Successful application of nonmyeloablative transplantation for paroxysmal nocturnal hemoglobinuria

Kentaro Suenaga, Yoshinobu Kanda, Hironari Niiya, Kunihisa Nakai, Takeshi Saito, Akiko Saito, Mutsuko Ohnishi, Toshio Takeuchi, Ryuji Tanosaki, Atsushi Makimoto, Shuichi Miyawaki, Toshihiro Ohnishi, Sachiyo Kanai, Kensei Tobinai, Yoichi Takaue, Shin Mineishi

Research output: Contribution to journalArticlepeer-review

43 Citations (Scopus)


Objective. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disorder that manifests as hemolytic anemia, venous thrombosis, and deficient hematopoiesis. Although allogeneic hematopoietic stem cell transplantation is considered the only curative therapeutic measure, transplant-related mortality is not negligible. Several studies supported the use of nonmyeloablative stem cell transplantation (NST) for patients of advanced age or with organ dysfunction. Hence, we used NST in a PNH patient who suffered from acute renal failure due to repeated episodes of hemolysis. Materials and Methods. We performed NST using a conditioning regimen consisting of cladribine 0.11 mg/kg × 6, busulfan 4 mg/kg × 2, and rabbit anti-thymocyte globulin 2.5 mg/kg × 2. He received peripheral blood stem cells from his human leukocyte antigen-matched brother. Prophylaxis against graft-vs-host disease was performed with cyclosporine A alone. Chimerism of peripheral blood mononuclear cells was evaluated serially using short tandem repeat analysis and flow cytometry. Results. No meaningful regimen-related toxicities were documented. Donor chimerism of 90 to 100% was achieved on day 14 and thereafter. The patient is doing well, without any recurrence of hemolysis 6 months after transplant. Follow-up chimerism studies confirmed stable and functioning donor-type hematopoiesis. Conclusions. NST may become a safe and curative approach in patients with PNH. Further studies are needed to establish the role of NST for treatment of PNH.

Original languageEnglish
Pages (from-to)639-642
Number of pages4
JournalExperimental Hematology
Issue number5
Publication statusPublished - 2001 May
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Biology
  • Hematology
  • Genetics
  • Cell Biology
  • Cancer Research


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