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Beta Thalassemia

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4. Genotype–phenotype correlation among beta-thalassemia and beta-thalassemia/HbE disease in Thai children: predictable clinical spectrum using genotypic analysis (PubMed)

Genotype–phenotype correlation among beta-thalassemia and beta-thalassemia/HbE disease in Thai children: predictable clinical spectrum using genotypic analysis Beta-thalassemia is a group of inherited hemolytic anemias and one of the most common genetic disorders in Thailand. The clinical spectrum of beta-thalassemia disease ranges from mild to severe clinical symptoms including mild beta-thalassemia intermedia (TI) and severe beta-thalassemia major (TM).This study aimed to determine (...) the correlation between beta-globin gene (HBB) mutations and their phenotypic manifestations by evaluating patients' clinical characteristics, transfusion requirements, growth and hematologic parameters, and hemoglobin typing among pediatric patients treated at Phramongkutklao Hospital.Seventy beta-thalassemia patients, including 63 with beta-thalassemia/hemoglobin E (HbE) and 7 with either homozygous or compound heterozygous beta-thalassemia, were enrolled in this study. Their clinical presentation, growth

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2018 Journal of blood medicine

5. Newborn blood spot screening for galactosemia, tyrosiemia type I, homocystinuria, sickle cell anemia, sickle cell/beta-thalassemia, sickle cell/hemoglobin C disease and severe combined immunodeficiency

Newborn blood spot screening for galactosemia, tyrosiemia type I, homocystinuria, sickle cell anemia, sickle cell/beta-thalassemia, sickle cell/hemoglobin C disease and severe combined immunodeficiency Newborn blood spot screening for galactosemia, tyrosiemia type I, homocystinuria, sickle cell anemia, sickle cell/beta-thalassemia, sickle cell/hemoglobin C disease and severe combined immunodeficiency Newborn blood spot screening for galactosemia, tyrosiemia type I, homocystinuria, sickle cell (...) anemia, sickle cell/beta-thalassemia, sickle cell/hemoglobin C disease and severe combined immunodeficiency Institute of Health Economics Record Status This is a bibliographic record of a published health technology assessment from a member of INAHTA. No evaluation of the quality of this assessment has been made for the HTA database. Citation Institute of Health Economics. Newborn blood spot screening for galactosemia, tyrosiemia type I, homocystinuria, sickle cell anemia, sickle cell/beta

2016 Health Technology Assessment (HTA) Database.

6. Long-term Safety and Efficacy Follow-up of Subjects Treated With GSK2696277 for Transfusion Dependent Beta-thalassemia in San Raffaele Telethon Institute of Gene Therapy-Beta Thalassemia (TIGET-BTHAL) Study

Long-term Safety and Efficacy Follow-up of Subjects Treated With GSK2696277 for Transfusion Dependent Beta-thalassemia in San Raffaele Telethon Institute of Gene Therapy-Beta Thalassemia (TIGET-BTHAL) Study Long-term Safety and Efficacy Follow-up of Subjects Treated With GSK2696277 for Transfusion Dependent Beta-thalassemia in San Raffaele Telethon Institute of Gene Therapy-Beta Thalassemia (TIGET-BTHAL) Study - Full Text View - ClinicalTrials.gov Hide glossary Glossary Study record managers (...) : refer to the if submitting registration or results information. Search for terms x × Study Record Detail Saved Studies Save this study Warning You have reached the maximum number of saved studies (100). Please remove one or more studies before adding more. Long-term Safety and Efficacy Follow-up of Subjects Treated With GSK2696277 for Transfusion Dependent Beta-thalassemia in San Raffaele Telethon Institute of Gene Therapy-Beta Thalassemia (TIGET-BTHAL) Study The safety and scientific validity

2017 Clinical Trials

7. Double heterozygocity for hemoglobin C and beta thalassemia dominant: A rare case of thalassemia intermedia (PubMed)

Double heterozygocity for hemoglobin C and beta thalassemia dominant: A rare case of thalassemia intermedia 29383226 2018 11 13 2038-8322 9 4 2017 Dec 22 Hematology reports Hematol Rep Double heterozygocity for hemoglobin C and beta thalassemia dominant: A rare case of thalassemia intermedia. 7447 10.4081/hr.2017.7447 Agapidou Alexandra A Hemoglobinopathy Service, Homerton University Hospital NHS Foundation Trust, London, UK. King Paul P Hemoglobinopathy Service, Homerton University Hospital (...) NHS Foundation Trust, London, UK. Ng Cecilia C Hemoglobinopathy Service, Homerton University Hospital NHS Foundation Trust, London, UK. Tsitsikas Dimitris A DA Hemoglobinopathy Service, Homerton University Hospital NHS Foundation Trust, London, UK. eng Journal Article 2018 01 03 Italy Hematol Rep 101556723 2038-8322 DNA analysis HbC Thalassemia electrophoresis 2017 10 13 2017 11 20 2017 11 21 2018 2 1 6 0 2018 2 1 6 0 2018 2 1 6 1 epublish 29383226 10.4081/hr.2017.7447 PMC5768097 Genet Med. 2010

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2018 Hematology reports

8. Sotatercept, a novel transforming growth factor beta ligand trap, improves anemia in beta-thalassemia: a phase 2, open-label, dose-finding study. (PubMed)

Sotatercept, a novel transforming growth factor beta ligand trap, improves anemia in beta-thalassemia: a phase 2, open-label, dose-finding study. β-thalassemia, a hereditary blood disorder caused by defective synthesis of hemoglobin β globin chains, leads to ineffective erythropoiesis and chronic anemia that may require blood transfusions. Sotatercept (ACE-011) acts as a ligand trap to inhibit negative regulators of late-stage erythropoiesis in the transforming growth factor β superfamily (...) , correcting ineffective erythropoiesis. In this phase II, open-label, dose-finding study, 16 patients with transfusion-dependent β -thalassemia and 30 patients with non-transfusion-dependent β-thalassemia were enrolled at seven centers in four countries between November 2012 and November 2014. Patients were treated with sotatercept at doses of 0.1, 0.3, 0.5, 0.75, or 1.0 mg/kg to determine a safe and effective dose. Doses were administered by subcutaneous injection every 3 weeks. Patients were treated

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2018 Haematologica

9. A comprehensive review of the prevalence of beta globin gene variations and the co-inheritance of related gene variants in Saudi Arabians with beta-thalassemia (PubMed)

A comprehensive review of the prevalence of beta globin gene variations and the co-inheritance of related gene variants in Saudi Arabians with beta-thalassemia Beta-thalassemia is a genetic disorder that is caused by variations in the beta-hemoglobin (HBB) gene. Saudi Arabia is among the countries most affected bybeta-thalassemia, and this is particularly problematic in the Eastern regions. This review article is an attempt to compile all the reported mutations to facilitate further national (...) regions to create a well-documented molecular data bank. This data bank can be used to develop a premarital screening program and lead to the best treatment and prevention strategies for beta-thalassemia.

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2018 Saudi medical journal

10. Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with beta-thalassemia. (PubMed)

Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with beta-thalassemia. Beta-thalassemia is a hereditary disorder with limited approved treatment options; patients experience anemia and its complications, including iron overload. This study aim was to determine whether luspatercept could improve anemia and disease complications in patients with beta-thalassemia. This open-label, nonrandomized, uncontrolled study (NCT01749540 and NCT02268409 (...) versus baseline. Twenty-six (81%) with transfusion dependence achieved ≥20% reduction in RBC transfusion burden. The most common grade 1-2 adverse events were bone pain, headache, and myalgia. As of the cutoff, 33 patients remain on study. In this study, a high percentage of beta-thalassemia patients receiving luspatercept had improvements in hemoglobin or transfusion burden. These findings support a randomized clinical trial to assess efficacy and safety.Copyright © 2019 American Society

2019 Blood

11. Global analysis of erythroid cells redox status reveals the involvement of Prdx1 and Prdx2 in the severity of beta thalassemia. (PubMed)

Global analysis of erythroid cells redox status reveals the involvement of Prdx1 and Prdx2 in the severity of beta thalassemia. β-thalassemia is a worldwide distributed monogenic red cell disorder, characterized by an absent or reduced beta globin chain synthesis. The unbalance of alpha-gamma chain and the presence of pathological free iron promote severe oxidative damage, playing crucial a role in erythrocyte hemolysis, exacerbating ineffective erythropoiesis and decreasing the lifespan of red (...) promotes the molecular chaperone activity of these proteins. Some studies have demonstrated the importance of Prdx1 and Prdx2 for the development and maintenance of erythrocytes in hemolytic anemia. Now, we performed a global analysis comparatively evaluating the expression profile of several antioxidant enzymes and their physiological reducing agents in patients with beta thalassemia intermedia (BTI) and healthy individuals. Furthermore, increased levels of ROS were observed not only in RBC, but also

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2018 PLoS ONE

12. Haplotypes inside the beta-globin gene: use as new biomarkers for beta-thalassemia prenatal diagnosis in north of Iran (PubMed)

Haplotypes inside the beta-globin gene: use as new biomarkers for beta-thalassemia prenatal diagnosis in north of Iran Beta-thalassemia is common in the Mediterranean area as well as the Middle East and India. Official report in Iran revealed the average prevalence rate of carriers about 4%. More than 20 restriction fragment length polymorphisms (RFLPs) are known in the beta-globin gene cluster and used in the prenatal diagnosis (PND) services. Some of these locations may have low allele (...) frequency and are not informative in the prenatal diagnosis. The current study aims to find new haplotypes and polymorphisms with high allele frequency in the local population.Two thousand three hundred fifty samples (1,321 male and 1,029 female) from the northern Iran, whom suspected to be the carriers either for alpha or beta thalassemia and referred to the local diagnostic laboratory as a routine services were investigated during five years, (2010-2015). The beta-globin gene was sequenced for all

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2017 Journal of biomedical science

13. Premarital Genetic Diagnosis Revealed Co-heredity Nature of Beta Globin Gene 25-26 del AA and 3’UTR+101 G-C Variants in Two Beta Thalassemia Heterozygotes (PubMed)

Premarital Genetic Diagnosis Revealed Co-heredity Nature of Beta Globin Gene 25-26 del AA and 3’UTR+101 G-C Variants in Two Beta Thalassemia Heterozygotes 27751971 2018 10 23 2018 11 13 1308-5263 34 1 2017 03 01 Turkish journal of haematology : official journal of Turkish Society of Haematology Turk J Haematol Premarital Genetic Diagnosis Revealed Co-heredity Nature of Beta Globin Gene 25-26 del AA and 3'UTR+101 G-C Variants in Two Beta Thalassemia Heterozygotes. 105-106 10.4274/tjh.2016.0069 (...) beta-Globins genetics beta-Thalassemia genetics 2016 10 19 6 0 2018 10 24 6 0 2016 10 19 6 0 ppublish 27751971 10.4274/tjh.2016.0069 PMC5451673 Am J Hematol. 1998 Aug;58(4):306-10 9692395 Eur J Haematol. 2014;92(5):444-9 24401016 Hemoglobin. 1993 Dec;17(6):551-5 8144356 Blood. 2001 Jul 1;98(1):250-1 11439976 Int J Lab Hematol. 2013 Feb;35(1):26-30 22862814

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2017 Turkish Journal of Hematology

14. Role of serum cystatin-C and beta-2 microglobulin as early markers of renal dysfunction in children with beta thalassemia major (PubMed)

Role of serum cystatin-C and beta-2 microglobulin as early markers of renal dysfunction in children with beta thalassemia major Although advancements have been made in the management of thalassemic patients, many unrecognized complications have emerged, such as renal abnormalities.To measure serum levels of cystatin-C and β-2 microglobulin in children with beta-thalassemia major (β-TM) and investigate their significance as early markers of glomerular and tubular dysfunctions.The study

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2017 International journal of nephrology and renovascular disease

15. Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine. (PubMed)

Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine. Iron overload is the primary cause of mortality and morbidity in thalassemia major (TM) despite advances in chelation therapy. The aim of this study was to compare the effectiveness and safety of deferasirox (DFX) and deferoxamine (DFO) as iron-chelating agents in patients with transfusion-dependent β-thalassemia major.This prospective randomized study (...) ). There were no mortalities or any serious adverse effects, neutropenia, arthropathy, or pulmonary toxicity. Gastrointestinal upset and skin rash occurred more frequently with DFX than with DFO (p = 0.254 and 0.095, respectively).With appropriate dosing and compliance with drugs, both DFX and DFO are generally well tolerated, safe, and effective in reducing serum ferritin levels in iron-overloaded, regularly-transfused thalassemia major patients. Therefore, oral DFX is recommended for more convenience

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2016 Electronic physician

16. Efficacy and safety of resveratrol, an oral hemoglobin F-augmenting agent, in patients with beta-thalassemia intermedia. (PubMed)

Efficacy and safety of resveratrol, an oral hemoglobin F-augmenting agent, in patients with beta-thalassemia intermedia. Recently, resveratrol showed induction of γ-globin mRNA synthesis in human erythroid precursors and reducing oxidative stress in red cells of thalassemia patients in many in vitro studies. We aimed to investigate the efficacy and safety of resveratrol, for the first time, in non-transfusion-dependent beta-thalassemia intermedia (B-TI) in Southern Iran. In this double-blind

2018 Annals of hematology

17. Late effects after hematopoietic stem cell transplantation for beta-thalassemia major: the French national experience. (PubMed)

Late effects after hematopoietic stem cell transplantation for beta-thalassemia major: the French national experience. In this retrospective study, we evaluate long-term complications in nearly all β-thalassemia-major patients who successfully received allogeneic hematopoietic stem cell transplantation in France. Ninety-nine patients were analyzed with a median age of 5.9 years at transplantation. The median duration of clinical follow up was 12 years. All conditioning regimens were

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2018 Haematologica

18. Impaired acylcarnitine profile in transfusion-dependent beta-thalassemia major patients in Bangladesh (PubMed)

Impaired acylcarnitine profile in transfusion-dependent beta-thalassemia major patients in Bangladesh Patients with beta-thalassemia major (BTM) suffer from fatigue, poor physical fitness, muscle weakness, lethargy, and cardiac complications which are related to an energy crisis. Carnitine and acylcarnitine derivatives play important roles in fatty acid oxidation, and deregulation of carnitine and acylcarnitine metabolism may lead to an energy crisis. The present study aimed to investigate

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2018 Journal of advanced research

19. Long-term safety of deferiprone treatment in children from the Mediterranean region with beta-thalassemia major: the DEEP-3 multi-center observational safety study (PubMed)

Long-term safety of deferiprone treatment in children from the Mediterranean region with beta-thalassemia major: the DEEP-3 multi-center observational safety study 29079595 2018 12 10 1592-8721 103 1 2018 01 Haematologica Haematologica Long-term safety of deferiprone treatment in children from the Mediterranean region with beta-thalassemia major: the DEEP-3 multi-center observational safety study. e1-e4 10.3324/haematol.2017.176065 Botzenhardt Sebastian S Department of Paediatrics

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2018 Haematologica

20. The Correlation of Cardiac and Hepatic Hemosiderosis as Measured by T2*MRI Technique with Ferritin Levels and Hemochromatosis Gene Mutations in Iranian Patients with Beta Thalassemia Major (PubMed)

The Correlation of Cardiac and Hepatic Hemosiderosis as Measured by T2*MRI Technique with Ferritin Levels and Hemochromatosis Gene Mutations in Iranian Patients with Beta Thalassemia Major Organ-specific hemosiderosis and iron overload complications are more serious and more frequent in some patients with beta thalassemia major (BTM) compared with others. We investigated whether coinheritance of HFE H63D or C282Y gene mutations in patients with BTM contributes to the phenotypic variation

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2018 Oman medical journal

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