• Simone Cesaro, Maria Vittoria Gazzola, Piero Marson, Elisabetta Calore, Luciana Caenazzo, Roberta Destro, Giustina De Silvestro, Stefania Varotto, Marta Pillon, Luigi Zanesco, and Chiara Messina.
• Clinic of Pediatric Oncology-Hematology, Department of Pediatrics, University of Padua, Via Giustiniani 3, 35128 Padua, Italy. simone.cesaro@unipd.it
• Am. J. Hematol. 2003 Feb 1; 72 (2): 143-6.

AbstractAllogeneic hematopoietic stem cell transplantation (HSCT) represents the only curative option for primary hemophagocytic lymphohistiocytosis (HLH), a rare disease of infants and young children, characterized by recurrent fever, hepatosplenomegaly, and cytopenia. We report a case of successful engraftment and stable full-donor chimerism in a patient with HLH who underwent peripheral allogeneic CD34-selected HSCT. The donor was his 1-antigen-HLA-mismatched grandmother. After a conditioning regimen based on the combination of thiotepa, fludarabine, melphalan, and rabbit antilymphocyte serum, the patient received a megadose of 26.3 x 10(6)/kg of CD34(+) peripheral blood cells. Neutrophil (>0.5 x 10(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 or chronic GVHD was observed. Infectious complications were the main causes of re-hospitalization in the first year after transplantation, but no significant morbidity was observed thereafter. Thirty-two months after HSCT, the patient is alive and well, still in complete clinical remission of his underlying disease with a durable engraftment, normal NK activity and full donor chimerism. This case suggests that a fludarabine-based conditioning regimen and CD34-selected peripheral allogeneic HSCT may be a feasible option in case of unavailability of a fully HLA-matched related or unrelated donor.Copyright 2003 Wiley-Liss, Inc.

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