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Sequential Related Donor Partial Liver Transplantation and Bone Marrow Transplantation

Development stage
Unknown
Lead developer
Sidney Kimmel Comprehensive Cancer Center
Modality
Small Molecules, Antibody-Based Therapeutics, Stem Cell Therapies → Cell Therapies
Administration
Intravenous, Surgical
01

Overview

This specialized therapeutic regimen, developed by the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, is designed for patients with hepatocellular carcinoma (HCC), including the fibrolamellar subtype. The protocol involves a sequential approach: first, a partial liver transplantation from a Human Leukocyte Antigen (HLA)-matched or haploidentical living related donor, followed by a bone marrow transplantation (BMT) from the same donor. The regimen utilizes a reduced-intensity conditioning (RIC) protocol that includes total body irradiation (TBI), fludarabine, and antithymocyte globulin. A hallmark of this approach is the use of high-dose post-transplantation cyclophosphamide (PTCy) to prevent graft-versus-host disease (GVHD) and induce hematopoietic chimerism. This chimerism is intended to promote immune tolerance to the liver allograft, potentially allowing for the withdrawal of long-term immunosuppression and providing a graft-versus-tumor effect to reduce the risk of cancer recurrence.

Other names
BMT after Liver Transplant for HCCSequential Related Donor Partial Liver Transplantation Followed by Bone Marrow Transplantation for Fibrolamellar or Non-fibrolamellar Hepatocellular Carcinoma

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