NYU Langone doctors often recommend a stem cell transplant for people with primary myelofibrosis. It is the only treatment that has the potential to cure this disorder. The procedure is usually an option for people younger than age 70 who are otherwise healthy. At NYU Langone, the procedure is performed at the Rita J. and Stanley H. Kaplan Stem Cell and Bone Marrow Transplant Center.
Stem cells are immature cells that are made inside the bone marrow. They mature into many different kinds of blood cells, including red blood cells, which carry oxygen and other nutrients to the body’s tissues and organs; white blood cells, which help fight infection; and platelets, which help with blood clotting.
In stem cell transplantation for myelofibrosis, a doctor delivers a high dose of a chemotherapy drug. This medication, given through a vein with intravenous (IV) infusion for a period of a few days, destroys rapidly multiplying cells throughout the body. This treatment is called conditioning. It destroys the bone marrow, including the swiftly dividing, immature blood-producing stem cells responsible for myelofibrosis.
After conditioning, the bone marrow is replaced with healthy stem cells provided by a donor. This is called allogeneic stem cell transplantation. You are given the stem cells through a vein with IV infusion in the hospital. After being infused, the stem cells travel through the bloodstream and enter the bone marrow, replacing the destroyed stem cells. Then, they mature into healthy red blood cells, white blood cells, and platelets.
Often, doctors may be able to use lower doses of chemotherapy for stem cell transplantation. These doses may not completely destroy the bone marrow, but the transplanted healthy stem cells from a donor may help your body mount an immune attack against myelofibrosis.
Finding a Stem Cell Donor
An allogeneic stem cell transplant is a donation of stem cells from a healthy person who is a “match.” This means the person’s genetic makeup and tissue types are compatible with yours. Siblings are usually ideal matches.
If necessary, an NYU Langone doctor can help locate an unrelated match or matches using national bone marrow registries. It can take several weeks or months to find the appropriate donor or donors.
Stem Cell Collection and Transplantation
Stem cells are typically collected from the matching donor using a procedure called peripheral blood stem cell harvest. A doctor collects blood from the donor through a catheter, or hollow tube, that’s inserted into a vein in the arm. Peripheral stem cell collection takes place either in an NYU Langone infusion center or at the hospital. The donor can go home that same day.
Rarely, doctors collect stem cells through bone marrow retrieval surgery, in which a needle is inserted into the donor’s hip bone to remove the cells. More than one insertion is required at times, and local anesthesia is used. The procedure takes place in the hospital or at a special apheresis unit, and the donor can usually go home the same day.
After conditioning, the collected stem cells are infused into the recipient.
Recovery from Stem Cell Transplantation
Because high doses of chemotherapy given before stem cell transplantation cause a drop in the levels of white and red blood cells and platelets, the risk of infection and bleeding increases in the days after the procedure. As a result, you stay in a hospital isolation room, and doctors and specialists monitor you closely. Lower doses of chemotherapy may result in a lower likelihood of these side effects, but staying in an isolation room is still necessary.
Regardless of the chemotherapy dose, your doctor prescribes IV antibiotics to prevent or manage infections. He or she also gives medications to prevent graft-versus-host disease, which occurs when the donated cells attack healthy tissues in the body. Lower doses of chemotherapy can result in a reduced risk of developing graft-versus-host-disease.
You may also need red blood cell and platelet transfusions until the donor’s stem cells start multiplying in your body. You can expect to stay in the hospital for about three to four weeks, while the stem cells start producing new blood cells.
You may return home when your blood cell levels return to normal and your doctor determines you are healthy enough.
Full recovery from a stem cell transplant can take several months. During this time, you have frequent follow-up appointments, and doctors perform blood tests to determine the success of the transplant.
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