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INTRODUCTION: In addition to the "gold standard" of therapy-steroids and gene therapy-there are experimental trials using granulocyte-colony stimulating factor (G-CSF) for patients with Duchenne muscular dystrophy (DMD). The aim of this study was to present the biochemical changes in blood after repeating cycles of granulocyte-colony stimulating factor G-CSF therapy in children with DMD. MATERIALS AND METHODS: Nineteen patients, aged 5 to 15 years, with diagnosed DMD confirmed by genetic tests, participated; nine were in wheelchairs, and ten were mobile and independent. Patients had a clinical assessment and laboratory tests to evaluate hematological parameters and biochemistry. G-CSF (5µg/kg/day) was given subcutaneously for five days during five nonconsecutive months over the course of a year. RESULTS: We found a significant elevation of white blood cells, and the level of leucocytes returned to norm after each cycle. No signs of any inflammatory process were found by monitoring C-reactive protein. We did not detect significant changes in red blood cells, hemoglobin, and platelet levels or coagulation parameters. We found a significant elevation of uric acid, with normalization after finishing each treatment cycle. A significant decrease of the mean value activity of aspartate transaminase (AST) and alanine transaminase (ALT) of the G-CSF treatment was noted. After each five days of therapy, the level of cholesterol was significantly lowered. Also, glucose concentration significantly decreased after the fourth cycle. CONCLUSIONS: G-SCF decreased the aminotransferases activity, cholesterol level, and glucose level in patients with DMD, which may be important for patients with DMD and metabolic syndrome.

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PURPOSE: The aim of this study was to assess the effectiveness of long-term therapy with multiply botulinum neurotoxin (BoNT) injections. METHODS: In 2004-2010, 60 children with spastic cerebral palsy aged 2-16 were treated multiple botulinum toxin sessions (injections in gastrocnemius muscle and soleus muscles). In each patient, we rated muscle tone by Modified Ashworth Scale, passive range of motion in ankle joint with extended, and flexed knee joint and gait using the Physician Rating Scale. Assessment was done before and after injection, up to eight BoNT sessions. RESULTS: The generalized additive models showed that a single treatment effect was visible for 3 months. The number of injections did not impact the effectiveness. Improvement in muscle tone was greater in children with hemiplegia than diplegia (ß = - 0.294; p = 0.014). Improvement in range of motion with extended knee joint was greater in hemiplegic than diplegic types (ß = 0.414; p =0.002), and improvement in range of motion with flexed knee was greater in children with more severe impairment (Gross Motor Function Classification System III vs. I, ß = 0.0603, p = 0.025; V vs. I, ß = 0.691, p = 0.023). The gait improvement rate decreased with patient age (p = 0.007). CONCLUSIONS: BoNT therapy is effective regardless of the number of injection sessions and duration of treatment. However, it is affected by the patient's age, type of cerebral palsy, and degree of impairment.

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INTRODUCTION: Previous studies of cerebral palsy (CP) suggest that it seasonal variations in the incidence of CP. The purpose of this paper was to compare seasonal variations in the incidence of cerebral palsy (CP) in Podlaskie Province, Poland, between 1990-1999 (study 2005) and 2000-2014 (study 2017) in a retrospective case-controlled study. MATERIALS AND METHODS: Data were obtained from the hospital database. We compared CP births between January 1, 1990, and December 31, 1999, n = 212 (116 boys, 96 girls) and January 1, 2000, and December 31, 2014, n = 205 (114 boys, 91 girls). We used Cosinor analysis to examine the seasonality of CP births. RESULTS: The highest number of CP births occurred in spring and the lowest in winter, with intermediate values in summer and autumn. This seasonal pattern was significant for spring vs. winter. The peaks in the numbers of CP births occurred in May and August; the lowest numbers of CP births occurred in February, December, and November. In the 2017 study, we observed a slight increase in spastic tetraplegia and a decrease in mixed CP. No significant corrections between mean temperature and Apgar score, low birth weight, and asphyxia were found. CONCLUSIONS: Our study confirmed the existence of seasonal patterns for CP births.

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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.

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Muscular dystrophies (MD) are heterogeneous group of diseases characterized by progressive muscle dysfunction. There is a large body of evidence indicating that angiogenesis is impaired in muscles of MD patients. Therefore, induction of dystrophic muscle revascularization should become a novel approach aimed at diminishing the extent of myocyte damage. Recently, we and others demonstrated that administration of granulocyte colony-stimulating factor (G-CSF) resulted in clinical improvement of patients with neuromuscular disorders. To date, however, the exact mechanisms underlying these beneficial effects of G-CSF have not been fully understood. Here we used flow cytometry to quantitate numbers of CD34+ cells, endothelial progenitor cells, and different monocyte subsets in peripheral blood of pediatric MD patients treated with repetitive courses of G-CSF administration. We showed that repetitive cycles of G-CSF administration induced efficient mobilization of above-mentioned cells including cells with proangiogenic potential. These findings contribute to better understanding the beneficial clinical effects of G-CSF in pediatric MD patients.

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Duchenne muscular dystrophy is a genetically determined X-linked disease and the most common, progressive pediatric muscle disorder. For decades, research has been conducted to find an effective therapy. This review presents current therapeutic methods for Duchenne muscular dystrophy, based on scientific articles in English published mainly in the period 2000 to 2014. We used the PubMed database to identify and review the most important studies. An analysis of contemporary studies of stem cell therapy and the use of granulocyte colony-stimulating factor (G-CSF) in muscular dystrophy was performed.

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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.

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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.

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Sufficient vitamin D levels are required for normal skeletal development and mineralization. This is particularly important in children with meningomyelocele who are at an increased risk of osteoporosis. The purpose of this study was to assess serum 25-hydroxyvitamin D [25(OH)D] and the biochemical markers of bone metabolism (parathormone, osteocalcin, alkaline phosphatase, and electrolytes) in children with meningomyelocele. The patient group comprised 33 children with meningomyelocele. The mean 25(OH)D was 11.51 ± 7.87 ng/mL. A total of 97% of the subjects had a 25(OH)D level in the insufficient range (< 30 ng/mL) and 48.5% had a 25(OH)D level less than 10 ng/mL. Almost all patients had serum osteocalcin and phosphorus concentrations above the normal limits. The level of 25(OH)D negatively correlated with age and body weight. There were no correlations between the biochemical markers of bone metabolism and the ambulatory status. A significant correlation between serum 25(OH)D and osteoporosis was found.

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INTRODUCTION: Low back pain (LBP) is one of the most common disorders affecting office employees working with a computer, which inevitably leads to lower quality of life. The aim of this study was to analyze the quality of life of patients with LBP after application of TENS (Transcutaneous Electrical Nervous Stimulation). MATERIAL AND METHODS: The study group included 30 computer workers (26 females and 4 males) aged 30-60 years (45 +/- 12). The quality of life was assessed using the World Health Organization Quality of Life instrument-Abbreviated version (WHOQOL-Bref) before and after 10 applications of TENS. Outcomes were evaluated with a visual analog score (VAS) for pain and Schober's test to measure the ability of a patient to flex his or her lower back. RESULTS: The mean VAS value decreased significantly from 3.83 +/- 1.31 cm at baseline to 3.36 +/- 1.21 cm after treatment. Patients reported pain relief. The highest quality of life scores in terms of social relationships were found in the social category evaluating personal relationships, social support and sexual activity (15.91 +/- 2.07) and mental health (14.32 +/- 1.59). After treatment a significant increase in the flexion of lower back was observed in the majority of patients. No significant correlations between the quality of life and the intensity of pain and the flexion of lower back before and after treatment were found. CONCLUSIONS: TENS therapy is an effective technique for pain relief in patients with LBP. TENS can also be used with other methods of LBP treatment and may improve the patients' quality of life.

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The aim of this study was to compare health-related quality of life in children with cerebral palsy and with myelomeningocele. Fifty-seven children with spastic cerebral palsy and 34 patients with myelomeningocele aged 5-16 years were included in the study. Their mothers completed standardized measures on the Revidierter Kinder Lebensqualitätsfragebogen (KINDL-R) parent questionnaire. The 2 groups were demographically comparable. The children with cerebral palsy were classified more frequently into levels II (n = 24) and III (n = 18) of the Gross Motor Function Classification System. Other patients were classified into levels IV (n = 5) and V (n = 10). Three patients with myelomeningocele were community walkers, 10 could walk with assistive devices, and 21 used a wheelchair. Lesion level was thoracic in 13 patients, lumbar in 17, and sacral in 4. Twenty-nine patients (85.3%) with myelomeningocele had hydrocephalus, and 27 had a shunt. Parents in the both studied groups reported similar overall quality of life of their children in the dimensions of physical and emotional well-being, self-esteem, family, friends, and school. No significant correlations between the quality-of-life scores and age, walking ability, and mental development of the studied groups were found.

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