts were blind to brand of wipe.
RESULTS
Of 737 women enrolled, 15 were excluded (admitted to neonatal intensive care, premature or other infant health issues). Of the 722 eligible babies, 698 (97%) remained in the study for the full 8-week duration, 24.6% of whom had IDD at some point during the study. Mothers using the brand with the fewest ingredients reported fewer days of clinically significant nappy rash (score≥3) than participants using the two other brands (p = 0.002 and p < 0.001). Severe IDD (grades 4 and 5) was rare (2.4%).
CONCLUSIONS
Rarity of severe IDD suggested that sensitive formula baby wipes are safe when used in cleansing babies from birth to eight weeks during nappy changes. The brand with fewest ingredients had significantly fewer days of clinically significant IDD. Daily observations recorded on a smartphone application proved to be a highly acceptable method of obtaining real-time data on IDD.
CLINICAL TRIAL REGISTRATION
This study was not designed or registered as a clinical trial as no intervention in normal patterns of infant care took place. Mothers who had already decided to use disposable nappies and a baby wipe product agreed to observe and report on their infants' skin condition; in return they received a 9-week supply of free nappies and wipes.
Topics: Adult; Diaper Rash; Female; Humans; Incidence; Infant; Infant, Newborn; Male; Prospective Studies; Random Allocation; Skin Care
PubMed: 33221203
DOI: 10.1016/j.pedneo.2020.10.003 -
The Journal of Pediatrics Mar 2021To examine the possible association between diaper need, difficulty affording an adequate amount of diapers, and pediatric care visits for urinary tract infections and...
OBJECTIVE
To examine the possible association between diaper need, difficulty affording an adequate amount of diapers, and pediatric care visits for urinary tract infections and diaper dermatitis.
STUDY DESIGN
This cross-sectional analysis using nationally representative survey data collected July-August 2017 using a web-based panel examined 981 parents of children between 0 and 3 years of age in the US (response rate, 94%). Survey weighting for differential probabilities of selection and nonresponse was used to estimate the prevalence of diaper need and to perform multivariable logistic regression of the association between parent reported diaper need and visits to the pediatrician for diaper rash or urinary tract infections within the past 12 months.
RESULTS
An estimated 36% of parents endorsed diaper need. Both diaper need (aOR 2.37; 95% CI 1.69-3.31) and visiting organizations to receive diapers (aOR 2.14; 95% CI 1.43-3.21) were associated with diaper dermatitis visits. Similar associations were found for diaper need (aOR 2.63; 95% CI 1.54-4.49) and visiting organizations to receive diapers (aOR 4.50; 95% CI 2.63-7.70) for urinary tract infection visits.
CONCLUSIONS
Diaper need is common and associated with increased pediatric care visits. These findings suggest pediatric provider and policy interventions decreasing diaper need could improve child health and reduce associated healthcare use.
Topics: Adult; Child, Preschool; Cross-Sectional Studies; Diaper Rash; Diapers, Infant; Facilities and Services Utilization; Female; Health Services Needs and Demand; Humans; Infant; Infant, Newborn; Male; Parents; United States; Urinary Tract Infections
PubMed: 33130154
DOI: 10.1016/j.jpeds.2020.10.061 -
Journal of Paediatrics and Child Health Oct 2020
PubMed: 33099825
DOI: 10.1111/jpc.2_14923 -
Journal of Paediatrics and Child Health Oct 2020
PubMed: 33099821
DOI: 10.1111/jpc.1_14923