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Simple Summary
This article summarises the international Frontline and Relapsed Rhabdomyosarcoma (FaR-RMS) clinical trial for patients with rhabdomyosarcoma. The trial has multiple research questions relating to chemotherapy and radiotherapy and biological and imaging studies as well as to the introduction of novel drugs for patients with very high...
Citations
... Residual masses post-treatment are often scar tissue or non-malignant cells, with minimal impact on OS. Early anatomical tumor size or volume changes after neo-adjuvant chemotherapy are poor predictors of long-term survival, highlighting the increasing importance of PET/CT scans for monitoring and the need for validating new early surrogate markers of outcome in RMS [27]. ...
Purpose
Pediatric Rhabdomyosarcoma (RMS) is a morbid and often lethal condition characterized by a paucity of clinical data. Beyond a detailed risk categorization system, it is unclear if genitourinary (GU) sites (bladder/prostate, paratesticular, female organs) have outcomes distinct from non-GU sites. This study pools primary data from phase-3 clinical trials involving pediatric RMS to evaluate this question.
Methods and materials
We obtained primary data from three Children’s Oncology Group pediatric RMS trials (NCT00075582, NCT00354835, NCT00354744) evaluating low- (LR), intermediate- (IMR), and high risk (HR) Pediatric RMS. Survival analysis was conducted using the Kaplan-Meier method, with Event-Free Survival (EFS) defined per protocol specifications.
Results
599 subject records were included in the analysis (111 GU RMS, 488 non-GU RMS). For subjects with GU RMS, overall survival (OS) was superior to non-GU RMS (HRR 0.55 [95% CI, 0.35–0.87], p = 0.009). In this same group, EFS superiority was not statistically significant compared to non-GU RMS (HRR 0.88 [95% CI, 0.63–1.22], p = 0.43). In the LR population, GU primary site was associated with improved OS that was not statistically significant (HRR 0.37 [95% CI 0.07–1.84], p = 0.21) and less favorable EFS (HRR 2.84 [95% CI, 1.13–7.12], p = 0.02).
Conclusions
In RMS, a GU primary site is linked to improved OS compared to non-GU sites, although LR GU RMS shows less favorable EFS. Our findings reinforce the association between GU primary sites and better OS outcomes in RMS, warranting further investigation into the surrogacy of EFS for OS in GU RMS.
Clinical trial registration
Not applicable.
... The authors wish to make corrections to the authorship and title of [1]. The corrected authorship is as follows: Julia Chisholm and Henry Mandeville contributed equally to the first authorship. ...
The authors wish to make corrections to the authorship and title of [...]
... Patients were afterward reassigned to four risk categories: low-risk (LR), intermediate-risk (IR), high-risk (HR), and very-high-risk (VHR). This current scheme is being applied to the running Frontline and Relapsed Rhabdomyosarcoma (FAR-RMS) Clinical Trial of the same EpSSG evaluating novel regimes for VHR, HR, and relapse patients, optimal duration of maintenance chemotherapy, and the impact of radiotherapy and surgery (local therapy) on health-related quality of life (HRQoL) [7,10]. ...
... Some authors claim that, since even a minimal incision of the mass to establish a definitive diagnosis and subsequent referral for radiotherapy and chemotherapy has proven highly effective, the procedure should be as minimally invasive as possible [23]. On the other hand, some authors claim that when well-circumscribed masses favorably located are present, a complete or near complete excision should be attempted [7]. Nonetheless, it is not still clear whether a complete removal of the orbital RMS could avoid the need for further treatments; therefore, especially in pediatric patients, oncologists advocate for a more aggressive approach including irradiation and chemotherapy, given the malignant nature of the disease [36]. ...
Background
Rhabdomyosarcoma (RMS) is the most prevalent soft tissue sarcoma in children, with approximately 30% of head and neck RMS occurring in the orbit. The management of orbital RMS is complex, requiring a multidisciplinary approach and careful surgical planning. The objective of the present paper is to provide the neurosurgeon with an update on this challenging tumor.
Case description
A 12-year-old boy was admitted to our department after the onset of diplopia in his right eye, associated with quickly worsening ipsilateral eyelid edema and mild proptosis. MRI revealed an anterior superomedial, extraconal, and intraorbital expansive lesion. Complete tumor removal was performed through a trans-orbital approach. Histological diagnosis of embryonal RMS was done. The patient underwent radio-chemotherapy in accordance with the EpSSG RMS2005 subgroup C protocol. The patient is alive and disease-free.
Discussion and conclusion
The intricacies of treating orbital tumors necessitate meticulous planning to safeguard vital structures while guaranteeing comprehensive oncological treatment. A multidisciplinary approach with a specific protocol depending on the location and characteristics of the tumor is required. The available treatment options include surgical intervention or combination therapies, such as chemotherapy and radiotherapy. The successful management of these diseases depends on the careful coordination and application of the available techniques.
... In RMS, residual masses of mature rhabdomyoblasts generally do not indicate persistent malignancy and rarely require further surgery. [27] Lastly, despite the small sample sizes, we compared BP with FO GU RMS, acknowledging the inherent risk of selection bias (as BP tumors are almost automatically excluded from the LR group compared to FO RMS). Both OS and EFS were comparable between the groups. ...
Purpose:
Pediatric Rhabdomyosarcoma (RMS) is a morbid and often lethal condition characterized by a paucity of clinical data. Beyond a detailed risk categorization system, it is unclear if genitourinary (GU) sites (bladder/prostate, paratesticular, female organs) have outcomes distinct from non-GU sites. This study pools primary data from phase-3 clinical trials involving pediatric RMS to evaluate this question.
Methods and Materials:
We obtained primary data from three Children’s Oncology Group pediatric RMS trials
(NCT00075582, NCT00354744, NCT00354835) evaluating low- (LR), intermediate- (IMR), and
high risk (HR) Pediatric RMS. Survival analysis was conducted using the Kaplan-Meier method, with Event-Free Survival (EFS) defined per protocol specifications.
Results:
599 subject records were included in the analysis (111 GU RMS, 488 non-GU RMS). For
subjects with GU RMS, overall survival (OS) was superior to non-GU RMS (HRR 0.55, 95% CI 0.35-0.87, p=0.009). In this same group EFS superiority was not statistically significant compared to non-GU RMS (HRR 0.88 95% CI, 0.63-1.22, p=0.43). In the LR population, GU primary site was associated with improved OS that was not statistically significant (HRR 0.37 95% CI 0.07-1.84, p=0.21) and less favorable EFS (HRR 2.84 95% CI, 1.13-7.12, p=0.02).
Conclusions:
In RMS, a GU primary site is linked to improved OS compared to non-GU sites, although LR GU RMS shows less favorable EFS. Our findings reinforce the association between GU primary sites and better OS outcomes in RMS, warranting further investigation into the surrogacy of EFS for OS in GU RMS.
Radiotherapy is an indispensable treatment modality in the management of rhabdomyosarcoma. Numerous efforts have been made to improve outcomes. The current thinking and future developments in the radiation oncology field about how to raise cure rates, especially in the highest-risk patients, are presented.
In 2005, the Paediatric Radiation Oncology Society (PROS), a multi‐professional association to promote radiotherapy for children and young people, was created. Its mission has been to educate those involved in this field, and to improve the standards of radiation oncology care for children, adolescents, and young adults across the globe, not least in less privileged settings where the majority of the world's children, and most with cancer, live. Through regular congresses, and joint meetings with the International Society of Paediatric Oncology (SIOP), PROS has facilitated a focus on this treatment modality, and advocated on behalf of those who might benefit from it.
