title: Strategy for bone marrow transplantation in eculizumab-treated paroxysmal nocturnal hemoglobinuria.
abstract: Although the recent introduction of eculizumab has had a significant impact on the management of paroxysmal nocturnal hemoglobinuria (PNH), bone marrow transplantation (BMT) remains the only therapeutic option for patients who develop severe aplasia in the clinical course of PNH. However, information regarding BMT for eculizumab-treated PNH patients is scarce, and two major points-the optimal duration of eculizumab therapy, and the optimal BMT conditioning regimen-remain unclear. Here, we describe the clinical course of a PNH patient who was successfully treated with unrelated reduced-intensity BMT. Eculizumab was discontinued 2 weeks prior to the initiation of the conditioning regimen, which consisted of fludarabine 180 mg/m(2), cyclophosphamide 100 mg/kg, rabbit anti-thymocyte globulin 2 mg/kg, and TBI 3 Gy. Complete donor chimerism was rapidly achieved in association with a rapid decrease in the proportion of PNH erythrocytes. The patient became transfusion-free immediately after BMT, and had no recurrence of hemolysis. The present case suggests that discontinuation of eculizumab before BMT and the use of a highly lymphoablative conditioning regimen may act as a successful treatment strategy in BMT for PNH. Further studies are warranted to evaluate the efficacy and safety of this treatment strategy.
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Kyoko
authors.initials: K
authors.lastname: Taniguchi
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Masaya
authors.initials: M
authors.lastname: Okada
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan. yoshihar@hyo-med.ac.jp.
authors.forename: Satoshi
authors.initials: S
authors.lastname: Yoshihara
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Akihiro
authors.initials: A
authors.lastname: Sawada
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Tazuko
authors.initials: T
authors.lastname: Tokugawa
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Shinichi
authors.initials: S
authors.lastname: Ishii
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Katsuji
authors.initials: K
authors.lastname: Kaida
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Kazuhiro
authors.initials: K
authors.lastname: Ikegame
authors.affiliation: Division of Hematology, Department of Medicine, Kobe University Graduate School of Medicine, Kobe, Japan.
authors.forename: Kentaro
authors.initials: K
authors.lastname: Minagawa
authors.affiliation: Division of Hematology, Department of Medicine, Kobe University Graduate School of Medicine, Kobe, Japan.
authors.forename: Toshimitsu
authors.initials: T
authors.lastname: Matsui
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
authors.forename: Hiroyasu
authors.initials: H
authors.lastname: Ogawa
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000379:methods*; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000628:therapy*; D006801:Humans; D008875:Middle Aged; D012074:Remission Induction; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome
chemical_list: D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab