• 2/10/2005
  • Rebekah Cin
  • Hem/Onc Today

Avoiding a painful complication of chemotherapy and radiotherapy be-fore bone marrow transplantation may be easier with an FDA approval of palifermin (Kepivance, Amgen).

The approval followed a placebo-controlled, double-blind, phase-3 study that found the IV drug reduced the risk of mucositis during pre-transplant regimens.

The study, which appeared in The New England Journal of Medicine, also found that palifermin shortened the duration of mucositis in patients who developed the adverse effect.

Mucositis development
High-dose chemotherapy and radiotherapy followed by hematopoietic stem-cell support is a well-established treatment for hematologic cancers, said Ricardo Spielberger, MD, of the City of Hope National Medical Center in Duarte, Calif. and lead researcher of the study.

Unfortunately, oral mucositis commonly develops. About 70% to 80% of patients undergoing high-dose chemotherapy, radiotherapy and hematopoietic stem-cell support experience the adverse effect.

Spielberger and his colleagues predicted that palifermin would mimic naturally occurring keratinocyte growth factor by stimulating cells on the surface layer of the mouth to grow. This would lead to fast replacement of the cells killed by the cancer treatments as well as accelerating the healing process of mouth ulcers.

Mucositis alleviated
Researchers enrolled 212 eligible patients with leukemia or lymphoma who received high doses of chemotherapy and radiation treatments associated with bone marrow transplantation. The patients were randomized to two treatment arms.

In one group, 106 patients received IV palifermin for three days before treatment. In the other group, 106 patients received a placebo in place of palifermin.

Ninety-eight percent of the patients who did not receive palifermin developed severe mucositis, compared with 63% of those who received the drug.

In patients who received palifermin, severe mucositis lasted an average of three days. Patients receiving a placebo experienced severe mucositis for an average of nine days.

The most common adverse effects of palifermin were skin rash; unusual sensations in the mouth, such as tingling; and increases in blood proteins suggesting pancreatic irritation. There were no reported serious adverse events.