Long-Term Adherence to Onabotulinum Toxin-A Intradetrusor Injections for Neurogenic Dysfunction in Children-A Retrospective Single-Center Evaluation

• Published in: Toxins Vol. 16; no. 7; p. 303
• Main Authors: Pellegrino, Chiara, Forlini, Valentina, Capitanucci, Maria Luisa, Della Bella, Gessica, Mosiello, Giovanni
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 01.07.2024
• Subjects: Adolescent; Adults; Anesthesia; Anticholinergics; Bladder; botulinum toxin-A; Botulinum Toxins, Type A - administration & dosage; Botulinum Toxins, Type A - therapeutic use; Boxes; Child; Child, Preschool; Children; Compliance; Drugs; Effectiveness; Female; Health aspects; Humans; Kidney diseases; Male; Medication Adherence; neurogenic bladder; neurogenic lower urinary tract dysfunction; Neuromuscular Agents - administration & dosage; Neuromuscular Agents - therapeutic use; neurotoxin; Patients; pediatric; pediatric urology; Pediatrics; Retrospective Studies; Surgery; Therapy; Toxins; Urinary Bladder, Neurogenic - drug therapy; Urogenital system; neurogenic bladder; pediatric urology; neurogenic lower urinary tract dysfunction; spina bifida; neurotoxin; botulinum toxin-A; neurogenic detrusor overactivity; pediatric
• ISSN: 2072-6651; 2072-6651
• DOI: 10.3390/toxins16070303

Onabotulinum Toxin-A (BTX-A) is a second-line treatment for neurogenic bladder (NB). It requires repeated injections over time, which is a possible limit for long-term adherence, especially in children, as general anesthesia is required. Almost 50% of adults discontinue therapy; few data on pediatric patients are present. The aim of this study is to share our long-term experience of BTX-A adherence in children. This study is a retrospective review of 230 refractory NB patients treated with BTX-A. The inclusion criteria were ≥3 treatments and the first injection performed ≥10 years before the study endpoint. Fifty-four patients were included. Mean follow-up was 10.2 years; mean treatment number was 6.4 for each patient. During follow-up, 7% did not need BTX-A anymore; 76% discontinued therapy, with a prevalence of acquired NB (64% acquired vs. 34% congenital; = 0.03); sex-based and urodynamic findings did not influence the discontinuation rate ( = 0.6, = 0.2, respectively). Considering those who withdrew from the therapy, 43% were lost to follow-up/died after a mean of 7.5 years (although 33% still experienced clinical efficacy); 33% changed therapy after a mean of 5.8 years (with reduced efficacy in 22%, persistent efficacy in 11%). BTX-A is a safe and effective therapy for pediatric patients. The treatment abandonment rate is higher for children than for adults; no specific reasons were highlighted. It is necessary to evaluate any age-specific factors to explain these data.