cometadata/datacite-titles-descriptions-related-identifiers huggingface dataset:
DOI: 10.57982/gzpy-qt80
title: Validation and Human Factor Analysis of Mercy’s babyTAPE for Weight Estimation in Neonates and Young Infants

description: The babyTAPE device was developed to use head and chest circumference measures to predict infant weight. TAP03 objectives were to evaluate user performance of critical tasks in a device-use environment, confirm predictive performance, determine user subjective experience and define inter-rater reliability (IRR). A total of 486 infants were enrolled at three sites, stratified to include a cross-section of newborn ages and sizes including preterm. Infant height/weight were taken using a calibrated infant scale, and head/chest circumference measured with a masked reference tape and babyTAPE. Rater performance and confidence in results were reported. Actual weights were compared with estimations using the reference tape and babyTAPE. For both methods, there was tight correlation between actual and estimated weights and high IRR. Most raters found the babyTAPE device to be reliable and easy to use, and human factor error magnitude was reduced compared to the reference tape. No adverse device events were reported.
DOI: 10.57982/fa16-ty93
title: Safety and Pharmacokinetics of Multiple-Dose Intravenous and Oral Clindamycin in Pediatric Subjects with BMI ?85th Percentile

description: The primary objective of CLN01 was to determine the pharmacokinetics (PK) of intravenous (IV) clindamycin in overweight and obese children and adolescents. This study was a prospective, open-label safety and PK study of multiple doses of IV and oral clindamycin in overweight and obese children 2 to <18 years of age. Clindamycin PK samples to develop the population PK model were also collected from NICHD-2011-POP01 and NICHD-2012-STA01 and analyzed as part of CLN01. Overall, clindamycin was very well tolerated in this open-label study. Three AEs (including one SAE) were reported in 2 (9%) CLN01 patients; none were considered related to study drug. After accounting for size-based differences using total body weight and physiologic differences using age, only volume of distribution and terminal elimination half-life were significantly different between obese and non-obese children. Clindamycin may be dosed based on total body weight (max dose 2.7 g/day) without dose adjustment based solely on obesity.
DOI: 10.57982/yjay-3487
title: Optimizing Cooling Strategies at <6 Hours of Age for Neonatal Hypoxic-Ischemic Encephalopathy

description: The Optimizing Cooling trial compared four whole-body cooling treatments for infants born at 36 weeks gestational age or later with hypoxic-ischemic encephalopathy: (1) cooling for 72 hours to 33.5�C; (2) cooling for 120 hours to 33.5�C; (3) cooling for 72 hours to 32.0�C; and (4) cooling for 120 hours to 32.0�C. The objective of this study was to evaluate whether whole-body cooling initiated at less than 6 hours of age and continued for 120 hours and/or a depth at 32.0�C would reduce death and disability at 18-22 months corrected age.  This trial was terminated early for emerging safety profiles and futility reasons.  Longer duration of cooling was associated with more arrhythmia and anuria and longer hospital days, whereas deeper cooling was associated with higher use of inhaled nitric oxide therapy, ECMO, more days of oxygen, and higher incidence of bradycardia.
DOI: 10.57982/kf2r-v263
title: Pelvic Symptoms and Patient Satisfaction After Colpocleisis

description: This study describes the postoperative course of women who undergo colpocleisis, with particular attention to the persistence or recurrence of urinary incontinence and patient satisfaction after the colpocleisis prolapse surgery.
DOI: 10.57982/3q4x-7p11
title: Ensuring Accurate Weight Assessment in Newborns and Young Infants (babyTAPE)

description: TAP02 objectives were to obtain anthropometric data to allow modification of the Mercy Method and Mercy TAPE to develop the babyTAPE for weight estimation in newborns and young infants. A total of 2097 infants were enrolled at eight sites, stratified to include a cross-section of newborn ages and sizes including preterm. Singular length and girth surrogates correlating most closely with weight were identified and a mathematical model for weight estimation in this population was developed and internally validated. Head circumference and chest circumference demonstrated the best correlations with weight and length and were among the most reproducible as reflected by intraclass correlation coefficients. The final model predicted weight within 10% and 15% of actual for 84% and 94% of infants, respectively, without bias for age measures or sex. No adverse device events were reported.
DOI: 10.57982/788q-c982
title: The Effect of Obesity on the Pharmacokinetics of Pantoprazole in Children and Adolescents

description: This was a prospective, multi-center, open-label pharmacokinetic (PK) and safety study of pantoprazole in obese children 6 to 17 years of age requiring treatment with an acid-modifying agent. The primary objective was to evaluate the PK of pantoprazole following administration of a single oral dose. All enrolled participants received study medication, were included in the safety population, and submitted at least one fresh plasma PK sample. The PK of pantoprazole was affected by obesity, with higher exposures observed in obese children and adolescents relative to non-obese historical controls. However, if weight tiered dosing regimens are used according to the FDA label, obese children and adolescents may be given the same dose as non-obese peers. Pantoprazole was well tolerated in this open-label study, with 9 AEs reported in 7 (17%) of the 41 safety participants during the study. Two AEs (headache and hiccups) in 2 participants (5%) were considered related to study drug. No SAEs were reported.
DOI: 10.57982/f1s4-mv35
title: The Critical Illness Stress-induced Immune Suppression Prevention Trial

description: Despite strict hand washing, sterile technique, and antibiotic-coated catheters, nosocomial infection and sepsis remain the leading acquired causes of morbidity and mortality in critically ill children. Subsequent use of antibiotics to treat nosocomial infection and sepsis is considered a major attributable factor in the rise of antibiotic-resistant organisms in this population of children. Presently, “prophylaxis” strategies are used to prevent stress-induced gastrointestinal bleeding; however, no “prophylaxis” strategy is used to prevent stress-induced nosocomial infection and sepsis. This study used a double-blind randomized controlled trial design to test the hypothesis that daily prophylaxis with metoclopramide, zinc, selenium, and glutamine will reduce nosocomial infection and sepsis in critically ill children.
DOI: 10.57982/62gh-cs85
title: Taking the Guesswork out of Pediatric Weight Estimation (TAPE): Validation of the Mercy TAPE

description: The main TAP01 objective was to evaluate the predictive performance of the 2D and 3D Mercy TAPE, and evaluate the equivalence of the 2D and 3D Mercy TAPE relative to the Mercy method of weight estimation. A total of 664 children were enrolled, stratified by year of age. Height/length, weight, humeral length and mid-upper arm circumference were obtained for each child using calibrated scales and measures. Measurements were then taken using blinded versions of the 2D- and 3D-TAPEs, and the estimated weights were contrasted with results utilizing reference methods. Concordance between both TAPE devices and the Mercy Method was >0.99. The proportion of children predicted within 10% and 20% of actual weight was 76% and 98% for the 2D TAPE, and 65% and 93% for the 3D TAPE. The 2D- and 3D-Mercy TAPEs outperformed reference methods for pediatric weight estimation and can be used in a wider range of children. No adverse device events were reported.
DOI: 10.57982/e8bm-m516
title: Antenatal Late Preterm Steroids: A Randomized Placebo Controlled Trial

description: The Antenatal Late Preterm Steroids Trial (ALPS) was a randomized, multicenter, double-blind trial to determine whether antenatal corticosteroids given to women at high risk for late preterm birth (34 weeks to 36 weeks) reduces stillbirth or the need for neonatal respiratory support of preterm birth or neonatal death in the first 72 hours. Women with a singleton pregnancy at 34-36 weeks who were at high risk for preterm delivery were randomized to 2 injections of betamethasone or matching placebo 24 hours apart. There were 2831 women randomized; 4 were lost to follow-up. The primary outcome occurred in 165 infants (11.6%) in the betamethasone group and 202 (14.4%) in the placebo group (RR 0.80; 95% CI, 0.66-0.97; p=0.02). Severe respiratory complications, transient tachypnea of the newborn, surfactant use, and bronchopulmonary dysplasia also occurred significantly less frequently in the betamethasone group. Neonatal hypoglycemia was more common in the betamethasone group (p<0.001).
DOI: 10.57982/pfek-5359
title: The Impact of Personal Protective Equipment on Pediatric Procedures

description: This was a randomized, cross-over, multi-center, simulation-based study to evaluate the impact of various Personal Protective Equipment (PPE) types on the time to successful completion of simulated, life-saving pediatric emergency medical procedures, including chest compression performance recorded from simulation manikins. There were two study arms: a pre-hospital arm, including registered Emergency Medical Service (EMS) providers, and a hospital arm, including registered nurses and licensed physicians. Our data suggest that the recommendation from the American Heart Association that compressors switch every 2 minutes during CPR need not be altered in the setting of PPE use. Overall, we found no significant differences in the conduct of procedures by pre-hospital medical providers while wearing PPE. Our findings also suggest that major changes to the planning or execution of most considered routine emergency procedures performed in the hospital setting are not needed when wearing PPE.
DOI: 10.57982/mq0w-1e77
title: Safety and Pharmacokinetics of Multiple Dose Metronidazole in Premature Infants

description: The primary objective of this prospective, open-label, multi-center, pharmacokinetic (PK) study was to evaluate the PK and safety of IV metronidazole in premature infants <32 weeks gestational age with suspected serious infection. There were 24 participants enrolled in two age groups based on postnatal age. Each participant received metronidazole and was included in the PK population. Plasma PK was evaluated using a limited sampling scheme. A new dosing strategy based on PMA was developed to account for developmental changes in metronidazole disposition as well as for simplicity in clinical application. The dosing of 7.5 mg/kg Q12 hours in infants PMA <34 weeks and 7.5 mg/kg Q8 hours in infants with a PMA 34-40 weeks, with a loading dose of 15 mg/kg for both age cohorts would achieve therapeutic metronidazole concentrations in the majority (>75%) of infants <90 days of age. In addition, metronidazole was well tolerated in this open-label study. None of the AEs and SAEs reported was related to study drug.
DOI: 10.57982/ewwb-h395
title: Measuring Opioid Tolerance Induced by Fentanyl (or Other Opioids)

description: To reduce pain, anxiety, or agitation, critically ill children routinely receive opioids for analgesia and sedation. Routine use of opioid therapy often leads to tolerance and dependence. The objective of this study was to examine the clinical factors associated with increased opioid dose among mechanically ventilated children. This study was carried out in 7 pediatric intensive care units from tertiary care children’s hospitals. 500 children receiving intravenous opioids between 5/09 and 10/09 were enrolled. Data on opioid use, concomitant therapy, demographic, and exploratory variables were collected for the duration of opioid therapy, 14 days after enrollment, and until PICU discharge or death. Conclusions from this study indicate that mechanically ventilated children require increasing opioid doses, often associated with prolonged opioid exposure or the need for additional sedation. Efforts to avoid prolonged opioid exposure and reduce clinical practice variation may prevent opioid therapy complications.
DOI: 10.57982/69rh-3166
title: Pharmacokinetics of Antistaphylococcal Antibiotics in Infants - Ticarcillin

description: NICHD-2012-STA01 was a multiple center, open label, multiple-dose pharmacokinetic study. The primary objective of this study was to determine the pharmacokinetics (PK) of rifampin, ticarcillin-clavulanate, and clindamycin in infants to address gaps in pediatric labeling of these drugs. Only the data for ticarcillin-clavulanate are reported here. Fifteen premature infants <30 weeks gestational age were enrolled under ticarcillin-clavulanate, all of whom reported at least one evaluable fresh plasma sample for PK analysis. All participants were included in the safety population. One mild adverse event was reported which was unrelated to ticarcillin-clavulanate.
DOI: 10.57982/x64h-gx77
title: Early Copper Histidine Therapy in Menkes Disease

description: This study evaluated the safety and efficacy of daily subcutaneous Copper Histidine (CuHis) injections given over three years to impact the neurodevelopmental outcomes in infants with Menkes disease. The impact of early (beginning within 1 month of birth corrected for prematurity) versus late CuHis treatment (beginning beyond 1 month of age and after onset of neurological symptoms) was evaluated.
DOI: 10.57982/6qhr-1243
title: Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency

description: Menkes disease and occipital horn syndrome (OHS) are forms of copper deficiency that must be diagnosed and treated very early in life to prevent serious developmental problems. However, these and other forms of copper deficiency are not very well understood, and further research is needed to determine whether certain treatments are useful in treating copper deficiency. One such treatment is copper histidinate, a new molecular entity that can be injected directly under the skin to avoid absorption through the gastrointestinal tract. This study investigated the effectiveness, side effects, and dosage of copper histidinate treatment for patients with Menkes, OHS, and unexplained copper deficiency.
DOI: 10.57982/96b0-4n94
title: Clinical Biomarkers in Alpha-Mannosidosis

description: Alpha-mannosidosis (AMD) is an inherited lysosomal storage disorder caused by mutations in the LAMAN gene, which encodes lysosomal alpha-mannosidase and is characterized by neurodevelopmental delay, mild immune deficiency, facial and skeletal abnormalities, hearing impairment, intellectual disability, muscle weakness and ataxia. Neuromuscular and skeletal deterioration progresses over decades, rendering most patients wheel-chair dependent. No consistently successful treatment is available. To better characterize the biochemical phenotype and natural history of this disorder, patients with AMD were recruited, ranging in age from five to 60 years, mainly in the US and Canada. Participants in the study visited the NIH Clinical Center for 2-3 days during which they underwent clinical and biochemical evaluations to establish reliable clinical benchmarks and to identify cerebrospinal fluid biomarkers that could serve as candidate surrogate markers of treatment effect in future clinical trials.
DOI: 10.57982/d4gq-vt04
title: Preconception Maternal Nutrition Trial Women First (Enrollment to 24 Months After Birth)

description: Multi-country three-arm, individually randomized, non-masked, controlled trial to ascertain the benefits of ensuring optimal maternal nutrition before conception and providing an evidence base for programmatic priority directed to minimizing the risk of malnutrition in all females of reproductive age. The objective is to determine the benefits to the offspring of women in poor, food-insecure environments of commencing a daily comprehensive maternal nutrition supplement (with additional balanced calorie/protein supplement for underweight participants) ? 3 months prior to conception versus the benefits of commencing the same supplement at 12 weeks gestation and also to compare offspring outcomes with those of a control group which receives no supplement.
DOI: 10.57982/4z3c-2015
title: The Molecular Basis of Inherited Reproductive Disorders

description: The aims of this study are: 1) to identify genes that play a role in human pubertal development and reproduction, 2) to characterize the phenotypic spectrum of patients with these gene defects, and 3) to discern the mode of inheritance for disorders caused by these gene defects. We are specifically interested in genes that cause Kallmann syndrome, idiopathic hypogonadotropic hypogonadism (IHH), precocious (early) puberty, and delayed puberty.
DOI: 10.57982/q0r0-vw20
title: Safety and Pharmacokinetics of Lisinopril in Pediatric Kidney Transplant Recipients

description: NICHD-2011-HTN01 was a prospective, open-label, multiple center, pharmacokinetics (PK) study of lisinopril in pediatric kidney transplant recipients, aged 2-17 years of age, with stable allograft function and need of medication therapy to control hypertension. The primary objective was to evaluate the safety profile and PK of oral lisinopril. Out of 29 screened participants, 26 received study medication at varying doses and were included in the safety population. Fifteen participants were lisinopril naive and received protocol specific treatment, while eleven received continuing lisinopril as part of standard of care treatment. Twenty-two participants had evaluable PK measurements and were included in the PK analysis population. Overall, lisinopril PK in children with a kidney transplant were comparable to historical groups of children and adults without a kidney transplant. One SAE was reported as unrelated to lisinopril, and one AE led to study drug discontinuation. Otherwise, AE rates were low.
DOI: 10.57982/xgt1-0q90
title: Pharmacokinetics of Sildenafil in Premature Infants

description: This was a phase I, open-label, pharmacokinetic (PK) study with a primary objective of determining PK of sildenafil in premature infants. The trial enrolled 25 premature infants ?28 weeks gestational age (GA) who were receiving sildenafil per local standard of care at doses between 0.5-2.25 mg/kg in cohort 1, as well as 9 premature infants ?28 weeks GA and postnatal age range 7-40 days who received a single intravenous dose of sildenafil 0.25 mg/kg or 0.125 mg/kg in cohort 2. A population PK model of sildenafil and metabolite N-desmethyl sildenafil (DMS) was developed. Fluconazole, a CYP3A4 inhibitor, was the only covariate found to explain a significant amount of the variability in sildenafil clearance. Although there is no widely accepted exposure target for sildenafil in the setting of bronchopulmonary dysplasia, simulated exposures obtained here are in agreement with previous population PK analysis performed in infants.
DOI: 10.57982/4fvt-6984
title: Critical Asthma Mortality and Morbidity Planning Study

description: A retrospective study was conducted across the 8 children’s hospitals affiliated with the NICHD CPCCRN. All children between 1 and 18 years of age with critical asthma admitted to a PICU between January 1, 2005 and December 31, 2009 who required intubation and mechanical ventilation or who died were included. Critical asthma is defined as an acute exacerbation of asthma or status asthmaticus as the primary cause for PICU admission. Patients with cystic fibrosis or bronchiolitis were excluded. If a patient was admitted to the hospital more than once during the study period, each hospitalization for critical asthma in which the patient was intubated and mechanically ventilated was included as a case. If a patient was admitted to the PICU more than once during the same hospitalization, only the first PICU admission in which the patient was intubated and mechanically ventilated was included. The study was approved and waiver of informed consent granted by the Institutional Review Board at each site.
DOI: 10.57982/57zs-q947
title: Life After Pediatric Sepsis Evaluation

description: The purpose of this prospective observational study was to describe short and long-term outcomes among children surviving septic shock. LAPSE investigated the intensity and duration of sepsis-associated morbidity that persists following the acute septic event. LAPSE described the post sepsis illness trajectory through Health Related Quality of Life (HRQL) and subject functional status (FS). LAPSE examined organ dysfunction, as well as individual and environmental characteristics that influenced these outcomes. We hypothesized that variation of HRQL and FS would be seen following survival of severe sepsis, enabling development of composite outcome measures for future trials of pediatric sepsis. This study found that 11% of the children died within 3 months of their septic shock episode. Additionally, duration of stay, cumulative vasoactive-inotropic scores, duration of mechanical ventilation, and other factors were associated with adverse outcomes.
DOI: 10.57982/aktm-zb15
title: Development of a Quantitative Functional Status Scale (FSS) for Pediatric Patients

description: The purpose of the FSS (Functional Status Scale) study was to create a functional status outcome measure for large outcome studies that is well defined, quantitative, rapid, reliable, minimally dependent on subjective assessments, and applicable to hospitalized pediatric patients across a wide range of ages and inpatient environments. This study was designed with the intention that FSS would be used throughout the world of pediatric medicine as an assessment tool. FSS domains of functioning included mental status, sensory functioning, communication, motor functioning, feeding, and respiratory status, categorized from normal (score=1) to very severe dysfunction (score=5). The Adaptive Behavior Assessment System II (ABAS II) established construct validity and calibration within domains. Primary care nurses completed the ABAS II.
DOI: 10.57982/y5cz-pp38
title: Extremely Low Birth Weight (ELBW) Infants Exposed to Furosemide or Bumetanide in the Neonatal Intensive Care Unit

description: This was a retrospective observational chart review to determine the incidence of events of special interest for hospitalized, extremely low birth weight infants admitted to one of two neonatal intensive care units and exposed to furosemide or bumetanide. The trial enrolled 679 infants, with 521 receiving furosemide treatment; 166 of these infants received bumetanide in addition to furosemide (no infants received bumetanide only). There were profound dosing differences between the two sites. Infants administered furosemide tended to be sicker than infants not administered furosemide at both sites. No association between diuretic use and death was found. Increased drug exposure was associated with higher rates of bronchopulmonary dysplasia diagnosis. Rates of hearing loss, cerebral palsy diagnosis, and abnormal Bayley scores were low (<15%) at each site. Rates of both hearing loss and kidney stones differed between sites.