BackgroundThe objective of this study is to explore the off-label use of targeted therapies (TTs) for patients with osteosarcoma registered within the French Sarcoma Group – Bone Tumor Study Group (GSF-GETO) national registry.MethodsAll patients with an osteosarcoma, registered between January 1, 2009 and July 15, 2013 were analyzed.ResultsTwenty-nine patients with refractory relapsed osteosarcomas received 33 treatment lines of TTs. The median age at the beginning of treatment was 19 years (range 9–72). The median number of previous lines of chemotherapy was 3 (range 1–8). Before inclusion, 3 patients were in second complete remission, 26 were in progression for metastatic relapse. Twenty-three patients received sirolimus (in combination with cyclophosphamide for 18); 5, sunitinib; 4, sorafenib; and one, pazopanib. Stable disease was observed for 45.5 % of patients (95 % Confidence Interval (CI) [20–52.8]). The median Progression-Free Survival (PFS) was 3 months (95 % CI [2–5.4]) for patients treated by sirolimus and 1.8 months (95 % CI [1.3–2.8]) for patients receiving multi-targeted tyrosine kinase inhibitors; 6-month PFS 15 %. The median Overall Survival (OS) was 6.8 months (95 % CI [4.7–12.1]), and one-year OS was 24 %. In a multivariate analysis, PFS was superior for patients receiving sirolimus compared to other TTs (Hazard Ratio (HR) = 2.7, 95 % CI [1.05–7.1]). No toxic death was reported. Grade 3 and 4 toxicities were observed in 27 and 6 % of cases respectively.ConclusionOff-label TTs, especially sirolimus, reported benefit in the treatment of refractory osteosarcomas with an acceptable toxicity profile, including in pediatric population.

BackgroundDesmoplastic small round cell tumor (DSRCT) is a very rare mesenchymal tumor that mainly affects teenagers and young adults with a mean age at diagnosis around 20–25 years. Although initial management still needs standardization, many centers will use multimodal treatment including intensive chemotherapy, extensive surgical resection followed by radiotherapy. Despite this, prognosis remains very poor and the median overall survival is 25 months. Recurrent disease is mainly treated by chemotherapy. Recently, due to the unmet medical need for recurrent disease, targeted therapies were explored for DSRCT.MethodsIn this study, we assessed the response rate and progression free survival in nine cases of progressive DSRCT included in the OUTC’s registry and treated with antiangiogenics targeted agents (sunitinib, sorafenib and bevacizumab). OUTC’s, a French national registry, collects data about the use of off-label targeted therapy in sarcoma.ResultsEight males and one woman were included, with median age at diagnosis of 27.3 years (range from 9 to 48 years). They received a mean 3 lines (2–5) of treatment before antiangiogenic agent initiation. Six patients received sunitinib, two received sorafenib and one bevacizumab. Median progression free survival was 3.1 months (range 2–5.5 months) and best response observed was 5.5 months stable disease. Most patients had manageable low-grade toxicities, mainly fatigue, abdominal pain and skin toxicity.ConclusionsDespite very limited activity of antiangiogenics in our study, prospective collection of cases of these rare tumors together with molecular data should guide therapeutic decision and enhance outcome.Electronic supplementary materialThe online version of this article (doi:10.1186/s13569-017-0076-4) contains supplementary material, which is available to authorized users.

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Objective: In the COVID-19 crisis context, the main objective of the study is to investigate factors associated with perceived concerns of change in long-term cancer care in patients currently under treatment. Methods: A French population-based cross-sectional study was performed using an online questionnaire in April 2020. All persons currently receiving cancer treatment and belonging to the Seintinelles Association (https://www.seintinelles.com) were included in this present analysis. Individual sociodemographic characteristics, medical status and information regarding cancer care were collected. Multivariate binomial logistic regression analysis was performed. Results: We included 298 women in the analysis. Younger participants (OR = 0.96 [0.94-0.99]), the need to visit healthcare facilities to receive treatment (OR = 2.93 [1.16-8.52]), deterioration in the quality of communication with the medical team since the beginning of the COVID-19 crisis (OR = 3.24 [1.61-7.02]) and being cared for by a university hospital or a public hospital (OR = 2.19 [1.16-4.23] versus comprehensive cancer centre) were associated with a perceived fear of change in long-term cancer care. Conclusion: To address patients' concerns regarding changes in their long-term cancer care, medical teams should consider the patients' own perceptions of the situation and provide clear, appropriate, precise information on cancer care, especially in the centres mostly affected by the COVID-19 crisis.

Targeted therapies (TT) are used in pediatric patients based on the data from adult literature. In 2008, the French Sarcoma Study Group and the Bone Tumor Group (Groupe Sarcome Fran?ais-Groupe d'?tude des Tumeurs Osseuses) opened Observatoire de l'Utilisation des Th?rapies Cibl?es dans les Sarcomes (OUTC'S), a national registry of targeted off-label sarcomas therapies. All patients registered in the OUTC'S database and treated in pediatric oncology units were included in this analysis. We describe TTs using off-label and off clinical trial practices for children with sarcoma. We analyzed TT tolerability and efficacy for 34 patients with osteosarcoma (n?=?20), Ewing sarcoma (n?=?9), clear cell sarcoma (n?=?1), synovialsarcoma (n?=?1), epithelioid sarcoma (n?=?1), myofibroblastic tumor (n?=?1), and desmoid tumor (n?=?1) who were registered from six pediatric centers. In total, 38 different TT courses were administered. The median age was 15 years (5?23) and 18.5 years (7?27) at diagnosis and at the time of starting TT, respectively. The decision to initiate TT was taken in a multidisciplinary board in 92% of the cases. TT included sirolimus (alone or in association with other treatments), sunitinib, sorafenib, cetuximab, imatinib, and crizotinib. The median duration of treatment was 109 days (21?515). Of the 34 patients, 6 had a partial response with a median response duration of 3.72 months (2.08?30.8) and 10 had a stabilization of the disease with a median duration of 3.63 months (0.75- 16.89). In our cohort, overall survival and progression-free survival were 8.68 months (95% confidence interval [CI]: 5.85?11.50) and 3.29 months (95% CI: 2.69?3.88), respectively. Grades 3 and 4 toxicities were reported for seven patients (26%) and were most commonly hematological. Patients under 15 years of age did not show severe toxicity. Hence, TT is an acceptable therapeutic option for refractory pediatric sarcomas. It is very important to continue collecting data and develop phase I/II protocols.

Findings support the need for guidelines that are sensitive to sociocultural context and promote autonomous decision-making.

Background and Aims: During the first lockdown of the coronavirus disease 2019 (COVID-19) pandemic, we developed a teleconsultation model and 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. Materials and Methods: This is a prospective monocentric exploratory study based on synchronous, video consultations between HWs and pediatric patients treated for blood or solid malignancies or blood benign diseases. Every HW completed an online survey which covered technical aspects, data concerning satisfaction, quality of the interaction with the patient, and emotions felt after the teleconsultation. A score was calculated for each aspect. 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 to 10), a global feeling score of 8.79 (3.33 to 10.00), and a quality score of 7.34 (2.50 to 10.00). A strong correlation between the quality of 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.