Efficacy and the Safety of Granulocyte Colony-Stimulating Factor Treatment in Patients with Muscular Dystrophy: A Non-Randomized Clinical Trial

IntroductionThe current standard treatment for patients with Duchenne muscular dystrophy (DMD) involves corticosteroids. Granulocyte colony-stimulating factor (G-CSF) induces the proliferation of satellite cells and myoblasts and, in turn, muscle regeneration. Beneficial effects of G-CSF were also described for skeletal muscle disorders.AimWe assessed the safety and effects of using G-CSF to promote muscle strength in patients with DMD.Materials and methodsInclusion criteria were as follows: patients aged 5–15 years with diagnosed with DMD confirmed by genetic test or biopsy. Fourteen patients were treated with steroids, and their use was not changed in this study. Diagnoses were confirmed by genetic tests: deletions were detected in 11 patients and duplications in 5 patients. Nineteen 5- to 15-year-old patients diagnosed with DMD—9 were in wheelchairs, whereas 10 were mobile and independent—completed an open study. Participants received a clinical examination and performed physiotherapeutic and laboratory tests to gage their manual muscle strength, their isometric force using a hand dynamometer, and aerobic capacity [i.e., 6-min walk test (6MWT)] before and after therapy. Each participant received G-CSF (5 µg/kg/body/day) subcutaneously for five consecutive days during the 1st, 2nd, 3rd, 6th, and 12th month. Laboratory investigations that included full blood count and biochemistry were performed. Side effects of G-CSF treatment were assessed during each visit. During each cycle of G-CSF administration in the hospital, rehabilitation was also applied. All patients received regular ambulatory rehabilitation.ResultsThe subcutaneous administration of G-CSF improved muscle strength in participants. We recorded a significant increase in the distance covered in the 6MWT, either on foot or in a wheelchair, increased muscle force in isometric force, and a statistically significant decrease in the activity of the muscle enzyme creatine kinase after nearly every cycle of treatment. We observed no side effects of treatment with G-CSF.ConclusionOur findings suggest that G-CSF increases muscle strength in patients with DMD, who demonstrated that G-CSF therapy is safe and easily tolerable

Effects of granulocyte colony-stimulating factor therapy for osteogenesis imperfecta: a case report

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

Quality of life of children suffering from motor disabilities as evaluated by their parents

Purpose: We assessed the quality of life of children with motor disabilities in comparison with healthy children, as evaluated by their parents, using the CHQ-PF28 questionnaire (Child Health Questionnaire—Parent Form). Materials and methods: In a prospective study, we evaluated the quality of life of 105 children with motor disabilities. Results: Our research showed lower quality of life in the group of children with motor disabilities compared with controls, both in terms of physical and psychosocial health. Significant correlations between independent walking and physical functioning, general behavior, and mental health of children suffering from motor disabilities were found. According to the average indices of quality of life of children suffering from motor disabilities, depending on sex, the greatest differences occurred in behavior and change of health status, while the smallest differences in self-esteem and parental involvement, compared with controls. In the case of healthy children, the largest differences appeared in the perception of pain, behavior, and self-esteem; whereas, the smallest variations occurred in the change of health status and physical activity. Conclusion: Children suffering from motor disabilities demonstrate lower quality of life compared with healthy children

Quality of life of children suffering from motor disabilities as evaluated by their parents

Purpose: We assessed the quality of life of children with motor disabilities in comparison with healthy children, as evaluated by their parents, using the CHQ-PF28 questionnaire (Child Health Questionnaire—Parent Form). Materials and methods: In a prospective study, we evaluated the quality of life of 105 children with motor disabilities. Results: Our research showed lower quality of life in the group of children with motor disabilities compared with controls, both in terms of physical and psychosocial health. Significant correlations between independent walking and physical functioning, general behavior, and mental health of children suffering from motor disabilities were found. According to the average indices of quality of life of children suffering from motor disabilities, depending on sex, the greatest differences occurred in behavior and change of health status, while the smallest differences in self-esteem and parental involvement, compared with controls. In the case of healthy children, the largest differences appeared in the perception of pain, behavior, and self-esteem; whereas, the smallest variations occurred in the change of health status and physical activity. Conclusion: Children suffering from motor disabilities demonstrate lower quality of life compared with healthy children

Effects of granulocyte colony-stimulating factor therapy for osteogenesis imperfecta: a case report

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

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

Introduction: Recent reports have revealed that 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 observation

Granulocyte colony-stimulating factor therapy for facioscapulohumeral dystrophy: a case report

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