Samenvatting (Frans of Engels)
Hersenverlamming is een levenslange neurologische ontwikkelingsstoornis die voortkomt uit niet-progressieve hersenbeschadiging bij foetussen of jonge kinderen. Het resulteert in bewegingsstoornissen en overmatige spiertonus, en gaat vaak gepaard met spasticiteit (spierstijfheid), wat de motoriek hindert en contracturen en misvormingen veroorzaakt. Wanneer orale medicijnen om de spieren te ontspannen (anti-spasmodica) of injecties met botulinumtoxine in de spieren de spasticiteit niet voldoende onder controle houden of ondraaglijke bijwerkingen veroorzaken, worden neurochirurgische procedures voorgesteld. Klassiek wordt bij niet-ambulante kinderen een pomp geïmplanteerd om baclofen rechtstreeks toe te dienen aan het hersenvocht rond het ruggenmerg (intrathecale baclofenpomp of ITB). Zorgvuldig geselecteerde ambulante kinderen kunnen ook worden onderworpen aan selectieve dorsale rhizotomie (SDR), die bestaat uit het selectief doorsnijden van zenuwwortels rond het onderste deel van de wervelkolom.
| Title | Intrathecal baclofen pump versus selective dorsal rhizotomy for non-ambulatory children with cerebral palsy (ITB vs. SDR) |
| Participants (P) | Children aged ≥ 4 years and < 18 years with spastic cerebral palsy (CP). Who have gross motor function classification system (GFMCS) grade 4 or 5. And whose spasticity is insufficiently controlled with conservative therapy and/or oral baclofen or who have intolerable side effects. |
| Intervention (I) | Selective Dorsal Rhizotomy (SDR) is a neurosurgical intervention which is exclusively performed in large university hospitals. In brief, a L1 laminectomy and durotomy is performed and approximately one third of each dorsal root between L2 and S1 is cut bilaterally under neurophysiological monitoring, followed by a ~1 week inpatient rehabilitation period. |
| Control (C) | Intrathecal baclofen (ITB) is a neurosurgical intervention which is typically performed in large academic centres in NL and BE. In brief, in the low lumbar area, the thecal sac is punctured and a spinal catheter is inserted under fluoroscopic guidance. Next, an abdominal wall pocket is created, and a subcutaneous catheter is tunnelled between both incisions and connected to a subcutaneous pump. Postoperative schedule is similar to SDR. |
| Outcome (O) | 1. Change in CP-CHILD caregiver version (in absolute value) during the 1st year postoperatively vs. immediately preoperatively (comparing SDR to ITB, non-inferiority design, 5/100 as clinically meaningful difference and noninferiority margin). 2. AE, SAE and SUSAR frequency, severity and causality in the first year postoperatively (comparing SDR to ITB). 3. Cost-effectiveness and cost-utility during the first postoperative year (comparing SDR to ITB). |
| Trial Design | Randomized (1:1), international, multicentric, parallel group, unblinded, pragmatic controlled trial testing non-inferiority (for the primary outcome CP-CHILD) / superiority (for all other outcomes). |
| Sample Size | 48 participants |
| Trial duration | 81 months from FPI to CSR (excluding LT-FU) and 148 months including LT-FU |
| Budget | 730.000 € |
| Sponsor | UZ Leuven |
| Chief Investigator | Prof. Dr. Philippe De Vloo |
| External partners | Annemieke Buizer, Amsterdam UMC, The Netherlands |
| Number of participating sites in Belgium | UZ Leuven, UZ Antwerpen, UZ Gent, Cliniques Universitaires Saint-Luc & UZ Brussel |
