TY - JOUR
T1 - Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years
AU - Hollak, Carla E. M.
AU - Belmatoug, Nadia
AU - Cole, J. Alexander
AU - vom Dahl, Stephan
AU - Deegan, Patrick B.
AU - Goldblatt, Jack
AU - Rosenbloom, Barry
AU - van Dussen, Laura
AU - Tylki-Szymańska, Anna
AU - Weinreb, Neal J.
AU - Zimran, Ari
AU - Cappellini, Maria Domenica
PY - 2012
Y1 - 2012
N2 - The characteristics of Gaucher disease (GD) associated with persistent thrombocytopenia despite imiglucerase enzyme therapy in type 1 GD (GD1) were investigated by retrospective analysis of International Collaborative Gaucher Group (ICGG) Registry data. The study involved 1016 GD1 patients with an intact spleen for whom date of diagnosis, therapy initiation, and platelet counts were known, and who received continuous imiglucerase therapy for 4 to 5 years. These patients were stratified by last platelet count: >= 120 x 10(9)/l (n = 772); >= 100 to <120 x 10(9)/l (n = 94); =80 to <100 x 10(9)/l (n = 80); and <80 x 10(9)/l (n = 70; 20 with <60 x 109/l) and characterized by initial and cumulative average imiglucerase dose, body mass index, platelet count, anaemia, hepatomegaly, splenomegaly, and skeletal assessments at baseline and after 45 years of therapy. Statistically significant associations were found between persistent thrombocytopenia and baseline platelet count ( <80 x 10(9)/l), splenomegaly, and anaemia (all P <0.0001). After 45 years, statistically significant associations were found with splenomegaly (P <0.0001), anaemia (P <0.0001), white blood cell count (P = 0.049), hepatomegaly (P = 0.004) and bone pain (P = 0.035). Exponential platelet decay in relation to splenomegaly suggests that platelets increase only when spleen volume decreases substantially
AB - The characteristics of Gaucher disease (GD) associated with persistent thrombocytopenia despite imiglucerase enzyme therapy in type 1 GD (GD1) were investigated by retrospective analysis of International Collaborative Gaucher Group (ICGG) Registry data. The study involved 1016 GD1 patients with an intact spleen for whom date of diagnosis, therapy initiation, and platelet counts were known, and who received continuous imiglucerase therapy for 4 to 5 years. These patients were stratified by last platelet count: >= 120 x 10(9)/l (n = 772); >= 100 to <120 x 10(9)/l (n = 94); =80 to <100 x 10(9)/l (n = 80); and <80 x 10(9)/l (n = 70; 20 with <60 x 109/l) and characterized by initial and cumulative average imiglucerase dose, body mass index, platelet count, anaemia, hepatomegaly, splenomegaly, and skeletal assessments at baseline and after 45 years of therapy. Statistically significant associations were found between persistent thrombocytopenia and baseline platelet count ( <80 x 10(9)/l), splenomegaly, and anaemia (all P <0.0001). After 45 years, statistically significant associations were found with splenomegaly (P <0.0001), anaemia (P <0.0001), white blood cell count (P = 0.049), hepatomegaly (P = 0.004) and bone pain (P = 0.035). Exponential platelet decay in relation to splenomegaly suggests that platelets increase only when spleen volume decreases substantially
U2 - https://doi.org/10.1111/j.1365-2141.2012.09175.x
DO - https://doi.org/10.1111/j.1365-2141.2012.09175.x
M3 - Article
C2 - 22640238
SN - 0007-1048
VL - 158
SP - 528
EP - 538
JO - British journal of haematology
JF - British journal of haematology
IS - 4
ER -