title: Use of deferiprone for iron chelation in patients with transfusion-dependent thalassaemia.
abstract: OBJECTIVE
  To conduct a retrospective case analysis of the clinical efficacy and adverse effects of deferiprone in our population.
  
  
  METHODS
  All patients with transfusion-dependent thalassaemia at KK Hospital who have been on deferiprone were included in the study. Outcomes measured include the change in ferritin levels and cardiac T2* values during deferiprone therapy, and incidence of side effects.
  
  
  RESULTS
  Thirty-three (47.1%) of the total cohort of 70 patients have been on deferiprone, out of which 26 were on combination therapy with desferrioxamine. Majority of the patients (76%) had stable cardiac iron load during deferiprone therapy, and four patients with moderate to severe cardiac iron load showed improvement. Ten patients (30.3%) had improvement in their ferritin levels. Three patients (9.1%) developed mild neutropenia at 3, 18 and 26 months, respectively, and two patients (6.1%) had agranulocytosis at 4 and 10 months, respectively. Their neutrophil counts improved spontaneously after cessation of deferiprone. Thrombocytopenia developed in 27.3% of the patients and was transient in majority (77.8%) of the patients. Five patients (15.2%) developed arthritis that improved after cessation of deferiprone therapy, and one patient had transient arthralgia that resolved spontaneously. Three patients (9.1%) had nausea and abdominal pain.
  
  
  CONCLUSIONS
  Deferiprone effectively reduced or stabilised cardiac iron load in our patients. Thrombocytopenia, arthropathy, neutropenia and agranulocytosis are the most important side effects. It is recommended that patients on deferiprone have their full blood counts monitored weekly for the first year of therapy and subsequently fortnightly as long as they are on deferiprone.
authors: Genetics Service, Department of Paediatric Medicine, KK Women's and Children's Hospital, Singapore. saumya.s.jamuar@kkh.com.sg
authors: Saumya Shekhar
authors: SS
authors: Jamuar
authors: Angeline Hwei Meeng
authors: AH
authors: Lai
authors: Ah Moy
authors: AM
authors: Tan
authors: Mei Yoke
authors: MY
authors: Chan
authors: Ee Shien
authors: ES
authors: Tan
authors: Ivy Swee Lian
authors: IS
authors: Ng
mesh_terms: D000293:Adolescent; D000328:Adult; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D000077543:Deferiprone; D005260:Female; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects* / Q000627:therapeutic use; D019190:Iron Overload / Q000188:drug therapy; D017063:Outcome Assessment, Health Care; D011728:Pyridones / Q000009:adverse effects* / Q000627:therapeutic use; D012189:Retrospective Studies; D012846:Singapore; D013789:Thalassemia / Q000188:drug therapy* / Q000503:physiopathology; D055815:Young Adult
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone