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International Journal of Hematology

, Volume 94, Issue 4, pp 403–407 | Cite as

Strategy for bone marrow transplantation in eculizumab-treated paroxysmal nocturnal hemoglobinuria

  • Kyoko Taniguchi
  • Masaya Okada
  • Satoshi YoshiharaEmail author
  • Akihiro Sawada
  • Tazuko Tokugawa
  • Shinichi Ishii
  • Katsuji Kaida
  • Kazuhiro Ikegame
  • Kentaro Minagawa
  • Toshimitsu Matsui
  • Hiroyasu Ogawa
Case Report

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/m2, 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.

Keywords

Paroxysmal nocturnal hemoglobinuria Bone marrow transplantation Eculizumab Reduced-intensity conditioning 

Notes

Acknowledgments

We are grateful to Ms. Aya Yano, Ms. Kimiko Yamamoto, and Ms. Junko Ikemoto for their excellent technical assistance.

Conflict of interest

The authors declare that they have no conflicts of interest.

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Copyright information

© The Japanese Society of Hematology 2011

Authors and Affiliations

  • Kyoko Taniguchi
    • 1
  • Masaya Okada
    • 1
  • Satoshi Yoshihara
    • 1
    Email author
  • Akihiro Sawada
    • 1
  • Tazuko Tokugawa
    • 1
  • Shinichi Ishii
    • 1
  • Katsuji Kaida
    • 1
  • Kazuhiro Ikegame
    • 1
  • Kentaro Minagawa
    • 2
  • Toshimitsu Matsui
    • 2
  • Hiroyasu Ogawa
    • 1
  1. 1.Division of Hematology, Department of Internal MedicineHyogo College of MedicineNishinomiyaJapan
  2. 2.Division of Hematology, Department of MedicineKobe University Graduate School of MedicineKobeJapan

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