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Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia
Jagdish Chandra1, Nupur Parakh2, Sidharth1
1Division of Pediatric Hematology, Department of Pediatrics, Lady Hardinge Medical College and associated Kalawati Saran Children Hospital, New Delhi.
Insights
Thalidomide significantly reduced transfusion needs and improved hemoglobin levels in children with transfusion-dependent thalassemia. The treatment showed a satisfactory safety profile with manageable side effects like constipation and neutropenia.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Clinical Trials
Background:
- Transfusion-dependent thalassemia requires lifelong blood transfusions, leading to iron overload and complications.
- Current treatment options for thalassemia have limitations, necessitating exploration of novel therapeutic agents.
Purpose of the Study:
- To evaluate the efficacy and safety of thalidomide in pediatric patients with transfusion-dependent thalassemia.
- To assess the impact of thalidomide on transfusion requirements, hemoglobin levels, and adverse events.
Main Methods:
- A prospective, single-center, open-label study involving 37 children aged 12-18 years.
- Thalidomide was administered for 6 months at a starting dose of 2-3 mg/kg/day.
- Efficacy was measured by changes in transfusion needs and hemoglobin fall rate; safety was monitored through adverse event recording.
Main Results:
- A significant reduction in the rate of hemoglobin fall was observed (1.0 g/week to 0.58 g/week, P<0.001).
- Major or moderate responses were achieved in 83.7% of children, with 40.5% remaining transfusion-free for a median of 6 weeks.
- Mean serum ferritin decreased (P<0.001), and mean HbF increased significantly (P<0.001).
- Common adverse events included constipation and neutropenia, mostly mild.
Conclusions:
- Thalidomide demonstrated significant efficacy in improving transfusion requirements and hematological parameters in children with transfusion-dependent thalassemia.
- The drug exhibited a satisfactory safety profile, with manageable adverse effects, suggesting its potential as a therapeutic option.
Objective:
To assess the efficacy and safety of thalidomide in children with transfusion-dependent thalassemia.
Methods:
This prospective, single center, open-label study enrolled children aged 12-18 years, and who received thalidomide for a duration of 6 months at a starting dose of 2-3 mg/kg/day. Efficacy was assessed by reduction in transfusion requirement and rate of fall of hemoglobin. Efficacy was classified as major, moderate and minimal/no response depending on the reduction in transfusion requirement. Safety was assessed by adverse effects related to thalidomide.
Results:
37 children [mean (SD) age, 14.7 (1.8) years were included. Rate of fall of hemoglobin reduced from a mean of 1.0 (0.24) g/week pre-thalidomide therapy to 0.58 (0.26) g/week after 6 months of thalidomide (P<0.001). 19 children (51.3%) had major response and 12 (32.4%) had moderate response. In 13.5% and 32.4% children response was observed within the first and second month of therapy, respectively. 15 (40.5%) children remained transfusion - free for a median (IQR) time of 6 (3-10) weeks of thalidomide therapy. Mean serum ferritin (SD) decreased from 1758.9 (835.1) to 1549.6(1016.9) (P<0.001). Mean HbF (SD) showed an increase from 2.95(2.6) to 49.2(33.3) (P<0.001). In 32 children, 47 adverse events were observed. Common adverse events were constipation and neutropenia (mostly mild).
Conclusions:
Thalidomide resulted in major/moderate response in majority of children with transfusion-dependent thalassemia with satisfactory adverse effect profile.
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