PMID- 10676915
OWN - NLM
STAT- MEDLINE
DCOM- 20000225
LR  - 20191103
IS  - 0037-1963 (Print)
IS  - 0037-1963 (Linking)
VI  - 37
IP  - 1
DP  - 2000 Jan
TI  - Cyclophosphamide and other new agents for the treatment of severe aplastic 
      anemia.
PG  - 102-9
AB  - Severe aplastic anemia (SAA) has a poor prognosis in the absence of treatment. 
      Current accepted therapeutic strategies include allogeneic stem-cell 
      transplantation and immunosuppression, both resulting in long-term survival in 
      the majority of patients. Although human leukocyte antigen (HLA)-matched sibling 
      stem-cell transplantation is highly effective, the 25% probability of finding a 
      suitable sibling donor within a family renders this approach available to only a 
      minority of patients. Transplantation using HLA-matched, unrelated donors carries 
      a high risk of treatment failure along with considerable toxicity. While combined 
      immunosuppression with both antithymocyte globulin (ATG) and cyclosporine A (CSA) 
      produces hematologic improvement in most patients, relapse is common. Late 
      evolution of aplastic anemia to other serious hematologic disorders, including 
      paroxysmal nocturnal hemoglobinuria (PNH), myelodysplasia, and acute leukemia, is 
      also a significant problem following treatment with ATG/CSA. Recently, results of 
      immunosuppression in SAA with another potent immunosuppressive agent, 
      cyclophosphamide, were reported in a small number of patients. The overall 
      response rate was similar to that seen with ATG/CSA, but relapse and late clonal 
      disease were not observed during a long period of follow-up. A larger randomized 
      trial comparing sustained hematologic response rates to either conventional 
      immunosuppression with ATG/CSA or high-dose cyclophosphamide and CSA is now 
      underway; secondary end points include response duration, event-free survival, 
      and overall survival. Additionally, a number of protocols designed to test the 
      efficacy of alternative immunosuppressive or immunomodulatory agents are being 
      developed.
FAU - Tisdale, J F
AU  - Tisdale JF
AD  - Molecular and Clinical Hematology Branch, National Institute of Diabetes and 
      Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, 
      USA.
FAU - Dunn, D E
AU  - Dunn DE
FAU - Maciejewski, J
AU  - Maciejewski J
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Semin Hematol
JT  - Seminars in hematology
JID - 0404514
RN  - 0 (Antibodies, Monoclonal)
RN  - 0 (Immunosuppressive Agents)
RN  - 0 (Receptors, Interleukin-2)
RN  - 8N3DW7272P (Cyclophosphamide)
RN  - HU9DX48N0T (Mycophenolic Acid)
RN  - W36ZG6FT64 (Sirolimus)
RN  - WM0HAQ4WNM (Tacrolimus)
SB  - IM
MH  - Anemia, Aplastic/*drug therapy
MH  - Antibodies, Monoclonal/therapeutic use
MH  - Cyclophosphamide/*therapeutic use
MH  - Humans
MH  - Immunosuppressive Agents/*therapeutic use
MH  - Mycophenolic Acid/analogs & derivatives/therapeutic use
MH  - Receptors, Interleukin-2/antagonists & inhibitors/immunology
MH  - Sirolimus/therapeutic use
MH  - Tacrolimus/therapeutic use
RF  - 75
EDAT- 2000/02/17 09:00
MHDA- 2000/03/04 09:00
CRDT- 2000/02/17 09:00
PHST- 2000/02/17 09:00 [pubmed]
PHST- 2000/03/04 09:00 [medline]
PHST- 2000/02/17 09:00 [entrez]
AID - S003719630000010X [pii]
AID - 10.1016/s0037-1963(00)90034-9 [doi]
PST - ppublish
SO  - Semin Hematol. 2000 Jan;37(1):102-9. doi: 10.1016/s0037-1963(00)90034-9.