Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 Dec 27;6(24):6162-6168.
doi: 10.1182/bloodadvances.2022007031.

Evaluation of the combination therapy of hydroxyurea and thalidomide in β-thalassemia

Saqib H Ansari  1 , Iqra Ansari  2   3 , Misbah Wasim  4 , Amjad Sattar  5   6 , Shariqa Khawaja  2 , Muhammad Zohaib  7 , Zeeshan Hussain  7 , Syed Omair Adil  8 , Ali H Ansari  2 , Usman H Ansari  2 , Fawad Farooq  2   9 , Noor-Un-Nisa Masqati  1   10
Affiliations

Evaluation of the combination therapy of hydroxyurea and thalidomide in β-thalassemia

Saqib H Ansari et al. Blood Adv. .

Abstract

Transfusion-related complications and lack of resources in low-to-middle-income countries have led to a search for novel therapies to reduce the need for blood transfusions in patients with β-thalassemia. Hydroxyurea (HU) has demonstrated promising outcomes; additionally, thalidomide has also shown improvement in hemoglobin (Hb) levels for patients with β-thalassemia in some studies. This study presents the findings of a single-arm nonrandomized trial to evaluate the efficacy of combination therapy of HU and thalidomide in children with β-thalassemia. A total of 135 patients (median age, 6 [interquartile range, 3-10] years), 77 (57%) males and 58 (43%) females, were followed first using HU alone, for 6 months, and then using the combination of HU and thalidomide for another 6 months. The primary outcome was a response to therapy, as measured by the number of transfusions required and Hb levels, for patients while receiving HU alone and then while using the combination therapy. Study findings showed a significant decline in blood transfusion volume (P < .001) and a significant increase in median Hb levels within 3 and 6 months of the combination therapy (P < .001). Eighty-nine (65.93%) participants were good responders, 16 (11.85%) were responders, and 30 (22.22%) were nonresponders, whereas the responders had variable genetic mutations. A total of 38 adverse events were reported that resolved on supportive treatment or temporary hold of the intervention. The combination therapy demonstrated promising results and could be considered for a diverse patient population with β-thalassemia. This trial was registered at www.clinicaltrials.gov as #NCT05132270.

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Comparison of response with respect to mutations (n = 135).
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010;5:11. - PMC - PubMed
    1. Origa R. β-Thalassemia. Genet Med. 2017;19(6):609–619. - PubMed
    1. Pinto VM, Poggi M, Russo R, Giusti A, Forni GL. Management of the aging beta-thalassemia transfusion-dependent population - the Italian experience. Blood Rev. 2019;38 - PubMed
    1. Shamshirsaz AA, Bekheirnia MR, Kamgar M, et al. Metabolic and endocrinologic complications in beta-thalassemia major: a multicenter study in Tehran. BMC Endocr Disord. 2003;3(1):4. - PMC - PubMed
    1. Tubman VN, Fung EB, Vogiatzi M, et al. Thalassemia Clinical Research Network Guidelines for the standard monitoring of patients with thalassemia: report of the Thalassemia Longitudinal Cohort. J Pediatr Hematol Oncol. 2015;37(3):e162–e169. - PMC - PubMed

Associated data