Thalidomide and Thalassemia

Hi,

I was wondering if anyone, either Thal major or intermmedia taking thalodomide or have heard of patients taking this medicine to help treatment? If so, what feedback do you have?

Thalidomide is helping many patients in India, but it is not for everyone, as some patients do have side effects that can't be ignored. There are a couple doctors using this therapy in India. The therapy usually includes other complementary drugs and supplements, like hydroxyurea, wheatgrass. folate and B12.

This was just published about the use of thalidomide in thalassemics.
https://sciforschenonline.org/journals/blood-disorders-medicine/JBDM-2-119.php

Role of Thalidomide in Treatment of Beta Thalassemia
  Vijay Ramanan*      Ketki Kelkar 

Abstract
Objective: To assess the effect of thalidomide on ferritin levels and duration of blood transfusion in patients with β-thalassemia.

Background: Thalassemia is the result of defective hemoglobin production due to reduced or absent expression of β- globin gene. Currently, the main therapy in β-thalassemia patients is a regular blood transfusion schedule and use of iron chelating agents. However, they are associated with limitations and severe therapeutic complications. Thalidomide can reduce α-globin chain production in erythroid progenitors and improve α:β chain imbalance. Few case reports indicate that thalidomide should be considered in cases of thalassemia which cannot be treated with transfusions and do not respond to hydroxyurea.

Design: Retrospective study

Subjects and method: Medical records of thalassemia patients who received thalidomide between January 2006 and April 2016 in our institution were reviewed. Data gathered included age, sex, splenectomy status, ferritin levels before and after treatment, duration of off blood transfusion, first blood transfusion was analyzed.

Results: the study included 104 subjects (34 females and 70 male) with an age ranging from 1 to 36 years (mean age: 13.04 years). Out of this, 27 patients had undergone splenectomy. Data of ferritin levels before and after treatment were available for 59 patients. Ferritin levels reduced to 51% in all patients (4037 to 2086 ng/ml). Ferritin levels reduced to 55% in patients who had undergone splenectomy and 49% in patients who had not undergone splenectomy. Age at which first blood transfusion started ranged from 3 months to 7.5 years (mean: 16.6 months). Duration of off blood transfusion ranged from 1 month to 42 months (mean: 10.22 months). There were 9 cases with βº- Thal Homozygotes and 4 cases of β+-Thal Homozygotes or β+/βº Compound Heterozygotes.

Conclusion: Thalidomide treatment reduced ferritin levels. Further studies are required to define the potential use of thalidomide in thalassaemia.