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.

Allogeneic 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.

Cesaro S ，Gazzola MV ，Marson P ，Calore E ，Caenazzo L ，Destro R ，De Silvestro G ，Varotto S ，Pillon M ，Zanesco L ，Messina C ... -  《AMERICAN JOURNAL OF HEMATOLOGY》

Fludarabine and melphalan-based conditioning for patients with advanced hematological malignancies relapsing after a previous hematopoietic stem cell transplant.

Devine SM ，Sanborn R ，Jessop E ，Stock W ，Huml M ，Peace D ，Wickrema A ，Yassine M ，Amin K ，Thomason D ，Chen YH ，Devine H ，Maningo M ，van Besien K ... -  《BONE MARROW TRANSPLANTATION》

Fludarabine/melphalan conditioning for allogeneic transplantation in patients with multiple myeloma.

Giralt S ，Aleman A ，Anagnostopoulos A ，Weber D ，Khouri I ，Anderlini P ，Molldrem J ，Ueno NT ，Donato M ，Korbling M ，Gajewski J ，Alexanian R ，Champlin R ... -  《BONE MARROW TRANSPLANTATION》

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler).

Grigull L ，Beilken A ，Schrappe M ，Das A ，Luecke T ，Sander A ，Stanulla M ，Rehe K ，Sauer M ，Schmid H ，Welte K ，Lukacs Z ，Gal A ，Sykora KW ... -  《-》

Rapid achievement of complete donor chimerism and low regimen-related toxicity after reduced conditioning with fludarabine, carmustine, melphalan and allogeneic transplantation.

Wäsch R ，Reisser S ，Hahn J ，Bertz H ，Engelhardt M ，Kunzmann R ，Veelken H ，Holler E ，Finke J ... -  《BONE MARROW TRANSPLANTATION》