Non-parameningeal head and neck rhabdomyosarcoma in children, adolescents, and young adults: Experience of the European paediatric Soft tissue sarcoma Study Group (EpSSG) – RMS2005 study

Glosli, Heidi; Bisogno, Gianni; Kelsey, Anna; Chisholm, Julia; Gaze, Mark; Kolb, F.; McHugh, Kieran; Shipley, Janet; Gallego, Soledad; Merks, Johannes H. M.; Smeele, Ludi E.; Mandeville, Henry; Ferrari, Andrea; Minard‐Colin, Véronique; Corradini, Nadège; Jenney, Meriel; Zanetti, Ilaria; Salvo, Gian Luca De; Orbach, Daniel; members, EpSSG

doi:10.1016/j.ejca.2021.04.007

Cited by 31 publications

(28 citation statements)

References 27 publications

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“…Rarer subtypes such as sclerosing/spindle‐cell RMS (Rudzinski et al , 2015) will be exciting to include, as also indicated by a recent case report (Acanda De La Rocha et al , 2021). Compared to patient incidence rates (Glosli et al , 2021), the collection has an underrepresentation of head and neck RMS. Although RMS tumor samples from this region were acquired, models from such samples failed, regardless of subtype, clinical stage, sample quantity, or quality.…”

Section: Discussionmentioning

confidence: 99%

Mesenchymal tumor organoid models recapitulate rhabdomyosarcoma subtypes

et al. 2022

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Rhabdomyosarcomas (RMS) are mesenchyme-derived tumors and the most common childhood soft tissue sarcomas. Treatment is intense, with a nevertheless poor prognosis for high-risk patients. Discovery of new therapies would benefit from additional preclinical models. Here, we describe the generation of a collection of 19 pediatric RMS tumor organoid (tumoroid) models (success rate of 41%) comprising all major subtypes. For aggressive tumors, tumoroid models can often be established within 4-8 weeks, indicating the feasibility of personalized drug screening. Molecular, genetic, and histological characterization show that the models closely resemble the original tumors, with genetic stability over extended culture periods of up to 6 months. Importantly, drug screening reflects established sensitivities and the models can be modified by CRISPR/Cas9 with TP53 knockout in an embryonal RMS model resulting in replicative stress drug sensitivity. Tumors of mesenchymal origin can therefore be used to generate organoid models, relevant for a variety of preclinical and clinical research questions.

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Section: Discussionmentioning

confidence: 99%

Mesenchymal tumor organoid models recapitulate rhabdomyosarcoma subtypes

et al. 2022

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“…For the patient who displayed a nodal progression after secondary surgery and before the end of chemotherapy, the irradiation field targeted only LN areas. Of the 41 irradiation fields targeting the primary tumor, 32…”

Section: Radiation Therapymentioning

confidence: 99%

“…In a series of 140 localized non-PM RMS including 40 RMSA from 1984 to 2004, Orbach et al ( 29) observed a 5-year OS of 66% and a 5-year systemic treatment, as well as to a greater efficiency of the local and regional treatment with conformational and proton beam radiation therapy and the development of free flaps, allowing large surgical resections for "ghost surgeries." Several authors suggest that OS and EFS are better in patients who underwent surgical resection in addition to treatment with chemotherapy and RT (15,(30)(31)(32). Multidisciplinary approach including "ghost surgery" for PM sarcoma is feasible and yields promising local control.…”

Section: Survival and Prognostic Factorsmentioning

confidence: 99%

Locoregional Control and Survival in Children, Adolescents, and Young Adults With Localized Head and Neck Alveolar Rhabdomyosarcoma—The French Experience

et al. 2022

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IntroductionThe head and neck (HN) are the most frequent sites of pediatric rhabdomyosarcoma (RMS). Alveolar RMS (ARMS) represents ~20% of all RMS cases and frequently spread to lymph nodes (LNs). The aim was to report locoregional control, event-free survival (EFS), and overall survival (OS), according to clinical and pathological features, LN staging, and treatment modalities.MethodsThe study included all patients prospectively enrolled in EpSSG RMS 2005 study under 21 years of age with localized HN ARMS and diagnosed between 2005 and 2016 in France. Medical data including imaging, surgical report, and radiation therapy planes were analyzed.ResultsForty-eight patients (median age 6 years; range 4 months−21 years), corresponding to 30 parameningeal and 18 non-parameningeal ARMS, were included. There were 33 boys (69%). Tumor locations included the following: orbit (n = 7) among which four cases had bone erosion, paranasal sinuses and nasal cavity (n = 16), deep facial spaces (n = 10), nasolabial fold (n = 8), and other non-parameningeal HN sites (n = 7). A fusion transcript of PAX3-FOXO1 or PAX7-FOXO1 was expressed in 33 of the 45 cases (73%) with molecular analysis. At diagnosis, 10 patients had primary resection of the primary tumor (PRPT) (none with microscopic complete resection) and 9 had LN staging. After induction chemotherapy, 26 patients (54%) had secondary resection of the primary tumor (SRPT) and 13 patients (27%) had cervical LN dissection. A total of 43 patients (90%) were treated with radiation therapy.With a median follow-up of 7 years (range 2–13 years), 5-year OS and EFS were 78% (95% CI, 63–88%) and 66% (95% CI, 51–78%), respectively. We observed 16 events (10 deaths): 4 local, 4 regional, 1 local and regional, and 7 metastatic. In univariate analysis, OS was only superior for patients under 10 years of age (p = 0.002), while FOXO1-negative ARMS, SRPT for parameningeal ARMS, and LN surgery were associated with significantly better EFS.ConclusionOur study confirms a better outcome for fusion-negative ARMS and ARMS in children under 10 years. Moreover, LN surgery and SRPT of parameningeal tumor may improve EFS of ARMS. Larger studies are needed to confirm our findings.

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“…Rhabdomyosarcoma (RMS) is a primitive malignant soft tissue sarcoma of the skeletal muscle phenotype that originates from mesenchymal cells [ 1 ]. It is the most common childhood and adolescent soft tissue sarcoma (3–5% of childhood tumours, 50% of paediatric soft tissue sarcomas) [ 1 , 2 , 3 , 4 ]. The median age at diagnosis of RMS patients is 6.5 years, and there is a slight male predominance (male/female ratio 1.3/1) [ 5 ].…”

Section: Introductionmentioning

confidence: 99%

“…The median age at diagnosis of RMS patients is 6.5 years, and there is a slight male predominance (male/female ratio 1.3/1) [ 5 ]. Different subtypes exist, including embryonal (70%), alveolar (20%), and spindle cell/sclerosing (10%) RMS [ 1 , 2 , 3 , 4 , 5 ]. Local treatment options for RMS include external beam radiotherapy with photons (RT), external beam radiotherapy with protons (PT) [ 6 ], Ablative surgery followed by the MOuld technique after loading brachytherapy and surgical REconstruction (AMORE) [ 7 ], and the combination of RT or PT with conventional surgery [ 8 ].…”

Section: Introductionmentioning

confidence: 99%

Patterns of Hearing Loss in Irradiated Survivors of Head and Neck Rhabdomyosarcoma

Diepstraten

Wiersma

Schoot

et al. 2022

Cancers

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Purpose: The frequency and patterns of HL in a HNRMS survivor cohort were investigated. A dose–effect relationship between the dose to the cochlea and HL was explored. Methods: Dutch survivors treated for HNRMS between 1993 and 2017 with no relapse and at least two years after the end of treatment were eligible for inclusion. The survivors were evaluated for HL with pure-tone audiometry. HL was graded according to the Muenster, Common Terminology Criteria for Adverse Events (CTCAE) v4.03 and International Society for Paediatric Oncology (SIOP) classification. We defined deleterious HL as Muenster ≥ 2b, CTCAE ≥ 2, and SIOP ≥ 2. Mixed-effects logistic regression was used to search for the dose–effect relationship between the irradiation dose to the cochlea and the occurrence of HL. Results: Forty-two HNRMS survivors underwent pure-tone audiometry. The Muenster, CTCAE and SIOP classification showed that 19.0% (n = 8), 14.2% (n = 6) and 11.9% (n = 5) of survivors suffered from HL, respectively. A low-frequency HL pattern with normal hearing or milder hearing loss in the higher frequencies was seen in four survivors. The maximum cochlear irradiation dose was significantly associated with HL (≥Muenster 2b) (p = 0.047). In our series, HL (≥Muenster 2b) was especially observed when the maximum dose to the cochlea exceeded 19 Gy. Conclusion: HL occurred in up to 19% of survivors of HNRMS. More research is needed on HL patterns in HNRMS survivors and on radiotherapy dose–effect relationships.

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Non-parameningeal head and neck rhabdomyosarcoma in children, adolescents, and young adults: Experience of the European paediatric Soft tissue sarcoma Study Group (EpSSG) – RMS2005 study

Cited by 31 publications

References 27 publications

Mesenchymal tumor organoid models recapitulate rhabdomyosarcoma subtypes

Mesenchymal tumor organoid models recapitulate rhabdomyosarcoma subtypes

Locoregional Control and Survival in Children, Adolescents, and Young Adults With Localized Head and Neck Alveolar Rhabdomyosarcoma—The French Experience

Patterns of Hearing Loss in Irradiated Survivors of Head and Neck Rhabdomyosarcoma

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