Despite the fact that currently there is a lack of effective treatment for SCA, there are many ongoing clinical studies that contain many therapeutic agents and medications to aid SCA sufferers.
Treating the disease remains supportive but symptomatic. So for now, there are the following treatment approaches:
- Hydroxyurea: It has many satisfying results during the last 30 years, as a therapeutic solution to any patient who has been hospitalized repeatedly due to acute pain or acute chest syndrome or other conditions. Its mechanism lies in the increase of fetal hemoglobin which is connected much more easily to the oxygen and thus it reduces vaso-occlusive episodes. As for today, it is by far the best treatment with the fewer side effects.
- the tactic of blood transfusions, and/or blood transfusions and/or blood cell transfusions are used to prevent complications. In particular, red blood cell transfusion may be required as an urgent, life-saving option for the treatment of acute complications
- hematopoietic cell transplantation (HCT) is a potentially curative treatment for sickle cell disease (SCD), but is not without risks. Most blood cell transplantations that are being carried out today to SCA patients, use identical siblings as carriers and they are concerning mainly children and teenagers.
- Inflammation and oxidative stress reduction: Recently, glutamine administration as a dietary supplement has begun. L-Glutamine is an essential amino acid of the body and some research has shown that it helps to deal with oxidative stress in Sickle Cell patients, possibly contributing to fewer painful attacks, more energy and well-being and a better health and therefore quality of life. Additional studies are being carried out about its effectiveness.
- Reactivation of the fetal hemoglobin. Although it’s the main activity of hydroxyurea there are also other ways that are being investigated.
- Monoclonal antibodies are a brand new injectable treatment (approved in 28/10/2020 by FDA with conditional authorization and still subject to additional monitoring.) It is indicated for the prevention of recurring vaso-occlusive episodes in SCA patients, aged 16 and over. It may be an additional treatment in hydroxyurea/hidroxycarbamide (HU/HC) or by its own for patients whose HU/HC is inadequate.
- `Voxelotor is a regulatory factor for hemoglobin that increases the affinity between hemoglobin and oxygen, resulting in the reduction of polymerization and hem-agglutination. It is taken orally and it’s addressed to patients aged 12 and over. The first results of the most recent clinical trials of the medicine showed that it increases the hemoglobin levels and reduces hemolysis. In January 2021, FDA approved the application for the marketing authorization of this medicine.
- gene therapy, is done by transplanting stem hematopoietic cells. Studies are being carried out and so far, the results are discouraging. Furthermore, it involves high technical requirements and cost.