Stem cells as a therapeutic modality in pediatric malformations

The aim of this study was to explore stem cell use in congenital anomalies.

During July 2005 through July 2006, autologous stem cells were used in 29 patients: 12 with liver cirrhosis and 17 with meningomyelocele. Stem cells were injected into the hepatic artery and the portal vein or into the hepatobiliary radicals for liver cirrhosis, or into the spinal cord and caudal space for meningomyelocele. Preoperative status served as the control condition.

The ages of patients with liver cirrhosis ranged between 1.5 and 9 months (mean, 4.12 months). The etiology was extra hepatic biliary atresia (EHBA) versus neonatal cholestasis and choledochal cyst in 8; 2 and 2 patients, respectively. Five patients died due to ongoing cirrhosis. Follow-up at 3 to 12 months (N = 7) showed absence of cholangitis (4/7), yellow stools (5/7), decreased liver firmness (3/7), improved liver function (6/7), and better appetite (6/7). Hepatobiliary scan was excretory in 6 of 7 with improved uptake in 4 of 7. Histopathology demonstrated comparative improvement in fibrosis among 3 patients. Meningomyelocele patients were between 0 and 1 month, 1-5 months, and 1-4 years in 5, 8, and 2 cases respectively. Five had a history of rupture. Three had undergone meningocele repair in the past with neurological deficits. Redo surgery for a tethered cord was performed in 1 patient. Follow-up at 3 to 11 months in 14 cases showed improved power in 7 with dramatic recovery in 3 (22%) and status quo in 7 (50%).

Initial stem cell use in liver cirrhosis and meningomyelocele has suggested beneficial results. However, long-term evaluation in randomized controlled trials is essential to draw further conclusions.