Materiały źródłowe

• #1 Rhabdomyosarcoma – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK507721/

  Rhabdomyosarcoma (RMS) is a rare condition making up 3% of all pediatric cancers. However, RMS is the most common childhood and adolescent soft tissue sarcoma, comprising 50% of soft tissue sarcomas in individuals younger than 20. In the US, there are approximately 350 newly diagnosed patients each year. All histological subtypes of RMS have also been shown to be significantly more prevalent in males. RMS has a much lower incidence in adults, accounting for approximately 1% of solid cancers. […] Although the various subtypes of RMS can arise anywhere in the body, the embryonal histological type is the most common, and the head and neck is the most frequently involved area. RMS involving the extremities is more frequently observed with the alveolar subtype. The pleomorphic and alveolar subtypes have the highest rates of metastases and, consequently, the poorest prognosis compared to other histologic types. The most common metastatic sites include the lungs, bone marrow, and lymph nodes.

• #2 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8125975/

  Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children, yet little is known about its etiology. Studies that examine either environmental exposures or germline genetic predisposition in RMS have begun to identify factors that contribute to this malignancy. Here, we summarize epidemiological reports of RMS incidence in terms of several factors, including age at diagnosis, biological sex, and geographic location. […] RMS has an incidence of approximately 4.71 per million children and adolescents less than 20 years of age in the United States. […] Importantly, survival for RMS is poor. In the most recently completed Childrens Oncology Group (COG) intermediate-risk study, the four-year event-free survival was 63%. For individuals with metastatic disease, the three-year event-free survival is less than 20%, despite multi-agent therapies.

• #2 Key Statistics for Rhabdomyosarcoma | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/about/key-statistics.html

  About 400 to 500 new cases of rhabdomyosarcoma (RMS) occur each year in the United States. The number of new cases has not changed much over the past few decades. […] Most rhabdomyosarcomas are diagnosed in children and teens, with more than half of them in children younger than 10 years old. […] About 3% of all childhood cancers are RMS. […] RMS is slightly more common in boys than in girls. […] No particular race or ethnic group seems to have an unusually high rate of RMS.

• #3 Rhabdomyosarcoma in childhood and adolescence: Epidemiology, pathology, and molecular pathogenesis – UpToDate

  https://www.uptodate.com/contents/rhabdomyosarcoma-in-childhood-and-adolescence-epidemiology-pathology-and-molecular-pathogenesis

  RMS is the most common soft tissue sarcoma of childhood, accounting for one-half of all diagnoses in this age group. However, they are rare, representing only 3 to 4 percent of pediatric cancers overall. Approximately 350 new cases are diagnosed in the United States each year, and the annual incidence in children, adolescents, and young adults under the age of 20 is 4.58 cases per one million. […] Two-thirds of cases are diagnosed in children younger than six years of age, and there is a small male predominance (male to female ratio between 1.3 and 1.5). The incidence in Black patients is higher than in White, most notably in those 15 to 19 years. The incidence appears to be lower in Asian (Indian sub-continent and West Indian ethnic origin) when compared with predominantly White populations.

• #3 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Childhood rhabdomyosarcoma is a soft tissue malignant tumor of mesenchymal origin. It accounts for approximately 2.7% of cancer cases among children aged 0 to 14 years and 1.4% of the cases among adolescents and young adults aged 15 to 19 years. The incidence is 4.6 cases per 1 million children younger than 20 years, which translates into about 350 new cases per year. Fifty percent of these cases are seen in the first decade of life. […] The 2020 World Health Organization classification distinguishes four histological subtypes of rhabdomyosarcoma, including embryonal, alveolar, spindle cell/sclerosing, and pleomorphic. While these subtypes classify rhabdomyosarcoma into prognostically useful histological categories, FOXO1 gene fusions uniquely occur in alveolar histology tumors; however, not all tumors that have been classified as alveolar histology have a FOXO1 fusion.

• #4 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://www.mdpi.com/2077-0383/10/9/2028

  Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children, yet little is known about its etiology. Studies that examine either environmental exposures or germline genetic predisposition in RMS have begun to identify factors that contribute to this malignancy. Here, we summarize epidemiological reports of RMS incidence in terms of several factors, including age at diagnosis, biological sex, and geographic location. […] RMS has an incidence of approximately 4.71 per million children and adolescents less than 20 years of age in the United States. This is similar to other sarcomas, including osteosarcoma (5.09 per million) and Ewing sarcoma (2.95 per million), but lower than the most frequent pediatric cancer—acute lymphoblastic leukemia (ALL, 32.59 per million). […] A primary objective of epidemiology is to study the distribution and determinants of disease in human populations. Assessments evaluating the distribution of disease (e.g., incidence, overall survival) are considered descriptive epidemiology, whereas studies evaluating the determinants of disease (e.g., environmental exposures, nutritional status) are considered analytic epidemiology.

• #5 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8125975/

  Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children, yet little is known about its etiology. Studies that examine either environmental exposures or germline genetic predisposition in RMS have begun to identify factors that contribute to this malignancy. Here, we summarize epidemiological reports of RMS incidence in terms of several factors, including age at diagnosis, biological sex, and geographic location. […] RMS has an incidence of approximately 4.71 per million children and adolescents less than 20 years of age in the United States. […] Importantly, survival for RMS is poor. In the most recently completed Childrens Oncology Group (COG) intermediate-risk study, the four-year event-free survival was 63%. For individuals with metastatic disease, the three-year event-free survival is less than 20%, despite multi-agent therapies.

• #6 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://www.mdpi.com/2077-0383/10/9/2028

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. This is in contrast with the steady increases in the incidences observed for other pediatric cancers. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.21–1.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.27–1.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. Based on a report including 59 cancer registries from 19 European countries, the overall incidence of RMS in Europe was 5.4 cases per million in children <15 years of age from 1978–1997. [...] In sub-Saharan Africa, the RMS incidence is highly variable. For example, a study of 16 population-based registries within the African Cancer Registry Network estimated that the incidence rate of childhood RMS in Western Africa ranged from 0.6 to 8.6 per million compared to 2.4 to 2.5 per million in Southern Africa and 2.6 to 16.3 per million in Eastern Africa. [...] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #7 Orphanet: Rhabdomyosarcoma

  https://www.orpha.net/en/disease/detail/780

  The annual incidence is 1/170,000. In children younger than 15 years, the annual incidence is estimated at 1/244,000. […] Rhabdomyosarcoma can develop anywhere in the body, including in sites where striated muscle does not normally occur. […] Rhabdomyosarcoma has a high risk of local relapse, a high risk of local extension to lymph nodes and a lower risk of metastases. […] The objective of current protocols is to avoid radiotherapy, if possible, in order to reduce long term sequelae.

• #8 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://www.mdpi.com/2077-0383/10/9/2028

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. This is in contrast with the steady increases in the incidences observed for other pediatric cancers. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.21–1.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.27–1.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. Based on a report including 59 cancer registries from 19 European countries, the overall incidence of RMS in Europe was 5.4 cases per million in children <15 years of age from 1978–1997. [...] In sub-Saharan Africa, the RMS incidence is highly variable. For example, a study of 16 population-based registries within the African Cancer Registry Network estimated that the incidence rate of childhood RMS in Western Africa ranged from 0.6 to 8.6 per million compared to 2.4 to 2.5 per million in Southern Africa and 2.6 to 16.3 per million in Eastern Africa. [...] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #9 Rhabdomyosarcoma epidemiology and demographics – wikidoc

  https://www.wikidoc.org/index.php/Rhabdomyosarcoma_epidemiology_and_demographics

  Rhabdomyosarcoma is considered as a most common soft tissue cancers among children and adolescents and it is the third most common extracranial solid tumors during childhood which comes after neuroblastoma and wilms tumor. Rhabdomyosarcoma is responsible for 50% of soft tissue tumors during childhood and 5% of all pediatrics cancers. Almost 350 new cases of rhabdomyosarcoma are diagnosed annually. The incidence of rhabdomyosarcoma is about 0.43 per 100,000 annually among children, adolescents, and young adults under 20 years old. The incidence may depend on the histologic subtype of rhabdomyosarcoma: Embryonal rhabdomyosarcoma peaks in 0 to 4 year age group at approximately 0.4 cases per 100,000 children, with a lower rate in adolescents, approximately 0.15 cases per 100,000 adolescents. The incidence of alveolar rhabdomyosarcoma does not vary by gender and is constant from ages 0 to 19 years at approximately 0.1 case per 100,000 children and adolescents. In patients with localized disease, the estimated 5-year survival rate is greater than 80% after using surgery, radiation therapy, and chemotherapy for treatment. In patients with metastatic lesions, the estimated 5-year event-free survival is less than 30%. Approximately two-third of all cases happen under 6 years old. The peak incidence of rhabdomyosarcoma is between 2-6 years and 10-18 years. The incidence of rhabdomyosarcoma is higher among black patients than white patients particularly between 15-19 years. The incidence of rhabdomyosarcoma is a little more predominant in males than females (about 1.3:1-1.5:1). The incidence of rhabdomyosarcoma is higher among Caucasian population in comparison to Asian population.

• #10 Pediatric Rhabdomyosarcoma: Practice Essentials, Background, Pathophysiology

  https://emedicine.medscape.com/article/988803-overview

  The incidence is 6 cases per 1,000,000 population per year (approximately 250 cases) in children and adolescents younger than 15 years. […] No notable geographic predilection is reported. […] No racial predilection is obvious. Overall, the male-to-female ratio is 1.2-1.4:1. Differences are observed according to the site of primary disease. […] Approximately 87% of patients are younger than 15 years, and 13% of patients are aged 15-21 years. Rhabdomyosarcoma rarely affects adults. Age-related differences are observed for the different sites of primary disease. Two age peaks tend to be associated with different locations. Patients aged 2-6 years tend to have head and neck or GU tract primary tumors, whereas adolescents aged 14-18 years tend to have primary tumors in extremity, truncal, or paratesticular locations. […] In an analysis of data collected by the Surveillance, Epidemiology, and End Results (SEER) program, mortality was highly related to age, site, and histology.

• #11 Rhabdomyosarcoma epidemiology and demographics – wikidoc

  https://www.wikidoc.org/index.php/Rhabdomyosarcoma_epidemiology_and_demographics

  Rhabdomyosarcoma is considered as a most common soft tissue cancers among children and adolescents and it is the third most common extracranial solid tumors during childhood which comes after neuroblastoma and wilms tumor. Rhabdomyosarcoma is responsible for 50% of soft tissue tumors during childhood and 5% of all pediatrics cancers. Almost 350 new cases of rhabdomyosarcoma are diagnosed annually. The incidence of rhabdomyosarcoma is about 0.43 per 100,000 annually among children, adolescents, and young adults under 20 years old. The incidence may depend on the histologic subtype of rhabdomyosarcoma: Embryonal rhabdomyosarcoma peaks in 0 to 4 year age group at approximately 0.4 cases per 100,000 children, with a lower rate in adolescents, approximately 0.15 cases per 100,000 adolescents. The incidence of alveolar rhabdomyosarcoma does not vary by gender and is constant from ages 0 to 19 years at approximately 0.1 case per 100,000 children and adolescents. In patients with localized disease, the estimated 5-year survival rate is greater than 80% after using surgery, radiation therapy, and chemotherapy for treatment. In patients with metastatic lesions, the estimated 5-year event-free survival is less than 30%. Approximately two-third of all cases happen under 6 years old. The peak incidence of rhabdomyosarcoma is between 2-6 years and 10-18 years. The incidence of rhabdomyosarcoma is higher among black patients than white patients particularly between 15-19 years. The incidence of rhabdomyosarcoma is a little more predominant in males than females (about 1.3:1-1.5:1). The incidence of rhabdomyosarcoma is higher among Caucasian population in comparison to Asian population.

• #12 Pediatric Rhabdomyosarcoma: Practice Essentials, Background, Pathophysiology

  https://emedicine.medscape.com/article/939156-overview

  The incidence is 6 cases per 1,000,000 population per year (approximately 250 cases) in children and adolescents younger than 15 years. […] No notable geographic predilection is reported. […] No racial predilection is obvious. […] Overall, the male-to-female ratio is 1.2-1.4:1. Differences are observed according to the site of primary disease. […] Approximately 87% of patients are younger than 15 years, and 13% of patients are aged 15-21 years. Rhabdomyosarcoma rarely affects adults. Age-related differences are observed for the different sites of primary disease. Two age peaks tend to be associated with different locations. Patients aged 2-6 years tend to have head and neck or GU tract primary tumors, whereas adolescents aged 14-18 years tend to have primary tumors in extremity, truncal, or paratesticular locations. […] In an analysis of data collected by the Surveillance, Epidemiology, and End Results (SEER) program, mortality was highly related to age, site, and histology.

• #13 Rhabdomyosarcoma in childhood and adolescence: Epidemiology, pathology, and molecular pathogenesis – UpToDate

  https://www.uptodate.com/contents/rhabdomyosarcoma-in-childhood-and-adolescence-epidemiology-pathology-and-molecular-pathogenesis

  RMS is the most common soft tissue sarcoma of childhood, accounting for one-half of all diagnoses in this age group. However, they are rare, representing only 3 to 4 percent of pediatric cancers overall. Approximately 350 new cases are diagnosed in the United States each year, and the annual incidence in children, adolescents, and young adults under the age of 20 is 4.58 cases per one million. […] Two-thirds of cases are diagnosed in children younger than six years of age, and there is a small male predominance (male to female ratio between 1.3 and 1.5). The incidence in Black patients is higher than in White, most notably in those 15 to 19 years. The incidence appears to be lower in Asian (Indian sub-continent and West Indian ethnic origin) when compared with predominantly White populations.

• #14 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8125975/

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. […] While there has been an increase in the incidence of ARMS (annual percent change (APC) = 4.20, 95% confidence interval (CI) = 2.605.82), this is likely due to changes in diagnostic criteria. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.211.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.271.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. […] In sub-Saharan Africa, the RMS incidence is highly variable. […] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #15 Key Statistics for Rhabdomyosarcoma | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/about/key-statistics.html

  About 400 to 500 new cases of rhabdomyosarcoma (RMS) occur each year in the United States. The number of new cases has not changed much over the past few decades. […] Most rhabdomyosarcomas are diagnosed in children and teens, with more than half of them in children younger than 10 years old. […] About 3% of all childhood cancers are RMS. […] RMS is slightly more common in boys than in girls. […] No particular race or ethnic group seems to have an unusually high rate of RMS.

• #16 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://www.mdpi.com/2077-0383/10/9/2028

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. This is in contrast with the steady increases in the incidences observed for other pediatric cancers. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.21–1.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.27–1.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. Based on a report including 59 cancer registries from 19 European countries, the overall incidence of RMS in Europe was 5.4 cases per million in children <15 years of age from 1978–1997. [...] In sub-Saharan Africa, the RMS incidence is highly variable. For example, a study of 16 population-based registries within the African Cancer Registry Network estimated that the incidence rate of childhood RMS in Western Africa ranged from 0.6 to 8.6 per million compared to 2.4 to 2.5 per million in Southern Africa and 2.6 to 16.3 per million in Eastern Africa. [...] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #17 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://www.mdpi.com/2077-0383/10/9/2028

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. This is in contrast with the steady increases in the incidences observed for other pediatric cancers. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.21–1.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.27–1.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. Based on a report including 59 cancer registries from 19 European countries, the overall incidence of RMS in Europe was 5.4 cases per million in children <15 years of age from 1978–1997. [...] In sub-Saharan Africa, the RMS incidence is highly variable. For example, a study of 16 population-based registries within the African Cancer Registry Network estimated that the incidence rate of childhood RMS in Western Africa ranged from 0.6 to 8.6 per million compared to 2.4 to 2.5 per million in Southern Africa and 2.6 to 16.3 per million in Eastern Africa. [...] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #18 Key Statistics for Rhabdomyosarcoma | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/about/key-statistics.html

  About 400 to 500 new cases of rhabdomyosarcoma (RMS) occur each year in the United States. The number of new cases has not changed much over the past few decades. […] Most rhabdomyosarcomas are diagnosed in children and teens, with more than half of them in children younger than 10 years old. […] About 3% of all childhood cancers are RMS. […] RMS is slightly more common in boys than in girls. […] No particular race or ethnic group seems to have an unusually high rate of RMS.

• #19 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Male patients have a higher incidence of embryonal tumors, and Black patients have a slightly higher incidence of alveolar tumors. […] The Children’s Oncology Group (COG) performed retrospective exome sequencing on germline DNA to determine the prevalence of 63 autosomal dominant cancer-predisposing genes in 615 patients with newly diagnosed rhabdomyosarcoma. They identified germline cancer-predisposition (pathogenic or likely pathogenic) variants in 45 patients with rhabdomyosarcoma (7.3%; all FOXO1 fusion negative) across 15 autosomal dominant genes. […] The prognosis for children or adolescents with rhabdomyosarcoma is related to many clinical and biological factors, including age, site of origin, tumor size, resectability, histopathological subtype, PAX3::FOXO1 or PAX7::FOXO1 gene fusion status, metastases at diagnosis, lymph node involvement at diagnosis, bone marrow involvement, biological characteristics, response to therapy, circulating tumor DNA and RNA.

• #20 Rhabdomyosarcoma in childhood and adolescence: Epidemiology, pathology, and molecular pathogenesis – UpToDate

  https://www.uptodate.com/contents/rhabdomyosarcoma-in-childhood-and-adolescence-epidemiology-pathology-and-molecular-pathogenesis

  RMS is the most common soft tissue sarcoma of childhood, accounting for one-half of all diagnoses in this age group. However, they are rare, representing only 3 to 4 percent of pediatric cancers overall. Approximately 350 new cases are diagnosed in the United States each year, and the annual incidence in children, adolescents, and young adults under the age of 20 is 4.58 cases per one million. […] Two-thirds of cases are diagnosed in children younger than six years of age, and there is a small male predominance (male to female ratio between 1.3 and 1.5). The incidence in Black patients is higher than in White, most notably in those 15 to 19 years. The incidence appears to be lower in Asian (Indian sub-continent and West Indian ethnic origin) when compared with predominantly White populations.

• #21 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8125975/

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. […] While there has been an increase in the incidence of ARMS (annual percent change (APC) = 4.20, 95% confidence interval (CI) = 2.605.82), this is likely due to changes in diagnostic criteria. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.211.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.271.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. […] In sub-Saharan Africa, the RMS incidence is highly variable. […] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #22 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://www.mdpi.com/2077-0383/10/9/2028

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. This is in contrast with the steady increases in the incidences observed for other pediatric cancers. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.21–1.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.27–1.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. Based on a report including 59 cancer registries from 19 European countries, the overall incidence of RMS in Europe was 5.4 cases per million in children <15 years of age from 1978–1997. [...] In sub-Saharan Africa, the RMS incidence is highly variable. For example, a study of 16 population-based registries within the African Cancer Registry Network estimated that the incidence rate of childhood RMS in Western Africa ranged from 0.6 to 8.6 per million compared to 2.4 to 2.5 per million in Southern Africa and 2.6 to 16.3 per million in Eastern Africa. [...] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #23 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8125975/

  Between 1975 and 2005, there was no significant change in the incidence of RMS overall or ERMS in the United States. […] While there has been an increase in the incidence of ARMS (annual percent change (APC) = 4.20, 95% confidence interval (CI) = 2.605.82), this is likely due to changes in diagnostic criteria. […] The incidence of RMS also varies by biological sex. Specifically, RMS incidence is higher in males compared to females (rate ratio (RR) = 1.37, 95% CI = 1.211.56); this phenomenon is driven by the predominance of male ERMS diagnoses (RR = 1.51 male to female, 95% CI = 1.271.80). […] The RMS incidence is modestly elevated in European countries compared to the United States. […] In sub-Saharan Africa, the RMS incidence is highly variable. […] Despite the literature showing racial disparities in other pediatric cancers, there is little evidence to suggest that the incidence of RMS varies by race/ethnicity.

• #24 Survival Rates for Rhabdomyosarcoma by Risk Group | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/detection-diagnosis-staging/staging-survival-rates.html

  Survival rates for rhabdomyosarcoma can give you an idea of what percentage of people in a similar situation (such as with the same type and stage of cancer) are still alive a certain amount of time after they were diagnosed. […] For a person with RMS, the risk group is important in estimating their outlook. […] The overall 5-year survival for children with RMS is about 70%, while survival in adults is lower. […] Overall, the survival rate for children in the low-risk group ranges from about 70% to over 90%. […] For those in the intermediate-risk group, the survival rates range from about 50% to about 70%. […] The survival rate in this group is generally around 20% to 30%. […] Even when taking risk groups and other factors into account, survival rates are at best rough estimates.

• #25 Rhabdomyosarcoma: Symptoms, Prognosis & Treatment

  https://my.clevelandclinic.org/health/diseases/6226-rhabdomyosarcoma

  Rhabdomyosarcoma mostly affects children and teenagers but can affect adults as well. Each year, between 400 and 500 people in the U.S. receive a diagnosis of rhabdomyosarcoma. […] Rhabdomyosarcoma symptoms may resemble less serious conditions. […] Oncologists who care for children with rhabdomyosarcoma classify the condition by risk group. […] Risk group classifications are low risk, intermediate risk and high risk. […] Survival rates vary widely depending on factors like the type of rhabdomyosarcoma, risk group classification and whether the condition comes back after treatment. Overall, 70% of children with this condition were alive five years after diagnosis. The five-year survival rate for adults is 20%. […] Rhabdomyosarcoma is a very rare cancer.

• #26 Survival Rates for Rhabdomyosarcoma by Risk Group | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/detection-diagnosis-staging/staging-survival-rates.html

  Survival rates for rhabdomyosarcoma can give you an idea of what percentage of people in a similar situation (such as with the same type and stage of cancer) are still alive a certain amount of time after they were diagnosed. […] For a person with RMS, the risk group is important in estimating their outlook. […] The overall 5-year survival for children with RMS is about 70%, while survival in adults is lower. […] Overall, the survival rate for children in the low-risk group ranges from about 70% to over 90%. […] For those in the intermediate-risk group, the survival rates range from about 50% to about 70%. […] The survival rate in this group is generally around 20% to 30%. […] Even when taking risk groups and other factors into account, survival rates are at best rough estimates.

• #27 Survival Rates for Rhabdomyosarcoma by Risk Group | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/detection-diagnosis-staging/staging-survival-rates.html

  Survival rates for rhabdomyosarcoma can give you an idea of what percentage of people in a similar situation (such as with the same type and stage of cancer) are still alive a certain amount of time after they were diagnosed. […] For a person with RMS, the risk group is important in estimating their outlook. […] The overall 5-year survival for children with RMS is about 70%, while survival in adults is lower. […] Overall, the survival rate for children in the low-risk group ranges from about 70% to over 90%. […] For those in the intermediate-risk group, the survival rates range from about 50% to about 70%. […] The survival rate in this group is generally around 20% to 30%. […] Even when taking risk groups and other factors into account, survival rates are at best rough estimates.

• #28 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8125975/

  Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children, yet little is known about its etiology. Studies that examine either environmental exposures or germline genetic predisposition in RMS have begun to identify factors that contribute to this malignancy. Here, we summarize epidemiological reports of RMS incidence in terms of several factors, including age at diagnosis, biological sex, and geographic location. […] RMS has an incidence of approximately 4.71 per million children and adolescents less than 20 years of age in the United States. […] Importantly, survival for RMS is poor. In the most recently completed Childrens Oncology Group (COG) intermediate-risk study, the four-year event-free survival was 63%. For individuals with metastatic disease, the three-year event-free survival is less than 20%, despite multi-agent therapies.

• #29 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8125975/

  Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children, yet little is known about its etiology. Studies that examine either environmental exposures or germline genetic predisposition in RMS have begun to identify factors that contribute to this malignancy. Here, we summarize epidemiological reports of RMS incidence in terms of several factors, including age at diagnosis, biological sex, and geographic location. […] RMS has an incidence of approximately 4.71 per million children and adolescents less than 20 years of age in the United States. […] Importantly, survival for RMS is poor. In the most recently completed Childrens Oncology Group (COG) intermediate-risk study, the four-year event-free survival was 63%. For individuals with metastatic disease, the three-year event-free survival is less than 20%, despite multi-agent therapies.

• #30 Rhabdomyosarcoma – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK507721/

  Recommendations concerning continued surveillance imaging for recurrent disease also vary between Europe and the US. Current guidelines in Europe recommend surveillance imaging every 4 months for 2 years. In the US, there are no set guidelines; however, most clinicians follow current Children’s Oncology Group recommendations to repeat imaging every 4 months for 4 years. Due to known complications from radiation exposure, particularly in children, many experts have recently advocated for US clinicians to adopt European surveillance guidelines as both protocols have similar outcomes. The management of patients with RMS is continuously evolving as new, evidence-based results emerge from clinical trials of treatment advancements.

• #31

  https://link.springer.com/article/10.1007/s00247-023-05596-8

  The current European imaging guidelines advise that surveillance imaging should be performed three times a year in the first 2 years after the end of therapy, reducing the period of surveillance of the primary tumour from 5 to 2 years. […] However, taking into account the limited evidence for a beneficial effect of surveillance imaging on survival, we would like to echo the words of McHugh and Roebuck: „To resolve the question of the value of ongoing imaging surveillance, patients should be randomized to either of two arms once therapy is complete; one with routine surveillance imaging, the other without surveillance imaging (relying solely on clinical follow-up).”

• #32 Rhabdomyosarcoma – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK507721/

  Recommendations concerning continued surveillance imaging for recurrent disease also vary between Europe and the US. Current guidelines in Europe recommend surveillance imaging every 4 months for 2 years. In the US, there are no set guidelines; however, most clinicians follow current Children’s Oncology Group recommendations to repeat imaging every 4 months for 4 years. Due to known complications from radiation exposure, particularly in children, many experts have recently advocated for US clinicians to adopt European surveillance guidelines as both protocols have similar outcomes. The management of patients with RMS is continuously evolving as new, evidence-based results emerge from clinical trials of treatment advancements.

• #33 Rhabdomyosarcoma – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK507721/

  Recommendations concerning continued surveillance imaging for recurrent disease also vary between Europe and the US. Current guidelines in Europe recommend surveillance imaging every 4 months for 2 years. In the US, there are no set guidelines; however, most clinicians follow current Children’s Oncology Group recommendations to repeat imaging every 4 months for 4 years. Due to known complications from radiation exposure, particularly in children, many experts have recently advocated for US clinicians to adopt European surveillance guidelines as both protocols have similar outcomes. The management of patients with RMS is continuously evolving as new, evidence-based results emerge from clinical trials of treatment advancements.

• #34 No additional benefit with intensive surveillance in relapsed rhabdomyosarcoma – BJMO

  https://www.bjmo.be/no-additional-benefit-with-intensive-surveillance-in-relapsed-rhabdomyosarcoma/

  Intensive surveillance after therapy is needed in pediatric patients treated for rhabdomyosarcoma (RMS). This is done as around a third of these patients experience relapse, and three-year survival after relapse is only 37%. […] The usefulness of this protocol for patient benefit is unclear. A recent retrospective study has analyzed the clinical value of surveillance in relapsed RMS group. […] The study found no survival advantage for RMS patients in intensive surveillance of clinical symptoms. The results show no benefit from off-therapy surveillance as repeated imaging can also harm the patient.

• #35 Is Detection of Relapse by Surveillance Imaging Associated With L…: Ingenta Connect

  https://www.ingentaconnect.com/content/wk/jpho/2022/00000044/00000006/art00005

  We investigated whether surveillance imaging had an impact on post-relapse survival in patients with rhabdomyosarcoma (RMS). […] We hypothesized that relapse detected by imaging (group IM) would be associated with longer survival compared with relapse detected with a clinical sign or symptom (group SS). […] These results on whether surveillance imaging provides survival benefit in patients with relapsed RMS are inconclusive. Larger studies are needed to justify current surveillance recommendations.

• #36 No additional benefit with intensive surveillance in relapsed rhabdomyosarcoma – BJMO

  https://www.bjmo.be/no-additional-benefit-with-intensive-surveillance-in-relapsed-rhabdomyosarcoma/

  Intensive surveillance after therapy is needed in pediatric patients treated for rhabdomyosarcoma (RMS). This is done as around a third of these patients experience relapse, and three-year survival after relapse is only 37%. […] The usefulness of this protocol for patient benefit is unclear. A recent retrospective study has analyzed the clinical value of surveillance in relapsed RMS group. […] The study found no survival advantage for RMS patients in intensive surveillance of clinical symptoms. The results show no benefit from off-therapy surveillance as repeated imaging can also harm the patient.

• #37

  https://link.springer.com/article/10.1007/s00247-023-05596-8

  The current European imaging guidelines advise that surveillance imaging should be performed three times a year in the first 2 years after the end of therapy, reducing the period of surveillance of the primary tumour from 5 to 2 years. […] However, taking into account the limited evidence for a beneficial effect of surveillance imaging on survival, we would like to echo the words of McHugh and Roebuck: „To resolve the question of the value of ongoing imaging surveillance, patients should be randomized to either of two arms once therapy is complete; one with routine surveillance imaging, the other without surveillance imaging (relying solely on clinical follow-up).”

• #38 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Male patients have a higher incidence of embryonal tumors, and Black patients have a slightly higher incidence of alveolar tumors. […] The Children’s Oncology Group (COG) performed retrospective exome sequencing on germline DNA to determine the prevalence of 63 autosomal dominant cancer-predisposing genes in 615 patients with newly diagnosed rhabdomyosarcoma. They identified germline cancer-predisposition (pathogenic or likely pathogenic) variants in 45 patients with rhabdomyosarcoma (7.3%; all FOXO1 fusion negative) across 15 autosomal dominant genes. […] The prognosis for children or adolescents with rhabdomyosarcoma is related to many clinical and biological factors, including age, site of origin, tumor size, resectability, histopathological subtype, PAX3::FOXO1 or PAX7::FOXO1 gene fusion status, metastases at diagnosis, lymph node involvement at diagnosis, bone marrow involvement, biological characteristics, response to therapy, circulating tumor DNA and RNA.

• #39 Rhabdomyosarcoma – Pediatrics – Merck Manual Professional Edition

  https://www.merckmanuals.com/professional/pediatrics/pediatric-cancers/rhabdomyosarcoma

  Approximately 15 to 25% of children present with metastatic disease. The lung is the most common site of metastasis; bone, bone marrow, and lymph nodes are other possible sites. […] Prognosis is based on cancer location (eg, prognosis is better with nonparameningeal head/neck and nonbladder/nonprostate genitourinary cancers), completeness of resection, presence of metastasis, age (prognosis is worse for children 10 years of age), and histology (embryonal histology is associated with a better outcome than alveolar histology). Combinations of these prognostic factors place children in a low-risk, intermediate-risk, or high-risk category. A complex risk stratification system exists. Treatment intensifies with each risk category, and overall survival ranges from 90% in children with low-risk disease to 50% in children with high-risk disease.

• #40 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Male patients have a higher incidence of embryonal tumors, and Black patients have a slightly higher incidence of alveolar tumors. […] The Children’s Oncology Group (COG) performed retrospective exome sequencing on germline DNA to determine the prevalence of 63 autosomal dominant cancer-predisposing genes in 615 patients with newly diagnosed rhabdomyosarcoma. They identified germline cancer-predisposition (pathogenic or likely pathogenic) variants in 45 patients with rhabdomyosarcoma (7.3%; all FOXO1 fusion negative) across 15 autosomal dominant genes. […] The prognosis for children or adolescents with rhabdomyosarcoma is related to many clinical and biological factors, including age, site of origin, tumor size, resectability, histopathological subtype, PAX3::FOXO1 or PAX7::FOXO1 gene fusion status, metastases at diagnosis, lymph node involvement at diagnosis, bone marrow involvement, biological characteristics, response to therapy, circulating tumor DNA and RNA.

• #41 Epidemiology, Incidence, and Survival of Rhabdomyosarcoma Subtypes: SEER and ICES Database Analysis – Rutgers University

  https://scholarship.libraries.rutgers.edu/esploro/outputs/journalArticle/Epidemiology-Incidence-and-Survival-of-Rhabdomyosarcoma/991031665245704646

  Rhabdomyosarcoma is the most common soft-tissue sarcoma in children and adolescents and accounts for 3% of all pediatric tumors. The National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) database was queried for patients diagnosed with any type of rhabdomyosarcoma between 1973 and 2014. Survival was modeled with Kaplan–Meier survival curves and Cox proportional hazards models were used to assess the effect of age and gender on survival. Pleomorphic and alveolar rhabdomyosarcoma had the worst overall survival with a 26.6% and 28.9% 5-year survival, respectively. Embryonal rhabdomyosarcoma had the highest 5-year survival rate (73.9%). Presence of higher-grade tumors and metastatic disease at presentation were negatively correlated with survival months (p<0.05). No significant differences in the survival were found between gender or race between all of the subtypes (p>0.05). This study highlights key differences in the demographic and survival rates of the different types of rhabdomyosarcoma that can be used for more tailored patient counseling. […] We also demonstrate that large, population-level databases provide sufficient data that can be used in the analysis of rare tumors.

• #42 Rhabdomyosarcoma – Pediatrics – Merck Manual Professional Edition

  https://www.merckmanuals.com/professional/pediatrics/pediatric-cancers/rhabdomyosarcoma

  Approximately 15 to 25% of children present with metastatic disease. The lung is the most common site of metastasis; bone, bone marrow, and lymph nodes are other possible sites. […] Prognosis is based on cancer location (eg, prognosis is better with nonparameningeal head/neck and nonbladder/nonprostate genitourinary cancers), completeness of resection, presence of metastasis, age (prognosis is worse for children 10 years of age), and histology (embryonal histology is associated with a better outcome than alveolar histology). Combinations of these prognostic factors place children in a low-risk, intermediate-risk, or high-risk category. A complex risk stratification system exists. Treatment intensifies with each risk category, and overall survival ranges from 90% in children with low-risk disease to 50% in children with high-risk disease.

• #43 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Male patients have a higher incidence of embryonal tumors, and Black patients have a slightly higher incidence of alveolar tumors. […] The Children’s Oncology Group (COG) performed retrospective exome sequencing on germline DNA to determine the prevalence of 63 autosomal dominant cancer-predisposing genes in 615 patients with newly diagnosed rhabdomyosarcoma. They identified germline cancer-predisposition (pathogenic or likely pathogenic) variants in 45 patients with rhabdomyosarcoma (7.3%; all FOXO1 fusion negative) across 15 autosomal dominant genes. […] The prognosis for children or adolescents with rhabdomyosarcoma is related to many clinical and biological factors, including age, site of origin, tumor size, resectability, histopathological subtype, PAX3::FOXO1 or PAX7::FOXO1 gene fusion status, metastases at diagnosis, lymph node involvement at diagnosis, bone marrow involvement, biological characteristics, response to therapy, circulating tumor DNA and RNA.

• #44 Genomic assessment of cancer-predisposition landscape of pediatric rhabdomyosarcoma | BCM

  https://www.bcm.edu/news/genomic-assessment-of-cancer-predisposition-landscape-of-pediatric-rhabdomyosarcoma

  Rhabdomyosarcoma (RMS) is a highly malignant tumor and the most common soft tissue sarcoma in children. […] The study included 615 patients with newly diagnosed RMS from Childrens Oncology Group institutions across North America. […] The results showed that 7.3% of patients with RMS had clinically significant variant changes in cancer-predisposition genes compared to 1.4% of controls. […] Understanding the frequency of these variants in children with RMS could influence strategies for genetic testing and future surveillance of patients with a pathogenic variant, as some of these variants may predispose children to multiple cancers later in life. […] These results may change our current decisions about when to offer genetic testing for children with RMS, which right now is focused on very young patients. […] The 7.3% prevalence rate we found is probably the lower boundary. […] We need these large-scale studies and collaborations to move the needle when it comes to improving outcomes for these children.

• #45 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Because treatment and prognosis partly depend on the histology and molecular characterization of the tumor, it is necessary that the tumor tissue be reviewed by expert pathologists with experience in the evaluation and diagnosis of tumors in children. […] The COG reviewed the correlation between anaplastic histology and germline TP53 pathogenic variants in 239 patients with rhabdomyosarcoma. Among the 46 patients with anaplastic rhabdomyosarcoma, 11% (n = 5) carried a germline TP53 pathogenic variant, compared with 1% (n = 2) of the patients without anaplasia. […] The COG performed a retrospective analysis of patients with rhabdomyosarcoma who had bone marrow metastasis at initial presentation and were treated in COG protocols. Rhabdomyosarcoma metastatic to bone was identified in 154 patients (median age at diagnosis, 14.9 years).

• #46 Risks for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/risks

  Rhabdomyosarcoma (RMS) is most common in children younger than 10 years old. It can also occur in teens and adults. It is slightly more common in boys than in girls. […] People who have certain genetic conditions have a higher than average risk for RMS. […] The genetic conditions listed below can increase your child’s risk for RMS. […] Li-Fraumeni syndrome is an inherited condition that is associated with an increased risk of developing certain types of cancers, including breast cancer, brain tumours, acute leukemia, bone and soft tissue sarcomas (such as RMS), and adrenal cortical carcinomas. […] Neurofibromatosis type 1 increases the risk for neuroendocrine tumours, soft tissue sarcoma, such as RMS, brain tumours, leukemia and neuroblastoma. […] Beckwith-Wiedemann syndrome is associated with an increased risk of developing cancer, including RMS, Wilms tumour, neuroblastoma and adrenal gland tumours. […] They also have a higher risk of developing certain types of cancers, including RMS and neuroblastoma. […] They have a higher risk for RMS.

• #47 Germline predisposition to genitourinary rhabdomyosarcoma – Schneider – Translational Andrology and Urology

  https://tau.amegroups.org/article/view/49299/html

  Multiple genetic conditions predispose to the development of rhabdomyosarcoma. […] Therefore, data are limited on genitourinary (GU) specific associations with certain genetic syndromes. […] In this article, we summarize possible genetic considerations for children with rhabdomyosarcoma of the genitourinary system. […] Genetic evaluation with counseling and/or testing may help identify an underlying condition. […] When a germline pathogenic DICER1 variant is identified, testing and surveillance recommendations are available for individuals and family members. […] Given the substantial lifetime cancer risk for LFS patients, intensive surveillance guidelines including routine whole-body MRI have been published to reduce morbidity and mortality for LFS patients. […] While data is limited, RMS in mosaic variegated aneuploidy is highly likely to occur in the GU tract, primarily in the vagina and bladder, and is more likely to be embryonal and exhibit botryoid features.

• #48 Risks for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/risks

  Rhabdomyosarcoma (RMS) is most common in children younger than 10 years old. It can also occur in teens and adults. It is slightly more common in boys than in girls. […] People who have certain genetic conditions have a higher than average risk for RMS. […] The genetic conditions listed below can increase your child’s risk for RMS. […] Li-Fraumeni syndrome is an inherited condition that is associated with an increased risk of developing certain types of cancers, including breast cancer, brain tumours, acute leukemia, bone and soft tissue sarcomas (such as RMS), and adrenal cortical carcinomas. […] Neurofibromatosis type 1 increases the risk for neuroendocrine tumours, soft tissue sarcoma, such as RMS, brain tumours, leukemia and neuroblastoma. […] Beckwith-Wiedemann syndrome is associated with an increased risk of developing cancer, including RMS, Wilms tumour, neuroblastoma and adrenal gland tumours. […] They also have a higher risk of developing certain types of cancers, including RMS and neuroblastoma. […] They have a higher risk for RMS.

• #49 Germline predisposition to genitourinary rhabdomyosarcoma – Schneider – Translational Andrology and Urology

  https://tau.amegroups.org/article/view/49299/html

  The most common genitourinary tumor seen in children with BWS is Wilms tumor, however, RMS accounts for 5% of the tumors found in the BWS population. […] While certain tumors are common in NF1, RMS is rare. […] Approximately 50% of the time, NF1 is inherited from an affected parent while the other 50% of the time it is brand new in an individual, or de novo. […] The risk is reported to be about 0.5%, or 1 in 200, for an individuals with NF1 to develop a RMS. […] Overall, although the incidence of any RMS in NF1 is rare, the risk of developing RMS is thought to be 20 times greater than in the general population. […] There is limited information regarding RMS in RASopathies other those mentioned above. […] CMMRD, like Li-Fraumeni syndrome, is a very aggressive cancer predisposition syndrome increasing the risk for Lynch syndrome type cancers in childhood as well as leukemia, lymphoma, and sarcoma, among others.

• #50 Germline predisposition to genitourinary rhabdomyosarcoma – Schneider – Translational Andrology and Urology

  https://tau.amegroups.org/article/view/49299/html

  Referral for genetic counseling is appropriate for any individual diagnosed with a GU RMS. […] Identification of features listed in Table 1 in addition to a tumor diagnosis, may highlight the need for genetic counseling referral and/or genetic testing. […] Thus, identifying a genetic predisposition and detecting further tumors that develop early may improve disease-related prognosis and spare individuals (and potentially their family members) the toxicities of chemotherapy and radiation.

• #51 Rhabdomyosarcoma – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK507721/

  Recommendations concerning continued surveillance imaging for recurrent disease also vary between Europe and the US. Current guidelines in Europe recommend surveillance imaging every 4 months for 2 years. In the US, there are no set guidelines; however, most clinicians follow current Children’s Oncology Group recommendations to repeat imaging every 4 months for 4 years. Due to known complications from radiation exposure, particularly in children, many experts have recently advocated for US clinicians to adopt European surveillance guidelines as both protocols have similar outcomes. The management of patients with RMS is continuously evolving as new, evidence-based results emerge from clinical trials of treatment advancements.

• #52

  https://link.springer.com/article/10.1007/s00247-023-05596-8

  Rhabdomyosarcoma, although rare, is the most frequent soft tissue sarcoma in children and adolescents. […] The incidence is higher in boys compared to girls (ratio: 1.4, 95% confidence interval (CI): 1.2-1.6). […] Rhabdomyosarcoma most often occurs in the head and neck, genitourinary (GU) tract and extremities, but can also occur at other sites. […] The rarity of rhabdomyosarcoma makes collaborative studies imperative, which in Europe are governed by the European paediatric Soft tissue sarcoma Study Group (EpSSG) and the Cooperative Weichteilsarkom Studiengruppe (CWS), including patients in clinical trials in well over 100 clinical centres. […] For high-quality rhabdomyosarcoma imaging, collaborative groups of the EpSSG, the CWS and the Oncology Task Force of the European Society of Paediatric Radiology (ESPR) have taken the initiative to develop an international guideline, including technical guidance and standardised reporting forms.

• #53 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  Although practically difficult (due to tumors rarity), there is a pressing need for high quality randomized controlled trials to provide further guidance. […] The rarity of adult RMS hindered accrual to randomized controlled trials, which in turn resulted in a lack of established treatment guidelines. […] Consequently, there exists an ongoing controversy on how to best manage these patients, and whether a modification of protocols for pediatric RMS, adult soft-tissue sarcoma or custom regimens should be adopted. […] Many potential explanations for the dismal survival were proposed through retrospective analyses, including age, higher incidence of unfavorable tumor sites, higher rates of alveolar/pleomorphic/undifferentiated histologies, and higher IRS-stage. […] Recent studies have also shown that adults treated with multidisciplinary approaches -adopted from pediatric protocols-, often have significantly better outcomes, although not as good as pediatric patients.

• #54 Epidemiology, Incidence, and Survival of Rhabdomyosarcoma Subtypes: SEER and ICES Database Analysis – Rutgers University

  https://scholarship.libraries.rutgers.edu/esploro/outputs/journalArticle/Epidemiology-Incidence-and-Survival-of-Rhabdomyosarcoma/991031665245704646

  Rhabdomyosarcoma is the most common soft-tissue sarcoma in children and adolescents and accounts for 3% of all pediatric tumors. The National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) database was queried for patients diagnosed with any type of rhabdomyosarcoma between 1973 and 2014. Survival was modeled with Kaplan–Meier survival curves and Cox proportional hazards models were used to assess the effect of age and gender on survival. Pleomorphic and alveolar rhabdomyosarcoma had the worst overall survival with a 26.6% and 28.9% 5-year survival, respectively. Embryonal rhabdomyosarcoma had the highest 5-year survival rate (73.9%). Presence of higher-grade tumors and metastatic disease at presentation were negatively correlated with survival months (p<0.05). No significant differences in the survival were found between gender or race between all of the subtypes (p>0.05). This study highlights key differences in the demographic and survival rates of the different types of rhabdomyosarcoma that can be used for more tailored patient counseling. […] We also demonstrate that large, population-level databases provide sufficient data that can be used in the analysis of rare tumors.

• #55 Model to predict the survival benefit of radiation for patients with rhabdomyosarcoma after surgery: A population-based study

  https://www.spandidos-publications.com/10.3892/ijo.2014.2466

  Rhabdomyosarcoma (RMS) is a rare form of cancer with an incidence of 0.50 per 100,000 in children 014 years old, and 0.23 per 100,000 for the overall population in 2010 according to Surveillance, Epidemiology, and End Results (SEER) statistics. […] The 5-year overall survival (OS) rate for patients with RMS has increased from approximately 35% in the 1970s to ~50% in the 2000s, according to SEER statistics. […] The study cohort comprised 1578 patients. […] The aim of this study was to provide a decision aid to the clinician that can give an individual estimation of the prognostic benefit of adjuvant radiotherapy, to facilitate the decision of whether adjuvant radiotherapy is appropriate. […] The study population comprised all patients with a diagnosis of RMS between 1990 and 2010. […] The SEER program collects information on incidence, prevalence and survival. Currently, registry in SEER covers approximately 28% of the US population, and the characteristics of the SEER population are comparable with the general US population.

• #56 Survival stratification in childhood rhabdomyosarcoma of the extremities: a derivation and validation study | Scientific Reports

  https://www.nature.com/articles/s41598-020-62656-x

  Due to a lack of data, to date, optimal prognostic stratification for childhood RMS of the extremities remains far from being established. […] In this study, we performed a population-based analysis of data from the Surveillance, Epidemiology, and End Results (SEER) registry to identify prognostic factors and to develop a prognostic stratification model in children with extremity RMS. […] The new survival stratification model was developed from a median 46.0 months of follow-up in the SEER-based cohort. […] The calibration plots demonstrated good concordance between the predicted and actual survival at 2, 3, and 5 years, and the bootstrap-adjusted c-index of 0.76 (95% CI, 0.730.80) suggested that the nomogram had a good capacity to discriminate among patients. […] Prognostic stratification is important for informing treatment decisions, and the treatment strategy also greatly impacts patient survival.

• #57 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  The prognosis of intermediate-risk group patients still improved if their disease responded to chemotherapy. […] The highest risk of local recurrence was seen in patients with IRS stage-II (unfavorable site), intermediate-risk disease, residual tumor (Group II-III), anthracycline-based regimen and PR to initial CT. […] Clinical group-III patients were compared based on whether they underwent delayed primary excisions (DPE) or not. […] The only significant difference between controlled and recurrent tumors among non-PM group-I patients was in their risk group distribution. […] Our findings highlight the local, distant and overall PFS benefit with the use of chemotherapy regimens, adapted from the pediatric RMS protocols, in adults with non-metastatic RMS. […] Development and assessment of novel chemotherapeutic agents is critical, especially for patients with intermediate-risk disease who could not benefit from conventional chemotherapeutic regimens.

• #58 Pediatric Rhabdomyosarcoma: Epidemiology and Genetic Susceptibility

  https://www.mdpi.com/2077-0383/10/9/2028

  Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children, yet little is known about its etiology. Studies that examine either environmental exposures or germline genetic predisposition in RMS have begun to identify factors that contribute to this malignancy. Here, we summarize epidemiological reports of RMS incidence in terms of several factors, including age at diagnosis, biological sex, and geographic location. […] RMS has an incidence of approximately 4.71 per million children and adolescents less than 20 years of age in the United States. This is similar to other sarcomas, including osteosarcoma (5.09 per million) and Ewing sarcoma (2.95 per million), but lower than the most frequent pediatric cancer—acute lymphoblastic leukemia (ALL, 32.59 per million). […] A primary objective of epidemiology is to study the distribution and determinants of disease in human populations. Assessments evaluating the distribution of disease (e.g., incidence, overall survival) are considered descriptive epidemiology, whereas studies evaluating the determinants of disease (e.g., environmental exposures, nutritional status) are considered analytic epidemiology.

• #59 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Male patients have a higher incidence of embryonal tumors, and Black patients have a slightly higher incidence of alveolar tumors. […] The Children’s Oncology Group (COG) performed retrospective exome sequencing on germline DNA to determine the prevalence of 63 autosomal dominant cancer-predisposing genes in 615 patients with newly diagnosed rhabdomyosarcoma. They identified germline cancer-predisposition (pathogenic or likely pathogenic) variants in 45 patients with rhabdomyosarcoma (7.3%; all FOXO1 fusion negative) across 15 autosomal dominant genes. […] The prognosis for children or adolescents with rhabdomyosarcoma is related to many clinical and biological factors, including age, site of origin, tumor size, resectability, histopathological subtype, PAX3::FOXO1 or PAX7::FOXO1 gene fusion status, metastases at diagnosis, lymph node involvement at diagnosis, bone marrow involvement, biological characteristics, response to therapy, circulating tumor DNA and RNA.

• #60 Is Detection of Relapse by Surveillance Imaging Associated With L…: Ingenta Connect

  https://www.ingentaconnect.com/content/wk/jpho/2022/00000044/00000006/art00005

  We investigated whether surveillance imaging had an impact on post-relapse survival in patients with rhabdomyosarcoma (RMS). […] We hypothesized that relapse detected by imaging (group IM) would be associated with longer survival compared with relapse detected with a clinical sign or symptom (group SS). […] These results on whether surveillance imaging provides survival benefit in patients with relapsed RMS are inconclusive. Larger studies are needed to justify current surveillance recommendations.

• #61

  https://link.springer.com/article/10.1007/s00247-023-05596-8

  The current European imaging guidelines advise that surveillance imaging should be performed three times a year in the first 2 years after the end of therapy, reducing the period of surveillance of the primary tumour from 5 to 2 years. […] However, taking into account the limited evidence for a beneficial effect of surveillance imaging on survival, we would like to echo the words of McHugh and Roebuck: „To resolve the question of the value of ongoing imaging surveillance, patients should be randomized to either of two arms once therapy is complete; one with routine surveillance imaging, the other without surveillance imaging (relying solely on clinical follow-up).”

Zobacz więcej