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Tumour volume reduction after neoadjuvant chemotherapy impacts outcome in localised embryonal rhabdomyosarcoma
Author(s) -
Dantonello Tobias M.,
Stark Monika,
Timmermann Beate,
Fuchs Jörg,
Selle Barbara,
Linderkamp Christin,
Handgretinger Rupert,
Hagen Rudolf,
Feuchtgruber Simone,
Kube Stefanie,
Kosztyla Daniel,
Kazanowska Bernarda,
Ladenstein Ruth,
Niggli Felix,
Ljungman Gustaf,
Bielack Stefan S.,
Klingebiel Thomas,
Koscielniak Ewa
Publication year - 2015
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.25207
Subject(s) - medicine , chemotherapy , rhabdomyosarcoma , embryonal rhabdomyosarcoma , induction chemotherapy , multivariate analysis , oncology , surgery , gastroenterology , sarcoma , pathology
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. Pediatr Blood Cancer 2015;62:16–23. © 2014 Wiley Periodicals, Inc.

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