title: Long-term assessment of efficacy and safety of L1, an oral iron chelator, in transfusion dependent thalassaemia: Indian trial.
abstract: From August 1989 to May 1991, 52 patients with transfusion dependent thalassaemia major received L1 (1,2-dimethyl-3- hydroxypyrid-4-one), the oral iron chelator, for a period of 3-21 months (mean +/- SD: 14.2 +/- 6.8). Mean (+/- SD) urinary iron excretion varied from 6.2 +/- 4.6 mg/d on 25 mg/kg/d of L1 to 42.3 +/- 37.1 mg/d on 100 mg/kg/d of L1. Mean (+/- SD) drop in S ferritin was 1465 +/- 990 micrograms/l after 5.0 +/- 0.8 months to 3641.2 +/- 2299.3 micrograms/l after 20.1 +/- 0.9 months of therapy. There was no evidence of neutropenia, thrombocytopenia, ear or eye toxicity. L1-related arthralgia, which was reversible on dose reduction or stoppage, was seen in 20 patients (38.5%), while minor gastrointestinal (GI) tract symptoms occurred in seven (3.5%) cases. We conclude that although L1 is an effective iron chelator, further studies are required to understand the mechanism of L1 related arthralgia and also to find a safer but effective dose on which incidence of L1 related arthralgia is minimal.
authors.affiliation: Department of Haematology, L.T.M.G. Hospital, Bombay, India.
authors.forename: M B
authors.initials: MB
authors.lastname: Agarwal
authors.forename: S S
authors.initials: SS
authors.lastname: Gupte
authors.forename: C
authors.initials: C
authors.lastname: Viswanathan
authors.forename: D
authors.initials: D
authors.lastname: Vasandani
authors.forename: J
authors.initials: J
authors.lastname: Ramanathan
authors.forename: N
authors.initials: N
authors.lastname: Desai
authors.forename: R R
authors.initials: RR
authors.lastname: Puniyani
authors.forename: A T
authors.initials: AT
authors.lastname: Chhablani
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use; D006801:Humans; D007501:Iron / Q000652:urine; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D007596:Joints; D010146:Pain / Q000139:chemically induced; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D013997:Time Factors; D017086:beta-Thalassemia / Q000150:complications / Q000188:drug therapy* / Q000652:urine
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D007501:Iron; D003676:Deferoxamine