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Management: Hematopoietic Stem Cell Transplant Gene Therapy

The document discusses treatment options for beta thalassemia major, highlighting standard therapies such as blood transfusions, iron chelation, and hematopoietic stem cell transplant (HSCT). HSCT is noted as the only curative treatment, improving health-related quality of life for patients, while gene therapy shows promise in providing a permanent erythropoietic effect. However, concerns regarding long-term risks associated with gene therapy and HSCT complications are also mentioned.

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Amanda Scarlet
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103 views1 page

Management: Hematopoietic Stem Cell Transplant Gene Therapy

The document discusses treatment options for beta thalassemia major, highlighting standard therapies such as blood transfusions, iron chelation, and hematopoietic stem cell transplant (HSCT). HSCT is noted as the only curative treatment, improving health-related quality of life for patients, while gene therapy shows promise in providing a permanent erythropoietic effect. However, concerns regarding long-term risks associated with gene therapy and HSCT complications are also mentioned.

Uploaded by

Amanda Scarlet
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
Available Formats
Download as DOCX, PDF, TXT or read online on Scribd

MANAGEMENT anemia.

Earlier transfusion with HSCT can attain higher


combined with iron chelation health-related quality of life
Standard therapy includes blood therapy was recommended as a (HRQoL) than that with
transfusions, folic acid, iron treatment of choice to reduce late conventional treatments.
chelating agents, and complications. Moreover, transplanted patients
hematopoietic cell transplant. have similar HRQoL with the
Treatment choices depend on the Hematopoietic Stem Cell general population.
patient’s clinical manifestation Transplant
Major medications Gene Therapy
 Iron chelating agents Hematopoietic stem cell
 Antipyretics transplant (HSCT) is currently the Gene therapy holds promise
 Vitamins only curative treatment for beta repairing one’s own bone marrow
 Hydroxyurea thalassemia major. HSCT cells by transferring the normal b-
 Antihistamines replaces the impaired endogenous globin or g-globin gene into
 Vaccines hematopoietic cells with effective hematopoietic stem cells (HSCs)
Blood transfusion allogeneic alternatives to to provide permanent
permanently produce normal red erythropoietic effect. However,
Blood transfusion indication is blood cells. Transplanted patients the oncogenic risk of lentiviral
mandatory for patients with beta do not need blood transfusion and vector integration is a long-term
thalassemia major but is still avoid consequent iron concern. A recent study reported
controversial for whom with accumulation complications. On safe mobilization of CD34+ cells
intermediate. Though blood the other hand, side effects after in adults with beta thalassemia
transfusion can prevent serious HSCT include graft-versus host and validated the effectiveness of
complications, it increases diseases, infections related to globin gene transfer for clinical
morbidity due to accumulation of abnormal immune reconstitution, investigation. For the first time, a
transfused complications. The tissue dysfunction, secondary patient with severe beta-
common approach is to delay cancers, and changes in quality of thalassemia has been independent
transfusion until the patient life.In the long-term, beta from transfusion for 5 years after
demonstrates complications of thalassemia major patients treated gene therapy.

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