Extramammary Paget Disease Treatment & Management

Updated: Apr 18, 2017
  • Author: Blanca Anais Estupiñan; Chief Editor: Dirk M Elston, MD  more...
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Three separate reports describe successful treatment of extramammary Paget disease (EMPD) with 5-fluorouracil, [15] imiquimod, [16, 17, 18, 19, 20] and a combination of paclitaxel and trastuzumab. [21]

Specifically, imiquimod 5% cream applied 3 times weekly for 16 weeks induced complete resolution in a patient with perineal EMPD. One case report describes two patients with recurrent and extensive EMPD achieving complete remission after 5-aminolevulinic acid (5-ALA) photodynamic therapy and topical imiquimod. [22] Lesions were treated with 20% 5-ALA photodynamic therapy every 2 weeks for a total of 6 cycles followed by topical imiquimod every other day for 3 months. Topical imiquimod is considered a possible treatment option, especially when surgery is a challenge or contraindicated. However, more studies are needed to confirm the use of topical therapies for patients with EMPD. [23]


Surgical Excision

Margin-controlled surgical excision of all the involved epidermis is the most effective treatment. Extramammary Paget disease (EMPD) extends beyond the visibly involved margins. Obviously involved skin should be examined by using transverse frozen sections or serial vertical sections (see Workup). Multiple scouting biopsies performed before surgery may aid in planning a more precise initial excision. [3]

Multifocal disease is a challenge for any surgical method that relies on contiguous tumor spread for effective margin control—even micrographic surgery. Currently, wide radical excision with 5-cm margins or Mohs micrographic surgery are the recommended excision options. When operating in the anogenital region, providers must consider the aesthetic and functional consequences patients may face after radical excision. However, Mohs micrographic surgery offers lower recurrence rates after excision of primary tumors, with a smaller margin of normal skin removed. [24, 25] The recurrence rate of primary tumors after standard surgical excision is 30-60%. The rate after excision with Mohs micrographic surgery is 8-26%. The average time to recurrence is 2.5 years, with case reports of more than 10 years follow-up. Although long-term outcomes are improved using Mohs micrographic surgery, some patients may not be able to afford the higher treatment costs. Additional studies have explored minimal surgical therapy as a third excision treatment option and have reported success with 1-cm excisional borders in well-circumscribed EMPD. [26]



Consultations and Long-Term Monitoring

Depending on the anatomic location of extramammary Paget disease (EMPD), treatment should be coordinated with an appropriate surgical subspecialist (eg, a urologist, a colorectal surgeon, or a gynecologist). Optimally, the consultant would have some experience treating this specific condition. Further consultation with a radiologist and a gastroenterologist may also be required to order appropriate screening examinations for internal malignancy.

Patients with EMPD require follow-up examination every 3 months after surgery to assess possible recurrence. This routine should continue for at least 24 months, after which time examinations may be done annually. Consider repeating other endoscopic or imaging studies on a regular basis according to the specific recommendations of the consultants.

A 2017 study found serum cytokeratin 19 fragment 21-1 (CYFRA 21-1) levels to be useful in monitoring tumor burden and treatment response. Compared with carcinoembryonic antigen (CEA) monitoring, CYFRA 21-1 levels were more sensitive in detecting tumor reduction as well as recurrence, with a corresponding decrease and increase in serum concentration, respectively. [27]