PMID- 15706582
OWN - NLM
STAT- MEDLINE
DCOM- 20051228
LR  - 20090112
IS  - 1545-5009 (Print)
IS  - 1545-5009 (Linking)
VI  - 45
IP  - 6
DP  - 2005 Nov
TI  - Diagnosis and treatment of children with aplastic anemia.
PG  - 770-80
AB  - BACKGROUND: Long-term survival rates among children diagnosed with severe 
      aplastic anemia (SAA) are excellent due to the success of human leukocyte antigen 
      (HLA)-identical related hematopoietic stem cell transplantation (HSCT), 
      concurrent advances in immunosuppressive treatment (IST), and improved supportive 
      care. The challenge in making treatment recommendations for children with SAA, 
      therefore, is to balance the apparent chronicity and morbidity following IST, 
      with the potential up-front toxicity and complications of HSCT. METHODS: This 
      review provides an update on the diagnosis and a risk-based treatment algorithm 
      for children with acquired SAA. Recent experience using alternative donor HSCT 
      and efforts to extend HSCT eligibility through advances in donor matching, 
      de-escalation of conditioning regimens, and potential marrow graft engineering 
      are highlighted. We discuss IST response rates, risks of relapse, and 
      complications including clonal evolution. CONCLUSIONS: While good treatment 
      options exist for a majority of children diagnosed with SAA, novel 
      non-transplantation treatments for unresponsive and relapsed patients without 
      suitable transplant donors are needed. Further improvements in outcome will 
      ultimately require a more complete understanding of the pathophysiology of 
      aplastic anemia (AA).
CI  - (c) 2005 Wiley-Liss, Inc.
FAU - Kurre, Peter
AU  - Kurre P
AD  - Division of Pediatric Hematology/Oncology, Department of Pediatrics, Oregon 
      Health & Science University, Portland, OR 97239-3098, USA. kurrepe@ohsu.edu
FAU - Johnson, F Leonard
AU  - Johnson FL
FAU - Deeg, H Joachim
AU  - Deeg HJ
LA  - eng
GR  - CA 087948/CA/NCI NIH HHS/United States
GR  - HL 36444/HL/NHLBI NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Review
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Immunosuppressive Agents)
SB  - IM
MH  - Anemia, Aplastic/complications/*diagnosis/*therapy
MH  - Child
MH  - Hematopoietic Stem Cell Transplantation/methods
MH  - Humans
MH  - Immunosuppressive Agents/therapeutic use
MH  - Risk Assessment
MH  - Salvage Therapy/methods
MH  - Treatment Outcome
RF  - 91
EDAT- 2005/02/12 09:00
MHDA- 2005/12/29 09:00
CRDT- 2005/02/12 09:00
PHST- 2005/02/12 09:00 [pubmed]
PHST- 2005/12/29 09:00 [medline]
PHST- 2005/02/12 09:00 [entrez]
AID - 10.1002/pbc.20322 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2005 Nov;45(6):770-80. doi: 10.1002/pbc.20322.