In young patients with transfusion-dependent beta-thalassemia (TDT), the use of early-start deferiprone appeared to be well tolerated and was associated with efficacy in reducing iron overload, according to the results of a recent study. Study findings were reported in the American Journal of Hematology.
The study was the START study (ClinicalTrials.gov Identifier: NCT03591575), which enrolled children below 10 years of age with TDT. Patients were randomly assigned 1:1 to receive either deferiprone oral solution or placebo for 12 months. In patients receiving deferiprone, the starting dose was 25 mg/kg/day, and this was increased to 50 mg/kg/day or to 75 mg/kg/day based on serum ferritin status.
The primary endpoint of the study was the proportion of patients in the intention-to-treat population with an iron load below the threshold for starting chelation therapy, which in this study was characterized by a serum ferritin level of ≥1000 mg/L at 2 consecutive visits by month 12. Secondary endpoints included the time to reach the serum ferritin iron overload threshold, the proportion of patients below this threshold each month, and the mean serum ferritin or transferrin saturation (TSAT) level each month.
There were 32 children assigned to receive deferiprone and 32 assigned to receive placebo. Patients had median ages of 2.0 years (range, 0.61-9.00) in the deferiprone group and 3.0 years (range, 0.76-8.00) in the placebo group. The mean rates of treatment adherence were 95% (SD, 14%) with deferiprone and 93% (SD, 10%) with placebo.
In primary endpoint analysis, by month 12, 66% of patients in the deferiprone group remained below the serum ferritin threshold, compared with 38% in the placebo group (P =.045). Patients in the placebo group also tended to reach the serum threshold of ≥1000 mg/L in less time than the deferiprone group did (P =.041). Neither group showed evidence of iron depletion by month 12.
Mean % TSAT levels were significantly higher in patients of the deferiprone group than in the placebo group at months 3, 4, 6, 9, and 11 (P <.05). Patients of the deferiprone group also reached a level of ≥60% TSAT faster than the placebo group did. Significantly more patients of the deferiprone group also reached the ≥60% TSAT level than those in the placebo group did in months 3 through 6 (P <.05).
There were adverse events reported in 91% of patients in either treatment group. Serious adverse reactions were reported in 5 patients who received deferiprone and in 2 patients who received placebo.
“Early-start deferiprone was efficacious at maintaining low iron levels; it was generally well-tolerated with a safety profile comparable to deferiprone treatment in older patients,” the study investigators wrote in their report.
Disclosures: Some study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of disclosures.
Elalfy MS, Hamdy M, Adly A, et al. Efficacy and safety of early-start deferiprone in infants and young children with transfusion-dependent beta thalassemia: evidence for iron shuttling to transferrin in a randomized, double-blind, placebo-controlled, clinical trial (START). Am J Hematol. Published online July 4, 2023. doi:10.1002/ajh.27010