MANAGEMENT anemia.

Earlier transfusion with HSCT can attain higher

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