Understanding the Goals for Treating Sickle Cell Disease
- The main goals when treating sickle cell disease (SCD), no matter the type, are to reduce pain and organ damage.
- For some types of the disease, a drug called hydroxyurea can be used to decrease pain, lower the risk of a complication known as acute chest syndrome, and reduce the need for blood transfusions.
- In recent years, more drugs have come on the market to treat SCD and reduce symptoms and complications.
- Other possible treatment options include pain medications, blood transfusions, vaccines and antibiotics, folic acid, and lifestyle interventions like drinking more water and managing stress.
“Our goal is to prevent these episodes of pain … and the other is to protect organs over the long-term,” Dr. Sophie Lanzkron, Director of the Sickle Cell Center for Adults at Johns Hopkins, tells SurvivorNet.
Read MoreTreatment Options for Sickle Cell Disease
Dr. Lanskron explains that the standard therapy for SCD, which has been around for many years, is hydroxyurea (sold under brand names Hydrea and Droxia). The drug was approved by the Food and Drug Administration in 1998, according to Dr. Lanskron, and it can be used for several types of SCD.Hydroxyurea works by helping red blood cells stay rounder and flexible, which can help:
- Reduce the number of sickle cells in the blood
- Decrease pain
- Lower the risk of acute chest syndrome (a possible SCD complication where sickle cells stick together and block the flow of oxygen in vessels in the lungs)
- Reduce the need for blood transfusions
This in turn reduces the numbers of doctor or hospital visits and improves quality of life for patients.
“It’s really a potentially life-transforming drug,” Dr. Lanskron says.
Three other drugs L-glutamine (such as Endari), crizanlizumab (Adakveo), and voxelotor (Oxbryta) have been approved more recently to treat SCD.
According to Hopkins Medicine, other potential treatment options include:
- Pain medications
- Staying well hydrated (to help prevent pain crises)
- Blood transfusions
- Vaccines or antibiotics (to prevent infection)
- Folic acid
- Regular eye exams
- Bone marrow transplant
Stress Management and Sickle Cell Disease
Dr. Lanskron highlights that it is also crucial for patients with SCD to manage their stress levels. This is because stress can lead to extreme episodes of pain (also known as a pain crisis).
“The goal of the therapy is to … try to get people to live with life’s stressors and still be able to do the daily things,” without having a pain crisis, she explains.
SurvivorNet has mental health resources tools specifically designed to help people struggling to cope with emotions while also dealing with a health condition.
Questions to Ask Your Doctor About Sickle Cell Disease
If you’ve been diagnosed with sickle cell disease, here are some questions you may consider asking your doctor to help understand your situation:
- Which SCD drug works best with my specific type and condition?
- What lifestyle changes should I make to help keep my SCD symptoms under control?
- Is there anything I should avoid doing because of my condition?
- What resources can I use to help manage stress?
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