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January 2, 2019

Imatinib Treatment for Locally Advanced or Metastatic Dermatofibrosarcoma Protuberans: A Systematic Review

Author Affiliations
  • 1Department of Dermatology, Facultad de Medicina, Pontificia Universidad Católica de Chile, Santiago, Chile
  • 2Dermatology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York
  • 3Department of Radiation Oncology, Memorial Sloan Kettering Cancer Center, New York, New York
  • 4Sarcoma Oncology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, Weill Cornell Medical College, New York, New York
JAMA Dermatol. 2019;155(3):361-369. doi:10.1001/jamadermatol.2018.4940
Key Points

Question  What are the outcomes associated with the use of imatinib in the treatment of metastatic or locally advanced dermatofibrosarcoma protuberans (DFSP)?

Findings  This systematic review finds that imatinib is associated with a complete or partial DFSP response in more than 60% of advanced cases, regardless of 400-mg or 800-mg daily dose. Severe adverse events occurred in about 15% of all cases.

Meaning  Imatinib should be considered a safe and effective therapy for advanced DFSP at a 400-mg/d starting dose.


Importance  Dermatofibrosarcoma protuberans (DFSP) has the potential for local destruction and recurrence, although it carries a low risk of metastasis. Complete surgical resection with negative margins is considered the gold standard for treatment; however, there are cases that are unresectable owing to tumor extension or size or owing to risk of cosmetic and/or functional impairment. Imatinib treatment has been used for locally advanced or metastatic DFSP.

Objective  To evaluate the usefulness of imatinib for treating DFSP.

Evidence Review  We conducted a systematic review on the PubMed and Embase databases for articles published from September 2002 through October 2017 using the key words “dermatofibrosarcoma” or “dermatofibrosarcoma protuberans” AND “therapy” AND “imatinib.” References within retrieved articles were also reviewed to identify additional studies. Studies of adults with histologically proven DFSP treated with imatinib as monotherapy or as an adjuvant or neoadjuvant therapy to surgery were included. Extracted data were analyzed using descriptive statistics. PRISMA guidelines were followed. All analysis took place October through December 2017.

Findings  Nine studies met inclusion criteria; 152 patients were included. The calculated mean patient age was 49.3 years (range, 20-73 years). Calculated mean tumor diameter was 9.9 cm (range, 1.2-49.0 cm). When COL1A1-PDGFβ protein translocation (collagen, type 1, alpha 1–platelet-derived growth factor β) was reported, it was present in 90.9% of patients (111 of 122). Complete response was seen in 5.2% of patients (8 of 152), partial response in 55.2% (84 of 152), stable disease in 27.6% (42 of 152), and progression in 9.2% (14 of 152). Four of the 152 patients (2.6%) were excluded from the analysis owing to unknown or unevaluable response. There were no differences in response rate using 400-mg or 800-mg daily doses (67.5% or 27 of 40 patients for 400-mg dose vs 67.1% or 49 of 73 patients for 800-mg dose complete or partial response; P > .99). Adverse events were present in at least 73.5% of cases (78 of 106); severe adverse events were present in 15.1% of cases (20 of 132).

Conclusions and Relevance  Imatinib is a useful directed therapy in patients with DFSP who are not surgical candidates owing to disease extension or significant cosmetic or functional impairment. There seems to be no difference between 400- or 800-mg daily doses.

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