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The Desmoid Tumor Research Foundation

December 2017

The EpSSG NRSTS 2005 treatment protocol for desmoid-type fibromatosis in children: an international prospective case series

Author Information

Daniel Orbach, Bernadette Brennan, Gianni Bisogno, Max Van Noesel, Ve_ronique Minard-Colin, Julia Daragjati, Michela Casanova,Nadege Corradini, Ilaria Zanetti, Gian Luca De Salvo, Anne Sophie Defachelles, Anna Kelsey, Myriam Ben Arush, Nadine Francotte, Andrea Ferrari

a) SIREDO Oncology Center, Institut Curie, Paris, Franceb)Department of Paediatric Oncology, Royal Manchester Children's Hospital, Manchester, UKc)Department of Diagnostic Paediatric Histopathology, Royal Manchester Children's Hospital, Manchester, UKd)Pediatric Hematology and Oncology Division, Padova University, Padova, Italye)Princess Maxima Center for Pediatric Oncology, Utrecht, Netherlandsf)Department of Paediatric and Adolescent Oncology, Gustave-Roussy, Villejuif, Franceg)Clinical Trials and Biostatistics Unit, IRCCS Istituto Oncologico Veneto, Padova, Italyh)Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italyi)Institut d'Hematologie et d'Oncologie Pediatrique, Centre Leon Berard, Lyon, Francej)Pediatric Oncology Department, Centre Oscar Lambret, 59020 Lille, Francek)Pediatric Department, Rambam Health Care Campus Haifa, Israell)Department of Pediatrics, CHC-Clinique Esperance, Montegnee, Belgium

Publication

Lancet Child Ado Health

Summary

In 2005, the European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) proposed a conservative treatment algorithmÑconsisting of an initial wait-and-see strategy, non-mutilating surgery, and minimal-morbidity chemotherapy (in the case of tumour progression)Ñfor paediatric patients with desmoid-type fibromatosis. We aimed to investigate the outcomes of this algorithm. In this case series, patients (<25 years) with desmoid-type fibromatosis from 57 centres in eight countries were prospectively registered through a web-based system. Diagnosis was based on histological analysis of the tumour specimen after biopsy or surgery, and we classified patients by tumour site, clinical stage (TNM system), and post-surgical stage (Intergroup Rhabdomyosarcoma Study system). From Oct 1, 2005, to July 31, 2016, 173 patients (median age 11.4 years [IQR 4.0Ð14.1], 88 [51%] male patients) were registered. After excluding patients with missing data, 54 (35%) patients had no immediate therapy (wait-and-see strategy), 47 (31%) had immediate surgery, and 53 (34%) had immediate chemotherapy after diagnosis. 5-year progression-free survival was 36.5% (95% CI 27.8Ð45.2) overall, 26.7% (14.2Ð41.0) in the wait-and-see group, 41.2% (25.8Ð55.9) in the surgery group, and 42.8% (27.2Ð57.6) in the chemotherapy group (overall log-rank p=0.17; wait-and-see vs surgery p=0.12; wait-and-see vs chemotherapy p=0.13). In multivariable analysis, large tumour size (>5 cm) was associated with worse progression-free survival (hazard ratio 2.25, 95% CI 1.34Ð3.76; p=0.0021). Apart from one patient in the chemotherapy group who died from a secondary tumour (head and neck anaplastic embryonal rhabdomyosarcoma), all patients were alive at the time of analysis. 13 (8%) patients had biopsy only (no further treatment), 65 (42%) had chemotherapy only, 31 (20%) had surgery only, 36 (23%) had both chemotherapy and surgery, and nine (6%) had radiotherapy in addition to other therapies. In paediatric patients with desmoid-type fibromatosis, the EpSSG conservative strategy did not compromise outcomes and could be adopted to reduce treatment burden.

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