title: Efficacy and side effects of deferiprone (L1) in thalassemia patients not compliant with desferrioxamine.
abstract: We report our experience with deferiprone (L1) (DFP) in 17 thalassemic patients, followed up in one center in Lebanon, who were initially on desferrioxamine and then shifted to DFP at a dose of 50-75 mg/kg/day as the sole chelator during 1-year follow-up. All 17 patients were compliant with therapy and there was no change in physical examination over the study period. Eight patients (47.1%) were positive for hepatitis C virus (HCV) antibodies. Urinary iron excretion was 21.8 +/- 14 mg/24 h (mean +/- SD) 1 week after starting DFP and dropped 12 months later to 13 +/- 7.4 mg/24 h (p = 0.009, paired t test). The initial serum ferritin level was 3,863 +/- 2,344 microg/l which dropped to 3,179 +/- 2,075 at 12 months after starting therapy (p = 0.07). HCV-negative patients as a group exhibited a significant decrease in serum ferritin after 6 and 12 months of DFP therapy (3,942 +/- 2,739 vs. 2,341 +/- 1,179 and 2,681 +/- 1,519 microg/l; p < 0.03 and p < 0.05, respectively). The most frequent side effects were joint pain, stiffness or swelling in 6 patients (35.3%), and nausea in 7 patients (41.2%), but these were well tolerated and did not require stopping treatment.
authors.affiliation: Chronic Care Center, American University of Beirut Medical Center, Beirut, Lebanon.
authors.forename: A
authors.initials: A
authors.lastname: Taher
authors.forename: F M
authors.initials: FM
authors.lastname: Chamoun
authors.forename: S
authors.initials: S
authors.lastname: Koussa
authors.forename: M A
authors.initials: MA
authors.lastname: Saad
authors.forename: A I
authors.initials: AI
authors.lastname: Khoriaty
authors.forename: R
authors.initials: R
authors.lastname: Neeman
authors.forename: F H
authors.initials: FH
authors.lastname: Mourad
mesh_terms: D000293:Adolescent; D000328:Adult; D018771:Arthralgia / Q000139:chemically induced; D002648:Child; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D005293:Ferritins / Q000097:blood; D005500:Follow-Up Studies; D018937:Hepatitis C Antibodies / Q000097:blood; D006801:Humans; D007501:Iron / Q000652:urine; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects; D008297:Male; D009325:Nausea / Q000139:chemically induced; D011728:Pyridones / Q000008:administration & dosage* / Q000009:adverse effects; D013789:Thalassemia / Q000188:drug therapy* / Q000821:virology; D016896:Treatment Outcome; D016312:Treatment Refusal
chemical_list: D018937:Hepatitis C Antibodies; D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D007501:Iron; D003676:Deferoxamine