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

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.

Some of the currently approved treatments available today include:

• An oral medication to treat people with sickle cell disease to help increase production of a working type of hemoglobin. Doing so may help make red blood cells less likely to turn into a sickle shape and stick together to block blood flow in blood vessels.
• An intravenous (IV) infusion for patients with sickle cell aged 16 years and older that is a type of medication known as a monoclonal antibody. This medication blocks the interaction of protein called P-selectin with blood cell lining and certain blood cells, which may help reduce severe pain crises, also known as "vaso-occlusive crises" or VOCs.
• Another oral medication was approved for patients aged 4 years and older. This is a type of medicine called a hemoglobin S polymerization inhibitor. This medication works by binding to hemoglobin and increasing its affinity to oxygen to prevent it from clumping and sickling, which can help improve hemoglobin level and reduce hemolysis.
• Another oral therapy to treat patients aged 5 years and older with sickle cell disease that plays various roles in cell metabolism, including antioxidant production and effects, which can help reduce episodes of sickle-cell related pain.

Please note, this list is not comprehensive of all currently approved treatments and therapies for sickle cell disease.

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 recommended to people who are under the age of 16 and who have a matched-sibling donorMatched-sibling 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.

Gene Therapy

Gene therapy is a treatment approach that aims to treat sickle cell at the genetic, or DNA, level. Two examples of gene therapy in sickle cell are gene addition and gene editing. In both types, your own cells are collected, modified, and reintroduced to your body with the goal of providing new instructions to create hemoglobin. Learn more about gene therapy and how it works.