Deferiprone or deferoxamine vs. combination therapy in patients with beta-thalassemia major: a case study in Taiwan.
Deferiprone (L1) has been recommended as an effective oral chelation therapy for patients with beta-thalassemia major (TM). From 1999 to 2004, 114 patients with TM from five treatment centers were enrolled in this program: iron (Fe) was chelated with L1 in 57 patients, deferoxamine (DFO) in 26, and combined L1/DFO therapy in 31. We found that serum ferritin (SF) was significantly lower in nine patients receiving L1 for more than 5 years (p = 0.04), 22 patients receiving L1 for 1-2 years (p < 0.01) and 31 receiving the combined therapy (p = 0.01), yet significantly higher in those receiving DFO only (p < 0.01). One patient showed transient neutropenia; arthropathy in one patient and gastrointestinal upset in two were noted, with no significant change in alanine aminotransferase (ALT) level. Of 17 patients who were submitted to a liver biopsy, 15 showed no significant change in hepatic fibrosis scores after therapy with L1. None of the 88 patients, including 31 who received the combined therapy, have abandoned oral L1 treatment due to adverse effects. Results of this study proved that L1 or combined therapy with L1 and DFO is effective in reducing SP; incidence of adverse events was low in patients with TM.
Department of Pediatrics, China Medical University Hospital, Taichung, Taiwan.
Ching-Tien
CT
Peng
Kang-Hsi
KH
Wu
Shu-Fen
SF
Wu
Der-Cherng
DC
Liang
Chao-Ping
CP
Yang
Ren-Chin
RC
Jang
Lin-Yen
LY
Wang
Chih-Cheng
CC
Hsiao
D000293:Adolescent; D000077543:Deferiprone; D003676:Deferoxamine / Q000009:adverse effects / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D008297:Male; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D013624:Taiwan / Q000453:epidemiology; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000175:diagnosis / Q000188:drug therapy*
D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine