TY - JOUR
T1 - A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia
AU - Kapelushnik, J.
AU - Or, R.
AU - Slavin, S.
AU - Nagler, A.
PY - 1997/12/2
Y1 - 1997/12/2
N2 - Allogeneic bone marrow transplantation (BMT) is an effective therapy for Fanconi's anemia (FA). However, mortality and transplant-related complications are usually high due to increased sensitivity to the alkylating agents and radiation commonly used for pre-transplant conditioning. Fludarabine monophosphate is a purine analogue that has been proven effective as a conditioning agent for chronic lymphocytic leukemia patients. We report a child with FA in leukemic transformation with thrombocytopenia and 20% myeloblasts who underwent successful BMT following conditioning with fludarabine/ATG/cyclophosphamide. The regimen was well tolerated, no transplant-related complications were observed, and engraftment was rapid. The child is currently 10 months post-BMT, in excellent clinical condition with a normal blood count, 100% chimerism and no sign of graft-versus-host disease (GVHD). We suggest that this fludarabine-based regimen may be effective in the conditioning of standard, as well as transforming, FA patients for BMT.
AB - Allogeneic bone marrow transplantation (BMT) is an effective therapy for Fanconi's anemia (FA). However, mortality and transplant-related complications are usually high due to increased sensitivity to the alkylating agents and radiation commonly used for pre-transplant conditioning. Fludarabine monophosphate is a purine analogue that has been proven effective as a conditioning agent for chronic lymphocytic leukemia patients. We report a child with FA in leukemic transformation with thrombocytopenia and 20% myeloblasts who underwent successful BMT following conditioning with fludarabine/ATG/cyclophosphamide. The regimen was well tolerated, no transplant-related complications were observed, and engraftment was rapid. The child is currently 10 months post-BMT, in excellent clinical condition with a normal blood count, 100% chimerism and no sign of graft-versus-host disease (GVHD). We suggest that this fludarabine-based regimen may be effective in the conditioning of standard, as well as transforming, FA patients for BMT.
KW - Bone marrow transplantation
KW - Engraftment
KW - Fanconi's anemia
KW - Fludarabine
KW - Toxicity
UR - http://www.scopus.com/inward/record.url?scp=0031436199&partnerID=8YFLogxK
U2 - 10.1038/sj.bmt.1701016
DO - 10.1038/sj.bmt.1701016
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C2 - 9466288
AN - SCOPUS:0031436199
SN - 0268-3369
VL - 20
SP - 1109
EP - 1110
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
IS - 12
ER -