Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy

Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy Compared to a Matched Control Group

Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Selective dorsal rhizotomy (SDR) is a surgical method used by some hospitals to permanently reduce spasticity in order to prevent further morbidities. Better understanding of the long-term outcomes of SDR is essential for clinicians and families. The results of this study will have direct clinical impact by equipping providers with the necessary information to counsel families during medical decision making.

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy
• Intervention / Treatment: Diagnostic test: Gait and Motion Analysis; Diagnostic test: Gross Motor Function Measure (GMFM); Behavioral: Surveys

Detailed Description

Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Excessive spasticity is thought to be uncomfortable, reduce function, cause gait deviations (e.g. equinus), and contribute toward musculoskeletal deformity and an elevated energy cost while walking. As such, SDR is a surgical method used by some hospitals to permanently reduce spasticity in order to prevent the aforementioned morbidities.

Treatment philosophies differ widely in regards to how aggressively to manage spasticity. Some centers (e.g. Gillette) aggressively treat spasticity early in life through a variety of measures such as SDR, intrathecal baclofen pumps, and botulinum toxin injections. Other centers (e.g. Shriners Hospitals for Children - Salt Lake City and Spokane) offer little in the way of spasticity reduction treatments.

There are several compelling reasons to conduct the proposed research study. First, emerging evidence suggests that the elimination of spasticity during childhood via SDR does not prevent contractures and only partially explains poor gross motor function, both previously thought to be clear outcomes of the surgery. Additionally, many of the longitudinal cohort studies that examined SDR outcomes have shown many outcome measures peak 1-3 years after surgery, and then decline toward baseline (i.e. pre-SDR) levels. Lastly, the quality of the SDR outcome literature is poor. Rarely are outcomes looked at in context of a proper control group. Either a control group is absent or comprised of typically developing children. This limits our ability to understand how patients with cerebral palsy may age without undergoing an SDR.

Better understanding of the long-term outcomes of SDR is essential for clinicians and families. The surgery, in general, is costly to families (time, expense, risk, etc.) and clinicians should have every confidence in the intended outcomes for any intervention.

• Study Type: Observational
• Enrollment (Actual): 78

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Saint Paul, Minnesota, United States, 55101
      • Gillette Children's Specialty Healthcare
  • Utah
    • Salt Lake City, Utah, United States, 84103
      • Shriners Hospitals for Children - Salt Lake City
  • Washington
    • Spokane, Washington, United States, 99204
      • Shriners Hospitals for Childrens - Spokane

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years and older (Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Former/current patients at Gillette Children's Specialty Healthcare, Shriners Hospitals for Children - Salt Lake City, or Shriners Hospitals for Children - Spokane

Description

Inclusion Criteria for Controls (-SDR) and Cases (+SDR):

• Able to speak and read English
• Diagnosed with bilateral cerebral palsy (i.e. no hemiplegics)
• Minimum age of 21 years presently
• Had a baseline gait and motion analysis

Controls (-SDR):

• No SDR
• No history of intrathecal baclofen (ITB) pump implantation for > 1 year
• No ITB pump at time of long-term follow-up (explant > or = 6 months)
• No history of oral baclofen for > 1 year
• No oral baclofen use at time of long-term follow-up
• No more than 10 sessions on botulinum toxin, phenol, or alcohol injection

Cases (+SDR):

• History of SDR > 5 years ago
• Had a baseline gait analysis < or = 18 months before SDR

Exclusion Criteria:

• none

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cases (+SDR); Patients with cerebral palsy that underwent an SDR	Diagnostic test: Gait and Motion Analysis; Gait and motion analysis is comprised of 3-dimensional kinematics and kinetics, electromyography, energy expenditure, and physical exam (range of motion, strength, spasticity, etc.); Diagnostic test: Gross Motor Function Measure (GMFM); Gross Motor Function Measure (GMFM) is an assessment used to evaluate gross motor function over time in individuals with cerebral palsy. The assessment is comprised of movement activities like standing, walking, running, and jumping; Behavioral: Surveys; Six surveys are used to assess function, activity, participation, pain, quality of life, and treatment history
Controls (-SDR); Matched patients with cerebral palsy but did not undergo an SDR	Diagnostic test: Gait and Motion Analysis; Gait and motion analysis is comprised of 3-dimensional kinematics and kinetics, electromyography, energy expenditure, and physical exam (range of motion, strength, spasticity, etc.); Diagnostic test: Gross Motor Function Measure (GMFM); Gross Motor Function Measure (GMFM) is an assessment used to evaluate gross motor function over time in individuals with cerebral palsy. The assessment is comprised of movement activities like standing, walking, running, and jumping; Behavioral: Surveys; Six surveys are used to assess function, activity, participation, pain, quality of life, and treatment history

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Three-dimensional gait and motion analysis	Compare three-dimensional gait kinematics and kinetics across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Energy expenditure	Compare energy expenditure across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Spasticity	Compare spasticity, measured by Modified Ashworth Score (0 no increase in tone - 4 rigid in flexion or extension), across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Passive range of motion	Compare passive range of motion, measured by lower extremity physical exam, across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Selective motor control	Compare selective motor control (0 patterned movement - 2 complete isolated movement) across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Lower extremity strength	Compare lower extremity strength, measured by the manual muscle test, across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Gross Motor Function Measure (GMFM-66)	Assess function using portions of the GMFM-66 (0 low function - 100 high function) and compare across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Gillette Functional Assessment Questionnaire (Gillette FAQ)	Assess function and activity using the Gillette FAQ (self-reported survey, 0 low function - 10 high function) and compare across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Functional Mobility Scale (FMS)	Assess function and activity using the FMS (self-reported survey, 1 uses wheelchair - 6 independent) and compare across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Participation Enfranchisement survey	Assess participation using the Participation Enfranchisement survey (self-reported survey, true/false) and compare across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Diener Satisfaction with Life Scale	Assess satisfaction using the Diener Satisfaction with Life Scale (self-reported survey, 5 dissatisfied- 35 satisfied) and compare across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
World Health Organization (WHO) Quality of Life Scale	Assess satisfaction using the WHO Quality of Life Scale (self-reported survey, 0 low quality of life - 100 high quality of life) and compare across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)
Multiple Sclerosis Spasticity Scale (MSSS-88)	Assess pain using portions of the MSSS-88 (self-reported survey, 21 not at all bothered - 84 extremely bothered) and compare across groups	Time of long-term follow-up research visit (on average 10 years post-baseline)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in gait and motion analysis	Compare change in gait kinematics and kinetics within groups and if the change is similar between groups	Baseline (qualifying exam for cases and controls) compared to long-term follow-up research visit (on average 10 years post-baseline)
Incidence of prior surgery and anti-spastic treatments	Incidence of prior surgery and anti-spastic treatments	Time of long-term follow-up research visit (on average 10 years post-baseline)
Cost of prior surgery and anti-spastic treatments	Cost of prior surgery and anti-spastic treatments	Time of long-term follow-up research visit (on average 10 years post-baseline)

Collaborators and Investigators

• Sponsor: Gillette Children's Specialty Healthcare
• Collaborators: Shriners Hospitals for Children
• Investigators: Principal Investigator: Michael Schwartz, PhD, Gillette Children's Specialty Healthcare

Publications and helpful links

General Publications

• Munger ME, Chen BP, MacWilliams BA, McMulkin ML, Schwartz MH. Comparing the effects of two spasticity management strategies on the long-term outcomes of individuals with bilateral spastic cerebral palsy: a multicentre cohort study protocol. BMJ Open. 2019 Jun 20;9(6):e027486. doi: 10.1136/bmjopen-2018-027486.

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2018
• Primary Completion (Actual): January 31, 2020
• Study Completion (Actual): January 31, 2020

Study Registration Dates

• First Submitted: November 20, 2018
• First Submitted That Met QC Criteria: December 26, 2018
• First Posted (Actual): December 31, 2018

Study Record Updates

• Last Update Posted (Actual): October 13, 2023
• Last Update Submitted That Met QC Criteria: October 12, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Outcomes; Selective Dorsal Rhizotomy
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Damage, Chronic; Cerebral Palsy
• Other Study ID Numbers: STUDY00002353

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No