The EpSSG NRSTS 2005 treatment protocol for desmoid-type fibromatosis in children: an international prospective case series

Orbach, Daniel; Brennan, Bernadette; Bisogno, Gianni; Noesel, Max M. van; Minard‐Colin, Véronique; Daragjati, Julia; Casanova, Michela; Corradini, Nadège; Zanetti, Ilaria; Salvo, Gian Luca De; Defachelles, Anne‐Sophie; Kelsey, Anna; Arush, Myriam Ben; Francotte, Nadine; Ferrari, Andrea

doi:10.1016/s2352-4642(17)30045-7

Cited by 45 publications

(42 citation statements)

References 32 publications

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“…There were no grade 3/4 toxicities observed. Schedule modification occurred in four (8%) patients, due to nausea alone, (17) Tamoxifen alone 15 (53) NSAIDS alone 2 (7) Surgery and NSAIDS 1 (3) Surgery and Tamoxifen 2 (7) Tamoxifen and NSAIDS 2 (7) Surgery, radiation and NSAIDS 1 (3) Multiple drug treatments (including doxorubicin) 1 (n = 2), nausea and anemia, (n = 1), and abdominal cramping, (n = 1).…”

Section: Treatment and Toxicitymentioning

confidence: 99%

“…Management of DF has evolved to eliminate unnecessary morbidity from surgery and radiation, toward first‐line active surveillance . An initial watchful waiting approach has not been shown to compromise outcomes when compared with upfront systemic treatment …”

Section: Introductionmentioning

confidence: 99%

“…[2][3][4][5][6] An initial watchful waiting approach has not been shown to compromise outcomes when compared with upfront systemic treatment. 6,7 When there is persistent progression of DF, many expert centers currently advocate for the use of medical therapy for initial intervention. [8][9][10] Although there are several systemic therapeutic DF options, in the absence of comparative studies, most institutions have selected a regimen based on historical data and local experience.…”

Section: Introductionmentioning

confidence: 99%

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Clinical benefit of methotrexate plus vinorelbine chemotherapy for desmoid fibromatosis (DF) and correlation of treatment response with MRI

et al. 2019

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Background Desmoid fibromatosis (DF) is a rare fibroblastic proliferation that was historically treated with surgery. We report (a) outcomes using low‐dose chemotherapy, methotrexate (MTX), and vinorelbine (VNL) for patients with progressing disease (PD) and (b) whether tumor volume ( V tumor ) and T2 signal on magnetic resonance imaging (MRI) are more reflective of treatment response compared with maximum tumor dimension ( D max ) defined by RECIST1.1. Methods Patients with biopsy‐proven DF, treated with MTX/VNL from 1997 to 2015 were reviewed. MRI for a subset of patients was independently re‐evaluated for response by RECIST, V tumor , and quantitative T2 hyperintensity. Results Among 48 patients treated for a median 19 months MTX/VNL, only nine (19%) had previous surgery. RECIST‐based overall response rate was complete response (CR) 20 (42%) + partial response (PR) 19 (39%), stable disease (SD) 8 (17%), for a clinical benefit rate of 98%. The median progression‐free survival (PFS) was 120 months, (95%CI 84‐155 months). Thirty‐six (75%) patients had not progressed at a median 38 months from treatment completion. Most common grade 1/2 toxicities included nausea (n = 12, 25%) and fatigue (n = 9,19%) with no grade 3/4 toxicities. In 22 patients with serial MRIs, there was a decrease in D max mean by 30%, V tumor by 76%, and in 19/22 (86%) a decrease in T2 signal intensity. Conclusion Low‐dose MTX/VNL for a defined duration has high efficacy with sustained benefit and minimal toxicity for treating DF. V tumor and T2 signal might better predict treatment response than RECIST.

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Section: Treatment and Toxicitymentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Clinical benefit of methotrexate plus vinorelbine chemotherapy for desmoid fibromatosis (DF) and correlation of treatment response with MRI

et al. 2019

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“…Recent reports have reported a control rate of 60%-90% with wait and see, 7,8 but it was poorer at 27% in the young cohort. 9 Such patients require effective systemic treatment.…”

Section: Introductionmentioning

confidence: 99%

Desmoid with biweekly methotrexate and vinblastine shows similar effects to weekly administration: A phase II clinical trial

et al. 2020

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Low‐dose methotrexate (MTX) plus vinblastine (VBL) chemotherapy is an effective treatment for desmoid‐type fibromatosis (DF). However, previous reports have described a weekly regimen, with no reports available on a biweekly one. The aim of this study was to determine the clinical outcomes of a biweekly regimen in a cohort prospectively treated in our single institution. Since 2010, we have prospectively treated refractory DF patients with biweekly MTX (30 mg/m2) + VBL (6 mg/m2). Efficacy, progression‐free survival (PFS), and correlating factors were analyzed. Adverse events (AEs) were recorded. In total, 38 patients received low‐dose MTX + VBL therapy, and its efficacy was assessed in 37 of them. Nineteen (51%) patients showed partial response (PR). Clinical benefit rate was 95%. PFS at 5 y was 80.8%. In PR cases, median time to response was 10 mo. Longer duration of therapy was significantly associated with the response of PR (P = .007) by univariate analysis. There was no clear association between various clinicopathological factors, including tumor size, location, catenin beta‐1 (CTNNB1) mutation status with effect. Only 3 AEs of grade 3/4 were observed. Tumor regrowth after MTX + VBL discontinuation was observed in 5 (20%) of 25 patients. Biweekly administration of MTX + VBL chemotherapy was well tolerated compared with weekly administration, and its efficacy was anticipated in DF patents, although the time needed to achieve a response may be relatively long. The treatment interval should be determined taking into account both the condition of the tumor and the patient's preference.

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“…Vincristine/dactinomycin and vinblastine/methotrexate regimens have been used with good results in patients with IM [35,47]. Yet, similar to what has been observed in patients with aggressive fibromatosis [48][49][50], response to treatment tends to be slow. Alternatively, a few children with PDGFRB-mutated IM have been treated successfully with imatinib and sunitinib [8,15,20,28].…”

Section: Treatmentmentioning

confidence: 89%

Genetic testing and surveillance in infantile myofibromatosis: a report from the SIOPE Host Genome Working Group

et al. 2020

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Infantile myofibromatosis (IM), which is typically diagnosed in young children, comprises a wide clinical spectrum ranging from inconspicuous solitary soft tissue nodules to multiple disseminated tumors resulting in life-threatening complications. Familial IM follows an autosomal dominant mode of inheritance and is linked to PDGFRB germline variants. Somatic PDGFRB variants were also detected in solitary and multifocal IM lesions. PDGFRB variants associated with IM constitutively activate PDGFRB kinase activity in the absence of its ligand. Germline variants have lower activating capabilities than somatic variants and, thus, require a second cis-acting hit for full receptor activation. Typically, these mutant receptors remain sensitive to tyrosine kinase inhibitors such as imatinib. The SIOPE Host Genome Working Group, consisting of pediatric oncologists, clinical geneticists and scientists, met in January 2020 to discuss recommendations for genetic testing and surveillance for patients who are diagnosed with IM or have a family history of IM/PDGFRB germline variants. This report provides a brief review of the clinical manifestations and genetics of IM and summarizes our interdisciplinary recommendations.

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The EpSSG NRSTS 2005 treatment protocol for desmoid-type fibromatosis in children: an international prospective case series

Cited by 45 publications

References 32 publications

Clinical benefit of methotrexate plus vinorelbine chemotherapy for desmoid fibromatosis (DF) and correlation of treatment response with MRI

Clinical benefit of methotrexate plus vinorelbine chemotherapy for desmoid fibromatosis (DF) and correlation of treatment response with MRI

Desmoid with biweekly methotrexate and vinblastine shows similar effects to weekly administration: A phase II clinical trial

Genetic testing and surveillance in infantile myofibromatosis: a report from the SIOPE Host Genome Working Group

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