MANUSCRIPT
Dear Editor,
We have read the article entitled ”Trends in blood transfusion,
hydroxyurea use, and iron overload among children with sickle cell
disease enrolled in Medicaid, 2004–2019” by Amy et al. [1] with
great interest and appreciate the effort put into the research. However,
upon reviewing the manuscript, we have identified a few ideas that we
believe are worth discussing to enhance the overall quality of the
article. The study’s results indicate that hydroxyurea is effective in
reducing hospitalization rates, inpatient days, pain events, ER visits,
episodes of acute chest syndrome, and transfusions, and improving
hemoglobin levels. The research also demonstrates that hydroxyurea has a
greater impact on younger children than older ones and that the change
in Hb F levels varies in different studies. Despite some limitations,
the study provides valuable information on the effectiveness of
hydroxyurea in treating Sickle Cell Anemia (SCA) in real-life settings.
To enhance the current predominant treatment, additional therapeutic
choices such as l-glutamine, crizanlizumab, and voxelotor have become
accessible in addition to hydroxyurea. Hematopoietic Stem Cell
Transplantation (HSCT) with fully matched donors is regarded the most
effective treatment for SCD patients who are unresponsive to
hydroxyurea. [2]
In addition, an ongoing phase 1-2 trial of LentiGlobin gene therapy for
Sickle Cell Disease involves transplanting autologous hematopoietic stem
cells transduced with a lentiviral vector carrying a modified -globin
gene, resulting in the synthesis of anti-sickling hemoglobin. The study
included 35 individuals who had at least four severe vaso-occlusive
episodes within the previous 24 months. Engraftment happened in all
patients, with the majority of red cells continuing to produce HbAT87Q,
resulting in less hemolysis and complete remission of severe
vaso-occlusive disease. [3]
In conclusion, the article by Amy et al. provides valuable insights into
the effectiveness of hydroxyurea in treating sickle cell anemia in
real-life settings. However, with the emergence of additional
therapeutic options such as l-glutamine, crizanlizumab, and voxelotor,
as well as hematopoietic stem cell transplantation and gene therapy, the
current predominant treatment for sickle cell anemia can be enhanced.
These new treatments have shown promising results in reducing the
frequency and severity of vaso-occlusive episodes and improving overall
outcomes for patients with sickle cell disease. Future research should
focus on evaluating the long-term efficacy and safety of these therapies
and identifying the most appropriate treatment options for different
patient populations.