Ali H Algiraigri1, Nicola A M Wright2, Elizabeth Oddone Paolucci3, Aliya Kassam3. 1. Department of Hematology, King Abdulaziz University Hospital, Jeddah, Saudi Arabia; King Faisal Special Hospital and Research Center, Jeddah, Saudi Arabia; Department of Community Health Science, University of Calgary, Calgary, Alberta, Canada. Electronic address: aalgiraigri@gmail.com. 2. Department of Pediatrics, Alberta Children's Hospital, Calgary, Alberta, Canada. 3. Department of Community Health Science, University of Calgary, Calgary, Alberta, Canada.
Abstract
OBJECTIVE/ BACKGROUND: Nontransfusion-dependent β-thalassemia (NTDβT) syndromes consist of β-thalassemia intermedia and moderate hemoglobin E/β thalassemias. They are characterized by varying degrees of chronic anemia and a wide spectrum of complications due to ineffective erythropoiesis and iron overload from chronic transfusions. Hydroxyurea (HU), an oral chemotherapeutic drug, is anticipated to decrease disease severity. METHODS: We performed a meta-analysis to evaluate the clinical efficacy and safety of HU in NTDβT patients of any age. MEDLINE, EMBASE, Cochrane databases, and major conference proceedings for studies that assessed HU in NTDβT patients were searched. Qualities of eligible studies were assessed by National Institutes of Health tools. RESULTS: Seventeen studies, collectively involving 709 patients, fulfilled the eligibility criteria. HU was associated with a significant decrease in transfusion need in severe NTDβT with complete and overall (≥50%) response rates of 42% and 79%, respectively. For mild NTDβT, HU was effective in raising hemoglobin by 1g/L in 64% of patients. CONCLUSION: HU appears to be effective, well tolerated, and associated with mild and transient adverse events. NTDβT patients may benefit from a trial of HU, although large randomized clinical trials assessing its efficacy should be conducted to confirm the findings of this meta-analysis and to assess its long-term toxicity and response sustainability.
OBJECTIVE/ BACKGROUND: Nontransfusion-dependent β-thalassemia (NTDβT) syndromes consist of β-thalassemia intermedia and moderate hemoglobin E/β thalassemias. They are characterized by varying degrees of chronic anemia and a wide spectrum of complications due to ineffective erythropoiesis and iron overload from chronic transfusions. Hydroxyurea (HU), an oral chemotherapeutic drug, is anticipated to decrease disease severity. METHODS: We performed a meta-analysis to evaluate the clinical efficacy and safety of HU in NTDβT patients of any age. MEDLINE, EMBASE, Cochrane databases, and major conference proceedings for studies that assessed HU in NTDβT patients were searched. Qualities of eligible studies were assessed by National Institutes of Health tools. RESULTS: Seventeen studies, collectively involving 709 patients, fulfilled the eligibility criteria. HU was associated with a significant decrease in transfusion need in severe NTDβT with complete and overall (≥50%) response rates of 42% and 79%, respectively. For mild NTDβT, HU was effective in raising hemoglobin by 1g/L in 64% of patients. CONCLUSION:HU appears to be effective, well tolerated, and associated with mild and transient adverse events. NTDβT patients may benefit from a trial of HU, although large randomized clinical trials assessing its efficacy should be conducted to confirm the findings of this meta-analysis and to assess its long-term toxicity and response sustainability.
Authors: Hyun Ji Lee; Kyung Hwa Shin; Hyung Hoi Kim; Eu Jeen Yang; Kyung Hee Park; Min Ju Kim; Jeong Ran Kwon; Young Sil Choi; Jun Nyun Kim; Myung Geun Shin; Yong Gon Cho; Sun Jun Kim; Kyeong Hee Kim; Seri Jeong; Seom Gim Kong; Yu Jin Jung; Nayoung Lee; Man Jin Kim; Moon Woo Seong Journal: Ann Lab Med Date: 2019-03 Impact factor: 3.464