Safety and tolerability of intrathecal delivery of autologous bone marrow nucleated cells in children with cerebral palsy: an open-label phase I trial.

Autor: Mancías-Guerra C; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México. Electronic address: consuelomanciasg@gmail.com., Marroquín-Escamilla AR; Neuropediatrics Department, Hospital Universitario 'Dr. José Eleuterio González,' Universidad Autónoma de Nuevo León, Monterrey, México., González-Llano O; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Villarreal-Martínez L; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Jaime-Pérez JC; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., García-Rodríguez F; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Valdés-Burnes SL; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Rodríguez-Romo LN; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Barrera-Morales DC; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Sánchez-Hernández JJ; Universidad Autónoma de Madrid, Madrid, Spain., Cantú-Rodríguez OG; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Gutiérrez-Aguirre CH; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Gómez-De León A; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Elizondo-Riojas G; Radiology and Imaging Department, Hospital Universitario 'Dr. José Eleuterio González,' Universidad Autónoma de Nuevo León, Monterrey, México., Salazar-Riojas R; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México., Gómez-Almaguer D; Hematology Service, Internal Medicine Department, Hospital Universitario 'Dr. José Eleuterio González', Universidad Autónoma de Nuevo León, Monterrey, México.
Jazyk: angličtina
Zdroj: Cytotherapy [Cytotherapy] 2014 Jun; Vol. 16 (6), pp. 810-20. Date of Electronic Publication: 2014 Mar 15.
DOI: 10.1016/j.jcyt.2014.01.008
Abstrakt: Background Aims: Cerebral palsy (CP) is related to severe perinatal hypoxia with permanent brain damage in nearly 50% of surviving preterm infants. Cell therapy is a potential therapeutic option for CP by several mechanisms, including immunomodulation through cytokine and growth factor secretion.
Methods: In this phase I open-label clinical trial, 18 pediatric patients with CP were included to assess the safety of autologous bone marrow-derived total nucleated cell (TNC) intrathecal and intravenous injection after stimulation with granulocyte colony-stimulating factor. Motor, cognitive, communication, personal-social and adaptive areas were evaluated at baseline and 1 and 6 months after the procedure through the use of the Battelle Developmental Inventory. Magnetic resonance imaging was performed at baseline and 6 months after therapy. This study was registered in ClinicaTrials.gov (NCT01019733).
Results: A median of 13.12 × 10(8) TNCs (range, 4.83-53.87) including 10.02 × 10(6) CD34+ cells (range, 1.02-29.9) in a volume of 7 mL (range, 4-10.5) was infused intrathecally. The remaining cells from the bone marrow aspirate were administered intravenously; 6.01 × 10(8) TNCs (range, 1.36-17.85), with 3.39 × 10(6) cells being CD34+. Early adverse effects included headache, vomiting, fever and stiff neck occurred in three patients. No serious complications were documented. An overall 4.7-month increase in developmental age according to the Battelle Developmental Inventory, including all areas of evaluation, was observed (±SD 2.63). No MRI changes at 6 months of follow-up were found.
Conclusions: Subarachnoid placement of autologous bone marrow-derived TNC in children with CP is a safe procedure. The results suggest a possible increase in neurological function.
(Copyright © 2014 International Society for Cellular Therapy. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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