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

INTRODUCTION: The 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. AIM: We assessed the safety and effects of using G-CSF to promote muscle strength in patients with DMD. MATERIALS AND METHODS: Inclusion 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. RESULTS: The 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. CONCLUSION: Our findings suggest that G-CSF increases muscle strength in patients with DMD, who demonstrated that G-CSF therapy is safe and easily tolerable.

Use of botulinum toxin in the treatment of ankle plantar flexor spasticity in children with cerebral palsy.

The aim of this study was to assess the effects of botulinum on spasticity of gastrocnemius and soleus muscles. Forty-one children with spastic cerebral palsy were assessed (muscle tone, range of motion of ankle joint extension with straightened and bent knee, and gait pattern using the Physician Rating Scale) before administration and 2, 6, and 13 weeks after. Changes on Physician Rating Scale and dorsiflexion with extended knee were significant after 2, 6, and 13 weeks. Differences in the remaining parameters were significant after the first two check-ups. Over 90% of the changes were positive. This research confirms the effectiveness of botulinum in reducing spasticity, increasing the range of motion, and improving the gait pattern.

Potential beneficial effects of granulocyte colony-stimulating factor therapy for spastic paraparesis in a patient with kyphoscoliosis: a case report.

Congenital kyphosis and kyphoscoliosis are much less common than congenital scoliosis and more serious because these curves can progress rapidly and can lead to spinal cord compression and paraplegia. A 15-year-old boy presented with congenital kyphoscoliosis along with spastic paraparesis (American Spinal Injury Association Impairment Scale grade C). We examined the safety and effectiveness of a low dose of analog granulocyte colony-stimulating factor (G-CSF) in this patient. G-CSF 5 µg/kg was given subcutaneously, daily for 5 days per month for 3 months. Laboratory tests, including blood, biochemical tests, and CD34+ cells (marker hematopoietic progenitor cells) were performed, in addition to clinical examination. Clinical examination revealed an increase of muscle strength in the upper limbs and decrease spasticity in the lower limbs between baseline and day 90 and day 180. We found no serious adverse event, drug-related platelet reduction, or splenomegaly. Leukocyte levels remained below 21,000/µL. CD34+ increased significantly at day 5 of G-CSF administration. Low-dose G-CSF was safe and well tolerated by the patient. A significant increase in muscle strength in this patient with spastic paraparesis after 3 months of treatment may indicate beneficial effects of G-CSF factor in this disorder. These results are inspiring and warrant further studies.

Psychometric properties and validation of the Polish CP QOL-Child questionnaire: a pilot study.

AIM: Translate, determine the psychometric properties and validate the Polish CP QOL-Child questionnaire. MATERIALS AND METHODS: A double translation of the questionnaire from English into Polish and back was executed. The questionnaire was distributed to 55 parents/legal guardians of children with cerebral palsy aged 4-12 years. The psychometric properties of the questionnaire were determined on the basis of its internal consistency and the internal consistency of each of the investigated aspects, as well as on the assessment of the relationship between quality of life and such data as child's age, parent's age, place of residence and GMFCS level. RESULTS: The results showed high levels of internal consistency of the Polish version of the CP QOL-Child questionnaire - Cronbach's α was between 0.77 and 0.82, which is comparable to the original scale, where it was 0.74-0.92. In addition, we found no relationship between child's age and parent's age and the child's quality of life. Whereas we determined dependencies between the child's GMFCS level and quality of life in areas such as emotional state (p = 0.025), pain and the effects of disability (p = 0.033), and to a lesser extent participation in social life (p = 0.045). However, Spearman test presented that only domain pain and impact of disability reported positive correlation r = 0.43. CONCLUSION: Studies showed that English language the CP QOL-Child questionnaire was successful translated into Polish which is confirmed by the results of the assessment of the psychometric properties and validation of the Polish language questionnaire. The results of our study indicate that the Polish language version of the CP QOL-Child questionnaire is an appropriate tool to assess the quality of life of Polish-speaking children with cerebral palsy aged 4-12 years.