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!

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.

CPRN Investigators Invited to Apply for PCORI Funding

The CPRN investigators have been invited to submit a full Patient-Centered Outcomes Research Institute (PCORI) application for the Large Pragmatic Studies to Evaluate Patient-Centered Outcomes – Spring 2016 Cycle which closes on August 9, 2016. Richard Stevenson, M.D., of the University of Virginia, is the lead Principle Investigator in the study entitled “Comparative Effectiveness of Surgical and Non-Surgical Interventions for Ambulatory Children with Bilateral Spastic (Diplegic) Cerebral Palsy using Patient-Centered Outcomes.” The study funds requested are in excess of $7 million. This five-year study, if funded, will fill critical information gaps in the treatment of children with CP for clinicians and patient families. Children between the ages of 2 and 12 with ambulatory CP that are considered candidates for surgical interventions will be enrolled at the 18 sites within the network. Site selection will be completed before the end of June 2016. The study is expected to enroll 1,500 patients in the first two years of accrual.

This study is a revision of a study that was submitted last year. The PCORI review committee encouraged the investigators to narrow the scope of the study regarding inclusion criteria to limit the heterogeneity of the population being study and increase feasibility of enrolling the target number of patients. We are pleased to have the opportunity to submit this revised study and are optimistic about it being funded.

CPRN Registry Protocol Submitted at Nationwide Children’s Hospital

The CPRN Registry Protocol, a formal description of the plans, purpose and data to be collected, safety and monitoring for the CPRN Registry research project, was submitted to the Nationwide Children’s Hospital (NCH) Institutional Review Board (IRB) today. The IRB’s role is to assure the protection of human subjects in research such that it is in accordance with all federal, institutional, and ethical guidelines. The submission of this protocol is a significant milestone in the progress of CPRN to launch a national cerebral palsy registry. It is significant for numerous reasons:

The IRB protocol approval is a required step to begin the collection of patient data for research;

    • Nationwide Children’s Hospital, as part of PEDSnet, enables us to take advantage of the Master Reciprocal IRB Reliance Agreement among PEDSnet institutions of which several are also part of CPRN including AI duPont Hospital, Boston Children’s Hospital, Children’s Hospital of Colorado, Cincinnati Children’s Hospital and Seattle Children’s Hospital;
    • Sites that are not part of PEDSnet can do a project specific reliance agreement;
    • Once the IRB protocol is approved, all other CPRN sites will be able to submit the CP registry protocol to their IRBs;
    • When CPRN seeks to expand its registry, other sites will be able to quickly gain IRB approval through the reliance mechanism.

The CPRN Registry IRB Protocol was submitted at Nationwide Children’s Hospital by Dr. Garey Noritz who is the Director of the Cerebral Palsy Program at NCH and one of the leaders of CPRN. The IRB Protocol can be found on the CPRN Resources page. CPRN appreciates the efforts of Dr. Noritz and Nationwide Children’s Hospital in launching the CPRN Registry.

CPRN at NIH Workshop on Cerebral Palsy

Leaders, investigators, and advisors from the Cerebral Palsy Research Network (CPRN) are attending the National Institutes of Health (NIH) Workshop on Basic and Translational Research for CP this week on March 24, 2016. CPRN founder Paul Gross, a former National Institute of Neurological Disorders and Stroke (NINDS) Advisory Council member, helped organize the agenda for the meeting. Gross, along with numerous advocates in the CP community, have encouraged NINDS leadership to create a strategic plan for preventing, treating and curing cerebral palsy. The first step toward a plan began with the NIH Workshop on the State-of-Science and Treatment in Cerebral Palsy in November 2014. Of five key objectives identified by participants in that meeting, more focus on basic and translational research was an important area for NINDS to consider and this week’s meeting agenda will focus on basic and translational research goals.

The agenda and format will allow the attendees – clinicians, researchers and patient advocates — to discuss the latest scientific advances in promising areas with the potential to alter the course of CP including neuroprotection, neuroplasticity, imaging, stem cells and animal models. Several CPRN investigators and contributors including Dr. Yvonne Wu from UCSF and Dr. Shenandoah “Dody” Robinson from Boston Children’s are speakers in the workshop. The synthesis of this meeting should help NIH further hone a strategic plan for cerebral palsy. The meeting is being streamed live by NIH and can be viewed here.

CPRN views participation in this type of national government planning meeting as critical to its mission of improving outcomes for people with cerebral palsy. Many findings will eventually need a clinical trials infrastructure to bring the benefits to patients. The CPRN Registry will help inform researchers of the most important areas to focus on and determine how quickly a study could be conducted across our network. It will also enable following long term outcomes for people that receive a range of treatments.

CPRN Logo with a dark and light green ribbon next to the words Cerebral Palsy Research Network.

CPRN Confers with PCORI staff about application planning

Members of the CPRN leadership team joined Dr. Rich Stevenson, Principal Investigator for the Enhancing Surgical Decision Making study, in a teleconference with two program directors at Patient Centered Research Outcomes Institute (PCORI) to discuss how to improve the opportunity for funding. CPRN had submitted an application in July, 2015 and learned on January 29, 2016 that the application was not funded. While the application scored well, PCORI only funded 5 of 34 applications. We met with PCORI staff to better understand the feedback from reviewers and determine our path forward. The dialogue was very productive and the staff was encouraging about the importance of surgical decision making in cerebral palsy.

The next PCORI funding cycle that will open for this study requires a letter of intent to be submitted in May, 2016. The study team plans to revise the study incorporating the feedback from PCORI staff and reviewers and re-submitting. We will also consider submitting a variant of the study to NIH. We remain committed to the importance of studying surgical interventions for cerebral palsy and confident that there will be a public funder, either PCORI or NIH, for the study.

PCORI Cerebral Palsy Study Decision Imminent

The Patient Centered Outcomes Research Institute (PCORI) makes its funding decisions at public meetings of its board of governors. The next PCORI board meeting is scheduled for January 26, 2016 and will include a formal decision on the CPRN/University of Virginia application entitled Enhancing Surgical Decision Making for Families of Children with Cerebral Palsy: A Practice-Based Evidence Study. The CPRN leadership team members, all of whom are co-principal investigators (PIs) for this study, are eager to hear the results of this PCORI application process. Study funding will greatly accelerate the work of CPRN by providing additional support to bring the leadership team, site PIs and the study patient stakeholder partners together as soon as April, 2016 to kick off the cerebral palsy study.

In the interim, CPRN will announce charter members later this month. Charter members will play a key role in finalizing the infrastructure and governance for the CPRN registry and network operations. The PCORI study and future CPRN studies will build on the registry and network infrastructure to accelerate study planning and core data capture for research.