Bożena Okurowska-Zawada

Dysphagia in children with infantile cerebral palsy

PURPOSE Dysphagia is a significant health problem in children with infantile cerebral palsy (ICP), but not frequently discussed in the literature. The study objective was to analyse dysphagia symptoms in children with a pyramidal form of ICP, including the oral and pharyngeal phases of deglutition and dysarthria severity. We searched for a correlation between dysphagia severity and ICP type, mental development and occurrence of epilepsy. MATERIAL AND METHODS A total of 67 children with a pyramidal form of infantile cerebral palsy were studied. Data were obtained based on case history elicited from the mothers, analysis of medical and psychological documentation, and logopaedic examination, including an examination of the action of swallowing. RESULTS Dysphagia symptoms were found in 41 (61%) studied children, most frequently referring only to the oral phase (25 children), with concomitant mild and moderate dysarthria. Oral and pharyngeal dysfunctions were observed in 14 children and coexisted with more pronounced dysarthria symptoms. The most severe disorders were mainly found in the pharyngeal phase in 2 children. A statistically significant correlation was noted between the severity of dysphagia symptoms and the ICP type (p<0.044) and mental development (p<0.00002). CONCLUSIONS Swallowing dysfunctions occur in the majority of children (>50%) with ICP. More serious disorders involving the oral and pharyngeal phases mainly affect children with tetraplegia and profound mental impairment. These disorders continue from early infancy through childhood and adolescence and improvement has been mainly observed when only the oral phase of swallowing is affected. These are always accompanied by dysarthria symptoms, which are especially severe when dysphagia involves the oral and pharyngeal phases. Early assessment and stimulation of the swallowing function should be a common element in the rehabilitation and care of children with ICP.

Duchenne muscular dystrophy: current cell therapies:

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.

Quality of Life in Children and Adolescents With Cerebral Palsy and Myelomeningocele

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.

Clinical and neuroimaging profile of congenital brain malformations in children with spastic cerebral palsy.

PURPOSE Analysis of the incidence of congenital brain malformations in children with spastic cerebral palsy (CP) in a hospital-based study. MATERIAL AND METHODS The present study included 74 boys and 56 girls with spastic tetraplegia, diplegia, and hemiplegia CP. Magnetic resonance imaging MRI findings were analyzed in children with CP. RESULTS Significant abnormalities relevant to the CP were evident on MRI in 124 (95.3%) subjects. Periventicular leukomalacia (PVL) was detected more frequently in children with spastic diplegia than in patients with tetraplegia or hemiplegia. Cerebral atrophy was found more often in the tetraplegic group compared to the diplegic patients. Porencephalic cysts were detected more often in children with spastic hemiplegia. Congenital brain anomalies were evident in 15 (10.7%) children with spastic CP. Brain malformations included: schizencephaly (5), agenesis corpus callosum (4), polymicrogyria (2), holoprosencephaly (2) and lissencephaly (2). Intractable epilepsy and mental retardation were observed more often in children with brain anomalies. Twelve patients with congenital brain malformations were born at term and three born at preterm. CONCLUSIONS Neuroimaging results in children with CP may help determine the etiology and make better prognosis of CP.

Assessment of risk factors for osteoporosis and fractures in children with meningomyelocele

PURPOSE Our objective was to assess bone and muscular mass in children with meningomyelocele (MMC), and to analyze risk factors for osteoporosis and fractures based on densitometric examination. MATERIAL AND METHODS The study group included 30 patients (15 girls and 15 boys) with MMC, aged 6-17 years, treated in the Department of Pediatric Rehabilitation, University Hospital. Physiotherapeutic assessment and laboratory tests (serum parathormone, alkaline phosphatase levels, calcium, and phosphate levels, and urine calcium levels) were performed. Densitometry was measured by dual energy X-ray absorptiometry using a Lunar DPX-L apparatus. Lean mass (fat-free tissue content) and fat mass (% fat content) was evaluated. RESULTS Femur fractures were the most common 12/30 (40%); 5/30 (17%) of the children with MMC had multiple fractures. The incidence of fractures correlated significantly with BMI and body fat content (p = 0.03) Children with MMC and fractures had a tendency toward higher BMI, despite the same absolute value of body mass, compared to those without fractures. Body fat levels were higher in MMC patients with fractures than in those without fractures (BMI R = 0.393, p = 0.03). Children with MMC and fractures had significantly higher 24 h calcuria values, despite normal renal function indices (p = 0.03). CONCLUSIONS Low-energetic fractures in MMC children may result from metabolic disturbances that are a consequence of excessive renal calcium loss or excessive fatty tissue content.

Serum 25-hydroxyvitamin D, osteocalcin, and parathormone status in children with meningomyelocele.

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.

Schizencephaly as a cause of spastic cerebral palsy

PURPOSE The objective was to investigate the clinical features of schizencephaly in children with spastic cerebral palsy. MATERIAL AND METHODS The present study included 180 children with cerebral palsy, spastic tetraplegia, diplegia, and hemiplegia. All magnetic resonance (MR) scans were obtained using a 1.5 T MR scanner with the use of a standard circularly polarized head coil. RESULTS Significant abnormalities relevant to cerebral palsy were evident on MRI in 95%. Periventicular leukomalacia was detected more frequently in children with spastic diplegia than in other patients. Cerebral atrophy was found more often in tetraplegic patients. Porencephalic cysts were detected more often in children with spastic hemiplegia. Congenital brain anomalies were evident in 20 (11.1%) children with spastic cerebral palsy. Twelve patients had schizencephaly with cerebral palsy. Children with spastic diplegia and tetraplegia had bilateral schizencephaly; patients with spastic hemiplegia only had unilateral schizencephaly. Most patients with schizencephaly had epilepsy. CONCLUSIONS Schizencephaly occurred more often in patients with spastic hemiplegia. Early detection of brain abnormalities in children with cerebral palsy may help in the prognosis and in the introduction of appropriate therapy.

Effect of Periodic Granulocyte Colony-Stimulating Factor Administration on Endothelial Progenitor Cells and Different Monocyte Subsets in Pediatric Patients with Muscular Dystrophies.

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.

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.

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.