지원사업
학술연구/단체지원/교육 등 연구자 활동을 지속하도록 DBpia가 지원하고 있어요.
커뮤니티
연구자들이 자신의 연구와 전문성을 널리 알리고, 새로운 협력의 기회를 만들 수 있는 네트워킹 공간이에요.
논문 기본 정보
- 자료유형
- 학술저널
- 저자정보
- 발행연도
- 2015.1
- 수록면
- 378 - 384 (7page)
이용수
초록· 키워드
오류제보하기
Gaucher disease is a lysosomal storage disease for which enzyme replacement therapy hasproven to be effective. A switch-over clinical trial was performed to evaluate the efficacyand safety of Abcertin® (ISU Abxis, Seoul, Korea) in subjects with type 1 Gaucher diseasewho were previously treated with imiglucerase. Five Korean patients with type 1 Gaucherdisease were enrolled. Previous doses of imiglucerase ranged from 30 to 55 U/kg everyother week. The same dose of Abcertin® was administered to all patients for 24 weeks.
Primary efficacy endpoints were changes in hemoglobin levels and platelet counts, and thesecondary efficacy endpoints included changes in liver and spleen volumes, serumbiomarkers, skeletal status and bone mineral density (BMD). During the study period, nostatistically significant changes were observed in all parameters including hemoglobinlevels and platelet counts, liver and spleen volumes, skeletal status and BMD. Abcertin®administration was continued in three patients for another 24 weeks as an extension of thestudy. Hemoglobin levels and platelet counts were maintained in all three patients. Inconclusion, the efficacy and safety of Abcertin® are similar to those of imiglucerase, andAbcertin® is an effective therapeutic agent for patients with type 1 Gaucher disease(Clinical Trial Registry No. NCT02053896 at www.clinicaltrials.gov).
목차
등록된 정보가 없습니다.
참고문헌 (22)
참고문헌 신청
Cox TM / 1997 / Gaucher’s disease : clinical features and natural history / Baillieres Clin Haematol 10:657~689
Scriver CR / 2001 / The metabolic & molecular bases of inherited disease / McGraw-Hill
Balicki D / 1995 / Gaucher disease / Medicine (Baltimore) 74:305~323
Starzyk K / 2007 / The long-term international safety experience of imiglucerase therapy for Gaucher disease / Mol Genet Metab 90:157~163
Barton NW / 1991 / Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher’s disease / N Engl J Med 324:1464~1470
Scriver CR / 2001 / The metabolic & molecular bases of inherited disease / McGraw-Hill
Balicki D / 1995 / Gaucher disease / Medicine (Baltimore) 74:305~323
Starzyk K / 2007 / The long-term international safety experience of imiglucerase therapy for Gaucher disease / Mol Genet Metab 90:157~163
Barton NW / 1991 / Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher’s disease / N Engl J Med 324:1464~1470
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