SAFETY AND EFFICACY OF LOW-DOSE THALIDOMIDE IN PATIENTS WITH TRANSFUSION DEPENDENT THALASSEMIA: A CLINICO-HEMATOLOGICAL ASSESSMENT

Main Article Content

Muhammad Idrees
Muhammad Tariq Masood Khan
Waleed Bawazir
Mohannad S. Hazzazi
Saeed M. Kabrah
Malik A. Altayar
Soad Al- Jaouni
Majed N. Almashjary
Steve Harakeh
Yasar M Yousafzai

Keywords

β-Thalassemia, prevalence, thalidomide, efficacy, safety

Abstract

Background: β-thalassemia is an autosomal recessive hereditary hemoglobin production disorder characterized by abnormal hemoglobin synthesis. The efficient and safe therapies for transfusion-dependent β-thalassemia (TDT) patients are desperately required to minimize the requirement for blood transfusion.


Objective: To establish the safety and efficacy of low-dose thalidomide in TDT patients.


Methodology: The retrospective cohort study was conducted in Khyber Medical University, Peshawar and Blood Diseases Clinic, Peshawar to investigate the safety and efficacy of low-dose thalidomide therapy in TDT patients. Samples were collected from the diagnosed cases of TDT patients whose ages were ≥3 years and were on thalidomide treatment for a period of ≥6 months. Complete blood count CBC was performed on Sysmex XP-100, Japan and biochemical tests were performed on Cobas 6000 analyzer series. Comprehensive patients’ demographics, clinical history and prognosis data were recorded and analyzed using the SPPS 27.


Results: The patient characteristics, biochemical parameters and hematological response were assessed and statistically tested for 384 TDT patients using low-dose thalidomide therapy. Applying the criteria for the Hb level achievement, we marked the significant results (<0.001) for Excellent, Good, and Partial responders that were 184 (47.9%), 96 (25%), and 60 (15.6%), respectively. The common side effects observed with the drug included abdominal discomfort, nausea, vomiting, headache, constipation, dizziness, fatigue, anxiety, repeated infections, and skin rash, that were controlled with symptomatic treatment and/or dose adjustment.


Conclusion: Collectively, thalidomide is safe and effective in increasing Hb levels thus reducing the requirements of blood transfusion in TDT patients. Future clinical trials are suggested to strengthen the efficacy and safety of low-dose thalidomide and to establish clinical guidelines for its rational use in TDT patients.


 

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