melphalan and Histiocytosis--Non-Langerhans-Cell

Topics: Adult; Antineoplastic Agents, Alkylating; Female; Histiocytosis, Non-Langerhans-Cell; Histiocytosis, Sinus; Humans; Infusions, Intra-Arterial; Male; Melphalan; Middle Aged

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.

Topics: Antigens, CD34; Antineoplastic Combined Chemotherapy Protocols; Disease-Free Survival; Graft Survival; Histiocytosis, Non-Langerhans-Cell; Humans; Infant; Melphalan; Myeloablative Agonists; Peripheral Blood Stem Cell Transplantation; Remission Induction; Thiotepa; Transplantation Chimera; Transplantation Conditioning; Transplantation, Homologous; Vidarabine

We report an adult case of reactive hemophagocytic syndrome (RHS) associated with myelodysplastic syndrome (MDS) who received emergency bone marrow transplantation (BMT). Despite methylprednisolone pulse therapy, high-dose gamma-globulin, and chemotherapy containing etoposide, the pancytopenia progressed. After informed consent, the patient underwent syngeneic BMT using melphalan as the conditioning regimen. The patient has been well without relapse of RHS and MDS for more than 2 years after BMT. This result suggests that the above strategy, including BMT, should be considered for the treatment of adult RHS associated with hematological malignancy.

Topics: Adult; Antineoplastic Combined Chemotherapy Protocols; Bone Marrow Transplantation; Disease-Free Survival; Histiocytosis, Non-Langerhans-Cell; Humans; Japan; Male; Melphalan; Myelodysplastic Syndromes; Pancytopenia; Transplantation, Isogeneic