Safety and feasibility of countering neurological impairment by intravenous administration of autologous cord blood in cerebral palsy

Lee Young-Ho, Choi Kyung, Moon Jin, Jun Hyun-Joo, Kang Hye-Ryeong, Oh Se-In, Kim Hyung, Um Jang, Kim Mi, Choi Yun, Lee Young-Jun, Kim Hee-Jin, Lee Jong-Hwa, Son Su, Choi Soo-Jin, Oh Wonil, Yang Yoon-Sun

Description

s We conducted a pilot study of the infusion of intravenous autologous cord blood (CB) in children with cerebral palsy (CP) to assess the safety and feasibility of the procedure as well as its potential efficacy in countering neurological impairment. Methods Patients diagnosed with CP were enrolled in this study if their parents had elected to bank their CB at birth. Cryopreserved CB units were thawed and infused intravenously over 10~20 minutes. We assessed potential efficacy over 6 months by brain magnetic resonance imaging (MRI)-diffusion tensor imaging (DTI), brain perfusion single-photon emission computed tomography (SPECT), and various evaluation tools for motor and cognitive functions. Results Twenty patients received autologous CB infusion and were evaluated. The types of CP were as follows: 11 quadriplegics, 6 hemiplegics, and 3 diplegics. Infusion was generally well-tolerated, although 5 patients experienced temporary nausea, hemoglobinuria, or urticaria during intravenous infusion. Diverse neurological domains improved in 5 patients (25%) as assessed with developmental evaluation tools as well as by fractional anisotropy values in brain MRI-DTI. The neurologic improvement occurred significantly in patients with diplegia or hemiplegia rather than quadriplegia. Conclusions Autologous CB infusion is safe and feasible, and has yielded potential benefits in children with CP.

Sujets

• Cerebral palsy
• Cord blood
• Cell therapy

Informations

• Publié par: biomed
• Publié le: 01 janvier 2012
• Nombre de lectures: 14
• Langue: English
• Poids de l'ouvrage: 1 Mo

Extrait

Leeet al.Journal of Translational Medicine2012,10:58 http://www.translationalmedicine.com/content/10/1/58

R E S E A R C HOpen Access Safety and feasibility of countering neurological impairment by intravenous administration of autologous cord blood in cerebral palsy 1,5†1†1 21 1 YoungHo Lee, Kyung Vin Choi, Jin Hwa Moon , HyunJoo Jun , HyeRyeong Kang , SeIn Oh , 2 22* 34 56 Hyung Sun Kim , Jang Soo Um , Mi Jung Kim, Yun Young Choi , YoungJun Lee , HeeJin Kim , JongHwa Lee , 7 88 8 Su Min Son , SooJin Choi , Wonil Ohand YoonSun Yang

Abstract Backgrounds:We conducted a pilot study of the infusion of intravenous autologous cord blood (CB) in children with cerebral palsy (CP) to assess the safety and feasibility of the procedure as well as its potential efficacy in countering neurological impairment. Methods:Patients diagnosed with CP were enrolled in this study if their parents had elected to bank their CB at birth. Cryopreserved CB units were thawed and infused intravenously over 10~20 minutes. We assessed potential efficacy over 6 months by brain magnetic resonance imaging (MRI)diffusion tensor imaging (DTI), brain perfusion singlephoton emission computed tomography (SPECT), and various evaluation tools for motor and cognitive functions. Results:Twenty patients received autologous CB infusion and were evaluated. The types of CP were as follows: 11 quadriplegics, 6 hemiplegics, and 3 diplegics. Infusion was generally welltolerated, although 5 patients experienced temporary nausea, hemoglobinuria, or urticaria during intravenous infusion. Diverse neurological domains improved in 5 patients (25%) as assessed with developmental evaluation tools as well as by fractional anisotropy values in brain MRIDTI. The neurologic improvement occurred significantly in patients with diplegia or hemiplegia rather than quadriplegia. Conclusions:Autologous CB infusion is safe and feasible, and has yielded potential benefits in children with CP. Keywords:Cerebral palsy, Cord blood, Mononuclear cells, Cell therapy

Backgrounds Cord blood (CB) was introduced for the first time in humans to reconstitute the hematopoietic system in patient with Fanconi anemia [1]. Since the first cord blood transplantation (CBT), more than 20,000 CBTs have been reported worldwide and more than 400,000 CB units have been stored in more than 100 CB banks [2]. The clinical use of CB has expanded into various areas such as inherited metabolic disorders. CBT for Hurler syndrome resulted in either stabilization or

* Correspondence: [e-mail] †Contributed equally 2 Department of Rehabilitation Medicine, Hanyang University Medical Center, Seoul, Korea Full list of author information is available at the end of the article

improvement of neurocognitive function, and mainte nance of new skills [3]. CBT for infantile Krabbe disease was highly effective if patients received transplants early in the course of the disease [4]. In such patients, CBT can prevent demyelination in the central and, often, the peripheral nervous system, extending life and improving overall quality of life. Kurtzberg, et al found that donor cells could enter the brain and induce remyelination and improvement in neurologic function in demyelinat ing diseases. Cerebral palsy (CP) describes a group of permanent disorders of movement and posture limiting activity, due to nonprogressive disturbances that occurred in the developing fetal or infant brain [5]. The ultimate goal of any therapy program for CP is to help children achieve

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