Reaching Your Chronic Myeloid Leukemia (CML) Treatment Milestones
What is CML?
CML is a type of cancer that starts in certain blood-forming cells of the bone marrow and invades the blood.
In CML, your bone marrow produces too many immature, abnormal white blood cells (WBCs). These abnormal WBCs crowd out healthy blood cells
Common signs and symptoms
Many CML symptoms occur because CML cells crowd out the bone marrow's healthy red blood cells, white blood cells, and platelets
Lack of red blood cells
Weakness
Fatigue
Shortness of breath during basic everyday activities
Lack of normal white blood cells
Increased risk of infection
Lack of platelets
Excessive bruising or bleeding
Additional symptoms may include: Fever, bone pain, unexplained weight loss, night sweats, pain or a feeling of fullness below the ribs on the left side due to an enlarged spleen.
Please note that this is not a complete list of signs and symptoms of CML.
Philadelphia chromosome positive (Ph+) CML is caused by the Philadelphia Chromosome
CML is caused by a single, specific abnormal gene known as BCR::ABL1
The BCR::ABL1 gene is found on an abnormal chromosome called the Philadelphia chromosome
The BCR::ABL1 gene causes the bone marrow to make too many abnormal white blood cells, also known as 'leukemia cells' or 'CML cells'
Is CML treatable?
CML is highly treatable and may be curable in some patients. It is important to discuss your treatment goals and make a plan for your treatment with your doctor.
How is CML treated?
CML is typically treated with a type of medication called a tyrosine kinase inhibitor (TKI). TKIs are “targeted therapies”, which mean they attack cancer cells while causing less damage to normal cells compared with traditional chemotherapy treatments. In CML, TKIs work by targeting the abnormal BCR::ABL1 protein that causes uncontrolled cell growth, causing the cells to die.
Measuring how well your treatment is working
After you start your TKI treatment, your doctors will monitor how well your treatment is working. In general, the better your response to drug therapy, the longer your CML will be controlled.
The treatment goal is to hit milestones within a certain amount of time and maintain those milestones.
Treatment goals vary by patient. Not every patient will reach these treatment milestones. Talk to your doctor to discuss your treatment goals.
Key milestones in your
TKI treatment journey
3-month and 6-month Milestones:
Early Molecular Response (EMR)
10 or fewer out of every 100 cells have the BCR::ABL1 gene
12-month Milestone
Complete Cytogenic Response (CCyR)
1 or fewer out of every 100 cells have the BCR::ABL1 gene
Additional Treatment
Response Measurements
Major Molecular Response (MMR)
Less than 1 out of every 1000 cells has the BCR::ABL1 gene
Deep Molecular Response (DMR or MR4.5)
1 or fewer out of every 32,000 cells has the BCR::ABL1 gene (undetectable)
A patient who is responding well and meeting these treatment milestones can stay on the same TKI, while patients who have not hit their milestones might switch to another TKI. Patients who are able to stay on their first TKI usually have better outcomes.
One of the most important things you can do to help reach your milestones is to take your TKI as prescribed, without missing doses
Up Next:
Treatment-free Remission
IMPORTANT SAFETY INFORMATION
Danziten™ (nilotinib) tablets, for oral use
DANZITEN is a prescription medicine used to treat:
- adults who have been newly diagnosed with a certain type of leukemia called Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase.
- adults with chronic phase Ph+ CML or accelerated phase Ph+ CML who:
- are no longer benefiting from other treatments, including imatinib (Gleevec), or
- have taken other treatments, including imatinib (Gleevec), and cannot tolerate them.
WARNING: QT PROLONGATION and SUDDEN DEATHS
See Full Prescribing Information for complete Boxed Warning.
- Nilotinib prolongs the QT interval. Prior to DANZITEN administration and periodically, monitor for hypokalemia or hypomagnesemia and correct deficiencies. (5.3) Obtain ECGs to monitor the QTc at baseline, seven days after initiation, and periodically thereafter, and following any dose adjustments. (5.3, 5.4, 5.8, 5.12)
- Sudden deaths have been reported in patients receiving nilotinib. (5.4) Do not administer DANZITEN to patients with hypokalemia, hypomagnesemia, or long QT syndrome. (4, 5.3)
- Avoid use of concomitant drugs known to prolong the QT interval and strong CYP3A4 inhibitors. (7.1, 7.2)
ADDITIONAL IMPORTANT SAFETY INFORMATION
Do not take DANZITEN if you have:- low levels of potassium or magnesium in your blood
- long QTc syndrome
DANZITEN can cause serious side effects, including:
Low blood cell counts: Low blood cell counts (red blood cells, white blood cells, and platelets) are common with DANZITEN, but can also be severe. Your healthcare provider will check your blood counts regularly during treatment with DANZITEN. Call your healthcare provider or get medical help right away if you develop any signs or symptoms of low blood counts, including fever, unexplained bleeding or bruising, shortness of breath, chills or other signs of infection, and unexplained weakness.
Decreased blood flow to the leg, heart, or brain: People who have recently been diagnosed with Ph+ CML and take DANZITEN may develop decreased blood flow to the leg, the heart, or brain. Get medical help right away if you suddenly develop any of the following symptoms, including chest pain or discomfort, problems walking or speaking, your leg feels cold, numbness or weakness, leg pain, and change in the skin color of your leg.
Pancreas inflammation (pancreatitis): Tell your healthcare provider right away if you develop any symptoms of pancreatitis, including sudden stomach area pain with nausea and vomiting.
Liver problems: DANZITEN can increase your risk of liver problems. People who have had liver problems in the past may be at risk for getting liver problems with DANZITEN. Call your healthcare provider or get medical help right away if you develop any symptoms of liver problems, including stomach area (abdominal pain), yellow skin and eyes, and dark-colored urine.
Tumor Lysis Syndrome (TLS): TLS is caused by a fast breakdown of cancer cells. Your healthcare provider may do blood tests to check you for TLS. TLS can cause you to have kidney failure and the need for dialysis treatment or an abnormal heartbeat.
Bleeding problems: Serious bleeding problems and death have happened during treatment with DANZITEN. Tell your healthcare provider right away if you develop any signs and symptoms of bleeding during treatment with DANZITEN.
Fluid retention: Your body may hold too much fluid (fluid retention): Symptoms of fluid retention include shortness of breath, rapid weight gain, and swelling.
Abnormal growth or development in children: Effects on growth and development have happened in children with chronic phase Ph+ CML during treatment with nilotinib. Some children and adolescents may have slower than normal growth during treatment with nilotinib.
The most common side effects of DANZITEN include:- nausea
- rash
- headache
- tiredness
- vomiting
- diarrhea
- cough
- constipation
- muscle and joint pain
- runny or stuffy nose, sneezing, sore throat
- night sweats
If you and your healthcare provider decide that you can stop taking DANZITEN and try treatment free remission (TFR), you may have more muscle and bone (musculoskeletal) symptoms than before you stopped treatment. Symptoms may include:
- muscle pain
- arm and leg pain
- joint pain
- bone pain
- spine pain
These are not all the possible side effects of DANZITEN. Please see Full Prescribing Information for a full list.
Before taking DANZITEN, tell your doctor about all your medical conditions, including:- have heart problems
- have had a stroke or other problems due to decreased blood flow to the brain
- have irregular heartbeat
- have QTc prolongation or a family history of it
- have liver problems
- have had pancreatitis
- have low blood levels of potassium or magnesium in your blood
- have bleeding problems
- had a surgical procedure involving the removal of the entire stomach (total gastrectomy)
- are pregnant or plan to become pregnant. Nilotinib can harm your unborn baby. Tell your healthcare provider right away if you are pregnant, or if you become pregnant during treatment with DANZITEN.
- Your healthcare provider should do a pregnancy test before you start treatment with DANZITEN.
- Use effective birth control (contraception) during treatment with DANZITEN and for 14 days after the last dose
- are breastfeeding or plan to breastfeed. It is not known if Nilotinib passes into your breast milk. Do not breastfeed during treatment and for 14 days after your last dose of DANZITEN.
If you need to take antacids (medicines to treat heartburn) do not take them at the same time that you take DANZITEN. If you take:
- a medicine to block the amount of acid produced in the stomach (H2 blocker): Take these medicines about 10 hours before you take DANZITEN, or about 2 hours after you take DANZITEN.
- an antacid that contains aluminum hydroxide, magnesium hydroxide, and simethicone to reduce the amount of acid in the stomach: Take these medicines about 2 hours before or about 2 hours after you take DANZITEN.
The Important Safety Information does not include all the information needed to use DANZITEN safely and effectively. Please see Full Prescribing Information for DANZITEN.
To Report SUSPECTED ADVERSE REACTIONS, contact Azurity Pharmaceuticals, Inc. at 1-800-461-7449, or FDA at 1-800-FDA-1088 or www.fda.gov/MedWatch.