Haemostatic abnormalities in treatment-naïve patients with Type 1 Gaucher's disease

There is a paucity of data on the effects of enzyme replacement therapy (ERT) on the coagulation abnormalities and platelet function of patients with Gaucher's disease (GDPs) and much of this data are controversial. This study investigates the haemostatic parameters in treatment-naïve GDPs and...

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Bibliographic Details
Published in:Platelets (Edinburgh) 2012-03, Vol.23 (2), p.143-149
Main Authors: Mitrovic, Mirjana, Antic, Darko, Elezovic, Ivo, Janic, Dragana, Miljic, Predrag, Sumarac, Zorica, Nikolic, Tanja, Suvajdzic, Nada
Format: Article
Language:English
Subjects:
Adolescent
Adult
Aged
Blood Coagulation - drug effects
Child
Child, Preschool
Enzyme Replacement Therapy - methods
Female
Gaucher Disease - blood
Gaucher Disease - drug therapy
Gaucher's disease, bleeding, coagulation, platelet aggregation
Glucosylceramidase - therapeutic use
Hemorrhage - blood
Hemorrhage - etiology
Hemostasis - drug effects
Humans
Male
Middle Aged
Platelet Aggregation - drug effects
Platelet Count
Spleen - physiopathology
Young Adult
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Summary:There is a paucity of data on the effects of enzyme replacement therapy (ERT) on the coagulation abnormalities and platelet function of patients with Gaucher's disease (GDPs) and much of this data are controversial. This study investigates the haemostatic parameters in treatment-naïve GDPs and the effects of ERT. 31 Serbian treatment-naïve type 1 GDPs (M/F 17/14; median age 49 years, splenectomized 9/31) were studied. The complete blood count, prothrombin time (PT), activated partial tromboplastin time (aPTT) and coagulation factors were measured using the standard methods. Platelet aggregation was assessed with a whole-blood aggregometer. Splenic volumes were assessed using computer tomography. Twenty-one patients were treated with ERT (Imiglucerase). The haemostatic parameters were assessed after 6, 12 and 24 months (ERT6, 12, 24). Initially bleeding episodes were registered in 10/31 GDPs. Median platelet count was 108 × 109/L; 22/31 GDPs were thrombocytopenic. The PT and aPTT values were abnormal in 16/31 and 13/31 GDPs, respectively. Platelet aggregation abnormalities were recorded in 19/31GDPs. Median platelet aggregation was reduced in response to adenosine-diphosphate 5 µmol/L (ADP5 0.46) and collagen 5 µmol/L (Col5 0.47). Splenic volume inversely correlated with the platelet count and a reduced response to arachidonic acid (AA), Col5 and ADP5 (p 
ISSN:0953-7104
1369-1635
DOI:10.3109/09537104.2011.597526