title: [Cliniko-laboratory indicators of efficiency fermento-substitution therapy of Gaucher disease in Ukraine].
abstract: The analysis of efficiency of treatment of 17 patients with Gaucher disease (GD) in Ukraine who had received fermento-substitution therapy for 2 years and more was conducted on the basis of clinical and laboratory monitoring data. Regular infusions of recombinant glucocerebroside reduced signs of hepatosplenomegaly and pancytopenia, reduced a bone pain and a bone crisis at the majority of patients with GD I type that led to considerable improvement of health state and improvement of patients life quality. Efficiency of treatment depended on regularity of drug administration, dosage and severity level of the disease at the start of the therapy. Adult patients were not seen to have corrections of bones and neurologic disorders after the treatment that confirmed necessity of an early initiation of the treatment, before formation of irreversible changes in these organs and systems. Chitiotriodase activity in blood plasma is the most complex laboratory indicator which displays activity of pathological process in patients with GD, therefore it is necessary to use it for an estimation of treatment efficiency to correct a recombinant glucocerebroside dosage.
authors.forename: N V
authors.initials: NV
authors.lastname: Ol'khovych
authors.forename: O M
authors.initials: OM
authors.lastname: Gryshchenko
authors.forename: N O
authors.initials: NO
authors.lastname: Pichkur
authors.forename: A M
authors.initials: AM
authors.lastname: Nedoboĭ
authors.forename: N S
authors.initials: NS
authors.lastname: Trofimova
authors.forename: T P
authors.initials: TP
authors.lastname: Ivanova
authors.forename: N G
authors.initials: NG
authors.lastname: Gorovenko
mesh_terms: D000293:Adolescent; D000328:Adult; D015415:Biomarkers / Q000097:blood*; D001842:Bone and Bones; D002648:Child; D004334:Drug Administration Schedule; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D005776:Gaucher Disease / Q000097:blood / Q000188:drug therapy / Q000503:physiopathology; D005962:Glucosylceramidase / Q000008:administration & dosage / Q000172:deficiency / Q000627:therapeutic use; D005963:Glucosylceramides / Q000097:blood*; D006454:Hemoglobins / Q000032:analysis; D006529:Hepatomegaly / Q000097:blood / Q000503:physiopathology; D006801:Humans; D007262:Infusions, Intravenous; D007275:Injections, Intravenous; D008297:Male; D010146:Pain; D010198:Pancytopenia / Q000097:blood*; D011788:Quality of Life; D012720:Severity of Illness Index; D013156:Splenectomy; D013163:Splenomegaly / Q000097:blood / Q000503:physiopathology; D016896:Treatment Outcome; D014455:Ukraine
chemical_list: D015415:Biomarkers; D005963:Glucosylceramides; D006454:Hemoglobins; D005962:Glucosylceramidase