title: Salvage rates after progression of high-risk neuroblastoma with a soft tissue mass.
abstract: OBJECTIVE
  Treatment of progression in high-risk neuroblastoma remains challenging despite improved survival. We retrospectively evaluated outcomes in children with a first progression that included soft-tissue masses.
  
  
  METHODS
  We reviewed records of 903 consecutive children with high-risk neuroblastoma diagnosed between 2004 and 2014, and identified 42 whose first progression included soft-tissue masses. Data on demographics, disease characteristics, treatment, and survival were collected. Primary outcome was 5-year overall survival (OS) from time of first progression. Secondary outcomes were local disease-free progression (LDFR) and progression-free survival (PFS) postprogression. We evaluated the prognostic significance of concomitant bone/bone marrow involvement, MYCN status, and multifocality of soft tissue relapse.
  
  
  RESULTS
  Median age at diagnosis was 3.0 (range: 1-10.7) years. Median time to first relapse or progression was 1.2 (range: 0.1-4.5) years after complete remission or minimal stable residual disease. Twelve (29%) patients had concomitant bone or marrow involvement at progression/relapse. There were 11 (26%) patients with International Neuroblastoma Staging System stage 3 disease (all with MYCN amplification), and 31 (74%) with stage 4 disease (12 with MYCN amplification). Nine (21%) patients had multifocal soft tissue progression. R1 resection was achieved in 41 children (95%), 38 (95%) of whom also received salvage radiation therapy. Five-year OS postprogression was 35% (95% CI: 19-51%), 5-year LDFS was 52% (95% CI: 32-72%), and 5-year PFS postprogression was 20% (95% CI: 6-34%).
  
  
  CONCLUSIONS
  Among children with high-risk neuroblastoma who underwent aggressive treatment of a first soft-tissue recurrence, 5-year postprogression overall survival was 34%. Multifocality and MYCN amplification were the predominant prognostic correlates for worse survival.
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Jennifer M
authors.initials: JM
authors.lastname: Murphy
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Irene-Isabel P
authors.initials: II
authors.lastname: Lim
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Benjamin A
authors.initials: BA
authors.lastname: Farber
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Todd E
authors.initials: TE
authors.lastname: Heaton
authors.affiliation: Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Ellen M
authors.initials: EM
authors.lastname: Basu
authors.affiliation: Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Stephen S
authors.initials: SS
authors.lastname: Roberts
authors.affiliation: Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Shakeel
authors.initials: S
authors.lastname: Modak
authors.affiliation: Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Brian H
authors.initials: BH
authors.lastname: Kushner
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA. Electronic address: laquaglm@mskcc.org.
authors.forename: Michael P
authors.initials: MP
authors.lastname: LaQuaglia
mesh_terms: D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009447:Neuroblastoma / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D011379:Prognosis; D012074:Remission Induction; D012189:Retrospective Studies; D016879:Salvage Therapy; D012983:Soft Tissue Neoplasms / Q000401:mortality / Q000473:pathology / Q000628:therapy*
keywords: High-risk neuroblastoma; MYCN; Multifocal disease; Progression; Survival