Long‐term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

Abstract

ObjectiveWe studied the effect of long-term alglucerase/imiglucerase (Ceredase®/Cerezyme®, Genzyme, a Sanofi company, Cambridge, MA, USA) treatment on hematological, visceral, and bone manifestations of Gaucher disease type 1 (GD1).MethodsThe International Collaborative Gaucher Group (ICGG) Gaucher Registry identified GD1 patients treated with alglucerase/imiglucerase who had dose and clinical data at first infusion and after 10 years of follow-up. Data for hemoglobin, platelet count, organ volumes, bone pain, and bone crisis were analyzed. Tests of the null hypothesis (no change from first infusion to 10 years) were performed using t tests for within-patient absolute change in continuous measurements and McNemar/chi-square tests for change in distributions using categorical values. An alpha level of 0.05 designated statistical significance.ResultsAs of October 2011, 557 nonsplenectomized and 200 splenectomized patients met the inclusion criteria. The majority of GD1 patients had at least one N370S allele. Compared with nonsplenectomized patients at first infusion, splenectomized patients had lower percentages of anemia (26.0 % vs. 42.8 %) and thrombocytopenia (14.2 % vs. 76.3 %), similar percentages of moderate or severe hepatomegaly (81.2 % vs. 80.0 %), and higher percentages of bone pain (88.9 % vs. 52.4 %) and bone crises (38.3 % vs. 16.0 %). After 10 years, both groups showed significant (p < 0.05) improvements in mean hemoglobin levels, platelet count, liver, and spleen (nonsplenectomized) volumes, and bone crises. Initial dosing in both groups ranged from <15 U/kg to ≤90 U/kg every 2 weeks. After 10 years, the majority was receiving 15 to ≤45 U/kg every 2 weeks.ConclusionTen years of imiglucerase treatment results in sustainable improvements in all GD1 parameters.Electronic supplementary materialThe online version of this article (doi:10.1007/s10545-012-9528-4) contains supplementary material, which is available to authorized users.

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“…При развитии проявлений остеопороза для замедле-ния и прекращения потери костной массы, повышения ее прочности, предотвращения переломов костей в комплексной терапии применяют альфакальцидол ж,вк 8 (Код АТХ: А11СС03), соли кальция ж,вк (например, Код АТХ: А12АА).…”

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“…La literatura médica registra, precisamente, numerosos ensayos clínicos dirigidos a comprobar el beneficio de medicamentos de alto costo para la mayoría de las patologías seleccionadas en la ley 20.850 [4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20][21][22] .…”