title: First Attempt of Sequential Living Donor Liver and Hematopoietic Stem Cell Transplantation in a Child With Advanced Hepatocellular Carcinoma: Case Report.
abstract: Effective therapeutic options for advanced hepatocellular carcinoma are limited. Hematopoietic stem cell transplantation may offer a graft-versus-tumor effect. Combined liver and hematopoietic stem cell transplantation from the same donor with preparatory conditioning may promote tolerogenicity to the liver allograft and offers the potential for immunosuppression withdrawal. We report our experience with the use of this approach in a pediatric patient with invasive hepatocellular carcinoma and pulmonary metastases who underwent a living-donor liver transplantation followed by reduced-toxicity myeloablative conditioning and hematopoietic stem cell transplant from the same parental donor. Neutrophil engraftment and full donor chimerism was achieved without liver allograft dysfunction. Despite normal liver function and marrow engraftment, the patient succumbed to multisystem organ failure from disseminated toxoplasmosis. At autopsy, there was no histologic evidence of tumor recurrence. No pulmonary nodules were found. Regardless of the unfortunate overall result, this case demonstrates preliminary feasibility of sequential living-donor liver transplantation and hematopoietic stem cell transplantation for unresectable and metastasized hepatic tumors. Future studies in select pediatric patients require evaluation of the optimal conditioning regimen and prevention strategies for opportunistic infections to determine both graft-versus-tumor effect on hepatic tumors and durability of tolerogenicity and possible immunosuppression withdrawal.
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Columbia University Medical Center, New York, New York.
authors.forename: J A
authors.initials: JA
authors.lastname: Picoraro
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Columbia University Medical Center, New York, New York; Department of Pediatrics, Albert Einstein College of Medicine, Bronx, New York (present address).
authors.forename: N
authors.initials: N
authors.lastname: Ovchinsky
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Columbia University Medical Center, New York, New York.
authors.forename: M
authors.initials: M
authors.lastname: Martinez
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Columbia University Medical Center, New York, New York.
authors.forename: S J
authors.initials: SJ
authors.lastname: Lobritto
authors.affiliation: Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Columbia University Medical Center, New York, New York.
authors.forename: P
authors.initials: P
authors.lastname: Satwani
authors.affiliation: Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, Ontario.
authors.forename: R
authors.initials: R
authors.lastname: Ramphal
authors.affiliation: Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Columbia University Medical Center, New York, New York; Department of Pediatrics, New York Medical College, Valhalla, New York (present address).
authors.forename: M S
authors.initials: MS
authors.lastname: Cairo
authors.affiliation: Department of Surgery, Columbia University Medical Center, New York, New York. Electronic address: tk2388@cumc.columbia.edu.
authors.forename: T
authors.initials: T
authors.lastname: Kato
mesh_terms: D006528:Carcinoma, Hepatocellular / Q000601:surgery*; D002648:Child; D003131:Combined Modality Therapy / Q000379:methods*; D017809:Fatal Outcome; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D016867:Immunocompromised Host / Q000276:immunology; D008113:Liver Neoplasms / Q000601:surgery*; D016031:Liver Transplantation / Q000379:methods*; D019520:Living Donors; D008297:Male; D014123:Toxoplasmosis / Q000276:immunology; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous