Yazarlar : Ayas M, Siddiqui K, Al-Jefri A et al

Yayın : Biol Blood Marrow Transplant

Yayın Yılı : 2014

Pubmed Linki : http://www.docguide.com/factors-affecting-outcome-related-allogeneic-hematopoietic-cell-transplantation-patients-fanconi-ane?tsid=5

Konu : Anemi

Literatür İçeriği :  Hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi Anemia (FA), and it is generally accepted that these patients should receive low intensity conditioning due to the underlying DNA repair defect in their cells. Outcomes of recipients of matched related HCT have generally been favorable, but only few studies have scrutinized the factors that may impact on the eventual outcome of these patients. This retrospective analysis of 94 pediatric patients with FA who underwent related HCT at KFSHRC was carried out to attempt to identify factors that may impact on outcome. Results showed overall survival probabilities (OS) of 92.5%, 89%, and 86% at 1, 5, and 10 years, respectively. In univariate analysis, use of higher dose cyclophosphamide (CY; 60mg/kg) conditioning was associated with a better 10-year OS than lower dose CY (20mg/kg) conditioning (91 % vs. 82 %, respectively; P =0.035), and use of radiation-containing regimens was associated with a significantly lower 10-year OS than non-radiation regimens (76 % vs. 91 %, respectively; P =0.005). Of all the 4 regimens used in this study, the fludarabine based regimen was associated with the highest survival (95.2%; P= 0.034). The use of the higher CY dose (60mg/kg) was associated with a significantly increased incidence of hemorrhagic cystitis (20% vs. 5.6% respectively; P =0.049). Three patients (3%) developed squamous cell carcinoma (2 oropharyngeal and one genitourinary), at 9.5, 5.5, 14 years post HCT; 2 of them had radiation containing conditioning. In conclusion, our data suggest that while using a higher dose CY (60 mg/kg) conditioning regimen may be associated with better survival, it is also associated with a significantly increased risk of hemorrhagic cystitis. The addition of fludarabine to the low dose CY (20 mg/kg) is associated with the best survival. On the other hand, radiation-containing regimens are associated with significantly lower survival.

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