Skip to content

Beta Thalassemia Major (Pediatrics)

Pharmacological Induction of Fetal Hemoglobin Synthesis

Hydroxyurea (Hydroxycarbamide) Therapy

Efficacy of hydroxyurea in providing transfusion independence in β-thalassemia. (opens new window)

Ansari SH, Shamsi TS, Ashraf M, Perveen K, Farzana T, Bohray M, Erum S, Mehboob T.

Source‎: J Pediatr Hematol Oncol 2011;33(5):339-43.

Indexed‎: PubMed 21602718

DOI‎: 10.1097/MPH.0b013e31821b0770

https://www.ncbi.nlm.nih.gov/pubmed/21602718 (opens new window)

Gγ-Xmn I polymorphism: a significant determinant of β-thalassemia treatment without blood transfusion. (opens new window)

Ansari SH, Shamsi TS, Munzir S, Khan MT, Erum S, Perveen K, Farzana T, Ashraf M, Mehboob T, Moinuddin M.

Source‎: J Pediatr Hematol Oncol 2013;35(4):e153-6.

Indexed‎: PubMed 23389500

DOI‎: 10.1097/MPH.0b013e31827e8662

https://www.ncbi.nlm.nih.gov/pubmed/23389500 (opens new window)

Efficacy of hydroxyurea (HU) in reduction of pack red cell (PRC) transfusion requirement among children having beta-thalassemia major: Karachi HU trial (KHUT). (opens new window)

Ansari SH, Shamsi TS, Siddiqui FJ, Irfan M, Perveen K, Farzana T, Panjwani VK, Yousuf A, Mehboob T.

Source‎: J Pediatr Hematol Oncol 2007;29(11):743-6.

Indexed‎: PubMed 17984691

DOI‎: 10.1097/MPH.0b013e318157fd75

https://www.ncbi.nlm.nih.gov/pubmed/17984691 (opens new window)

Hydroxyurea can eliminate transfusion requirements in children with severe beta-thalassemia. (opens new window)

Bradai M, Abad MT, Pissard S, Lamraoui F, Skopinski L, de Montalembert M.

Source‎: Blood 2003;102(4):1529-30.

Indexed‎: PubMed 12702505

DOI‎: 10.1182/blood-2003-01-0117

https://www.ncbi.nlm.nih.gov/pubmed/12702505 (opens new window)

Decreased transfusion needs associated with hydroxyurea therapy in Algerian patients with thalassemia major or intermedia. (opens new window)

Bradai M, Pissard S, Abad MT, Dechartres A, Ribeil JA, Landais P, de Montalembert M.

Source‎: Transfusion 2007;47(10):1830-6.

Indexed‎: PubMed 17880608

DOI‎: 10.1111/j.1537-2995.2007.01399.x

https://www.ncbi.nlm.nih.gov/pubmed/17880608 (opens new window)

Genotype-phenotype relationship of patients with β-thalassemia taking hydroxyurea: a 13-year experience in Iran. (opens new window)

Karimi M, Haghpanah S, Farhadi A, Yavarian M.

Source‎: Int J Hematol 2012;95(1):51-6.

Indexed‎: PubMed 22180324

DOI‎: 10.1007/s12185-011-0985-6

https://www.ncbi.nlm.nih.gov/pubmed/22180324 (opens new window)

A randomised double-blind placebo-controlled clinical trial of oral hydroxyurea for transfusion-dependent β-thalassaemia. (opens new window)

Yasara N, Wickramarathne N, Mettananda C, Silva I, Hameed N, Attanayaka K, Rodrigo R, Wickramasinghe N, Perera L, Manamperi A, Premawardhena A, Mettananda S

Source‎: Sci Rep. 2022;12(1):2752.

Indexed‎: PubMed 35177777

DOI‎: 10.1038/s41598-022-06774-8

https://pubmed.ncbi.nlm.nih.gov/35177777/ (opens new window)

Response to hydroxyurea treatment in Iranian transfusion-dependent beta-thalassemia patients. (opens new window)

Yavarian M, Karimi M, Bakker E, Harteveld CL, Giordano PC.

Source‎: Haematologica 2004;89(10):1172-8.

Indexed‎: PubMed 15477200

https://www.ncbi.nlm.nih.gov/pubmed/15477200 (opens new window)

Hydroxyurea therapy in 49 patients with major beta-thalassemia. (opens new window)

Zamani F, Shakeri R, Eslami SM, Razavi SM, Basi A.

Source‎: Arch Iran Med 2009;12(3):295-7.

Indexed‎: PubMed 19400608

https://www.ncbi.nlm.nih.gov/pubmed/19400608 (opens new window)

Recombinant Erythropoietin (Alone or in Combination With Other Agents)

Use of hydroxyurea and recombinant erythropoietin in management of homozygous ß⁰ thalassemia. (opens new window)

Kohli-Kumar M, Marandi H, Keller MA, Guertin K, Hvizdala E.

Source‎: J Pediatr Hematol Oncol 2002;24(9):777-8.

Indexed‎: PubMed 12468925

https://www.ncbi.nlm.nih.gov/pubmed/12468925 (opens new window)

Other Agents

Fetal globin gene inducers: novel agents and new potential. (opens new window)

Perrine SP, Castaneda SA, Chui DH, Faller DV, Berenson RJ, Siritanaratku N, Fucharoen S.

Source‎: Ann N Y Acad Sci 2010;1202:158-64.

Indexed‎: PubMed 20712788

DOI‎: 10.1111/j.1749-6632.2010.05593.x

https://www.ncbi.nlm.nih.gov/pubmed/20712788 (opens new window)