Fanconi Anemia 1989
DOI: 10.1007/978-3-642-74179-1_5
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Bone Marrow Transplantation for Fanconi Anemia

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Cited by 154 publications
(216 citation statements)
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“…Patients frequently develop BM failure in the second decade of life [1]. HLA-matched BM or cord blood transplantation remains the only curative option; however, only approximately onethird of patients are able to find an HLA-matched donor [3]. Therefore, there is a great need to develop other treatments.…”
Section: Discussionmentioning
confidence: 99%
See 1 more Smart Citation
“…Patients frequently develop BM failure in the second decade of life [1]. HLA-matched BM or cord blood transplantation remains the only curative option; however, only approximately onethird of patients are able to find an HLA-matched donor [3]. Therefore, there is a great need to develop other treatments.…”
Section: Discussionmentioning
confidence: 99%
“…The progressive BM failure and the late developing myeloid malignancies account for 90% of the mortality in FA [1]. Aside from cord blood transplantation [2], the only cure for the hematopoietic manifestations of FA is an HLA identical allogeneic BM transplantation from a family member, a therapy available to only about 30% of patients [3]. Thirteen FA complementation types (FANCA, FANCB, FANCC, FANCD1, FANCD2, FANCE, FANCF, FANCG, FANCI, FANCJ, FANCL, FANCM and FANCN) have been identified and the cDNAs of twelve FA genes have been sequenced .…”
Section: Introductionmentioning
confidence: 99%
“…Flu, by virtue of its profound immunosuppressive effect, has improved engraftment without increasing the risk of toxicity. In the pre-Flu era, overall survival was dramatically down to around 30%, 8,10 but with the introduction of this agent, it has remarkably increased to 53% 17 with a top percentage of 96. 18 TRM, which in alternative donor HSCT historically had rates of about 70-80%, in the largest study ever performed on alternative donor transplants in FA, 17 has remarkably declined from 81 to 47% with the use of Flu.…”
Section: Hcst From Alternative Donorsmentioning
confidence: 99%
“…As transfusions increase the risk of graft failure, 10 it is better to perform the transplant before the patient becomes at increased risk for transfusions or has bleeding symptoms due to minor traumas, or has actually been transfused. This stage usually corresponds to that of a platelet count between 30 and 40 Â 10 9 /l and haemoglobin around 9 g/100 ml.…”
Section: Algorithm For Treatment Decisionmentioning
confidence: 99%
“…[1][2][3][4][5] For bone marrow failure, it has been universally accepted and proven that allogeneic SCT is the therapy of choice, [6][7][8][9][10][11][12][13][14][15] but the optimal conditioning regimen remains undetermined.…”
Section: Introductionmentioning
confidence: 99%