Successful stem cell transplantation following orthotopic liver transplantation from the same haploidentical family donor in a girl with hemophagocytic lymphohistiocytosis

• Published in: Blood Vol. 96; no. 12; pp. 3997 - 3999
• Main Authors: Matthes-Martin, Susanne, Peters, Christina, Königsrainer, Alfred, Fritsch, Gerhard, Lion, Thomas, Heitger, Andreas, Kapelari, Klaus, Kronberger, Martina, Offner, Felix, Wrba, Fritz, Margreiter, Raimund, Gadner, Helmut
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 01-12-2000; The Americain Society of Hematology
• Subjects: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Behavior Therapy; Biological and medical sciences; Bone marrow, stem cells transplantation. Graft versus host reaction; Female; Graft Rejection - prevention & control; Graft Survival - immunology; Haplotypes - immunology; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic Stem Cell Transplantation - standards; Histiocytosis, Non-Langerhans-Cell - complications; Histiocytosis, Non-Langerhans-Cell - surgery; Humans; Immune Tolerance; Immunophenotyping; Immunosuppression - methods; Infant; Liver Failure, Acute - etiology; Liver Failure, Acute - surgery; Liver Transplantation - immunology; Liver Transplantation - methods; Liver Transplantation - standards; Liver, biliary tract, pancreas, portal circulation, spleen; Medical sciences; Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases; Surgery of the digestive system; T-Lymphocyte Subsets; Tissue Donors; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation Chimera; Human; Transfusion; Stem cell; Liver; Related donor; Hematopoietic cell; Homograft; Hepatic disease; Hemopathy; Transplantation; Congenital disease; Case study; Hemophagocytic lymphohistiocytosis; Liver failure; Treatment; Mother; Surgery; Digestive diseases; Graft; Child; Lymphocyte
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V96.12.3997

The case of a 4-month-old girl with familial hemophagocytic lymphohistiocytosis is described. The patient underwent stem cell transplantation from her haploidentical mother 2 months after receiving a living-related liver transplant from the same donor for acute hepatic failure. Conditioning regimen consisted of 16 mg/kg busulfan, 200 mg/kg cyclophosphamide, 10 mg/kg thiothepa, and antithymocyte globulin. Myeloid engraftment occurred on day +10, but CD3+ cells of recipient origin remained. To convert the T-cell chimerism, the patient received donor lymphocyte infusion on day +43, and subsequently the allelic pattern changed to complete donor genotype on day +57. Four months after stem cell transplantation the patient is disease free, with complete donor chimerism in bone marrow and stable hepatic graft function without any immunosuppressive therapy.