Incidence of Peripheral Neuropathy and Cardiovascular Adverse Effects in β-Thalassemia Patients Following Off-Label Thalidomide Therapy

Author(s): Muhammad Azaz Ali Khan, Tanzeel Imran, Asad Ullah Amjad, Ume Amin, Uroosa Hasan Khan, Muhammad Usama Tahir, Areesha Fatima

Background: Thalidomide, used off-label as an HbF inducer, is associated with significant neurological and cardiovascular toxicity. This study examines the incidence of peripheral neuropathy and cardiovascular adverse effects among β-thalassemia patients using externally sourced thalidomide.

Methods: A retrospective review was conducted at JSF (jan 2024 to oct 2025) including patients who reported independent thalidomide use. Clinical data were analyzed to identify neurological and cardiovascular complications.

Results: Peripheral neuropathy and generalized body aches were the most prevalent neurological complications, while arrhythmias represented the most significant cardiovascular toxicity.

Conclusion: Off-label thalidomide poses substantial risks when used without clinical supervision. Improved regulation and patient education are required to prevent avoidable morbidity. However it has beneficial effect in thalassemic patient having allo antibodies.