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What to Expect From CML Treatment: An Overview

Many good CML treatments can help control your disease so you can lead a normal life. To find the best treatment for you, you'll work closely with a specialist called a hematologist-oncologist, a doctor with special training in blood diseases such as cancer.

The goal in treating CML is to destroy cells that contain the BCR-ABL gene, which leads to too many abnormal white blood cells.

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About This PDQ Summary

Purpose of This Summary This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of childhood non-Hodgkin lymphoma. It is intended as a resource to inform and assist clinicians who care for cancer patients. It does not provide formal guidelines or recommendations for making health care decisions. Reviewers and Updates This summary is reviewed regularly and updated as necessary by the PDQ Pediatric...

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Targeted Drugs: The First Step

Your CML treatment will probably start with a type of drug called a tyrosine kinase inhibitor (TKI). TKIs block tyrosine kinase, the protein made by the BCR-ABL gene that leads to too many abnormal blood cells. Your doctor will likely prescribe a TKI such as bosutinib (Bosulif), dasatinib (Sprycel), imatinib (Gleevec), nilotinib (Tasigna), or ponatinib (Iclusig).

Most people respond to these drugs quickly. Your doctor should know within 3 to 6 months if your treatment is working.

If your CML progresses after being treated with two or more other TKIs, your doctor may switch you to omacetaxine mepesuccinate (Synribo). It helps stop the growth of cancerous cells. It’s given by injection under the skin. 

You may go into remission while you're taking a TKI. That means that the abnormal gene is no longer in your cells. It doesn't mean that you're cured -- just that your CML is under control. 

Always tell your doctor about any side effects or new symptoms.

TKI side effects include:

  • Nausea and vomiting
  • Diarrhea
  • Rash
  • Headache
  • Fatigue
  • Lower blood cell counts

Is Treatment Working?

Among the treatment goals your doctors will seek:

  • Normal blood cell counts with no abnormal white blood cells -- called a complete hematologic response.
  • No blood or bone marrow cells containing the Philadelphia chromosome, which creates the BCR-ABL gene. This is called a complete cytogenetic response.
  • No sign of BCR-ABL in your blood -- also called a complete molecular response.

Regular Testing

While you're taking TKIs, you’ll have regular blood tests, including:

  • A complete blood count to check white blood cells, red blood cells, and platelets
  • A blood cell exam to determine the percentage of abnormal blood cells
  • Cytogenetic analysis to check for the abnormal Philadelphia chromosome
  • Polymerase chain reaction (PCR) test to look for the BCR-ABL gene

During the first 3 months, you'll likely have a complete round of blood tests every 2 weeks.

At 3 months, you’ll get a follow-up bone marrow study. After the third month, you'll have blood and bone marrow tests at least once every 6 months until you have a complete cytogenetic response.

Once you have a complete cytogenetic and molecular response, you'll get a PCR test every 3-6 months and cytogenetic testing once every year.

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