Thalidomide and Hydroxyurea in Transfusion-Dependent Thalassemia: Efficacy, Safety Profile and Impact on Quality of Life

Abstract

Transfusion-dependent thalassemia (TDT) is a major public health concern in India, requiring regular transfusions for survival. There is also significant morbidity caused by iron overload and transfusion related infections. Novel therapies targeting fetal hemoglobin induction are the need of the hour in resource-poor institutions for patients where transplant is not feasible for various reasons. This single arm, non-randomised prospective trial evaluated the efficacy and safety of a combination of low dose thalidomide and hydroxyurea in TDT along with the impact on quality of life (QoL). It included 41 TDT patients, who failed a reasonable trial of hydroxyurea. Complete response (CR) was defined as transfusion independence and partial response (PR) denoted at least a 50% reduction in transfusion requirement. The rest were defined as non-responders (NR). The mean age of the cohort was 20.78 years (range 12–45 years). There were 13 males and 28 females. Nineteen (46.3%), 7 (17.1%), and 15 (36.6%) patients achieved CR, PR, and no response respectively. The overall response rate (CR + PR) was 63.4%. There was a significant increase in hemoglobin levels with decrement in transfusion burden and ferritin levels. There were no significant adverse reactions. No significant predictors of response were found including amongst genetic modifiers. It improved the health related QoL amongst responders. The combination of thalidomide and hydroxyurea appear safe and effective in the reduction in transfusion requirement of TDT patients. The judicious use of these drugs can improve the quality of life and pave the way for patients not eligible for a stem cell transplant.

Keywords:

• Thalassemia
• transfusion dependent
• thalidomide
• hydroxyurea
• transplant ineligible

Acknowledgements

State Thalassemia Unit, IHTM, Kolkata

Ethics approval

Approved by the Institutional Ethical Committee.

Consent form

Informed consent taken from patients.

Authors contributions

Sukrita Bhattacharjee: Concept, clinical data curation, manuscript drafting, literature review, statistics

Shouriyo Ghosh: Manuscript drafting, statistics, manuscript editing

Maitreyee Bhattacharyya: concept, manuscript editing, overall supervision

Jyoti Shaw, Sunistha Bhattacharjee: Genetic and mutation data testing and evaluation

Disclosure statement

The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.

Keynote

The combination of thalidomide and hydroxyurea decreases transfusion requirement and increases hemoglobin in TDT patients

Data availability statement

Available for publication/review.

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.