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.

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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.

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.

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.