# Improving infant hydrocephalus outcomes in Uganda: Predicting developmental outcomes and identifying patients at risk for early treatment failure after ETV/CPC

> **NIH NIH R01** · BOSTON CHILDREN'S HOSPITAL · 2022 · $515,374

## Abstract

Project Summary/Abstract
Infant hydrocephalus is a serious global health problem, with an estimated 400,000 new cases annually. Nearly
half of these occur in sub-Saharan Africa, with neonatal infection being the most common cause there. Untreated,
hydrocephalus causes progressive brain injury and even death. The standard treatment has long been
placement of a ventriculoperitoneal shunt (VPS) but these devices require life-long maintenance and nearly all
fail multiple times. Because of this, shunt-dependence is more dangerous in LMIC where emergency access to
neurosurgical care is difficult or impossible. CURE Children’s Hospital of Uganda (CCHU) developed an alternate
treatment combining endoscopic third ventriculostomy with choroid plexus cauterization (ETV/CPC) to give
patients a shunt-free life. An NIH-funded, randomized controlled trial of ETV/CPC vs VPS for infants with post-
infectious hydrocephalus (PIH) at CCHU found no significant difference at one-year post-treatment in brain
growth or developmental outcomes. Treatment failure is a significant problem for both, occurring in a third.
Developmental outcomes were associated with the amount of brain growth, but not with traditional metrics of
head or ventricle size. Treatment has always focused on controlling ventricle size, but this has little association
with outcomes. Brain growth is a better indicator, and we hypothesize in PIH it depends in part on the integrity
of the underlying brain matter after infection and in part on the cerebral circulation’s ability to support regrowth.
Consequently, we hypothesize cerebral blood flow and metabolism are more representative of disease severity
and treatment response. We developed a novel, combined frequency-domain near-infrared and diffuse
correlation spectroscopies (FDNIRS/DCS) technology to measure cerebral blood flow (CBF) and oxygen
metabolism (CMRO2) non-invasively at the bedside. In pilot studies at BCH and CCHU, we found untreated
hydrocephalus progressively impedes cerebral perfusion and depresses metabolism, while successful treatment
immediately restores CBF and CMRO2. Moreover, greater CMRO2 immediately after surgery correlated with
better brain growth six months later. We now propose validating these results in a larger group with the goal of
leading hydrocephalus treatment away from targeting ventricle size and directing it instead towards measurable
improvements in the brain’s physiological health. Such a paradigm shift will have substantial clinical impact in
developing countries, the United States, and the rest of the world.

## Key facts

- **NIH application ID:** 10428554
- **Project number:** 5R01HD096693-05
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** Pei-Yi Lin
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $515,374
- **Award type:** 5
- **Project period:** 2018-09-13 → 2025-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10428554

## Citation

> US National Institutes of Health, RePORTER application 10428554, Improving infant hydrocephalus outcomes in Uganda: Predicting developmental outcomes and identifying patients at risk for early treatment failure after ETV/CPC (5R01HD096693-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10428554. Licensed CC0.

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