Leukemia and lymphoma patients who need a bone marrow transplant now have a drug to help them cope with one of the treatment's most common side effects: mouth sores (called oral mucositis). Most patients develop these mouth sores, which are often painful enough to need treatment with intravenous narcotics.

The US Food and Drug Administration has approved palifermin (sold as Kepivance), after a study showed it cut the severity of mouth sores and helped them heal faster. The study was published in the New England Journal of Medicine (Vol. 351, No. 25: 2590-2598).

The drug is the first to be approved to treat this painful side effect of the high-dose chemotherapy and radiation needed before a bone marrow transplant.

"Until now… the most we could do for our patients was to give them ice chips and narcotics to try and manage their pain," said Patrick Stiff, MD, in a statement. He was one of the study's lead investigators and is director of the Cardinal Bernardin Cancer Center at Loyola University Health System.

Stiff and his colleagues studied 212 people with leukemia, Hodgkin disease, non-Hodgkin lymphoma, or multiple myeloma who were having a bone marrow or stem cell transplant. Half the patients received palifermin, and half were given a placebo.

Nearly all the patients in the study developed some degree mouth sores. But severe mouth sores were less common and healed more quickly in the people who received palifermin. Just 68% of those patients developed grade 3 sores (where the patient can only swallow liquid) or grade 4 sores (where the patient can't eat at all and must be fed through a vein). By contrast, 98% of the people on placebo reported grade 3 or 4 sores. The sores lasted about 6 days in the patients on palifermin, and 9 days in those on placebo.

The people who took palifermin also had less pain than people on placebo; they used less narcotic pain medication and didn't need to use it for as long a time. And, fewer palifermin patients needed vein feeding.

Side effects of the drug included rash, itching, skin tingling and redness, taste changes, and tongue thickness. These side effects were not severe.

The research was funded by Amgen, the drug's maker.

Palifermin is a type of drug known as a keratinocyte growth factor. It is a form of a human protein. The drug helps the cells lining the mouth grow and multiply. This action counteracts the damage to the lining of the mouth caused by the high-dose chemotherapy and radiation given before a bone marrow transplant.

Palifermin is given directly into a vein. Patients get the drug on each of the 3 days just before high-dose chemotherapy and radiation, and for 3 more days right after the bone marrow transplant.

So far, palifermin is only approved for use in patients with leukemia, myeloma or lymphoma who receive high doses of chemotherapy and radiation therapy followed by stem cell rescue. Amgen said about 11,000 US adults with blood cancers receive bone marrow transplants each year.

Palifermin is being tested in patients with other types of cancer.