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What Is Ponatinib Used For?

If you have certain types of leukemia, you may receive ponatinib, a chemotherapy drug used to help slow down or stop the production of abnormal blood cells. Ponatinib is approved for use in people who are no longer responding to or cannot tolerate other leukemia medications. This drug is licensed for use in adults only.


An Overview of Uses for Ponatinib

Ponatinib (Iclusig®) is a prescription medication approved to treat people who have certain types of leukemia. It belongs to a group of medicines known as tyrosine kinase inhibitors. Specifically, ponatinib is approved to treat the following types of leukemia:
  • Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL)
  • Chronic myeloid leukemia (CML).
Leukemia is a cancer that starts in the blood-forming tissue, such as the bone marrow, and causes large numbers of abnormal blood cells to be produced. Over time, the abnormal blood cells (also called leukemia cells) build up in the blood and bone marrow and crowd out normal, healthy white blood cells, red blood cells, and platelets. This can cause serious problems, such as infections, anemia, and bleeding.
There are several different types of leukemia. In general, they are named by how quickly the disease develops and gets worse (acute versus chronic) and the type of blood cell affected (lymphoid versus myeloid). Acute leukemia progresses rapidly, while chronic leukemia advances more slowly.
Chronic myeloid leukemia (also known as chronic myelogenous leukemia) is a slowly progressing type of leukemia that mainly affects older adults. In CML, the bone marrow makes too many of a type of white blood cell known as granulocytes (see Types of Leukemia for information about the other types).
Acute lymphoblastic leukemia is a type of leukemia in which a large amount of abnormal lymphoblasts (immature lymphocytes) are made by the bone marrow. These abnormal lymphoblasts do not function properly and cannot fight infection well. ALL is the most common type of leukemia in children. It is also sometimes referred to as acute lymphocytic leukemia.
Most people with CML and some people with ALL have an abnormal chromosome known as the Philadelphia chromosome (abbreviated as Ph chromosome). Chromosomes are threadlike structures found in cells that contain DNA. The Philadelphia chromosome occurs when a piece of one chromosome breaks off and trades places with a piece that has broken off from another chromosome. The Ph chromosome causes the bone marrow to make an enzyme known as tyrosine kinase. Tyrosine kinase, in turn, causes the production of large amounts of abnormal granulocytes.
Leukemia generally progresses through three phases, or stages. These stages include:
  • Chronic phase
  • Accelerated phase
  • Blast phase.
In the chronic phase, symptoms are usually mild or not present. As the number of leukemia cells increases, the disease moves into the accelerated phase, and eventually the blast phase. In the blast phase, leukemia is much more aggressive and symptoms may be significant.
Ponatinib is approved to treat adults with CML in any phase or Ph-chromosome-positive ALL. Because of the very high risk of blood clots or other serious problems with this medication, its use has been restricted only to people with T315I-positive CML or ALL, or for those who cannot take other tyrosine kinase leukemia medications.
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Ponatinib Drug Information

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