Long-term safety and tolerability of repeated treatments with onabotulinumtoxinA in children with neurogenic detrusor overactivity
Isreal Franco, MD1, Eric Dobremez, MD, PhD2, Piet B. Hoebeke, MD3, Wilson Titanji, PhD4, Irina Yushmanova, MD4, Brenda Jenkins, BS4, Paul F. Austin, MD5.
1Yale New Haven Children's Hospital, New Haven, CT, USA, 2H˘pital Pellegrin Enfants, Bordeaux, France, 3Ghent University Hospital, Ghent, Belgium, 4Allergan (an AbbVie company), Irvine, CA, USA, 5Texas Children's Hospital, Houston, TX, USA.
BACKGROUND: OnabotulinumtoxinA is approved for the treatment of neurogenic detrusor overactivity (NDO) in adults inadequately managed with anticholinergics and was shown to be effective and well tolerated in children in a randomized double-blind study (Clinicaltrials.gov, NCT01852045). Given the paucity of long-term data in children we report on the continued safety in these patients after repeated onabotulinumtoxinA treatments.
METHODS: This was a multicenter, double-blind, long-term, repeat-treatment extension study (Clinicaltrials.gov, NCT01852058) in patients who entered from the preceding study where they received one onabotulinumtoxinA treatment. Data were integrated across both studies. All patients (5-17 years) were on clean intermittent catheterization and could receive dose escalations based on their response to the preceding treatment (50U, 100U, or 200U onabotulinumtoxinA [not to exceed 6U/kg] studied).
RESULTS: Among patients enrolled, 95, 90, 55, and 11 patients received at least 1, 2, 3, or 4 treatments with study medication, respectively. The safety profile was similar across doses and consistent after repeat treatments (Table). Most commonly reported treatment emergent adverse events (TEAEs) within the first 12 weeks were urinary tract infection (UTI) and bacteriuria. There were no cases of autonomic dysreflexia, neutralizing antibodies, and no TEAEs related to distant spread of toxin. Two cases of pyelonephritis (100U; cycle 2) occurred ≥ 231 days after treatment and were not considered treatment related. Six cases of nonrelated hydronephrosis occurred between 85 to 309 days after injection. No meaningful changes in renal function (based on estimated glomerular filtration rate) were noted in any patient.
CONCLUSIONS: OnabotulinumtoxinA continued to be well tolerated in up to four repeated treatments in pediatric patients with NDO with similar safety profiles across dose groups. TEAEs were primarily urological with no new safety concerns.
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