Allogeneic hematopoietic stem cell transplantation (HSCT) represents the onlycurative option for primary hemophagocytic lymphohistiocytosis (HLH), a raredisease of infants and young children, characterized by recurrent fever,hepatosplenomegaly, and cytopenia. We report a case of successful engraftment andstable full-donor chimerism in a patient with HLH who underwent peripheralallogeneic CD34-selected HSCT. The donor was his 1-antigen-HLA-mismatchedgrandmother. After a conditioning regimen based on the combination of thiotepa,fludarabine, melphalan, and rabbit antilymphocyte serum, the patient received amegadose of 26.3 x 10(6)/kg of CD34(+) peripheral blood cells. Neutrophil (>0.5 x10(9)/L) and platelet (>50 x 10(9)/L) engraftment was observed on days +16 and+12, respectively, and the patient was discharged home on day +24. No acute orchronic GVHD was observed. Infectious complications were the main causes ofre-hospitalization in the first year after transplantation, but no significantmorbidity was observed thereafter. Thirty-two months after HSCT, the patient isalive and well, still in complete clinical remission of his underlying diseasewith a durable engraftment, normal NK activity and full donor chimerism. Thiscase suggests that a fludarabine-based conditioning regimen and CD34-selectedperipheral allogeneic HSCT may be a feasible option in case of unavailability of a fully HLA-matched related or unrelated donor.
Successful engraftment and stable full donor chimerism after myeloablation with thiotepa, fludarabine, and melphalan and CD34-selected peripheral allogeneic stem cell transplantation in hemophagocytic lymphohistiocytosis
CESARO, SIMONE;
2003-01-01
Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) represents the onlycurative option for primary hemophagocytic lymphohistiocytosis (HLH), a raredisease of infants and young children, characterized by recurrent fever,hepatosplenomegaly, and cytopenia. We report a case of successful engraftment andstable full-donor chimerism in a patient with HLH who underwent peripheralallogeneic CD34-selected HSCT. The donor was his 1-antigen-HLA-mismatchedgrandmother. After a conditioning regimen based on the combination of thiotepa,fludarabine, melphalan, and rabbit antilymphocyte serum, the patient received amegadose of 26.3 x 10(6)/kg of CD34(+) peripheral blood cells. Neutrophil (>0.5 x10(9)/L) and platelet (>50 x 10(9)/L) engraftment was observed on days +16 and+12, respectively, and the patient was discharged home on day +24. No acute orchronic GVHD was observed. Infectious complications were the main causes ofre-hospitalization in the first year after transplantation, but no significantmorbidity was observed thereafter. Thirty-two months after HSCT, the patient isalive and well, still in complete clinical remission of his underlying diseasewith a durable engraftment, normal NK activity and full donor chimerism. Thiscase suggests that a fludarabine-based conditioning regimen and CD34-selectedperipheral allogeneic HSCT may be a feasible option in case of unavailability of a fully HLA-matched related or unrelated donor.
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