GTI 0101 (04/08/93) 
Page 16 of 35 
Upon completion of study treatment (marrow or PBSC), patients will continue to be 
followed monthly for up to 1 year, every six months for the next year. 
0 PATIENT SELECTION 
3.1 Inclusion Criteria 
Patients must meet the following criteria within 14 days prior to study entry (Day 0) 
unless otherwise noted: 
3.11 Men or women, age >_ 18 years of age with confirmed Gaucher disease 
3.12 Established diagnosis of Type I Gaucher disease as confirmed by: 
3.121 Glucocerebrosidase enzyme level determination, OR 
3.122 DNA sequence analysis 
3.13 Adequate baseline organ function as assessed by the following laboratory values 
within 30 days prior to study entry (day -30 to 0): 
o Adequate renal function with estimated creatinine clearance > 50 ml/min; 
which can be estimated by the following formula: 
CrCl male = (140-age in years x (body weight in kg) 
(SCr) x (72) 
CrCl female = 0.85 x (CrCl male) 
o Adequate liver function with SGOT, SGPT and alkaline phosphatase _< 
5 times the ULN [if transaminases > than the upper limit of normal 
(ULN), patients should have a hepatitis B surface antigen (HBsAG) test 
prior to study entry. Patients may not enter the study if HBsAG is 
positive.] 
o PT and PTT not more than 1.5 times the ULN 
o Hemoglobin >.9.0 g/dl (patients cannot be transfusion dependent) 
o Platelets J> 75,000/mm 3 
o WBC > 2,000/mm 3 
o Absolute Neutrophil Count (ANC) _> 1,000/mm 3 
o Serum Amylase <_ 1.5 times the ULN 
Recombinant DNA Research, Volume 17 
[801] 
