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Erschienen in: Indian Journal of Hematology and Blood Transfusion 2/2020

02.03.2020 | Short Communication

Thalidomide in Patients with Transfusion-Dependent E-Beta Thalassemia Refractory to Hydroxyurea: A Single-Center Experience

verfasst von: Arijit Nag, Vivek S. Radhakrishnan, Jeevan Kumar, Saurabh Bhave, Deepak Kumar Mishra, Reena Nair, Mammen Chandy

Erschienen in: Indian Journal of Hematology and Blood Transfusion | Ausgabe 2/2020

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Abstract

Transfusion-dependent E-Beta (EB) thalassemia is one of the major causes of hereditary hemoglobinopathies in India. Hydroxyurea has been tried for HbF induction and amelioration of the transfusion frequency in EB thalassemia. The primary objective of this retrospective study, conducted between January 2017 and December 2018, was to determine the efficacy of thalidomide in reducing transfusion frequency in patients with EB thalassemia who have failed a reasonable trial of hydroxyurea. Of the 21 patients studied, 15 (71.4%) attained transfusion independence (complete responders) and 1 (4.7%) attained partial response (50% decrease in transfusion requirement) while 5 (23.9%) were non-responders. 12 patients attained their response within 1 month, 2 patients achieved within 1–3 months, and 1 patient beyond 3 months. Median time to transfusion independence in complete responders was 1 month. The median time on thalidomide for the complete responders and partial responders was 16.48 months. No major grade 3/4 toxicities were documented. This approach needs larger randomised controlled studies. Thalidomide is a safe and effective strategy at reducing or abrogating transfusion requirement in patients with EB thalassemia. This approach requires further testing in systematic clinical trials.
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Metadaten
Titel
Thalidomide in Patients with Transfusion-Dependent E-Beta Thalassemia Refractory to Hydroxyurea: A Single-Center Experience
verfasst von
Arijit Nag
Vivek S. Radhakrishnan
Jeevan Kumar
Saurabh Bhave
Deepak Kumar Mishra
Reena Nair
Mammen Chandy
Publikationsdatum
02.03.2020
Verlag
Springer India
Erschienen in
Indian Journal of Hematology and Blood Transfusion / Ausgabe 2/2020
Print ISSN: 0971-4502
Elektronische ISSN: 0974-0449
DOI
https://doi.org/10.1007/s12288-020-01263-2

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