Developing therapies for rare tumors: opportunities, challenges and progress

Bradford, Diana; Reilly, Karlyne M.; Widemann, Brigitte C.; Sandler, Abby; Kummar, Shivaani

doi:10.1517/21678707.2016.1120663

Cited by 5 publications

(5 citation statements)

References 55 publications

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“…With respect to treatment, the challenges facing the treatment of vulvar malignancies can be addressed by changes in regulations for drug development and by national and international initiatives to promote the clinical research in the field of rare diseases [ 38 , 57 , 58 ]. There are promising lines of research, such as the investigation of potentially targetable markers (for example, the prostaglandin E2 receptor 4) [ 59 ] and of new potential immunotherapeutic approaches to the management of the disease (for example, the use of state-of-the-art immune checkpoint inhibitors) [ 60 ].…”

Section: Discussionmentioning

confidence: 99%

Trends in Net Survival from Vulvar Squamous Cell Carcinoma in Italy (1990–2015)

Mancini

Bucchi

Zamagni

et al. 2023

JCM

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(1) Objective: In many Western countries, survival from vulvar squamous cell carcinoma (VSCC) has been stagnating for decades or has increased insufficiently from a clinical perspective. In Italy, previous studies on cancer survival have not taken vulvar cancer into consideration or have pooled patients with vulvar and vaginal cancer. To bridge this knowledge gap, we report the trend in survival from vulvar cancer between 1990 and 2015. (2) Methods: Thirty-eight local cancer registries covering 49% of the national female population contributed the records of 6274 patients. Study endpoints included 1- and 2-year net survival (NS) calculated using the Pohar-Perme estimator and 5-year NS conditional on having survived two years (5|2-year CNS). The significance of survival trends was assessed with the Wald test on the coefficient of the period of diagnosis, entered as a continuous regressor in a Poisson regression model. (3) Results: The median patient age was stable at 76 years. One-year NS decreased from 83.9% in 1990–2001 to 81.9% in 2009–2015 and 2-year NS from 72.2% to 70.5%. Five|2-year CNS increased from 85.7% to 86.7%. These trends were not significant. In the age stratum 70–79 years, a weakly significant decrease in 2-year NS from 71.4% to 65.7% occurred. Multivariate analysis adjusting for age group at diagnosis and geographic area showed an excess risk of death at 5|2-years, of borderline significance, in 2003–2015 versus 1990–2002. (4) Conclusions: One- and 2-year NS and 5|2-year CNS showed no improvements. Current strategies for VSCC control need to be revised both in Italy and at the global level.

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Section: Discussionmentioning

confidence: 99%

Trends in Net Survival from Vulvar Squamous Cell Carcinoma in Italy (1990–2015)

Mancini

Bucchi

Zamagni

et al. 2023

JCM

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“… 7 Furthermore, for some very rare pediatric malignancies, natural history is not fully understood, there are no standard treatments, and conducting clinical trials is difficult because of small patient numbers. 8 , 9 These limitations, combined with the comparatively low incidence of pediatric compared with adult cancers, 1 , 2 underscore the necessity to collect, aggregate, and make accessible the research and clinical data that could enable a better understanding of the drivers of these cancers.…”

Section: Introductionmentioning

confidence: 99%

The Childhood Cancer Data Initiative: Using the Power of Data to Learn From and Improve Outcomes for Every Child and Young Adult With Pediatric Cancer

Flores-Toro,

Jagu,

Armstrong

et al. 2023

JCO

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Data-driven basic, translational, and clinical research has resulted in improved outcomes for children, adolescents, and young adults (AYAs) with pediatric cancers. However, challenges in sharing data between institutions, particularly in research, prevent addressing substantial unmet needs in children and AYA patients diagnosed with certain pediatric cancers. Systematically collecting and sharing data from every child and AYA can enable greater understanding of pediatric cancers, improve survivorship, and accelerate development of new and more effective therapies. To accomplish this goal, the Childhood Cancer Data Initiative (CCDI) was launched in 2019 at the National Cancer Institute. CCDI is a collaborative community endeavor supported by a 10-year, $50-million (in US dollars) annual federal investment. CCDI aims to learn from every patient diagnosed with a pediatric cancer by designing and building a data ecosystem that facilitates data collection, sharing, and analysis for researchers, clinicians, and patients across the cancer community. For example, CCDI's Molecular Characterization Initiative provides comprehensive clinical molecular characterization for children and AYAs with newly diagnosed cancers. Through these efforts, the CCDI strives to provide clinical benefit to patients and improvements in diagnosis and care through data-focused research support and to build expandable, sustainable data resources and workflows to advance research well past the planned 10 years of the initiative. Importantly, if CCDI demonstrates the success of this model for pediatric cancers, similar approaches can be applied to adults, transforming both clinical research and treatment to improve outcomes for all patients with cancer.

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“…Collectively, this results in 27% of cancers diagnosed in the United States qualifying as rare tumors, including all pediatric cancers (<18 years old). 1,2 Although pediatric cancers are rare, approximately 12,600 children less than 20 years old are diagnosed with cancer each year. Pediatric malignancies are the leading cause of disease-related mortality in children, more than cystic fibrosis, muscular dystrophy, asthma, and AIDS combined.…”

Section: Introductionmentioning

confidence: 99%

“…While the definition of a rare tumor varies across the world, the US National Cancer Institute (NCI) has defined a rare tumor as fewer than 15 cases per 100,000 people yearly or less than 200,000 prevalence in the United States and fewer than 40,000 new cases per year. Collectively, this results in 27% of cancers diagnosed in the United States qualifying as rare tumors, including all pediatric cancers (<18 years old) 1,2 …”

Section: Introductionmentioning

confidence: 99%

NCI intramural program approach to rare tumors: Natural history study of rare solid tumors in children and adults: A longitudinal, comprehensive data and biospecimen collection protocol

Wedekind,

Reilly,

Rivero

et al. 2023

Pediatric Blood & Cancer

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Rare tumors across the world are lacking adequate knowledge, resources, and community. Through partnership with patients, advocacy organizations, researchers, and clinicians, we have developed a comprehensive, longitudinal, prospective, and retrospective natural history protocol to collect, analyze, and share data on patients with rare tumors. A strong collaborative effort is vital to ensure success of enrollment, patient engagement, data collection, and analysis to ultimately develop clinical trials to improve outcomes for patients with rare cancers.

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Developing therapies for rare tumors: opportunities, challenges and progress

Cited by 5 publications

References 55 publications

Trends in Net Survival from Vulvar Squamous Cell Carcinoma in Italy (1990–2015)

Trends in Net Survival from Vulvar Squamous Cell Carcinoma in Italy (1990–2015)

The Childhood Cancer Data Initiative: Using the Power of Data to Learn From and Improve Outcomes for Every Child and Young Adult With Pediatric Cancer

NCI intramural program approach to rare tumors: Natural history study of rare solid tumors in children and adults: A longitudinal, comprehensive data and biospecimen collection protocol

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