MEDICAL RECORD 
CONTINUATION SHEET for either: 
NIH 2514-1, Consent to Participate In A Clinical Research Study 
NIH 2514-2, Minor Patient’s Assent to Participate In A Clinical Research Study 
STUDY NUMBER: CONTINUATION: page _JL of _L pages. 
Pre-Study Testing: Prior to being entered into this study you will 
undergo blood tests, bone marrow aspiration and biopsy. X-ray and 
other imaging studies, and physical examination to determine the 
status of your Gaucher disease and of your liver, kidneys and bone 
marrow. You will have 2-4 tablespoons of blood drawn. You will sign 
a separate consent form for the bone marrow aspiration and biopsy, 
which will be done with local anesthesia and has minimal risks, 
including pain, bleeding and infection at the biopsy site. 
Collection of Peripheral Blood Stem Cells: 
G-CSF Administration : To increase the number of immature stem cells 
in the blood, the drug G-CSF (granulocyte, colony-stimulating 
factor) will be administered either as an outpatient in the 
hematology clinic or as an inpatient on the hematology ward at the 
Clinical Center. This drug stimulates the bone marrow to release 
stem cells into the blood, where they can be more easily collected. 
G-CSF will be given as a daily injection under the skin for 7 days. 
G-CSF may cause fever, flu-like symptoms, muscle aches, bone pain, 
rash, redness at the injection site, decrease in platelet count, 
and temporary worsening of skin conditions such as psoriasis and 
eczema. You will be treated for any of these symptoms if they 
occur. These symptoms disappear immediately after discontinuation 
of the drug. It is also possible that G-CSF could temporarily 
worsen the bone symptoms caused by your Gaucher disease. If there 
is any evidence for these problems, the G-CSF would be stopped 
immediately and you would be taken off protocol. 
Central Line Placement : On the 4rth or 5th day after starting G- 
CSF , you will have a special intravenous line called a "central 
line" inserted into a large vein in your groin, neck^ or chest. 
This will be done using local anesthesia by a line insertion 
specialist. You will sign a separate consent for this procedure. 
This large intravenous line is necessary for collection of stem 
cells from your blood. You may have mild pain and bruising at the 
insertion site. Rarely, infections or more serious bleeding 
develop at the insertion site, which can be treated by removal of 
the line, antibiotics, or pressure to the site. This line will be 
removed on day 7, after completion of stem cell collection. 
Apheresis : On days 5, 6 and 7 after beginning G-CSF, you will 
undergo a procedure called "apheresis" to collect the stem cells in 
you blood. You will be hooked up to an apheresis machine via one 
lumen (opening) of your central line, and blood will slowly flow 
through the machine for 1-3 hours. The machine will separate out 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
[ 824 ] 
NIH-2514-1 (10-84) 
N 'J^UM6fWant DNA Research, Voluftfcff 1 ^ 9 
