How it is administered
Clofarabine is given as an intravenous (IV) infusion. The recommended pediatric dose is 52 mg/m², administered over 2 hours daily for 5 consecutive days in a 28-day cycle. Treatment cycles are usually repeated every 2 to 6 weeks, depending on how the patient responds and recovers from side effects. The medication comes in a clear, colorless solution supplied in single-dose vials and is diluted before administration.
Supportive care, such as IV fluids and medications to prevent uric acid buildup, is often provided during treatment to reduce the risk of complications.
How it works
Clofarabine is a type of chemotherapy known as a purine nucleoside metabolic inhibitor. Once inside the body, it is converted into an active form that interferes with the DNA of cancer cells. Clofarabine works by blocking enzymes needed for DNA synthesis and repair, which are essential for cancer cells to grow and divide. It also disrupts the energy supply within the cancer cells' mitochondria, leading to cell death (apoptosis).
Because it targets rapidly dividing cells, clofarabine is especially effective against certain types of blood cancers, such as acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), particularly in cases where the disease has returned or not responded to other treatments.
Common side effects
- Vomiting
- Nausea
- Diarrhea
- Febrile neutropenia (fever with low white blood cell count)
- Pruritus (itching)
- Headache
- Bacteremia (bacteria in the blood)
- Fever (pyrexia)
- Rash
- Rapid heartbeat (tachycardia)
- Abdominal pain
- Chills
- Fatigue
- Loss of appetite (anorexia)
- Pain in extremities
- Low blood pressure (hypotension)
- Nosebleeds (epistaxis)
- Small red or purple spots on the skin (petechiae)
Other serious side effects can include myelosuppression (low blood counts), infections, liver and kidney problems, severe skin reactions, and bleeding.
Who Should take it
Clofarabine is indicated for pediatric patients aged 1 to 21 years who have relapsed or refractory acute lymphoblastic leukemia (ALL) after at least two previous treatment regimens. It may also be used in some cases of acute myeloid leukemia (AML), although its primary FDA-approved use is for ALL.
This medication is generally considered when other standard treatments have not worked or when the leukemia has come back. It is given under the supervision of a healthcare professional experienced in treating blood cancers.
Who should not take it
There are no absolute contraindications listed for clofarabine, but it should be used with caution in certain situations. Patients who have severe liver or kidney problems, active uncontrolled infections, or who are pregnant should discuss the risks and benefits with their healthcare provider.
Clofarabine can cause serious side effects, so it may not be suitable for patients who cannot tolerate intensive chemotherapy or who have significant organ dysfunction. It is not approved for use in patients over 21 years old or in those with other types of cancers outside of the approved indications.
Commonly used with
Clofarabine may be used alone or in combination with other chemotherapy drugs, especially in clinical trials or specialized treatment protocols. In some studies, it has been combined with medications like etoposide and cyclophosphamide, particularly in patients who have previously received stem cell transplants or have aggressive disease.
Supportive medications, such as antiemetics (to prevent nausea), antibiotics, and medications to manage uric acid levels, are also commonly used alongside clofarabine.
Commonly tested with
Clofarabine has been tested in combination with other chemotherapy agents, such as etoposide and cyclophosphamide, in clinical trials for relapsed or refractory acute leukemias. These combinations are often used to enhance the effectiveness of treatment, especially in patients who have not responded to standard therapies.
It is also tested with supportive care measures, including hydration and medications to prevent tumor lysis syndrome, to reduce the risk of treatment-related complications.