Michela Casanova1, Bernadette Brennan2, Rita Alaggio3, Anna Kelsey4, Daniel Orbach5, Max M van Noesel6, Nadege Corradini7, Veronique Minard-Colin8, Ilaria Zanetti9, Gianni Bisogno9, Soledad Gallego10, Johannes H M Merks11, Gian Luca De Salvo12, Andrea Ferrari13. 1. Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy. 2. Paediatric Oncology, Royal Manchester Children's Hospital, Manchester, United Kingdom. 3. Pathology Department, Ospedale Pediatrico Bambino Gesù IRCCS, Roma, Italy. 4. Department of Paediatric Histopathology, Royal Manchester Children's Hospital, Manchester, United Kingdom. 5. SIREDO Oncology Center (Care, Innovation and Research for Children and AYA with Cancer), Institut Curie, PSL University, Paris, France. 6. Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands. 7. Department of Pediatric Oncology, Institut d'Hematologie et d'Oncologie Pédiatrique, Centre Léon Bérard, Lyon, France. 8. Department of Pediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France. 9. Hematology and Oncology Division, Department of Women's and Children's Health, University of Padova, Padova, Italy. 10. Paediatric Oncology, Hospital Universitario Vall d'Hebron, Barcelona, Spain. 11. Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands; Department of Pediatric Oncology, Emma Children's Hospital, Academic Medical Center Amsterdam, Netherlands. 12. Clinical Trials and Biostatistics Unit, IRCCS Istituto Oncologico Veneto, Padova, Italy. 13. Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy. Electronic address: andrea.ferrari@istitutotumori.mi.it.
Abstract
INTRODUCTION: We report the clinical findings and results of treatment in the cohort of patients with inflammatory myofibroblastic tumor (IMT) managed according to the European pediatric Soft Tissue Sarcoma Study Group (EpSSG) protocol from 2005 to 2016. METHODS: Patients (<25 years old) with IMT from 9 countries were prospectively registered via a web-based system. Their histology was reviewed by a national/international pathology panel. Immunohistochemistry for ALK assessment was mandatory. No adjuvant therapy was suggested for initially resected tumors. No specific systemic therapy was recommended for cases of unresectable disease. RESULTS: Among 80 cases of IMT registered, 20 were excluded because pathology review led to a revised diagnosis. Of the remaining 60 patients (median age 9.5 years), 59 had localized, and 1 had multifocal/metastatic disease. The lung was the primary site in 14 cases. IMT developed as a second tumor in 2 cases. Forty cases were ALK-positive, and 20 were ALK-negative. Five-year event-free survival (EFS) and overall survival (OS) were 82.9% and 98.1%, respectively. No clinical variables correlated statistically with the outcome: survival was the same for ALK-positive and ALK-negative cases. The overall response to systemic therapy was 64%: 8/10 cases responded to vinblastine-methotrexate chemotherapy, and 5/5 to ALK-inhibitors. CONCLUSIONS: This study demonstrated a good overall prognosis for IMT, even for initially unresectable disease and in ALK-negative cases. Chemotherapy is still a valid option for advanced disease. Larger studies involving both pediatric and adult patients are needed to clarify the role of ALK inhibitors.
INTRODUCTION: We report the clinical findings and results of treatment in the cohort of patients with inflammatory myofibroblastic tumor (IMT) managed according to the European pediatric Soft Tissue Sarcoma Study Group (EpSSG) protocol from 2005 to 2016. METHODS:Patients (<25 years old) with IMT from 9 countries were prospectively registered via a web-based system. Their histology was reviewed by a national/international pathology panel. Immunohistochemistry for ALK assessment was mandatory. No adjuvant therapy was suggested for initially resected tumors. No specific systemic therapy was recommended for cases of unresectable disease. RESULTS: Among 80 cases of IMT registered, 20 were excluded because pathology review led to a revised diagnosis. Of the remaining 60 patients (median age 9.5 years), 59 had localized, and 1 had multifocal/metastatic disease. The lung was the primary site in 14 cases. IMT developed as a second tumor in 2 cases. Forty cases were ALK-positive, and 20 were ALK-negative. Five-year event-free survival (EFS) and overall survival (OS) were 82.9% and 98.1%, respectively. No clinical variables correlated statistically with the outcome: survival was the same for ALK-positive and ALK-negative cases. The overall response to systemic therapy was 64%: 8/10 cases responded to vinblastine-methotrexate chemotherapy, and 5/5 to ALK-inhibitors. CONCLUSIONS: This study demonstrated a good overall prognosis for IMT, even for initially unresectable disease and in ALK-negative cases. Chemotherapy is still a valid option for advanced disease. Larger studies involving both pediatric and adult patients are needed to clarify the role of ALK inhibitors.
Authors: Giacomo Giulio Baldi; Mehdi Brahmi; Salvatore Lo Vullo; Elena Cojocaru; Olivier Mir; Michela Casanova; Bruno Vincenzi; Tommaso Martino De Pas; Giovanni Grignani; Maria Abbondanza Pantaleo; Jean Yves Blay; Robin Lewis Jones; Axel Le Cesne; Anna Maria Frezza; Alessandro Gronchi; Paola Collini; Angelo Paolo Dei Tos; Carlo Morosi; Luigi Mariani; Paolo Giovanni Casali; Silvia Stacchiotti Journal: Oncologist Date: 2020-07-12
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