What Is Busulfan Used For?

How Does This Medicine Work?

Busulfan is part of a group of medications called alkylating agents. In general, alkylating agents work by causing strands of DNA to bond to each other and become linked (this is known as "cross-linking"). The linked strands cannot uncoil and separate, which is necessary for the DNA to replicate. Because DNA replication is essential for cells to grow and multiply, alkylating agents like busulfan prevent cell growth and multiplication, and may cause cell death.

Can Children Use It?

Children can use busulfan. Dosing of busulfan based on weight is the same for children and adults. Juvenile chronic myelogenous leukemia (also called juvenile myelomonocytic leukemia), which is not associated with the Philadelphia chromosome, does not generally respond to treatment with busulfan. Talk with your child's healthcare provider about the particular benefits and risks of using busulfan in children.

Is It Safe for Older Adults to Use Busulfan?

There were not enough older adults in busulfan clinical trials to determine whether they would respond any differently to busulfan than younger age groups. This medication can be used in older adults. However, older adults may be more sensitive to the side effects of busulfan, and may need to be started on lower doses and monitored more closely.

What About Off-Label Uses?

On occasion, your healthcare provider may recommend this medicine for something other than the uses discussed in this article. This is called an "off-label" use. Busulfan may be prescribed off-label for the following uses:
  • Treatment of polycythemia vera, a bone marrow disease associated with abnormally high levels of red blood cells
  • Treatment of thrombocytosis (abnormally high platelet counts)
  • Treatment of myelofibrosis (a disease of the bone marrow)
  • Treatment of ovarian cancer
  • Prior to bone marrow transplantation (the injectable form of busulfan, Busulfex®, is approved for this use).
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