# Endoscopic versus Shunt Treatment of Hydrocephalus in Infants

> **NIH NIH U01** · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · 2022 · $1,492,187

## Abstract

Hydrocephalus primarily affects infants under one year of age. The traditional treatment for hydrocephalus is
cerebrospinal fluid (CSF) shunt. Life-time complications of CSF shunts are high and expensive, with annual
hospital charges of nearly $2.0 billion, accounting for 3.1% of all pediatric hospital charges. As shunt
complications also adversely impact quality of life, it is not surprising that surveys of families affected by
hydrocephalus show that they desperately desire shunt-free treatment options. The most promising shunt-free
treatment for infant hydrocephalus is endoscopic third ventriculostomy with choroid plexus cauterization
(ETV+CPC), which our Hydrocephalus Clinical Research Network (HCRN) and others have shown to be safe
and viable. However, the cognitive outcome of ETV+CPC compared to shunt is not known. Our data suggest
that most families would accept the risks of ETV+CPC for the chance to be free of shunt, but only if initial
treatment with ETV+CPC will not lead to noticeable sacrifice in cognitive outcome compared to shunting. With
this U01 proposal, we aim: 1. To determine, in infants ≤12 months corrected age, with hydrocephalus requiring
treatment at tertiary care pediatric neurosurgery centers in North America, does ETV+CPC compared to shunt
result in non-inferior cognitive outcome at 12 months from surgery, as measured by Bayley Scales of Infant and
Toddler Development (Bayley-III) Cognitive Scale with a non-inferiority margin of 1.5. 2a. To determine, in
the same cohort, if ETV+CPC compared to shunt results in non-inferior Bayley-III Motor/Language Scales or
Vineland-3 Scales. 2b. To compare other measures of treatment performance (brain/ventricle volume,
treatment failure, hospital days, repeat surgery, use of imaging) and complications between treatment arms.
3a. To determine the effect of ETV+CPC and shunt on cerebral structural connectivity, with diffusion MR
fractional anisotropy (FA) in the corpus callosum 12 months after surgery. 3b. To define the relationships
between pre-operative brain/ventricular volume and cerebrospinal fluid (CSF) NCAM-1 level to post-operative
corpus callosum FA and Bayley-III Cognitive Scale 12 months after surgery. 3c. To define the relationships
between change in brain/ventricular volume (pre-operatively to 12 months post-operatively) to FA in the
corpus callosum and corticospinal tract and Bayley-III Cognitive and Motor Scales 12 months post-operatively.
To do this, we plan an RCT comparing ETV+CPC and shunt in infants with hydrocephalus, within the HCRN, a
committed group of 14 leading North American pediatric neurosurgical centers with a long track-record of
successful collaborative clinical research and RCTs in hydrocephalus. Optimal cognitive outcome, the primary
concern of families, will therefore, be our primary outcome. Assessment of dMRI, a validated, non-invasive
method of measuring white matter microstructural integrity and structural connectivity in the developing
brain, will ...

## Key facts

- **NIH application ID:** 10242095
- **Project number:** 5U01NS107486-03
- **Recipient organization:** UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH
- **Principal Investigator:** Richard Holubkov
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $1,492,187
- **Award type:** 5
- **Project period:** 2019-09-15 → 2027-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10242095

## Citation

> US National Institutes of Health, RePORTER application 10242095, Endoscopic versus Shunt Treatment of Hydrocephalus in Infants (5U01NS107486-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10242095. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*