Efficacy and safety of oral deferasirox treatment in the posttransplant period for patients who have undergone allogeneic hematopoietic stem cell transplantation (alloHSCT)

Ann Hematol. 2012 May;91(5):743-749. doi: 10.1007/s00277-011-1358-1. Epub 2011 Nov 4.


Iron overload is considered to be associated with various complications in patients who undergo both allogeneic (allo) and autologous hematopoietic stem cell transplantation (HSCT). A total of 23 alloHSCT recipients who started deferasirox treatment due to hyperferritinemia (ferritin ≥1,000 ng/mL) were analyzed retrospectively. The demographic characteristics, data about deferasirox treatment, and history of phlebotomy were obtained from the patients' files. The reduction in posttreatment ferritin levels was found statistically significant compared with pretreatment ferritin levels in both def+phlebotomy and def+nonphlebotomy groups (p = 0.025 and 0.017, respectively). The liver enzymes, especially ALT and bilirubins, were significantly reduced after the treatment (p < 0.05). The deferasirox treatment reduced pretreatment ferritin levels below the level of 1,000 ng/mL in a median period of 94 days, and these data were found to be statistically significant (p < 0.05). The median treatment duration time with deferasirox was 94 days (72-122). The most common adverse effects were nausea and vomiting, which occurred in three of the patients (13%). In conclusion, our data suggest that oral deferasirox treatment may be used as a safe and effective alternative method for reducing iron overload in alloHSCT recipients, whether combined with or without phlebotomy.

MeSH terms

  • Adolescent
  • Adult
  • Benzoates / adverse effects
  • Benzoates / therapeutic use*
  • Deferasirox
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Iron Chelating Agents / adverse effects
  • Iron Chelating Agents / therapeutic use*
  • Iron Overload / drug therapy*
  • Iron Overload / etiology
  • Iron Overload / mortality
  • Kidney / physiopathology
  • Leukemia / complications
  • Leukemia / therapy
  • Leukemia, Myeloid, Acute / complications
  • Leukemia, Myeloid, Acute / therapy
  • Liver / pathology
  • Liver / physiopathology
  • Male
  • Middle Aged
  • Phlebotomy
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / complications
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy
  • Transplantation, Homologous
  • Triazoles / adverse effects
  • Triazoles / therapeutic use*
  • Young Adult


  • Benzoates
  • Iron Chelating Agents
  • Triazoles
  • Deferasirox