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Medulloblastoma in the Modern Era: Review of Contemporary Trials, Molecular Advances, and Updates in Management.

ABSTRACT: Critical discoveries over the past two decades have transformed our understanding of medulloblastoma from a single entity into a clinically and biologically heterogeneous disease composed of at least four molecularly distinct subgroups with prognostically and therapeutically relevant genomic signatures. Contemporary clinical trials also have provided valuable insight guiding appropriate treatment strategies. Despite therapeutic and biological advances, medulloblastoma patients across the age spectrum experience tumor- and treatment-related morbidity and mortality. Using an updated risk stratification approach integrating both clinical and molecular features, ongoing research seeks to (1) cautiously reduce therapy and mitigate toxicity in low-average risk patients, and (2) thoughtfully intensify treatment with incorporation of novel, biologically guided agents for patients with high-risk disease. Herein, we review important historical and contemporary studies, discuss management updates, and summarize current knowledge of the biological landscape across unique pediatric, infant, young adult, and relapsed medulloblastoma populations.

SUBMITTER: Lazow MA

PROVIDER: S-EPMC9723091 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature

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Medulloblastoma in the Modern Era: Review of Contemporary Trials, Molecular Advances, and Updates in Management.

Lazow Margot A MA Palmer Joshua D JD Fouladi Maryam M Salloum Ralph R

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics 20220720 6

Critical discoveries over the past two decades have transformed our understanding of medulloblastoma from a single entity into a clinically and biologically heterogeneous disease composed of at least four molecularly distinct subgroups with prognostically and therapeutically relevant genomic signatures. Contemporary clinical trials also have provided valuable insight guiding appropriate treatment strategies. Despite therapeutic and biological advances, medulloblastoma patients across the age spe ...[more]

PMID: 35859223

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Project description:ImportancePulmonary arterial hypertension (PAH) is characterized by severe remodeling of the distal pulmonary arteries, increased pulmonary vascular resistance, and right ventricular dysfunction that promotes heart failure. Once regarded as largely untreatable, evidence-based decision making now guides clinical management of PAH and improves outcomes. However, misconceptions regarding the approach to PAH in the modern era are common and associated with substandard clinical care.ObservationsThe clinical profile of PAH has changed substantially since its original description. Patients are older at diagnosis than previously reported; disease severity appears greater in men compared with women; and patients with PAH in association with connective tissue disease are identified as a particularly high-risk subgroup. Risk stratification scales for PAH are now available at point of care, which inform treatment goals, including a 6-minute walk distance of greater than 440 m, peak volume of oxygen consumption of greater than 15 mL/min/kg, right atrial area of less than 18 cm2, cardiac index of greater than 2.5 L/min/m2, and absent or low symptom burden with routine physical activity. At present, 14 therapies targeting 6 PAH-specific molecular intermediaries are used clinically. Recent landmark trial data have demonstrated the critical importance of initial combination therapy in treatment-naive patients. These findings underscore a global shift in PAH that couples early disease detection with aggressive pharmacotherapy. Indeed, recent longitudinal data from patients receiving combination therapy show that the 3-year survival rate in PAH may be as high as 84% compared with 48% from the original National Institutes of Health registry on idiopathic PAH (1980-1985). Despite these gains, incomplete clinical evaluation and misdiagnosis by referring clinicians is common and associated with inappropriate therapy.Conclusions and relevanceCompared with the original clinical experience, PAH has evolved into a contemporary and treatable disease characterized by improved survival and a high standard for defining therapeutic success. However, underawareness among clinicians regarding the importance of early and accurate PAH diagnosis persists and is a potentially reversible cause of adverse outcome in this disease.

Dataset Information

Medulloblastoma in the Modern Era: Review of Contemporary Trials, Molecular Advances, and Updates in Management.

Publications

Medulloblastoma in the Modern Era: Review of Contemporary Trials, Molecular Advances, and Updates in Management.

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