- Anemia is a lack of hemoglobin in blood. Having anemia can
makes feel tired and weak.
- There are many forms of anemia each with aits own cause ,
sickle cell anemia is one of them.
- Sickle cell disease is a hemoglobinopathy which affects
approximately 100,000 individual in United States.
- It is caused by point mutation in
- globin gene resulting in synthesis of pathological hemoglobin
S(HbS).
- Pathophysiological studies shown dense dehydrated red cell play
a significant role in acute and chronic clinical manifestations of
SCD.
- This is due to modulation of innate immunity and increased iNKT
lymphocytes, increase level of vascular active cytokine such as
endothelium combined with the final contribution of platelet.
- Hydroxyurea is the gold standard treatment for SCD.
- Various option for treatment are :
- Molecule targeting hemolysis induced vasculopathy.
- Agents that modulate the abnormal tone.
- Agents interfering with red cellvascular adhesion event.
- Molecules interfering with the physical properties of the red
cell endothelial adhesion process. Molecules specifically
interfering with sickle cell endothelial adhesive mechanism.
Molecule affecting platelet function. These are still under
progress.
- N- acetyl cysteine an exogenous thiol donor.
In the last two decades, progress in hemopoietic stem cell
transplantation strategies have allowed to offer a new curative
option.
Development of CRISPR/Cas9 genome editing has been reported to
represent a new potential therapeutic tool.
A new field of combination therapy require a holistic approach
considering the improvement of patient quality of life.
( Matte A et.al.2018.New therapeutic options fro the treatment
of sickle cell disease. MJHID;11:1-12)