A new study indicates that a short-course treatment with Kepivance (palifermin) prior to high-dose chemotherapy and stem cell transplantation may be sufficient to decrease mouth ulcers and the supportive care required by multiple myeloma patients after a stem cell transplant.

In multiple myeloma patients under the age of 65, high-dose chemotherapy followed by an autologous stem cell transplant is the preferred and most effective treatment option. However, the high dose of chemotherapy often causes severe side effects such as oral mucositis, which occurs when the mucous lining inside the mouth becomes inflamed and develops ulcers.

Oral mucositis can become severe enough to require hospitalization so patients can receive pain killers or nutrition intravenously. Patients who develop inflammation and ulceration in the digestive tract also have a higher risk of infection and ultimately, treatment-related death.

Kepivance is a drug that prevents and treats inflammation and ulceration in the mouth by stimulating the activity of cells that line and protect the mouth and digestive tract. It is normally administered for three consecutive days before and three consecutive days after stem cell transplantation.

In this retrospective study, researchers evaluated the effect of a short-course treatment of Kepivance prior to high-dose chemotherapy and stem cell transplantation.  No Kepivance was administered after transplantation.

Researchers analyzed the data of 67 multiple myeloma patients who received 60 µg/kg Kepivance for three consecutive days starting a week before their transplantation. Patients then received high-dose melphalan (Alkeran) – 56 patients were given 200 mg/m², while the 11 patients with impaired kidney function received 140 mg/m². After their transplants, patients were given granulocyte colony-stimulating factor (G-CSF) to promote the circulation of the infused stem cells.

Sixteen patients (24 percent) developed severe oral mucositis. Researchers found that the rate of severe oral mucositis was significantly lower in patients with normal kidney function (16 percent) than in patients with impaired kidney function (64 percent). In addition, patients with impaired kidney function experienced mouth ulcers longer (nine days) than patients with normal kidney function (seven days).

Side effects associated with Kepivance included taste distortion and mild to moderate skin reactions.

Researchers also compared the results of this study with results of two prior studies that involved 21 patients who had been treated with G-CSF and 21 patients treated with supportive care only.

They found that patients treated with short-course Kepivance had a shorter hospital stay (median of 18 days) than patients treated with just G-CSF (21 days) and those treated with neither (22 days). In addition, patients who received short-course Kepivance did not need any intravenous pain killers, as compared to patients treated with G-CSF who needed them for a median of four days and patients treated with supportive care only who needed them for a median of eight days.

Researchers concluded that a three-day treatment with Kepivance prior to high-dose chemotherapy and stem cell transplantation may suffice to reduce oral mucositis effectively in patients with normal kidney function. They suggest that the short-course treatment has the potential to become the standard of care.

They added that their results show that the short-course Kepivance treatment does not reduce the rate of severe oral mucositis in patients with impaired kidney function. However, they pointed to another recent study that recommends treating oral mucositis in patients with impaired kidney function aggressively by intensifying the Kepivance doses.

They recommend prospective studies to confirm their results.

For more information, please see the journal Annals of Oncology (abstract) and the clinical trial description.