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Thalassemia (severe form)

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchOnly the strongest evidence (53)Meta-analysis (12)Treatment guideline (1)Randomized trial (20)Systematic review (12)Phase 3 study (8)Clinical trial (379)Review article (138)Early study (phase 1 or 2) (18)Case report (134)Laboratory or animal research (17)Commentary or editorial (44)Retracted publication (2) Studies currently recruiting participants only (31)

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Publications and studies (1521)

• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia. (2025-08-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major (2025-08-08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion (2025-08-08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients (2025-08-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. (2025-08-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Impact of Virtual Reality on Pain and Anxiety During Intravenous Cannulation Procedures Among Thalassemia Patients in UAE. (2025-08-01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major (2025-06-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan (2025-06-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia (2025-05-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long Term Beta Thalassemia Treatment: Findings From The Extension Period (2025-05-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Effect of Laughter Yoga in Beta Thalassaemia Major Patients: a Randomised Controlled Trial (2025-05-20) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants (2025-05-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of IMR-687 in Healthy Adult Volunteers (2025-05-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of IMR-687 in Subjects With Beta Thalassemia (2025-05-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy (2025-04-09) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase (2025-04-06) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. International Registry of Patients With Alpha Thalassemia (2025-04-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) (2025-04-02) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 (2025-04-02) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia. (2025-03-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia (2025-03-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. To Evaluate Long- Term Safety and Efficacy of Luspatercept (2025-03-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure (2025-03-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Β-Thalassemia Treatment with KL003 Cell Injection (2025-03-10) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies (2025-03-03) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19 (2025-02-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) (2025-02-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel (2025-02-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long Term Outcomes in β Thalassemia Major (2025-02-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) (2025-01-27) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia (2025-01-14) ♡
• Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies. (2024/12/31) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Hemolysis due to anti-IH in a patient with beta-thalassemia and Mycoplasma pneumoniae infection. (2024/12/31) ♡
• Relevance of Next-Generation Sequencing in the Diagnosis of Thalassemia and Hemoglobinopathies: The Experience of Four Italian Diagnostic Hubs. (2024/12/27) ♡
• A novel conditioning regimen with pre-transplantation immunosuppression reduces the complication rates in hematopoietic stem cell transplantation in transfusion-dependent β-thalassemia. (2024/12/21) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Reproductive Health in Women with Major β-Thalassemia: Evaluating Ovarian Reserve and Endocrine Complications. (2024/12/20) ♡
• A fatal case of Vibrio cholerae-associated diarrhea and bacteremia in a 30-year-old carrier of beta-thalassemia. (2024/12/19) ♡
• Circulating biomarkers associated with pediatric sickle cell disease. (2024/12/19) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Application of third-generation sequencing technology in the genetic testing of thalassemia. (2024/12/18) ♡
• Demographics, clinical characteristics, and real-world treatment patterns among patients with beta-thalassemia: a retrospective medical record abstraction study. (2024/12/15) ♡
• [Clinical characteristics of cytokine release syndrome after haploidentical hematopoietic stem cell transplantation for thalassemia major]. (2024/12/15) ♡
• Exploring the functional and immune landscape of E-β thalassemia patients through RNA sequencing of peripheral blood mononuclear cells. (2024/12/14) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Beta-Thalassemia and Male Infertility: Unraveling the Oxidative Stress Connection-An Up-to-Date Review. (2024/12/12) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Luspatercept: a treatment for ineffective erythropoiesis in thalassemia. (2024/12/06) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A case of intrathoracic extramedullary hematopoiesis-associated pleural effusion in a beta-thalassemia intermedia patient and a short literature review. (2024/12/04) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. The genotype and phenotype analysis in 3 cases with the rare genotype of HBB:c.316-146T > G. (2024/12/01) ♡
• Outcomes of Hematopoietic Stem Cell Transplantation in Patients With Thalassemia Major: How Do Anti-HLA Antibodies Impact?: The Impact of Anti-HLA Antibodies on Transplantation Outcomes in Thalassemia Major. (2024/12/01) ♡
• The value of Mentzer index in the diagnosis of children thalassemia minor. (2024/12/01) ♡
• [Application Analysis of Screening for Thalassemia in the Population of Childbearing Age in Quanzhou]. (2024/12/01) ♡
• [Analysis of genetic diagnosis results of 1 501 suspected Cases of thalassemia patients from 2020 to 2022]. (2024/12/01) ♡

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