title: Immunosuppressive treatment in children with acquired aplastic anemia.
abstract: OBJECTIVE
  Immunosuppressive treatment (IST) is an alternative for children with acquired aplastic anemia (AA) that do not have HLA-matched donors. The objective of this study was to evaluate the outcome of IST in children with acquired AA.
  
  
  METHODS
  The study included 18 pediatric acquired AA patients that were retrospectively evaluated. The patients either did not have an HLA-matched related donor or were unable to undergo transplantation within 6 months despite having an HLA-matched donor.
  
  
  RESULTS
  In all, 6 of the patients were characterized as very severe AA, 6 as severe AA, and 6 as moderate AA. Mean duration of follow-up was 44.5 months. In total, 9 patients that could not be treated with equine anti-thymocyte globulin (hATG) following diagnosis received high-dose methylprednisolone treatment. Among the 6 very severe AA patients, 2 achieved complete remission (22%); the other 16 patients received hATG+cyclosporine and short-term methylprednisolone. In total, 4 of the patients died during the first month of treatment. Of the remaining 12 patients, 3 responded to the treatment (25%). Of the 9 patients that did not respond after 3 months of treatment, 7 received a second course of immunosuppressive treatment with rabbit ATG (rATG)+cyclosporine and short-term methylprednisolone; 2 of the 7 patients responded (22%), but 5 did not respond to any treatment. Median survival among the patients was as 64 ± 8 months
  
  
  CONCLUSIONS
  Combination IST with ATG+cyclosporine and low-dose methylprednisolone was an effective treatment in the pediatric acquired AA patients with non-identical HLA donors. In the patients that couldn't be treated with ATG high-dose methylprednisolone treatment was safe and effective.
authors.affiliation: Sisli Etfal Training and Research Hospital, Department of Pediatrics, İstanbul, Turkey.
authors.forename: Yıldız
authors.initials: Y
authors.lastname: Yıldırmak
authors.affiliation: Sisli Etfal Training and Research Hospital, Department of Pediatrics, İstanbul, Turkey.
authors.forename: Ela
authors.initials: E
authors.lastname: Erdem
authors.affiliation: Sisli Etfal Training and Research Hospital, Department of Pediatrics, İstanbul, Turkey.
authors.forename: Leyla
authors.initials: L
authors.lastname: Telhan
authors.affiliation: Sisli Etfal Training and Research Hospital, Department of Pediatrics, İstanbul, Turkey.
authors.forename: Laliz
authors.initials: L
authors.lastname: Kepekçi
keywords: Acquired aplastic anemia; Immunosuppressive treatment; children
references.citation: Br J Haematol. 2006 Jun;133(6):606-11
references.pmid: 16704434
references.citation: Pediatr Blood Cancer. 2005 Nov;45(6):770-80
references.pmid: 15706582
references.citation: Pediatr Blood Cancer. 2008 Jan;50(1):52-7
references.pmid: 17941069
references.citation: Exp Hematol. 2006 Jul;34(7):826-31
references.pmid: 16797409
references.citation: Blood. 2010 Jun 24;115(25):5131-6
references.pmid: 20237318
references.citation: Blood. 2000 Mar 15;95(6):1931-4
references.pmid: 10706857
references.citation: Blood. 2005 Sep 15;106(6):2102-4
references.pmid: 15933058
references.citation: Eur J Haematol. 1988 Nov;41(5):508
references.pmid: 3208873
references.citation: Haematologica. 2007 Jan;92(1):2-5
references.pmid: 17229628
references.citation: Bone Marrow Transplant. 2008 Aug;42 Suppl 1:S42-S44
references.pmid: 18724299
references.citation: Br J Haematol. 2008 Jan;140(2):197-205
references.pmid: 18173756
references.citation: Pediatr Hematol Oncol. 2009 Oct-Nov;26(7):487-95
references.pmid: 19863204
references.citation: Blood. 2002 Aug 1;100(3):799-803
references.pmid: 12130489
references.citation: Blood. 2002 Dec 15;100(13):4668-70
references.pmid: 12393567
references.citation: JAMA. 2003 Mar 5;289(9):1130-5
references.pmid: 12622583
references.citation: Haematologica. 2004 Sep;89(9):1054-61
references.pmid: 15377466
references.citation: Blood. 2006 Oct 15;108(8):2509-19
references.pmid: 16778145