Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report

Abstract Background The incidence of gemcitabine-induced hemolytic uremic syndrome has already been described in adults. Several approaches have been employed in the treatment of gemcitabine-induced hemolytic uremic syndrome with different outcomes. One of the most promising agents is eculizumab, wh...

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Main Authors: Ludovica Facchini, Maurizio Lucchesi, Alessia Stival, Rosa Maria Roperto, Francesca Melosi, Marco Materassi, Silvia Farina, Veronica Tintori, Maurizio de Martino, Iacopo Sardi
Format: Article
Language:English
Published: BMC 2017-07-01
Series:Journal of Medical Case Reports
Subjects:
Online Access:http://link.springer.com/article/10.1186/s13256-017-1373-5
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author Ludovica Facchini
Maurizio Lucchesi
Alessia Stival
Rosa Maria Roperto
Francesca Melosi
Marco Materassi
Silvia Farina
Veronica Tintori
Maurizio de Martino
Iacopo Sardi
author_facet Ludovica Facchini
Maurizio Lucchesi
Alessia Stival
Rosa Maria Roperto
Francesca Melosi
Marco Materassi
Silvia Farina
Veronica Tintori
Maurizio de Martino
Iacopo Sardi
author_sort Ludovica Facchini
collection DOAJ
description Abstract Background The incidence of gemcitabine-induced hemolytic uremic syndrome has already been described in adults. Several approaches have been employed in the treatment of gemcitabine-induced hemolytic uremic syndrome with different outcomes. One of the most promising agents is eculizumab, which is a monoclonal antibody directed against C5 complement protein. Case presentation We reported the case of a 3-year-old white boy with medulloblastoma who underwent high-dose chemotherapy and craniospinal irradiation. Afterwards he started maintenance chemotherapy with gemcitabine and oxaliplatin. After five courses he presented a progressive clinical worsening, which resulted in a systemic thrombotic microangiopathy. Initially he was treated with rituximab without clinical improvement. Therefore he started therapy with repeated cycles of eculizumab. After seven infusions he showed a gradual improvement and finally a complete remission of gemcitabine-induced hemolytic uremic syndrome. Conclusions Eculizumab prevents serious complement-mediated vascular damage for chemotherapy-induced thrombotic microangiopathy in pediatric cases.
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spelling doaj.art-e31775af1ec44382abf83a5d0b0592aa2022-12-21T18:20:37ZengBMCJournal of Medical Case Reports1752-19472017-07-011111610.1186/s13256-017-1373-5Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case reportLudovica Facchini0Maurizio Lucchesi1Alessia Stival2Rosa Maria Roperto3Francesca Melosi4Marco Materassi5Silvia Farina6Veronica Tintori7Maurizio de Martino8Iacopo Sardi9Neuro-Oncology Unit, Department of Pediatric Oncology, Meyer Children’s HospitalNeuro-Oncology Unit, Department of Pediatric Oncology, Meyer Children’s HospitalNeuro-Oncology Unit, Department of Pediatric Oncology, Meyer Children’s HospitalNephrology Unit, Meyer Children’s Hospital, University of FlorenceIntensive Care Unit, Meyer Children’s HospitalNephrology Unit, Meyer Children’s Hospital, University of FlorenceNeuro-Oncology Unit, Department of Pediatric Oncology, Meyer Children’s HospitalTransplantation Unit, Department of Pediatric Oncology, Meyer Children’s HospitalDepartment of Health Sciences, University of Florence, Meyer Children’s HospitalNeuro-Oncology Unit, Department of Pediatric Oncology, Meyer Children’s HospitalAbstract Background The incidence of gemcitabine-induced hemolytic uremic syndrome has already been described in adults. Several approaches have been employed in the treatment of gemcitabine-induced hemolytic uremic syndrome with different outcomes. One of the most promising agents is eculizumab, which is a monoclonal antibody directed against C5 complement protein. Case presentation We reported the case of a 3-year-old white boy with medulloblastoma who underwent high-dose chemotherapy and craniospinal irradiation. Afterwards he started maintenance chemotherapy with gemcitabine and oxaliplatin. After five courses he presented a progressive clinical worsening, which resulted in a systemic thrombotic microangiopathy. Initially he was treated with rituximab without clinical improvement. Therefore he started therapy with repeated cycles of eculizumab. After seven infusions he showed a gradual improvement and finally a complete remission of gemcitabine-induced hemolytic uremic syndrome. Conclusions Eculizumab prevents serious complement-mediated vascular damage for chemotherapy-induced thrombotic microangiopathy in pediatric cases.http://link.springer.com/article/10.1186/s13256-017-1373-5MicroangiopathyEculizumabGemcitabineMedulloblastomaRadiotherapyBrain tumors
spellingShingle Ludovica Facchini
Maurizio Lucchesi
Alessia Stival
Rosa Maria Roperto
Francesca Melosi
Marco Materassi
Silvia Farina
Veronica Tintori
Maurizio de Martino
Iacopo Sardi
Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report
Journal of Medical Case Reports
Microangiopathy
Eculizumab
Gemcitabine
Medulloblastoma
Radiotherapy
Brain tumors
title Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report
title_full Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report
title_fullStr Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report
title_full_unstemmed Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report
title_short Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report
title_sort role of eculizumab in a pediatric refractory gemcitabine induced thrombotic microangiopathy a case report
topic Microangiopathy
Eculizumab
Gemcitabine
Medulloblastoma
Radiotherapy
Brain tumors
url http://link.springer.com/article/10.1186/s13256-017-1373-5
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