Alterations in Stem Cell Populations in IGF-1 Deficient Pediatric Patients Subjected to Mecasermin (Increlex) Treatment.

Pathway involving insulin-like growth factor 1 (IGF-1) plays significant role in growth and development. Crucial role of IGF-1 was discovered inter alia through studies involving deficient patients with short stature, including Laron syndrome individuals. Noteworthy, despite disturbances in proper growth, elevated values for selected stem cell populations were found in IGF-1 deficient patients. Therefore, here we focused on investigating role of these cells-very small embryonic-like (VSEL) and hematopoietic stem cells (HSC), in the pathology. For the first time we performed long-term observation of these populations in response to rhIGF-1 (mecasermin) therapy. Enrolled pediatric subjects with IGF-1 deficiency syndrome were monitored for 4-5 years of rhIGF-1 treatment. Selected stem cells were analyzed in peripheral blood flow cytometrically, together with chemoattractant SDF-1 using immunoenzymatic method. Patients' data were collected for correlation of experimental results with clinical outcome. IGF-1 deficient patients were found to demonstrate initially higher levels of VSEL and HSC compared to healthy controls, with their gradual decrease in response to therapy. These changes were significantly associated with SDF-1 plasma levels. Correlations of VSEL and HSC were also reported in reference to growth-related parameters, and IGF-1 and IGFBP3 values. Noteworthy, rhIGF-1 was shown to efficiently induce development of Laron patients achieving at least proper rate of growth (compared to healthy group) in 80% of subjects. In conclusion, here we provided novel insight into stem cells participation in IGF-1 deficiency in patients. Thus, we demonstrated basis for future studies in context of stem cells and IGF-1 role in growth disturbances.

Botulinum toxin injection as primary treatment for esotropia in patients with cerebral palsy.

PURPOSE: Botulinum toxin type A is a potent neurotoxin that blocks the release of acetylocholine at the neuromuscular junction of cholinergic nerves. Cerebral palsy is cause of ocular disorders. There is an increased presence of strabismus, refractive errors, and reduced visual acuity. The purpose of this study was to assess the efficacy of botulinum toxin injection in the treatment of esotropia in patients with cerebral palsy. MATERIAL AND METHODS: Seven patients were included in the study. All patients had a full ophthalmic examination on initial visit, including cycloplegic refraction and duction. The angle of esotropic deviation at distance was recorded in prism diopters. The botulinum toxin type A was administered into the medial rectus muscle under general anesthesia. RESULTS: Mean age of the patients was 12 years. The mean angle of deviation pretreatment was 36.6 PD. Successful motor alignment (orthotropia +/- 10 PD) was achieved in the botulinum toxin type A group in 57.1% of patients. CONCLUSIONS: The use of botulinum toxin in the treatment of esotropia in children and adolescents with cerebral palsy is an alternative to conventional surgical therapy.