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-naïve 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 ± 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.
journal_name
Medicine (Baltimore)journal_title
Medicineauthors
Lee BH,Abdalla AF,Choi JH,Beshlawy AE,Kim GH,Heo SH,Megahed AMH,Elsayed MAL,Barakat TEM,Eid KMAE,El-Tagui MH,Mahmoud MMH,Fateen E,Park JY,Yoo HWdoi
10.1097/MD.0000000000008492subject
Has Abstractpub_date
2017-11-01 00:00:00pages
e8492issue
45eissn
0025-7974issn
1536-5964pii
00005792-201711100-00036journal_volume
96pub_type
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