Sickle Cell Treatment
Sickle Cell Pharmacologic Treatments Blood Transfusions  Hematopoietic Stem Cell Transplant (HSCT) Gene Therapy

Match-related donor a donor who matches a minimum of 6 human leukocyte antigen (HLA) proteins with a transplant patient

Gene therapy a method of treating genetic diseases at the DNA level (the source) with the goal of changing the course of a disease

Learning about the current treatment options for sickle cell will help you develop a comprehensive care plan with your healthcare team. Knowing how to manage symptoms and complications can help you or your loved ones better
navigate sickle cell care.

Treatment Options

Sickle cell affects everyone differently. Current therapies mainly focus on managing the symptoms of sickle cell and require lifelong use. Recent progress in sickle cell care has led to treatment options that haven't been seen in decades and have the potential to help manage acute and chronic symptoms of the disease.

Treatment options include:

Pharmacologic Treatments

Current available sickle cell treatments mainly focus on relieving pain and managing acute symptoms and complications. They aim to lessen the frequency and severity of pain crises, reduce blood transfusions and hospitalizations, or improve hemoglobin levels. Chronic pain is commonly treated with over-the-counter pain relievers. Pain medications may also be prescribed by a doctor to treat acute and chronic pain.

Blood Transfusions

Blood transfusions can help relieve symptoms of sickle cell and potentially prevent stroke and other complications in high-risk patients. There are 2 types of transfusions used for people with sickle cell: A simple transfusion and a red blood cell exchange. In a simple transfusion, donated blood is given to a patient; in a red blood cell exchange, a patient’s red blood cells are removed and replaced with healthy red blood cells. Blood transfusions are a common procedure but risks can include an immune response to the donated blood, infection, and excess iron buildup in the body (iron overload).

Hematopoietic Stem Cell Transplant (HSCT)

An HSCT (also called a bone marrow transplant or stem cell transplant) is a type of treatment that replaces blood stem cells with cells from a donor. Bone marrow transplants are currently the only treatment that can lead to a cure; however, they are mostly limited to people who are under the age of 18 and have a Match-related donora donor who matches a minimum of 6 human leukocyte antigen (HLA) proteins with a transplant patient available. Bone marrow transplants do not treat the underlying gene mutation that causes sickle cell.

Treatment Opportunities

Significant advancements have been made in the treatment of sickle cell, but it is not enough; serious medical and social burdens remain. There is still a need for treatments that can eliminate lifelong symptom management and its unwanted side effects.

Research in Sickle Cell Disease

Scientists continue to explore how to address unmet needs in the management and treatment of sickle cell. As of February 2021, there have been more than 350 clinical studies on sickle cell disease completed, with over 250 active or actively recruiting trials happening to date. From collecting information about diagnoses to improving current treatments and exploring new options, scientists aim to improve care for sickle cell every day.

Improving and Developing Therapies

Pharmacologic treatments

Scientists are continuing to explore ways to expand available pharmacologic treatments to target and address symptoms and complications of sickle cell throughout the body. There are many different ways in which these treatments work and what they target in the body. Some methods being studied include treatments that aim to:

stabilize sickled red blood cells to try to prevent them from breaking apart (hemolysis)
reduce the frequency and effects of vaso-occlusion by impacting how different cells bind and stick to one another
decrease the inflammation caused by damage from sickled red blood cells that contributes to organ damage

HSCT

Scientists are working to develop alternative approaches to help improve safety and reduce the transplant-related risks involved with HSCT, such as finding a less toxic regimen. They are also investigating how to expand the amount of donors available to a patient, as having a matched-related donor is a limitation for this treatment. As of February 2021, there were about 60 active or recruiting trials studying HSCT in patients with sickle cell disease.

Gene therapy

Gene therapya method of treating genetic diseases at the DNA level (the source) with the goal of changing the course of a disease is a type of treatment that uses genetic material with the goal of changing the course of a disease, and it has been studied for more than 40 years. It is a therapeutic approach that is being investigated for the treatment of multiple diseases. Although many gene therapies are currently in early research or clinical trials, some have already been approved by the US Food and Drug Administration. To date, there are no approved gene therapies for sickle cell.

Overall, there are two types of gene therapy being studied: gene addition and gene editing. Both of these types of gene therapy are being explored in sickle cell. There are several techniques within these types that aim to give the cell a new set of instructions and treat the disease at the genetic level. In sickle cell, this can include:

adding a gene for a functioning form of hemoglobin (Hb) that can compensate for dysfunctioning hemoglobin S (HbS)
turning on or off a different gene that regulates the type of Hb the body produces
correcting the Hb gene mutation so the body produces a functioning form of Hb instead

In any type of gene therapy, the genetic material needs a way to get inside the cells of the person with the disease. This happens with a delivery system called a vector and can be done either inside the body (in vivo) or outside (ex vivo).

As with any treatment, there are risks associated with gene therapy. Learn more about the different techniques and how gene therapy works by exploring the following resources:

genehome: a source for comprehensive and objective information about gene therapy, from beginners to advanced learners
American Society of Gene and Cell Therapy: provides information on science, technology, as well as gene and cell therapy for patients and their families
National Human Genome Research Institute: an organization that collaborates with the scientific and medical communities to provide the latest information about the human genome
oneSCDvoice: a sickle cell–specific platform aiming to connect the sickle cell community and provide educational resources, including a gene therapy education center

Discussing your needs with your doctor is a great way to strengthen your partnership and can help you create a long-term care plan to meet your goals. Don’t be afraid to ask your doctor about future treatments opportunities for sickle cell—they may be able to provide you with more information about these options.

Screening and data collection

The Centers for Disease Control and Prevention (CDC) has partnered with other groups to help enhance newborn screening programs to include sickle cell screening throughout the United States. This initiative aims to improve screening results to help patients and their families get connected to healthcare resources to better manage their conditions and improve long-term health. The CDC has also created a data collection program to study long-term trends in diagnosis, treatment, and healthcare access for people with sickle cell.

Healthcare worker training

In 2014, the CDC began working with partners to better understand health issues that can occur for patients receiving blood transfusions. In addition to improving overall quality and length of life, the program aimed to develop training on transfusion guidelines for healthcare providers. In 2020, the Department of Health and Human Services launched the Sickle Cell Disease Training and Mentoring Program (STAMP) to train healthcare workers in sickle cell care and promote physicians and specialists working together to support their patients.

The Sickle Cell Disease Association of America offers a Sickle Cell Disease Community Health Worker Training program. This online course offers a concentration in sickle cell disease in addition to covering the core competencies of community health workers.

Photo of Cory, who lives with sickle cell, discussing the importance of being proactive

Be proactive. Don’t wait. We must remember to always LISTEN to our bodies. Such as knowing your hemoglobin levels, and how you feel they fluctuate. Because not knowing, and just waiting till something tragic happens could be the difference in a warriors' life.”

CORY

Living With Sickle Cell

What Can You Do?

Building a comprehensive healthcare team that focuses on specialists from different areas may help you and your loved ones manage your acute and chronic complications before your complications progress. It’s important to work with your care team to design a plan that works for you.

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