Les sarcomes osseux, incluant les ostéosarcomes (OS) et les sarcomes d’Ewing (ES), sont des tumeurs rares mais agressives, nécessitant une prise en charge spécialisée. Parmi les techniques d’imagerie disponibles, la TEP-TDM au FDG (TEP FDG) a pris une place majeure ces dernières années dans ce champ oncologique. Elle s’est imposée pour le bilan d’extension initial, grâce à sa sensibilité élevée pour la détection des métastases extrapulmonaires, notamment pour la détection des atteintes osseuses et ostéomédullaires. L’utilisation de la TEP FDG se développe pour l’évaluation de la réponse thérapeutique, à la chimiothérapie néoadjuvante, et aux traitements systémiques chez les patients présentant une maladie métastatique avancée. La TEP FDG est plus facilement accessible et présente moins de contraintes d’acquisition que l’IRM corps entier, éléments d’importance pour la prise en charge d’une population pédiatrique. En outre, les dernières avancées technologiques permettront d’améliorer la radioprotection de ces jeunes patients.

Bone sarcomas, including osteosarcomas (OS) and Ewing sarcomas (ES), are rare but aggressive tumors requiring specialized care. Among the available imaging techniques, FDG PET-CT (PET FDG) has taken a significant role in this oncological field in recent years. It has been established for the initial staging due to its high sensitivity for detecting extrapulmonary metastases, particularly for detecting bone and bone marrow involvement. The use of PET FDG is also developing for the evaluation of therapeutic response to neoadjuvant chemotherapy and systemic treatments in patients with advanced metastatic disease. PET FDG offers better availability and fewer acquisition constraints compared to whole-body MRI, which is important for managing a pediatric population. Furthermore, the latest technological advances will improve radiation protection for these young patients. (c) 2025 Elsevier Masson SAS. All rights are reserved, including those for text and data mining, AI training, and similar technologies.

Background and aims: During the first lockdown of the novel Coronavirus pandemic (COVID-19), we developed a teleconsultation model to replace in-person visits. We conducted a survey-based study with the aim to evaluate satisfaction and emotions of health workers (HWs), to assess the feasibility of teleconsultations and to evaluate technical issues. Methods: This is a prospective monocentric exploratory study in which data were collected from March until May 2020 in the Lyon Pediatric Hematology and Oncology institute (IHOPe). We realized synchronous, video-based consultations between HWs and pediatric patients, treated for blood or solid malignancies or blood benign diseases. Every HW completed an online, pseudo-anonymized questionnaire which covered technical aspects, data concerning satisfaction, perception of the quality of the interaction with the patient and emotions felt after the teleconsultation. A score was calculated for each aspect. In order to study the relationship between the scores, the correlation coefficient method was used. Results: Eleven specialists participated in the study and we selected 84 questionnaires. With a satisfaction rate of 74%, HWs felt mostly calm (80%), relaxed (70%), stress-free (69%) and relieved (65%). We calculated the following median scores: an overall satisfaction score of 6.67 (0-10), a global feeling score of 8.79 (3.33-10.00), and a quality score of 7.34 (2.50-10.00). A strong correlation between the quality of the teleconsultation and the satisfaction of the HWs has been highlighted (r = 0,588). Conclusion: Our series is an encouragingly positive experience from the perspective of the HWs, their feelings and perceptions.

Le sarcome d’Ewing est un sarcome agressif de l’os et des tissus mous, avec un pic d’incidence chez l’adolescent et le jeune adulte. Trente pour cent des patients sont métastatiques au diagnostic et environ 20 % d’entre eux présentent une atteinte ostéomédullaire et/ou osseuse. En l’absence de métastases, la survie globale à 5 ans est de 65 à 75 %. Elle diminue à 50 % en cas d’atteinte pulmonaire isolée et chute à moins de 25 % en cas d’atteinte ostéomédullaire. Actuellement, le myélogramme et la biopsie ostéomédullaire constituent le gold standard pour le diagnostic de l’atteinte ostéomédullaire, mais ce sont deux techniques invasives. La TEP-TDM FDG est plus sensible que la TDM seule et que l’IRM conventionnelle pour la détection des métastases osseuses et extra-osseuses, incluant l’atteinte ostéomédullaire. Les recommandations récentes de l’ESMO rapportent que le myélogramme n’est pas nécessaire dans le bilan d’extension initial des sarcomes localisés ayant une TEP-TDM FDG négative. Cependant, plusieurs études et méta-analyses présentent des résultats contradictoires quant à la corrélation entre la TEP-TDM FDG et les prélèvements ostéomédullaires. Ces études sont hétérogènes, et de faibles effectifs. Nous présentons les résultats d’une étude rétrospective monocentrique ayant analysé les dossiers de 180 patients pour lesquels un sarcome d’Ewing a été diagnostiqué entre janvier 2010 et janvier 2020 au Centre Léon Bérard et ayant bénéficié à la fois d’une TEP-TDM FDG et de prélèvements ostéomédullaires (myélogramme et/ou biopsie) au diagnostic. Une relecture centralisée et en aveugle des TEP-TDM FDG positives ou douteuses a été réalisée par un médecin nucléaire expérimenté. Cette étude représente la plus grosse cohorte de patients avec sarcome d’Ewing analysée avec un objectif de comparer les modalités du bilan d’extension initial ostéomédullaire. Sur les 180 patients, 13 patients avaient un prélèvement de moelle positif, parmi lesquels 12 avaient une TEP positive pour une atteinte ostéomédullaire. Parmi les 167 patients avec prélèvement de moelle négatif, 166 avaient une TEP négative pour une atteinte ostéomédullaire. La sensibilité et la spécificité de la TEP FDG dans cet objectif sont de 92,3 % et 99,4 %. Compte tenu des résultats de cette étude et de la littérature actualisée disponible, le GSF-GETO/GROUPOS suggère de ne plus réaliser de prélèvement de moelle osseuse de façon systématique au diagnostic initial de sarcome d’Ewing lorsqu’une TEP-TDM FDG est réalisée. Un algorithme décisionnel est proposé : le prélèvement de moelle osseuse n’est plus nécessaire en cas d’aspect scintigraphique caractéristique d’atteinte ostéomédullaire, de sarcome d’Ewing localisé, d’atteinte secondaire pulmonaire ou d’atteinte oligométastatique osseuse focale (moins de 5 lésions). En cas d’atteinte secondaire extra-pulmonaire ou osseuse plurifocale, une IRM du squelette axial et du pelvis est recommandée.

BACKGROUND:Osteosarcoma stratification relies on clinical parameters and histological response. We developed a new personalized stratification using less invasive circulating tumor DNA (ctDNA) quantification. PATIENTS AND METHODS:Plasma from patients homogeneously treated in the prospective protocol OS2006, at diagnosis, before surgery and end of treatment, were sequenced using low-passage whole-genome sequencing (lpWGS) for copy number alteration detection. We developed a prediction tool including ctDNA quantification and known clinical parameters to estimate patients' individual risk of event. RESULTS:ctDNA quantification at diagnosis (diagCPA) was evaluated for 183 patients of the protocol OS2006. diagCPA as a continuous variable was a major prognostic factor, independent of other clinical parameters, including metastatic status [diagCPA hazard ratio (HR) = 3.5, P = 0.002 and 3.51, P = 0.012, for progression-free survival (PFS) and overall survival (OS)]. At the time of surgery and until the end of treatment, diagCPA was also a major prognostic factor independent of histological response (diagCPA HR = 9.2, P < 0.001 and 11.6, P < 0.001, for PFS and OS). Therefore, the addition of diagCPA to metastatic status at diagnosis or poor histological response after surgery improved the prognostic stratification of patients with osteosarcoma. We developed the prediction tool PRONOS to generate individual risk estimations, showing great performance ctDNA quantification at the time of surgery and the end of treatment still required improvement to overcome the low sensitivity of lpWGS and to enable the follow-up of disease progression. CONCLUSIONS:The addition of ctDNA quantification to known risk factors improves the estimation of prognosis calculated by our prediction tool PRONOS. To confirm its value, an external validation in the Sarcoma 13 trial is underway.

Sclerosing Epithelioid Fibrosarcoma (SEF) and Low Grade Fibromyxoid Sarcoma (LGFMS) are ultrarare sarcomas sharing common translocations whose natural history are not well known. We report on the nationwide exhaustive series of 330 patients with SEF or LGFMS in NETSARC+ since 2010. PATIENTS AND METHODS:NETSARC (netsarc.org) is a network of 26 reference sarcoma centers with specialized multidisciplinary tumor boards (MDTB). Since 2010, (i) pathological review has been mandatory for sarcoma,and (ii) tumour/patients' characteristics have been collected in the NETSARC+ nationwide database. The characteristics of patients with SEF and LGFMS and their outcome are compared. RESULTS:35/73 (48%) and 125/257(49%) of patients with SEF and LGFMS were female. More visceral, bone and trunk primary sites were observed in SEF (p < 0.001). 30% of SEF vs 4% of LGFMS patients had metastasis at diagnosis (p < 0.0001). Median size of the primary tumor was 51 mm (range 10-90) for LGFMS vs 80 (20-320) for SEF (p < 0.001). Median age for LGFMS patients was 12 years younger than that of SEF patients (43 [range 4-98] vs 55 [range 10-91], p < 0.001). Neoadjuvant treatment was more often given to SEF (16% vs 9%, p = 0.05). More patients with LGFMS were operated first in reference centers (51% vs 26%, p < 0.001). The R0 rate on the operative specimen was 41% in LGFMS vs 16% in SEF (p < 0.001). Median event-free survival (EFS) of patients with SEF and LGFMS were 32 vs 136 months (p < 0.0001). The median overall survival (OS) was not reached. Fifty-months OS was 93% vs 81% for LGFMS vs SEF (p = 0.05). Median OS was 77 months after first relapse, similar for SEF and LGFMS. In multivariate analysis, age, tumor size, metastasis at diagnosis were independent prognostic factors for OS in LGFMS. CONCLUSIONS:Although sharing close molecular alterations, SEF and LGFMS have a different natural history, clinical presentation and outcome, with a higher risk of metastatic relapse in SEF. Survival after relapse is longer than with other sarcomas, and similar for SEF and LGFMS.

Background: The prognosis of relapsed and unresectable Ewing sarcoma and osteosarcoma is dismal and unchanged over the last decades. Management of patients is based on the used of various cytotoxic regimens. However, pharmacologic inhibition of Met signaling and of aberrant angiogenesis has shown promising results in several preclinical models of Ewing sarcoma and osteosarcoma. This study aims to investigate the activity of the MET/VEGFR2 inhibitor, cabozantinib in patients with advanced Ewing and osteosarcoma. Methods: These are two multi-centre single-arm two-stage phase 2 trials assessing the efficacy and safety of cabozantinib in patients with advanced Ewing sarcoma or osteosarcoma. Main eligibility criteria included: age ≥ 12 years, ECOG Performance status ≤ 1, metastatic or unresectable locally advanced disease and documented disease progression (as per RECIST v1.1) before study entry. The number of previous lines of treatment was not limited. Patients received cabozantinib (oral route; adults: 60 mg, children: 40 mg/m 2 ), daily until progressive disease or unacceptable toxicity. The primary endpoint was objective response for Ewing sarcoma and a dual one based on a 6-month objective response and non-progression of osteosarcoma. Findings: From April 16 2015 to July 12 2018, 90 patients were recruited (Ewing sarcoma: 45; Osteosarcoma: 45). Median follow-up was 31.3 months (95%CI: [12.4–35.4]) and 31.1 months (95%CI: [24.4–31.7]), for Ewing sarcomas and osteosarcomas, respectively. Thirty-nine (86.7%) Ewing sarcoma and 42 (93.3%) osteosarcoma were assessable for efficacy after histological and radiological review. Seven patients with osteosarcoma (16.7%) had partial response and 14 (33.3%) had stable disease. Ten patients with Ewing sarcoma (25.6%) had partial response and 15 (38.4%) had stable disease. Fourteen osteosarcoma patients (33.3%) and 10 Ewing sarcoma patients (25.6%) were progression-free at six months. Therapy was well tolerated, although grade 1 or grade 2 fatigue, diarrhea, mucositis and were common. The most common grade 3 or 4 adverse events were hypophosphatemia (n=8, 8.9%), aspartate aminotransferase increase (n=5,5.6%), syndrome (n=5, 5.6%), pneumothorax (=5, 5.6%), neutropenia (n=5, 5.6%). At least one serious adverse event was reported in 61 patients (67.8%). Clinical Trial Interpretation: In this study, cabozantinib showed marked antitumor activity in patients with advanced Ewing sarcoma and osteosarcoma and may represent a new therapeutic option in this setting.

Abstract Background/Objectives About 1,000,000 new cases of cancer in Adolescent and Young Adults (AYAs) are diagnosed annually worldwide. . While their long term survival is about 80%, they are six times more likely to develop a second primary cancer (SPC) compared to their peers. This risk is multifactorial and depends on the type of first cancer, treatment received and prevalence of risk factors. PREVAPAJA aimed to implement a clinical program based on physical activity (PA) and cancer prevention recommendations for AYAs with cancer at Centre Léon Bérard-AYAs Department. Methods The study was conducted at Leon Berard Comprehensive Cancer Centre among patients aged 15-25 years. AYAs attended PA sessions during the active treatment period and were individually informed on SPC risk prevention. PA, sedentary, anthropometrics, quality of life and fatigue were assessed at baseline (T1) and at the end of treatment (T2). PA level and intention of changes in health behaviors were assessed by phone 1 year after T1. Results 68 AYAs (median age=19 years) were enrolled in 2016-2017). The results showed an improvement in PA level during and at distance of the intervention, with also a reduction of sitting time. Fatigue decreased between T1 and T2 (p>0.003) and overall quality of life improved significantly between T1 and T2 (p>0.001). Conclusions This study showed the feasibility of implementing a clinical program based on PA intervention and cancer prevention recommendations for AYAs with cancer. It responded to AYAs' needs for support and discussions regarding PA recommendations and ways to prevent SPC. Beneficial outcomes of this program should encourage to systematically proposing PA intervention in combination with information exchanges with AYAs with cancer.

CIC-DUX4 sarcoma (CDS) represent the second most prevalent subset of high grade round cell sarcomas after Ewing sarcomas. The natural history might be diverse and probably underscores a heterogeneous group. Moreover, treatment strategy can differ according to institutions. The main goals of this retrospective study was to describe characteristics, treatments and outcome for patients with CDS included in the French NETSARC database.

Introduction: Survival of adolescents and young adults (AYA) with sarcoma is lower than in younger patients. The objective of this study was to describe the regional healthcare circuits, the differences in the management between adult, paediatric and mixed units and to assess the prognostic impact of compliance with clinical practice guidelines (CPGs) on overall survival (OS) and on relapse free survival (RFS). Materials and methods: Retrospective analysis of the management and long term follow-up of all 13-25 year old patients with a sarcoma diagnosed in the Rhone-Alpes area between 2000 and 2005. Results: 140 patients satisfied inclusion criteria and were selected. The majority of 13-25 year old patients were treated in paediatric units. Joint management resulted in a higher rate of discussion in multidisciplinary tumour board, inclusion in clinical trials, and fertility preservation. Non-compliance with guidelines was observed in 65% of cases. Overall compliance was not reported to correlate to survival. Compliance of radiotherapy with CPG's seemed associated with a better prognosis for OS (HR = 0.20, 95% CI = [0.10-0.40]; p < 0.0001) and RFS (HR = 0.18, 95% CI = [0.09-0.37; p < 0.0001) as well as compliance of surgery for OS (HR = 0.43, 95% CI = [0.23-0.81]; p = 0.01). Multivariate Cox regression analysis revealed other independent predictors of OS like age at diagnosis, stage and histological subtype. Conclusions: Management of AYA in joint units seems to improve the quality of care. Compliance of surgery and radiotherapy with CGP's seems to improve survival. (C) 2020 Elsevier Ltd, BASO - The Association for Cancer Surgery, and the European Society of Surgical Oncology. All rights reserved.

Desmoid type fibromatosis (DTF) are rare intermediate malignancies with unpredictable becoming. Guidelines recommend active surveillance (AS) first if few symptoms are present. The aim of this retrospective multicentric study is to describe the French population of patients <40 years with DTF registered in 2006-2017 in netSARC and CONTICABASE databases. Of 1526 patients registered, 964 patients were selected (median age (MA) 31 years, 83 children, F/M: 3.3). APC constitutional pathogenic variant was described in 4.6%. Primary tumor sites were abdominal wall (41%), trunk (23%), extremity (19%), mesentery (14%) and head and neck (HN) (3%); Median size was 55mm. MA was higher in patients with abdominal wall and mesentery sites (32 and 31.5 years) than in those with HN site (21 years, p=0.0001). Patients with familial adenomatous polyposis were 3 times more frequent in multifocal DTF group, and in mesenteric primary (p<0.001). Women of childbearing age were more frequent in the group "abdominal wall" than in the other groups (99.5% vs 96.8%, p=0.007) without increase of DTF diagnosis during pregnancy. First line strategy was known for 487 patients: AS (n=189, MA 31 years; treatment needed in a second time in 77 of them) versus front line treatment (n=298, MA 30 years), including most often surgery (280 patients, MA 31 years) or non surgical therapies (systemic treatment n=14, or cryotherapy n=4) (18 patients, MA 15 years, p<0.0001). With a median follow-up of 17 months [0; 142 months], the 1-year progression free survival (PFS) rate was 83.2% [80.2%-85.8%]. In univariate analysis, PFS was worse for young patients, male, tumor≥5cm, extremity and trunk sites, and APC constitutional pathogenic variant status. In multivariate analysis, size ≥5cm (HR=1.447, p=0.0923) and extremity primary (HR=1.6, p=0.0113) remained associated with worse PFS. Among the 487 informative patients, AS was associated with a better PFS (HR=0.694, p=0.0848). This series of patients identifies age, gender and constitutional related characteristics. AS seems to be a valid first line option. Such databases improve knowledge about risk factors for DTF and highlight the patients requiring care in expert centers.

PURPOSE:There are some lines of evidence suggesting a potential role of immunotherapy for treating patients with osteosarcomas. PATIENTS AND METHODS:This was an open-label, multicentre, phase 2 study of pembrolizumab in combination with metronomic cyclophosphamide in patients with advanced osteosarcomas. All patients received 50 mg b.i.d. of cyclophosphamide one week on and one week off and 200 mg of intravenous pembrolizumab (every 3 weeks). There was a dual primary end-point, encompassing both the non-progression and objective responses at 6 months per Response Evaluation Criteria in Solid Tumours (RECIST), version 1.1. An objective response rate of 20% and/or a 6-month non-progression rate of 60% were determined as reasonable objectives for treatment with meaningful effect. Correlative studies of immune biomarkers were planned from the patients' tumour samples. RESULTS:Between October 13 2015 and July 3 2017, 17 patients were included. Fifty were assessable for the efficacy end-point. Four patients experienced tumour shrinkage, resulting in a partial response (PR) in one patient (6.7%). The 6-month non-progression rate was 13.3% (95% confidence interval [CI]: 1.7-40.5). The most frequent adverse events were grade I or II nausea, anaemia, anorexia and fatigue. programmed death-ligand 1 (PD-L1) expression rate was low, observed in only 2 cases of 14 with available tumour material. The only patient who experienced PR had a PD-L1-negative tumour. CONCLUSION:Programmed cell death 1 (PD-1) inhibition has limited activity in osteosarcomas. Further studies investigating PD-1 inhibitor in combination with agents modulating the microenvironment are warranted. TRIAL REGISTRATION:This study is registered with ClinicalTrials.gov, number NCT02406781.