Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case reportReportar como inadecuado

Role of eculizumab in a pediatric refractory gemcitabine-induced thrombotic microangiopathy: a case report - Descarga este documento en PDF. Documentación en PDF para descargar gratis. Disponible también para leer online.

Journal of Medical Case Reports

, 11:209

First Online: 27 July 2017Received: 26 December 2016Accepted: 02 July 2017


BackgroundThe 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 presentationWe 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.

ConclusionsEculizumab prevents serious complement-mediated vascular damage for chemotherapy-induced thrombotic microangiopathy in pediatric cases.

KeywordsMicroangiopathy Eculizumab Gemcitabine Medulloblastoma Radiotherapy Brain tumors Case report AbbreviationsADAMTS 13A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13

aHUSAtypical hemolytic uremic syndrome

ARDSAcute respiratory distress syndrome

CNSCentral nervous system

CSFCerebrospinal fluid

CTComputed tomography

GiHUSGemcitabine-induced hemolytic uremic syndrome

HSCT-TMAHematopoietic stem cell transplantation-associated thrombotic microangiopathy

HUSHemolytic uremic syndrome

TMAThrombotic microangiopathy

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Autor: Ludovica Facchini - Maurizio Lucchesi - Alessia Stival - Rosa Maria Roperto - Francesca Melosi - Marco Materassi - Silvia 


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