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What do I need to know about Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) and ICLUSIG® (ponatinib)?

The following is an overview of Ph+ ALL and how it is treated.

What is Ph+ ALL?

Ph+ ALL (or “Philadelphia chromosome-positive ALL”) is a fast-growing cancer of the blood that starts in young white blood cells (lymphoblasts or simply, blast cells). In people with Ph+ ALL, an abnormal change in a cell’s DNA (mutation) creates the “Philadelphia” (or Ph) chromosome. The Ph chromosome causes the body to produce the BCR-ABL1 protein. This protein causes the bone marrow to produce too many lymphoblasts, leading to leukemia.

How is Ph+ ALL treated?

Treatment usually consists of chemotherapy, corticosteroids, and a tyrosine kinase inhibitor (TKI). Many people with Ph+ ALL at some point may need a stem cell transplant. Your healthcare provider will find the treatment that is right for you.

What should I know about how Ph+ ALL treatment goals
are monitored?

If you have Ph+ ALL, your healthcare provider will track how your disease is responding to treatment on a schedule depending on the regimen she or he has you on, your age, and overall health.

  • The goal of treatment is complete remission, which means that your blood counts are normal, your disease symptoms are gone, and there is no evidence of disease in your bone marrow.
    • Even if you have complete remission, there may still be a small number of leukemia cells left behind called minimal residual disease (MRD) that can only be detected with sensitive tests.
  • Your healthcare provider may monitor the levels of BCR-ABL1 in your blood.
  • If your Ph+ ALL does not go into complete remission after the first phase of treatment, your healthcare provider may order mutation testing because new BCR-ABL1 mutations can appear during treatment.
  • If a new mutation is found, your doctor may consider a change in TKI therapy.

Always consult with your healthcare provider if you need more information about Ph+ ALL treatment goals.

Why am I starting a new therapy?

Ph+ ALL is often treated with a combination of chemotherapy, steroids, and tyrosine kinase inhibitor (TKI) therapy.
Throughout your treatment, your healthcare provider will monitor how well your disease is responding. If complete remission is not achieved on a certain treatment plan, or if you once achieved complete remission and your disease starts to progress again, your healthcare provider may consider changing parts of your treatment plan, such as your TKI therapy.
No response or a loss of response may be a sign that your disease is resistant to your current treatment plan, and your healthcare provider may make changes to help avoid a relapse.

Monitoring can help healthcare providers determine if you are resistant to treatment

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 see how you are responding to treatment.

Response monitoring test

Cytogenetic testing helps measure the number of cells in your bone marrow or blood that contains the Philadelphia chromosome. Your healthcare provider may perform this test before you start treatment.

  • Cytogenetic testing may be repeated during treatment
  • If your tests show complete remission, your healthcare provider may continue to look for minimal residual disease (MRD)
  • Cytogenetic tests can help determine if the Ph+ ALL has become resistant to your current treatment

Molecular testing helps measure small amounts of BCR-ABL1 in your bone marrow or blood. One type of molecular test is PCR, and it can be used to evaluate MRD.

  • Molecular tests can help determine if your disease is responding to treatment
  • If molecular tests show signs of MRD, your healthcare provider may modify your treatment program
  • If your disease has stopped responding to treatment, molecular tests can help determine if there is a BCR-ABL1 mutation that is affecting your TKI therapy

How do mutations affect treatment options?

Part of the difficulty of treating any type of cancer, including Ph+ ALL, is that cancer cells can change (or mutate) over time. In Ph+ ALL, 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 Ph+ ALL. ICLUSIG is currently the only TKI that is indicated for people with Ph+ ALL who have the T315I mutation.

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

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What has the study of ICLUSIG shown in Ph+ ALL?

The clinical trial included 32 people with Ph+ ALL who were resistant or intolerant to other TKI treatments. The study looked at the safety and effectiveness of ICLUSIG in Ph+ ALL.

The effects of ICLUSIG were evaluated based on the following types of responses:

Complete Hematologic Response (CHR)

Blood counts improve and no young abnormal blood cells can be detected.

Major Hematologic Response (MaHR)

A combination of CHR and another type of hematologic response called no evidence of leukemia.

ICLUSIG was found to be effective in Ph+ ALL

The response rates below show the effectiveness of ICLUSIG. Your personal experience may be different. Talk to your healthcare provider about these results and any questions about potential side effects of ICLUSIG.

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

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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.