Table 4.

Recent studies reporting hematopoietic stem cell transplant outcomes for patients with Fanconi anemia and pre-HSCT evolution to myeloid malignancies

ReferencePatients (n)Era of HSCTConditioningSurvival
Mitchell et al. (2014)35  MM: 21 1988-2011 Various All MM: 5-year OS 33% 
Bierings et al. (2018)13  Total: 199
MM: 54 
1991-2014 Various MDS: 4-year OS 48%
AML: 4-year OS 17% 
Giardino et al. (2020)32  MM: 74 1999-2016 Various All MM: 5-year OS 42%
5-year EFS 39% 
Bernard et al. (2021)33  Total: 82
MM: 11 
1999-2018 Most fludarabine + cyclophosphamide All MM: 5-year OS 40% 
Mehta et al. (2017)15  Total: 45
MM: 11 
2009-2014 Low-dose busulfan + cyclophosphamide, fludarabine, ATG MDS: 3-year OS 63.6% 
Chattopadhyay et al. (2023)34  Total: 60
MM: 10 
1990-2021 Low-dose busulfan + fludarabine All MM: 5-year OS 46% 
ReferencePatients (n)Era of HSCTConditioningSurvival
Mitchell et al. (2014)35  MM: 21 1988-2011 Various All MM: 5-year OS 33% 
Bierings et al. (2018)13  Total: 199
MM: 54 
1991-2014 Various MDS: 4-year OS 48%
AML: 4-year OS 17% 
Giardino et al. (2020)32  MM: 74 1999-2016 Various All MM: 5-year OS 42%
5-year EFS 39% 
Bernard et al. (2021)33  Total: 82
MM: 11 
1999-2018 Most fludarabine + cyclophosphamide All MM: 5-year OS 40% 
Mehta et al. (2017)15  Total: 45
MM: 11 
2009-2014 Low-dose busulfan + cyclophosphamide, fludarabine, ATG MDS: 3-year OS 63.6% 
Chattopadhyay et al. (2023)34  Total: 60
MM: 10 
1990-2021 Low-dose busulfan + fludarabine All MM: 5-year OS 46% 

ATG, antithymocyte globulin; EFS, event-free survival.

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