FLUDARABINE-BASED LOW-INTENSITY CONDITIONING FOR FANCONI ANEMIA IS ASSOCIATED WITH GOOD OUTCOMES IN APLASTIC ANEMIA BUT NOT IN MDS - A SINGLE-CENTER EXPERIENCE

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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

Keywords

Fanconi Anemia, Fludarabine, low-dose Cyclophosphamide, Low-dose Busulfan, Graft Versus Host Disease, Secondary Malignancies

Abstract

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.

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