Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: The optimal care study

Ali T. Taher, Khaled M. Musallam, Mehran Karimi, Amal El-Beshlawy, Khawla Belhoul, Shahina Daar, Mohamed Salaheldin Saned, Abdul Hamid El-Chafic, Maria R. Fasulo, Maria D. Cappellini

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Abstract

Despite recent advances in understanding the pathophysiologic mechanisms behind the thalassemia intermedia (TI) phenotype, data on the effects of treatment are deficient. To provide such data, we evaluated 584 TI patients for the associations between patient and disease characteristics, treatment received, and the rate of complications. The most common disease-related complications were osteoporosis, extramedullary hematopoeisis (EMH), hypogonadism, and cholelithiasis, followed by thrombosis, pulmonary hypertension (PHT), abnormal liver function, and leg ulcers. Hypothyroidism, heart failure, and diabetes mellitus were less frequently observed. On multivariate analysis, older age and splenectomy were independently associated with an increased risk of most disease-related complications. Transfusion therapy was protective for thrombosis, EMH, PHT, heart failure, cholelithiasis, and leg ulcers. However, transfusion therapy was associated with an increased risk of endocrinopathy. Iron chelation therapy was in turn protective for endocrinopathy and PHT. Hydroxyurea treatment was associated with an increased risk of hypogonadism yet was protective for EMH, PHT, leg ulcers, hypothyroidism, and osteoporosis. Attention should be paid to the impact of age on complications in TI, and the beneficial role of splenectomy deserves revisiting. This study provides evidence that calls for prospective evaluation of the roles of transfusion, iron chelation, and hydroxyurea therapy in TI patients.

Original languageEnglish
Pages (from-to)1886-1892
Number of pages7
JournalBlood
Volume115
Issue number10
DOIs
Publication statusPublished - Mar 11 2010

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ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Taher, A. T., Musallam, K. M., Karimi, M., El-Beshlawy, A., Belhoul, K., Daar, S., ... Cappellini, M. D. (2010). Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: The optimal care study. Blood, 115(10), 1886-1892. https://doi.org/10.1182/blood-2009-09-243154