Cerebral Palsy Research Network Blog

Archive for October 2016

CPRN Completes Review of Cerebral Palsy Common Data Elements

The Cerebral Palsy Research Network (CPRN) has completed a comprehensive comparison of the National Institute of Neurological Disorders and Stroke (NINDS) Common Data Elements (CDE) for cerebral palsy to the CPRN CP registry. The CP CDEs were developed by the American Academy for Cerebral Palsy and Developmental Medicine (AACPDM) for the purpose of standardizing data collection for NINDS funded studies in CP. Standardized data collection allows the data from multiple studies to be compared to create new learning in the future.

NINDS released the CP CDEs for public review in September, 2016. CPRN had shared its CP registry elements with the NINDS/AACPDM working groups back in January, 2016 for consideration in the development of the CP CDEs. After our extensive review, we are pleased to see that the CPRN registry is very much aligned with the NINDS CDEs for CP. There were a few elements that were developed by NINDS/AACPDM that are more substantive than ours, which we have decided to adopt. There were a small number of other elements where we have recommended changes to the CDEs as part of the public comment period that closes this month. And there are some areas of necessary difference because of our focus on longitudinal research that differ from single study designs. CPRN also offered our neurosurgical and orthopedic data elements to NINDS/AACPDM for inclusion in the CDEs.

Overall, our conclusion is that the CPRN Registry will provide a rich set of patient data that can be combined with future study data funded by NIH to expand discoveries in cerebral palsy.

CPRN’s Path to a Learning Health System

Dr. Garey Noritz, one of the Cerebral Palsy Research Network’s (CPRN) Co-founder’s, co-authored an article published in Developmental Medicine and Children Neurology about his participation in a “Learning Health System” for cerebral palsy. Entitled “’Learn From Every Patient’: implementation and early results of a Learning Health System1,” describes the convergence of three major trends in medicine: electronic health/medical records (EHR/EMR), the prioritization of translational research and the need to better control health care costs.

These trends came together in a project pioneered at Nationwide Children’s Hospital (NCH) for its population with cerebral palsy. “Learn from Every Patient” (LFEP) is a single-center registry effort that sought to capture the patient information from CP clinic visits to conduct research while improving the quality of care. The CP provider team at NCH defined a set of data points that were common in each visit from a CP patient, e.g., diagnosis, type of CP, treatments, etc. These data points, historically included in the text of the doctor’s note, were represented on screens in the hospital EMR and were used during patient clinic visits.

By collecting the patient information in a structured format in the EMR rather than text in the doctor’s note, the authors were able to analyze trends in the data over a 12-month period and compare patient health outcomes among various groups that either participated in the project or were seen in another clinic.

Clinicians using the system found they were equally or more efficient than their prior method for patient care. NCH was able to increase its CP clinic volume by 20% with the same number of providers over the 12-month period studied. The study included a group of 131 patients with cerebral palsy. The impact on the health of these children was significant:

“Children treated in the LFEP Program for a 12-month period experienced a 43% reduction in total inpatient days (p=0.030 vs prior 12mo period), a 27% reduction in inpatient admissions, a 30% reduction in emergency department visits (p=0.001), and a 29% reduction in urgent care visits (p=0.046). “

In the month following the publication of this article, Dr. Rich Stevenson, a CPRN member, wrote a commentary that was published in the journal entitled “Integration of research and clinical practice: the future is now2” where he not only extolled the virtues of LFEP but also its extension in implementation in CPRN. He concluded his commentary by saying:

“Through their foundational work, the ‘Learn from Every Patient’ Study Group has in fact built a template for the data collection in both efforts. They have successfully created what others throughout the field of medicine have been calling for, and in so doing have fast-forwarded the integration of research and clinical practice for children with CP and other childhood disabilities. The future is now.”

CPRN is helping NCH to facilitate innovation in patient care. This study highlights that research can help children with CP through innovation in care.

1Lowes, L. P., Noritz, G. H., Newmeyer, A., Embi, P. J., Yin, H., Smoyer, W. E., ‘Learn from Every Patient’ Study Group (2016), ‘Learn From Every Patient’: implementation and early results of a learning health system. Developmental Medicine & Child Neurology. doi: 10.1111/dmcn.13227
2Stevenson, R. D. (2016), Integration of research and clinical practice: the future is now. Dev Med Child Neurol. doi:10.1111/dmcn.13281

CPRN Investigator Receives NIH Funding for CP Prevention Trial

Dr. Yvonne Wu, a pediatric neurologist from the University of California San Francisco, received significant funding ($2.3M over the next 12 months) from the National Institute of Neurological Disorders and Stroke (NINDS) to conduct a multi-center clinical trial to prevent cerebral palsy from a specific type of brain injury — hypoxic ischemic encephalopathy — in full term babies. While this study is not a Cerebral Palsy Research Network (CPRN) study, we are excited by the potential that the study holds to reduce the incidence of CP. Dr. Wu is the site Principal Investigator (PI) for Benioff Children’s Hospital/UCSF for CPRN. Her experience in clinical epidemiology and clinical trials makes her an invaluable asset to CPRN and we look forward to the results of this study. Her Co-PI, Dr. Sandra Juul of the University of Washington, is the lead PI on a similar trial for very low birthweight premature babies (see the PENUT trial for more information on this study).

Hypoxic-ischemic encephalopathy (HIE) refers to brain injury resulting from reduced blood and oxygen flow to a baby’s brain near the time of birth. HIE affects up to 12,000 newborns each year in the U.S. Half of affected infants have a bad outcome including death, cerebral palsy and cognitive impairment despite receiving hypothermia, the only available treatment. Cerebral palsy is the most common long term neurodevelopmental impairment in survivors of HIE. Erythropoietin (Epo) is a cytokine with remarkable neuroprotective and neuroregenerative effects demonstrated in animal models of neonatal brain injury. In a phase II trial, we randomized 50 cooled infants to receive Epo or placebo. Infants treated with hypothermia + Epo had less brain injury on MRI than infants who received hypothermia alone. Epo is commercially available, relatively inexpensive, and safe in neonates. We hypothesize that Epo given to cooled infants with moderate/severe HIE will reduce the combined outcome of death or neurodevelopmental impairment from 49 to 33%. To test this hypothesis, we will perform a randomized, double-blind, placebo-controlled trial of Epo therapy in 500 infants with HIE. We anticipate that Epo will confer improved 2-year neurodevelopmental outcome, will be safe, and will decrease brain injury severity as determined by early biomarkers. If successful, we anticipate that Epo treatment has the potential to reduce the burden of CP caused by HIE from 1368 babies to 760 babies per year in the U.S.