Factors Affecting the Outcome of Related Allogeneic Hematopoietic Cell Transplantation in Patients with Fanconi Anemia
| Autor: | Amal Al-Seraihy, Ashraf Khairy, Abdulrahman Al-Musa, Hasan Shahin, Samer Markiz, Mouhab Ayas, Hassan El-Solh, Abdullah Al-Jefri, Khawar Siddiqui, Ali Al-Ahmari |
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| Rok vydání: | 2014 |
| Předmět: |
Male
medicine.medical_specialty Transplantation Conditioning Cyclophosphamide Adolescent Hemorrhage Gastroenterology Fanconi anemia Internal medicine Cystitis medicine Humans Transplantation Homologous Child Retrospective Studies Univariate analysis Transplantation Radiation business.industry Histocompatibility Testing Siblings Bone marrow failure Stem cell transplantation Hematopoietic Stem Cell Transplantation Infant Hematology Myeloablative Agonists medicine.disease Survival Analysis Fludarabine Surgery Regimen Oropharyngeal Neoplasms Fanconi Anemia Gamma Rays Child Preschool Carcinoma Squamous Cell Female business Unrelated Donors Vidarabine Hemorrhagic cystitis medicine.drug |
| Zdroj: | Biology of Blood and Marrow Transplantation. 20(10):1599-1603 |
| ISSN: | 1083-8791 |
| DOI: | 10.1016/j.bbmt.2014.06.016 |
| Popis: | 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 because of the underlying DNA repair defect in their cells. Outcomes for recipients of matched related HCT have generally been favorable, but only a few studies have scrutinized the factors that may affect the eventual outcome of these patients. This retrospective analysis of 94 pediatric patients with FA who underwent related HCT at King Faisal Specialist Hospital & Research Center was carried out to attempt to identify factors that may affect outcome. Results showed overall survival (OS) probabilities of 92.5%, 89%, and 86% at 1, 5, and 10 years, respectively. In univariate analysis, use of higher dose cyclophosphamide (CY) (60 mg/kg) conditioning was associated with a better 10-year OS than lower dose CY (20 mg/kg) conditioning (91% versus 82%, respectively; P = .035), and use of radiation-containing regimens was associated with a significantly lower 10-year OS than nonradiation regimens (76% versus 91%, respectively; P = .005). Of the 4 regimens used in this study, the fludarabine-based regimen was associated with the highest survival (95.2%; P = .034). The use of the higher dose CY (60 mg/kg) was associated with a significantly increased incidence of hemorrhagic cystitis (HC) (20% versus 5.6% respectively; P = .049). Three patients (3%) developed squamous cell carcinoma (2 oropharyngeal and 1 genitourinary), at 9.4, 5.4, and 13.3 years after HCT; 2 of them had radiation-containing conditioning. In conclusion, our data suggest that although 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 HC. 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. |
| Databáze: | OpenAIRE |
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