Finding the right sickle cell disease (SCD) care isn’t always simple. Some people have trouble reaching specialists. Others have had their symptoms dismissed or treatment delayed.

Treatment isn’t one-size-fits-all. Some treatment options lower the chance of pain crises or other complications. Others help prevent stroke, fight infection, or offer a potential cure.

This article covers six types of SCD treatment options and what each may involve.

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Treating Sickle Cell Disease

A treatment plan for SCD may include three types of care: supportive care, medicines that change how the disease acts, and treatments that aim for a cure.

• Supportive care — These treatments help prevent or manage problems. They can include vaccines and antibiotics to prevent infection, regular checkups, and pain management.
• Disease-modifying treatment — These treatments can reduce sickling, pain crises, and other complications.
• Curative-intent treatment — These treatments try to replace or change the blood-forming cells that cause SCD. These include stem cell transplants and gene therapy.

Disease-Modifying Treatments for Sickle Cell Disease

These four treatments act on different aspects of sickle cell disease to help limit or manage pain, complications, blood cell damage, and the need for hospitalization.

Hydroxyurea helps the body make more fetal hemoglobin, which makes red blood cells less likely to sickle. It can prevent pain crises, acute chest syndrome (a serious lung complication), hospital stays, and the need for transfusions.

Children and adults may use hydroxyurea. Some babies start taking it at 9 months old. It’s usually taken once a day as a capsule, tablet, or liquid.

Hydroxyurea can lower blood cell counts, so you’ll need regular blood work. Possible side effects include headache, nausea, and low appetite. Call your care team if you develop a fever or signs of infection.

This medication can harm a fetus. If you or your partner can become pregnant, ask your care team about contraception and pregnancy precautions before starting hydroxyurea.

L-glutamine, sold as Endari, is an amino acid powder. It may help limit red blood cell damage and reduce pain crises and acute chest syndrome.

Endari can be used starting at age 5 and older. Each dose is mixed with food or a drink and taken twice daily. Some people take it as well as hydroxyurea.

Possible side effects include nausea, tiredness, chest pain, and bone or muscle pain. Tell your care team if a symptom is severe or doesn’t improve.

Crizanlizumab, sold as Adakveo, blocks a protein called P-selectin. This makes platelets and white blood cells less likely to stick to the inside of blood vessels and cause a painful blockage. It’s approved for people aged 16 and older.

Crizanlizumab is infused through a vein. After the first dose, the next visit is two weeks later. Later doses are usually given every four weeks. The infusion itself lasts about 30 minutes, and your healthcare team will watch for a reaction.

Possible side effects include fever, chills, nausea, dizziness, trouble breathing, or worse pain during or after treatment. These can be signs of an infusion reaction, and you should tell your healthcare provider if you notice them.

A transfusion adds red blood cells with normal hemoglobin A to your blood. This can improve anemia (a low red blood cell count) and lower the share of sickled cells in the blood for a while. The blood is given through an IV, and the visit takes between two and five hours.

A transfusion may be used to treat severe anemia or acute chest syndrome, before surgery, or to help prevent strokes. Some people may need just one transfusion to treat a new problem. Others receive them on a regular schedule.

Regular transfusions can cause iron overload, which can harm organs over time. You may need tests to check your iron levels and medicine to remove extra iron.

Your care team will watch for transfusion reactions and alloimmunization, when your immune system makes antibodies against donor blood. This reaction can make it harder to find blood that is a good match for you.

Tell your care provider right away about fever, chills, itching, chest or back pain, or trouble breathing.

Curative-Intent Treatments for Sickle Cell Disease

The next two options aim to stop SCD at its source. They’re much more involved than taking a daily medicine or getting a monthly infusion, and deciding whether to pursue them can bring up fear, hope, and uncertainty.

These treatments may involve chemotherapy, fertility concerns, a hospital stay, months of follow-up, insurance or travel barriers, and questions about what life may look like afterward.

A stem cell transplant, or bone marrow transplant, replaces the blood stem cells that produce your blood with blood stem cells from a suitable donor. Treatment works best when the donor is a close match. A fully matched brother or sister is usually the best option.

Transplant is considered more often for people with severe SCD, such as after a stroke or repeated serious complications. Age, organ health, and the availability of a matched donor also help shape the decision.

Before transplant, you’ll receive conditioning treatment, which may include chemotherapy or other medicines to make room for the donor cells and suppress your immune system. The transplant is followed by a hospital stay, and recovery can last for months.

Risks include infection, stem cell rejection, and graft-versus-host disease, which means the cells from the donor attack your body. Call your transplant team right away if you have fever, rash, diarrhea, or yellow skin or eyes.

Gene therapy starts with your own cells rather than cells from a donor. The care team collects your blood stem cells, the cells are modified in a lab, and chemotherapy makes room in your bone marrow before the modified stem cells are infused back.

Because the cells are collected from you, a match isn’t needed and there is no risk of graft-versus-host disease.

The process can take months, and it involves the risks of strong chemotherapy. Gene therapy may be an option for someone who lacks a donor match, but it requires care at a specialized center and a hospital stay of four to six weeks.

It’s also important to know that gene therapy may reduce or stop some major SCD symptoms, but it may not erase every health issue a person has developed over time. It’s reasonable to ask what the treatment can and can’t change before deciding whether it feels right for you.

Casgevy

Casgevy uses CRISPR gene editing. The treatment helps the blood stem cells to make more fetal hemoglobin, which can keep red blood cells from sickling. It may be used for people ages 2 and older who have had repeated sickle cell crises.

Treatment includes stem cell collection, lab work, powerful chemotherapy, a one-time infusion, and then recovery for around four to six weeks in the hospital.

Common serious complications include:

• Low blood counts requiring frequent transfusions of red blood cells and platelets
• Painful mouth sores
• Fevers
• Serious infections
• Bleeding

Call your care team right away if you experience fever, chills, unusual bleeding, or a severe headache. Long-term follow-up is needed for at least 15 years after taking Casgevy because gene editing is still a new treatment.

Lyfgenia

Lyfgenia adds genetic material to blood stem cells collected from you. The modified cells then make a form of hemoglobin that’s less likely to sickle. It may be used for people age 12 and older who have had vaso-occlusive events, including pain crises.

Treatment involves collecting cells, giving powerful chemotherapy, returning the modified cells in a one-time infusion, and then recovering in the hospital for around four to six weeks.

Lyfgenia carries the strongest safety warning from the U.S. Food and Drug Administration (FDA) because a few people who took it later developed blood cancer. After taking Lyfgenia, you may need follow-up monitoring for signs of blood cancer throughout your life.

During recovery, fever or unusual bleeding needs quick medical attention. Ask the gene therapy team about the side effects, recovery time, and the risk of reduced fertility.

Which Treatment Is Best for You?

The best treatment option depends on your age, the type of SCD you have, any SCD complications, and your current health, support system, and goals. A daily medicine or a months-long hospital treatment can affect your life in very different ways.

Access to sickle cell care can also shape the decision. People with SCD may face access barriers, insurance delays, long travel, or increased time away from work or school. Those concerns can be especially important with transplants and gene therapy.

Bring your questions to your hematologist or sickle cell care team. Ask what symptoms the treatment may improve, how long it takes, what monitoring you’ll need, and which risks matter most for you. It’s also reasonable to seek a second opinion.

Your needs may change, and treatment choices can be revisited. Learning what causes SCD may help you prepare questions.

New Research for Sickle Cell Disease

Researchers are testing new medicines, transplant techniques, and ways to edit genes to help treat SCD. Clinical trials may offer treatments that aren’t otherwise available, although their benefits and risks may still be uncertain. Your care team can help you decide whether a trial is worth exploring.