Heidi Castillo

Effects of bisphosphonates in children with osteogenesis imperfecta: an AACPDM systematic review

This systematic review of the effects of bisphosphonate treatment in children with osteogenesis imperfecta was conducted using the American Academy for Cerebral Palsy and Developmental Medicine methodology for developing systematic reviews of treatment interventions (Revision 1.1) 2004. Despite a large body of published literature, there have been only eight studies with a sufficiently high level of internal validity to be truly informative. These studies confirm improvement in bone density. Many, but not all studies, demonstrate reduction in fracture rate and enhanced growth. There has been extremely limited evaluation of broader treatment impacts such as deformity, need for orthopedic surgery, pain, functioning, or quality of life. Short‐term side effects were minimal. Which medication and dosing regimen is optimal and how long patients should be treated are unclear. This body of evidence would be strengthened by a larger controlled trial, because many studies lacked adequate power to evaluate stated outcomes. These studies do not address the impacts of bisphosphonates in children with milder forms of osteogenesis imperfecta and severe forms that are not due to mutations in the type I pro‐collagen gene (e.g. types VII and VIII). Additional research is needed into treatment of infants. More studies evaluating medication choices, optimal dosing, duration of treatment, post‐treatment impacts, and long‐term side effects are necessary.

Testing the feasibility of a National Spina Bifida Patient Registry

BACKGROUND The purpose of this study was to describe the development and early implementation of a national spina bifida (SB) patient registry, the goal of which is to monitor the health status, clinical care, and outcomes of people with SB by collecting and analyzing patient data from comprehensive SB clinics. METHODS Using a web-based, SB-specific electronic medical record, 10 SB clinics collected health-related information for patients diagnosed with myelomeningocele, lipomyelomeningocele, fatty filum, or meningocele. This information was compiled and de-identified for transmission to the Centers for Disease Control and Prevention (CDC) for quality control and analysis. RESULTS A total of 2070 patients were enrolled from 2009 through 2011: 84.9% were younger than 18 years of age; 1095 were women; 64.2% were non-Hispanic white; 6.5% were non-Hispanic black or African American; and 24.2% were Hispanic or Latino. Myelomeningocele was the most common diagnosis (81.5%). CONCLUSIONS The creation of a National Spina Bifida Patient Registry partnership between the CDC and SB clinics has been feasible. Through planned longitudinal data collection and the inclusion of additional clinics, the data generated by the registry will become more robust and representative of the population of patients attending SB clinics in the United States and will allow for the investigation of patient outcomes.

The Resident Decision-Making Process in Global Health Education Appraising Factors Influencing Participation

The globalization of pediatric graduate medical education is ongoing; thus, this study was conducted to begin to explore the nature of resident interest in global health (GH) training and to further identify potentially modifiable factors influencing participation in away rotations. The authors surveyed all residents at Cincinnati Children’s Hospital Medical Center to identify factors influencing participation in education efforts and away rotations. With a participation rate of 79.4% (n = 143), 5 key factors emerged as most significant in the decision-making process amid all participants. Among residents who had previous experience, 82.1% were interested in participating in an away elective compared with 58.3% of those without experience (P = .002). Residents with previous experience abroad were also more likely to plan to integrate GH into their careers (61.7% vs 26.7%, P < .0001). This article describes specific obstacles to resident participation in GH education and documents the association between previous experience and significant interest in long-term involvement.

Global health opportunities within pediatric subspecialty fellowship training programs: surveying the virtual landscape

BackgroundThere is growing interest in global health among medical trainees. Medical schools and residencies are responding to this trend by offering global health opportunities within their programs. Among United States (US) graduating pediatric residents, 40% choose to subspecialize after residency training. There is limited data, however, regarding global health opportunities within traditional post-residency, subspecialty fellowship training programs. The objectives of this study were to explore the availability and type of global health opportunities within Accreditation Council for Graduate Medical Education (ACGME)-accredited pediatric subspecialty fellowship training programs, as noted by their online report, and to document change in these opportunities over time.MethodsThe authors performed a systematic online review of ACGME-accredited fellowship training programs within a convenience sample of six US pediatric subspecialties. Utilizing two data sources, the American Medical Association-Fellowship and Residency Electronic Interactive Database Access (AMA-FREIDA) and individual program websites, all programs were coded for global health opportunities and opportunity types were stratified into predefined categories. Comparisons were made between 2008 and 2011 using Fisher exact test. All analyses were conducted using SAS Software v. 9.3 (SAS Institute Inc., Cary, NC).ResultsOf the 355 and 360 programs reviewed in 2008 and 2011 respectively, there was an increase in total number of programs listing global health opportunities on AMA-FREIDA (16% to 23%, p=0.02) and on individual program websites (8% to 16%, p=0.004). Nearly all subspecialties had an increased percentage of programs offering global health opportunities on both data sources; although only critical care experienced a significant increase (p=0.04, AMA-FREIDA). The types of opportunities differed across all subspecialties.ConclusionsGlobal health opportunities among ACGME-accredited pediatric subspecialty fellowship programs are limited, but increasing as noted by their online report. The availability and types of these opportunities differ by pediatric subspecialty.

Longitudinal Study of Bladder Continence in Patients with Spina Bifida in the National Spina Bifida Patient Registry

Purpose Achieving bladder continence in individuals with spina bifida is a lifetime management goal. We investigated bladder continence status through time and factors associated with this status in patients with spina bifida. Materials and Methods We used National Spina Bifida Patient Registry data collected from 2009 through 2015 and applied generalized estimating equation models to analyze factors associated with bladder continence status. Results This analysis included 5,250 participants with spina bifida in a large, multi‐institutional patient registry who accounted for 12,740 annual clinic visit records during the study period. At last followup mean age was 16.6 years, 22.4% of participants had undergone bladder continence surgery, 92.6% used some form of bladder management and 45.8% reported bladder continence. In a multivariable regression model the likelihood of bladder continence was significantly greater in those who were older, were female, were nonHispanic white, had a nonmyelomeningocele diagnosis, had a lower level of lesion, had a higher mobility level and had private insurance. Continence surgery history and current management were also associated with continence independent of all other factors (adjusted OR and 95% CI 1.9, 1.7–2.1 and 3.8, 3.2–4.6, respectively). The association between bladder management and continence was stronger for those with a myelomeningocele diagnosis (adjusted OR 4.6) than with nonmyelomeningocele (adjusted OR 2.8). Conclusions In addition to demographic, social and clinical factors, surgical intervention and bladder management are significantly and independently associated with bladder continence status in individuals with spina bifida. The association between bladder management and continence is stronger in those with myelomeningocele.

Early postnatal bladder function in fetoscopic myelomeningocele repair patients

PURPOSE Prenatal repair of myelomeningocele (MMC) via hysterotomy has demonstrated neurosurgical and motor benefits, when compared to postnatal repairs. Urologic benefits, however, remain to be seen. The purpose of this study was to review early postnatal bladder function in patients undergoing a novel endoscopic approach for MMC repair using an exteriorized uterus. METHODS A prospective urologic assessment of patients undergoing fetoscopic MMC repair and receiving subsequent care at our facility, was performed. Patients were managed and urodynamic studies risk-stratified according to the Centers for Disease Control and Prevention Urologic and Renal Protocol for the Newborn and Young Child with Spina Bifida. RESULTS Fetoscopic MMC repair was performed in 14 patients. No patients had hydronephrosis or bladder thickening at birth. Detrusor overactivity was observed in nine (64.3%) patients. Impaired compliance was seen in eight (57.1%) patients. No patients had a detrusor leak point pressure of > 40 cm H2O or evidence of detrusor sphincter dyssynergia. Three (21.4%) patients had vesicoureteral reflux, seven (50.0%) had an open bladder neck, and none had trabeculated bladders. CONCLUSION In this early experience with fetoscopic MMC repair, postnatal bladder function does not appear to be any worse than that of previously reported prenatal or postnatal closures.

Decompression for Chiari malformation type II in individuals with myelomeningocele in the National Spina Bifida Patient Registry

OBJECTIVEThe purpose of this study was to determine the rate of decompression for Chiari malformation type II in individuals with myelomeningocele in the National Spina Bifida Patient Registry (NSBPR). In addition, the authors explored the variation in rates of Chiari II decompression across NSBPR institutions, examined the relationship between Chiari II decompression and functional lesion level of the myelomeningocele, age, and need for tracheostomy, and they evaluated for temporal trends in rates of Chiari II decompression.METHODSThe authors queried the NSBPR to identify all individuals with myelomeningocele between 2009 and 2015. Among these patients, they identified individuals who had undergone at least 1 Chiari II decompression as well as those who had undergone tracheostomy. For each participating NSBPR institution, the authors calculated the proportion of patients enrolled at that site who underwent Chiari II decompression. Logistic regression was performed to analyze the relationship between Chiari II decompression, functional lesion level, age at decompression, and history of tracheostomy.RESULTSOf 4448 individuals with myelomeningocele identified from 26 institutions, 407 (9.15%) had undergone at least 1 Chiari II decompression. Fifty-one patients had undergone tracheostomy. Logistic regression demonstrated a statistically significant relationship between Chiari II decompression and functional lesion level of the myelomeningocele, with a more rostral lesion level associated with a higher likelihood of posterior fossa decompression. Similarly, children born before 2005 and those with history of tracheostomy had a significantly higher likelihood of Chiari II decompression. There was no association between functional lesion level and need for tracheostomy. However, among those children who underwent Chiari II decompression, the likelihood of also undergoing tracheostomy increased significantly with younger age at decompression.CONCLUSIONSThe rate of Chiari II decompression in patients with myelomeningocele in the NSBPR is consistent with that in previously published literature. There is a significant relationship between Chiari II decompression and functional lesion level of the myelomeningocele, which has not previously been reported. Younger children who undergo Chiari II decompression are more likely to have undergone tracheostomy. There appears to be a shift away from Chiari II decompression, as children born before 2005 were more likely to undergo Chiari II decompression than those born in 2005 or later.

Variation in bowel and bladder continence across US spina bifida programs: A descriptive study

PURPOSE Continence is low in individuals with spina bifida, but published prevalence varies markedly across studies. The objective of this study was to examine bladder and bowel continence among patients served by multidisciplinary clinics participating in the National Spina Bifida Patient Registry and to examine whether variation in prevalence exists across clinics. METHODS Data were obtained from patients 5 years and older from March 2009 to December 2012. Data were gathered at clinic visits using standardized definitions. RESULTS Data from 3252 individuals were included. Only 40.8% of participants were continent of urine; 43% were continent of stool. Bladder and bowel continence differed by spina bifida type, with those with myelomeningocele having significantly lower reported prevalence of continence than those with other forms of spina bifida. Bladder and bowel continence varied across registry sites. Adjustment based on demographic and condition-specific variables did not make substantive differences in prevalence observed. CONCLUSION Less than half of spina bifida patients served in multidisciplinary clinics report bladder or bowel continence. Variability in prevalence was observed across clinics. Further research is needed to examine if clinic-specific variables (e.g., types of providers, types of interventions used) account for the observed variation.