@article {150454, title = {A multicenter, open-label, phase III study of Abcertin in Gaucher disease.}, journal = {Medicine}, volume = {96}, year = {2017}, month = {2017 Nov}, pages = {e8492}, abstract = {

BACKGROUND: Gaucher disease (GD) is caused by a deficiency in the lysosomal enzyme glucocerebrosidase. Enzyme replacement therapy (ERT) is recommended for clinical improvement.

METHODS: The efficacy and safety of a new imiglucerase, Abcertin, were assessed in 7 Egyptian patients with treatment-na{\"\i}ve type 1 GD. Each patient was administered a biweekly 60 U/kg dose of Abcertin for 6 months. The primary endpoint was the change in hemoglobin concentration. The secondary endpoints were changes from baseline in platelet counts, spleen and liver volumes, biomarker levels, skeletal parameters, and bone mineral density.

RESULTS: The hemoglobin concentration increased by a mean of 1.96 {\textpm} 0.91 g/dL (range 1.11-2.80 g/dL) or 20.6\% (P = .001). Statistically significant increases in the platelet count and decreases in the spleen volume and biomarker levels were also observed. There were no severe drug-related adverse events. One patient developed anti-imiglucerase antibodies without neutralizing activity.

CONCLUSION: Our study results demonstrate the efficacy and safety of Abcertin in patients with type 1 GD. This suggests that Abcertin can be an alternative ERT option for type 1 GD.

}, keywords = {Adolescent, Area Under Curve, Biomarkers, Bone Density, Child, Child, Preschool, Egypt, Enzyme Replacement Therapy, Gaucher Disease, Glucosylceramidase, Half-Life, Hemoglobins, Humans, Liver, Male, Metabolic Clearance Rate, Organ Size, Recombinant Proteins, Spleen}, issn = {1536-5964}, doi = {10.1097/MD.0000000000008492}, author = {Lee, Beom Hee and Abdalla, Ahmed Fathy and Choi, Jin-Ho and Amal ElBeshlawy and Kim, Gu-Hwan and Heo, Sun Hee and Megahed, Ahmed Megahed Hassan and Elsayed, Mona Abdel Latif and Barakat, Tarik El-Sayed Mohammad and Eid, Khaled Mohamed Abd El-Azim and El-Tagui, Mona Hassan and Mahmoud, Mona Mohamed Hamdy and Fateen, Ekram and Park, June-Young and Yoo, Han-Wook} }