title: Autologous cell seeded biodegradable scaffold for augmentation cystoplasty: phase II study in children and adolescents with spina bifida.
abstract: OBJECTIVE
  Augmentation cystoplasty using gastrointestinal segments in children/adolescents with medically refractory neurogenic bladder is associated with significant complications. We evaluated an autologous cell seeded biodegradable scaffold (Tengion®) for bladder augmentation as an alternative to traditional enterocystoplasty in this population.
  
  
  METHODS
  A phase II prospective study was performed in children with neurogenic bladder due to spina bifida requiring enterocystoplasty for detrusor pressure 40 cm H2O or greater despite maximum antimuscarinic medication. Following open bladder biopsy, urothelial and smooth muscle cells were grown ex vivo and seeded onto a biodegradable scaffold to form a regenerative augment as the foundation for bladder tissue regeneration. Bladder neck sling was the only concomitant surgical procedure permitted. Bladders were cycled postoperatively to promote regeneration. Primary and secondary outcomes at 12 months included change in bladder compliance, bladder capacity and safety. Long-term assessment was done with similar outcomes at 36 months.
  
  
  RESULTS
  Compliance improved in 4 patients at 12 months and in 5 patients at 36 months, although the difference was not clinically or statistically significant. There was no clinical or statistical improvement in bladder capacity at 12 or 36 months in any patient. Adverse events occurred in all patients, and most were easily treated. Two patients had low cell growth following bladder biopsy, of whom 1 withdrew from the study and 1 underwent a second biopsy. Serious adverse events of bowel obstruction and/or bladder rupture occurred in 4 patients.
  
  
  CONCLUSIONS
  Our autologous cell seeded biodegradable scaffold did not improve bladder compliance or capacity, and our serious adverse events surpassed an acceptable safety standard.
authors.affiliation: University of Alabama at Birmingham, Birmingham, Alabama. Electronic address: dbjoseph@uab.edu.
authors.forename: David B
authors.initials: DB
authors.lastname: Joseph
authors.affiliation: Boston Children's Hospital, Boston, Massachusetts.
authors.forename: Joseph G
authors.initials: JG
authors.lastname: Borer
authors.affiliation: Children's Hospital Los Angeles, Los Angeles, California.
authors.forename: Roger E
authors.initials: RE
authors.lastname: De Filippo
authors.affiliation: Wake Forest Baptist Medical Center, Winston-Salem, North Carolina.
authors.forename: Steve J
authors.initials: SJ
authors.lastname: Hodges
authors.affiliation: University of California, Irvine, Irvine, California.
authors.forename: Gordon A
authors.initials: GA
authors.lastname: McLorie
mesh_terms: D020341:Absorbable Implants; D000293:Adolescent; D000714:Anastomosis, Surgical; D017690:Cell Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008297:Male; D009130:Muscle, Smooth / Q000166:cytology; D011446:Prospective Studies; D016135:Spinal Dysraphism / Q000150:complications; D054457:Tissue Scaffolds; D014182:Transplantation, Autologous; D001743:Urinary Bladder / Q000601:surgery*; D001750:Urinary Bladder, Neurogenic / Q000209:etiology / Q000601:surgery*; D013520:Urologic Surgical Procedures / Q000379:methods; D019459:Urothelium / Q000166:cytology
keywords: autologous; neurogenic; regenerative medicine; spina bifida cystica; transplantation; urinary bladder; urologic surgical procedures