title: Clinical features and survival of asian pediatric patients with paroxysmal nocturnal hemoglobinuria: results from a single center in China.
abstract: OBJECTIVE
  Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease, especially in children. To characterize the clinical presentations and survival, we performed a retrospective analysis of pediatric patients.
  
  
  METHODS
  We reviewed 55 pediatric patients with PNH referred to our hospital from January 1990 through June 2012 to assess clinical presentations, survival, and differences among subcategories.
  
  
  RESULTS
  The overall survival 10 years after diagnosis estimated via the Kaplan-Meier method was 77.6%. The cohort of patients was divided into subcategories of classic PNH, PNH/aplastic anemia (AA), and subclinical PNH (PNH-sc)/AA based on the recently proposed PNH working clinical classification. We found that patients with classic PNH and PNH/AA had larger PNH clones and many more parameters of hemolysis, but patients with PNH-sc/AA had smaller PNH clones, fewer parameters of hemolysis, and a higher rate of bone marrow failure. Our results revealed a high rate of bone marrow failure and a low rate of hemoglobinuria at presentation. Furthermore, thrombotic events were not observed in our patients, which is significantly different from the rate seen in Caucasian patients. Additionally, pediatric patients with PNH may develop bone marrow cytogenetic abnormalities.
  
  
  CONCLUSIONS
  This study provides insight into Chinese pediatric PNH patients and may aid in setting up individualized therapeutic regimens.
authors.affiliation: State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Tianjin, PR China.
authors.forename: Meili
authors.initials: M
authors.lastname: Ge
authors.forename: Jun
authors.initials: J
authors.lastname: Shi
authors.forename: Xingxin
authors.initials: X
authors.lastname: Li
authors.forename: Yingqi
authors.initials: Y
authors.lastname: Shao
authors.forename: Jinbo
authors.initials: J
authors.lastname: Huang
authors.forename: Zhendong
authors.initials: Z
authors.lastname: Huang
authors.forename: Jing
authors.initials: J
authors.lastname: Zhang
authors.forename: Neng
authors.initials: N
authors.lastname: Nie
authors.forename: Yizhou
authors.initials: Y
authors.lastname: Zheng
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic; D001855:Bone Marrow Diseases; D000080983:Bone Marrow Failure Disorders; D002648:Child; D002681:China; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications / Q000209:etiology / Q000401:mortality / Q000473:pathology / Q000503:physiopathology; D006461:Hemolysis; D006801:Humans; D008297:Male; D015996:Survival Rate