PMID- 19148946
OWN - NLM
STAT- MEDLINE
DCOM- 20090406
LR  - 20131121
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 52
IP  - 5
DP  - 2009 May
TI  - Tacrolimus as an alternative to cyclosporine in the maintenance phase of 
      immunosuppressive therapy for severe aplastic anemia in children.
PG  - 626-30
LID - 10.1002/pbc.21926 [doi]
AB  - OBJECTIVE: Given the paucity of data on the use of agents other than cyclosporine 
      (CsA) in the maintenance phase of immunosuppressive therapy (IST) for severe 
      aplastic anemia (SAA) in children, we sought to describe our experience with 
      tacrolimus in pediatric SAA, and to compare outcomes with a preceding series of 
      patients who received CsA. METHODS: Eight patients with SAA diagnosed between 
      2003 and 2008 for whom no human leukocyte antigen (HLA)-matched sibling donor was 
      identified underwent tacrolimus-based IST. These children were compared with a 
      previously described series of 13 patients who had undergone CsA-based IST at our 
      institution between 1990 and 2003. All patients initially received equine 
      antithymocyte globulin (ATG) and corticosteroids. RESULTS: Complete response (CR) 
      rate was 88% for tacrolimus and 85% for CsA. Median time to CR was approximately 
      7 months in both groups. Median follow-up duration was 2.4 years for tacrolimus 
      and 8.4 years for CsA. Among responders with de novo SAA, relapse rate was 25% (n 
      = 1) at 2 years for tacrolimus and 0% at 2 years and 23% (n = 3) at 5 years for 
      CsA; no significant difference in relapse-free survival was detected between the 
      two groups (P = 0.07). Paroxysmal nocturnal hemoglobinuria was seen in one 
      patient on tacrolimus who had relapsed after CsA-based IST. Tacrolimus-based IST 
      was well-tolerated. CONCLUSION: These data provide evidence that tacrolimus may 
      be a suitable alternative to CsA as part of an IST regimen for SAA in children 
      who lack an HLA-matched sibling and may have a more favorable profile of side 
      effects than CsA.
CI  - (c) 2009 Wiley-Liss, Inc.
FAU - Alsultan, Abdulrahman
AU  - Alsultan A
AD  - Center for Cancer and Blood Disorders, The Children's Hospital, University of 
      Colorado Denver, Aurora, Colorado 80045, USA. aalsultan@gmail.com
FAU - Goldenberg, Neil A
AU  - Goldenberg NA
FAU - Kaiser, Nicole
AU  - Kaiser N
FAU - Graham, Douglas K
AU  - Graham DK
FAU - Hays, Taru
AU  - Hays T
LA  - eng
PT  - Controlled Clinical Trial
PT  - Journal Article
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Immunosuppressive Agents)
RN  - 83HN0GTJ6D (Cyclosporine)
RN  - WM0HAQ4WNM (Tacrolimus)
SB  - IM
MH  - Adolescent
MH  - Anemia, Aplastic/complications/*drug therapy/*pathology
MH  - Child
MH  - Child, Preschool
MH  - Cyclosporine/*therapeutic use
MH  - Disease-Free Survival
MH  - Humans
MH  - Immunosuppressive Agents/*therapeutic use
MH  - Tacrolimus/*therapeutic use
MH  - Treatment Outcome
MH  - Young Adult
EDAT- 2009/01/17 09:00
MHDA- 2009/04/07 09:00
CRDT- 2009/01/17 09:00
PHST- 2009/01/17 09:00 [entrez]
PHST- 2009/01/17 09:00 [pubmed]
PHST- 2009/04/07 09:00 [medline]
AID - 10.1002/pbc.21926 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2009 May;52(5):626-30. doi: 10.1002/pbc.21926.