1497P - Efficacy and Safety of Patients Treated Long-Term with Trabectedin (T) on the Expanded Access Program: A Retrospective Analysis

Date 11 September 2017
Event ESMO 2017 Congress
Session Poster display session
Topics Cytotoxic agents
Soft Tissue Sarcomas
Biological therapy
Presenter Elizabeth Davis
Citation Annals of Oncology (2017) 28 (suppl_5): v521-v538. 10.1093/annonc/mdx387
Authors E. Davis1, S. Schuetze2, S. Patel3, R. Maki4, R.L. Jones5, C.R. Shin6, R. Knoblauch7, G. Wang8, M. Smith7, G.D.S. Demetri9, P. Merriam10
  • 1Division Of Hematology/oncology, Vanderbilt University Medical Center, 37232 - Nashville/US
  • 2Division Of Hematology/oncology, University of Michigan, Ann Arbor/US
  • 3Department Of Sarcoma Medical Oncology, The University of Texas, MD Anderson Cancer Center, Houston/US
  • 4Medical Oncology And Hematology, Monter Cancer Center, Lake Success/US
  • 5Medical Oncology, Royal Marsden Hospital, SW3 6JJ - London/GB
  • 6Med Group Oncology, Janssen Scientific Affairs, LLC, Horsham/US
  • 7Clin Oncology, Janssen Pharmaceuticals, 8869 - Raritan/US
  • 8Biostats, Janssen Research & Development, LLC, Spring House/US
  • 9Center For Sarcoma And Bone Oncology, Dana-Farber Cancer Institute and Ludwig Center at Harvard, Boston/US
  • 10Center For Sarcoma And Bone Oncology, Dana-Farber Cancer Institute, 02215 - Boston/US



Treatment of soft tissue sarcoma (STS) with long-term systemic therapy can be limited by cumulative toxicity. Treatment with T for prolonged courses without the cumulative toxicity has been previously described from clinical trials. Here we report the efficacy and safety for patients (pts) treated long term (≥ 6 months) in a real world setting in the T Expanded Access Program from 2005-2010.


In this retrospective analysis of pts with pre-treated, relapsed/refractory STS of multiple histologies treated ≥6 mo with T (1.5 mg/m2 iv q3wk), we compared pts treated 6-12 mo and >12 mo.


Of 1803 pts, 401 (21.6%) remained on treatment ≥6 mos; 268 (14.5%) for 6-12 mo and 133 (7.2%) >12 mo. Demographics did not differ. Leiomyosarcoma or liposarcoma were the most common histologies. The mOS (mo) was 18.1 and 47.0, ORR was 7.8% and 6.8%, and clinical benefit rate (CR+PR+SD) (95%CI) was 47.4% (41.3;53.6) and 38.3% (30.1;47.2) in the 6-12 mo and >12 mo groups, respectively. The incidence of adverse events (AE)s and serious adverse events (SAE)s were similar in both groups (Table). The most common grade 3/4 AEs occurring in ≥ 5% were neutropenia, thrombocytopenia, anemia, ALT/AST increase, fatigue and nausea. A majority received dose reduction or delay; the primary reason for treatment discontinuation was disease progression. The longest observed duration of treatment was 55 mo (64 cycles; synovial sarcoma) and 54 mo (73 cycles; uterine leiomyosarcoma).Table:

1497P Safety and Efficacy

6-12 Months>12 Months
(N = 268)(N = 133)
Median Treatment Duration (mo), range8.4(6; 12)16.3 (12; 55)
Treatment Response,
Complete response, n (%)1 (0.4)3 (2.3)
Partial response, n (%)20 (7.5)6 (4.5)
Stable disease, n (%)106 (39.6)42 (31.6)
Progressive disease, n (%)20 (7.5)12 (9.0)
Not available, n (%)121 (45.1)70 (52.6)
Treatment-emergent adverse events (TEAEs)225 (84.0)119 (89.5)
Serious TEAEs88 (32.9)47 (35.3)
Treatment discontinued255(95.1)103 (77.4)
Due to disease progression192(71.6)72 (54.1)
Due to adverse event10 (3.7)2 (1.5)
Patients with cycle delay154 (57.5)82 (61.7)
Patients with dose reduction172 (64.2)104 (78.2)


T can be safely administered and well tolerated in pts who receive a prolonged duration (≥6 mo) of therapy. Improved mOS may be achieved in pts who experience prolonged disease stabilization following T but adjustments in dose or schedule is frequently required.

Clinical trial identification


Legal entity responsible for the study

Janssen Research & Development, LLC


Janssen Research & Development, LLC


S. Schuetze: Honorarium for Janssen ad hoc scientific advisory board to me. Research funding to institution (University of Michigan) from Janssen. S. Patel: Consultant to: Janssen, Eisai, Novartis, CytRx, Epizyme, Bayer, Eli Lilly Grants for clinical trial from: Janssen, Eisai, Morphotek. R. Maki: Consulting or advisory role: SARC, ASCO, AADX, ARCUS, Bayer, GSAI, GEM, Novartis, GSK, Immune Design, Janssen, Kyropharm, Lilly Tracon and Presage Research Funding and Travel, Accommodations, Expenses: Tracon, Immune Design, Lilly, and SARC. R.L. Jones: Consultant for: Adaptimmune, Blueprint, Eisai, Epizyme, Daichii, Deciphera, Janssen Scientific Affairs, LLC, Immunedesign, Lilly, Merck, Pharmamar. C.R. Shin, R. Knoblauch, G. Wang, M. Smith: Employee of Janssen and own stock in Johnson & Johnson. G.D.S. Demetri: Consulting: Novartis, Janssen, PharmaMar, Daiichi-Sankyo, Adaptimmune, Eisai Patent licensed to Novartis from Dana-Farber with royalty paid to Dana-Farber Research support to Dana-Farber: Novartis, Janssen. All other authors have declared no conflicts of interest.