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131I-Meta-iodobenzylguanidine followed by Busulfan and Melphalan and autologous stem cell transplantation in high-risk neuroblastoma
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  • Stefano Giardino,
  • Arnoldo Piccardo,
  • Massimo Conte,
  • Matteo Puntoni,
  • Enrica Bertelli,
  • Stefania Sorrentino,
  • Mariapina Montera,
  • Marco Risso,
  • Ilaria Caviglia,
  • Vania Altrinetti,
  • Edoardo Lanino,
  • Maura Faraci,
  • Alberto Garaventa
Stefano Giardino
Istituto Giannina Gaslini

Corresponding Author:[email protected]

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Arnoldo Piccardo
Galliera Hospital Trust
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Massimo Conte
Istituto Giannina Gaslini
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Matteo Puntoni
Galliera Hospital Trust
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Enrica Bertelli
Istituto Giannina Gaslini
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Stefania Sorrentino
Istituto Giannina Gaslini
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Mariapina Montera
Istituto Giannina Gaslini
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Marco Risso
Istituto Giannina Gaslini
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Ilaria Caviglia
Istituto Giannina Gaslini
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Vania Altrinetti
Galliera Hospital Trust
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Edoardo Lanino
Istituto Giannina Gaslini
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Maura Faraci
Istituto Giannina Gaslini
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Alberto Garaventa
Istituto Giannina Gaslini
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Abstract

Introduction.Despite progress obtained with current treatments,the event-free survival of high-risk neuroblastoma(HR-NB)patients does not exceed 40-50% and the prognosis in refractory or relapsed patients is poor,still representing a challenge for pediatric oncologist.Therapeutic Iodine-131 meta-iodobenzylguanidine(Th-131I-MIBG) is a recognized safe and potentially effective treatment in NB.Materials.In this retrospective study,we report outcome of 28 MIBG-avid NB patients with advanced disease,because refractory or relapsed,underwent,from 1996 to 2014,to Th-131I-MIBG administered shortly before(median of 17 days) high-dose chemotherapy with Busulfan and Melphalan(HD-BuMel) and autologous stem cell transplantation(ASCT)at Gaslini Institute in Genoa,with the aim to analyze feasibility,safety and efficacy of this approach.Results.Engraftment occurred in all patients after a median of 14(11-29)and 30 days(13-80)from ASCT for neutrophil and platelet respectively.No treatment-related deaths were observed.The main high grade(3-4)toxicity observed was oral and gastrointestinal mucositis in 78.6% and 7.1% of patients respectively,while high grade hepatic toxicity was observed in 10.7%;two patients developed veno-occlusive-disease(7.1%),completely responsive to defibrotide.Hypothyroidism was the main late complication occurred in 9 patients(31.1%).After Th-131MIBG and HD-BuMel, 19 patients(67.8%) showed an improvement of disease status.Over a median follow-up of 15.9 years,the 3-year and 5-year overall survival(OS)probability were 53%(CI 0.33-0.69)and 41%(CI 0.22-0.59)and the 3-year and 5-year rates of cumulative risk of progression/relapse were 64%(CI 0.47-0.81)and 73%(CI 0.55-0.88),respectively.MYCN amplification emerged as the only risk factor significantly associated with OS(HR 3.58;p0.041).Conclusion.Th-131I-MIBG administered shortly before HD-BuMel turned out to be a safe and effective regimen,suggesting it should be included in a sequential approach in patients with advanced MIBG-avid NB.These patients could be benefit to be manage in centers with proven expertise in these treatments.
27 Jun 2020Submitted to Pediatric Blood & Cancer
27 Jun 2020Submission Checks Completed
27 Jun 2020Assigned to Editor
29 Jun 2020Reviewer(s) Assigned
18 Jul 2020Review(s) Completed, Editorial Evaluation Pending
18 Jul 2020Editorial Decision: Revise Major
24 Sep 20201st Revision Received
24 Sep 2020Submission Checks Completed
24 Sep 2020Assigned to Editor
25 Sep 2020Reviewer(s) Assigned
05 Oct 2020Review(s) Completed, Editorial Evaluation Pending
06 Oct 2020Editorial Decision: Accept