Prognostic role of pleural effusion or ascites in localized rhabdomyosarcoma

Daniela Di Carlo, Andrea Ferrari, Tiziana Toffolutti, Giuseppe Maria Milano, Carla Manzitti, Antonio Ruggiero, Patrizia Dall'Igna, Fraia Melchionda, Ilaria Zanetti, Giovanni Scarzello, Gianni Bisogno

Research output: Contribution to journalArticle

Abstract

PURPOSE: The presence of pleural effusion or ascites at the time of diagnosis is generally considered a poor prognostic factor for children with rhabdomyosarcoma (RMS), and treatment is usually intensified despite the fact that there are no published studies to support this decision. We investigated the prognostic role of the presence of pleural effusion or ascites at diagnosis in patients with localized RMS consecutively enrolled in the Italian Soft Tissue Sarcoma Committee protocols over a 30-year period.

METHODS: We reviewed the radiological reports at diagnosis of 150 children with supradiaphragmatic and infradiaphragmatic RMS, noting any presence of effusion and its extent (minimal, moderate, or massive). All patients received intensive chemotherapy, surgery, and standard or hyperfractionated radiotherapy.

RESULTS: Effusion was identified in 32 children (21.3%), 14 with pleural effusion and 18 with ascites. As for its extent, 13 children presented with minimal, 12 with moderate, and 7 with massive effusion. The 5-year progression-free survival (PFS) rate was 49.8% (confidence interval [CI] 31.7-65.5) and 49.5% (CI 40-58.2) for patients with and without effusion, respectively (P = .5). When only patients with moderate or massive effusion were considered, however, their PFS was 36.8% (CI 16.5-57.5) versus 51.2% (CI 42.2-59.5) in patients with minimal or no effusion (P = .01). On the whole, patients with pleural effusion had a very poor outcome with a 5-year PFS of 35.7% (CI 13-59.4).

CONCLUSIONS: The presence of moderate or massive effusion seems to be an unfavorable prognostic factor in children with RMS, and justifies their inclusion in experimental studies.

Original languageEnglish
Pages (from-to)e27932
JournalPediatric Blood and Cancer
Volume66
Issue number11
DOIs
Publication statusPublished - Nov 2019

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Rhabdomyosarcoma
Pleural Effusion
Ascites
Confidence Intervals
Disease-Free Survival
Sarcoma
Radiotherapy
Survival Rate
Drug Therapy

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Prognostic role of pleural effusion or ascites in localized rhabdomyosarcoma. / Di Carlo, Daniela; Ferrari, Andrea; Toffolutti, Tiziana; Milano, Giuseppe Maria; Manzitti, Carla; Ruggiero, Antonio; Dall'Igna, Patrizia; Melchionda, Fraia; Zanetti, Ilaria; Scarzello, Giovanni; Bisogno, Gianni.

In: Pediatric Blood and Cancer, Vol. 66, No. 11, 11.2019, p. e27932.

Research output: Contribution to journalArticle

Di Carlo, Daniela ; Ferrari, Andrea ; Toffolutti, Tiziana ; Milano, Giuseppe Maria ; Manzitti, Carla ; Ruggiero, Antonio ; Dall'Igna, Patrizia ; Melchionda, Fraia ; Zanetti, Ilaria ; Scarzello, Giovanni ; Bisogno, Gianni. / Prognostic role of pleural effusion or ascites in localized rhabdomyosarcoma. In: Pediatric Blood and Cancer. 2019 ; Vol. 66, No. 11. pp. e27932.
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abstract = "PURPOSE: The presence of pleural effusion or ascites at the time of diagnosis is generally considered a poor prognostic factor for children with rhabdomyosarcoma (RMS), and treatment is usually intensified despite the fact that there are no published studies to support this decision. We investigated the prognostic role of the presence of pleural effusion or ascites at diagnosis in patients with localized RMS consecutively enrolled in the Italian Soft Tissue Sarcoma Committee protocols over a 30-year period.METHODS: We reviewed the radiological reports at diagnosis of 150 children with supradiaphragmatic and infradiaphragmatic RMS, noting any presence of effusion and its extent (minimal, moderate, or massive). All patients received intensive chemotherapy, surgery, and standard or hyperfractionated radiotherapy.RESULTS: Effusion was identified in 32 children (21.3{\%}), 14 with pleural effusion and 18 with ascites. As for its extent, 13 children presented with minimal, 12 with moderate, and 7 with massive effusion. The 5-year progression-free survival (PFS) rate was 49.8{\%} (confidence interval [CI] 31.7-65.5) and 49.5{\%} (CI 40-58.2) for patients with and without effusion, respectively (P = .5). When only patients with moderate or massive effusion were considered, however, their PFS was 36.8{\%} (CI 16.5-57.5) versus 51.2{\%} (CI 42.2-59.5) in patients with minimal or no effusion (P = .01). On the whole, patients with pleural effusion had a very poor outcome with a 5-year PFS of 35.7{\%} (CI 13-59.4).CONCLUSIONS: The presence of moderate or massive effusion seems to be an unfavorable prognostic factor in children with RMS, and justifies their inclusion in experimental studies.",
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T1 - Prognostic role of pleural effusion or ascites in localized rhabdomyosarcoma

AU - Di Carlo, Daniela

AU - Ferrari, Andrea

AU - Toffolutti, Tiziana

AU - Milano, Giuseppe Maria

AU - Manzitti, Carla

AU - Ruggiero, Antonio

AU - Dall'Igna, Patrizia

AU - Melchionda, Fraia

AU - Zanetti, Ilaria

AU - Scarzello, Giovanni

AU - Bisogno, Gianni

N1 - © 2019 Wiley Periodicals, Inc.

PY - 2019/11

Y1 - 2019/11

N2 - PURPOSE: The presence of pleural effusion or ascites at the time of diagnosis is generally considered a poor prognostic factor for children with rhabdomyosarcoma (RMS), and treatment is usually intensified despite the fact that there are no published studies to support this decision. We investigated the prognostic role of the presence of pleural effusion or ascites at diagnosis in patients with localized RMS consecutively enrolled in the Italian Soft Tissue Sarcoma Committee protocols over a 30-year period.METHODS: We reviewed the radiological reports at diagnosis of 150 children with supradiaphragmatic and infradiaphragmatic RMS, noting any presence of effusion and its extent (minimal, moderate, or massive). All patients received intensive chemotherapy, surgery, and standard or hyperfractionated radiotherapy.RESULTS: Effusion was identified in 32 children (21.3%), 14 with pleural effusion and 18 with ascites. As for its extent, 13 children presented with minimal, 12 with moderate, and 7 with massive effusion. The 5-year progression-free survival (PFS) rate was 49.8% (confidence interval [CI] 31.7-65.5) and 49.5% (CI 40-58.2) for patients with and without effusion, respectively (P = .5). When only patients with moderate or massive effusion were considered, however, their PFS was 36.8% (CI 16.5-57.5) versus 51.2% (CI 42.2-59.5) in patients with minimal or no effusion (P = .01). On the whole, patients with pleural effusion had a very poor outcome with a 5-year PFS of 35.7% (CI 13-59.4).CONCLUSIONS: The presence of moderate or massive effusion seems to be an unfavorable prognostic factor in children with RMS, and justifies their inclusion in experimental studies.

AB - PURPOSE: The presence of pleural effusion or ascites at the time of diagnosis is generally considered a poor prognostic factor for children with rhabdomyosarcoma (RMS), and treatment is usually intensified despite the fact that there are no published studies to support this decision. We investigated the prognostic role of the presence of pleural effusion or ascites at diagnosis in patients with localized RMS consecutively enrolled in the Italian Soft Tissue Sarcoma Committee protocols over a 30-year period.METHODS: We reviewed the radiological reports at diagnosis of 150 children with supradiaphragmatic and infradiaphragmatic RMS, noting any presence of effusion and its extent (minimal, moderate, or massive). All patients received intensive chemotherapy, surgery, and standard or hyperfractionated radiotherapy.RESULTS: Effusion was identified in 32 children (21.3%), 14 with pleural effusion and 18 with ascites. As for its extent, 13 children presented with minimal, 12 with moderate, and 7 with massive effusion. The 5-year progression-free survival (PFS) rate was 49.8% (confidence interval [CI] 31.7-65.5) and 49.5% (CI 40-58.2) for patients with and without effusion, respectively (P = .5). When only patients with moderate or massive effusion were considered, however, their PFS was 36.8% (CI 16.5-57.5) versus 51.2% (CI 42.2-59.5) in patients with minimal or no effusion (P = .01). On the whole, patients with pleural effusion had a very poor outcome with a 5-year PFS of 35.7% (CI 13-59.4).CONCLUSIONS: The presence of moderate or massive effusion seems to be an unfavorable prognostic factor in children with RMS, and justifies their inclusion in experimental studies.

U2 - 10.1002/pbc.27932

DO - 10.1002/pbc.27932

M3 - Article

VL - 66

SP - e27932

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

SN - 1545-5009

IS - 11

ER -