Soft-tissue sarcomas in children and adolescents with neurofibromatosis type 1

Andrea Ferrari, Gianni Bisogno, Alessandra Macaluso, Michela Casanova, Paolo D'Angelo, Paolo Pierani, Ilaria Zanetti, Rita Alaggio, Giovanni Cecchetto, Modesto Carli

Research output: Contribution to journalArticle

Abstract

BACKGROUND. Patients affected by neurofibromatosis type 1 (NF1) are at higher risk of developing soft-tissue sarcomas (STS) than the general population. The clinical findings and outcome in 43 children and adolescents with NF1 treated for STS in the Italian protocols between 1988 and 2004 are reported. METHODS. The study included 37 patients with neurogenic sarcomas (36 malignant peripheral nerve sheath tumors [MPNST], 1 triton tumor) and 6 cases of rhabdomyosarcoma (RMS). The prevalence of NF1 observed during the study period was 43% in the MPNST population and 1% in the RMS group. RESULTS. Most patients with neurogenic sarcomas had large, invasive tumors. Five-year event-free and overall survival rates were 19% and 28%, respectively. Two of 16 patients with evaluable disease responded to chemotherapy. All 6 RMS patients were ≤3 years old and had embryonal subtype, 5 of 6 arising in the genitourinary tract or pelvis (paravesical); 4 were alive in first remission at the time of the analysis, 1 was alive in second remission after a local recurrence, and 1 died of disease. CONCLUSIONS. The occurrence of STS in pediatric patients with NF1 syndrome in Italy is discussed, confirming that NF1 patients have a high risk of developing STS, and particularly MPNST, often with an aggressive clinical presentation and poor outcome. Cases of RMS tended to have particular features (early age, embryonal histotype, genitourinary site) and their outcome seemed to resemble that of the general RMS population.

Original languageEnglish
Pages (from-to)1406-1412
Number of pages7
JournalCancer
Volume109
Issue number7
DOIs
Publication statusPublished - Apr 1 2007

Fingerprint

Neurofibromatosis 1
Sarcoma
Rhabdomyosarcoma
Neurilemmoma
Neurofibrosarcoma
Population
Neurofibromatoses
Pelvis
Italy
Disease-Free Survival
Neoplasms
Survival Rate
Pediatrics
Recurrence
Drug Therapy

Keywords

  • Malignant peripheral nerve sheath tumor
  • Neurofibromatosis
  • Pediatric soft tissue sarcomas
  • Rhabdomyosarcoma

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

Cite this

Ferrari, A., Bisogno, G., Macaluso, A., Casanova, M., D'Angelo, P., Pierani, P., ... Carli, M. (2007). Soft-tissue sarcomas in children and adolescents with neurofibromatosis type 1. Cancer, 109(7), 1406-1412. https://doi.org/10.1002/cncr.22533

Soft-tissue sarcomas in children and adolescents with neurofibromatosis type 1. / Ferrari, Andrea; Bisogno, Gianni; Macaluso, Alessandra; Casanova, Michela; D'Angelo, Paolo; Pierani, Paolo; Zanetti, Ilaria; Alaggio, Rita; Cecchetto, Giovanni; Carli, Modesto.

In: Cancer, Vol. 109, No. 7, 01.04.2007, p. 1406-1412.

Research output: Contribution to journalArticle

Ferrari, A, Bisogno, G, Macaluso, A, Casanova, M, D'Angelo, P, Pierani, P, Zanetti, I, Alaggio, R, Cecchetto, G & Carli, M 2007, 'Soft-tissue sarcomas in children and adolescents with neurofibromatosis type 1', Cancer, vol. 109, no. 7, pp. 1406-1412. https://doi.org/10.1002/cncr.22533
Ferrari, Andrea ; Bisogno, Gianni ; Macaluso, Alessandra ; Casanova, Michela ; D'Angelo, Paolo ; Pierani, Paolo ; Zanetti, Ilaria ; Alaggio, Rita ; Cecchetto, Giovanni ; Carli, Modesto. / Soft-tissue sarcomas in children and adolescents with neurofibromatosis type 1. In: Cancer. 2007 ; Vol. 109, No. 7. pp. 1406-1412.
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abstract = "BACKGROUND. Patients affected by neurofibromatosis type 1 (NF1) are at higher risk of developing soft-tissue sarcomas (STS) than the general population. The clinical findings and outcome in 43 children and adolescents with NF1 treated for STS in the Italian protocols between 1988 and 2004 are reported. METHODS. The study included 37 patients with neurogenic sarcomas (36 malignant peripheral nerve sheath tumors [MPNST], 1 triton tumor) and 6 cases of rhabdomyosarcoma (RMS). The prevalence of NF1 observed during the study period was 43{\%} in the MPNST population and 1{\%} in the RMS group. RESULTS. Most patients with neurogenic sarcomas had large, invasive tumors. Five-year event-free and overall survival rates were 19{\%} and 28{\%}, respectively. Two of 16 patients with evaluable disease responded to chemotherapy. All 6 RMS patients were ≤3 years old and had embryonal subtype, 5 of 6 arising in the genitourinary tract or pelvis (paravesical); 4 were alive in first remission at the time of the analysis, 1 was alive in second remission after a local recurrence, and 1 died of disease. CONCLUSIONS. The occurrence of STS in pediatric patients with NF1 syndrome in Italy is discussed, confirming that NF1 patients have a high risk of developing STS, and particularly MPNST, often with an aggressive clinical presentation and poor outcome. Cases of RMS tended to have particular features (early age, embryonal histotype, genitourinary site) and their outcome seemed to resemble that of the general RMS population.",
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AU - Ferrari, Andrea

AU - Bisogno, Gianni

AU - Macaluso, Alessandra

AU - Casanova, Michela

AU - D'Angelo, Paolo

AU - Pierani, Paolo

AU - Zanetti, Ilaria

AU - Alaggio, Rita

AU - Cecchetto, Giovanni

AU - Carli, Modesto

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N2 - BACKGROUND. Patients affected by neurofibromatosis type 1 (NF1) are at higher risk of developing soft-tissue sarcomas (STS) than the general population. The clinical findings and outcome in 43 children and adolescents with NF1 treated for STS in the Italian protocols between 1988 and 2004 are reported. METHODS. The study included 37 patients with neurogenic sarcomas (36 malignant peripheral nerve sheath tumors [MPNST], 1 triton tumor) and 6 cases of rhabdomyosarcoma (RMS). The prevalence of NF1 observed during the study period was 43% in the MPNST population and 1% in the RMS group. RESULTS. Most patients with neurogenic sarcomas had large, invasive tumors. Five-year event-free and overall survival rates were 19% and 28%, respectively. Two of 16 patients with evaluable disease responded to chemotherapy. All 6 RMS patients were ≤3 years old and had embryonal subtype, 5 of 6 arising in the genitourinary tract or pelvis (paravesical); 4 were alive in first remission at the time of the analysis, 1 was alive in second remission after a local recurrence, and 1 died of disease. CONCLUSIONS. The occurrence of STS in pediatric patients with NF1 syndrome in Italy is discussed, confirming that NF1 patients have a high risk of developing STS, and particularly MPNST, often with an aggressive clinical presentation and poor outcome. Cases of RMS tended to have particular features (early age, embryonal histotype, genitourinary site) and their outcome seemed to resemble that of the general RMS population.

AB - BACKGROUND. Patients affected by neurofibromatosis type 1 (NF1) are at higher risk of developing soft-tissue sarcomas (STS) than the general population. The clinical findings and outcome in 43 children and adolescents with NF1 treated for STS in the Italian protocols between 1988 and 2004 are reported. METHODS. The study included 37 patients with neurogenic sarcomas (36 malignant peripheral nerve sheath tumors [MPNST], 1 triton tumor) and 6 cases of rhabdomyosarcoma (RMS). The prevalence of NF1 observed during the study period was 43% in the MPNST population and 1% in the RMS group. RESULTS. Most patients with neurogenic sarcomas had large, invasive tumors. Five-year event-free and overall survival rates were 19% and 28%, respectively. Two of 16 patients with evaluable disease responded to chemotherapy. All 6 RMS patients were ≤3 years old and had embryonal subtype, 5 of 6 arising in the genitourinary tract or pelvis (paravesical); 4 were alive in first remission at the time of the analysis, 1 was alive in second remission after a local recurrence, and 1 died of disease. CONCLUSIONS. The occurrence of STS in pediatric patients with NF1 syndrome in Italy is discussed, confirming that NF1 patients have a high risk of developing STS, and particularly MPNST, often with an aggressive clinical presentation and poor outcome. Cases of RMS tended to have particular features (early age, embryonal histotype, genitourinary site) and their outcome seemed to resemble that of the general RMS population.

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