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– For patients with nonchallenging cases of basal cell carcinoma, surgery remains the gold standard of treatment, but imiquimod is another feasible option, according to Ervin H. Epstein, MD.

Doug Brunk/MDedge News
Dr. Ervin Epstein

In a noninferiority trial, researchers in the Netherlands randomized 601 patients with superficial BCCs to one of three medical treatments: two treatments of photodynamic therapy (PDT) 1 week apart (group 1), imiquimod 5 days per week for 6 weeks with no occlusion (group 2), and 5-fluorouracil (5-FU) twice per day for 4 weeks with no occlusion (group 3). They followed the patients for 5 years to see which tumors came back (J Invest Dermatol. 2018 Mar;138[3]:527-33). At 5 years, tumor-free survival was 63% in group 1, 81% in group 2, and 70% in group 3. Based on this analysis the authors concluded that imiquimod is the first choice for noninvasive treatment of most primary BCCs.

“Yes, imiquimod is helpful, but it’s not as good as surgery,” Dr. Epstein said at the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

Researchers like Dr. Epstein, a dermatologist at Brown & Toland Physicians in Oakland, Calif., have been exploring ways to reduce the recurrence of BCCs in those at high risk. One approach is to stay out of the sun, but that is not practical for most people, “and the data are not very supportive,” he said. “Very few people can actually avoid sunlight. What about topical 5-FU? Suppose you put on 5-FU and then study people for the next year, to see what the likelihood is of them getting a BCC? Why it has taken us so long to do this kind of study is in some ways shameful.” He mentioned one study in which patients at risk of superficial BCC recurrence applied topical 5-FU twice a day for 2-4 weeks. The regimen led to an 11% decrease in the development of BCCs, a trend that did not reach statistical significance.



Another approach to warding off BCC recurrence is to take oral hedgehog pathway inhibitors, which are highly effective. “The problem is, hedgehog pathway inhibitors have side effects that are not catastrophic, but they’re annoying,” Dr. Epstein said. “Patients can lose their hair, they get muscle cramps, and can lose their taste.”

To date, there have been at least three trials evaluating the feasibility of a topical hedgehog pathway inhibitor in adult subjects. In one of the trials, researchers evaluated the efficacy of LDE225, a selective antagonist of Smoothened, in eight patients with nevoid basal cell carcinoma syndrome. They were instructed to apply LDE225 twice per day for 4 weeks (J Invest Dermatol 2011 Aug;131[8]:1735-44). Of 13 BCCs treated in the patients, 12 had a partial to complete response. In fact, eight BCCs had an average of 56% volume reduction but none had complete histologic clearing.

At PellePharm, a biotechnology that Dr. Epstein cofounded, researchers are developing patidegib topical gel, 2%, for the reduction of disease burden of persistently developing BCCs in subjects with Gorlin syndrome. In a phase 3 trial, which just completed recruitment, adults are instructed to apply the gel twice daily to the face for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new surgically eligible BCCs that develop over the 12-month period. “We want to see if we can reduce the number of surgically eligible BCCs,” Dr. Epstein said. “We’re very hopeful.”

SDEF and this news organization are owned by the same parent company. Dr. Epstein spoke during a forum on cutaneous malignancies at the meeting.

Dr. Epstein disclosed that he has been a consultant for Genentech, Novartis, Amgen, and Infinity. He is also cofounder/director and stockholder in PellePharm.

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– For patients with nonchallenging cases of basal cell carcinoma, surgery remains the gold standard of treatment, but imiquimod is another feasible option, according to Ervin H. Epstein, MD.

Doug Brunk/MDedge News
Dr. Ervin Epstein

In a noninferiority trial, researchers in the Netherlands randomized 601 patients with superficial BCCs to one of three medical treatments: two treatments of photodynamic therapy (PDT) 1 week apart (group 1), imiquimod 5 days per week for 6 weeks with no occlusion (group 2), and 5-fluorouracil (5-FU) twice per day for 4 weeks with no occlusion (group 3). They followed the patients for 5 years to see which tumors came back (J Invest Dermatol. 2018 Mar;138[3]:527-33). At 5 years, tumor-free survival was 63% in group 1, 81% in group 2, and 70% in group 3. Based on this analysis the authors concluded that imiquimod is the first choice for noninvasive treatment of most primary BCCs.

“Yes, imiquimod is helpful, but it’s not as good as surgery,” Dr. Epstein said at the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

Researchers like Dr. Epstein, a dermatologist at Brown & Toland Physicians in Oakland, Calif., have been exploring ways to reduce the recurrence of BCCs in those at high risk. One approach is to stay out of the sun, but that is not practical for most people, “and the data are not very supportive,” he said. “Very few people can actually avoid sunlight. What about topical 5-FU? Suppose you put on 5-FU and then study people for the next year, to see what the likelihood is of them getting a BCC? Why it has taken us so long to do this kind of study is in some ways shameful.” He mentioned one study in which patients at risk of superficial BCC recurrence applied topical 5-FU twice a day for 2-4 weeks. The regimen led to an 11% decrease in the development of BCCs, a trend that did not reach statistical significance.



Another approach to warding off BCC recurrence is to take oral hedgehog pathway inhibitors, which are highly effective. “The problem is, hedgehog pathway inhibitors have side effects that are not catastrophic, but they’re annoying,” Dr. Epstein said. “Patients can lose their hair, they get muscle cramps, and can lose their taste.”

To date, there have been at least three trials evaluating the feasibility of a topical hedgehog pathway inhibitor in adult subjects. In one of the trials, researchers evaluated the efficacy of LDE225, a selective antagonist of Smoothened, in eight patients with nevoid basal cell carcinoma syndrome. They were instructed to apply LDE225 twice per day for 4 weeks (J Invest Dermatol 2011 Aug;131[8]:1735-44). Of 13 BCCs treated in the patients, 12 had a partial to complete response. In fact, eight BCCs had an average of 56% volume reduction but none had complete histologic clearing.

At PellePharm, a biotechnology that Dr. Epstein cofounded, researchers are developing patidegib topical gel, 2%, for the reduction of disease burden of persistently developing BCCs in subjects with Gorlin syndrome. In a phase 3 trial, which just completed recruitment, adults are instructed to apply the gel twice daily to the face for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new surgically eligible BCCs that develop over the 12-month period. “We want to see if we can reduce the number of surgically eligible BCCs,” Dr. Epstein said. “We’re very hopeful.”

SDEF and this news organization are owned by the same parent company. Dr. Epstein spoke during a forum on cutaneous malignancies at the meeting.

Dr. Epstein disclosed that he has been a consultant for Genentech, Novartis, Amgen, and Infinity. He is also cofounder/director and stockholder in PellePharm.

– For patients with nonchallenging cases of basal cell carcinoma, surgery remains the gold standard of treatment, but imiquimod is another feasible option, according to Ervin H. Epstein, MD.

Doug Brunk/MDedge News
Dr. Ervin Epstein

In a noninferiority trial, researchers in the Netherlands randomized 601 patients with superficial BCCs to one of three medical treatments: two treatments of photodynamic therapy (PDT) 1 week apart (group 1), imiquimod 5 days per week for 6 weeks with no occlusion (group 2), and 5-fluorouracil (5-FU) twice per day for 4 weeks with no occlusion (group 3). They followed the patients for 5 years to see which tumors came back (J Invest Dermatol. 2018 Mar;138[3]:527-33). At 5 years, tumor-free survival was 63% in group 1, 81% in group 2, and 70% in group 3. Based on this analysis the authors concluded that imiquimod is the first choice for noninvasive treatment of most primary BCCs.

“Yes, imiquimod is helpful, but it’s not as good as surgery,” Dr. Epstein said at the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

Researchers like Dr. Epstein, a dermatologist at Brown & Toland Physicians in Oakland, Calif., have been exploring ways to reduce the recurrence of BCCs in those at high risk. One approach is to stay out of the sun, but that is not practical for most people, “and the data are not very supportive,” he said. “Very few people can actually avoid sunlight. What about topical 5-FU? Suppose you put on 5-FU and then study people for the next year, to see what the likelihood is of them getting a BCC? Why it has taken us so long to do this kind of study is in some ways shameful.” He mentioned one study in which patients at risk of superficial BCC recurrence applied topical 5-FU twice a day for 2-4 weeks. The regimen led to an 11% decrease in the development of BCCs, a trend that did not reach statistical significance.



Another approach to warding off BCC recurrence is to take oral hedgehog pathway inhibitors, which are highly effective. “The problem is, hedgehog pathway inhibitors have side effects that are not catastrophic, but they’re annoying,” Dr. Epstein said. “Patients can lose their hair, they get muscle cramps, and can lose their taste.”

To date, there have been at least three trials evaluating the feasibility of a topical hedgehog pathway inhibitor in adult subjects. In one of the trials, researchers evaluated the efficacy of LDE225, a selective antagonist of Smoothened, in eight patients with nevoid basal cell carcinoma syndrome. They were instructed to apply LDE225 twice per day for 4 weeks (J Invest Dermatol 2011 Aug;131[8]:1735-44). Of 13 BCCs treated in the patients, 12 had a partial to complete response. In fact, eight BCCs had an average of 56% volume reduction but none had complete histologic clearing.

At PellePharm, a biotechnology that Dr. Epstein cofounded, researchers are developing patidegib topical gel, 2%, for the reduction of disease burden of persistently developing BCCs in subjects with Gorlin syndrome. In a phase 3 trial, which just completed recruitment, adults are instructed to apply the gel twice daily to the face for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new surgically eligible BCCs that develop over the 12-month period. “We want to see if we can reduce the number of surgically eligible BCCs,” Dr. Epstein said. “We’re very hopeful.”

SDEF and this news organization are owned by the same parent company. Dr. Epstein spoke during a forum on cutaneous malignancies at the meeting.

Dr. Epstein disclosed that he has been a consultant for Genentech, Novartis, Amgen, and Infinity. He is also cofounder/director and stockholder in PellePharm.

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