Daniel Orbach1, Bernadette Brennan2, Gianni Bisogno3, Max Van Noesel4, Véronique Minard-Colin5, Julia Daragjati6, Michela Casanova7, Nadege Corradini8, Ilaria Zanetti6, Gian Luca De Salvo6, Anne Sophie Defachelles9, Anna Kelsey10, Myriam Ben Arush11, Nadine Francotte12, Andrea Ferrari7. 1. SIREDO Oncology Center (Care, Innovation and Research for Children, Adolescents and Young Adults with Cancer), Institut Curie, Paris, France. Electronic address: daniel.orbach@curie.fr. 2. Department of Paediatric Oncology, Royal Manchester Children's Hospital, Manchester, UK. 3. Pediatric Hematology and Oncology Division, Padova University, Padova, Italy. 4. Princess Maxima Center for Pediatric Oncology, Utrecht, Netherlands. 5. Department of Paediatric and Adolescent Oncology, Gustave-Roussy, Villejuif, France. 6. Clinical Trials and Biostatistics Unit, IRCCS Istituto Oncologico Veneto, Padova, Italy. 7. Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy. 8. Institut d'Hematologie et d'Oncologie Pediatrique, Centre Leon Berard, Lyon, France. 9. Pediatric Oncology Department, Centre Oscar Lambret, 59020 Lille, France. 10. Department of Diagnostic Paediatric Histopathology, Royal Manchester Children's Hospital, Manchester, UK. 11. Pediatric Department, Rambam Health Care Campus Haifa, Israel. 12. Department of Pediatrics, CHC-Clinique Esperance, Montegnee, Belgium.
Abstract
BACKGROUND: In 2005, the European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) proposed a conservative treatment algorithm-consisting of an initial wait-and-see strategy, non-mutilating surgery, and minimal-morbidity chemotherapy (in the case of tumour progression)-for paediatric patients with desmoid-type fibromatosis. We aimed to investigate the outcomes of this algorithm. METHODS: 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). Progression-free survival was defined as the time from diagnosis until disease progression (clinical or radiological progressive disease, relapse, or death from any cause). FINDINGS: From Oct 1, 2005, to July 31, 2016, 173 patients (median age 11·4 years [IQR 4·0-14·1], 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 27·8-45·2) overall, 26·7% (14·2-41·0) in the wait-and-see group, 41·2% (25·8-55·9) in the surgery group, and 42·8% (27·2-57·6) 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 1·34-3·76; 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. INTERPRETATION: In paediatric patients with desmoid-type fibromatosis, the EpSSG conservative strategy did not compromise outcomes and could be adopted to reduce treatment burden. FUNDING: S Wisnia and la Città della Speranza Foundation.
BACKGROUND: In 2005, the European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) proposed a conservative treatment algorithm-consisting of an initial wait-and-see strategy, non-mutilating surgery, and minimal-morbidity chemotherapy (in the case of tumour progression)-for paediatric patients with desmoid-type fibromatosis. We aimed to investigate the outcomes of this algorithm. METHODS: 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). Progression-free survival was defined as the time from diagnosis until disease progression (clinical or radiological progressive disease, relapse, or death from any cause). FINDINGS: From Oct 1, 2005, to July 31, 2016, 173 patients (median age 11·4 years [IQR 4·0-14·1], 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 27·8-45·2) overall, 26·7% (14·2-41·0) in the wait-and-see group, 41·2% (25·8-55·9) in the surgery group, and 42·8% (27·2-57·6) 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 1·34-3·76; 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. INTERPRETATION: In paediatric patients with desmoid-type fibromatosis, the EpSSG conservative strategy did not compromise outcomes and could be adopted to reduce treatment burden. FUNDING: S Wisnia and la Città della Speranza Foundation.
Authors: Katrina M Ingley; Sally M Burtenshaw; Nicole C Theobalds; Lawrence M White; Martin E Blackstein; Rebecca A Gladdy; Seng Thipphavong; Abha A Gupta Journal: Cancer Med Date: 2019-07-13 Impact factor: 4.452
Authors: Andrea Ferrari; Bernadette Brennan; Michela Casanova; Nadege Corradini; Pablo Berlanga; Reineke A Schoot; Gema L Ramirez-Villar; Akmal Safwat; Gabriela Guillen Burrieza; Patrizia Dall'Igna; Rita Alaggio; Lisa Lyngsie Hjalgrim; Susanne Andrea Gatz; Daniel Orbach; Max M van Noesel Journal: Cancer Manag Res Date: 2022-09-23 Impact factor: 3.602