title: Long-term response to deferiprone therapy in Asian Indians.
abstract: Deferiprone (L1) has been used in several countries for iron chelation therapy for over one decade. Long-term results on the drug are lacking. In the present study, data of 110 patients on deferiprone (L1) for up to 17 years were analyzed. On a mean L1 dose of 70.2 mg/kg/day (range 44-100), serum ferritin level showed a very steady decrease with time from an initial mean (+/-SD) of 3,033.61 +/- 1,468.04 ng/ml to final of 1,665.08 +/- 949.93 ng/ml after a mean (+/-SD) of 6.1 +/- 3.8 years. In total, 13 patients discontinued L1 therapy. Major complications of L1 requiring permanent discontinuation of treatment included arthropathy (n = 8, 7.2%) and neutropenia/agranulocytosis (n = 5, 4.5%). Lesser complications permitting continued L1 treatment included transient mild leucopenia or thrombocytopenia (n = 3) and gastrointestinal problems (n = 5). There were a total of three deaths attributed to agranulocytosis. Although the complications associated with L1 treatment are significant and require close monitoring, they do not preclude effective long-term therapy in the vast majority of patients. A longer duration of therapy is required for effective response in chronically iron-overloaded patients. Further well-controlled prospective studies of L1 are required to identify factors affecting individual response to therapy.
authors: Hemato-Oncology & Genetics Units, Department of Pediatrics, Advanced Pediatrics Centre (APC), Post-Graduate Institute of Medical Education & Research (PGIMER), Chandigarh, India. inupan@yahoo.com
authors: Inusha
authors: I
authors: Panigrahi
authors: Ram K
authors: RK
authors: Marwaha
authors: Rashmi R
authors: RR
authors: Das
mesh_terms: D000293:Adolescent; D000328:Adult; D000380:Agranulocytosis / Q000139:chemically induced; D044466:Asian People; D015913:Chelation Therapy / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D000077543:Deferiprone; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007194:India; D007502:Iron Chelating Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy*; D008297:Male; D009503:Neutropenia / Q000139:chemically induced; D011728:Pyridones / Q000009:adverse effects* / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins