Study Title: Enhancing Surgical Decision Making for Families of Children with Cerebral Palsy: A Practice-Based Evidence Study
Principal Investigator: Richard Stevenson, MD
Sponsoring Institution: University of Virginia
Amy Bailes, PT PhD, Co-Investigator, Cincinnati Children’s Hospital
Diane Damiano, PT PhD, Co-Principal Investigator, National Institute of Health
Paul Gross, Co-Principal Investigator, Cerebral Palsy Research Network
Susan Horn, PhD, Co-Principal Investigator, University of Utah
Unni Narayanan, MD, Co-Principal Investigator, Hospital for Sick Children
Garey Noritz, MD, Co-Investigator, Nationwide Children’s Hospital
Jerry Oakes, MD, Co-Principal Investigator, Children’s of Alabama
Michele Shusterman, Co-Principal Investigator, CP Daily Living
Background and Significance
Cerebral palsy (CP) refers to a variable group of disorders affecting movement and posture, attributable to non-progressive injuries to the developing brain. CP is the most common physical disability of childhood, impacting individuals across their lifetime. No cure is imminent. Many therapies are commonly used, but for most children, neurological and/or orthopedic surgery is a central part of the treatment plan. A recent (2013) comprehensive “systematic review of systematic reviews” of interventions for children with CP documented the state of the evidence; while data are sufficient to justify their use, the practice gap for surgical procedures is that available data are often inconclusive and trials that involve direct comparisons between invasive options are virtually non-existent. Scientific evidence is insufficient to match the best procedure with each child, leading to wide practice variation. For this project, Co-PI Gross engaged an on-line community of parents of children with CP to survey their views of surgery and surgical decision-making with 1214 respondents. Median age of children with CP was 8 years with 55% having had at least one surgical procedure. Parents overwhelmingly reported difficulty in making the decision for surgery; indications and recommendations about timing, type, and extent of surgery vary widely between physicians, with no clear guidelines in the medical literature to guide definitive course of action. Importantly, surgical treatments carry the greatest risks, elevating the burden of these decisions. Finally, there is lack of evidence that these procedures result in meaningful improvements related to the ultimate goals for their children.
Study Purpose and Aims
Purpose: to explore the effectiveness of surgical interventions that address motor disorders in CP using a comparative effectiveness paradigm to compare surgical and non-surgical alternatives, and outcome measures most relevant to the patient and family goals. The existing Cerebral Palsy Research Network or CPRN (cpresearch.net) provides the foundation for data collection in the proposed study. The CPRN was founded in early 2015 by a parent of a child with CP and co-PI, to establish a national center-based registry that would enable high quality clinical research to improve treatment. The long-term goals are to create new evidence to improve care and to utilize the CPRN to implement improved care, and to monitor outcomes through continuous quality improvement. The fundamental study question is: which surgical procedure is associated with best outcomes for each group of subjects (classified by functional severity).
- Aim 1: Compare the effectiveness of different surgical treatments with each other and non-operative treatments and natural history based on the primary outcomes (GOAL and CPCHILD) and secondary outcomes at 1 and 2 years (primary endpoints).
- Aim 2: Determine which patient characteristics are associated with best (and worst) outcomes at 1 and 2 years for each surgical procedure to better define the appropriate indications for each procedure.
- Aim 3: Explore the association between changes in ICF measures of body structure and function with changes in the child and parent-reported goal based primary outcome and other measures of activity, participation, and health related quality of life.
This study will utilize Practice Based Evidence-Comparative Effectiveness methodology (PBE-CE), developed by Susan Horn (Co-PI). Horn’s research group and Data Coordinating Center (DCC) at the University of Utah’s Health Sciences Center have partnered to provide data coordinating services for both the CPRN registry and this study. PBE methodology uses detailed descriptions of actual clinical practices or processes to examine relationships between patient characteristics (demographics, GMFCS level, type of motor disorder, co-morbid severity of illness, etc), details of interventions (surgical procedures), non-surgical interventions, and their associations with favorable and unfavorable outcomes.
The study will enroll 3,000 subjects at 18 sites across the US and Canada with the expectation of having at least 2,500 with 2 years of follow up data by the end of the study period. We anticipate that 2,000 will undergo one of three common surgical procedures that the study is evaluating (selective dorsal rhizotomy, intrathecal baclofen pump, lower extremity orthopedic surgery). We anticipate at least 200 subjects in each of three surgical groups for each of severity groups (GMFCS I – II, GMFCS III, GMFCS IV – V). All other non-surgical interventions will be recorded carefully and serve as comparators. Interventions (surgical or non-surgical) introduced will be part of routine clinical care and carefully documented. Primary outcome measures are validated, parent reported measures of function, participation, and quality of life: CPCHILD and GOAL. Secondary outcomes include: PEDI-CAT, Pediatric PROMIS, and impairment measures such as spasticity, range of motion. All children with the diagnosis of CP of either gender and any racial or ethnic groups will be included. Only children with surgery in the previous two years and underlying genetic anomalies will be excluded. We will conduct descriptive analyses, bivariate analyses using 2-sample tests, ANOVA, and correlations, propensity score, inferential association, causal inference, and various sensitivity analyses. Analyses for each aim will incorporate some or all of these approaches. The investigative team is an experienced group; nine Co-PIs and Co-Is include two parent stakeholders, two neurodevelopmental pediatricians, two physical therapists, an orthopedic surgeon, a neurosurgeon, and a biostatistician. Together with the scientific steering committee consisting of site PIs and a Patient and Stakeholder Partner committee, the team has worked collaboratively to create a research environment for success.
Cerebral Palsy (CP) is the most common physical disability of childhood. CP is a permanent disorder caused by an injury to the developing brain, which causes problems with the motor system-walking, posture, and balance. There is a variety of severity; some children walk independently, others use canes or walkers, and some use wheelchairs.
Children with CP often have tight muscles which can lead to difficulty walking, abnormalities of the joints, and chronic pain. These may interfere with a child’s ability to participate in and enjoy life. There are several operations that can be performed to improve these conditions, but the medical evidence is not clear on which is best for a particular child, and when. Parents of children with CP face difficult choices in deciding which surgery, if any, their child should have. While one family may be trying to help with pain, another may be focused on improving walking. They count on their medical team to recommend the surgery that will meet these goals. However, the medical team does not have clear evidence to address parents’ concerns and help them understand what options are best suited for their child. This creates tremendous stress for families who are eager to support their child’s development and comfort, yet fear putting their child through an operation that may not help or may even worsen their condition. This study will focus on finding the “best” surgery for children with CP, based on age, severity, and goals.
This study will compare surgeries that are common for children with CP. Common operations include surgery on the bones or joints, to the muscles directly, or to the spinal cord to relax the muscles. These surgeries are performed frequently, despite a lack of clear evidence to guide the choice of surgery.
The study team consists of doctors and therapists from 18 centers across the US and Canada, and parents from around North America dedicated to improving the care given to children with CP. We will recruit 2500 children with CP from our centers who might undergo one of the CP operations, and follow them for 2 years. We will collect information about how their parents and doctors decide whether the child should have surgery or not. If they have surgery, we will collect information about it from the surgeon. Throughout the study, we will follow each child’s medical status, physical function, and how they are participating as part of a family and community. The child’s quality of life, as reported by the family, is one of the most important outcome measures in the study. The parents on our study team have been full partners in the creation and design of the study and have played an important role in helping us understand what outcomes are most meaningful to a child and their family.
Our goal is to help doctors and parents make better decisions about which children with CP should have which operations, and at what ages different surgical procedures offer the best outcomes.
|Site Investigator||Study Site|
|Freeman Miller, MD||AI Dupont Children’s Hospital|
|Elizabeth Barkoudah, MD||Boston Children’s Hospital|
|Dennis Matthews, MD||Children’s Hospital of Colorado|
|W Jerry Oakes, MD||Children’s of Alabama|
|Amy Bailes, PT, PhD/Jilda Vargus-Adams, MD, MSc||Cincinnati Children’s Hospital|
|David P. Roye Jr., MD||Columbia|
|Michael Partington, MD||Gillette Children’s Specialty Healthcare|
|Unni G. Narayanan, MBBS, MSc, FRCS(C)||Holland Bloorview Kids Rehabilitation Hospital & The Hospital for Sick Children, University of Toronto|
|Garey Noritz, MD
Jeff Leonard, MD
|Nationwide Children’s Hospital|
|Robert Bollo, MD, MS||Primary Children’s Hospital|
|Deborah Gaebler, MD
Gadi Revivo, DO
|Rehabilitation Institute of Chicago|
|Kristie F. Bjornson, PT PhD||Seattle Children’s Hospital|
|William Oppenheim, MD||UCLA|
|Amy Viehoever, MD, PhD
Yvonne Wu, MD, MPH
|Aga Lewelt, MD, MS||University of Florida Jacksonville|
|David A. Yngve, MD||UTMB Galveston|
|Edward Hurvitz, MD||University of Michigan|
|Mark Romness, MD||UVA|
|James A. Blackman, MD, MPH||Stakeholder||Chief Medical Officer||Cerebral Palsy Foundation|
|Jon Davids, MD||Stakeholder||Orthopedic Surgeon||Shriners of Northern California|
|Lizette Dunay||Stakeholder and Parent||Executive Director||CURE CP|
|Richard Ellenson||Stakeholder and Parent||CEO||Cerebral Palsy Foundation|
|Cynthia Frisina||Stakeholder and Parent||Executive Director||Reaching for the Stars|
|Paul H. Gross||Stakeholder and Parent||Founder||Cerebral Palsy Research Network|
|Nancy Murphy, MD||Stakeholder||Director, Complex Care||Primary Childrens Hospital|
|Robert Sawin, MD||Stakeholder||Surgeon-in-chief||Seattle Childrens|
|Nancy D. Schmidt||Stakeholder||SVP||Spaulding Rehab|
|Michele Shusterman||Stakeholder and Parent||Founder||CP Daily Living|
|Lisa Thornton, MD||Stakeholder||Physiatrist||University of Chicago|
|Ann Tilton, MD||Stakeholder||Pediatric Neurologist||Children’s Hospital New Orleans Louisana|
|Marion “Jack” Walker, MD||Stakeholder||Pediatric Neurosurgeon||Retired University of Utah|
|Brad Wilson||Stakeholder||CEO||Blue Cross Blue Shield of North Carolina|