OBJECTIVE: The study objective was to evaluate the safety and efficacy of a transaortic approach to midventricular and apical septal myectomy in patients with hypertrophic cardiomyopathy with left ventricular outflow tract or midventricular obstruction. METHODS: From January 2018 to August 2023, 940 patients underwent transaortic septal myectomy at the Cleveland Clinic, of whom 682 (73%) had midventricular or apical resection. Patients who underwent isolated basal myectomies were excluded. Templated operative reports designated septal regions resected as basal (opposition to mitral valve up to the leaflet tips), midventricular (leaflet tips to just beyond the papillary muscle heads), and apical (apical third of the ventricle). Myocardial resection specimen weights, intraventricular gradients, and clinical outcomes were assessed. RESULTS: Of the 682 patients, 582 (85%) had basal plus midventricular resection and 78 (11%) had basal, midventricular, and apical resection. Mean preoperative intraventricular gradient was 102 ± 41 mm Hg. Median resection weight was 10 g (15th, 85th percentiles: 7, 15), and mean postoperative intraventricular gradient was 16 ± 10 mm Hg, with 625 (96%) patients achieving gradients 36 mm Hg or less. There were no iatrogenic mitral or aortic valve injuries. Permanent pacemaker placement was required in 38 patients (5.6%), of whom 8 (1.2%) had normal preoperative conduction. Operative mortality occurred in 1 patient (0.1%) after an intraoperative ventricular septal defect. CONCLUSIONS: Most patients undergoing septal myectomy for relief of obstruction required resection beyond the basal septum. With specialized instrumentation, detailed imaging and knowledge of variable septal anatomy, resecting midventricular and apical septal muscle can be safely and effectively achieved through a transaortic approach.
BACKGROUND: In retrospective analyses, the Pediatric Oncology Group [POG) and the Federation National des Centres de Lutte Contre le Cancer (FNCLCC) histologic grade predict outcome in pediatric non-rhabdomyosarcoma soft tissue sarcoma (NRSTS), but prospective data on grading, clinical features, and outcomes of low-grade NRSTS are limited. METHODS: We analyzed patients less than 30 years of age enrolled on Children's Oncology Group (COG) study ARST0332 (NCT00346164) with POG grade 1 or 2 NRSTS. Low-risk patients were treated with surgery alone. Intermediate-/high-risk patients received ifosfamide/doxorubicin and radiotherapy, with definitive resection either before or after 12 weeks of chemoradiotherapy. RESULTS: Estimated 5-year event-free and overall survival were 90% and 100% low risk (n = 80), 55% and 78% intermediate risk (n = 15), and 25% and 25% high risk (n = 4). In low-risk patients, only local recurrence was seen in 10%; none with margins greater than 1 mm recurred locally. Sixteen of 17 intermediate-/high-risk patients who completed neoadjuvant chemoradiotherapy underwent gross total tumor resection, 80% with negative margins. Intermediate-/high-risk group events included one local and seven metastatic recurrences. Had the FNCLCC grading system been used to direct treatment, 29% of low-risk (surgery alone) patients would have received radiotherapy ± chemotherapy. CONCLUSIONS: Most low-risk patients with completely resected POG low-grade NRSTS are successfully treated with surgery alone, and surgical margins greater than 1 mm may be sufficient to prevent local recurrence. Patients with intermediate- and high-risk low-grade NRSTS have outcomes similar to patients with high-grade histology, and require more effective therapies. Use of the current FNCLCC grading system may result in overtreatment of low-risk NRSTS curable with surgery alone.
Background: Cardiac resynchronization therapy (CRT) is recommended for patients with symptomatic heart failure in sinus rhythm with left ventricular ejection fraction (LVEF) ≤ 35%, QRS duration ≥ 150â ms, and left bundle branch block (LBBB) morphology. However, when severe left ventricular dysfunction and cardiogenic shock are present, treatment paradigms are often limited to palliative medical therapy or advanced therapies with durable left ventricular assist device or heart transplant as the functional and survival benefit of CRT in these patients remains uncertain. Case summary: A 77-year-old white man with long-standing LBBB with dyssynchrony, severely reduced LVEF of 4%, and severe bicuspid aortic stenosis (AS) presented with worsening heart failure symptoms. After multidisciplinary heart team evaluation and pre-operative optimization, the patient underwent a surgical aortic valve replacement with simultaneous intraoperative initiation of CRT with pacemaker (CRT-P) and temporary mechanical circulatory support. Echocardiography at 44 days and 201 days post-discharge showed an LVEF of 29% and 40%, respectively. Discussion: This case demonstrates that reverse remodelling and native heart recovery were successfully achieved in a patient with advanced structural heart disease, presenting with cardiogenic shock, through an early and aggressive approach involving multidisciplinary heart team evaluation, treatment of severe AS with surgical aortic valve replacement, prophylactic intraoperative initiation of temporary mechanical circulatory support, and early initiation of CRT-P.
This study evaluated the nationwide associations between concomitant left atrial appendage clip (LAAC) placement during cardiac surgery and postoperative outcomes. We identified 1,260,999 patients who underwent coronary artery bypass grafting, valve, and aortic surgeries in the 2016 to 2020 Nationwide Readmissions Database and stratified by concomitant LAAC versus no LAAC placement. Patients who underwent surgical ablation were excluded. Mortality and complications were compared during index admissions and for patients readmitted within 30 and 90 days of the index discharge date for unmatched and propensity score-matched groups. Overall, 6.7% (84,293) of patients underwent cardiac surgery and concomitant LAAC placement without surgical ablation. After propensity score matching, the index admission mortality and overall complications were not different in patients with LAAC versus patients without LAAC. LAAC placement was associated with increased any-cause 30-day readmissions (15% vs 13%, p <0.01). In patients with LAAC, within 30 days, there were no differences in mortality (3.9% vs 3.8%, p = 0.60) or overall complications (64% vs 63%, p = 0.20), whereas stroke was lower (5.3% vs 6.5%, p <0.01) and heart failure was higher (35% vs 30%, p <0.01). For patients readmitted within 90 days, similar findings were observed for any-cause readmissions, mortality, overall complications, stroke, and heart failure. In conclusion, concomitant LAAC placement during cardiac surgery was associated with lower early postdischarge incidence of stroke and a favorable overall risk-benefit profile. Given these short-term findings in a real-world population of all patients who underwent cardiac surgery, longer-term studies with more granular data are needed to evaluate the potential benefit of this practice.
BACKGROUND: Novel therapies are needed for relapsed and refractory rhabdomyosarcoma (RRMS). Phase II clinical trials in RRMS have typically utilized radiologic response as the primary activity endpoint, an approach that poses several limitations in RRMS. In this analysis, we aimed to estimate an event-free survival (EFS) endpoint for RRMS that could be used as a benchmark for future studies. PROCEDURE: We performed a retrospective study of patients with RRMS enrolling on 13 single-agent phase II Children's Oncology Group and legacy group trials from 1997 to 2016. All included trials used radiographic response as their primary activity endpoint. Six-month EFS was estimated from time of trial enrollment with 95% confidence intervals. Clinical characteristics, including trial of enrollment, sex, age, race, histology, number of prior chemotherapies, and radiographic response were evaluated for their impact on 6-month EFS. RESULTS: We identified 175 patients across 13 trials. The 6-month EFS was 16.8% (11.6%-22.8%). No differences were seen in 6-month EFS based on age, sex, race, or histology. There were nonsignificant trends toward improved 6-month EFS for patients with less than or equal to two prior lines of therapy versus higher than two, for patients enrolled on trials that achieved their primary radiographic response endpoint versus trials that did not, and for patients who achieved complete or partial response compared to those achieving stable disease. CONCLUSIONS: The prognosis of RRMS enrolled on single-agent phase II trials is poor. This pooled 6-month EFS of RRMS on single-agent trials may be used as a RRMS-specific benchmark for future single-agent phase II trials.
BACKGROUND: Embryonal sarcoma of the liver (ESL) is a rare mesenchymal tumor most common in childhood; the optimal treatment approach is uncertain. The clinical features and outcomes of patients with ESL enrolled in a Children's Oncology Group (COG) clinical trial that evaluated a risk-based strategy for treating soft tissue sarcomas in patients aged <30 years were evaluated. METHODS: This subset analysis included patients with ESL enrolled in COG study ARST0332. Central review of records, pathology, and imaging confirmed the diagnosis, presenting features, and surgery extent and complications. All patients received dose-intensive ifosfamide/doxorubicin chemotherapy, with cycle timing dependent on surgery and radiotherapy. Tumor resection occurred before study entry or after four cycles of chemotherapy; radiotherapy for residual tumor was optional. RESULTS: Thirty-nine eligible/evaluable patients with ESL were analyzed. All tumors were >10 cm in diameter; four were metastatic. Tumor resection was performed upfront in 23 and delayed in 16. Positive surgical margins (n = 6) and intraoperative tumor rupture (n = 6) occurred only in upfront resections. Eight patients received radiotherapy. Estimated 5-year event-free and overall survival were 79% (95% confidence interval [CI], 65%-93%) and 95% (95% CI, 87%-100%), respectively. Positive margins increased the local recurrence risk. One of 13 patients with documented hemorrhagic ascites and/or tumor rupture developed extrahepatic intra-abdominal tumor recurrence. CONCLUSIONS: The treatment strategy used in ARST0332 achieved favorable outcomes for patients with ESL despite a substantial proportion having high-risk disease features. Deferring tumor resection until after neoadjuvant chemotherapy may decrease the risk of intraoperative tumor rupture and improve the likelihood of adequate surgical margins.
BACKGROUND: The aim of this study was to estimate the event-free survival (EFS) of children and young adults with relapsed or refractory nonrhabdomyosarcoma soft tissue sarcoma (NRSTS) treated in nonrandomized phase 2 studies conducted by the Children's Oncology Group (COG) and predecessor groups to establish a benchmark EFS for future phase 2 NRSTS trials evaluating the activity of novel agents. METHODS: A retrospective analysis of patients with recurrent or refractory NRSTS prospectively enrolled in nonrandomized phase 2 COG and predecessor group trials between 1994 and 2015 was conducted. EFS was defined as disease progression/relapse or death and calculated via the Kaplan-Meier method. The log-rank test and relative risk regression were used to compare EFS distribution by age at enrollment, sex, race, NRSTS histology, prior lines of therapy, calendar year of trial, and type of radiographic response. RESULTS: In total, 137 patients were enrolled in 13 phase 2 trials. All trials used radiographic response rate as a primary outcome, and none of the agents used were considered active on the basis of trial-specified thresholds. The estimated median EFS and 6-month EFS of the entire study cohort was 1.5 months (95% confidence interval [CI], 1.3-1.8 months) and 19.4% (95% CI, 12.7%-26%), respectively. No difference in EFS was observed by age at enrollment, sex, race, NRSTS histology subtype, prior lines of therapies, and trial initiation year. EFS significantly differed by radiographic response. CONCLUSIONS: The EFS for children and young adults with relapsed or refractory NRSTS remains suboptimal. Established EFS can be referenced as a benchmark for future single-agent phase 2 trials incorporating potentially active novel agents in this population.
Transcatheter aortic valve replacement is not widely used in patients with congenital heart disease. We describe our single-center experience of transcatheter aortic valve replacement in congenital heart disease, demonstrating short-term feasibility and safety, role in lifetime management of congenital aortic valve disease, and use as a bridge to recovery, future surgery, or transplantation.
INTRODUCTION: The impact of established prognostic factors on survival outcomes for childhood rhabdomyosarcoma (RMS) have not been well described in the adolescent and young adult (AYA) RMS patient population. METHODS: This is a retrospective analysis of patients with newly diagnosed RMS enrolled between 1997 and 2016 on seven previously reported Children's Oncology Group (COG) clinical trials. Demographics, clinical features, treatment details, and outcome data were collected. Five-year event-free survival (EFS) and overall survival (OS) were estimated for patients diagnosed at age 15-39 years and those diagnosed under age 15 years using the Kaplan-Meier method. Log-rank test was used to compare prognostic factors for EFS and OS. Factors significant in the univariable analysis were included in a Cox proportional hazards regression model. Nonsignificant covariates were removed from the multiple regression model. RESULTS: Total 2151 patients including 402 AYAs were analyzed. AYAs were more likely to present with primary tumors ≥5 cm in size, metastatic disease, alveolar histology, and have FOXO1 fusions compared to children. Five-year EFS for the AYA cohort was 44.2% versus 67% for children (p < .001), and 5-year OS was 52% for the AYA cohort versus 78% for children (p < .001). Multivariable analysis revealed tumor site, size and invasiveness, clinical group, and histology were prognostic in AYAs. CONCLUSION: AYAs with RMS have a poorer prognosis compared to younger children due to multiple factors. Further research focused on AYAs to better understand RMS biology and improve treatments is critical to improve survival.
Rhabdomyosarcoma, Embryonal , Rhabdomyosarcoma , Soft Tissue Neoplasms , Child , Humans , Adolescent , Young Adult , Adult , Retrospective Studies , Rhabdomyosarcoma/pathology , Prognosis , Proportional Hazards Models
Soft tissue sarcomas (STS) represent a heterogeneous group of extraskeletal mesenchymal tumors that affect individuals throughout the entire age continuum. Despite this pervasive influence, key differences exist in the presentation of these sarcomas across varying age groups that have prevented a more uniform approach to management. Notably, rhabdomyosarcoma (RMS) is more common in children, while most nonrhabdomyosarcoma soft tissue sarcoma (NRSTS) subtypes are more prevalent in adults. Older patients with NRSTS appear to have more molecularly complex biology and often present with more advanced disease compared with children. Poorer outcome disparities are observed in older patients with RMS despite receiving similar treatment as younger patients. In this review, we highlight differences in epidemiology, biology, and management paradigms for pediatric and adult patients with STS and explore opportunities for a unified approach to enhance the care and outcomes within the AYA population.
Rhabdomyosarcoma , Sarcoma , Soft Tissue Neoplasms , Child , Humans , Adolescent , Young Adult , Aged , Sarcoma/therapy , Sarcoma/drug therapy , Rhabdomyosarcoma/epidemiology , Rhabdomyosarcoma/genetics , Rhabdomyosarcoma/therapy , Soft Tissue Neoplasms/epidemiology , Soft Tissue Neoplasms/genetics , Soft Tissue Neoplasms/therapy
Patients with advanced ischemic cardiomyopathy manifesting as left ventricular dysfunction exist along a spectrum of severity and risk, and thus decision-making surrounding optimal management is challenging. Treatment pathways can include medical therapy as well as revascularization through percutaneous coronary intervention or coronary artery bypass grafting. Additionally, temporary and durable mechanical circulatory support, as well as heart transplantation, may be optimal for select patients. Given this spectrum of risk and the complexity of treatment pathways, patients may not receive appropriate therapy given their perceived risk, which can lead to sub-satisfactory outcomes. In this review, we discuss the identification of high-risk ischemic cardiomyopathy patients, along with our programmatic approach to patient evaluation and perioperative optimization. We also discuss our strategies for therapeutic decision-making designed to optimize both short- and long-term patient outcomes.
Cardiomyopathies , Myocardial Ischemia , Percutaneous Coronary Intervention , Ventricular Dysfunction, Left , Humans , Myocardial Ischemia/complications , Myocardial Ischemia/therapy , Coronary Artery Bypass , Ventricular Dysfunction, Left/surgery , Cardiomyopathies/therapy , Cardiomyopathies/surgery , Treatment Outcome
OBJECTIVE: Patient characteristics, risks, and outcomes associated with reoperative multivalve cardiac surgery are poorly characterized. Effect of patient variables and surgical components of each reoperation were evaluated with regard to operative mortality. METHODS: From January 2008 to January 2022, 2324 patients with previous cardiac surgery underwent 2352 reoperations involving repair or replacement of multiple cardiac valves at Cleveland Clinic. Mean age was 66 ± 14 years. Number of surgical components representing surgical complexity (valve procedures, aortic surgery, coronary artery bypass grafting, and atrial fibrillation procedures) ranged from 2 to 6. Random forest for imbalanced data was used to identify risk factors for operative mortality. RESULTS: Surgery was elective in 1327 (56%), urgent in 1006 (43%), and emergency in 19 (0.8%). First-time reoperations were performed in 1796 (76%) and 556 (24%) had 2 or more previous operations. Isolated multivalve operations comprised 54% (1265) of cases; 1087 incorporated additional surgical components. Two valves were operated on in 80% (1889) of cases, 3 in 20% (461), and 4 in 0.09% (2). Operative mortality was 4.2% (98 out of 2352), with 1.7% (12 out of 704) for elective, isolated multivalve reoperations. For each added surgical component, operative mortality incrementally increased, from 2.4% for 2 components (24 out of 1009) to 17% for ≥5 (5 out of 30). Predictors of operative mortality included coronary artery bypass grafting, surgical urgency, cardiac, renal dysfunction, peripheral artery disease, New York Heart Association functional class, and anemia. CONCLUSIONS: Elective, isolated reoperative multivalve surgery can be performed with low mortality. Surgical complexity coupled with key physiologic factors can be used to inform surgical risk and decision making.
A palpable rectal mass associated with gastrointestinal (GI) symptoms immediately raises concern for colorectal cancer, but rarely can represent distant metastatic disease. The incidence of symptomatic colorectal metastasis from a primary lung cancer without any pulmonary symptom is extremely rare. We report a rare case of constipation as the presenting symptom in a patient ultimately found to have metastatic squamous cell carcinoma of the lung. A rectal mass was readily palpable on examination, illustrating the importance of digital rectal examination. In addition, GI clinicians should maintain a high index of suspicion when evaluating patients at risk of non-GI malignancies.
Rhabdomyosarcoma is the most common soft-tissue neoplasm in the pediatric population. The survival of children with rhabdomyosarcoma has only marginally improved over the past 25 years and remains poor for those with metastatic disease. A significant challenge to advances in treatment of rhabdomyosarcoma is the relative rarity of this disease, necessitating years to complete clinical trials. Progress can be accelerated by international cooperation and sharing national experiences. This necessitates agreement on a common language to describe patient cohorts and consensus standards to guide diagnosis, treatment, and response assessment. These goals formed the premise for creating the INternational Soft Tissue saRcoma ConsorTium (INSTRuCT) in 2017. Multidisciplinary members of this consortium have since developed international consensus statements on the diagnosis, treatment, and management of pediatric soft-tissue sarcomas. Herein, members of the INSTRuCT Diagnostic Imaging Working Group present international consensus recommendations for imaging of patients with rhabdomyosarcoma at diagnosis, at staging, and during and after completion of therapy. The intent is to promote a standardized imaging approach to pediatric patients with this malignancy to create more-reliable comparisons of results of clinical trials internationally, thereby accelerating progress in managing rhabdomyosarcoma and improving survival.
Rhabdomyosarcoma , Sarcoma , Soft Tissue Neoplasms , Child , Humans , Sarcoma/pathology , Rhabdomyosarcoma/diagnostic imaging , Rhabdomyosarcoma/therapy , Combined Modality Therapy , Soft Tissue Neoplasms/pathology , Diagnostic Imaging
Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned coprimary or secondary analyses are not yet available. Clinical trial updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.Long-term outcomes from Children's Oncology Group study AEWS0031 were assessed to determine whether the survival advantage of interval-compressed chemotherapy (ICC) was maintained over 10 years in patients with localized Ewing sarcoma (ES). AEWS0031 enrolled 568 eligible patients. Patients were randomly assigned to receive vincristine-doxorubicin-cyclophosphamide and ifosfamide-etoposide alternating once every 3 weeks (standard timing chemotherapy [STC]) versus once every 2 weeks (ICC). For this updated report, one patient was excluded because of uncertainty of original diagnosis. The 10-year event-free survival (EFS) was 70% with ICC compared with 61% with STC (P = .03), and 10-year overall survival (OS) was 76% with ICC compared with 69% with STC (P = .04). There was no difference in the 10-year cumulative incidence of second malignant neoplasms (SMNs; PC [see Data Supplement, online only] = .5). A test for interaction demonstrated that ICC provided greater risk reduction for patients with tumor volume ≥200 mL than for patients with tumors <200 mL, but no evidence for a significant interaction in other subgroups defined by age, primary site, and histologic response. With longer-term follow-up, ICC for localized ES is associated with superior EFS and OS without an increased risk for SMN compared with STC. ICC is associated with improved outcomes even in adverse-risk patient groups.
Bone Neoplasms , Sarcoma, Ewing , Humans , Child , Sarcoma, Ewing/pathology , Bone Neoplasms/therapy , Etoposide , Ifosfamide , Antineoplastic Combined Chemotherapy Protocols/adverse effects , Doxorubicin , Vincristine
Background: The Society of Thoracic Surgeons risk scores are widely used to assess risk of morbidity and mortality in specific cardiac surgeries but may not perform optimally in all patients. In a cohort of patients undergoing cardiac surgery, we developed a data-driven, institution-specific machine learning-based model inferred from multi-modal electronic health records and compared the performance with the Society of Thoracic Surgeons models. Methods: All adult patients undergoing cardiac surgery between 2011 and 2016 were included. Routine electronic health record administrative, demographic, clinical, hemodynamic, laboratory, pharmacological, and procedural data features were extracted. The outcome was postoperative mortality. The database was randomly split into training (development) and test (evaluation) cohorts. Models developed using 4 classification algorithms were compared using 6 evaluation metrics. The performance of the final model was compared with the Society of Thoracic Surgeons models for 7 index surgical procedures. Results: A total of 6392 patients were included and described by 4016 features. Overall mortality was 3.0% (n = 193). The XGBoost algorithm using only features with no missing data (336 features) yielded the best-performing predictor. When applied to the test set, the predictor performed well (F-measure = 0.775; precision = 0.756; recall = 0.795; accuracy = 0.986; area under the receiver operating characteristic curve = 0.978; area under the precision-recall curve = 0.804). eXtreme Gradient Boosting consistently demonstrated improved performance over the Society of Thoracic Surgeons models when evaluated on index procedures within the test set. Conclusions: Machine learning models using institution-specific multi-modal electronic health records may improve performance in predicting mortality for individual patients undergoing cardiac surgery compared with the standard-of-care, population-derived Society of Thoracic Surgeons models. Institution-specific models may provide insights complementary to population-derived risk predictions to aid patient-level decision making.
Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.ARST1321 was a phase II study designed to compare the near complete pathologic response rate after preoperative chemoradiation with/without pazopanib in children and adults with intermediate-/high-risk chemotherapy-sensitive body wall/extremity non-Rhabdomyosarcoma Soft Tissue Sarcoma (ClinicalTrials.gov identifier: NCT02180867). Enrollment was stopped early following a predetermined interim analysis that found the rate of near complete pathologic response to be significantly greater with the addition of pazopanib. As a planned secondary aim of the study, the outcome data for this cohort were analyzed. Eight-five eligible patients were randomly assigned to receive (regimen A) or not receive (regimen B) pazopanib in combination with ifosfamide and doxorubicin + preoperative radiotherapy followed by primary resection at week 13 and then further chemotherapy at week 25. As of December 31, 2021, at a median survivor follow-up of 3.3 years (range, 0.1-5.8 years), the 3-year event-free survival for all patients in the intent-to-treat analysis was 52.5% (95% CI, 34.8 to 70.2) for regimen A and 50.6% (95% CI, 32 to 69.2) for regimen B (P = .8677, log-rank test); the 3-year overall survival was 75.7% (95% CI, 59.7 to 91.7) for regimen A and 65.4% (95% CI, 48.1 to 82.7) for regimen B (P = .1919, log-rank test). Although the rate of near complete pathologic response was significantly greater with the addition of pazopanib, outcomes were not statistically significantly different between the two regimens.
Sarcoma , Soft Tissue Neoplasms , Adult , Humans , Child , Sarcoma/drug therapy , Soft Tissue Neoplasms/pathology , Ifosfamide/therapeutic use , Doxorubicin/therapeutic use , Antineoplastic Combined Chemotherapy Protocols/therapeutic use
In the United States, approximately 850-900 children and adolescents each year are diagnosed with soft tissue sarcomas (STS). STS are divided into rhabdomyosarcoma (RMS) and non-rhabdomyosarcoma STS (NRSTS). RMS and NRSTS are risk stratified into low-, intermediate-, and high-risk categories, with 5-year survival rates of approximately 90%, 50%-70%, and 20%, respectively. Recent key achievements from the Children's Oncology Group (COG) STS Committee include the identification of new molecular prognostic factors for RMS, development and validation of a novel risk stratification system for NRSTS, successful completion of a collaborative NRSTS clinical trial with adult oncology consortia, and collaborative development of the INternational Soft Tissue SaRcoma ConsorTium (INSTRuCT). Current COG trials for RMS are prospectively evaluating a new risk stratification system that incorporates molecular findings, de-intensification of therapy for a very low-risk subgroup, and augmented therapy approaches for intermediate- and high-risk RMS. Trials for NRSTS exploring novel targets and local control modalities are in development.
Rhabdomyosarcoma , Sarcoma , Soft Tissue Neoplasms , Adult , Adolescent , Child , Humans , Sarcoma/drug therapy , Rhabdomyosarcoma/therapy , Soft Tissue Neoplasms/therapy , Soft Tissue Neoplasms/diagnosis , Survival Rate , Medical Oncology
