Monika Sparber-Sauer1, Sabine Stegmaier1, Christian Vokuhl2, Guido Seitz3, Thekla von Kalle4, Monika Scheer1, Marc Münter5, Stefan S Bielack1,6, Jadwiga Weclawek-Tompol7, Ruth Ladenstein8, Felix Niggli9, Gustaf Ljungman10, Joerg Fuchs11, Simone Hettmer12, Ewa Koscielniak1,13, Thomas Klingebiel14. 1. Klinikum Stuttgart - Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pediatrics 5 (Oncology, Hematology, Immunology), Stuttgart, Germany. 2. Section of Pediatric Pathology, Department of Pathology, Kiel Pediatric Tumor Registry, Kiel, Germany. 3. Department of Pediatric Surgery, University Children's Hospital Marburg, Marburg, Germany. 4. Klinikum Stuttgart - Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Olgahospital, Institute of Radiology, Stuttgart, Germany. 5. Klinikum Stuttgart, Institute of Radiotherapy, Stuttgart, Germany. 6. Department of Pediatric Hematology and Oncology, University of Muenster, Muenster, Germany. 7. Department of Pediatric Hematology/Oncology and BMT, University of Wroclaw, Wroclaw, Poland. 8. Pediatric Oncology, St. Anna Kinderspital, Wien, Austria. 9. Department of Pediatric Oncology, University of Zurich, Zurich, Switzerland. 10. Department of Women's and Children's Health, Children's University Hospital, University of Uppsala, Uppsala, Sweden. 11. Department of Pediatric Surgery and Urology, University Children's Hospital, Tuebingen, Germany. 12. Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Faculty of Medicine, University of Freiburg, Freiburg, Germany. 13. Department of Pediatric Hematology and Oncology, Children's Hospital, Tuebingen, Germany. 14. Department for Children and Adolescents, University Hospital of Frankfurt, Frankfurt/M., Germany.
Abstract
BACKGROUND: Rhabdomyosarcoma (RMS) diagnosed during the first year of life is reported to have poor outcome. Little is known about treatment and outcome data of relapsed disease (RD). METHODS: Characteristics, treatment, and outcome of 155 patients ≤ 12 months registered within the Cooperative Weichteilsarkom Studiengruppe (CWS) between 1981 and 2016 were evaluated. RESULTS: Localized disease (LD) was diagnosed in 144 patients and metastatic disease (MD) in 11. The histological diagnosis was alveolar (RMA) (n = 38, 23/25 examined patients PAX7/3:FOXO1-positive), embryonal (RME) (n = 100), botryoid (n = 10), anaplastic (n = 1), and spindle-cell RMS (n = 6). Multimodal treatment including conventional (age-adjusted) chemotherapy (CHT) (n = 150), resection (n = 137), and radiotherapy (RT) (n = 37) was administered. Complete remission was achieved in 129 of 144 patients with LD. RD occurred in 51 infants at a median age of 1.7 years (range, 0.3-8.8). Sixty-three percent of patients with RMA suffered RD, in contrast to 28% of patients with RME. Relapse treatment consisted of conventional CHT (n = 48), resection (n = 28), and RT (n = 21). The pattern of relapse and best resection were significant prognostic factors for patients with RD (P = 0.000 and P = 0.002). Late effects occurred as secondary malignancies in 6%, long-term toxicity in 21%, and resection-related impairment in 33% of the 105 surviving patients. The 5-year event-free survival and overall survival for infants with initial LD were 51% and 69%, 14% and 14% for patients with initial MD and 39% and 41% for relapsed patients, respectively. CONCLUSION: Multimodal treatment including microscopically complete resection is strongly recommended to achieve a good prognosis in LD and RD of infants with RMS.
BACKGROUND:Rhabdomyosarcoma (RMS) diagnosed during the first year of life is reported to have poor outcome. Little is known about treatment and outcome data of relapsed disease (RD). METHODS: Characteristics, treatment, and outcome of 155 patients ≤ 12 months registered within the Cooperative Weichteilsarkom Studiengruppe (CWS) between 1981 and 2016 were evaluated. RESULTS:Localized disease (LD) was diagnosed in 144 patients and metastatic disease (MD) in 11. The histological diagnosis was alveolar (RMA) (n = 38, 23/25 examined patientsPAX7/3:FOXO1-positive), embryonal (RME) (n = 100), botryoid (n = 10), anaplastic (n = 1), and spindle-cell RMS (n = 6). Multimodal treatment including conventional (age-adjusted) chemotherapy (CHT) (n = 150), resection (n = 137), and radiotherapy (RT) (n = 37) was administered. Complete remission was achieved in 129 of 144 patients with LD. RD occurred in 51 infants at a median age of 1.7 years (range, 0.3-8.8). Sixty-three percent of patients with RMA suffered RD, in contrast to 28% of patients with RME. Relapse treatment consisted of conventional CHT (n = 48), resection (n = 28), and RT (n = 21). The pattern of relapse and best resection were significant prognostic factors for patients with RD (P = 0.000 and P = 0.002). Late effects occurred as secondary malignancies in 6%, long-term toxicity in 21%, and resection-related impairment in 33% of the 105 surviving patients. The 5-year event-free survival and overall survival for infants with initial LD were 51% and 69%, 14% and 14% for patients with initial MD and 39% and 41% for relapsed patients, respectively. CONCLUSION: Multimodal treatment including microscopically complete resection is strongly recommended to achieve a good prognosis in LD and RD of infants with RMS.
Authors: Sarah Whittle; Rajkumar Venkatramani; Anton Schönstein; Svetlana D Pack; Rita Alaggio; Christian Vokuhl; Erin R Rudzinski; Anna-Lena Wulf; Angelica Zin; Juliana R Gruver; Michael A Arnold; Johannes H M Merks; Simone Hettmer; Ewa Koscielniak; Frederic G Barr; Douglas S Hawkins; Gianni Bisogno; Monika Sparber-Sauer Journal: Eur J Cancer Date: 2022-04-19 Impact factor: 10.002