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Rhabdomyosarcoma of the female genitourinary tract: Primary and relapsed disease in infants and older children. Treatment results of five Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry
Author(s) -
SparberSauer Monika,
Matle Malin,
Vokuhl Christian,
Hallmen Erika,
Kalle Thekla,
Münter Marc,
Timmermann Beate,
Bielack Stefan S.,
Klingebiel Thomas,
Koscielniak Ewa,
Seitz Guido
Publication year - 2021
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28889
Subject(s) - medicine , rhabdomyosarcoma , genitourinary system , radiation therapy , pediatrics , surgery , chemotherapy , multimodal therapy , stage (stratigraphy) , sarcoma , pathology , paleontology , biology
Background Rhabdomyosarcoma (RMS) of the female genitourinary tract (FGU‐RMS) located at the vagina or uterus is one of the most favorable RMS sites. Little is known about treatment and outcome in infants and relapsed disease (RD). Methods Characteristics, treatment, and outcome of 71 children with FGU‐RMS registered within five Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry (1981‐2019) were evaluated. Results FGU‐RMS was diagnosed in 67 patients with localized disease (LD) at a median age of 2.89 years (0.09‐18.08). Multimodal treatment consisted of chemotherapy (CHT) (n = 66), secondary surgery (n = 32), and radiotherapy (n = 11). Age at diagnosis ≤12 months was the only significant negative prognostic factor influencing the event‐free survival (EFS). Ten‐year EFS and overall survival (OS) for infants ≤12 months were 50% and 81%, respectively. In contrast, children with LD >1 year and ≤10 years had a 10‐year EFS and OS of 78% and 94% ( P = .038), and >10 years of 82% and 88%, respectively ( P = .53). Metastatic disease was observed in four patients of which three are alive. RD occurred in five of 12 infants ≤1 year and 10/55 children at a median of 1.38 years (0.53‐2.97) after initial diagnosis. Treatment of patients with RD consisted of multimodal treatment (n = 13) or resection only (n = 2). Nine patients (60%) were alive in clinical remission at a median of 7.02 years (1.23‐16.72) after diagnosis of RD. Conclusion Infants with FGU‐RMS have a higher relapse rate than older children with FGU‐RMS, but prognosis is fair.