A multicenter, open-label, phase III study of Abcertin in Gaucher disease.

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

Medicine

authors

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 HW

doi

10.1097/MD.0000000000008492

subject

Has Abstract

pub_date

2017-11-01 00:00:00

pages

e8492

issue

45

eissn

0025-7974

issn

1536-5964

pii

00005792-201711100-00036

journal_volume

96

pub_type

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