Advances in Hematology / 2013 / Article / Tab 1

Research Article

Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience

Table 1

Baseline values for surveyed parameters and after 4 years ( ), value between value of difference at four years from baseline, and percent of patients achieving goal for that parameter at four years (Student's -test and logistic regression with significance level of 5% were predetermined.). Therapeutic goals are as described by Pastores et al. (2004) [7] and as presented in Table 2.

Hemoglobin (gm/dL)Platelet counts
(×10/mm3)
Spleen (US) index volume ( )Liver (US) index volumeBone painNew bone crisesNew osteonecrosisLS BMDLS -score ( )FN BMDFN -score ( )Pediatric heightPuberty

Mean SD baseline 0.00
Mean SD 4 years
value 0.0000.0000.0000.0000.0000.0000.0000.0000.0000.0000.0000.0000.000
% at goal at 4 years81.7%68.9%60.0%53.1%59.9%93.8%92.6%NA69.9%NA70.6%100%48.8%

US: ultrasound; NA: not applicable.

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