Paul Teng, Sidney W. Gross, and Charles M. Newman
Paul Gross, Gavin T. Reed, Rachel Engelmann, and John R. W. Kestle
Funding of hydrocephalus research is important to the advancement of the field. The goal of this paper is to describe the funding of hydrocephalus research from the National Institutes of Health (NIH) over a recent 10-year period.
The NIH online database RePORT (Research Portfolio Online Reporting Tools) was searched using the key word “hydrocephalus.” Studies were sorted by relevance to hydrocephalus. The authors analyzed funding by institute, grant type, and scientific approach over time.
Over $54 million was awarded to 59 grantees for 66 unique hydrocephalus proposals from 48 institutions from 2002 to 2011. The largest sources of funding were the National Institute of Neurological Disease and Stroke and the National Institute of Child Health and Human Development. Of the total, $22 million went to clinical trials, $15 million to basic science, and $10 million to joint ventures with small business (Small Business Innovation Research or Small Business Technology Transfer). Annual funding varied from $2.3 to $8.1 million and steadily increased in the second half of the observation period. The number of new grants also went from 15 in the first 5 years to 27 in the second 5 years. A large portion of the funding has been for clinical trials. Funding for shunt-device development grew substantially. Support for training of hydrocephalus investigators has been low.
Hydrocephalus research funding is low compared with that for other conditions of similar health care burden. In addition to NIH applications, researchers should pursue other funding sources. Small business collaborations appear to present an opportunity for appropriate projects.
James P. McAllister II, Michael A. Williams, Marion L. Walker, John R. W. Kestle, Norman R. Relkin, Amy M. Anderson, Paul H. Gross, and Samuel R. Browd
Building on previous National Institutes of Health-sponsored symposia on hydrocephalus research, “Opportunities for Hydrocephalus Research: Pathways to Better Outcomes” was held in Seattle, Washington, July 9–11, 2012. Plenary sessions were organized into four major themes, each with two subtopics: Causes of Hydrocephalus (Genetics and Pathophysiological Modifications); Diagnosis of Hydrocephalus (Biomarkers and Neuroimaging); Treatment of Hydrocephalus (Bioengineering Advances and Surgical Treatments); and Outcome in Hydrocephalus (Neuropsychological and Neurological). International experts gave plenary talks, and extensive group discussions were held for each of the major themes.
The conference emphasized patient-centered care and translational research, with the main objective to arrive at a consensus on priorities in hydrocephalus that have the potential to impact patient care in the next 5 years. The current state of hydrocephalus research and treatment was presented, and the following priorities for research were recommended for each theme. 1) Causes of Hydrocephalus—CSF absorption, production, and related drug therapies; pathogenesis of human hydrocephalus; improved animal and in vitro models of hydrocephalus; developmental and macromolecular transport mechanisms; biomechanical changes in hydrocephalus; and age-dependent mechanisms in the development of hydrocephalus. 2) Diagnosis of Hydrocephalus—implementation of a standardized set of protocols and a shared repository of technical information; prospective studies of multimodal techniques including MRI and CSF biomarkers to test potential pharmacological treatments; and quantitative and cost-effective CSF assessment techniques. 3) Treatment of Hydrocephalus—improved bioengineering efforts to reduce proximal catheter and overall shunt failure; external or implantable diagnostics and support for the biological infrastructure research that informs these efforts; and evidence-based surgical standardization with longitudinal metrics to validate or refute implemented practices, procedures, or tests. 4) Outcome in Hydrocephalus—development of specific, reliable batteries with metrics focused on the hydrocephalic patient; measurements of neurocognitive outcome and quality-of-life measures that are adaptable, trackable across the growth spectrum, and applicable cross-culturally; development of comparison metrics against normal aging and sensitive screening tools to diagnose idiopathic normal pressure hydrocephalus against appropriate normative age-based data; better understanding of the incidence and prevalence of hydrocephalus within both pediatric and adult populations; and comparisons of aging patterns in adults with hydrocephalus against normal aging patterns.
William E. Whitehead, Jay Riva-Cambrin, John C. Wellons III, Abhaya V. Kulkarni, David D. Limbrick Jr., Vanessa L. Wall, Curtis J. Rozzelle, Todd C. Hankinson, Patrick J. McDonald, Mark D. Krieger, Ian F. Pollack, Mandeep S. Tamber, Jonathan Pindrik, Jason S. Hauptman, Robert P. Naftel, Chevis N. Shannon, Jason Chu, Eric M. Jackson, Samuel R. Browd, Tamara D. Simon, Richard Holubkov, Ron W. Reeder, Hailey Jensen, Jenna E. Koschnitzky, Paul Gross, James M. Drake, and John R. W. Kestle
The primary objective of this trial was to determine if shunt entry site affects the risk of shunt failure.
The authors performed a parallel-design randomized controlled trial with an equal allocation of patients who received shunt placement via the anterior entry site and patients who received shunt placement via the posterior entry site. All patients were children with symptoms or signs of hydrocephalus and ventriculomegaly. Patients were ineligible if they had a prior history of shunt insertion. Patients received a ventriculoperitoneal shunt after randomization; randomization was stratified by surgeon. The primary outcome was shunt failure. The planned minimum follow-up was 18 months. The trial was designed to achieve high power to detect a 10% or greater absolute difference in the shunt failure rate at 1 year. An independent, blinded adjudication committee determined eligibility and the primary outcome. The study was conducted by the Hydrocephalus Clinical Research Network.
The study randomized 467 pediatric patients at 14 tertiary care pediatric hospitals in North America from April 2015 to January 2019. The adjudication committee, blinded to intervention, excluded 7 patients in each group for not meeting the study inclusion criteria. For the primary analysis, there were 229 patients in the posterior group and 224 patients in the anterior group. The median patient age was 1.3 months, and the most common etiologies of hydrocephalus were postintraventricular hemorrhage secondary to prematurity (32.7%), myelomeningocele (16.8%), and aqueductal stenosis (10.8%). There was no significant difference in the time to shunt failure between the entry sites (log-rank test, stratified by age < 6 months and ≥ 6 months; p = 0.061). The hazard ratio (HR) of a posterior shunt relative to an anterior shunt was calculated using a univariable Cox regression model and was nonsignificant (HR 1.35, 95% CI, 0.98–1.85; p = 0.062). No significant difference was found between entry sites for the surgery duration, number of ventricular catheter passes, ventricular catheter location, and hospital length of stay. There were no significant differences between entry sites for intraoperative complications, postoperative CSF leaks, pseudomeningoceles, shunt infections, skull fractures, postoperative seizures, new-onset epilepsy, or intracranial hemorrhages.
This randomized controlled trial comparing the anterior and posterior shunt entry sites has demonstrated no significant difference in the time to shunt failure. Anterior and posterior entry site surgeries were found to have similar outcomes and similar complication rates.
Syed Hassan A. Akbari, Asad A. Rizvi, Travis S. CreveCoeur, Rowland H. Han, Jacob K. Greenberg, James Torner, Douglas L. Brockmeyer, John C. Wellons III, Jeffrey R. Leonard, Francesco T. Mangano, James M. Johnston, Manish N. Shah, Bermans J. Iskandar, Raheel Ahmed, Gerald F. Tuite, Bruce A. Kaufman, David J. Daniels, Eric M. Jackson, Gerald A. Grant, Alexander K. Powers, Daniel E. Couture, P. David Adelson, Tord D. Alden, Philipp R. Aldana, Richard C. E. Anderson, Nathan R. Selden, Karin Bierbrauer, William Boydston, Joshua J. Chern, William E. Whitehead, Robert C. Dauser, Richard G. Ellenbogen, Jeffrey G. Ojemann, Herbert E. Fuchs, Daniel J. Guillaume, Todd C. Hankinson, Brent R. O’Neill, Mark Iantosca, W. Jerry Oakes, Robert F. Keating, Paul Klimo Jr., Michael S. Muhlbauer, J. Gordon McComb, Arnold H. Menezes, Nickalus R. Khan, Toba N. Niazi, John Ragheb, Chevis N. Shannon, Jodi L. Smith, Laurie L. Ackerman, Andrew H. Jea, Cormac O. Maher, Prithvi Narayan, Gregory W. Albert, Scellig S. D. Stone, Lissa C. Baird, Naina L. Gross, Susan R. Durham, Stephanie Greene, Robert C. McKinstry, Joshua S. Shimony, Jennifer M. Strahle, Matthew D. Smyth, Ralph G. Dacey Jr., Tae Sung Park, and David D. Limbrick Jr.
The goal of this study was to assess the social determinants that influence access and outcomes for pediatric neurosurgical care for patients with Chiari malformation type I (CM-I) and syringomyelia (SM).
The authors used retro- and prospective components of the Park-Reeves Syringomyelia Research Consortium database to identify pediatric patients with CM-I and SM who received surgical treatment and had at least 1 year of follow-up data. Race, ethnicity, and insurance status were used as comparators for preoperative, treatment, and postoperative characteristics and outcomes.
A total of 637 patients met inclusion criteria, and race or ethnicity data were available for 603 (94.7%) patients. A total of 463 (76.8%) were non-Hispanic White (NHW) and 140 (23.2%) were non-White. The non-White patients were older at diagnosis (p = 0.002) and were more likely to have an individualized education plan (p < 0.01). More non-White than NHW patients presented with cerebellar and cranial nerve deficits (i.e., gait ataxia [p = 0.028], nystagmus [p = 0.002], dysconjugate gaze [p = 0.03], hearing loss [p = 0.003], gait instability [p = 0.003], tremor [p = 0.021], or dysmetria [p < 0.001]). Non-White patients had higher rates of skull malformation (p = 0.004), platybasia (p = 0.002), and basilar invagination (p = 0.036). Non-White patients were more likely to be treated at low-volume centers than at high-volume centers (38.7% vs 15.2%; p < 0.01). Non-White patients were older at the time of surgery (p = 0.001) and had longer operative times (p < 0.001), higher estimated blood loss (p < 0.001), and a longer hospital stay (p = 0.04). There were no major group differences in terms of treatments performed or complications. The majority of subjects used private insurance (440, 71.5%), whereas 175 (28.5%) were using Medicaid or self-pay. Private insurance was used in 42.2% of non-White patients compared to 79.8% of NHW patients (p < 0.01). There were no major differences in presentation, treatment, or outcome between insurance groups. In multivariate modeling, non-White patients were more likely to present at an older age after controlling for sex and insurance status (p < 0.01). Non-White and male patients had a longer duration of symptoms before reaching diagnosis (p = 0.033 and 0.004, respectively).
Socioeconomic and demographic factors appear to influence the presentation and management of patients with CM-I and SM. Race is associated with age and timing of diagnosis as well as operating room time, estimated blood loss, and length of hospital stay. This exploration of socioeconomic and demographic barriers to care will be useful in understanding how to improve access to pediatric neurosurgical care for patients with CM-I and SM.