Treatment with deferasirox, an oral iron-chelating agent, may be feasible and beneficial for patients with acute myeloid leukemia (AML), even when initiated early after allogeneic hematopoietic stem cell transplantation (alloHSCT), according to a retrospective study published in Cancer Medicine.

Researchers investigated the effect of deferasirox on long-term survival in 326 patients with AML (median age, 41 years) who underwent alloHSCT in remission. The cohort was 52.8% male (172 patients). The transplant donor was a matched sibling for 54.9% of patients, unrelated for 33.7%, and haploidentical-related for 11.3%.

Among 198 patients who did not receive deferasirox, hyperferritinemia (1000 ng/mL or greater) before and after alloHSCT was found to have a negative prognostic value for survival. This was mainly attributed to an increase in relapse.

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At 1 month after alloHSCT, 128 patients (46%) with hyperferritinemia received deferasirox. The researchers found that deferasirox resulted in a faster decline in serum ferritin level and had a manageable safety profile compared with not receiving deferasirox. Deferasirox was also associated with better overall survival (66.6% vs 50.1%; P<.001) and disease-free survival (65.4% vs 47.5%; <.001) compared with patients not receiving deferasirox, although no significant differences in nonrelapse mortality (19.1% vs 22.8%; =.285) were found.

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The authors concluded that hyperferritinemia was negatively prognostic for survival not only before but also after alloHSCT, primarily through compromised graft-versus-leukemia effects. They noted that when initiated early after alloHSCT, treatment with deferasirox may be associated with improved survival and restoration of graft-versus-leukemia effects.

The authors cautioned that these findings are limited because of their retrospective nature and imperfect balance between the 2 groups and noted that randomized, controlled studies are warranted to further investigate the potential benefits of deferasirox after alloHSCT.


1. Cho B-S, Jeon Y-W, Hahn A-R, et al. Improved survival outcomes and restoration of graft‐vs‐leukemia effect by deferasirox after allogeneic stem cell transplantation in acute myeloid leukemia [published online January 24, 2019]. Cancer Medicine. doi: 10.1002/cam4.1928