Overview | Schedule | Side Effects | Monitoring | Tips | Patient Assistance | Emotional Wellness | References
Treatment Name: Asciminib (Scemblix®)
How does Asciminib (Scemblix®) work?
Asciminib is designed to stop cancerous white blood cells from growing and dividing. It may also permit normal, non-cancerous blood cells (such as red blood cells and platelets) in the bone marrow to grow normally.
More specifically, Asciminib binds to a protein in the cancer cell known as BCR-ABL1. Unlike other medications for CML, it binds to a unique site on this protein called the myristoyl site, which locks the protein into an inactive state. With the protein locked, it prevents more cancer cells from growing and dividing. Clinicians may refer to Asciminib as an “allosteric inhibitor”, simply meaning that it binds to the cancer protein in a unique, alternative way compared to conventional tyrosine kinase inhibitor (TKI) therapies such as imatinib, bosutinib, dasatinib, nilotinib, or ponatinib. Importantly, it targets both native and mutated BCR-ABL1, including the gatekeeper mutation known as T315I.
Asciminib is also known as its study drug name: ABL001.
Goals of Asciminib (Scemblix®) therapy:
Asciminib is taken to reduce symptoms from CML or prevent them from ever occurring, but is not commonly given with the goal of cure.
Schedule
How is Asciminib (Scemblix®) therapy for CML taken?
Asciminib may be offered to patients who have taken at least two different TKIs (such as imatinib, bosutinib, dasatinib, nilotinib, or ponatinib) and had either relapsed or refractory disease, or had unacceptable side effects from two prior TKIs.
The recommended dose of Asciminib (Scemblix®) for patients with Ph+ CMLin chronic phase is:
- Asciminib 80 mg by mouth ONCE daily at approximately the same time each day
OR
- Asciminib 40 mg by mouth TWICE daily at approximately 12-hour intervals.
- Asciminib (Scemblix®) should be taken by mouth without food, but may be taken with water. Avoid eating for at least 2 hours before and 1 hour after taking asciminib
Importantly, patients with Ph+ CML in chronic phase that also have the T315I gatekeeper mutation may require higher doses than those without this mutation:
- 200 mg taken by mouth TWICE daily at approximately 12-hour intervals
- Asciminib (Scemblix®) should be taken by mouth without food, but may be taken with water. Avoid eating for at least 2 hours before and 1 hour after taking asciminib
Asciminib is a pill that is taken at home. Some patients may require hospitalization when initially diagnosed and begin treatment before leaving the hospital. Typical duration of therapy is usually life-long.
It is important that you do not miss doses, unless your doctor specifically says it is okay. Missing doses may prevent you from achieving a response and increase the risk of asciminib no longer working.
Note: Individual doses may vary based upon your Doctor's recommendation, or drug availability
Side Effects
What are the most common side effects of Asciminib (Scemblix®) when taken by patients for CML?
In clinical studies, the most commonly reported Asciminib side effects are shown here:
- Fatigue (29%)
- Headache (28%)
- Increase in serum lipase (27%)
- Joint pain (24%)
- Nausea (24%)
- Diarrhea (23%)
- Rash (23%)
- Low platelet count (22%)
- Vomiting (21%)
- High blood pressure (19%)
- Upper respiratory tract infection (18%)
- Abdominal (belly) pain (17%)
- Pain in arm or leg (16%)
- Itching (16%)
- Back pain (15%)
- Constipation (14%)
- Fever (1%)
- Dizziness (13%)
- Cough (13%)
- Shortness of breath (13%)
- Muscle pain (13%)
- Low red blood cells [anemia] (11%)
- High triglycerides (11%)
- Sinus infection (11%)
- Low white blood cell count [neutropenia] (11%)
- Bone pain (10%)
- Swelling in extremities [peripheral edema] (10%)
- Trouble sleeping (10%)
- Increased sweating (9%)
- Low phosphorous in blood (9%)
- Noncardiac chest pain (9%)
- High blood sugar (9%)
- Decreased appetite (8%)
- Depression (8%)
- Dry eye (8%)
- Elevated uric acid levels (8%)
- Musculoskeletal pain (8%)
- Blurry vision (8%)
- Anxiety (7%)
- Dry skin (7%)
- Muscle spasms (7%)
- Mouth pain (7%)
- Weight gain (7%)
- Heartburn (6%)
- Low blood potassium levels (6%)
- Pleural effusion (6%)
- Urinary tract infection (5%)
On average, 9% (6 out of 68) of patients discontinue treatment due to unacceptable side effects
Importantly, not all people who experience a side effect from asciminib will experience it in the same way. It may be mild in some or severe in others, depending upon the individual. Everybody is different. Additionally, side effects may vary over time. For some, side effects may be a reason to delay or switch treatment, reduce the dose, or avoid treatment with a certain medication altogether.
Side effects may be treatable when they occur or preventable by taking certain medications before they happen. When medications are taken to prevent a problem, this is known as prophylaxis, or "prophy" for short.
After starting treatment with asciminib be sure to come back and watch all of the side effect videos shown below. Each of these videos contain valuable information about side effect management that will hopefully help you to both feel better and stay out of the hospital.
Watch videos on common Asciminib (Scemblix®) side effects below
Monitoring
How often is monitoring needed with Asciminib (Scemblix®) treatment?
Labs (blood tests) may be checked before treatment, once weekly during the first month, then every other week for the first three months, then monthly until stable. Labs at diagnosis often include: Complete Blood Count (CBC), Comprehensive Metabolic Panel (CMP), Lactate Dehydrogenase (LDH), magnesium, phosphorus, and uric acid, plus any others your doctor may order. Tumor lysis syndrome is usually not seen after the first two weeks of therapy. Additionally, your doctor will order a test known as BCR-ABL1. Your doctor uses the BCR-ABL1% to monitor your response to Asciminib.
How do I know if I am responding to Asciminib (Scemblix®)?
It is important for you to know what your BCR-ABL1% is. This way, if it begins to increase, you will know and can look for reasons why.
In the clinical trial, bone marrow samples were taken to assess response in patients with BCR-ABL1 transcript levels of more than 1% on the International Scale (IS) or for those who lost complete hematologic response, and every 3 months in patients without
a complete cytogenetic response, and as clinically indicated in all other patients.
How often is imaging needed with Asciminib (Scemblix®) therapy?
Imaging may be checked before treatment, and periodically thereafter. Imaging may include: a chest X-ray, or CT scan of the lungs. These are most often done to look for fluid in the lungs or an infection causing pneumonia.
How might blood test results/imaging affect treatment with Asciminib (Scemblix®) therapy?
Depending upon the results, your doctor may advise to interrupt asciminib therapy for a short period of time (several days), decrease the dose if the normal blood cells are too low, or switch therapy if goals of therapy are not being met.
In a small subset of patients who develop two mutations within the same BCR-ABL1 allele (compound mutations), one of which being the T315I mutation, the combination of ponatinib plus asciminib may result in a response whereas the use of either agent alone may not.
Questions to Ask Your...
A better understanding of your treatments will allow you to ask more questions of your healthcare team. We then hope that with the answers, you will get better results and have greater satisfaction with your care. Because we know it's not always easy to know what questions to ask, we've tried to make it easy for you!
Choose any healthcare provider below to see common questions that you may want to ask of this person. Then, either print each list to bring to your clinic visits, or copy the questions and send them as a message to your healthcare team through your electronic medical record.
ChemoExperts Tips
What are the most important things to know about Asciminib (Scemblix®) while receiving therapy?
- Tell your doctor if you develop abdominal pain or a change in bowel movements, including loose stools. This may be a sign that your pancreas is inflamed, a condition known as pancreatitis, as a result of taking Asciminib. Doctors will monitor your blood for the development of pancreatitis using a lab called the serum lipase. Importantly, patients with an elevated or high serum lipase level. May not develop signs or symptoms of pancreatitis. If it does develop, All cases in the clinical trial resolved within 5 - 10 days after Asciminib discontinuation or dosage interruption, and one patient was able to restart Asciminib at a lower dose.
- Common adverse events included fatigue, headache, joint pain, high blood pressure, and low platelet count.
- Although the development of mutations and resistance to asciminib is relatively rare (occurring in 4 of 86 in the phase III clinical trial), it is possible. Therefore, it is important to not miss any doses, unless directed to do so by your doctor.
- Bring all of your pill bottles to every clinic visit! This will make it easier for your healthcare team to make sure Asciminib can be taken safely with all other medications you are taking. A pharmacist should ALWAYS review your medication list to ensure that drug interactions are prevented or managed appropriately
- Clinical trials may exist for CML. Ask your doctor if any studies are currently enrolling in your area. If not, go to clinicaltrials.gov to search for other centers offering study medications
Patient Assistance & Co-payment Coverage
Patients under the age of 65 years, or those with private insurance plans:
If you have insurance and are looking for patient assistance or copay assistance for Asciminib (Scemblix®), we have provided links that may help.
Visit our Patient Assistance page and click the links to various patient assistance programs for help paying for Asciminib (Scemblix®). Depending upon your income, they may be able to help cover the cost of:
For Branded medications (may be available for generic medications too), check with the manufacturer to determine if a co-pay card is offered and if it could reduce your monthly copay.
- If you are uninsured, check with the manufacturer to determine if you are eligible to receive medication at no cost.
Medicare and Medicaid patients (Patients 65 years or older):
The clinic providing treatment will likely pre-authorize medications and immune therapies such as Asciminib (Scemblix®) and are the best source to help you understand drug cost.
- Ask to speak with a patient assistance technician or financial counselor at the clinic or hospital administering this therapy.
Emotional Wellness
What is Emotional Wellness?
Emotional wellness is having a positive outlook balanced with a realistic understanding of current life events. This requires both an awareness and acceptance of your emotions. It is with this knowledge that you can develop a plan to take the necessary actions to positively impact your life.
Emotional wellness uses an ongoing process to continually reflect on the stressors of life in a constructive manner to move forward and create happiness.
Because emotional wellness is deeply connected with physical, social, and spiritual wellness, pursuing it often becomes particularly difficult in times of major illness. Despite this difficulty, working toward emotional wellness has been connected to improved treatment outcomes and a higher likelihood of achieving goals of therapy.
Learn more about pursuing emotional wellness while receiving treatment with Asciminib (Scemblix®)
