A pilot study of anti-CTLA4 antibody ipilimumab in patients with synovial sarcoma Journal Article


Authors: Maki, R. G.; Jungbluth, A. A.; Gnjatic, S.; Schwartz, G. K.; D'Adamo, D. R.; Keohan, M. L.; Wagner, M. J.; Scheu, K.; Chiu, R.; Ritter, E.; Kachel, J.; Lowy, I.; Old, L. J.; Ritter, G.
Article Title: A pilot study of anti-CTLA4 antibody ipilimumab in patients with synovial sarcoma
Abstract: Background. Patients with recurrent synovial sarcomas have few options for systemic therapy. Since they express large amounts of endogenous CT (cancer testis) antigens such as NY-ESO-1, we investigated the clinical activity of single agent anti-CTLA4 antibody ipilimumab in patients with advanced or metastatic synovial sarcoma. Methods. A Simon two-stage phase II design was used to determine if there was sufficient activity to pursue further. The primary endpoint was tumor response rate by RECIST 1.0. Patients were treated with ipilimumab 3 mg/kg intravenously every 3 weeks for three cycles and then restaged. Retreatment was possible for patients receiving an extra three-week break from therapy. Sera and peripheral blood mononuclear cells were collected before and during therapy to assess NY-ESO-1-specific immunity. Results. Six patients were enrolled and received 1-3 cycles of ipilimumab. All patients showed clinical or radiological evidence of disease progression after no more than three cycles of therapy, for a RECIST response rate of 0%. The study was stopped for slow accrual, lack of activity, and lack of immune response. There was no evidence of clinically significant either serologic or delayed type hypersensitivity responses to NY-ESO-1 before or after therapy. Conclusion. Despite high expression of CT antigens by synovial sarcomas of patients treated in this study, there was neither clinical benefit nor evidence of anti-CT antigen serological responses. Assessment of the ability of synovial sarcoma cell lines to present cancer-germ cell antigens may be useful in determining the reason for the observed lack of immunological or clinical activity. © 2013 Robert G. Maki et al.
Keywords: adult; cancer survival; clinical article; treatment response; overall survival; disease course; fatigue; diarrhea; drug efficacy; hypophosphatemia; side effect; treatment duration; cancer staging; antigen expression; ipilimumab; metastasis; multiple cycle treatment; phase 2 clinical trial; nausea; vomiting; clinical assessment; creatinine; hemoglobin; creatinine blood level; abdominal pain; alanine aminotransferase blood level; aspartate aminotransferase blood level; hyperglycemia; lymphocytopenia; rash; alanine aminotransferase; alkaline phosphatase; aspartate aminotransferase; hypoalbuminemia; thorax pain; immune response; pilot study; ny eso 1 antigen; acne; muscle weakness; thrombocyte count; pleura effusion; alkaline phosphatase blood level; hyperbilirubinemia; leukocyte count; synovial sarcoma; peripheral blood mononuclear cell; bronchitis; hypocalcemia; gastritis; musculoskeletal chest pain; proctalgia
Journal Title: Sarcoma
Volume: 2013
ISSN: 1357-714X
Publisher: Hindawi Publishing Corporation  
Date Published: 2013-01-01
Start Page: 168145
Language: English
DOI: 10.1155/2013/168145
PROVIDER: scopus
PMCID: PMC3608267
PUBMED: 23554566
DOI/URL:
Notes: --- - "Export Date: 3 June 2013" - "CODEN: SARCF" - "Source: Scopus"
Altmetric Score
MSK Authors
  1. Gary Schwartz
    364 Schwartz
  2. David R D'Adamo
    37 D'Adamo
  3. Robert Maki
    211 Maki
  4. Mary Louise Keohan
    66 Keohan
  5. Sacha Gnjatic
    111 Gnjatic
  6. Achim Jungbluth
    341 Jungbluth
  7. Gerd Ritter
    158 Ritter
  8. Erika Ritter
    34 Ritter
  9. Lloyd J Old
    386 Old
  10. Rita Jou-Chien Chiu
    2 Chiu
  11. Kelly Scheu
    6 Scheu
  12. Jennifer Kachel
    1 Kachel