Cerebral palsy (CP) is a lifelong neurodevelopmental condition arising from non-progressive disorders occurring in the foetal or infant brain. It results in movement disorders and excessive muscle tone, and often presents with spasticity (muscle stiffness), which impairs motor function and causes contractures and deformities. When oral medications to relax the muscles (antispasmodics) or botulinum toxin muscle injections do not adequately control spasticity or cause intolerable side effects, neurosurgical procedures are proposed. Classically, in non-ambulatory children, a pump will be implanted to deliver baclofen directly to the cerebrospinal fluid surrounding the spinal cord (intrathecal baclofen pump or ITB). Carefully selected ambulatory children can also be subjected to selective dorsal rhizotomies (SDR), which consists in selectively cutting nerve rootlets around the lower part of the spine.
Although ITB is an intensive chronic therapy requiring pump refills, dose adjustments, replacements and revisions, it is reversible and considered less invasive than SDR, which is not reversible or adjustable.
Nonetheless, both internationally and in the Netherlands and Belgium, some clinical groups have recently started to use SDR in non-ambulatory spastic CP patients as well, first as an alternative in case of repeated ITB complications, but also as a primary treatment.
This study aims to assess whether SDR improves patients at least as much as ITB, while having fewer side effects and offering better value for money.
| 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 |
