title: Hepatoblastoma in a patient with methylmalonic aciduria.
abstract: Childhood malignant tumors and their treatment are not well described in the natural history of methylmalonic aciduria (MMA). Here we present a case of hepatoblastoma occurring in the native liver of a 19-month-old male with MMA. His tumor was unresectable at diagnosis and he received neoadjuvant chemotherapy with cisplatin, 5-fluorouracil and vincristine. He developed metabolic acidosis and hyperglycemia during chemotherapy. In addition, he developed anemia, thrombocytopenia and febrile neutropenia. He underwent a combined liver-kidney transplant for local control of his tumor and to treat MMA. He remains in remission more than five years after his transplant. In addition, his transplant has cured his MMA and he is able to tolerate a regular diet without developing metabolic crises.
authors.affiliation: Division of Pediatric Hematology/Oncology, Los Angeles County+University of Southern California Medical Center, Los Angeles, California; Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California.
authors.forename: Randall
authors.initials: R
authors.lastname: Chan
authors.forename: Leo
authors.initials: L
authors.lastname: Mascarenhas
authors.forename: Richard G
authors.initials: RG
authors.lastname: Boles
authors.forename: Nanda
authors.initials: N
authors.lastname: Kerkar
authors.forename: Yuri
authors.initials: Y
authors.lastname: Genyk
authors.forename: Rajkumar
authors.initials: R
authors.lastname: Venkatramani
mesh_terms: D000592:Amino Acid Metabolism, Inborn Errors / Q000150:complications* / Q000175:diagnosis / Q000628:therapy; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use; D001706:Biopsy; D018197:Hepatoblastoma / Q000150:complications* / Q000175:diagnosis* / Q000628:therapy; D006801:Humans; D007223:Infant; D016030:Kidney Transplantation; D016031:Liver Transplantation; D008297:Male; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome
keywords: chemotherapy; hepatoblastoma; kidney transplantation; liver transplantation; methylmalonic aciduria