| Autor: |
Biju George, Sohini Chattopadhyay, Sharon Lionel, Sushil Selvarajan, Anup Devasia, Anu Korula, Uday Kulkarni, Fouzia Aboobacker, Eunice Sindhuvi, Kavitha Lakshmi, Alok Srivastava, Aby Abraham, Vikram Mathews |
| Jazyk: |
angličtina |
| Rok vydání: |
2023 |
| Předmět: |
|
| Zdroj: |
Mediterranean Journal of Hematology and Infectious Diseases, Vol 15, Iss 1 (2023) |
| Druh dokumentu: |
article |
| ISSN: |
2035-3006 |
| DOI: |
10.4084/MJHID.2023.039 |
| Popis: |
Background: Hematopoietic stem cell transplantation [HSCT] is the only curative option for Fanconi Anemia (FA) patients with hematological abnormalities. Materials and Methods: This is a retrospective analysis of patients with FA who underwent a matched-related donor HSCT. Results: Sixty patients underwent 65 transplants between 1999-2021 using a fludarabine-based low-intensity conditioning regimen. The median age at transplant was 11 years (range: 3-37). Aplastic anemia (AA) was the underlying diagnosis in 55 (84.6%) while 8 (12.4%) had myelodysplastic syndrome (MDS) and 2 (3%) had acute myeloid leukemia (AML). The conditioning regimen used was fludarabine with low-dose cyclophosphamide for aplastic anemia and Fludarabine with low-dose busulfan for MDS/AML. Graft versus host disease (GVHD)prophylaxis consisted of cyclosporine and methotrexate. Peripheral blood stem cells was the predominant graft source (86.2%). Engraftment occurred in all but 1 patient. The median time to neutrophil and platelet engraftment was 13 days (range: 9-29) & 13 days (range: 5-31), respectively. Day 28 chimerism analysis showed complete chimerism in 75.4 % and mixed chimerism in 18.5%. Secondary graft failure was encountered in 7.7%. Grade II–IV acute GVHD occurred in 29.2% while Grade III-IV acute GVHD occurred in 9.2%. Chronic GVHD was seen in 58.5% and was limited in most patients. The median follow-up is 55 months [range: 2-144] & the 5-year estimated overall survival (OS) is 80.2 ± 5.1%. Secondary malignancies were noted in 4 patients. The 5-year OS was significantly higher in patients undergoing HSCT for AA (86.6 + 4.7%) as compared to MDS/AML (45.7+16.6%) (p= 0.001). Conclusion: SCT using a fully matched donor provides good outcomes with low-intensity conditioning regimens in patients with FA who have an aplastic marrow. |
| Databáze: |
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| Externí odkaz: |
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