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NCCN Melanoma Guidelines: New Drugs, Less Testing

March 23, 2012 (Hollywood, Florida) — Information on 2 new systemic therapies for metastatic disease and a reduced emphasis on testing for metastases in early-stage disease headline the changes to the melanoma guidelines from the National Comprehensive Cancer Network (NCCN).
The guidelines were discussed here at the NCCN 17th Annual Conference.
In his opening remarks to the NCCN audience, Daniel Coit, MD, from the Memorial Sloan-Kettering Cancer Center in New York City, highlighted the recently approved vemurafenib (Zelboraf, Plexxikon/Roche) and ipilimumab (Yervoy, Bristol-Myers Squibb), calling them "very exciting new agents for the treatment of systemic disease."
Last year during the NCCN annual meeting, ipilimumab was on the cusp of being reviewed by the US Food and Drug Administration (FDA). Upon its approval, it was integrated into the NCCN guidelines.
More recently, a footnote was added to the section in the guidelines on metastatic disease concerning the use of ipilimumab in patients who fail after an initial response to the therapy. "Reinduction with ipilimumab may be considered for select patients who experienced no significant toxicity during [previous] ipilimumab therapy and who relapse after initial clinical response or progress after stable disease [for more than] 3 months," reads the new guidance.
This guidance is not FDA indicated, Dr. Coit told Medscape Medical News at the meeting. However, the recommendation, which was unanimously supported by the melanoma panel, is based on observations from a clinical trial, he said.
John Thompson, MD, from the University of Washington and the Seattle Cancer Care Alliance, reviewed 2 registration trials of ipilimumab for the NCCN audience.
In the second-line therapy trial, patients receiving ipilimumab plus a peptide vaccine (glycoprotein 100) had a longer median survival than patients receiving the vaccine alone (10 vs 6.4 months; P < .001). In the first-line therapy trial, ipilimumab plus dacarbazine improved overall survival in patients with previously untreated metastatic melanoma, compared with dacarbazine plus placebo. Specifically, overall survival was significantly longer in the ipilimumab group than in the placebo group (11.2 vs 9.1 months; hazard ratio for death, 0.72; P < .001).
Responses to ipilimumab "may be delayed," said Dr. Thompson. Therefore, clinicians should select patients for treatment who will have "sufficient" time to develop a response.
According to Dr. Thompson, immune-related adverse events are a concern with the immunotherapy, and it is "essential" for clinicians to take the Risk Evaluation and Mitigation Strategies (REMS) program for the drug. Nursing staff also need to be trained in the adverse events. It is advisable for patients to carry a wallet card in the event that they are taken to a hospital that is not familiar with ipilimumab.
The toxic effects associated with ipilimumab can in "most cases" be treated satisfactorily, Dr. Thompson said.
The NCCN guidelines now include vemurafenib as an option for patients with advanced or metastatic melanoma. Vemurafenib is "recommended for patients with V600 mutation of the BRAF gene."
"We are stressing the importance of screening metastatic melanoma patients for the presence of this mutation," said Dr. Coit.
The guidelines also alert clinicians to adverse events associated with the targeted therapy. "Vemurafenib has the potential for significant dermatologic complications, including cutaneous squamous cell carcinoma and extreme photosensitivity. Regular dermatologic evaluation with referral to a dermatologist is clinically indicated. Patients should also be carefully monitored for the development of other adverse reactions, such as joint pain and swelling," according to the guidelines.
Dr. Thompson pointed out that vemurafenib provides a median survival of nearly 16 months, according to recently published data. He explained that this is "better than expected" in the pretreated population in that study.
Less Testing in Early-Stage Disease
Dr. Coit also reviewed the guidelines' recommendations for working-up patients with early-stage disease.
"We are working very hard to define subsets of patients at very low risk for either distant disease or nodal disease who should not undergo extensive staging procedures," he said.
In stage IB and IIA disease, routine imaging and lab tests are not recommended in the new guidance. For stage IIB and IIC disease, these are also not recommended, and chest x-ray is optional. In another bit of new guidance, sentinel lymph node biopsy is "generally not recommended" in these patients unless there are specific adverse features.
"We live in a culture of overtesting," summarized Dr. Coit about these changes, adding that the "yield is too low" to justify this work-up.
He reminded the audience that the value of sentinel lymph node biopsy in early-stage disease is limited to providing prognostic information and does not result in improved survival. "To date, sentinel lymph node biopsy is a staging procedure without any proven benefit on overall survival," said Dr. Coit.
Currently, there is an ongoing international phase 3 clinical trial, known as MSLT-II, that Dr. Coit believes is one of the most important trials in all of surgical oncology. It is designed to determine if, in patients with positive sentinel nodes (stage III), there is a survival benefit for completion lymph node dissection, compared with observation with nodal ultrasound.
Dr. Coit observed that one of the surprises in melanoma is that even when there is a positive lymph node, and thus stage III disease, the 5-year overall survival rate is about 80%, which is much higher than in other stage III cancers.
Dr. Thompson reports receiving research support from Bristol-Myers Squibb, GlaxoSmithKline, and Millennium.
National Comprehensive Cancer Network (NCCN) 17th Annual Conference. Presented March 15, 2012.

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