Sickle Cell Treatment (Treatment)

There is no standard cure for sickle cell disease, but there are treatments to help manage it. Treatments can include:

Medicine For Sickle Cell Pain

There are many painkillers for sickle cell crisis including:

Hydroxyurea (Hydrea): An oral medication that can reduce the frequency of pain crises and the need for hospital stays. It can also help improve anemia. However, it can increase the risk of infections and pregnant women should not take it.

Crizanlizumab (Adakveo): An injectable medication that can help reduce the frequency of pain crises in adults and children older than 16. Side effects can include nausea, joint pain, back pain, and fever

Voxelotor: An oral medication that can lower the risk of anemia and improve blood flow throughout the body. Side effects can include headache, nausea, diarrhea, fatigue, rash, and fever.

Non-opioid pain relievers: Most of the time, over-the-counter pain medicines like acetaminophen, aspirin, paracetamol, or ibuprofen can ease pain from an episode

Weak opioids: For mild to moderate pain, you can try codeine phosphate, dihydrocodeine, or tramadol.

Strong opioids: For moderate to severe pain, you can try morphine, oxycodone, or fentanyl.

Blood transfusion

A blood transfusion can be a life-saving treatment for sickle cell disease (SCD), but it also carries some risks. A clinician with expertise in SCD should be involved in the decision to transfer. This type of intervention is mainly done to treat serious anemia complications, after a stroke or to prevent complications before surgery.

Here, red blood cells are removed from a donor and given to the patient through a vein. Transfusion in Sickle cell disease aims to increase the oxygen-carrying capacity and decrease the proportion of sickle hemoglobin (HbS) relative to hemoglobin A (HbA) to reverse the complications of sickle cell crises. This increases the number of flexible red blood cells, which can reduce symptoms and complications.

The risk remains an immune response to the donor blood, infection, excess iron buildup, and other complications. Transfusions can be given as a top-up or exchange transfusion. The red blood cells used should be ABO compatible, Rh and Kell matched, and HbS negative.

Vaccinations

Children with sickle cell disease should have all routine vaccinations, and possibly additional vaccinations. Children under 5 years of age with SCD should also take daily penicillin or another antibiotic prescribed by a doctor.

People with SCD are considered at high risk for certain infections, such as the flu, meningitis, pneumonia, and viral hepatitis. Getting immunizations can help protect people with SCD from developing serious infections.

Several vaccinations are recommended for people with Sickle Cell disease. The following are Sickle cell vaccine names.

Pneumovax is given because it gives greater protection against a number of bacterial types that cause pneumococcal infection. The GP gives the first dose of the vaccine when the individual is around two years of age and then a booster dose is given every five years thereafter and right through adulthood.

Meningivac gives protection against meningococcus types A and C which cause meningitis. Even if a person has been given Men C which protects against meningococcus type C it is recommended that they have Meningivac before traveling to some parts of the world e.g. Africa and Asia because it will protect against meningitis A which occurs more commonly in those parts of the world.

Influenza (Flu) vaccine is recommended that people who do not have good immunity and prone to getting infections easily should be offered a flu vaccination every year. Getting the flu virus can be hazardous in those with sickle cell disease and this vaccination is highly recommended. In the UK it is offered by the GP usually in autumn or early winter, if it has not been offered contact your GP.

Hepatitis B vaccination: People can contract Hepatitis B by using contaminated needles, for example, whilst injecting illegal drugs, through sexual contact with an infected person, contamination from a blood transfusion, and less commonly from an infected mother to her unborn baby. Blood transfusion services in the UK are extremely careful to screen all blood donors for hepatitis B; this reduces the risk of contamination via that route. If an individual with sickle cell disease requires blood transfusion, it is recommended that they should have this vaccination to give them protection. If you are unsure whether you have received or need any vaccinations, please speak to your Haematology Consultant.

Antibiotics

People with sickle cell disease are more vulnerable to infections.

Most people need to take a daily dose of antibiotics, usually Penicillin prophylaxis, often for the rest of their lives. If the person is allergic to penicillin, oral erythromycin should be given.

Long-term use of antibiotics will not pose any serious risks to your health.

Gene therapies

Gene therapies are a promising treatment for sickle cell disease (SCD) that can reduce or eliminate the frequency of acute pain crises. The two gene therapies approved by the FDA in December 2023 are:

• Casgevy (exagamglogene autotemcel)Uses CRISPR gene editing to silence the BCL11A gene in red blood cells, which allows the body to produce fetal hemoglobin.
• Lyfgenia (lovotibeglogene autotemcel)Uses a harmless virus to introduce a healthy copy of the beta-globin gene into blood stem cells. 

Gene therapies for SCD work by either adding new DNA or modifying existing DNA. While they don’t correct the mutation that causes SCD, they can significantly reduce the frequency of pain crises.

Bone Marrow Transplant For Sickle Cell

A bone marrow transplant, also known as a blood and bone marrow transplant or hematopoietic stem cell transplant, is a potential cure for sickle cell disease. It involves replacing unhealthy blood-forming cells with healthy ones from a donor

A bone marrow transplant for sickle cell disease is typically only considered for children under 16 years old. This is because the transplant is usually only considered for children who have severe complications from the disease, and who have a donor with closely matching bone marrow. 

A bone marrow transplant for sickle cell disease involves:

• Treating the recipient: The recipient is given drugs to destroy their bone marrow cells. 
• Collecting the donor’s cells: The donor is given a “mobilizing” medicine to increase the number of stem cells in their blood, which are then collected. 
• Infusing the donor’s cells into the recipient: The donor’s cells are infused into the recipient’s blood, where they travel to the bone marrow and produce healthy red blood cells. 

Bone Marrow Transplant For Sickle Cell Risks

Bone marrow transplants for sickle cell disease can have several risks, including:

• InfectionThe recipient is at risk of infection while the new stem cells are growing in the bone marrow, which can be severe and sometimes fatal. 
• Graft-versus-host disease (GVHD)A serious and potentially life-threatening complication that occurs when the donor’s immune system attacks the recipient’s organs. 
• Graft failureThe transplant may not take hold in the marrow, which can be caused by infection, recurrent disease, or an insufficient number of stem cells. 
• Damage to organsThe liver and heart can be permanently or temporarily damaged by infection, GVHD, chemotherapy, radiation, or fluid overload. 
• InfertilityThe transplant can affect fertility, but newer methods are reducing this risk. 
• Seizures and bleeding in the brainThese can occur as complications of a bone marrow transplant. 
• Long-term painA transplant may not cure long-term pain associated with sickle cell disease. 

The conditioning process to prepare the recipient for the transplant can also cause side effects, such as nausea, vomiting, diarrhea, hair loss, mouth sores, bleeding, anemia, fatigue, and cataracts. 

A bone marrow transplant is the only way to cure sickle cell disease, but it’s a long process that involves several weeks in the hospital and months of follow-up care.

Other treatments include:

• Incentive spirometry to prevent acute chest syndrome (ACS)
• Bronchodilators for patients with asthma or acute bronchospasm