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What do I need to know about chronic myeloid leukemia (CML) and ICLUSIG® (ponatinib)?

On this page you will find a brief overview of CML and how it is treated.

What is CML?

CML starts with an abnormal change (or mutation) in a cell’s DNA. This mutation occurs in an immature blood cell and creates the “Philadelphia” or Ph chromosome. The Ph chromosome causes the cell to make a protein called BCR-ABL1. This protein then causes the bone marrow to start making abnormal white blood cells. These are CML cells, and they crowd healthy white blood cells out of your bone marrow to cause leukemia.
CML has 3 phases: chronic phase (CP), accelerated phase (AP), blast phase (BP). Most people with CML are diagnosed in CP. An important goal of CP-CML treatment is to prevent progression to AP and BP.

Your healthcare provider is your primary source of information.
Always consult with your healthcare provider if you have questions about your disease.

What are my treatment goals?

Your healthcare provider will look for specific responses in your disease at different times in your treatment. These milestones may help your healthcare provider better understand if your treatment is working. Your healthcare provider will tell you which specific responses (often referred to as “milestones”) they are looking for and how you may be monitored while you are on treatment.

These response milestones show decreasing levels of CML in the body

Early Molecular Response (EMR)

BCR-ABL1 levels are 10% or less

Complete Cytogenetic Response (CCyR)

Absence of Ph chromosome
(or typically BCR-ABL1 levels are 1% to 0.1%)

Major Molecular Response (MMR)

BCR-ABL1 levels are less than 0.1%

Other milestone terms you may hear about

  • Complete hematologic response (CHR): Blood cell counts are normal, and no abnormal blood cells are detected.

  • Complete molecular response (CMR): BCR-ABL1 cannot be detected.

  • Major cytogenetic response (MCyR): When 0-35% of tested cells have the Ph chromosome.

Why am I starting a new therapy?

Some people will need to change their tyrosine kinase inhibitor (TKI) treatment over time. There are 2 main reasons why a person may need to change TKI therapy—resistance and intolerance. Resistance and intolerance can appear at any time.

Resistance: When a TKI does not work well for a person’s CML, or it stops working over time.

Intolerance: When a TKI causes side effects that result in a healthcare provider recommending a patient not continue taking it.

TKIs can stop the growth of leukemic cells by blocking the abnormal BCR-ABL1 protein. Many people with CML respond well to the first TKI they try and may stay on that treatment for years.

Monitoring can help healthcare providers determine if you are resistant to other treatments

Regular monitoring can help your healthcare provider find early signs of resistance. Your healthcare provider will use different tests to monitor your disease levels and check how your CML is responding to treatment.

Response monitoring tests

Molecular monitoring is sometimes called QPCR. It is a test that can measure small amounts of BCR-ABL1 in the blood.

  • Molecular monitoring is recommended every 3 to 6 months throughout treatment
  • Many laboratories use an International Scale to make sure measurements are the same from lab to lab

Cytogenetic testing measures the number of cells in your bone marrow that contain the Philadelphia chromosome.

  • Your healthcare provider may do this test before you start treatment
  • This test may be repeated if your healthcare provider thinks you may be resistant to treatment

How do mutations affect treatment options?

Part of the difficulty of treating any type of cancer, including CML, is that cancer cells can change (or mutate) over time. In CML, mutations can appear in BCR-ABL1 during TKI treatment. This can cause you to stop responding to the TKI.

If you develop a new mutation while on a TKI, your healthcare provider may want to change your therapy to a different TKI that may work better against the new mutation.

T315I is a specific type of mutation that can occur in CML. ICLUSIG is currently the only TKI that works in people with CML who have the T315I mutation.

Please consult with your healthcare provider if you want to learn more about mutations, monitoring, and treatment.

Other milestone terms you may hear about

  • Complete hematologic response (CHR): Blood cell counts are normal, and no abnormal blood cells are detected.

  • Complete molecular response (CMR): BCR-ABL1 cannot be detected.

  • Major cytogenetic response (MCyR): When 0-35% of tested cells have the Ph chromosome.

What has the study of ICLUSIG shown in CML?

ICLUSIG was studied in a large clinical trial of more than 400 people, most of whom had been treated with at least 2 TKIs. It included mostly people with CML.

The clinical trial included 267 people with CP-CML. The goal of this trial was for these patients to have a major cytogenetic response (MCyR) by 12 months, which includes patients having a complete cytogenetic response (CCyR) and patients having a partial cytogenetic response.

ICLUSIG was found to be effective in resistant
or intolerant CP-CML

The response rates below show the effectiveness of ICLUSIG, but they should always be balanced against the side effects that were experienced. You should discuss this information with your healthcare provider.

  • Talk to your healthcare provider about these results and others, including major molecular response (MMR).
  • Most people who had a response and stayed on treatment still maintained a response 4 years after starting ICLUSIG.

Talk to your healthcare provider about these ICLUSIG results. Your personal experience may be different.

ICLUSIG is the only TKI indicated for people with the
T315I mutation

The T315I mutation in CML may affect treatment decisions. See some of the results from the ICLUSIG trial.

  • 64 people with CP-CML in the ICLUSIG trial had the T315I mutation. This has been linked to resistance to other TKIs.
  • Many of these patients were able to achieve MCyR by 12 months with ICLUSIG.
  • Responses lasted for at least 4 years in most patients who stayed on treatment.

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Glossary

Accelerated phase CML (AP-CML):
The second phase of CML progression, when the number of blast cells is increased.
BCR-ABL1:
An abnormal protein that is made by the BCR-ABL1 fusion gene and causes too many abnormal white blood cells (leukemia cells) to be made.
BCR-ABL1 mutations:
Changes to the BCR-ABL1 protein that prevent certain TKIs from working.
Blast cell:
Abnormal, immature blood cell.
Blast phase CML (BP-CML):
The third and final phase of CML progression, which has the highest number of blast cells in the blood and bone marrow and can be life-threatening.
Chemotherapy:
Medicines that kill fast-growing cells, including cancer cells and normal cells.
Chronic myeloid leukemia (CML):
The first phase of CML, when there are more white blood cells than normal but may not cause symptoms.
Chronic phase CML (CP-CML):
The first phase of CML, when there are more white blood cells than normal but may not cause symptoms.
Clinical trial:
Research on a test or treatment to assess its safety or how well it works.
Complete cytogenetic response (CCyR):
Treatment response when no Ph chromosomes are seen in a bone marrow sample.
Complete hematologic response (CHR):
Treatment response when blood cell counts decrease to normal and no young abnormal blood cells are seen in the blood.
Complete molecular response (CMR):
Treatment response when BCR-ABL1 cannot be detected in the blood.
Complete remission:
When no leukemia cells are found in the blood or bone marrow and all signs and symptoms of the cancer are gone.
Corticosteroids:
Medicines used to reduce redness, swelling, and pain, but also to kill leukemia cells; also called steroids.
Cytogenetic testing:
A test used to look for changes in chromosomes (the part of the cell that contains genetic information).
Deoxyribonucleic acid (DNA):
The genetic information carried by cells.
Early molecular response (EMR):
Treatment response when BCR-ABL1 levels decrease to ≤10% within 3 to 6 months of starting treatment.
International Scale (IS):
A standardized scale for measuring and reporting results of a very sensitive test that measures the number of cells that have the BCR-ABL1 gene.
Intolerance:
When treatment with a drug must be stopped because of side effects.
Major cytogenetic response (MCyR):
Treatment response when about one-third (35% or less) of cells have the Ph chromosome.
Major hematologic response (MaHR):
Treatment response that is either a complete hematologic response or another type of hematologic response called no evidence of leukemia (NEL).
Major molecular response (MMR):
Treatment response when BCR-ABL1 levels decrease to <0.1% by 18 months.
Minimal residual disease (MRD):
A very small amount of cancer cells left in the body after treatment.
Molecular monitoring:
A very sensitive test that can measure very small amounts of BCR-ABL1 in the blood; sometimes referred to as QPCR.
Mutation:
Abnormal change in a cell's genetic material (DNA).
Mutation testing:
Tests used to see if samples of deoxyribonucleic acid (DNA; the genetic information carried by cells) have changed (mutated).
Partial cytogenetic response (PCyR):
reatment response when 1% to 35% of bone marrow cells still have the Ph chromosome.
Philadelphia (Ph) chromosome:
An abnormal chromosome that forms when pieces of chromosomes 9 and 22 switch places with each other. This forms a longer chromosome 9 and a shorter chromosome 22. The shorter chromosome 22 contains the BCR-ABL1 gene and is known as the Philadelphia chromosome.
Quantitative reverse transcriptase polymerase chain reaction (QPCR):
A very sensitive test that measures the number of cells in the blood or bone marrow that have BCR-ABL1.
QT prolongation:
Electrical signals that help the heart pump are slowed, causing the heart to pump inefficiently.
Resistance (or resistant):
When cancer cells do not respond to a treatment.
Stem cell transplant:
Treatment that replaces damaged or diseased cells in the bone marrow—soft tissue in the center of bones where blood cells are made—with healthy blood-forming cells called blood stem cells.
T315I:
A type of BCR-ABL1 mutation that causes cancer cells to not respond to certain TKIs.
Tyrosine kinase inhibitor (TKI):
A type of medicine that stops the growth of leukemic cells by blocking BCR-ABL1.

The information provided on this site is intended for informational purposes and cannot replace individualized guidance from a healthcare provider. Be sure to talk with your healthcare provider about making any change to your healthcare regimen. The information on this site is intended for residents of the United States only. Product labeling may be different in other countries.