The process of blood stem cell transplantation involves total body irradiation (TBI) and high-dose Cytoxan. The goal is to destroy the immune cells that are thought to be causing scleroderma and to supply your body with selected cells to reset your immune system. While previous studies of stem cell transplantation are encouraging, this procedure is investigational. It has never been directly compared to other treatments for scleroderma until this study.
Before Your Stem Cell Transplant
In order to avoid experiencing numerous needle and intravenous (IV) sticks, a central venous catheter will be inserted under local anesthesia. The catheter will be tunneled under the skin on your chest and then advanced from a large vein near your neck until the tip (opening) of the catheter is in a vein just above the heart. This catheter will remain in place throughout your transplant. You will be taught how to care for your central venous catheter.
Stem Cell Mobilization
Injections of blood stimulants called growth colony stimulating factor (G-CSF) will be given to stimulate your bone marrow to make and release stem cells into your bloodstream.
Leukapheresis
You will be connected to a pheresis machine via your central venous catheter. As the blood circulates through the machine, your blood stem cells will be collected and stored for future use. The remainder of your blood will be returned to your body through the catheter. In total, about 20 to 40 pints of blood will be removed and returned. Between 1 and 5 leukapheresis collections will be needed, and each collection will take about 4 hours.
CD34 Selection
Your blood cell samples will undergo a process where specific types of stem cells that express a protein called CD34 are selected. Cells thought to be associated with activating scleroderma will be discarded.
Cryopreservation
The cells are stored in a freezer until the time of your transplant.
Conditioning
In order to prepare your body for the stem cell transplant, you will be admitted to the hospital and given total body irradiation (TBI), a chemotherapy drug called Cytoxan, and ATGAM.
- TBI will be given twice daily for 2 days in a row. Lung and kidney shielding will be done to reduce the risk of damage to these organs.
- Cytoxan will be given through your central venous catheter for 2 consecutive days.
- You will also be given ATGAM, if you are not allergic to it. Doses of ATGAM will be given through your vein before and shortly after the transplant.
- In addition to medications given to you as part of your standard medical care, you will be given corticosteroids and an ACE inhibitor.
Stem Cell Transplantation
On the day of transplant, your blood stem cells will be removed from the freezer and thawed. The cells will be infused through your central venous catheter over about 30 minutes.
After Your Stem Cell Transplant
You likely will be hospitalized until your white blood cell levels recover. This may take approximately 14–21 days after transplantation. During this time, you will be given antibiotics to protect against infection, and you will continue to receive ATGAM and corticosteroids. Also, you will receive at least 1 blood product transfusion. After hospital discharge, you will need to stay close to the transplant center for another 1–2 months after your transplant for follow-up visits. Your central venous catheter will be removed when the process is complete.
Risks and Side Effects
Serious side effects have been associated with autologous stem cell transplantation. Possible risks and side effects are listed on this web site; however, all interested participants should carefully review the SCOT research consent form for further detail.
