Hematology Service, Internal Medicine Department, Hospital Universitario "Dr. José Eleuterio González", Universidad Autónoma de Nuevo León, Monterrey, México.
Neuropediatrics Department, Hospital Universitario "Dr. José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México.
Cytotherapy. 2014 Jun;16(6):810-20. doi: 10.1016/j.jcyt.2014.01.008. Epub 2014 Mar 15.
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.
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).
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.
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.
脑瘫（CP）与近 50%存活的早产儿围产期严重缺氧导致的永久性脑损伤有关。细胞疗法是通过多种机制治疗 CP 的一种潜在治疗选择，包括通过细胞因子和生长因子分泌进行免疫调节。
在这项 I 期开放性临床试验中，纳入了 18 名 CP 儿科患者，以评估在粒细胞集落刺激因子刺激下进行自体骨髓来源的总核细胞（TNC）鞘内和静脉注射的安全性。通过使用 Battelle 发育性婴儿测试量表，在基线、治疗后 1 个月和 6 个月评估运动、认知、沟通、个人-社会和适应领域。在基线和治疗后 6 个月进行磁共振成像。该研究在 ClinicaTrials.gov 注册（NCT01019733）。
鞘内输注了中位数为 13.12×10^8^ TNC（范围，4.83-53.87），包括 10.02×10^6^ CD34+细胞（范围，1.02-29.9），体积为 7 mL（范围，4-10.5）。骨髓抽吸物中的剩余细胞经静脉输注；6.01×10^8^ TNC（范围，1.36-17.85），其中 3.39×10^6^细胞为 CD34+。早期不良反应包括 3 名患者出现头痛、呕吐、发热和颈项强直。未记录到严重并发症。根据 Battelle 发育性婴儿测试量表，观察到发育年龄总体增加了 4.7 个月，包括所有评估领域（±SD 2.63）。在 6 个月的随访中未发现 MRI 变化。
CP 儿童蛛网膜下腔自体骨髓来源 TNC 的放置是一种安全的操作。结果表明神经功能可能有所增强。
