Clinical trial NCT04792606: Long-Term Outcomes of Hip Interventions for Children With Cerebral Palsy
Official title: Long-Term Outcomes of Hip Interventions for Children With Cerebral Palsy - An International Multi-Centre Prospective Comparative Cohort Study (CHOP-II)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cerebral Palsy
Keywords: non-ambulatory, non-ambulatory cerebral palsy, patient reported outcomes measures, PROMS, outcomes, CPCHILD
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
Collaborators: name: Royal Children's Hospital
name: Shriners Hospitals for Children,Canada
name: Provincial Health Services Authority
name: Aarhus University Hospital
name: Sheba Medical Center
name: Seoul National University Bundang Hospital
name: Starship Children's Hospital of New Zealand
name: Poznan University of Medical Sciences
name: Skane University Hospital
name: Karolinska University Hospital
name: Great Ormond Street Hospital for Children NHS Foundation Trust
name: The Royal London Hospital
name: Nuffield Orthopaedic Centre NHS Trust
name: Children's Hospital Medical Center, Cincinnati
name: Boston Children's Hospital
name: Columbia University
name: Shriners Hospitals for Children, Sacramento
name: Gillette Children's Specialty Healthcare
name: Alfred I. duPont Hospital for Children
name: IWK Health Centre
name: Alder Hey Children's NHS Foundation Trust
name: Children's Hospital Colorado
name: Hospital for Special Surgery, New York
name: Shriners Hospitals for Children, Honolulu
name: Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
name: Morgan Stanley Children's Hospital
name: Children's Health Queensland Hospital and Health Service
name: University of Nebraska
First posted: 2021-03-11
Enrollment: 1000
Sex: ALL
Minimum age (years): 2
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children between the ages of 2 and 19 with severe cerebral palsy (defined as a GMFCS level or IV or V) experiencing hip displacement (with radiographic evidence of a migration percentage (MP) or 30%) will be recruited to be apart of the CHOP II study.

Brief summary: Children with severe cerebral palsy (CP) are at high risk for progressive hip displacement, associated with pain and contractures interfering with many aspects of care, comfort and quality of life. These children undergo many types of interventions, the optimal timing and effectiveness of which, remain unclear. In 2014, CIHR funded the CP Hip Outcomes Project (CHOP), an international multi-centre prospective longitudinal cohort study of children with severe (non-ambulant) CP with evidence of hip displacement defined as a Reimer's migration percentage (MP) of at least 30%. The study was designed to evaluate the comparative effectiveness of different treatment strategies to prevent or relieve symptoms associated with hip instability, using the validated Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD ) questionnaire as the primary outcome measure of health-related quality of life © (HRQL) for this population. 650 patients enrolled from 28 sites in 11 countries, are actively being followed and will reach at least 2 years of follow-up at the end of 2019. This project, will study the impact of hip instability and its management in children with severe CP using the CPCHILD questionnaire that was developed specifically for this purpose. Although CHOP will define outcomes at 24 months, the outcomes are not expected to remain stable while the child is still growing. The inception cohort will need follow-up until skeletal maturity to track their long-term outcome trajectories.

Detailed description: Background: Children with severe cerebral palsy (CP) are at high risk for progressive hip displacement, associated with pain and contractures interfering with many aspects of care, comfort and quality of life. These children undergo many types of interventions, the optimal timing and effectiveness of which, remain unclear. In 2014, CIHR funded the CP Hip Outcomes Project (CHOP), an international multi-centre prospective longitudinal cohort study of children with severe (non-ambulant) CP with evidence of hip displacement defined as a Reimer's migration percentage (MP) of at least 30%. The study was designed to evaluate the comparative effectiveness of different treatment strategies to prevent or relieve symptoms associated with hip instability, using the validated Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD ) questionnaire as the primary outcome measure of health-related quality of life © (HRQL) for this population. 650 patients enrolled from 28 sites in 11 countries, are actively being followed and will reach at least 2 years of follow-up at the end of 2019. This project, unique in scale and design, will study the impact of hip instability and its management in children with severe CP using a meaningful outcome measure that was developed specifically for this purpose. Although CHOP will define outcomes at 24 months, the outcomes are not expected to remain stable while the child is still growing. The inception cohort will need follow-up until skeletal maturity to track their long-term outcome trajectories. The international network of investigators/sites and the infrastructure established for CHOP will facilitate the long-term follow-up of the participants in this study. In addition, the results of CHOP will inform the design of nested trials to evaluate the effectiveness of future interventions aimed at improving the quality of life of children with severe disabilities.

Study Aims Aim 1: Measure the post 24-month effectiveness of different treatment strategies for hip displacement in children with severe CP using the CPCHILD as the primary outcome measure.

Aim 2: Compare the rates of recurrence, relapse of symptoms, additional interventions and other longer term complications associated with each of the treatment cohorts.

Methods

Study Design: Multi-centre longitudinal cohort study of children with severe (non-ambulant) CP, ages 3 to 18 years with hip MP ≥ 30% enrolled in one of 5 cohorts:

A. "Natural" History or watchful waiting B. Serial botulinum toxin injections C. Adductor (+/- psoas) muscle releases alone D. Hip reconstructive surgery E. Salvage hip surgery Data collected include demographics, prognostic factors, including co-morbid conditions, parental reports of HRQL as measured by the CPCHILD, and radiographic measures of MP and acetabular index (AI). The primary outcome measure CPCHILD, as well as the MP \& AI will be assessed every 12 months after the 24 month follow-up since inception in CHOP. The cohort of 650 children enrolled in Groups B, C, D, \& E will be compared with each other as well as with their respective propensity matched counterparts of untreated children (Group A), using repeated measures ANOCOVA at 3yrs, and annually thereafter until at least 5 years of follow-up or skeletal maturity.

Significance: CHOP I \& II will establish the superiority of either prophylactic or reactive intervention strategies for hip displacement in severe CP and provide justification for hip surveillance.

Eligibility criteria: Inclusion Criteria:

* Children with severe, non-ambulant (GMFCS IV \& V) Cerebral Palsy
* between 2 and 19 years of age
* with radiographic evidence of hip displacement (previously untreated) as measured by Reimer's MP ≥ 30%
* and their parents or primary caregivers, defined as the individual most involved in the daily care of the child are willing to complete the CPCHILD in one of the available languages the CPCHILD is offered in

Exclusion Criteria:

* under the age of 2, or over the age of 19
* Reimer's migration percentage under 30% in both hips
* parents or primary caregivers unable to complete the CPCHILD in one of it's available languages

Primary outcomes:
measure: Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD)
timeFrame: The CPCHILD takes approximately 15 minutes to complete
Secondary outcomes:
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: The Hospital for Sick Children
name: Unni G Narayanan
role: PRINCIPAL_INVESTIGATOR
References: