Janusz Wojtkowski

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.

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 childs age, parents 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 - Cronbachs 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 childs age and parents age and the childs quality of life. Whereas we determined dependencies between the childs 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.

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.

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.

Recombinant Granulocyte Colony-Stimulating Factor Increases Muscle Strength in Neuromuscular Disorders

To the Editor: Granulocyte-colony stimulating factor is a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream. A human recombinant form of granulocyte colonystimulating factordfilgrastimdis commonly used to treat neutropenia after chemotherapy. The healing process of an injured muscle can be divided into three different phases.1 The first phase, or the necrosisdegeneration and inflammation phase, occurs within the firstminutes and continues for up to 2weeks after injury. The second phase, or the regeneration repair phase, begins in the firstweek after injuryandpeaks at 14days. Finally, scar-tissue formation (third phase) takes place during week 2 and increases over time for up to 4weeks after injury. Furthermore, the time between injury and initiation of the proliferation is affected by several factors and metabolic state of the muscle. Filgrastim induces also neurogenesis, exerts antiapoptotic effects in neurons and muscles, and has anti-inflammatory properties.2 Recent studies have demonstrated that granulocyte colony-stimulating factor has regenerating and repairing functions in skeletal-muscle regeneration therapy.3 It has been also demonstrated that, after injuries that crush the muscles, the injection of granulocyte-colony stimulating factor daily improves muscular regeneration. The findings that stem cells other than satellite cells could also contribute to muscle regeneration led to studies moving away from using satellite cells/myoblasts, towards atypical stem cells.4 Filgrastim can potentially be used for the treatment of spinal cord injury, stroke, and neurodegenerative diseases.5 Recently, we examined the safety and effectiveness of filgrastim in a 15-year-old boy with facioscapulohumeral dystrophy and in a 15-year-old boy with congenital kyphoscoliosis along with spastic paraparesis. Filgrastim 5 mg/kg was given subcutaneously daily for 5 days/month for 3 months.5 Laboratory tests, CD34þ cells, and electromyography were performed. Clinical examination revealed a significant increase of muscle strength in the lower and upper limbs in the patient with facioscapulohumeral dystrophy between baseline and day 90, and day 180. A significant increase of muscle strength in the upper limbs in the patient with spastic paraparesis was observed.

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 GCSF 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 welltolerated. 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 observations.

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.

Effects of short moderate exercise on hematological parameters and stem cells in healthy humans

_________________________________________________________________________ Introduction: Exercise at various durations and intensities impact on blood and stem cells. This pilot study examined the effects of 30 minutes of treadmill walking on hematological indices and progenitor stem cells CD34+ in healthy subjects. Materials and methods: A total of 17 non-smoking, healthy students, aged 20 to 22 years participated. Hemoglobin, hematocrit, white blood cells, platelets, and stem cell CD34+ numbers were assessed before and after moderate exercise. Statistical analyses examined the relationships between CD34+ cells versus hematological indices, age, and body mass index. Results: Following exercise, significant increases were observed in leukocytes, neutrophils, eosinophils, and CD34+ cells numbers. For CD34+ cells, a fourfold increase was seen. Significant correlations between CD34+ cells, white blood cells, and neutrophils were found. Conclusion: Our results suggest that moderate exercise has a physiological impact on hematologic parameters and stem cells CD34+ in healthy subjects. Furthermore, our findings suggest that brief treadmill exercise may enhance tissue repair mechanisms so important in physiotherapy.