Sickle Cell Disease Treatments
Advancements in medical research have led to the development of targeted treatments addressing specific complications associated with sickle cell disease and offering hope for improved outcomes and better management of symptoms.
Your Personalized Sickle Cell Disease Treatment Plan
The treatment plans we offer include a range of approaches tailored to manage the complexities of this genetic disorder. From leading-edge therapies to FDA-approved interventions, our patients benefit from a multidisciplinary approach aimed at enhancing their quality of life.
Sickle Cell Disease Treatments We Offer
Montefiore Einstein offers patients access to the most up-to-date, approved therapies, including curative therapies. Our dedicated team understands the intricate nature of sickle cell disease, employing comprehensive, individualized treatment strategies to ensure optimal care and improved quality of life for individuals facing the challenges of this condition.
Preventive treatment options for sickle cell disease aim to reduce the frequency and severity of painful crises, complications and organ damage, promoting better overall health and quality of life for patients. Our experts will monitor and evaluate your:
- Diet
- Hydration
- Sleep maintenance
- Folic acid levels
- Antibiotics use
- Cardiac, eye and kidney conditions through routine screening
- Vaccinations
Hydroxyurea
Hydroxyurea has been shown to reduce the frequency of painful crises, alleviate symptoms, and improve overall quality of life by raising hemoglobin F levels and lowering neutrophil count.This medication comes in an oral tablet.
Oxbryta (Voxelotor)
Oxbryta improves anemia by modifying hemoglobin which increases oxygen binding in red cells, preventing their deformation. This medication comes in an oral tablet.
Endari (L-glutamine)
Endari is a therapeutic supplement that demonstrates effectiveness in reducing the frequency of pain crises and improving overall health outcomes by decreasing inflammation and harmful oxidation within red blood cells. This medication comes in an oral powder.
Crizanlizumab
Crizanlizum is given once a month to help prevent pain and blockages in blood vessels, especially for those whose symptoms persist despite using hydroxyurea. This is an intravenous infusion that decreases the stickiness of sickle red cells.
Blood transfusions can lead to excess iron in the body which causes additional health issues. Chelation therapy involves eliminating this surplus iron. It can be administered both orally and intravenously.
Doctors may administer simple or exchange blood transfusions to increase normal red blood cell levels in patients with sickle cell disease, easing complications like acute chest syndrome and preventing strokes. Typically lasting one to four hours, these transfusions occur every three to four weeks, with ongoing monitoring for potential complications such as allergic reactions or iron buildup. A patient may need an IV line to be placed permanently.
Our Medical Cannabis Clinic offers potential relief from pain and other symptoms associated with sickle cell disease, providing patients with alternative management options. For more information, contact our specialists for a referral.
A stem cell transplant, also referred to as a bone marrow transplant, involves replacing bone marrow affected by sickle cell anemia with healthy marrow from a donor, often a sibling. Working with the BMT Clinical Trials Network, we provide care to patients who may be best served with a transplant from a haploidentical source or for whom reduced-intensity preparation is the best course of action.
There are currently two different approaches to gene therapy that can provide a potential cure for sickle cell disease.
In stem cell gene addition therapy, a person’s own stem cells are removed, and a gene for producing normal hemoglobin is introduced. The modified stem cells are then returned to the patient through an autologous transplant. This is a favorable option for individuals with sickle cell disease who are lacking a well-matched donor.
Gene editing therapy involves modifying the DNA in a person’s stem cells to address the sickle gene, restoring the cells’ capacity to produce healthy red blood cells. This process, known as editing, occurs outside the body as stem cells are removed, treated, and then infused back into the bloodstream. While FDA-approved for individuals aged 12 and older, the long-term effects of this innovative treatment are still under investigation.
With several clinical trials in progress, we offer patients access to innovative treatments and care that may not be available elsewhere.