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

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...

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Bibliographic Details
Published in:Journal of medical case reports 2017-07, Vol.11 (1), p.209-209, Article 209
Main Authors: Facchini, Ludovica, Lucchesi, Maurizio, Stival, Alessia, Roperto, Rosa Maria, Melosi, Francesca, Materassi, Marco, Farina, Silvia, Tintori, Veronica, de Martino, Maurizio, Sardi, Iacopo
Format: Article
Language:English
Subjects:
Anemia
Antibiotics
Antibodies, Monoclonal, Humanized - administration & dosage
Antimetabolites, Antineoplastic - adverse effects
Brain cancer
Brain tumors
Cancer therapies
Care and treatment
Case Report
Case reports
Cellular biology
Cerebellar Neoplasms - drug therapy
Chemotherapy
Child, Preschool
Complement Inactivating Agents - administration & dosage
Deoxycytidine - adverse effects
Deoxycytidine - analogs & derivatives
Dosage and administration
Drug dosages
Eculizumab
Fatal Outcome
Gemcitabine
Hemolytic-uremic syndrome
Hemolytic-Uremic Syndrome - chemically induced
Hemolytic-Uremic Syndrome - diagnosis
Hemolytic-Uremic Syndrome - drug therapy
Humans
Hypertension
Male
Medulloblastoma
Medulloblastoma - drug therapy
Microangiopathy
Monoclonal antibodies
NMR
Nuclear magnetic resonance
Patients
Pediatrics
Radiation therapy
Radiotherapy
Stem cells
Targeted cancer therapy
Transplants & implants
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Summary: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. 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. Eculizumab prevents serious complement-mediated vascular damage for chemotherapy-induced thrombotic microangiopathy in pediatric cases.
ISSN:1752-1947
1752-1947
DOI:10.1186/s13256-017-1373-5