Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia
Summary
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations. In Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure. The standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.
Timeline
- Start
- 2024-01-01
- Primary completion
- 2025-12-30
- Completion
- 2025-12-30
Publications
- Results Li X, Hu S, Liu Y, Huang J, Hong W, Xu L, Xu H, Fang J. Efficacy of Thalidomide Treatment in Children With Transfusion Dependent beta-Thalassemia: A Retrospective Clinical Study. Front Pharmacol. 2021 Aug 12;12:722502. doi: 10.3389/fphar.2021.722502. eCollection 2021.
- Results Garg A, Patel K, Shah K, Trivedi D, Raj A, Yadav R, Shah S. Safety and Efficacy of Thalidomide and Hydroxyurea Combination in Beta Thalassemia Patients. Indian J Hematol Blood Transfus. 2023 Jan;39(1):85-89. doi: 10.1007/s12288-022-01536-y. Epub 2022 Apr 21.
- Results Chen J, Zhu W, Cai N, Bu S, Li J, Huang L. Thalidomide induces haematologic responses in patients with beta-thalassaemia. Eur J Haematol. 2017 Nov;99(5):437-441. doi: 10.1111/ejh.12955. Epub 2017 Sep 27.
- Results Nag A, Radhakrishnan VS, Kumar J, Bhave S, Mishra DK, Nair R, Chandy M. Thalidomide in Patients with Transfusion-Dependent E-Beta Thalassemia Refractory to Hydroxyurea: A Single-Center Experience. Indian J Hematol Blood Transfus. 2020 Apr;36(2):399-402. doi: 10.1007/s12288-020-01263-2. Epub 2020 Mar 2.
- Results Ansari SH, Ansari I, Wasim M, Sattar A, Khawaja S, Zohaib M, Hussain Z, Adil SO, Ansari AH, Ansari UH, Farooq F, Masqati NU. Evaluation of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia. Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Hydroxyurea | Other / unclassified | 20 mg/kg | — |
| Subject | Thalidomide | Other / unclassified | 2.5 mg/kg | — |
| Subject | Thalidomide | Other / unclassified | 3 mg/kg | — |
| Subject | Thalidomide | Other / unclassified | 5 mg/kg | — |
| Background | Aspirin | Small molecule | 2 mg/kg | — |
| Background | Aspirin | Small molecule | 4 mg/kg | — |
| Background | Deferasirox | Unknown | — | — |
| Background | Deferiprone | Small molecule | — | — |
| Background | Deferoxamine | Other / unclassified | — | — |
| Background | folic acid | Other / unclassified | 2 mg | — |
| Background | folic acid | Other / unclassified | 5 mg | — |