Tumour volume reduction after neoadjuvant chemotherapy impacts outcome in localised embryonal rhabdomyosarcoma.

Abstract

BACKGROUND:

Response (tumour volume reduction) to induction chemotherapy has been used to stratify secondary local and systemic treatment of Intergroup Rhabdomyosarcoma Study Group III (IRSG-III) embryonal rhabdomyosarcoma (RME) in consecutive CWS-trials. To evaluate its actual impact we studied response-related treatment and outcomes.

PROCEDURE:

Patients with IRSG-III RME <21 years and non-response (NR, <33% volume reduction) in five consecutive CWS-trials were analysed and compared with partial responders (PAR, ≥ 33% reduction). The NR was reviewed and sub-classified as Objective Response (OR, <0%-33% reduction) or Stable/Progressive Disease (SPD).

RESULTS:

Fifty-nine of 529 patients had NR (n = 34 OR, n = 25 SPD). Primary risk-factors including age, tumour size, and TN-classification did not differ between NR and PAR groups but NR had more patients with unfavourable sites comparatively (P = 0.04). There were no differences in primary risk-factors between OR and SPD. Significant factors associated with poor outcome in multivariate analysis were NR, TN-classification, age >10 years, tumour size >5 cm and therapy in older trials. After response assessment n = 24 NR continued to receive induction chemotherapy, n = 32 received other combinations and n = 3 no further chemotherapy. Forty-two non-responders were irradiated, and the tumours were completely resected in n = 20. After a median follow-up of 8 years, 34 NR are alive. Seventeen of 21 failures leading to disease-related deaths were locoregional. The five-year overall survival rate (OS) was 76 ± 4% for PAR, 79 ± 14% for OR, but only 40 ± 19% for SPD (P < 0.001).

CONCLUSION:

Response to induction chemotherapy appears to be an important surrogate marker of poor outcome in patients with SPD largely due to ineffective local control.

• Authors: Tobias M Dantonello, Monika Stark, Beate Timmermann, Jörg Fuchs, Barbara Selle, Christin Linderkamp, Rupert Handgretinger, Rudolf Hagen, Simone Feuchtgruber, Stefanie Kube, Daniel Kosztyla, Bernarda Kazanowska, Ruth Ladenstein, Felix Niggli, Gustaf Ljungman, Stefan S Bielack, Thomas Klingebiel, Ewa Koscielniak, Cooperative Weichteilsarkom Studiengruppe [CWS]
• Journal: Pediatric blood & cancer (Pediatr Blood Cancer) Vol. 62 Issue 1 Pg. 16-23 (Jan 2015) ISSN: 1545-5017 [Electronic] United States
• PMID: 25263634 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2014 Wiley Periodicals, Inc.

Topics

• Adolescent
• Adult
• Antineoplastic Combined Chemotherapy Protocols (therapeutic use)
• Child
• Child, Preschool
• Female
• Follow-Up Studies
• Humans
• Infant
• Infant, Newborn
• Lymphatic Metastasis
• Male
• Neoadjuvant Therapy
• Neoplasm Recurrence, Local (drug therapy, mortality, pathology)
• Neoplasm Staging
• Prognosis
• Retrospective Studies
• Rhabdomyosarcoma, Embryonal (drug therapy, mortality, pathology)
• Survival Rate
• Tumor Burden
• Young Adult