How I treat non-transfusion-dependent β-thalassemia
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Elsevier B.V.
Abstract
The intricate interplay of anemia and iron overload under the pathophysiological umbrella of ineffective erythropoiesis in non-transfusion-dependent β-thalassemia (NTDT) results in a complex variety of clinical phenotypes that are challenging to diagnose and manage. In this article, we use a clinical framework rooted in pathophysiology to present 4 common scenarios of patients with NTDT. Starting from practical considerations in the diagnosis of NTDT, we delineate our strategy for the longitudinal care of patients who exhibit different constellations of symptoms and complications. We highlight the use of transfusion therapy and novel agents, such as luspatercept, in the patient with anemia-related complications. We also describe our approach to chelation therapy in the patient with iron overload. Although tackling every specific complication of NTDT is beyond the scope of this article, we touch on the management of the various morbidities and multisystem manifestations of the disease. © 2023 The American Society of Hematology
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Beta-thalassemia, Chelation therapy, Humans, Iron chelating agents, Iron overload, Thalassemia, Acetylsalicylic acid, Alanine aminotransferase, Ambrisentan, Antibiotic agent, Anticoagulant agent, Antithrombocytic agent, Beta adrenergic receptor blocking agent, Bilirubin, Bisphosphonic acid derivative, Bosentan, Calcium channel blocking agent, Deferasirox, Deferiprone, Deferoxamine, Dexamethasone, Dipeptidyl carboxypeptidase inhibitor, Ferritin, Ferrous sulfate, Glecaprevir plus pibrentasvir, Haptoglobin, Hemoglobin, Hemoglobin a2, Hemoglobin alpha chain, Hepatitis vaccine, Hydroxyurea, Influenza vaccine, Iron, Iron chelating agent, Lactate dehydrogenase, Ledipasvir plus sofosbuvir, Levothyroxine, Low molecular weight heparin, Luspatercept, Macitentan, Pentoxifylline, Pneumococcus vaccine, Riociguat, Ruxolitinib, Sildenafil, Sodium nitrite, Sofosbuvir plus velpatasvir, Tadalafil, Adult, Analgesia, Anemia, Antibiotic therapy, Anticoagulant therapy, Antiviral therapy, Beta thalassemia, Blood transfusion, Case report, Cerebrovascular accident, Clinical article, Drug dose titration, Dual energy x ray absorptiometry, Endocrine bone disease, Erythrocyte transfusion, Exertional dyspnea, Extramedullary hematopoiesis, Fatigue, Female, Ferritin blood level, Functional status, Gallstone, Gene insertion, Genotype, Heart left ventricle ejection fraction, Hemoglobin determination, Hepatitis a, Hepatitis b, Hepatitis c, Hepatosplenomegaly, Human, Hypothyroidism, Iron deficiency anemia, Knee pain, Leg pain, Leg ulcer, Liver cell carcinoma, Liver cirrhosis, Liver fibrosis, Low back pain, Lung artery pressure, Male, Mean corpuscular hemoglobin concentration, Mean corpuscular volume, Microcytic anemia, Morbidity, Multiple cycle treatment, Non transfusion dependent thalassemia, Nuclear magnetic resonance imaging, Osteopenia, Pathophysiology, Patient care, Patient referral, Phase 2 clinical trial (topic), Phenotype, Platelet count, Pregnancy, Protein electrophoresis, Pseudotumor, Pulmonary hypertension, Randomized controlled trial (topic), Reticulocyte count, Review, Symptom, Syndrome delineation, Thalassemia intermedia, Thrombosis, Transthoracic echocardiography, Treatment indication, Treatment refusal, Tricuspid regurgitation velocity, Ulcer healing