Hematology and Blood Disorders

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Mini Review - Hematology and Blood Disorders (2024) Volume 7, Issue 2

Current therapies and emerging treatments for thalassemias: A comprehensive review

Dusk Wilder *

Department of Hematology and Blood Transfusion, Karolinska Institutet, Sweden

*Corresponding Author:
Dusk Wilder
Department of Hematology and Blood Transfusion, Karolinska Institutet, Sweden
E-mail: Wilder45@ki.se

Received: 01-June-2024, Manuscript No. AAHBD-24-137803; Editor assigned: 04-June-2024, PreQC No AAHBD-24-137803 (PQ) Reviewed:15-June-2024, QC No. AAHBD-24-137803Revised:20-June-2024, Manuscript No. AAHBD-24-137803 (R); Published:27-June-2024, DOI:10.35841/aahbd-7.2.184

Citation: Wilder D. Current therapies and emerging treatments for thalassemias: A comprehensive review. Hematol Blood Disord. 2024;7(2):184

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Introduction

Thalassemias, a group of inherited blood disorders characterized by abnormal hemoglobin production, present significant challenges in diagnosis and management. With advances in medical research and technology, the landscape of thalassemia treatment is constantly evolving. This article provides a comprehensive review of current therapies and emerging treatments for thalassemias, highlighting their efficacy, limitations, and potential impact on patient care [1].

Blood Transfusions: Regular blood transfusions remain the cornerstone of treatment for individuals with severe forms of thalassemia, such as beta-thalassemia major. Transfusions help alleviate anemia by providing healthy red blood cells with normal hemoglobin levels. However, long-term transfusion therapy can lead to iron overload, necessitating concurrent iron chelation therapy [2].

Iron Chelation Therapy: Iron chelators, such as deferoxamine, deferiprone, and deferasirox, are used to remove excess iron from the body in patients undergoing chronic blood transfusions. These medications help prevent iron accumulation in vital organs, reducing the risk of complications such as heart disease, liver damage, and endocrine dysfunction [3].

Folic Acid Supplementation: Folic acid supplementation is often prescribed to individuals with thalassemia to support red blood cell production and mitigate the effects of chronic anemia. Folic acid helps stimulate the production of new red blood cells, which can improve energy levels and reduce symptoms of fatigue [4].

Gene Therapy: Gene therapy holds promise as a potential curative treatment for thalassemias by addressing the underlying genetic defect responsible for abnormal hemoglobin production. Techniques such as gene addition, gene editing (e.g., CRISPR-Cas9), and lentiviral vector-mediated gene transfer are being investigated in clinical trials. Early results have shown encouraging outcomes, including increased hemoglobin levels and reduced transfusion requirements [5].

Fetal Hemoglobin Induction: Fetal hemoglobin (HbF) has a higher oxygen-binding capacity than adult hemoglobin (HbA) and can compensate for defective HbA in individuals with thalassemia [6].

Therapies aimed at inducing HbF production, such as hydroxyurea and gene modulation agents, are being explored as potential treatments for thalassemias. These therapies have shown promise in increasing HbF levels and improving clinical outcomes in some patients [7].

Bone Marrow Transplantation: Allogeneic bone marrow transplantation, also known as hematopoietic stem cell transplantation (HSCT), remains the only curative treatment for thalassemia. HSCT involves replacing the patient's diseased bone marrow with healthy donor stem cells capable of producing normal red blood cells. However, HSCT is associated with risks, including graft rejection, graft-versus-host disease (GVHD), and transplant-related complications [8].

Thalassemias pose significant challenges to patients and healthcare providers due to their chronic nature and potential complications. While current therapies such as blood transfusions and iron chelation therapy are effective in managing symptoms and improving quality of life, they are not curative and require lifelong adherence [9].

Emerging treatments, including gene therapy, fetal hemoglobin induction, and bone marrow transplantation, offer hope for a potential cure and improved long-term outcomes for individuals with thalassemias [10].

Conclusion

As research continues to advance and new treatment modalities become available, it is essential to prioritize early diagnosis, access to specialized care, and comprehensive support services for patients with thalassemias. Collaborative efforts between clinicians, researchers, advocacy groups, and policymakers are crucial in advancing the field of thalassemia treatment and ultimately improving the lives of affected individuals.

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