title: [Outcomes of allogeneic hematopoietic stem cell transplantation for 18 patients with paroxysmal nocturnal haemoglobinuria].
abstract: OBJECTIVE
  To evaluate the outcomes of allogeneic hematopoietic stem cell transplantation（allo-HSCT）for paroxysmal nocturnal haemoglobinuria（PNH）and aplastic anemia（AA）- PNH syndrome.
  
  
  METHODS
  The clinical data of 18 PNH or AA-PNH patients, including 4 classic PNH and 14 AA-PNH, received allo-HSCT from Dec 2007 to Feb 2015 were analyzed retrospectively. Nine patients received HLA-haploidentical donor HSCT（1 patient received salvage HLA-haploidentical donor HSCT after the graft failure of double cord blood transplantation）, 7 patients received HLA-identical sibling donor HSCT, and 2 HLA-identical unrelated donor HSCT. The conditioning regimens were as follow: 13 patients received modified BU/CY- based regimens, 5 non- myeloablative regimens ［fludarabine （Flu） + antithymocyte globulin（ATG）+ cyclophosphamide（CY）or busulfan（BU）］. Prophylaxis for graft- versushost disease（GVHD）: the patients with HLA-identical sibling donor received cyclosporine（CsA）plus short-term methotrexate（MTX）, the patients with HLA -haploidentical donor or HLA-identical unrelated donor received CsA or tacrolimus（FK506）+ mycophenolate mofetil（MMF）+ short- term methotrexate （MTX）.
  
  
  RESULTS
  All patients were engrafted successfully（1 patient engrafted by haploidentical donor after the graft failure of double cord blood transplantation）. The median days of neutrophils（ANC）above 0.5 × 109/L and platelets （PLT） more than 20 × 10⁹/L were 11（10- 26）days and 15（11- 120）days, respectively. Three patients（17.6%）developed acute GVHD（aGVHD）, 2 for grade Ⅱ aGVHD, 1 for grade Ⅳ aGVHD. Of 16 patients, 2 occurred limited chronic GVHD（cGVHD）. After a median follow-up of 14.6（2.0-86.7）months, 3 patients（17.6%）died, out of which one died of severe aGVHD, one died of severe pulmonary infection, one pulmonary infection with transplant- associated thrombotic microangiopathy. The 5- year estimated disease free survival was（80.5 ± 10.2）%. No patient relapsed.
  
  
  CONCLUSIONS
  Allo-HSCT is an effective and curable therapy for PNH or AA-PNH with improved prognosis, and offers a valid therapeutic option for these patients before humanized monoclonal antibody against C5 are widely used clinically.
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Feng
authors.initials: F
authors.lastname: Chen
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Depei
authors.initials: D
authors.lastname: Wu
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Xiaowen
authors.initials: X
authors.lastname: Tang
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Miao
authors.initials: M
authors.lastname: Miao
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Chengcheng
authors.initials: C
authors.lastname: Fu
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Huiying
authors.initials: H
authors.lastname: Qiu
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Zhengming
authors.initials: Z
authors.lastname: Jin
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Shengli
authors.initials: S
authors.lastname: Xue
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Xiao
authors.initials: X
authors.lastname: Ma
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Aining
authors.initials: A
authors.lastname: Sun
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Weirong
authors.initials: W
authors.lastname: Chang
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Changgeng
authors.initials: C
authors.lastname: Ruan
mesh_terms: D000741:Anemia, Aplastic / Q000628:therapy*; D000961:Antilymphocyte Serum; D002066:Busulfan; D003520:Cyclophosphamide; D016572:Cyclosporine; D018572:Disease-Free Survival; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation; D006457:Hemoglobinuria, Paroxysmal / Q000628:therapy*; D006801:Humans; D008727:Methotrexate; D009173:Mycophenolic Acid / Q000031:analogs & derivatives; D012189:Retrospective Studies; D035781:Siblings; D016559:Tacrolimus; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D061349:Unrelated Donors; D014740:Vidarabine / Q000031:analogs & derivatives
chemical_list: D000961:Antilymphocyte Serum; D016572:Cyclosporine; D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; D009173:Mycophenolic Acid; C024352:fludarabine; D016559:Tacrolimus; D008727:Methotrexate