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. 2023 Dec 8;2023(1):556-562. doi: 10.1182/hematology.2023000489

Table 4.

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

Reference Patients (n) Era of HSCT Conditioning Survival
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.