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About 7,885 results

ALLMedicine™ Thalassemia Center

Research & Reviews  2,848 results

Identification of a Novel Hb H Disease with Glucose-6-Phosphate Dehydrogenase Deficienc...
https://doi.org/10.1080/03630269.2022.2070072
Hemoglobin Ren ZM, Xing ZH et. al.

May 19th, 2022 - With the development of sequencing technology, more and more rare thalassemia types have been found. In this article, we found a novel Hb H disease combined with glucose-6-phosphate dehydrogenase (G6PD) deficiency through whole genome sequencing (...

Hb Westport β121 (GH4) Glu>Asp; HBB: c.366A>C: A novel β-globin variant interfering wit...
https://doi.org/10.1016/j.clinbiochem.2022.05.003
Clinical Biochemistry; Moore JA, Li BV et. al.

May 18th, 2022 - To describe a novel β-globin variant that interferes with HbA1c analysis by cation exchange HPLC. Diabetes screening by HbA1c measurement was assessed using cation exchange HPLC and an immunoassay point-of-care analyzer. Routine hemoglobinopathy s...

A Triple-Heterozygous β-Thalassemia Patient Demonstrated an Unusual Electrophoresis Pat...
https://doi.org/10.1080/03630269.2022.2072322
Hemoglobin Liao J, Li Q et. al.

May 17th, 2022 - β-Thalassemia (β-thal) is caused by mutations on the β-globin genes, causing reduced (β+) or absent (β0) synthesis of the β chains of hemoglobin (Hb). In this report, a 28-year-old male patient with anemia and jaundice, was diagnosed with triple-h...

Diagnostic Dilemma of Hemoglobinopathies Using High Performance Liquid Chromatography A...
https://doi.org/10.1080/03630269.2022.2072324
Hemoglobin Sen A, Sen A et. al.

May 17th, 2022 - Hemoglobinopathies are quite common in India, and multiple awareness and screening initiatives exist for detection of thalassemia in the population. One of the most common and successfully used method for thalassemia screening is the high performa...

HLA-E*01:01 allele is associated with better response to anti-HCV therapy while homozyg...
https://doi.org/10.1016/j.humimm.2022.04.010
Human Immunology; Hosseini E, Sarraf Kazerooni E et. al.

May 16th, 2022 - HLA-E binding to NKG2A/CD94 induces inhibitory signals that modulate NK cells cytotoxicity against infected targets. HCV-derived peptides stabilize HLA-E molecule that favours its higher expression. However, HLA-E stability and expression vary in ...

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Guidelines  5 results

Red blood cell specifications for patients with hemoglobinopathies: a systematic review...
https://doi.org/10.1111/trf.14611
Transfusion Compernolle V, Chou ST et. al.

Apr 27th, 2018 - Red blood cell (RBC) transfusions remain essential in the treatment of patients with sickle cell disease (SCD) and β-thalassemia. Alloimmunization, a well-documented complication of transfusion, increases the risk of delayed hemolytic transfusion ...

Guidelines for the Standard Monitoring of Patients With Thalassemia: Report of the Thal...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4511957
Journal of Pediatric Hematology/oncology; Tubman VN, Fung EB et. al.

Jul 23rd, 2015 - Chronic transfusion therapy has played a central role in extending life expectancy for patients with hemoglobinopathies such as thalassemia. However, this life-saving therapy is associated with numerous complications that now comprise the bulk of ...

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: i...
https://doi.org/10.3324/haematol.2013.099747
Haematologica Angelucci E, Matthes-Martin S et. al.

May 3rd, 2014 - Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention an...

Cardiovascular function and treatment in β-thalassemia major: a consensus statement fro...
https://doi.org/10.1161/CIR.0b013e31829b2be6
Circulation Pennell DJ, Udelson JE et. al.

Jun 19th, 2013 - This aim of this statement is to report an expert consensus on the diagnosis and treatment of cardiac dysfunction in β-thalassemia major (TM). This consensus statement does not cover other hemoglobinopathies, including thalassemia intermedia and s...

Management of chronic viral hepatitis in patients with thalassemia: recommendations fro...
https://doi.org/10.1182/blood-2009-11-248724
Blood Di Marco V, Capra M et. al.

Jun 17th, 2010 - Chelation therapy with new drugs prevents cardiac damage and improves the survival of thalassemia patients. Liver diseases have emerged as a critical clinical issue. Chronic liver diseases play an important role in the prognosis of thalassemia pat...

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Drugs  46 results see all →

Clinicaltrials.gov  120 results

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
https://clinicaltrials.gov/ct2/show/NCT04610866

May 13th, 2022 - Sickle cell disease (SCD) is a multisystem disorder associated with episodes of acute clinical events and progressive organ damage. Episodic pain, triggered by microvascular vaso-occlusion induced by sickling of red blood cells (RBCs), is the most...

A Study of IMR-687 in Subjects With Beta Thalassemia
https://clinicaltrials.gov/ct2/show/NCT04411082

May 13th, 2022 - A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for 36 weeks in 2 populations of adult subjects wit...

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
https://clinicaltrials.gov/ct2/show/NCT04770753

May 11th, 2022 - The mitapivat group will include approximately 114 participants. The placebo group will include approximately 57 participants.

Preventing Sickle Cell Kidney Disease
https://clinicaltrials.gov/ct2/show/NCT02373241

May 9th, 2022 - Feasibility Trial of Losartan to Correct Abnormal Circadian Blood Pressure. Cohort participants (below) identified with in-clinic hypertension and abnormal nocturnal dipping on 24 hour ABPM will be asked to participate in a feasibility trial of lo...

Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients
https://clinicaltrials.gov/ct2/show/NCT03877809

May 3rd, 2022 - The general aim of the protocol is to demonstrate the applicability of a personalised and precision medicine approach in beta-thalassemia in a clinical trial setting for a repurposed drug, namely sirolimus. The presence of high level of Fetal Hemo...

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News  129 results

Gene Therapy for Beta Thalassemia Safe, Effective Up to 8 Years, but Concerns Emerge
https://www.medscape.com/viewarticle/966016

Jan 5th, 2022 - NEW YORK (Reuters Health) - Lentiviral globin gene therapy with reduced-intensity conditioning (RIC) was safe and effective in beta-thalassemia in a 6-8-year follow-up, although cautious monitoring is still necessary, researchers say. Beta-thalass...

Beta-Thalassemia Gene Therapy Achieves Transfusion Independence
https://www.medscape.com/viewarticle/964649

Dec 12th, 2021 - In patients with transfusion-dependent beta-thalassemia, a single gene therapy infusion is capable of yielding durable transfusion independence and substantial improvements in iron overload, an investigator reported at the annual meeting of the Am...

FDA Grants Priority Review to Luspatercept-aamt for Non–Transfusion Dependent Beta Thalassemia
https://www.onclive.com/view/fda-grants-priority-review-to-luspatercept-aamt-for-non-transfusion-dependent-beta-thalassemia

Dec 3rd, 2021 - The FDA has granted priority review to the supplemental biologics license application (sBLA) for luspatercept-aamt (Reblozyl) for the treatment of anemia in adults with non–transfusion dependent (NTD) β-thalassemia. The European Medicines Agency h...

FDA Grants Priority Review to Beti-Cel for β-thalassemia Requiring Transfusions
https://www.onclive.com/view/fda-grants-priority-review-to-beti-cel-for--thalassemia-requiring-transfusions

Nov 22nd, 2021 - The FDA has granted a priority review designation to a biologics license application (BLA) for betibeglogene autotemcel (beti-cel) for the treatment of adult, adolescent, and pediatric patients with β-thalassemia across all genotypes who require r...

Adult Nonmalignant Hematology Is an Endangered Field That Merits Protection
https://www.onclive.com/view/adult-nonmalignant-hematology-is-an-endangered-field-that-merits-protection

Oct 6th, 2021 - Hematologists use the terms classical, nonmalignant, and benign hematology to reference the same entity: the study and management of nonmalignant disorders of the blood. It is a discipline unto itself, distinct from its sister field, malignant hem...

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Patient Education  6 results see all →