Evidence-Based Clinical Practice Guidelines for Extramammary Paget Disease

Nour Kibbi, Joshua L. Owen, Brandon Worley, Jake X. Wang, Vishnu Harikumar, Malia B. Downing, Sumaira Z. Aasi, Phyu P. Aung, Christopher A. Barker, Diana Bolotin, Jeremy S. Bordeaux, Todd V. Cartee, Sunandana Chandra, Nancy L. Cho, Jennifer N. Choi, Kee Yang Chung, William A. Cliby, Oliver Dorigo, Daniel B. Eisen, Yasuhiro FujisawaNicholas Golda, Thorvardur R. Halfdanarson, Christos Iavazzo, Shang I.Brian Jiang, Jean Kanitakis, Ashraf Khan, John Y.S. Kim, Timothy M. Kuzel, Naomi Lawrence, Mario M. Leitao, Allan B. Maclean, Ian A. Maher, Bharat B. Mittal, Kishwer S. Nehal, David M. Ozog, Curtis A. Pettaway, Jeffrey S. Ross, Anthony M. Rossi, Sabah Servaes, Michael J. Solomon, Valencia D. Thomas, Maria Tolia, Bryan B. Voelzke, Abigail Waldman, Michael K. Wong, Youwen Zhou, Nobuo Arai, Alexandria Brackett, Sarah A. Ibrahim, Bianca Y. Kang, Emily Poon, Murad Alam

Research output: Contribution to journalReview articlepeer-review

10 Scopus citations


Importance: Extramammary Paget disease (EMPD) is a frequently recurring malignant neoplasm with metastatic potential that presents in older adults on the genital, perianal, and axillary skin. Extramammary Paget disease can precede or occur along with internal malignant neoplasms. Objective: To develop recommendations for the care of adults with EMPD. Evidence Review: A systematic review of the literature on EMPD from January 1990 to September 18, 2019, was conducted using MEDLINE, Embase, Web of Science Core Collection, and Cochrane Libraries. Analysis included 483 studies. A multidisciplinary expert panel evaluation of the findings led to the development of clinical care recommendations for EMPD. Findings: The key findings were as follows: (1) Multiple skin biopsies, including those of any nodular areas, are critical for diagnosis. (2) Malignant neoplasm screening appropriate for age and anatomical site should be performed at baseline to distinguish between primary and secondary EMPD. (3) Routine use of sentinel lymph node biopsy or lymph node dissection is not recommended. (4) For intraepidermal EMPD, surgical and nonsurgical treatments may be used depending on patient and tumor characteristics, although cure rates may be superior with surgical approaches. For invasive EMPD, surgical resection with curative intent is preferred. (5) Patients with unresectable intraepidermal EMPD or patients who are medically unable to undergo surgery may receive nonsurgical treatments, including radiotherapy, imiquimod, photodynamic therapy, carbon dioxide laser therapy, or other modalities. (6) Distant metastatic disease may be treated with chemotherapy or individualized targeted approaches. (7) Close follow-up to monitor for recurrence is recommended for at least the first 5 years. Conclusions and Relevance: Clinical practice guidelines for EMPD provide guidance regarding recommended diagnostic approaches, differentiation between invasive and noninvasive disease, and use of surgical vs nonsurgical treatments. Prospective registries may further improve our understanding of the natural history of the disease in primary vs secondary EMPD, clarify features of high-risk tumors, and identify superior management approaches..

Original languageEnglish (US)
Pages (from-to)618-628
Number of pages11
JournalJAMA Oncology
Issue number4
StatePublished - Apr 2022

Bibliographical note

Funding Information:
supported by unrestricted research funding from the Section of Cutaneous Surgery in the Northwestern University Department of Dermatology. This research was funded, in part, by National Institutes of Health/National Cancer Institute Cancer Center Support Grant P30 CA008748 (to Dr Leitao).

Funding Information:
reported serving as an advisory board member for Castle Biosciences outside of the submitted work. Dr Barker reported receiving grants from the National Institutes of Health through subcontracts from Physical Sciences Incorporated, EMD Serono, Alpha Tau Medical, Merck, and Amgen; personal fees from the American Society for Radiation Oncology, the American Brachytherapy Society, and Regeneron; and nonfinancial support from Alpha Tau Medical, H. Lee Moffitt Cancer Center, and Regeneron outside the submitted work. Dr Bolotin reported serving as an investigator without compensation on clinical trials for Replimune and Pelle Pharm outside the submitted work. Dr Chandra reported serving as an advisory board member for Exicure, Novartis, Bristol Myers Squibb, Sanofi Genzyme, and Regeneron outside the submitted work. Dr Choi reported receiving personal fees from Regeneron, Parexel, OnQuality Pharmaceuticals, Bristol Myers Squibb, and Kyowa Kirin International outside the submitted work. Dr Halfdanarson reported receiving personal fees from Curium, Lexicon, Terumo, and ScioScientific; receiving research support paid to the institution from Thermo Fisher Scientific, Basilea, Turnstone Biologics, Agios, and Novartis; and serving as a consultant with fees paid to the institution for ITM, Ipsen, and Novartis outside the submitted work. Dr Leitao reported receiving funding in part through the National Institutes of Health/National Cancer Institute Cancer Center Support Grant P30 CA008748; serving on the advisory board for and receiving personal fees from Ethicon/J&J and Takeda; and receiving grants to the institution from KCI/Acelity outside the submitted work. Dr Ross reported being an employee of Foundation Medicine Inc, being an equity owner in Roche Holdings, serving on the board of directors of and being an equity owner in Celsius Therapeutics Inc, and serving as a consultant to and being equity owner of Tango Therapeutics Inc. Dr Rossi reported serving as a consultant for Almirall, Merz, Dynamed, Canfield Scientific, Evolus, Biofrontera, Quantia MD, Lam Therapeutics, Regeneron, and Cutera; receiving travel support from Mavig and L’Oreal; serving on the advisory board for Allergan Inc and Skinfix; being founder of DAR Companies; receiving research grants from ASLMS: A Ward Memorial Research Grant and Skin Cancer Foundation; receiving research/study funding from Regen, LeoPharma, and Biofrontera; serving on the editorial boards of Lasers in Surgery and Medicine, Cutis, Journal of the American Academy of Dermatology, and Dermatologic Surgery; being a board member of ASDS; and being a committee member and/or chair for AAD, ASDS, and ASLMS. Dr Thomas reported serving as an advisory board committee member for Regeneron and Merck outside the submitted work. Dr Wong reported serving on the advisory board for Merck Pharmaceuticals, Bristol Myers Squibb, EMD Serona, Pfizer, Regeneron, and Castle Biosciences outside the submitted work. No other disclosures were reported.

Publisher Copyright:
© 2022 American Medical Association. All rights reserved.

PubMed: MeSH publication types

  • Journal Article
  • Systematic Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't


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