PMID- 18173756
OWN - NLM
STAT- MEDLINE
DCOM- 20080423
LR  - 20131121
IS  - 1365-2141 (Electronic)
IS  - 0007-1048 (Linking)
VI  - 140
IP  - 2
DP  - 2008 Jan
TI  - Cyclosporin A response and dependence in children with acquired aplastic anaemia: 
      a multicentre retrospective study with long-term observation follow-up.
PG  - 197-205
LID - 10.1111/j.1365-2141.2007.06903.x [doi]
AB  - Immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporin A 
      (CyA) is the standard treatment for children with acquired aplastic anaemia (AAA) 
      lacking a matched donor. Survival rates of more than 80% at 5 years are achieved, 
      but the response is drug-dependent in 15-25% of cases. This study, of 42 
      consecutive children with AAA treated with IST, assessed the incidence of 
      CyA-dependence, CyA and granulocyte colony-stimulating factor (G-CSF) tapering 
      schedules and the impact of drug accumulation on progression to 
      myelodysplasia/acute myeloid leukaemia (MDS/AML). Overall survival was 83% at 10 
      years. CyA-dependence without a predictive marker was observed in 18% of 
      responders. Probability of discontinuing CyA was 60.5% at 10 years; a slow CyA 
      tapering schedule was performed in 84% of patients; the cumulative incidence of 
      relapse was 16% at 10 years. Relapse risk was significantly associated with rapid 
      CyA discontinuation: 60% compared to 7.6% in the slow tapering group (P = 0.001). 
      Cumulative incidence of MDS/AML was 8% at 10 years, with a significant 
      correlation with both G-CSF cumulative dose and second IST. This long-term 
      follow-up of children with AAA shows that IST with a slow CyA tapering course is 
      an effective treatment with a low-relapse rate in these cases.
FAU - Saracco, Paola
AU  - Saracco P
AD  - Paediatric Haematology, University of Turin, Turin, Italy. paola.saracco@unito.it
FAU - Quarello, Paola
AU  - Quarello P
FAU - Iori, Anna Paola
AU  - Iori AP
FAU - Zecca, Marco
AU  - Zecca M
FAU - Longoni, Daniela
AU  - Longoni D
FAU - Svahn, Johanna
AU  - Svahn J
FAU - Varotto, Stefania
AU  - Varotto S
FAU - Del Vecchio, Gian Carlo
AU  - Del Vecchio GC
FAU - Dufour, Carlo
AU  - Dufour C
FAU - Ramenghi, Ugo
AU  - Ramenghi U
FAU - Bacigalupo, Andrea
AU  - Bacigalupo A
FAU - Locasciulli, Anna
AU  - Locasciulli A
CN  - Bone Marrow Failure Study Group of the AIEOP (Italian Association of Paediatric 
      Haematology Oncology)
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PT  - Research Support, Non-U.S. Gov't
PL  - England
TA  - Br J Haematol
JT  - British journal of haematology
JID - 0372544
RN  - 0 (Immunosuppressive Agents)
RN  - 143011-72-7 (Granulocyte Colony-Stimulating Factor)
RN  - 83HN0GTJ6D (Cyclosporine)
SB  - IM
MH  - Adolescent
MH  - Anemia, Aplastic/blood/*drug therapy
MH  - Child
MH  - Child, Preschool
MH  - Cyclosporine/administration & dosage/blood/*therapeutic use
MH  - Disease Progression
MH  - Drug Administration Schedule
MH  - Drug Evaluation
MH  - Drug Therapy, Combination
MH  - Epidemiologic Methods
MH  - Female
MH  - Granulocyte Colony-Stimulating Factor/administration & dosage/therapeutic use
MH  - Humans
MH  - Immunosuppressive Agents/administration & dosage/blood/*therapeutic use
MH  - Infant
MH  - Male
MH  - Recurrence
MH  - Treatment Outcome
EDAT- 2008/01/05 09:00
MHDA- 2008/04/24 09:00
CRDT- 2008/01/05 09:00
PHST- 2008/01/05 09:00 [pubmed]
PHST- 2008/04/24 09:00 [medline]
PHST- 2008/01/05 09:00 [entrez]
AID - BJH6903 [pii]
AID - 10.1111/j.1365-2141.2007.06903.x [doi]
PST - ppublish
SO  - Br J Haematol. 2008 Jan;140(2):197-205. doi: 10.1111/j.1365-2141.2007.06903.x.