Clinical trial NCT03789786: Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy
Official title: Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy Compared to a Matched Control Group
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cerebral Palsy
Keywords: Selective Dorsal Rhizotomy
Outcomes
Lead sponsor: Gillette Children's Specialty Healthcare
Sponsor class: OTHER
Collaborators: name: Shriners Hospitals for Children
First posted: 2018-12-31
Enrollment: 78
Sex: ALL
Minimum age (years): 21
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: OTHER
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

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Three-dimensional gait and motion analysis
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Energy expenditure
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Spasticity
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Passive range of motion
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Selective motor control
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Lower extremity strength
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Gross Motor Function Measure (GMFM-66)
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Gillette Functional Assessment Questionnaire (Gillette FAQ)
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Functional Mobility Scale (FMS)
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Participation Enfranchisement survey
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Diener Satisfaction with Life Scale
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: World Health Organization (WHO) Quality of Life Scale
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Multiple Sclerosis Spasticity Scale (MSSS-88)
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Secondary outcomes:
measure: Change in gait and motion analysis
timeFrame: Baseline (qualifying exam for cases and controls) compared to long-term follow-up research visit (on average 10 years post-baseline)
measure: Incidence of prior surgery and anti-spastic treatments
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
measure: Cost of prior surgery and anti-spastic treatments
timeFrame: Time of long-term follow-up research visit (on average 10 years post-baseline)
Other outcomes:
Locations:
city: Saint Paul
country: United States
facility: Gillette Children's Specialty Healthcare
state: Minnesota
city: Salt Lake City
country: United States
facility: Shriners Hospitals for Children - Salt Lake City
state: Utah
city: Spokane
country: United States
facility: Shriners Hospitals for Childrens - Spokane
state: Washington
Study officials:
affiliation: Gillette Children's Specialty Healthcare
name: Michael Schwartz, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.