Cytarabine is a chemotherapy agent used to treat certain types of cancer. The therapy is available as a generic from many companies. It is approved by the U.S. Food and Drug Administration to treat acute nonlymphocytic leukemia. Cytarabine may also be used to treat myeloma in combination with other medications.

How does cytarabine work?

Myeloma is a type of cancer that affects blood cells. It starts in the bone marrow and often spreads from one bone to another. As with many cancers, myeloma cells divide and spread rapidly. In order to divide, the cancerous cells need to make a copies of their genomes rapidly.

Cytarabine contains a small molecule that is metabolized in the cell to resemble a nucleotide, a building block of DNA. The active form of the therapy is incorporated into the DNA, where it blocks the synthesis of new DNA, halting cell division. Blocking cell division stops cancer from spreading, and makes cancerous cells more vulnerable to other chemotherapy agents.

Cytarabine in clinical trials for myeloma

Cytarabine is rarely used as a sole chemotherapy agent to treat myeloma.

A study published in the European Journal of Cancer and Clinical Oncology evaluated the efficacy of cytarabine treatment in refractory multiple myeloma. Out of 14 patients enrolled in the study, only one achieved partial remission. The treatment led to myelosuppression (the loss of activity in the bone marrow) and two patients died from infection and bleeding as a result. The researchers concluded that cytarabine had high toxicity and poor activity against myeloma.

In another study published in the British Journal of Haematology 42 myeloma patients were treated with cytarabine in combination with three other treatments (etoposide, methylprednisolone, and cisplatin). The combination was called ESHAP. Of 21 patients who had failed a previous treatment of vincristine, Adriamycin, and dexamethasone (VAD), 67% responded to ESHAP treatment. Kidney function decreased in 13 of the 42 patients following ESHAP treatment, but none required dialysis. The overall survival reported for all patients was 62% at four years after ESHAP treatment. The authors concluded that ESHAP had acceptable toxicity and effectively killed chemotherapy-resistant cancer cells in these patients.

Cytarabine can also be used to increase the number of circulating stem cells for autologous hematopoietic stem cell transplantation (ASCT) to treat myeloma. In ASCT, a person’s own stem cells are used to replace the cancerous bone marrow. In order to get enough healthy cells to transplant, patients are treated with a growth factor called G-CSF (granulocyte-colony stimulating factor) that causes stem cells to expand. The combination of G-CSF and cytarabine has been shown to significantly increase the number of circulating stem cells in myeloma patients in comparison to other treatments.

Other information

Cytarabine can cause side effects such as fatigue, appetite loss, bruising, abdominal pain, and inflammation around the heart (pericarditis). In women, cytarabine can cause menstruation to halt.

In rare cases, patients may experience a condition called cytarabine syndrome, which is characterized by a combination of symptoms such as fever, muscle and bone pain, rash, sore eyes, and extreme weakness.


Last updated: Feb. 13, 2020


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