title: Hematologic responses in patients with aplastic anemia treated with deferasirox: a post hoc analysis from the EPIC study.
abstract: Reports are emerging of hematologic responses associated with iron chelation therapy; however, studies are limited in aplastic anemia patients. Deferasirox reduced iron overload in aplastic anemia patients enrolled in the EPIC (Evaluation of Patients' Iron Chelation with Exjade(®)) study (n=116). A post hoc analysis of hematologic responses was conducted on 72 patients with evaluable hematologic parameters (according to UK guideline criteria), 24 of whom received deferasirox without concomitant immunosuppressive treatment. Partial hematologic responses were observed in 11 of 24 (45.8%) patients; all became transfusion-independent. One patient had an additional platelet response and one patient had an additional platelet and hemoglobin response. Mean serum ferritin levels at end of study were significantly reduced in partial hematologic responders (n=11; -3948 ± 4998 ng/mL; baseline 6693 ± 7014 ng/mL; percentage change from baseline -45.7%; P=0.0029). In non-responders, the reduction in serum ferritin was less pronounced (n=13; -2021 ± 3242 ng/mL; baseline 4365 ± 3063 ng/mL; % change from baseline -27.6%; P=0.0171). Alongside reduction in iron overload, deferasirox may, therefore, improve hematologic parameters in a subset of aplastic anemia patients. Further investigation is required to elucidate the mechanisms involved.
authors.affiliation: The Catholic University of Korea, Seoul, South Korea. jwlee@catholic.ac.kr
authors.forename: Jong Wook
authors.initials: JW
authors.lastname: Lee
authors.forename: Sung-Soo
authors.initials: SS
authors.lastname: Yoon
authors.forename: Zhi Xiang
authors.initials: ZX
authors.lastname: Shen
authors.forename: Arnold
authors.initials: A
authors.lastname: Ganser
authors.forename: Hui-Chi
authors.initials: HC
authors.lastname: Hsu
authors.forename: Ali
authors.initials: A
authors.lastname: El-Ali
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Nicolas
authors.initials: N
authors.lastname: Martin
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000097:blood* / Q000188:drug therapy* / Q000473:pathology; D001565:Benzoates / Q000627:therapeutic use*; D001792:Blood Platelets / Q000378:metabolism / Q000473:pathology; D002648:Child; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000037:antagonists & inhibitors / Q000097:blood; D006454:Hemoglobins / Q000378:metabolism; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009504:Neutrophils / Q000378:metabolism / Q000473:pathology; D011446:Prospective Studies; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D001565:Benzoates; D006454:Hemoglobins; D007166:Immunosuppressive Agents; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D000077588:Deferasirox