Predictive Factors for Treatment Success of Intrathecal Baclofen Therapy in Children With Cerebral Palsy - The Netherlands CP Register as a Unique Model for Practice Based, Long Term Data From Children With Cerebral Palsy.
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 75
- 主要终点
- Change in Goal Attainment Scaling (GAS)
研究概览
简要总结
For children with severe cerebral palsy (CP) in whom other options are insufficient, intrathecal baclofen (ITB) treatment could be an option. Literature provides some evidence for the effect of ITB, however we notice that not all children benefit equally. Furthermore long term effects of ITB in a prospective cohort of individuals with CP have not yet been described in literature. To improve selection for ITB physicians need to know which factors influence outcome on the long term. In order to accomplish these goals prospectively, real life data over a long period of time, and from a large cohort are needed.
The investigators will therefore conduct a prospective observational cohort study using the Netherlands CP register to collect data over a period of 4,5 years. The Netherlands CP register is an innovative register for data collection which was developed between 2016 and 2020 in a unique collaboration between children with CP, their parents, researchers and health care professionals.
All children and adolescents with CP, who start ITB treatment in the Netherlands under the age of 16, diagnosed with spastic or dyskinetic CP, are eligible for inclusion in the study.
The primary outcome measure is goal attainment scaling (GAS) in which achievement of individual goals can be quantified. Factors that potentially influence outcome including spasticity, dystonia, scoliosis and complications will be secondary outcome measures.
Measurements will be done before initiation of ITB, after 3 months, after 1 year and after 2 years.
Statistical analysis will include effect size Cohen's d for all outcome measures. To assess which factors are possibly associated with GAS T-score outcome at follow-up multiple regression analysis will be conducted.
详细描述
Background/rational:
Cerebral palsy (CP) is the most common cause of childhood disability with a prevalence of 2.11 per 1000 live births in high income countries. CP enfolds a group of developmental disorders of movement and posture, attributed to non-progressive disturbances which occurred in the developing foetal or infant brain.
An advanced treatment option for individuals with severe CP is Intrathecal Baclofen (ITB) treatment. This treatment can be considered when other options, such as oral medication, are insufficient or cause side effects. Previous studies have shown mostly low level evidence for the effect of ITB on spasticity in children with spastic CP. For children with dyskinetic CP, The Amsterdam UMC and Maastricht UMC previously conducted a randomized placebo-controlled trial, which provides evidence for the effect of ITB on attaining personal treatment goals after three months of ITB treatment. It was furthermore found that after one year of ITB treatment, personal treatment goals were at least partially achieved in 97% of children and adolescents.
Not all children show similar response to ITB: some benefit more than others in attaining treatment goals. The investigators previous research on the effect of ITB in children with dyskinetic CP, found that a higher level of dystonia prior to ITB and reduction of pain during ITB were associated, for a small part, with attainment of treatment goals. Sample size was not sufficient to look at specific factors associated with specific types of treatment goals. For example, goals on the level of personal care could be influenced by other factors than goals related to sitting or transfers.
Long-term effects of ITB in a prospective cohort of individuals with spastic and dyskinetic CP have not yet been described in literature. Furthermore, knowing which factors influence outcome on the long term, preferably related to specific types of treatment goals, can be used to inform and select children and adolescents with CP better before initiating ITB. In order to accomplish these goals prospectively, real life data over a long period of time, and from a large cohort are needed.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All children and adolescents diagnosed with CP,
- •who start ITB treatment in the Netherlands under the age of 16,
排除标准
- •no consent is given by the patient and/or caregiver for participation
结局指标
主要结局
Change in Goal Attainment Scaling (GAS)
时间窗: 3 months, 1 year, 2 years
Attainment of personal treatment goals are scored on a standardised scale where -2 is the baseline score and 0 is the goal. Scores can be given for deterioration (-3), improvement but not attainment (-1) and more (+1) or much more (+2) than goal achieved). change in these outcomes between the different time points is assessed.
次要结局
- range of motion(baseline, 3 months, 1 year, 2 years)
- Scoliosis(baseline, 3 months, 1 year, 2 years)
- Pediatric Evaluation of Disability Inventory (PEDI)(baseline, 3 months, 1 year, 2 years)
- Complications(3 months, 1 year, 2 years)
- spasticity(baseline, 3 months, 1 year, 2 years)
- Functional strength testing(baseline, 3 months, 1 year, 2 years)
- Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)(baseline, 3 months, 1 year, 2 years)
- Pain severity on Visual Analogue Scale (pain VAS)(baseline, 3 months, 1 year, 2 years)
- ITB pump details(3 months)
- Catheter tip heigth(3 months, 1 year, 2 years)
- Selective motor control testing(baseline, 3 months, 1 year, 2 years)
- Barry-Albright-Dystonia Scale (BADS)(baseline, 3 months, 1 year, 2 years)
- walking pattern/gait analysis(baseline, 3 months, 1 year, 2 years)
- Catheter type(3 months)
研究者
Laura Bonouvrié
MD PhD
Amsterdam UMC, location VUmc