Prevention of renal and bladder damage in children with spina bifida by means of early injections with Botulinum-
Toxin-A (Botox): a pilot study.

Background of the study:

Children with severe spina bifida almost always have serious bladder function problems, possibly resulting in renal damage. Taking into account (recent) discovered properties of botulinum toxin A it can be assumed that early treatment
with Botox in the bladder of children with spina bifida may have a protective effect on bladder function. This can result in
less operations and less incontinence later on in life, which undoubtedly will increase the quality of life of these children.



Also (further) deterioration of the renal function can be delayed.



Objective of the study:

- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder
and renal function?

- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an operation
(ileocystoplastic with appendicovesicostoma)?



Study design:

Open, controlled, prospective pilot study.



Study population:

All newborns with MMC can participate in the study. There will be made no distinction in gender and/or ethnical background.



Nature and extent of the burden and risks associated with participation, benefit and group relatedness:

With dosages of <10 U/kg hardly any side effects are described in literature. A relative frequent side effect in “non neurogenic” adults is urinary retention. In case of urinary retention the patient should be catheterized temporarily (approximately 4 weeks) to fully empty the bladder. Patients in this study however are already catheterized as of birth, so a temporarily retention is not relevant. Another known side effect is a urinary tract infection shortly after the injection. This side effect however is rather a result of the necessary cystoscopy than of the Botox itself. Besides this, the risk of a urinary tract infection can be strongly reduced by applying antibiotics peri-operative. In current literature no prove can be found that repetitive narocsis has a negative influence on the development of children. Based on the hypothesis serious functional restrictions can be prevented. This probably weigh up to the currently unknown effects of anesthetics in children.

Vroegtijdige Botox-injecties in de blaas bij MMC patiënten kunnen verdere achteruitgang van de blaas- en nierfunctie voorkomen.

Primary outcome: after 5 years of follow-up



- Clinical parameters: continuous

- Urodynamic parameters: at visits with urodynamic testing (age 2, 8,
17, 26, 35, 44, 53, and 62 months)

- Number and nature of operative interventions to the urinary tract: continuous.

Botulinum toxin A will be injected in the bladder at the age of approximately 5 months during a short-term narcosis (10 minutes). These injections will be repeated every 9 months during the study period.