Rare Tumors in Children: Progress Through Collaboration

Pappo, Alberto S.; Furman, Wayne L.; Schultz, Kris Ann P.; Ferrari, Andrea; Helman, Lee J.; Krailo, Mark

doi:10.1200/jco.2014.59.3632

Cited by 43 publications

(25 citation statements)

References 55 publications

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“…24 Notably, there has been tremendous success in the advancement of treatment for many childhood cancers, in large part because treatment for children's cancers has been concentrated in specialized centers and the joint research collaborations among these centers. [25][26][27] Among solid tumors, 59% of rare cancers are diagnosed at regional/distant stages compared with 45% of all common cancers. Progress in screening and detection of cancer has generally been limited to more common cancer sites.…”

Section: Discussionmentioning

confidence: 99%

The burden of rare cancers in the United States

DeSantis

Kramer

Jemal

2017

CA A Cancer J Clinicians

138

105

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There are limited published data on the burden of rare cancers in the United States. By using data from the North American Association of Central Cancer Registries and the Surveillance, Epidemiology, and End Results program, the authors provide information on incidence rates, stage at diagnosis, and survival for more than 100 rare cancers (defined as an incidence of fewer than 6 cases per 100,000 individuals per year) in the United States. Overall, approximately 20% of patients with cancer in the United States are diagnosed with a rare cancer. Rare cancers make up a larger proportion of cancers diagnosed in Hispanic (24%) and Asian/Pacific Islander (22%) patients compared with non-Hispanic blacks (20%) and non-Hispanic whites (19%). More than two-thirds (71%) of cancers occurring in children and adolescents are rare cancers compared with less than 20% of cancers diagnosed in patients aged 65 years and older. Among solid tumors, 59% of rare cancers are diagnosed at regional or distant stages compared with 45% of common cancers. In part because of this stage distribution, 5-year relative survival is poorer for patients with a rare cancer compared with those diagnosed with a common cancer among both males (55% vs 75%) and females (60% vs 74%). However, 5-year relative survival is substantially higher for children and adolescents diagnosed with a rare cancer (82%) than for adults (46% for ages 65-79 years). Continued efforts are needed to develop interventions for prevention, early detection, and treatment to reduce the burden of rare cancers. Such discoveries can often advance knowledge for all cancers. CA Cancer J Clin 2017. © 2017 American Cancer Society. CA Cancer J Clin 2017;67:261-272. © 2017 American Cancer Society.

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Section: Discussionmentioning

confidence: 99%

The burden of rare cancers in the United States

DeSantis

Kramer

Jemal

2017

CA A Cancer J Clinicians

138

105

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“…COG’s Rare Tumor Committee also includes 3 other tumor types: retinoblastoma, liver tumors, and germ cell tumors. These 4 categories account for 15% to 20% of all childhood malignancies …”

Section: Introductionmentioning

confidence: 99%

“…These 4 categories account for 15% to 20% of all childhood malignancies. 4,5 Initiatives to study rare childhood tumors also include the creation of international disease-specific registries and clinics. 6 These include registries for pleuropulmonary blastoma, 7 pediatric adrenocortical carcinoma, 8 nuclear protein of the testis (NUT) midline carcinoma (http://www.nmcregistry.org/), and ovarian and testicular stromal tumors Cancer August 1, 2019 (http://www.otstregistry.org/), as well as an annual centralized clinic for pediatric gastrointestinal stromal tumor (https://ccr.cancer.gov/gist).…”

Section: Introductionmentioning

confidence: 99%

Rare cancers in childhood and adolescence in Brazil: First report of data from 19 population‐based cancer registries

Balmant

Reis²,

Santos

et al. 2019

Cancer

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Background Rare childhood cancer is challenging to define. The Italian Pediatric Rare Tumor (TREP) Study considers rare tumors to include solid malignancies characterized by an annual incidence rate of <2 cases per 1 million and not enrolled in clinical trials. The objective of the current study was to analyze the population incidence rate of rare tumors among children and adolescents (those aged birth‐19 years) in Brazil. Methods Incidence data were obtained from 19 population‐based cancer registries covering the 5 geographic regions in Brazil. Newly diagnosed cases were selected according to the TREP definition, using the International Classification of Diseases for Oncology. To calculate the crude incidence rate, the numbers of incident children and adolescents with a specific rare cancer were divided by the corresponding person‐years lived for the population aged <20 years during the same period. Results Two tumors had an incidence rate that was >2 cases per 1 million (thyroid and skin cancers) in adolescents only. Several tumors demonstrated variations in incidence across the Brazilian regions. Adrenocortical carcinoma had a high incidence rate (4 cases per 1 million) in the south region among children aged <10 years. Thyroid and skin carcinoma had higher incidence rates in the midwest, southeast, and south regions. Conclusions Due to the extraordinary rarity of these events, networking is important for improving basic research, clinical studies, and trials. Centralization of diagnosis is the only way to improve the diagnosis and treatment of children affected by these rare diseases. The registration and surveillance of rare pediatric cancers are crucial from a public health point of view, and therefore the quality of registration has to be improved.

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“…Despite the well-elaborated management strategies for the most common childhood malignancies, childhood cancer remains the second leading cause of illness-related mortality among children in developed countries (2). 3 Pediatric very rare tumors (VRTs) represent a particular subset of childhood cancers, comprising approximately 9 to 11% of all malignancies occurring in children below the age of 20, 75% of them diagnosed between the ages of 15 to 19 years old (3,4). The European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) defines a VRT as any solid malignancy or borderline tumor that has an incidence rate of ≤ 2 per million per year and/or is not considered for treatment in clinical trials (5).…”

Section: Introductionmentioning

confidence: 99%

The impact of incorrect registration on survival rate of children with very rare tumors

Šalaševičius

Rutkauskienė

Vincerževskienė³

et al. 2019

Preprint

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Background: Pediatric very rare tumors (VRTs) represent a heterogeneous subset of childhood malignancies, with reliable survival rate estimations depending dramatically on each (un)registered case. The current study aimed to evaluate the number of VRTs among Lithuanian children and the change in treatment outcome over the 16 year study period as well as to assess the impact of the registration status on survival estimation. Methods . We performed a population-based retrospective analysis across children below 18 years old diagnosed with VRTs in Lithuania between the years 2000 to 2015. The identified cases were then crosschecked with the Lithuanian Cancer Registry (a population-based epidemiology cancer registry) for the registration and survival status. A five year overall survival (OS 5y ) was calculated using Kaplan-Meier estimation method. Results . Forty-four children affected by VRTs were identified within the defined time frame. Nine of them (20.5%) were not reported to the Lithuanian Cancer Registry at the time of diagnosis. The OS 5y of the entire cohort was 55.8%. The cure rate did not improve over the analyzed time periods – 54.2% in 2000-2007 vs 49.4% in 2008-2015. The OS 5y differed significantly between registered (n=35) and unregistered (n=9) cohorts: 45.1% vs 100%, respectively (p=0.016). The tumor progression was responsible for treatment failure in 95% of cases. Conclusions. The OS 5y of all analyzed children affected by VRT was lower as compared to the other childhood cancers. The survival rate of the unregistered patients was significantly superior that mislead interpretation of treatment outcome. Meticulous registration of VRTs is crucial for correct evaluation of treatment outcome, especially across small countries with fewer numbers of cases.

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Rare Tumors in Children: Progress Through Collaboration

Cited by 43 publications

References 55 publications

The burden of rare cancers in the United States

The burden of rare cancers in the United States

Rare cancers in childhood and adolescence in Brazil: First report of data from 19 population‐based cancer registries

The impact of incorrect registration on survival rate of children with very rare tumors

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