PMID- 21707222
OWN - NLM
STAT- MEDLINE
DCOM- 20111215
LR  - 20211203
IS  - 1521-0669 (Electronic)
IS  - 0888-0018 (Linking)
VI  - 28
IP  - 6
DP  - 2011 Sep
TI  - Immunosuppressive therapy without hematopoietic growth factor exposure in 
      pediatric acquired aplastic anemia.
PG  - 469-78
LID - 10.3109/08880018.2011.568043 [doi]
AB  - Immunosuppressive therapy (IST) is recommended for children with acquired 
      aplastic anemia (AA) who lack a human leukocyte antigen (HLA)-matched sibling 
      donor for hematopoietic cell transplantation (HCT). Hematopoietic growth factors 
      have often been included in IST supportive care, but prolonged exposure may 
      increase the risk of secondary clonal evolution. The authors evaluated response, 
      survival, and the incidence of clonal evolution following cyclosporine-based IST 
      without hematopoietic growth factor exposure in a population-based pediatric 
      cohort, identified retrospectively. Forty-five patients with a median age of 7.3 
      years (range 1.2-17.0 years) were included. Partial (PR) and complete (CR) 
      response was achieved in 82% and 64%, at a median of 55 days (range 11-414 days) 
      and 7.6 months (range 2.8-82.2 months), respectively. Patients with associated 
      seronegative hepatitis had an increased likelihood of PR and CR on multivariate 
      analyses (PR: hazard ratio [HR] 3.15, 95% confidence interval [CI] 1.40, 7.11; 
      CR: HR 2.99, 95% CI 1.35, 6.62), whereas older children were less likely to 
      achieve IST response than children younger than 5 years at diagnosis. Five- and 
      10-year overall survival was 96% +/- 4% and 90% +/- 7%, respectively, and 5-year 
      failure-free survival was 63% +/- 8%. There was no infection-related mortality, 
      although 16.4% of patients had at least 1 episode of documented bacteremia. The 
      5-year cumulative incidence of relapse was 12.9% and of clonal evolution was 
      3.2%. The authors conclude that children with AA who receive IST without 
      hematopoietic growth factor support have excellent response and survival outcomes 
      and a low incidence of clonal evolution.
FAU - Deyell, Rebecca J
AU  - Deyell RJ
AD  - Division of Pediatric Hematology/Oncology/Bone Marrow Transplantation, British 
      Columbia Children's Hospital, University of British Columbia, Vancouver, British 
      Columbia, Canada.
FAU - Shereck, Evan B
AU  - Shereck EB
FAU - Milner, Ruth A
AU  - Milner RA
FAU - Schultz, Kirk R
AU  - Schultz KR
LA  - eng
PT  - Journal Article
DEP - 20110627
PL  - England
TA  - Pediatr Hematol Oncol
JT  - Pediatric hematology and oncology
JID - 8700164
RN  - 0 (Hematopoietic Cell Growth Factors)
RN  - 0 (Immunosuppressive Agents)
RN  - 83HN0GTJ6D (Cyclosporine)
SB  - IM
MH  - Adolescent
MH  - Age Factors
MH  - Anemia, Aplastic/*drug therapy/*mortality
MH  - Child
MH  - Child, Preschool
MH  - Cyclosporine/*administration & dosage
MH  - Disease-Free Survival
MH  - Female
MH  - *Hematopoietic Cell Growth Factors
MH  - Humans
MH  - Immunosuppression Therapy/*methods
MH  - Immunosuppressive Agents/*administration & dosage
MH  - Infant
MH  - Male
MH  - Retrospective Studies
MH  - Survival Rate
EDAT- 2011/06/29 06:00
MHDA- 2011/12/16 06:00
CRDT- 2011/06/29 06:00
PHST- 2011/06/29 06:00 [entrez]
PHST- 2011/06/29 06:00 [pubmed]
PHST- 2011/12/16 06:00 [medline]
AID - 10.3109/08880018.2011.568043 [doi]
PST - ppublish
SO  - Pediatr Hematol Oncol. 2011 Sep;28(6):469-78. doi: 10.3109/08880018.2011.568043. 
      Epub 2011 Jun 27.