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Effects of granulocyte colony-stimulating factor treatment in children and patients with cerebral palsy: a preliminary report

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Paszko-Patej G., Kułak W., Okurowska-Zawada B., Sienkiewicz D., Wojtkowski J., Sochoń K., Kalinowska A., Okulczyk K., Topór E., Mirska A.
Progress in Health Sciences
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2017
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vol. 7(1)
78-82
EN
neuroinflammation and apoptosis in brains affected by cerebral palsy could be therapeutic targets. Granulocyte colony-stimulating factor (G-CSF) exerts anti-inflammatory and antiapoptosis effects and stimulates the proliferation of neural stem and progenitor cells in the brain. Purpose: To assess the efficacy and safety of G-CSF treatment in children and adolescents with CP. Materials and methods: Six patients with spastic tetraplegia CP aged 3-15 years were enrolled in this study. Five patients had GMFCS (Gross Motor Function Classification System) level at V, three children had epilepsy, and three had severe mental retardation. We used the gross motor function measure-66 (GMFM-66) to assess motor function.GCSF (5μg/kg/body/day) was administered subcutaneously for five consecutive days during the four months. The parents also evaluated the physical and mental development of their children. Results: We observed improvement in motor function in patients with CP on the GMFM-66 scale. Parents reported improvement in behavior, speech development, and a decrease in spasticity in children with CP. G-CSF therapy was well-tolerated. No side effects were observed during the study. Conclusions: Our preliminary report suggests that G-CSF treatment improves motor and mental function in patients with CP. Further studies are needed to confirm these observations
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Safety and efficacy of granulocyte colony stimulating factor in a patient with tetraplegia caused by cervical hyperextension injury: a case report

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Okurowska-Zawada B., Kułak W., Sienkiewicz D., Paszko-Patej G., Dmitruk E., Kalinowska A., Wojtkowski J., Korzeniecka–Kozerska, A
Progress in Health Sciences
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2014
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vol. 4
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issue 2
181-184
EN
he authors present the case of a 17-year-old boy who suffered a cervical spinal injury as a result of the sharp bending of the head after slipping (without falling). After about 30 minutes, he began to feel tingling in the limbs and he developed tetraparesis. He went through physical rehabili-tation, psychological rehabilitation, occupational therapy, and periodic catheterization. Additionally, we introduced to him a low dose of analog granulocyte colony-stimulating factor (G-CSF). G-CSF 5 μg/kg was given subcutaneously daily for 5 days per month for 3 months, again after 6 months, and again after 10 months. The boy could sit indecently and walk with assistance. A significant increase in muscle strength in this patient with tetraplegia after 10 months of treatment may indicate beneficial effects of G-CSF in this disorder.
3
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Effects of granulocyte colony-stimulating factor therapy for osteogenesis imperfecta: a case report

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Paszko-Patej G., Sienkiewicz D., Kułak W., Okurowska-Zawada B., Wojtkowski J., Kalinowska A., Okulczyk K., Sochoń K., Dmitruk E., Mirska A.
Progress in Health Sciences
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2017
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vol. 7(1)
205-208
EN
Introduction: Osteogenesis imperfecta (OI) is a genetic disorder of increased bone fragility and low bone mass. OI type IV. Materials and methods: We examined the safety and effectiveness of a low dose of analog granulocyte colony-stimulating factor (G-CSF) in a 15-year-old girl OI type IV. G-CSF 5 μg/kg was given subcutaneously, for 5 days/month for 3, 6 and 12 months. Laboratory tests, including blood, biochemical tests were performed, in addition to clinical examination.Results: Clinical examination revealed an increase of muscle strength in the upper and lower limbs between base line and day 6 and 12 months. We found no serious adverse events. Leukocyte levels remained below 38,000/μL. Low dose G-CSF was safe and well tolerated by the patient. Conclusions: A significant increase in muscle strength in this patient may indicate beneficial effects of G-CSF factor in this disorder. These results are inspiring and warrant further studies.
4
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Granulocyte colony-stimulating factor therapy for facioscapulohumeral dystrophy: a case report

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Sienkiewicz D., Kułak W., Okurowska-Zawada B., Paszko-Patej G., Wojtkowski J., Dmitruk E., Okulczyk K., Sochoń K., Kalinowska A., Żak J., Pogorzelski R.
Progress in Health Sciences
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2016
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vol. 6(2)
175-177
EN
We examined the safety and effectiveness of a low dose of analog granulocyte-colony stimulating factor in a 15-year-old boy with facioscapulohumeral dystrophy. The onset of disease was noted at 12 years of age. The physical examination noted general muscle atrophy more pronounced at left side of the body. He was able to walk 300 meters within 6 minute walk test. Granulocyte colony-stimulating factor 5 μg/kg was given subcutaneously daily for 5 days/month for 1, 2, 3, 6 and 12 months. Clinical examination, laboratory tests including blood, biochemical tests, and CD34+ cells were performed. A significant increase of muscle strength in the lower and upper limbs between baseline, and after 3 months of treatment, after 6, and after 12 months was found. He was able to walk 480 meters within 6 minutes after 12 months. Electromyography demonstrated increase of amplitude in the examined in upper and lower limbs after six months compared to baseline. Leukocyte levels remained below 25000/μL. CD34+ increased significantly at day 5 of granulocyte colony-stimulating factor admini-stration. It was safe and well tolerated by the patient. A significant increase in muscle strength in this patient with facioscapulohumeral dystrophy after 3 months of treatment, after 6, and after 12 months since the first treatment course was completed may indicate beneficial effects of granulocyte colony-stimulating factor in this disorder.
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