title: Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): an initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC).
abstract: BACKGROUND
  Randomized clinical trials in pediatric aplastic anemia (AA) are rare and data to guide standards of care are scarce.
  
  
  METHODS
  Eighteen pediatric institutions formed the North American Pediatric Aplastic Anemia Consortium to foster collaborative studies in AA. The initial goal of NAPAAC was to survey the diagnostic studies and therapies utilized in AA.
  
  
  RESULTS
  Our survey indicates considerable variability among institutions in the diagnosis and treatment of AA. There were areas of general consensus, including the need for a bone marrow evaluation, cytogenetic and specific fluorescent in situ hybridization assays to establish diagnosis and exclude genetic etiologies with many institutions requiring results prior to initiation of immunosuppressive therapy (IST); uniform referral for hematopoietic stem cell transplantation as first line therapy if an HLA-identical sibling is identified; the use of first-line IST containing horse anti-thymocyte globulin and cyclosporine A (CSA) if an HLA-identical sibling donor is not identified; supportive care measures; and slow taper of CSA after response. Areas of controversy included the need for telomere length results prior to IST, the time after IST initiation defining a treatment failure; use of hematopoietic growth factors; the preferred rescue therapy after failure of IST; the use of specific hemoglobin and platelet levels as triggers for transfusion support; the use of prophylactic antibiotics; and follow-up monitoring after completion of treatment.
  
  
  CONCLUSIONS
  These initial survey results reflect heterogeneity in diagnosis and care amongst pediatric centers and emphasize the need to develop evidence-based diagnosis and treatment approaches in this rare disease.
authors.affiliation: Division of Hematology/Oncology, Boston Children's Hospital and Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts.
authors.forename: David A
authors.initials: DA
authors.lastname: Williams
authors.forename: Carolyn
authors.initials: C
authors.lastname: Bennett
authors.forename: Alison
authors.initials: A
authors.lastname: Bertuch
authors.forename: Monica
authors.initials: M
authors.lastname: Bessler
authors.forename: Thomas
authors.initials: T
authors.lastname: Coates
authors.forename: Seth
authors.initials: S
authors.lastname: Corey
authors.forename: Yigal
authors.initials: Y
authors.lastname: Dror
authors.forename: James
authors.initials: J
authors.lastname: Huang
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Lipton
authors.forename: Timothy S
authors.initials: TS
authors.lastname: Olson
authors.forename: Ulrike M
authors.initials: UM
authors.lastname: Reiss
authors.forename: Zora R
authors.initials: ZR
authors.lastname: Rogers
authors.forename: Colin
authors.initials: C
authors.lastname: Sieff
authors.forename: Adrianna
authors.initials: A
authors.lastname: Vlachos
authors.forename: Kelly
authors.initials: K
authors.lastname: Walkovich
authors.forename: Winfred
authors.initials: W
authors.lastname: Wang
authors.forename: Akiko
authors.initials: A
authors.lastname: Shimamura
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000175:diagnosis* / Q000188:drug therapy* / Q000453:epidemiology; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D019538:Health Care Surveys; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D009656:North America / Q000453:epidemiology; D010818:Practice Patterns, Physicians'; D011379:Prognosis; D035781:Siblings
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine
keywords: NAPAAC; acquired aplastic anemia; diagnosis; treatment