Project Summary  

This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.

What is the research about?

Chiari type I malformation is a disorder where brain tissue extends into the spinal cord. Doctors usually diagnose this health problem in late childhood or early adulthood. Patients who have Chiari type I malformation and syringomyelia, a cyst or fluid-filled sac in the spinal cord, can have headaches and a hard time talking, swallowing, and breathing.

Doctors use two types of surgery to treat Chiari type I malformation and syringomyelia. In the first type, called posterior fossa decompression, the surgeon removes a small section of bone at the back of the skull to reduce pressure on the brain. In the second type of surgery, called duraplasty, the surgeon does the same procedure and opens the cover of the brain, called the dura. The surgeon then sews in a patch of tissue to make the dura bigger and relieve pressure on the brain.

One in five patients have complications from duraplasty, such as infection. To avoid complications, some patients opt not to have duraplasty. But if decompression without duraplasty does not ease symptoms, patients may need to have a second surgery.

Doctors don’t know whether either type of surgery is more effective for relieving symptoms. In this study, the research team is comparing decompression with and without duraplasty to see how well each type of surgery relieves symptoms and if either causes less harm. The team is also comparing the two approaches on quality of life after surgery.

Who can this research help?

Study results may help patients with Chiari type I malformation and syringomyelia make decisions with their doctors about surgery options.

What is the research team doing?

The research team is enrolling 148 patients ages 0 to 21 years with Chiari type I malformation and syringomyelia. The team is assigning patients, by chance, to have decompression surgery with or without duraplasty. Patients return to the surgeon’s office three times after surgery for a nervous system exam. At these visits, patients complete two surveys about their symptoms and quality of life. One year after surgery, patients have a brain and spine scan to measure the size of their cyst.

Clinicians, caregivers, and patients help plan and monitor the study and share results.

Research methods at a glance

Design Elements	Description
Design	Randomized controlled trial
Population	Children and young adults ≤21 years old with Chiari malformation type I ≥5 mm tonsillar ectopia, syrinx between 3 mm and 6 mm, and Chiari Severity Index classification 1
Interventions/ Comparators	Posterior fossa decompression surgery Posterior fossa decompression surgery with duraplasty
Outcomes	Primary: complications of surgery (e.g., cerebrospinal fluid leaks, infections, meningitis) Secondary: size of syrinx, quality of life
Timeframe	12-month follow-up for primary outcome

Project Details

Principal Investigator

David Limbrick, MD, MS, PhD

Project Status

In progress; Enrollment complete

Project Title

Posterior Fossa Decompression with or without Duraplasty for Chiari Type I Malformation with Syringomyelia

Board Approval Date

September 2015

Project End Date

June 2021

Organization

Washington University School of Medicine

Year Awarded

2015

State

Missouri

Project Type

Research Project

Health Conditions  

Neurological Disorders

Rare Diseases

Intervention Strategies

Other Clinical Interventions
Training and Education Interventions

Populations

Children -- 18 and under
Individuals with Rare Disease

Funding Announcement

Assessment of Prevention, Diagnosis, and Treatment Options

Project Budget

$2,590,076

Study Registration Information

HSRP20162124

NCT02669836