What was the research about?
Acute brain injuries from car crashes, falls, and tumors can cause long-term health problems in children and teens. After brain injuries, children may have a hard time speaking, moving, or thinking clearly. Different types of therapy may help. Therapy may include
- Physical therapy to help children move better
- Occupational therapy to help children do everyday things like dressing and playing
- Speech therapy to help children put words together and talk clearly
Most children with brain injuries first receive treatment in a pediatric intensive care unit, or PICU. Doctors don’t know if giving therapy during the PICU stay will help prevent future problems. In this study, the research team looked at whether giving rehab therapies to children with brain injuries within three days of being in the PICU was possible and safe. They wanted to compare children who received early therapy with children who received usual care. In usual care, children received therapy if and when their doctors recommended it. The team looked at the children’s ability to do everyday activities and their quality of life. They also asked about how the family was doing.
What were the results?
The number of children who joined and stayed in the study for six months was too small for the research team to compare children who received early therapy with those who received usual care. As a result, the research team couldn’t say whether early therapy helped the children and their families.
The study did show it was possible to give early therapy to children in the PICU. Therapists did not record any serious harm resulting from this care.
Who was in the study?
The study included 58 children ages 3 to 17 with acute brain injuries and their parents or guardians. The children were receiving care in PICUs in Pittsburgh, Pennsylvania; Cincinnati, Ohio; and Chicago, Illinois. Of the children, 69 percent were white, 17 percent were black, and 4 percent were Asian; 10 percent were Hispanic. The average age was 11, and 60 percent were boys. Most children had been in an accident or had a brain tumor.
What did the research team do?
The research team assigned children by chance to one of two groups. Children in one group got therapy within three days of arriving in the PICU. Those in the second group received therapy if and when their doctors recommended it.
Parents or guardians filled out surveys about their children’s quality of life and the everyday tasks they could do before their brain injuries. They also answered questions about how their families were doing before their children were injured. They filled out the same surveys six months later.
Parents of children who had brain injuries or other disabilities, doctors, and a patient organization worked with the research team on the study.
What were the limits of the study?
Only 39 parents or guardians filled out surveys six months after their children’s injuries. Because the study was small, the research team couldn’t compare the two groups as planned. When studies are small, it’s hard to tell whether any results are because of chance or differences in treatment.
Future research could look at how to increase the number of children receiving care in PICUs who take part in and stay in studies.
How can people use the results?
PICUs can use the results when considering early therapy for children with brain injuries.
To compare the effectiveness of early rehabilitation versus usual care on improving functional status, quality of life, and family functioning in children with acute brain injuries receiving treatment in a pediatric intensive care unit (PICU).
|Design||Randomized controlled trial|
|Population||58 patients between the ages of 3 and 17 admitted to a PICU with traumatic or nontraumatic acute brain injuries and their parents or guardians|
Primary: physical, cognitive, and emotional functional status; quality of life; family functioning
Secondary: feasibility, adverse events of early rehabilitation
|Timeframe||6-month follow-up for primary outcomes|
This multicenter, randomized controlled trial compared the effectiveness of early rehabilitation versus usual care rehabilitation on improving functioning and quality of life for children with acute brain injuries admitted to a PICU.
Researchers randomly assigned patients to early rehabilitation or to usual care. For patients in the early rehabilitation group, therapists offered physical therapy (PT), occupational therapy (OT), and speech-language therapy (SLT) within 72 hours of PICU admission. Therapists offered patients in the usual care group these therapies when recommended by a member of the treatment team. As a result, rehabilitation in the usual care group could occur in the PICU, in the ward after PICU discharge, or not at all.
The study included 58 patients ages 3 to 17 with acute brain injuries and their parents or guardians. The patients were receiving care at PICUs in Pittsburgh, Pennsylvania; Cincinnati, Ohio; and Chicago, Illinois. Of the patients, 69% were white, 17% were black, and 4% were Asian; 10% were Hispanic. The mean age was 11, and 60% were male. The most frequent diagnoses were traumatic brain injury and brain mass.
Parents or guardians completed surveys on their child’s functional status and quality of life and their family’s functioning before the injury based on their recollections. They completed the same surveys six months later.
For both groups, researchers recorded PT, OT, and SLT sessions via checklists and chart review during the entire hospital stay, including in the PICU and in the ward. Therapists recorded adverse events at every attempted therapy session using prespecified checklists.
Parents of children who had experienced acute brain injuries or had disabilities, clinicians, hospital administrators, and a patient advocacy organization worked with researchers to plan and conduct the study.
Researchers could not recruit the planned number of patients into the study, and only 39 parents or guardians completed functional status, quality-of-life, and family functioning surveys at six months. Therefore, the trial was underpowered to detect differences in primary outcomes.
However, the study demonstrated the feasibility of offering PICU-based early rehabilitation. Children in the early intervention group received PT, OT, and SLP in the PICU.
Therapists did not record any therapy-associated serious adverse events in either group.
The sample size was too small to detect differences in primary outcomes between the two groups.
Conclusions and Relevance
Researchers could not compare the difference in six-month functional status, quality of life, and family functioning between the early rehabilitation and usual care groups. Providing PICU-based early PT, OT, and SLT to children with acute brain injuries was feasible.
Future Research Needs
Future research could address the challenges of recruiting and retaining patients receiving care in the PICU for randomized trials.
Final Research Report
View this project's final research report.
Related Journal Citations
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:
- Reviewers asked how the researchers recruited participants for the survey portion of the study and how that would affect interpretation of survey results. The researchers added more detail about their survey methods to the report. However, they also admitted that they were unable to provide an accurate survey response rate since the survey link was on a publicly available website to encourage multi-national responses.
- Reviewers said the report was unclear in its description of the handling of missing data, and they expressed concern that missing data were not handled as expected in the PCORI Methodology Standards. The researchers responded that they did not impute missing data for the primary outcomes and that by far the largest reason for missing primary outcomes data was being unable to contact families to assess six-month outcomes. There were few missing data for other study elements. The researchers indicated that they were not able to complete imputation analyses at this time.
- Reviewers stressed that this study was not a pilot or feasibility study but was a clinical trial that was not able to test its hypotheses because of underenrollment and significant loss to follow-up. Given the limited conclusions that could be drawn from an incomplete trial, the reviewers considered the comments on clinical implications and generalizability to be unwarranted. The researchers revised their discussion of the clinical trial, focusing more on lessons for future research and less on the clinical implications of this study’s results.
- Reviewers had difficulty understanding the rationale for the biomarker component of this study, and particularly the advantages of assessing a functional outcome by biomarker rather than by less invasive means. The researchers indicated that brain injury biomarkers have been used as diagnostic and prognostic tools, and trajectories of biomarkers could be informative in understanding changes in functioning after brain injury.
Conflict of Interest Disclosures
Study Registration Information
- Has Results