TY - JOUR
T1 - Congenital rhabdomyosarcoma
T2 - A report from the European paediatric Soft tissue sarcoma Study Group
AU - Bisogno, Gianni
AU - Minard-Colin, Veronique
AU - Arush, Myriam Ben
AU - Daragjati, Julia
AU - Coppadoro, Beatrice
AU - Gallego, Soledad
AU - Alaggio, Rita
AU - Smeulders, Naima
AU - Mudry, Peter
AU - Zin, Angelica
AU - Merks, Johannes H.M.
AU - Slater, Olga
N1 - Publisher Copyright:
© 2021 Wiley Periodicals LLC
PY - 2022/2/1
Y1 - 2022/2/1
N2 - Procedure: Congenital rhabdomyosarcoma (RMS) represents a challenging disease due to its characteristics and the difficulties in delivering treatment in this immature population. Methods: We analyzed treatment and outcome of patients with congenital RMS, defined as tumor diagnosed in the first 2 months of life, enrolled in the European paediatric Soft tissue sarcoma Study Group protocols. Results: Twenty-four patients with congenital RMS were registered. All, except one patient (PAX3-FOXO1-positive metastatic RMS), had favorable histology and localized disease. Three patients had VGLL2-CITED2/NCOA2 fusion. Complete tumor resection was achieved in 10 patients. No radiotherapy was given. Chemotherapy doses were adjusted to age and weight. Only two patients required further dose reduction for toxicity. The 5-year event-free survival (EFS) and overall survival (OS) were 75.0% (95% confidence interval [CI] 52.6–87.9) and 87.3% (95% CI 65.6–95.7), respectively. Progressive disease was the main cause of treatment failure. Conclusion: Patients with congenital RMS presented with a favorable disease, allowing weight- and age-adjusted doses of chemotherapy and avoidance of irradiation, without compromising the outcome.
AB - Procedure: Congenital rhabdomyosarcoma (RMS) represents a challenging disease due to its characteristics and the difficulties in delivering treatment in this immature population. Methods: We analyzed treatment and outcome of patients with congenital RMS, defined as tumor diagnosed in the first 2 months of life, enrolled in the European paediatric Soft tissue sarcoma Study Group protocols. Results: Twenty-four patients with congenital RMS were registered. All, except one patient (PAX3-FOXO1-positive metastatic RMS), had favorable histology and localized disease. Three patients had VGLL2-CITED2/NCOA2 fusion. Complete tumor resection was achieved in 10 patients. No radiotherapy was given. Chemotherapy doses were adjusted to age and weight. Only two patients required further dose reduction for toxicity. The 5-year event-free survival (EFS) and overall survival (OS) were 75.0% (95% confidence interval [CI] 52.6–87.9) and 87.3% (95% CI 65.6–95.7), respectively. Progressive disease was the main cause of treatment failure. Conclusion: Patients with congenital RMS presented with a favorable disease, allowing weight- and age-adjusted doses of chemotherapy and avoidance of irradiation, without compromising the outcome.
UR - http://www.scopus.com/inward/record.url?scp=85115881803&partnerID=8YFLogxK
U2 - 10.1002/pbc.29376
DO - 10.1002/pbc.29376
M3 - Article
C2 - 34582098
AN - SCOPUS:85115881803
SN - 1545-5009
VL - 69
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
IS - 2
M1 - e29376
ER -