Dual Oral Iron Chelation (DOIC) in Children with Transfusion-Dependent Beta Thalassemia: Real-World Efficacy Data

Amitoj Singh Chadha1, Abaan Ul Haq Khazi Mohammad1, Naveen Kanth Dhãmi Nadakuditi1

  • 1Department of Pediatric Hematology Oncology, St. Johns Medical College Hospital, Bangalore, India.

Insights

Dual oral iron chelation using deferiprone and deferasirox effectively reduces iron overload in children with transfusion-dependent thalassemia. This combination therapy is well-tolerated and significantly lowers ferritin levels, especially in patients with higher initial values.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Iron overload is a common complication in children with Transfusion-dependent Thalassemia (TDT).
  • Chelation therapy is essential for managing iron overload in TDT patients.
  • Combination chelation with deferiprone (DFP) and deferasirox (DFX) offers a treatment option.

Purpose of the Study:

  • To evaluate the efficacy and safety of dual oral iron chelation (DOIC) in children with TDT.
  • To assess the impact of DOIC on serum ferritin levels over time.
  • To identify factors influencing ferritin reduction with DOIC.

Main Methods:

  • Retrospective chart review of TDT children initiated on DOIC between 2015 and 2022.
  • Analysis of serum ferritin levels before and after DOIC initiation.
  • Assessment of treatment duration and adverse effects.

Main Results:

  • Forty-five children with TDT received DOIC.
  • Mean serum ferritin decreased from 2929 ng/dl to 1962.10 ng/dl at 30 months.
  • 82.22% of children achieved ferritin < 2000 ng/dl, and 60% achieved < 1000 ng/dl.
  • Higher initial ferritin levels (> 2500 ng/dl) showed rapid reduction within 12-24 months.
  • DOIC was well-tolerated with no severe adverse effects.

Conclusions:

  • Dual oral iron chelation (DOIC) is an effective and safe treatment for reducing iron overload in children with TDT.
  • DOIC leads to significant reductions in serum ferritin levels.
  • The therapy is particularly effective in patients with high baseline ferritin levels and is well-tolerated.

Related Concept Videos

Extraction: Advanced Methods00:56

Extraction: Advanced Methods

Metal ions can be separated from one another by complexation with organic ligands–the chelating agent– to form uncharged chelates. Here, the chelating agent must contain hydrophobic groups and behave as a weak acid, losing a proton to bind with the metal. Since most organic ligands used in this process are insoluble or undergo oxidation in the aqueous phase, the chelating agent is initially added to the organic phase and extracted into the aqueous phase. The metal-ligand complex is...
403
Bone Marrow Sampling and Transplants01:22

Bone Marrow Sampling and Transplants

Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
276
Masking and Demasking Agents01:19

Masking and Demasking Agents

EDTA titrations may necessitate masking and demasking agents to temporarily protect a particular metal ion in a mixture from the EDTA reaction. These agents facilitate the sequential analysis of the metal ions by forming stable complexes with some—but not all—metal ions during certain steps.
There are many masking agents, such as cyanide, fluoride, triethanolamine, thiourea, and 2,3-bis(sulfanyl)propan-1-ol (formerly 2,3-dimercapto-1-propanol), with the masking agent chosen based on...
2.3K