Thalassemia 2016: Modern medicine battles an ancient disease

Neurological complications of beta-thalassemia

(2015) P. Nemtsas et al.   ANNALS OF HEMATOLOGY

Genomic approaches to identifying targets for treating β hemoglobinopathies

(2015) Duyen A. Ngo et al.   BMC Medical Genomics

Synergistic intracellular iron chelation combinations: mechanisms and conditions for optimizing iron mobilization

(2015) Evangelia Vlachodimitropoulou Koumoutsea et al.   BRITISH JOURNAL OF HAEMATOLOGY

Estimating tissue iron burden: current status and future prospects

(2015) John C. Wood   BRITISH JOURNAL OF HAEMATOLOGY

Accuracy of Magnetic Resonance Imaging in Diagnosis of Liver Iron Overload: A Systematic Review and Meta-analysis

(2015) Maria Sarigianni et al.   Clinical Gastroenterology and Hepatology

Macrophages and regulation of erythropoiesis

(2015) Rebecca N. Jacobsen et al.   CURRENT OPINION IN HEMATOLOGY

 -thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies

(2015) S. Rivella   HAEMATOLOGICA

Anemia: progress in molecular mechanisms and therapies

(2015) Vijay G Sankaran et al.   NATURE MEDICINE

 -thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies

(2015) S. Rivella   HAEMATOLOGICA

A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers

(2014) Kenneth M. Attie et al.   AMERICAN JOURNAL OF HEMATOLOGY

Conditioning regimens in allo-SCT for thalassemia major

(2014) V Mathews et al.   BONE MARROW TRANSPLANTATION

Hepatocellular carcinoma in thalassaemia: an update of the Italian Registry

(2014) Caterina Borgna-Pignatti et al.   BRITISH JOURNAL OF HAEMATOLOGY

Current approach to iron chelation in children

(2014) Yesim Aydinok et al.   BRITISH JOURNAL OF HAEMATOLOGY

Iron chelation

(2014) Sujit Sheth   CURRENT OPINION IN HEMATOLOGY

Bisphosphonates in the management of thalassemia-associated osteoporosis: a systematic review of randomised controlled trials

(2014) Andrea Giusti   JOURNAL OF BONE AND MINERAL METABOLISM

Thalassemia Bone Disease: A 19-Year Longitudinal Analysis

(2014) Phillip Wong et al.   JOURNAL OF BONE AND MINERAL RESEARCH

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia

(2014) Michael Dussiot et al.   NATURE MEDICINE

Preimplantation Diagnosis for Single Gene Disorders

(2014) Valerie Baker et al.   SEMINARS IN REPRODUCTIVE MEDICINE

Red blood cell transfusions for thalassemia: results of a survey assessing current practice and proposal of evidence-based guidelines

(2014) Cheryl Goss et al.   TRANSFUSION

Ferritin trends do not predict changes in total body iron in patients with transfusional iron overload

(2013) Mammen Puliyel et al.   AMERICAN JOURNAL OF HEMATOLOGY

A Novel Reduced-Intensity Conditioning Regimen for Unrelated Umbilical Cord Blood Transplantation in Children with Nonmalignant Diseases

(2013) Suhag H. Parikh et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies Using a Reduced-Intensity Conditioning Regimen and Third-Party Mesenchymal Stromal Cells

(2013) Sandhya Kharbanda et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Hepcidin and  -thalassemia major

(2013) E. Nemeth   BLOOD

Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach

(2013) J. Gaziev et al.   BLOOD

Clinical experience with fetal hemoglobin induction therapy in patients with  -thalassemia

(2013) K. M. Musallam et al.   BLOOD

Pain in thalassaemia: the effects of age on pain frequency and severity

(2013) Dru Haines et al.   BRITISH JOURNAL OF HAEMATOLOGY

Assessment and management of iron overload in β-thalassaemia major patients during the 21st century: a real-life experience from the Italian Webthal project

(2013) Antonio Piga et al.   BRITISH JOURNAL OF HAEMATOLOGY

Next generation sequencing of SNPs for non-invasive prenatal diagnosis: challenges and feasibility as illustrated by an application to β-thalassaemia

(2013) Thessalia Papasavva et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Preimplantation genetic diagnosis, an alternative to conventional prenatal diagnosis of the hemoglobinopathies

(2013) J. Traeger-Synodinos   International Journal of Laboratory Hematology

International Cooperation for the Cure and Prevention of Severe Hemoglobinopathies

(2013) Lawrence B. Faulkner et al.   JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY

Macrophages support pathological erythropoiesis in polycythemia vera and β-thalassemia

(2013) Pedro Ramos et al.   NATURE MEDICINE

Updates of the HbVar database of human hemoglobin variants and thalassemia mutations

(2013) Belinda Giardine et al.   NUCLEIC ACIDS RESEARCH

An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level

(2013) D. E. Bauer et al.   SCIENCE

Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME)

(2013) Elliott Vichinsky et al.   TRANSFUSION

The Prevention of Thalassemia

(2013) A. Cao et al.   Cold Spring Harbor Perspectives in Medicine

Genetic association studies in  -hemoglobinopathies

(2013) S. L. Thein   Hematology-American Society of Hematology Education Program

Hematopoietic Cell Transplantation for Thalassemia: A Global Perspective BMT Tandem Meeting 2013

(2012) Parinda A. Mehta et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

The definition and epidemiology of non-transfusion-dependent thalassemia

(2012) David J. Weatherall   BLOOD REVIEWS

Cerebral infarction in β-thalassemia intermedia: Breaking the silence

(2012) Khaled M. Musallam et al.   THROMBOSIS RESEARCH

Hypercoagulability in β-thalassemia: a status quo

(2012) Maria Domenica Cappellini et al.   Expert Review of Hematology

 -thalassemia: a model for elucidating the dynamic regulation of ineffective erythropoiesis and iron metabolism

(2011) Y. Ginzburg et al.   BLOOD

The challenge of haemoglobinopathies in resource-poor countries

(2011) David J. Weatherall   BRITISH JOURNAL OF HAEMATOLOGY

Bone loss caused by iron overload in a murine model: importance of oxidative stress

(2010) J. Tsay et al.   BLOOD

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Ineffective erythropoiesis and thalassemias

(2009) Stefano Rivella   CURRENT OPINION IN HEMATOLOGY

Update on Survival in Thalassemia Major

(2009) Paul Telfer   HEMOGLOBIN

Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in  -thalassemia

(2008) I. V. Libani et al.   BLOOD

Hematopoietic stem cell transplantation in thalassemia

(2008) J Gaziev et al.   BONE MARROW TRANSPLANTATION

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION

Single-Dose, Randomized, Double-Blind, Placebo-Controlled Study of ACE-011 (ActRIIA-IgG1) in Postmenopausal Women*

(2008) Jon Ruckle et al.   JOURNAL OF BONE AND MINERAL RESEARCH

Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of  -thalassemia

(2008) M. Uda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA