Congenital rhabdomyosarcoma: A report from the European paediatric Soft tissue sarcoma Study Group

Gianni Bisogno; Veronique Minard-Colin; Myriam Ben Arush; Julia Daragjati; Beatrice Coppadoro; Soledad Gallego; Rita Alaggio; Naima Smeulders; Peter Mudry; Angelica Zin; Johannes H M Merks; Olga Slater

doi:10.1002/pbc.29376

Congenital rhabdomyosarcoma: A report from the European paediatric Soft tissue sarcoma Study Group

Gianni Bisogno, Veronique Minard-Colin, Myriam Ben Arush, Julia Daragjati, Beatrice Coppadoro, Soledad Gallego, Rita Alaggio, Naima Smeulders, Peter Mudry, Angelica Zin, Johannes H M Merks, Olga Slater

Group Merks

Research output: Contribution to journal › Article › peer-review

3 Citations (Scopus)

Abstract

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.

Original language	English
Article number	e29376
Pages (from-to)	e29376
Journal	Pediatric blood & cancer
Volume	69
Issue number	2
DOIs	https://doi.org/10.1002/pbc.29376
Publication status	Published - Sept 2021

Keywords

Child
Gene Fusion
Humans
Progression-Free Survival
Repressor Proteins
Rhabdomyosarcoma/pathology
Rhabdomyosarcoma, Embryonal
Trans-Activators

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title = "Congenital rhabdomyosarcoma: A report from the European paediatric Soft tissue sarcoma Study Group",

abstract = "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.",

keywords = "Child, Gene Fusion, Humans, Progression-Free Survival, Repressor Proteins, Rhabdomyosarcoma/pathology, Rhabdomyosarcoma, Embryonal, Trans-Activators",

author = "Gianni Bisogno and Veronique Minard-Colin and Arush, {Myriam Ben} and Julia Daragjati and Beatrice Coppadoro and Soledad Gallego and Rita Alaggio and Naima Smeulders and Peter Mudry and Angelica Zin and Merks, {Johannes H M} and Olga Slater",

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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

PY - 2021/9

Y1 - 2021/9

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.

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KW - Repressor Proteins

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JF - Pediatric blood & cancer

IS - 2

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ER -

Congenital rhabdomyosarcoma: A report from the European paediatric Soft tissue sarcoma Study Group

Abstract

Keywords

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