Liver-Directed Melphalan (Hepzato Kit)
Cancer specialists can treat many types of cancer, including metastatic uveal melanoma, with medications that kill fast-growing cells in your body called chemotherapies. While chemotherapies are not often used in the treatment of cutaneous melanoma, they are re-emerging as a potential tool in the treatment of uveal melanoma. Liver-directed melphalan (Hepzato Kit) can help patients with metastatic uveal melanoma live longer by shrinking melanoma tumors that have spread to the liver.
What Is Liver-Directed Melphalan (Hepzato Kit)
- Hepzato Kit is used to treat uveal melanoma that has metastasized to the liver that is not surgically removable.
- Hepzato Kit delivers melphalan directly to the liver through a novel delivery system in a hospital setting.
How Does Liver-Directed Melphalan (Hepzato Kit) Work?
Hepzato Kit is a novel therapy that delivers melphalan, a well-studied chemotherapy drug first approved in 1964, directly to the liver through a novel delivery system called the Hepatic Delivery System (HDS). This allows oncologists to treat metastatic uveal melanoma liver tumors with much higher doses of melphalan than would otherwise be possible. By administering the therapy only to the liver, systemic side effects are significantly reduced.
Hepzato Kit was approved based on results from Delcath's Phase 3 FOCUS clinical trial. In this single arm, multicenter study, 91 patients with metastatic uveal melanoma with unresectable (ineligible for surgery) liver metastases received Hepzato Kit every 6 to 8 weeks, for up to 6 treatments. Together, these patients had an objective response rate (ORR) – meaning the percentage of patients that had a significant reduction in size or disappearance of their tumor – of 36.3% and a median duration of response (DoR) of 14 months. The Disease Control Rate (DCR) – meaning the percentage of patients whose tumors either shrink or remain stable – among the patient cohort was 73.6% and included 7 complete responses (7.7%) and 26 partial response (28.6%).1
The study enrolled patients with metastatic uveal melanoma with metastases predominately involving the liver (liver dominant). Patients with limited metastases beyond the liver, such as in the bone, subcutaneous sites, lymph nodes, or lung were permitted if the life-threatening component of the uveal melanoma was in the liver and the tumors outside the liver had a defined treatment plan. Fifty six percent of patients in the cohort had not received any previous systemic treatment for their melanoma.
Which Patients May Benefit from Liver-Directed Melphalan (Hepzato Kit)?
Patients with Metastatic Uveal Melanoma that has Metastasized to the Liver:
In 2023, the U.S. Food and Drug Administration (FDA) approved liver-directed melphalan (Hepzato Kit) for the treatment of:
- Patients with metastatic uveal melanoma with unresectable (ineligible for surgery) liver metastases affecting less than 50% of the liver with no extrahepatic (outside of the liver) metastases, and
- Patients with metastatic uveal melanoma with unresectable (ineligible for surgery) liver metastases affecting less than 50% of the liver with limited metastases beyond the liver, such as in the bone, subcutaneous sites, lymph nodes, or lung that is amenable to treatment with surgery or radiation.
Speak to your doctor to see if you are eligible to be treated with liver-directed melphalan (Hepzato Kit).
How Is Liver-Directed Melphalan (Hepzato Kit) Given for the Treatment of Metastatic Uveal Melanoma?
Patients receive liver-directed melphalan (Hepzato Kit) directly into the hepatic artery (the main source of blood to the liver) through a minimally invasive procedure known as percutaneous hepatic perfusion (PHP). During this procedure, the liver is temporarily isolated from the body’s circulatory system during which melphalan is infused directly to the liver over a 30-minute period. Upon exiting the liver, the patient’s blood is filtered before returning it to systemic circulation.
Treatments are administered in a hospital setting at select sites every six to eight weeks.
What Are the Goals of Liver-Directed Melphalan (Hepzato Kit) for the Treatment of Metastatic Uveal Melanoma?
The goals of using liver-directed melphalan (Hepzato Kit) include:
- Shrinking melanoma tumors that have spread to the liver and cannot be surgically removed, thereby preserving organ function
- Treating symptoms of metastatic melanoma
- Helping patients live longer
All melanoma treatments, including liver-directed melphalan (Hepzato Kit), have side effects, which can sometimes be serious. Patients should talk with their physician to learn more about the side effects of liver-directed melphalan (Hepzato Kit) and other melanoma treatment options.
What Should I Ask My Doctor About Liver-Directed Melphalan (Hepzato Kit) for the Treatment of Metastatic Uveal Melanoma?
Not all treatments work for all patients, because everyone is different. If you are interested in learning more about liver-directed melphalan (Hepzato Kit), here are some questions you should ask your care team:
- Am I eligible for liver-directed melphalan (Hepzato Kit)?
- What is your experience with liver-directed melphalan (Hepzato Kit)?
- Is liver-directed melphalan (Hepzato Kit)a good option for my melanoma treatment?
- Is there an alternative therapy for me?
- How successful has liver-directed melphalan (Hepzato Kit) been for patients like me?
- What are the side effects of liver-directed melphalan (Hepzato Kit)?
- How should I report any side effects or changes that I experience while undergoing therapy with liver-directed melphalan (Hepzato Kit)?
- Are there any clinical trials that I should consider?
- What other treatments are FDA-approved for treating advanced uveal melanoma?
- What are the risks and benefits of each of my available treatment options?
- What are the goals for my treatment?