Pediatric rhabdomyosarcoma incidence and survival in the United States: An assessment of 5656 cases, 2001–2017

Abstract

Background While rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children and adolescents, past epidemiology studies of this malignancy used data that covered <30% of the US population. Therefore, we evaluated RMS incidence using data from U.S. Cancer Statistics (USCS) and survival trends using the National Program of Cancer Registries (NPCR), which covers 100% and 94% of the U.S. population, respectively. Methods Incidence and survival were assessed for pediatric patients diagnosed with RMS during 2003–2017 and 2001–2016, respectively. Both demographic and clinical variables were evaluated. Age‐adjusted incidence rates, average annual percent change (AAPC), and 5‐year relative survival (RS) were calculated, all with corresponding 95% confidence intervals (CIs). Cox regression models were used to evaluate the impact of demographic and clinical variables on survival. Results We identified 5656 primary RMS cases in USCS during 2003–2017. The age‐adjusted incidence rate was 4.58 per 1 million (95% CI: 4.46–4.70) with an AAPC of 0.3% (95% CI: −0.7 to 1.2%). In NPCR, 5‐year RS for all cases was 68.0% (95% CI: 66.6–69.3%). In multivariable analyses, non‐Hispanic (NH) Black cases had worse survival compared with NH White cases (hazard ratio [HR] = 1.16, 95% CI: 1.01–1.33). Conclusion The incidence and survival rates were stable in the largest and most comprehensive population‐based analysis for pediatric RMS cases in the U.S. Additionally, we observed a survival disparity among NH Black cases. Findings from this study could inform interventions to address disparities, risk stratification strategies, and clinical trial design.

Impact of race and ethnicity on presentation and outcomes of patients treated on rhabdomyosarcoma clinical trials: A report from the Children's Oncology Group

“…In the study of IRS clinical trials, there was no evidence of a difference in 5‐year failure free survival by race or ethnicity 7 . In contrast, a recent population‐based study using US Cancer Statistics and National Program of Cancer Registries data found that among children with RMS, NHW patients had higher 5 year relative survival compared to NHB patients 16 . Similarly, a recent SEER‐based study of children with RMS under 10 years of age reported that Hispanic ethnicity was independently associated with worse survival 15 .…”

“… 7 In contrast, a recent population‐based study using US Cancer Statistics and National Program of Cancer Registries data found that among children with RMS, NHW patients had higher 5 year relative survival compared to NHB patients. 16 Similarly, a recent SEER‐based study of children with RMS under 10 years of age reported that Hispanic ethnicity was independently associated with worse survival. 15 Broader studies of pediatric solid tumor or sarcoma patients have yielded generally similar results.…”

“…12 Studies investigating the effect of race and ethnicity on outcomes in patients with pediatric solid tumors, sarcomas, or specifically RMS have produced opposing results. Some studies have shown that NHW patients experience improved survival compared to NHB and/or Hispanic patients, 8,9,[11][12][13][14][15][16] yet others have not reported differences in outcome by race and/or ethnicity. 7,17 Furthermore, a number of studies that sought to identify prognostic factors for RMS report no data on race and/or ethnicity.…”

Zebrafish—A Suitable Model for Rapid Translation of Effective Therapies for Pediatric Cancers

“…Rhabdomyosarcoma, which is the most common soft-tissue sarcoma diagnosed in children, constitutes approximately 3-4% of all pediatric cancers [97,98]. Alterations in the RAS/MAPK signaling pathway are reported in many patients with rhabdomyosarcoma [99] Kahsay et al demonstrated a significant downregulation of the RAS/MAPK pathway in pax3 double mutant zebrafish (pax3a −/− ; pax3b −/− ), which resulted in a delayed progression of kRAS-induced rhabdomyosarcoma [100].…”

A Rare Case of Rhabdosarcoma on the Upper Pole of the Kidney

“…They typically arise from chromosomal translocations involving the PAX3 gene on chromosome 2 and the FOXO1 gene on chromosome 13, leading to aberrant gene products that promote tumor formation [ 1 ]. The overall incidence of RMS in individuals below 20 years of age is 4.58 per million per year in the United States [ 2 ], with slight variations across different regions. In Europe, the incidence is slightly higher at 5.4 per million per year [ 3 ], while in Japan, it is lower at 3.4 per million per year [ 3 ].…”