Research presents cerebral palsy research updates, funding and funding opportunities, as well as new publications and the information offered within them, to keep the cerebral palsy community informed. Learn how you can be a part of the conversations by joining MyCP, an interactive cerebral palsy community forum, today!

CPRN Leadership Team Member Receives Grant

Amy Bailes PT PhD, PCS receives funding from the American Physical Therapy Association (APTA) Academy of Pediatric Physical Therapy.

A Cerebral Palsy Research Network leader, Dr. Amy Bailes, received notification of an award for her application entitled “Toward a practice based evidence model for studying physic

Amy Bailes, PT Ph.D.

Amy Bailes, PT Ph.D.

al therapy dose in cerebral palsy” from the Academy of Pediatric Physical Therapy on November 4, 2016. The study aims to establish the reliability of capturing specific dosing information about the physical therapy (PT) session from the Electronic Medical Record (EMR). EMR based data capture by clinicians treating people with cerebral palsy is critical to the vision and success of CPRN. This study will validate whether details of the individual PT session can be quickly and accurately captured from the EMR.

The grant will fund a pilot study at Cincinnati Children’s Hospital to test the process for capturing information about the PT session from the EMR. If the study yields positive results, the PT session form will be integrated into the CPRN Registry to more completely understand the impact of PT dosing and outcomes.

Adult Patient Reported Registry Nears Completion

Clinicians and researchers of the Adult Study Group have been meeting regularly to refine questions for the CPRN Patient Reported Outcomes registry. Input from the adults with cerebral palsy on the CPRN Community Advisory Committee this summer indicated that consumers wanted more information about pain management, pain prevention, optimizing function with aging, and issues with social and emotional function.

The study group adapted a survey widely used to study pain and pain management. It is developing a survey to ask individuals about perceived changes in function with aging, how they are managing these changes, social and physical supports, community and employment activities, satisfaction with life, anxiety, and depression.

Two initial surveys are being created. The first survey will focus on pain, and will allow answers to questions about pain location, interference, and management.  It will provide useful information for clinicians and consumers.

The second survey will focus on physical, social, and emotional function. It will allow the adult study group to describe self-reported functional changes, social challenges and successes, and emotional struggles and victories.  It will provide useful information for clinicians and consumers.

The study group is planning to have the surveys piloted tested in December by the community advisors for release to the broader CP community in Q1 2017. People interested in CPRN surveys should sign up for CPRN alerts.

This is an important step to begin to address the needs of adults with cerebral palsy. Studying the adult population with CP was determined to be a critical need at the 2014 Workshop on the State of the science and treatments in CP that was held by the National Institutes of Health.

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.

CPRN and UVA Describe PCORI CP Study in Webinar

Learn about CP Intervention Study Application to PCORI

Richard Stevenson, M.D., University of Virginia

Richard Stevenson, M.D., University of Virginia

University of Virginia’s Dr. Richard Stevenson and CPRN Chairman Paul Gross hosted a webinar entitled “Comparative Effectiveness of Surgical and Non-Surgical Interventions for Ambulatory Children with Bilateral Spastic Cerebral Palsy using Patient Centered Outcomes” with CPRN Site Investigators and its recently reconstituted Patient Stakeholder Partners committee on Wednesday, August 10, 2016. We have posted the webinar of the call and the accompanying slides on CPRN’s Resource Page for public consumption. The mega application to the Patient-Centered Outcomes Research Institute (PCORI) totaled 770 pages including a 45-page research plan and a 379-page budget. The application garnered 142 letters of support and collaboration from CP focused investigators, societies and academies, patients and stakeholders. Set expectation of when you might find out about funding from PCORI.

Listen to the webinar to hear investigators Stevenson, Gross, Horn and Shusterman describe the planned study, the analysis, the use of the CPRN network and how patients and stakeholders will be involved. The webinar is 40 minutes long including Q&A.

New CPRN Cerebral Palsy Study Posted

Next week, the Cerebral Palsy Research Network (CPRN) in partnership with the University of Virginia, will resubmit its application for funding for a CP study entitled “Comparative Effectiveness of Surgical and Non-Surgical Interventions for Ambulatory Children with Bilateral Spastic Cerebral Palsy using Patient Centered Outcomes” to the Patient-Centered Outcomes Research Institute (PCORI). The study team, led by Dr. Richard Stevenson of the University of Virgina and CPRN Chairman and Founder Paul Gross, brought together founding members of CPRN (Dr. Susan Horn, Dr. Unni Narayanan, Dr. Garey Noritz and Dr. Jerry Oakes) along with Dr. Diane Damiano from the National Institutes of Health Clinical Center and patient advocate Michele Shusterman of CP Daily Living and CP NOW Foundation as investigators. This multi-center observational study will enroll 2000 patients from 23 of the CPRN Network sites to study practice variation and outcomes for lower extremity surgery including orthopedic surgeries and neurosurgery for children with bilateral spastic CP. The need for comparative effectiveness research was identified as a key outcome from the NIH Workshop entitled “State of Science and Treatment Decisions in Cerebral Palsy” in November 2014.

We are excited by the focus and scope of this revised application to PCORI. We invite you to read about all about it on our Research study page. We will post the results from this application process in early 2017 when we hear from PCORI.

CPRN Finalizes Application for Largest Cerebral Palsy Study

Principal Investigators Rich Stevenson, M.D., of the University of Virginia (UVA) and Paul Gross of the Cerebral Palsy Research Network (CPRN) and their study team finalized the details for what will be the largest cerebral palsy study ever conducted in the U.S. The study team and site investigators have been hard at work to complete this Herculean effort to apply to the Patient-Centered Outcomes Research Institute (PCORI) for a 23 center study. The Study Executive Team includes:

Name Institution Role
Diane Damiano, Ph.D. National Institute of Health Clinical Center Co-Investigator
Paul Gross Cerebral Palsy Research Network Dual Principal Investigator
Susan Horn, Ph.D. University of Utah Co-Investigator
Jacob Kean, Ph.D. University of Utah Co-Investigator
Unni Narayanan, M.D., M.P.H. SickKids/Holland Bloorview Co-Investigator
Garey Noritz, M.D. Nationwide Children’s Hospital Co-Investigator
Jerry Oakes, M.D. Children’s of Alabama Co-Investigator
Michele Shusterman CP Daily Living/CP NOW Foundation Co-Investigator
Richard Stevenson, M.D. University of Virginia Dual Principal Investigator

The primary goal is to determine the effectiveness of surgical interventions for ambulatory children with CP to improve gait function as measured by child and parent reported outcomes that align with family goals when compared to standard non-surgical interventions. Our secondary goal is to change practice by identifying patient and surgical details that will help clinicians and parents choose the right treatment for the right child at the right time.
The Specific Aims are to:

  1. Compare effectiveness of orthopedic surgery, selective dorsal rhizotomy (SDR), neurotoxin injection, and physical therapy (PT) alone in improving outcomes in patients ages 3-12 with bilateral spastic cerebral palsy;
  2. Describe and compare adverse events associated with each treatment option;
  3. Explore intervention characteristics associated with positive or negative outcomes controlling for patient factors.

The study executive team appreciates the efforts of the site investigators and their grants administration teams and the patient and stakeholder partners. In addition, special recognition goes to the UVA grants administration team of Kerry Day, Michelle Haynes and Angela Rogers and University of Utah biostatistician Tom Greene for making this application possible. We look forward to a positive affirmation of our work from PCORI in early 2017!

Cerebral Palsy Nonprofits and Academies Support CPRN PCORI Application

As the Cerebral Palsy Research Network (CPRN) and Richard Stevenson, MD, of the University of Virginia finalize their application to the Patient-Centered Outcomes Research Institute for a large scale cerebral palsy trial, we have received tremendous support for the study from both the cerebral palsy nonprofit organizations and the provider membership organizations. The membership organizations represent a significant part of the “delivery system” for care and have agreed to help disseminate the findings from the proposed study with their membership.

Similarly, the nonprofit organizations provide access to a very broad array of patients, parents and caregivers for children with cerebral palsy that might benefit from the study findings. The study is directly aimed at providing more definitive answers to patients, parents and providers as to what treatments work best for which patients.

Many of the societies and academies whose members treat children with CP have agreed to disseminate our results including:

The nonprofit organizations that have agreed to help disseminate our results include:

These stakeholders are joined by a number of provider and payer stakeholders that are part of the “delivery system, ” including perspectives from members of the delivery system and administration help assure that the study and its results are adopted. The delivery system stakeholders include:

  • Jon Davids, M.D., Orthopedic
  • Surgeon, Shriners Hospital of Sacramento
  • Vedant Kulkarni, M.D., Orthopedic Surgeon, Shriners Hospital of Sacramento
  • Nancy Murphy, M.D., Medical Director, Comprehensive Care, Primary Children’s Hospital
  • Robert Sawin, M.D., Chief of Surgery, Seattle Children’s Hospital
  • Lisa Thorton, M.D., Physical Medicine and Rehabilitation, University of Chicago
  • Ann Tilton, M.D., Pediatric Neurologist, Children’s Hospital, New Orleans
  • Marion “Jack” Walker, M.D., Neurosurgeon, Primary Children’s Hospital
  • Brad Wilson, C.E.O., Blue Cross Blue Shield of North Carolina

We greatly appreciate the level of support we have received from the leadership of the various provider societies, the nonprofit organizations and the stakeholders in supporting this study endeavor.

Site Survey Speaks Volumes About CPRN Study Capacity

As the Cerebral Palsy Research Network (CPRN) prepares its application for the Patient-Centered Outcomes Research Institute (PCORI), we sought very detailed information from our partner Site Principal Investigators (Site PIs) about the children they see and treat in their hospitals. The proposed PCORI application is a study of lower extremity interventions for children with bilateral spasticity (spastic diplegia). We asked each site to review their volume of patients that fit our core “eligibility” criteria for the study — ages two to 12 years and Gross Motor Functional Classification Scale I-III. We asked how many children did they see last year with ambulatory CP that met those criteria. Subsequently, we asked how many of those children received:

  1. gait related neurosurgery surgery specifically selective dorsal rhizotomy (SDR);
  2. gait related orthopedic surgery;
  3. how many had medical treatments such as botulinum toxin (botox) or phenol injections;
  4. and how many only had physical therapy over that 12-month period.

We were gathering these data for planning the study but the results are fascinating unto themselves. Below are a few highlights from the 23 centers participating in this study application. Each of these data points are only for the cohort of patients that are between the ages of two and 12 years and have bilateral spasticity seen in the last year:

  • more than 4,200 of these children were seen in clinic;
  • there were 714 (16.7%) orthopedic surgeries performed to treat them;
  • another 157 (3.7%) had an SDR;
  • approximately 1,500 (35%) were treated with botox or phenol injections;
  • the largest treatment group was physical therapy only with more than 1,800 (43%).

While these numbers are interesting in the aggregate, the practice variation underlying them is quite significant and will be a key part of this study along with the associated patient outcomes. We appreciate the significant work that our study partners have done to contribute to this survey. At the outset of this process, the survey was quite daunting to the Site PIs. CPRN leadership team member, Dr. Ed Hurvitz quipped “I am pleased to tell you that I am currently involved in a major registry project called CPRN.  Once that is moving along and rolling, I will be able to pull out all of the data on the sheet without much effort!  Currently, it is hard to imagine where I get all of those numbers.” This statement speaks to the power of the cerebral palsy registry that CPRN is developing.

CPRN Invites Eight New Sites to Partner in PCORI Application

Twenty Three Sites Invited To Participate in Groundbreaking Study

The Cerebral Palsy Research Network (CPRN) has invited eight new study sites to partner with 15 of the existing CPRN network sites for the “Comparative Effectiveness of Surgical and Non-Surgical Interventions for Ambulatory Children with Bilateral Spastic (Diplegic) Cerebral Palsy using Patient Centered Outcomes” study application. The partner sites bring additional patient volume and diversity of region, practice and population to the research. CPRN welcomes the participation of the following hospitals and investigators for the Patient-Centered Outcomes Research Institute (PCORI) application:

Texas Children’s HospitalAloysia Schwabe, MDHouston, TX

Site Principal Investigator Location
Columbia/Weinberg Cerebral Palsy Center David Roye, MD New York, NY
Johns Hopkins Medical Institutes Ranjit Varghese, MD Baltimore, MD
Phoenix Children’s Hospital Taryn Bragg, MD
Michael Kruer, MD
Phoenix, AZ
Rehabilitation Institute of Chicago Gadi Revivo, DO Chicago, IL
St. Louis Children’s Hospital Toni Pearson, MD St. Louis, MO
University of Texas Health Center-Houston Manish Shah, MD Houston, TX
University of Texas Medical Branch-Galveston David Yngve, MD Galveston, TX

CPRN plans to enroll 1,500 patients in the study over a two-year enrollment period across the sites. If funded, it will be the first ever of this scale that will analyze the the most common surgical interventions for children with ambulatory CP. Using patient-centered outcomes, the study aims to yield new data to help guide both practitioners and parents when they must make critical surgical and medical decisions for children with bilateral CP. Below are all of the existing CPRN sites and principal investigators that are participating in this study application:

Site Principal Investigator Location
A.I. du Pont Children’s Hospital Freeman Miller, MD Newark, DE
Boston Children’s Hospital Benjamin Shore, MD, MPH Boston, MA
Children’s Hospital of Colorado Dennis Matthews, MD Denver, CO
Children’s Hospital of Alabama Brandon Roque, MD Birmingham, AL
Cincinnati Children’s Hospital Amy Bailes, PhD
Jilda Vargus-Adams, MD, MPH
Cincinnati, OH
Gillette Children’s Specialty Healthcare Michael Partington, MD Minneapolis , MN
Nationwide Children’s Hospital Jeffrey Leonard, MD
Garey Noritz, MD
Columbus, OH
Primary Children’s Hospital/University of Utah1 Robert Bollo, MD
Jacob Kean, PhD
Susan Horn, PhD
Salt Lake City, UT
Rady Children’s Hospital Pritha Dalal, MD San Diego, CA
Seattle Children’s Hospital Kristie Bjornson, PhD Seattle, WA
SickKids/Holland Bloorview Unni Narayanan, MD, MPH Toronto, ON Canada
Texas Scottish Rite Hospital Mauricio Delgado, MD Dallas, TX
University of Florida, Jacksonville Aga Lewelt, MD Jacksonville, FL
University of Michigan Ed Hurvitz, MD Ann Arbor, MI
University of Virginia2 Rich Stevenson, MD Charlottesville, VA

1Data Coordinating Center
2Clinical Coordinating Center

The application is due to PCORI on August 9, 2016 and CPRN will be informed of the funding decision in early 2017. The study will hopefully begin to enroll patients in October, 2017.