Treatment for Sickle Cell Disease

As a person living with sickle cell disease, your needs and the effects of your disease will change over time. Your care should change, too. The most important step you can take is to partner with health care providers who will plan treatment that is right for you right now. Individualized care helps you live a full, active and healthy life.

At Fred Hutch Cancer Center, we believe sickle cell disease does not have to define who you are or what you can do. Our sickle cell specialists work closely with you, your family and each other to understand and meet your needs. We provide all standard therapies. We also offer you access to the latest treatments through clinical trials. 

Our doctors provide the full range of sickle cell disease treatments. Based on your exact case, your treatment may include medicines, like hydroxyurea, crizanlizumab, L-glutamine and voxelotor, and blood transfusions. For some patients, a blood and marrow transplant may be an important option. Your Fred Hutch team works to reduce your risk for painful episodes and make sure you get the care you need when you do have pain. We also help to deal with any other symptoms you have, maintain your health and prevent or manage complications.

As you go through treatment, your needs may change. Your care team at Fred Hutch is with you each step of the way. For example, we will help you deal with any side effects you have. We may suggest adding a new therapy that was just approved. To protect your health over the long term, we will keep seeing you for as long as you need us.

Hear from Kleber Yotsumoto Fertrin, MD, PhD, the director of the Fred Hutch Sickle Cell Disease and Iron Overload Program, as he shares why it is so important to get individualized care.

Hear from Kleber Yotsumoto Fertrin, MD, PhD, the director of the Fred Hutch Sickle Cell Disease and Iron Overload Program, as he shares the services offered at the Sickle Cell Disease Program.

“With the array of medications that we have available, no sickle cell patient should be stuck with the same treatment for their whole life. We can always do better. We can always adjust over time to meet their changing needs.”

— Kleber Yotsumoto Fertrin, MD, PhD, hematologist

Treatment Plan

Sickle cell disease treatment is highly customized for each patient’s needs.

How Do We Create Your Treatment Plan?

Your Fred Hutch hematologist works with an entire group of physicians who specialize in blood disorders, like sickle cell disease. Together, they hold sickle cell rounds — meetings to discuss their patients’ treatment plans. If your care team is thinking about a blood or marrow transplant for you, they may meet with other types of team members to talk about this option, like a radiation oncologist and pathologist.

This approach means you benefit from the experience of the whole group.

With support from the larger team, your physician will:

  • Talk about all possible treatments for your situation
  • Decide which therapies will be most promising for you
  • Check if any clinical trials match your needs, so you can think about joining them

Your care team will walk you and your caregiver through the treatment plan we recommend for you. You will have a chance to share your personal preferences, and you will decide together what happens next.

Why Do Treatment Plans Differ?

The treatment plan we design for you depends on many things, including:

  • Your age
  • Your lifestyle, like your job and the activities you enjoy 
  • Your symptoms and how serious they are
  • How your disease has changed over time
  • If you have any complications of sickle cell disease
  • Your overall health
  • Your needs and preferences, like what type of treatment schedule works in your life and if you want to join a clinical trial

Factors like these help us make decisions about the standard therapies and clinical trials that are right for you. 

What Is The Standard Therapy For Sickle Cell Disease?

Because sickle cell disease can range from mild to severe and it can change over time, there is no single way to treat everyone. However, most people get medicines to prevent sickling (when red blood cells become crescent-shaped), to prevent or reduce pain or to manage other effects of the disease. Transfusions of healthy red blood cells are another common treatment. For people seeking a cure, a blood or marrow transplant may be an option.

At Fred Hutch, our standard always involves caring for you as a whole person. We help you get relief from side effects and provide many other forms of support, like integrative medicine, nutrition counseling and physical therapy. We also make sure you have a team of specialists to check for, prevent and manage complications that can affect different parts of your body, like your eyes, heart and kidneys.

Our patients can choose to have promising new therapies that you can get only through a clinical trial. Many people come to Fred Hutch to be part of these studies. Your care team will tell you about studies that might be right for you, so you can think about joining them.

Treatment Process

Each person with sickle cell disease is unique. We choose, combine and schedule your therapies based on what works for your needs. Your care team makes sure you understand each type of treatment and all of your choices.

Acute Chest Syndrome Treatment

Acute chest syndrome is a lot like pneumonia. It involves chest pain, coughing, breathing trouble and fever.

If you get acute chest syndrome, your team will choose treatments based on the cause. Common treatments include getting oxygen, taking medicine to fight an infection or open your airways and having a blood transfusion. 

Infection Prevention and Treatment

Sickle cell disease increases your risk of infections, like flu, hepatitis, meningitis and pneumonia.

Special attention to steps like washing your hands, handling food safely and getting recommended vaccinations can help prevent infections. If you do get an infection, it’s important to see your care team right away for treatment, like antibiotics.

Hydroxyurea

Hydroxyurea is a medicine taken every day to reduce pain episodes, recurrence of acute chest syndrome and the need for blood transfusions or hospital stays.

To make sure you are getting the right amount of hydroxyurea, your care team will check your level of blood cells (blood counts) from time to time. They will adjust your dose as needed.

L-glutamine

This oral medicine (taken by mouth) may help reduce the number of sickle cell crises you have.

It may make your red blood cells more flexible so they can flow through your bloodstream more easily.

Treatment for Hand-Foot Syndrome (Dactylitis)

Hand-foot syndrome is swelling of the hands and feet from blocked blood vessels.

You might also hear this syndrome called “dactylitis,” which refers to inflamed fingers or toes. You might have a fever as well. Pain medicine and getting more fluids are the main treatments. 

Blood Transfusions

Getting a red blood cell transfusion or red blood cell exchange can help with anemia or other effects of sickle cell disease.

Anemia can make you tired, irritable, dizzy and lightheaded. It can lead to a fast heart rate, breathing trouble and jaundice, and it can make your skin paler. In children, anemia can slow growth and delay puberty.

A red blood cell transfusion gives you healthy red blood cells from a donor. It can temporarily provide what your body needs to fight severe anemia. It may also help with other life-threatening effects of sickle cell, like acute chest syndrome or buildup of blood in the spleen (splenic sequestration). 

We offer red blood cell transfusions at our outpatient clinic on Lake Union. If you need a red blood cell exchange (to remove your red blood cells and replace them with healthy donor cells), our patients get this type of care at UW Medical Center – Montlake. 

Crizanlizumab

If you often have pain episodes, your doctor may recommend the medicine crizanlizumab.

The most common problem from sickle cell disease is pain from decreased oxygen to tissues in the body. It tends to flare up in what physicians call “pain episodes” or “crises.” Episodes can come on suddenly, be mild to severe and last for any length of time.

Crizanlizumab may help you have fewer pain episodes because of sickled red blood cells blocking the flow of blood. These are called vaso-occlusive crises, or VOCs. They can lead to other health problems, like damage to your organs. 

This medicine is given by infusion. For an infusion, liquid medicine is put into a vein through an intravenous (IV) line. You will get infusions in a special area of the clinic. Nurses who are experts in infusions give you these treatments. They also watch over you during the treatment. They will help with any medical issues that come up and will keep you comfortable.

Usually, the first two infusions are given two weeks apart. Then, you will get an infusion every four weeks. 

Blood or Marrow Transplant

A blood or marrow transplant resets your body’s ability to make healthy blood cells. Researchers at Fred Hutch pioneered this form of treatment.

So far, a blood or marrow transplant is the only treatment that may cure sickle cell disease. It is an important option for some adults, but it does have risks and side effects. 

Physicians at the Fred Hutch Blood and Marrow Transplant Program have done more than 17,000 transplants, including for people with sickle cell. 

We can talk with you and your family about the benefits and risks, if we recommend a transplant for you and why, and what to expect. Our transplant specialists will answer your questions and help you decide what’s right for you. 

If you have a transplant, a team of Fred Hutch transplant experts will care for you. Your team will include a transplant oncologist, transplant nurse, radiation oncologist, advanced practice providers, pharmacist, registered dietitian, patient care coordinator and social worker.

Doctors and researchers at Fred Hutch pioneered blood and marrow transplants decades ago. Today, we continue to improve transplant techniques and to develop new options.

Learn more about Blood and Marrow Tranplants

Pain Prevention and Management

Over-the-counter medicines, like ibuprofen or acetaminophen, may be enough to control pain from sickle cell disease. But sometimes you may need stronger opioid medicines to take at home or even during treatment at the hospital.

Your Fred Hutch team works to make sure you get the care you need for pain. We have an expert Pain Clinic team, including a dedicated nurse to help people with blood disorders. We can also help you learn to reduce your risk for painful episodes by avoiding triggers, like low fluid intake and extreme changes in temperature.

In the U.S., people with sickle cell disease are usually African Americans, who may have experienced unequal treatment for their health needs, especially their pain. For example, research shows that African Americans tend to receive less treatment for pain overall than white people do, even if they report more pain. Also, physicians may be more likely to see African Americans who say they are in pain as drug-seeking, even though white people are more likely to misuse opioids.1

At Fred Hutch, our sickle cell team is working to end health disparities and improve care for everyone with sickle cell disease by teaching other health care providers about these issues. With colleagues at UW School of Medicine, our sickle cell experts and some patient-advocates talk with medical students about the racial and socioeconomic inequities faced by some people with sickle cell disease. Together, they teach young medical students to care for all people who have sickle cell disease with compassion and respect.

1. Pain and Ethnicity, Virtual Mentor/AMA Journal Ethics.

Monitoring Your Health

Your sickle cell disease care team will see you regularly for exams and tests to check:

  • How well your treatment is working
  • If there is any reason to change your treatment
  • If you have any new problems with your spleen, liver, heart, kidneys, lungs, eyes or other parts of your body
  • If you need help with side effects or supportive care services, like nutrition care or mental health counseling

We update your treatment plan based on the best scientific evidence as well as how your disease responds and what you prefer.

Checklist for People with Sickle Cell Disease

You can take many steps to stay as healthy as possible with sickle cell disease. One of the most important steps is to work closely with your care team. Look over this checklist, and talk with your team about each recommendation.

Check Your Hydroxyurea Dose

Your team will check your blood counts (level of blood cells) from time to time to see if you are taking a safe dose of hydroxyurea. If your blood counts (especially neutrophils and platelets) get too low, your team will reduce your dose. The best dose decreases your blood counts a lot, but not too much. Taking the best dose can help prevent pain crises and keep you out of the hospital.

What you can do:

  • Take your hydroxyurea and folate every day as prescribed.
  • See your health care provider as often as they recommend to check your blood counts.

Keep Your Vaccines Up to Date

Because of sickle cell disease, your spleen might not work well, or it might have been removed. This puts you at higher risk for infection. To help protect you, we recommend getting certain vaccines.

Ask your health care provider if you need these or any other vaccines:

  • Influenza vaccine (flu shot) — every year
  • Pneumococcal polysaccharide vaccine (PPSV23 or Pneumovax) for pneumonia — 2 doses, 5 years apart
  • Meningococcal vaccine for meningitis — 2 doses, 2 months apart, followed by boosters every 5 years
  • Haemophilus influenzae type B (Hib) vaccine for meningitis — 1 dose
  • Tetanus booster — every 10 years
  • Hepatitis B vaccine — 3 doses
  • COVID-19 vaccine — 1–2 doses, depending on the brand, followed by boosters

Get Screened for Complications

Sickle cell could be harming your body in ways you don’t feel yet. Screenings can check for signs now that could turn into big problems later. This gives you the chance to get care to help prevent more damage. 

Ask your care team about these screenings for people with sickle cell disease:

  • Eye exams — every year to check for sickle cell retinopathy.
  • Lung function tests — to tell us if your body is getting enough oxygen. If it’s not, you could get oxygen to use at home.
  • A sleep study — for some people. If you have sleep apnea, your body doesn’t get enough oxygen at night, which can make sickling worse. A special device can help you get more oxygen while you sleep.
  • Echocardiogram (heart ultrasound) — to tell us how your heart is working and if you are getting pulmonary artery hypertension, which is a serious complication. 
  • Urinalysis — to check for protein in your urine. This helps us know if your kidneys might be injured.
  • Ferritin — a blood test to check for a high level of iron. If you’ve had more than 10 transfusions in your life, you are at risk for iron overload, which can harm your liver.
  • MRI — if your ferritin is high or if you have had iron overload. It shows how much iron is in your liver and if you need a treatment called chelation to remove some iron.

Have Routine Wellness Care

All adults need routine wellness care, like regular check-ups and tests to check for health problems before they cause symptoms.

  • See your health care provider as often as they recommend for routine wellness care.

Managing Side Effects

You might be wondering about possible side effects from treatment, like nausea from hydroxyurea or L-glutamine or iron overload from blood transfusions. 

You are always at the center of everything we do. Sickle cell physicians, nurses and advanced practice providers are here to help prevent or relieve side effects of treatment.

Get Help with Side Effects

With any treatment, we talk with you about what to expect, based on your treatment plan, and what can help if you do have side effects. We will work closely with you to make sure that side effects are managed.

At your appointments, we want you to tell us about any side effects you are having. If you have questions or concerns between appointments, you can call or email us. We will make sure you know how to reach care providers at Fred Hutch after hours, if that is when you need us.

We have many tools to help you feel better, such as:

  • Nutrition care and medicines to help with digestive problems
  • Antibiotics and antiviral drugs to prevent or treat infections
  • Medicines to remove excess iron (iron chelation therapy)
  • Conventional and integrative therapies for pain

Common Side Effects

Side effects are different depending on which treatment you get. These are some of the common side effects of sickle cell disease treatment:

  • Problems in your digestive tract, like nausea or constipation
  • Low levels of blood cells if your hydroxyurea dose is too high
  • Iron overload from having many blood transfusions
  • Developing antibodies to donor red blood cells (alloimmunization) 

Supportive Care Services

Along with treating your sickle cell disease, Fred Hutch provides a range of services to support you and your caregiver. This is part of how we take care of you — not just your disease.

From registered dietitians to social workers and spiritual health staff, we have experts who specialize in caring for people with blood disorders or cancer. We understand that living with sickle cell disease can be an intense and challenging experience for you and your family. We are here to provide the care you need. 

Learn more about Supportive Care

Caregiving During Treatment

If your loved one is having treatment for sickle cell disease, there are many ways you can help. Caregiving often means doing tasks like these:

  • Keeping track of their appointments and driving them to and from treatment
  • Watching for changes in their condition and telling their care team about any symptoms
  • Providing physical care, like helping them take medicines
  • Spending time with them and encouraging them
  • Taking care of things at home that they may not be able to do, like grocery shopping and cleaning

Caregiving for Transplant Patients

Caregivers have a special role in blood and marrow transplants. This intense treatment involves chemotherapy (and sometimes radiation) with serious side effects. During the initial recovery period, which often takes at least a month, your loved one will need daily help. We have classes to help transplant caregivers get ready. During recovery, a transplant registered nurse is available by phone 24 hours a day, 7 days a week, to help you.

Continuing Care

Throughout treatment, your team provides follow-up care and support on a schedule designed for you. We understand that your disease and treatment might impact nearly every aspect of your life. We’re here to help you cope with the physical, practical and emotional effects. 

At follow-up visits, you will see the same Sickle Cell Disease Clinic team. If you do not live close to Fred Hutch and you prefer to travel here less often, we will work with your local health care providers to make sure you get the follow-up care you need. 

For people who had a blood or marrow transplant, the Fred Hutch Long-Term Follow-Up Program provides lifelong monitoring and care. 

Schedule For Follow-up Visits

Just like we personalize your treatment plan for you, we personalize your follow-up schedule, too. Usually, people taking hydroxyurea have visits at least four times a year. People on crizanlizumab are seen once a month before their infusion. If you need a high dose of pain medicine, your team may want to see you every two weeks to check how well your pain is being managed. Your hematologist will base your schedule on many factors, including how your disease and the treatments are affecting you.

What Happens at Follow-up Visits

Follow-up for sickle cell disease usually means seeing your hematologist for a physical exam and having blood tests. It’s also important to see specialists who can check other parts of your health, like your eyes, lungs and heart.

Together, you and your care team will go over the benefits and risks of any follow-up options and decide what is right for you.

Meet the Sickle Cell Disease Care Team

Sickle Cell Team at Fred Hutch