Results Summary

What was the research about?

Transverse myelitis, or TM, is a rare disease that causes inflammation of part of the spinal cord. TM can cause pain, numbness, muscle weakness, paralysis, and problems with bladder and bowel control. Because TM is a rare disease, few studies have looked at how well treatments for TM work.

In this study, the research team looked at whether a treatment called plasma exchange, or PLEX, helped improve physical function in children with TM. PLEX filters certain proteins out of a person’s blood and replaces the proteins with man-made protein or with plasma from a donor. The team wanted to compare children with TM who received PLEX with children who didn’t receive it.

What were the results?

Fewer children with TM joined the study than planned. As a result, the study couldn’t compare children with TM who received PLEX with children who didn’t receive it.

Among children with TM in the study, 11 received PLEX. In eight of these children, PLEX followed another treatment. Four had improved physical function one year after treatment. Three children with TM had PLEX alone. All three children had improved physical function one year after treatment.

During the study, the research team found a new type of TM called acute flaccid myelitis, or AFM. The original type of TM and AFM differ. As a result, the study looked at the children with TM and AFM separately. The study found that PLEX may help some children with AFM. But given the small number of children with AFM who received treatment, these findings aren’t certain.

Who was in the study?

The study included 39 children with TM and 51 children with AFM ages 0–18 who were diagnosed between 2014 and 2018. To take part in the study, children had to be diagnosed in the previous six months. Of the children, 79 percent were white, 6 percent were African American, 6 percent were Asian, 6 percent indicated more than one race, and 4 percent didn’t report race; 17 percent were Hispanic or Latino. The average age at diagnosis was 7, and 60 percent were boys.

What did the research team do?

The research team worked with a patient advocacy group called the Transverse Myelitis Association to recruit children with TM. The association recruited children online and from seven hospitals that treat TM in the United States.

The research team reviewed health records to see whether children received PLEX. At 6 and 12 months after the study began, doctors looked at physical function in children who were recruited from hospitals.

Adult patients with TM, families of children with TM, and the Transverse Myelitis Association helped design the study.

What were the limits of the study?

Fewer patients joined the study than planned. Also, the study couldn’t look at data for children with TM and AFM together. As a result, the study had too few children to compare treatments.

Future research could continue to look at treatments for TM and AFM.

How can people use the results?

Researchers can use the results when planning studies for children with TM and AFM.

Final Research Report

View this project's final research report.

Peer-Review Summary

Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.

The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments. 

Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:

  • The reviewers noted that the original goals of this study, comparing different treatments for transverse myelitis (TM), could not be accomplished as planned because the researchers could not meet recruitment goals. However, the study did provide important information about a related disease, acute flaccid myelitis (AFM), that saw a surge in diagnoses during the study. The reviewers accepted the change in the study’s goal, from comparing treatments for TM to comparing AFM to two types of TM. The reviewers’ comments primarily asked the researchers to clarify how they conducted their analyses. The researchers also simplified some of the descriptions of diagnostic similarities and differences between AFM and TM.

Conflict of Interest Disclosures

Project Information

Benjamin M. Greenberg, MD, MHS
University of Texas Southwestern Medical Center
Collaborative Assessment of Pediatric Transverse Myelitis: Understand, Reveal, Educate (CAPTURE) Study

Key Dates

September 2013
October 2020

Study Registration Information


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Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
Populations Populations PCORI is interested in research that seeks to better understand how different clinical and health system options work for different people. These populations are frequently studied in our portfolio or identified as being of interest by our stakeholders. View Glossary
Intervention Strategy Intervention Strategies PCORI funds comparative clinical effectiveness research (CER) studies that compare two or more options or approaches to health care, or that compare different ways of delivering or receiving care. View Glossary
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Last updated: January 25, 2023