Florence Duffaud's research while affiliated with Aix-Marseille Université and other places

Publications (181)

Article
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Background The initial management of patients with sarcoma is a critical issue. We used the nationwide French National Cancer Institute-funded prospective sarcoma database NETSARC to report the management and oncologic outcomes in adolescents and young adults (AYAs) patients with sarcoma at the national level. Patients and methods NETSARC database...
Article
Cancer genetics has to date focused on epithelial malignancies, identifying multiple histotype-specific pathways underlying cancer susceptibility. Sarcomas are rare malignancies predominantly derived from embryonic mesoderm. To identify pathways specific to mesenchymal cancers, we performed whole-genome germline sequencing on 1644 sporadic cases an...
Article
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In leiomyosarcoma (LMS), a very aggressive disease, a relatively transcriptionally uniform subgroup of well-differentiated tumors has been described and is associated with poor survival. The question raised how differentiation and tumor progression, two apparently antagonist processes, coexist and allow tumor malignancy. We first identified the mos...
Article
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Background CIC-rearranged sarcomas (CIC-RS) represent the most frequent subset of “Ewing-like” undifferentiated small round cell sarcomas. These tumors tend to be more aggressive than Ewing sarcomas. Moreover, treatment strategy can differ according to teams. The primary aim of this retrospective study was to describe the characteristics, treatment...
Article
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Plain language summary Sarcomas are a type of cancer that start from the bones or soft tissues (such as muscle, blood vessel, or fat tissue) anywhere in the body. Sarcomas are rare – only about 1% of all cancers are sarcomas, and there are many different types. Sarcomas occur more often in children than in adults. People with sarcomas which cannot...
Article
Background Diffuse-type tenosynovial giant cell tumour (D-TGCT) is a non-malignant but locally aggressive tumour driven by overexpression of colony-stimulating factor-1 (CSF1). CSF1R inhibitors are potential therapeutic strategies for patients not amenable to surgery. We report here the long-term outcome of nilotinib in patients with advanced D-TGC...
Article
Background: The incidence of different soft tissue sarcoma (STS) histotypes among ethnic and geographic populations has not been comprehensively investigated. Methods: Data from 2013 to 2016 were obtained from national cancer registry databases in France and Taiwan. Liposarcoma (LPS), leiomyosarcoma (LMS), angiosarcoma (AS), synovial sarcoma (SS...
Article
Background Metastatic leiomyosarcomas have a poor prognosis, and currently doxorubicin alone is used as the standard first-line treatment. Doxorubicin combined with trabectedin has shown promising results in phase 1 and 2 studies. We aimed to identify and compare the progression-free survival of patients with metastatic or unresectable uterine or s...
Article
Objectives: To explore cancer patients' experience of pain and coping strategies as well as beliefs and representations associated with pain, pain management and treatments. Method and participants: A qualitative study based on semi-structured interviews was conducted among 16 patients with cancer. The inductive research strategy adopted derived...
Article
11571 Background: Perivascular Epithelioid Cell Neoplasms (PEComas) encompass a heterogeneous family of mesenchymal tumors. The current understanding of their natural history is limited. Previously described clinicopathological factors aimed to define benign or malignant variants, but there is a lack of prognostic factors associated with recurrence...
Article
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The national reference network NETSARC+ provides remote access to specialized diagnosis and the Multidisciplinary Tumour Board (MTB) to improve the management and survival of sarcoma patients in France. The IGéAS research program aims to assess the potential of this innovative organization to address geographical inequalities in cancer management....
Preprint
Background . The initial management of patients with sarcoma is critical. We used the French National Cancer Institute-funded nationwide sarcoma database NETSARC to report the management and oncologic outcomes in children, adolescents and young adults (AYAs) patients with sarcoma at the national level. Patients and methods . Data from patients with...
Article
Purpose: The COVID-19 vaccination campaign began in December 2020, in France, and primarily targeted the oldest people. Our study aimed to determine the level of acceptance of vaccination in a population of older patients with cancer. Methods: From January 2021, we offered vaccination with the BNT162b2 COVID-19 vaccine to all patients 70 years and...
Article
PURPOSE Antitumor activity in preclinical models and a phase I study of patients with dedifferentiated liposarcoma (DD-LPS) was observed with selinexor. We evaluated the clinical benefit of selinexor in patients with previously treated DD-LPS whose sarcoma progressed on approved agents. METHODS SEAL was a phase II-III, multicenter, randomized, dou...
Article
The benefit of chemotherapy (CT) in rare bone sarcomas is not documented in prospective studies. This retrospective study from the French sarcoma network for bone tumors ResOs was performed in adult patients (pts) from 1976 to 2014, with histologically verified diagnosis of leiomyosarcomas (LMS), undifferentiated pleomorphic (UPS), or radiation-ass...
Article
Treatment options for metastatic osteosarcomas are scarce. Following failure of standard first line therapy, patients who relapse present a challenging treatment dilemma, and have a poor prognosis. Surgical removal of all metastases is essential. A retrospective analysis of patients with metastatic osteosarcomas was conducted in 15 French Sarcoma G...
Article
Background: Functional Status (FS) is an important domain in Comprehensive Geriatric Assessment (CGA) and is most often evaluated using the Activities of Daily Living (ADL) and Instrumental Activities of Daily Living (IADL) scales separately. Method and objectives: This secondary analysis of a previous prospective cohort study was conducted betw...
Article
Background Retrospective analyses suggest that patients with advanced KIT exon 9-mutated gastrointestinal stromal tumors (GISTs) receiving imatinib 800 mg (rather than 400 mg) daily have better outcomes. In the adjuvant setting, the question of the optimal dose of imatinib remains unsettled.Objective We aimed to retrospectively assess the activity...
Article
Background This multi-cohort trial explored the efficacy and safety of regorafenib for patients with advanced sarcomas of bone origin; this report details the cohort of patients with metastatic or locally advanced chondrosarcoma (CS), progressing after prior chemotherapy. Patients and methods Patients with CS, progressing despite prior standard th...
Article
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Background Spatial inequalities in cancer management have been evidenced by studies reporting lower quality of care or/and lower survival for patients living in remote or socially deprived areas. NETSARC+ is a national reference network implemented to improve the outcome of sarcoma patients in France since 2010, providing remote access to specializ...
Article
TPS11576 Background: Pts with OS and non-OS of bone are treated with a multimodal sequence therapy of neoadjuvant chemotherapy (CT), surgery and adjuvant CT, followed by a close surveillance until recurrence. At recurrence, the prognosis remains poor with objective response rates of 3-29%, and a median Progression-Free Survival (PFS) of less than 4...
Article
e23537 Background: R0 surgery in reference centers is the cornerstone for sarcomas treatment with better local control and survival and is mandatory for ESMO-EURACAN and NCCN guidelines. The medical community is out on this issue for cutaneous sarcomas, if they have to follow suit. This retrospective descriptive study focused on margin status and l...
Article
Background Desmoid tumours (DTs) are rare tumours originating from musculoaponeurotic structures. Although benign, they may be locally aggressive, leading to pain and disability. European Society for Medical Oncology (ESMO) guidelines recommend frontline watchful waiting and medical treatment in progressing tumours. Cryoablation is an interventiona...
Preprint
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Based on the transcriptome analysis of 555 sarcomas, we identified a group of tightly clustered leiomyosarcomas (LMS) due to their gene expression homogeneity. We named them “hLMS” and the other LMS “oLMS”. We derived a transcriptional signature able to identify each group and used it to classify patients from two independent cohorts. In all cohort...
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Background: Polymorbidity induces polypharmacy in older patients may lead to potential drug-drug interactions (DDI) which can modify the tolerance and safety of oncological treatments and alter the intended therapeutic effect. The objective of our study was to describe the decision-making process for oncological treatment and related outcomes, in...
Article
Background: A Comprehensive Geriatric Assessment (CGA) has been proposed to assess prognosis and to adapt oncological care in older cancer patients. However, few biological markers are incorporated in the CGA. Methods: This comparative study on older cancer patients was realized before final therapeutic decision and during a CGA including biolog...
Article
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Opinion statement: Osteosarcomas (OS) belong to a large family of mesenchymal tumor entities which exhibit heterogenous histological, genetic, and molecular features. Current OS treatment regimen consists of the combination of surgery and intensive multi-agent chemotherapy. Ever since the introduction of chemotherapy, 5-year survival rate among OS...
Article
11506 Background: U-LMS and ST-LMS are rare tumors with poor prognosis when locally advanced or metastatic, and with moderate chemosensitivity. Overall response rates (ORR) given in the 1 st -line setting do not exceed 50% for U-LMS and 35% for ST-LMS with a mean response duration of 3- 6 months without impact on overall survival (OS). In 2015 we r...
Article
e16643 Background: Isocitrate dehydrogenase 1 mutations (mIDH1) are present in a variety of solid tumors resulting in production and accumulation of (R)-2-hydroxyglutarate causing DNA hypermethylation and promoting tumorigenesis. Olutasidenib is an oral, potent and selective inhibitor of mutated IDH1 protein. We report preliminary results from the...
Article
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Importance The association between quality of surgery and overall survival in patients affected by localized gastrointestinal stromal tumors (GIST) is not completely understood. Objective To assess the risk of death with and without imatinib according to microscopic margins status (R0/R1) using data from a randomized study on adjuvant imatinib. D...
Article
Background: The optimal treatment for advanced leiomyosarcoma is still debated. Given histotype-specific prospective controlled data lacking, this study retrospectively evaluated doxorubicin plus dacarbazine, doxorubicin plus ifosfamide, and doxorubicin alone as first-line treatments for advanced/metastatic leiomyosarcoma treated at European Organ...
Article
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Soft tissue sarcoma (STS) are rare and aggressive tumours. Their classification includes numerous histological subtypes of frequent poor prognosis. Liposarcomas (LPS) are the most frequent type among them, and the aggressiveness and deep localization of dedifferentiated LPS are linked to high levels of recurrence. Current treatments available today...
Article
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Background: Brain metastases from sarcomatous lesions pose a management challenge owing to their rarity and the histopathological heterogeneity. Prognostic indices such as the Graded Prognostic Assessment (GPA) index have been developed for several primary tumour types presenting with brain metastases (e.g. lung, breast, melanoma), tailored to the...
Article
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Background: Patients with Ewing sarcoma or osteosarcoma have a median overall survival of less than 12 months after diagnosis, and a standard treatment strategy has not yet been established. Pharmacological inhibition of MET signalling and aberrant angiogenesis has shown promising results in several preclinical models of Ewing sarcoma and osteosar...
Article
Aims : Patients suffering from cancer are often confronted with an awareness of their own finiteness and with the fact that their life is now ruled by the cancer. This existential upheaval can be accompanied by spiritual “work” which contributes to the adjustment to the disease and to the conditions of the end of life. Spirituality can be defined a...
Article
Background Metastatic phyllodes tumors have poor prognosis with median overall survival of 11.5 months. The objective of this study is to identify prognostic factors and the best options for management of metastatic malignant phyllode tumors (MMPTs). Patients and Methods A multicentric retrospective study, including cases of MMPT from 10 sarcoma c...
Article
Background: Intimal sarcoma (InS) is an exceedingly rare neoplasm with an unfavorable prognosis, for which new potentially active treatments are under development. We report on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in patients with InS. Methods: Seventeen sarcoma reference centers in Europe, the...
Article
In the OS2006 study, patients younger than 18 years were treated with a methotrexate‐based regimen (MTX), patients older than 25 years with a doxorubicin‐cisplatin‐ifosfamide–based regimen (API‐AI), whereas patients aged 18‐25 years received either API‐AI or MTX. We herein report the prespecified subgroup analysis of the outcome of 106 patients tre...
Article
11030 Background: Treatment (trt) options for metastatic OST are scarce. Following failure of standard 1st line therapy pts who relapse present a challenging trt dilemma, and have poor prognosis. Surgical removal of all mets is essential. Currently, there are no standardized 2nd line trt options in relapsed OST. Methods: Pts were identified from 2...
Article
TPS11078 Background: We have recently identified a gene expression signature so called “CINSARC” which is related with chromosomal instability and highly predictive of metastasis-free and overall survival in soft-tissue sarcoma (STS) patients. The prognostic relevance of this signature has been validated recently in an independent set of sarcomas f...
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Purpose: CDKN2A loss is frequent in gastrointestinal stromal tumors (GISTs) and associated with aggressive outcome. Palbociclib is a CDK4 inhibitor with preclinical antitumor efficacy in tumors with P16/CDKN2A loss. Patients and methods: This is a multicenter single-arm phase II clinical trial assessing safety and efficacy of palbociclib in pati...
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Soft tissue sarcomas (STS) are rare tumors accounting for less than 1% of human cancers. While the highest incidence of sarcomas is observed in elderly, this population is often excluded or poorly represented in clinical trials. The present study reports on clinico‐pathological presentation, and outcome of sarcoma patients over 90 recorded in the N...
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Introduction: Based on a mathematical model of trabectedin-induced neutropenia, we assessed the predictive value of absolute neutrophil count (ANC) on progression-free survival (PFS) in an independent validation cohort of patients treated with trabectedin. Methods: We collected data from 87 patients in two expert centers who received at least two c...
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Background: There is an unmet need for markers predicting the outcome of patients with advanced soft tissue sarcoma (STS) treated with pazopanib. Since toxicity might be related to the anti-tumor activity of the drug, the aim of this study was to determine whether pazopanib-induced proteinuria, hypothyroidism and cardiotoxicity grade 3-4 were assoc...
Article
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Renal collecting duct carcinoma (CDC) is a rare and highly aggressive subtype of kidney cancer with poor prognosis. We report a case of one patient, who was successfully treated with gemcitabine-platin based chemotherapy for polymetastatic renal CDC, and experienced a late and prolonged complete remission. In June 2014, a 69-year-old male patient w...
Conference Paper
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BODY: Objective: There is a strong genetic component to sarcomas and they are more common in hereditary cancer syndromes. The most recognised genetic drivers are germline mutations in theTP53 gene (Li Fraumeni syndrome) but other risk genes include NF1/2, Rb, SDH genes and BRCA2. A polygenic contribution to sarcoma risk has also been recognised. We...
Article
Aims: Gemcitabine has been associated with thrombotic microangiopathy (TMA). We conducted a national retrospective study of Gemcitabine-associated TMA (G-TMA). Methods: From 1998 to 2015, all cases of G-TMA reported to the French Pharmacovigilance Network and the French TMA Reference Center, and cases explored for complement alternative pathway...
Article
Background: Regorafenib has proven activity in patients with pretreated gastrointestinal stromal tumours and colorectal and hepatocellular carcinoma. We designed REGOBONE to assess the efficacy and safety of regorafenib for patients with progressive metastatic osteosarcoma and other bone sarcomas. This trial comprised four parallel independent coh...
Article
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Background Alternative endpoints to overall survival (OS) are frequently used to assess treatment efficacy in randomized controlled trials (RCT). Their properties in terms of surrogate outcomes for OS need to be assessed. We evaluated the surrogate properties of progression-free survival (PFS), time-to-progression (TTP) and time-to-treatment failur...
Article
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Background Gastrointestinal stromal tumors (GISTs) are rare, but represent the most common mesenchymal neoplasms of the gastrointestinal tract. EPIdemiology GIST, is an observational multicenter longitudinal follow-up cohort study reporting the prescribing patterns of imatinib in patients with GIST and the impact of the treatment in a real-world (s...
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Background Opinion about euthanasia has been explored among the general population and recently in patients receiving palliative care. 96% of the French population declared themselves in favor of euthanasia while less of 50% of palliative care patients are. The aim of the present study was to explore and identify potential determinant factors assoc...
Article
Sarcomas are a common type of tumor within the pediatric population. The utilization of proton therapy as a primary attribute the ability to spare adjacent healthy tissue, therefore, proton therapy has become a preferential indication in pediatrics compared to other photon irradiation modalities. Proton therapy is also a proven and historically val...
Article
Upfront screening for dihydropyrimidine dehydrogenase (DPD) deficiency in patients scheduled for 5-FU-based protocols should reduce the risk of treatment-related toxicities by preventive adaptive dosing. Our group has developed a simple, rapid and cheap functional testing that helps categorizing patients on their DPD status and detecting poor metab...
Article
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Five‐year overall survival (OS) of localized RMS exceeds 70% in children (<18) but is very poor in adult patients. We analyzed the outcome and prognostic factors (PF) of a national series of adult patients with RMS in a large study. The study population consisted of two different cohorts: a retrospective cohort (157 adult patients treated in 13 ref...
Article
Purpose: The objective of this study was to describe the outcome and prognostic factors for adults treated for localized myxofibrosarcoma. Methods and materials: We conducted a retrospective multicenter study of 425 nonmetastatic patients who underwent surgery between January 1996 and December 2015 in French National Group and were enrolled in t...
Article
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Since desmoid tumors (DT) exhibit an unpredictable clinical course, with stabilization and/or spontaneous regression, an initial “wait-and-see” policy is the new standard of care–thus, the actual challenge is to identify early factors of progression. We present a method of detection of CTNNB1 mutations using a targeted digital droplet PCR (ddPCR) o...
Article
Background: Pigmented villonodular synovitis (alternatively known as diffuse-type giant cell tumour) is a rare, locally aggressive tumour driven by a specific translocation resulting in the overexpression of colony-stimulating factor 1 (CSF1). CSF1 receptor (CSF1R) inhibitors (ie, tyrosine kinase inhibitors and antibodies) induce a response in pat...
Article
Radiotherapy forms a significant part of cancer treatment. Caregivers support and advise patients to help them to adapt to the treatment and limit the occurrence of side effects. A nurse and member of a mobile support care and palliative care unit shares her experience of working in a team using sophrology to relieve the anxiety resulting from the...
Article
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Constitutive activating mutations in KIT and platelet-derived growth factor receptor α (PDGFRα) are heavily involved in the pathobiology of gastrointestinal stromal tumors (GISTs). This disease has served as an effective “proof-of-concept” model for targeting gain-of-function kinase mutations in cancer. This review discusses the current standard of...
Article
Background: Upfront screening for dihydropyrimidine dehydrogenase (DPD) deficiency in patients scheduled for 5-FU should help reduce the risk of toxicities by preventive adaptive dosing. Our group has developed a simple functional testing categorizing patients upon their DPD status, i.e. extensive metabolizer (EM) or poor metabolizer (PM) patients...
Article
Background: Alveolar soft part sarcoma (ASPS) is an exceedingly rare and orphan disease, without active drugs approved in the front line. Pazopanib and trabectedin are licensed for sarcoma treatment from second-line, but very little and contradictory data are available on their activity in ASPS. Lacking ongoing and/or planned clinical trials, we c...
Article
Background: To assess the role of first-line Molecular Targeted Therapies (MTTs) in Advanced chordoma (AC) patients. Methods: Retrospective study of 80 patients treated between January 2004 and December 2015 at 15 major French Sarcoma or Neurooncology Centres. Results: The sex ratio M/F was 46/34. The median age was 59 (6-86) years. The primar...
Article
Cetuximab administration is contingent upon the upfront determination of Ras mutational status, because mutated patients are unlikely to respond to this therapy. In addition to tumor genomics, drug exposure levels could be a critical yet largely underestimated issue, because several reports have already demonstrated that cetuximab pharmacokinetic p...