and after multiple-dose administrations (30U/kg, n = 14; 60U/kg, n = 15) at week 38. PK parameters were determined using a noncompartmental analysis method. Mean maximum plasma concen- tration (Cmax), area under the plasma concentration curve (AUC)0-t, and AUC0-∞ values were higher after the 60U/kg dose than the 30U/kg dose on day 1 and at week 38. The mean time to Cmax (Tmax) ranged from 83-95 min, mean elimination half-life (t1/2) ranged from 25.0- 34.8 min, mean clearance rates (CL) ranged from 19.9-30.7 L/h, and mean volume of distribution (Vss) ranged from 11.7-17.5 L on day 1 and week 38, indicating no dependence on dose or sampling day. Pediatric PK were assessed in a multicenter trial of taliglucerase alfa safety and efficacy in pediatric patients (2-b 18 years of age) with GD. Serial blood samples for PK analysis in plasma were obtained from 10 pediatric patients who received taliglucerase alfa infusions of 30 and 60U/kg every 2 weeks for 10-27 mo. PK parameters were determined using a noncompartmental analysis method. Mean Cmax, AUC0-t, and AUC0-∞ values were higher at the 60U/kg (n = 4) than 30U/kg (n = 6) dose. After dose normalization, these values were similar. Mean values for Tmax and t1/2 were similar for the taliglucerase alfa at 30 and 60U/kg doses. Mean CL was 33% lower (17.0 vs 25.5 L/h, respectively) and Vss was 35% lower (10.7 vs 16.4 L, respectively) for the 60 vs 30U/kg dose groups. After normalization by body weight, these differences decreased to 16% and 23%, respectively, indicating linear PK. Following repeated infusions of taliglucerase alfa, observed mean t1/2 and CL values in pediatric patients (31.5-34.8 min and 17-25.5 L/h, respectively) were similar to those observed in adult patients, indicating comparable taliglucerase alfa PK in pediatric and adult patients with GD. These studies were sponsored by Protalix Biotherapeutics. Pediatric study PB- 06-006 is ongoing. Editorial and medical writing support was provided by Peloton Advantage, LLC, and was funded by Pfizer. doi:10.1016/j.ymgme.2013.12.014 3 Dental problems of Egyptian Gaucher disease children and infiltration of the dental pulp with Gaucher cells Magy Abdelwahab a , Gihan Abu Elniel b , a Cairo University Pediatric Hospital, Cairo, Egypt, b Cairo University, Cairo, Egypt Gaucher disease (GD) is the most prevalent autosomal recessive lysosomal disorder characterized by deficiency of acid β-glucosidase (glucocerebrosidase) resulting in accumulation of glucocerebroside- laden macrophages (Gaucher’s cells) in the reticuloendothelial system throughout the body, typically in the liver, spleen, bone marrow, lymph nodes, brain, and lungs. There are 3 phenotypes of GD; type I,the chronic non-neuronopathic variant and GD types 2 and 3, the neuronopathic variants. Some studies described man- dibulo-maxillofacial involvement in those patients and only a single recent report evaluated their oral health with none exclusively in children or describing dental abnormalities in dentin or enamel. Moreover, few reports described infiltration of the mandibular bone and rarely the maxilla with Gaucher cells with none referring to the dental pulp or other dental structures. However, in view of frequent dental complaints affecting the quality of life of some patients’ and the unusual severe dental problems observed we evaluated their dental condition. So, we assessed the general dental condition, hygiene and gingiva of GD children presenting with frequent recurrent blackish discolouration of multiple teeth and spontaneous breakage and evaluated them for any other abnormalities in shape, structure or cells to explain the underlying pathology. The study included eighteen children diagnosed as GD with enzyme assay and genotype, 17 GD type3 (L444P/L444P) and one GD type1 (R359Q/ R359Q) complaining of teeth problems with age ranging from 3 to 13 years,14 males and 4 females. All are on enzyme replacement therapy, 40-60 IU/kg/2 weeks and had complete oral and periodontal dental evaluation. Gingiva was assessed by gingival index and caries by caries indices (DMF for permanent teeth, DMF and def for mixed dentition stage and dmf for complete primary dentition). Five cases had teeth indicated for extraction and oral biopsy was done with ground and decalcified sections to evaluate enamel, dentin, cemen- tum and pulp tissue for significant pathological abnormalities. Our pediatric cohort of GD had no abnormalities in structure or outer appearance of the tooth. 88% of children showed gingivitis ranging from moderate to severe whilst all had carious lesions, as severe form of early childhood caries. Biopsy of permenant teeth revealed variable abnormalities in dentin structure with cell entrapment and loss of interdigitation in dentinoenamel junction while that of primary teeth showed fibrosed pulps and infiltration of dental pulp with Gaucher cells(fig). All type 3 GD have moderate to severe neurological involvement, 1 is epileptic, 1 has severe bony manifes- tations and 1 died of severe pulmonary involvement while type I GD patient had severe bony involvement. In conclusion, dental tissue is another site that can be infiltrated with Gaucher cells and one of the tissues that should be assessed in evaluating GD patients especially type 3 in the pediatric age group as the dental findings can be one of the clinical criteria assessing the disease severity. doi:10.1016/j.ymgme.2013.12.015 4 Lung involvement of Egyptian children with type 1 and 3 Gaucher disease: does enzyme replacement therapy have a role in reversing symptoms and can it be considered an index of disease severity? Magy Abdelwahab, Hadeel Seif, Rania Hesham, Cairo University Pediatric Hospital, Cairo, Egypt Gaucher disease (GD) is characterized by deficiency of gluco- cerebrosidase resulting in accumulation of glucocerebroside-laden macrophages (Gaucher’s cells) typically in the liver, spleen, bone marrow and brain. There are 3 phenotypes of GD; type I, the chronic non-neuronopathic variant and GD types 2 and 3, the neuronopathic variants. Lung involvement is a relatively rare manifestation of GD with variable severity,sometimes fatal and its response to Enzyme replacement therapy (ERT) is unclear. It is usually reported in severe type1 GD and GD type 3 (L444P/L444P). There are very few reports of children with no large prospective GD type 3 studies describing the clinical spectrum of lung involvement, radiological findings and response to ERT. Interstitial lung disease, ground-glass pattern, Abstracts S16