Deferasirox for managing transfusional iron overload in people with sickle cell disease

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#anemia-sickle-cell #benzoates #chelation-therapy #deferasirox #deferoxamine

Deferasirox for managing transfusional iron overload in people with sickle cell disease

Meerpohl et al., 2010 | Cochrane Database Syst Rev | Systematic Review

Citation

Meerpohl Joerg J, Antes Gerd, ... Bassler Dirk. Deferasirox for managing transfusional iron overload in people with sickle cell disease. Cochrane Database Syst Rev. 2010-Aug-04(8):CD007477. doi:10.1002/14651858.CD007477.pub2

Abstract

BACKGROUND: Sickle cell disease (SCD) is a group of genetic haemoglobin disorders. Increasingly, some people with SCD develop secondary iron overload due to occasional red blood cell transfusions or are on long-term transfusion programmes for e.g. secondary stroke prevention. Iron chelation therapy can prevent long-term complications.Deferoxamine and deferiprone have been found to be efficacious. However, questions exist about the effectiveness and safety of the new oral chelator deferasirox. OBJECTIVES: To assess the effectiveness and safety of oral deferasirox in people with SCD and secondary iron overload. SEARCH STRATEGY: We searched the Cystic Fibrosis & Genetic Disorders Group's Haemoglobinopathies Trials Register (06 April 2010).We searched MEDLINE, EMBASE, EBMR, Biosis Previews, Web of Science, Derwent Drug File, XTOXLINE and three trial registries: www.controlled-trials.com; www.clinicaltrials.gov; www.who.int./ictrp/en/. Most recent searches: 22 June 2009. SELECTION CRITERIA: Randomised controlled trials comparing deferasirox with no therapy or placebo or with another iron chelating treatment schedule. DATA COLLECTION AND ANALYSIS: Two authors independently assessed study quality and extracted data. We contacted the study author for additional information. MAIN RESULTS: One study (203 people) was included comparing the efficacy and safety of deferasirox and deferoxamine after 12 months. Data were not available on mortality or end-organ damage. Using a pre-specified dosing algorithm serum ferritin reduction was similar in both groups, mean difference (MD) 375.00 microg/l in favour of deferoxamine; (95% confidence interval (CI) -106.08 to 856.08). Liver iron concentration measured by superconduction quantum interference device showed no difference for the overall group of patients adjusted for transfusion category, MD -0.20 mg Fe/g dry weight (95% CI -3.15 to 2.75).Mild stable increases in creatine were observed more often in people treated with deferasirox, risk ratio 1.64 (95% CI 0.98 to 2.74). Abdominal pain and diarrhoea occurred significantly more often in people treated with deferasirox. Rare adverse events (less than 5% increase) were not reported; long-term adverse events could not be measured in the included study (follow-up 52 weeks). Patient satisfaction with, and convenience of treatment were significantly better with deferasirox. AUTHORS' CONCLUSIONS: Deferasirox appears to be as effective as deferoxamine. However, only limited evidence is available assessing the efficacy regarding patient-important outcomes. The short-term safety of deferasirox seems to be acceptable, however, follow-up was too short to exclude long-term side effects and thus treatment with deferasirox cannot be judged completely safe. Future studies should assess long-term outcomes for safety and efficacy, and also evaluate rarer adverse effects.

Key Findings

One study (203 people) was included comparing the efficacy and safety of deferasirox and deferoxamine after 12 months. Data were not available on mortality or end-organ damage. Using a pre-specified dosing algorithm serum ferritin reduction was similar in both groups, mean difference (MD) 375.00 microg/l in favour of deferoxamine; (95% confidence interval (CI) -106.08 to 856.08). Liver iron concentration measured by superconduction quantum interference device showed no difference for the overall

Outcomes Measured

  • Requires manual extraction

Population

Field Value
Population See abstract
Sample Size 203
Age Range See abstract
Condition See abstract

MeSH Terms

  • Anemia, Sickle Cell
  • Benzoates
  • Chelation Therapy
  • Deferasirox
  • Deferoxamine
  • Erythrocyte Transfusion
  • Ferritins
  • Humans
  • Iron Chelating Agents
  • Iron Overload
  • Randomized Controlled Trials as Topic
  • Triazoles

Evidence Classification

  • Level: Systematic Review
  • Publication Types: Journal Article, Systematic Review
  • Vertical: iron

Provenance

Source extracted via PubMed E-utilities API on 2026-04-09