One‐year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation

Abstract

Deferasirox treatment or phlebotomy reduces iron burden in pediatric patients with β- thalassemia major post-HSCT, with a manageable safety profile.

Efficacy and safety of deferasirox in non-thalassemic patients with elevated ferritin levels after allogeneic hematopoietic stem cell transplantation

“…[9][10][11] Besides iron chelation, phlebotomy is another safe, convenient and cost-effective option. 10,[14][15][16][17] Yet, data on phlebotomy are hitherto limited. One restriction is that phlebotomy could not be initiated early after HSCT in every patient, which might hamper its value as an instrument to improve outcome assuming that the impact of elevated ferritin on outcome might be clearest early on after HSCT (although this needs to be proved).…”

“…[22][23][24][25][26][27][28] However, data regarding the use of deferasirox after allogeneic HSCT are limited. 15,17,19,20 This prospective multicenter trial is, to the best of our knowledge, the largest trial evaluating the efficacy and safety of deferasirox after allogeneic HSCT. The impact on cyclosporine absorption as well as surrogate markers of iron stores and hepcidin dynamics were analyzed.…”

“…13 Data suggest that post-HSCT SF is comparable to pre-HSCT values, 12 and hyperferritinemia up to day +720 post HSCT could significantly be associated with decreased survival. 8 Hence, lowering ferritin after HSCT through phlebotomy 10,[14][15][16][17] and/or iron chelation 15,[17][18][19][20][21] seems rational, although the impact on survival is not yet known and needs to be studied in controlled trials.…”

Iron Overload in Hematopoietic Stem Cell Transplantation

“…However, in clinical practice, the problem is not common and improvement of the patients after transplantation is reported. According to the recent report by Inati et al [2], with standard chelation therapy, the outcome of thalassemic patients undergoing stem cell transplantation is usually favorable. The use of the standard dosage of deferoxamine, with or without phlebotomy, accompanied with close iron status monitoring can be effective [2,3].…”

“…According to the recent report by Inati et al [2], with standard chelation therapy, the outcome of thalassemic patients undergoing stem cell transplantation is usually favorable. The use of the standard dosage of deferoxamine, with or without phlebotomy, accompanied with close iron status monitoring can be effective [2,3]. It can be seen that stem cell transplantation can be problematic despite there being a need of hypertransfusion during the process even though the patient might have an underlying severe iron overload condition such as thalassemia.…”