Health Maintenance To Prevent Complications
                
Babies with sickle cell disease (SCD) should be referred to a doctor or provider group that has experience taking care of people with this disease. The doctor might be a hematologist (a doctor with special training in blood diseases) or an experienced general pediatrician, internist, or family practitioner.
                
For infants, the first SCD visit should take place before 8 weeks of age.
                
If someone was born in a country that doesnt perform newborn SCD screening, he or she might be diagnosed with SCD later in childhood. These people should also be referred as soon as possible for special SCD care.
                

                

                
Examining the person
                
Giving medicines and immunizations
                
Performing tests
                
Educating families about the disease and what to watch out for
                

                
Preventing Infection
                
In SCD, the spleen doesnt work properly or doesnt work at all. This problem makes people with SCD more likely to get severe infections.
                
Penicillin
                
In children with SCD, taking penicillin two times a day has been shown to reduce the chance of having a severe infection caused by the pneumococcus bacteria. Infants need to take liquid penicillin. Older children can take tablets.
                
Many doctors will stop prescribing penicillin after a child has reached the age of 5. Some prefer to continue this antibiotic throughout life, particularly if a person has hemoglobin SS or hemoglobin S0 thalassemia, since people with SCD are still at risk. All people who have had surgical removal of the spleen, called a splenectomy, or a past infection with pneumococcus should keep taking penicillin throughout life.
                
Immunizations
                
People with SCD should receive all recommended childhood vaccines. They should also receive additional vaccines to prevent other infections.
                
Pneumococcus. Even though all children routinely receive the vaccine against pneumococcus (PCV13), children with SCD should also receive a second kind of vaccine against pneumococcus (PPSV23). This second vaccine is given after 24 months of age and again 5 years later. Adults with SCD who have not received any pneumococcal vaccine should get a dose of the PCV13 vaccine. They should later receive the PPSV23 if they have not already received it or it has been more than 5 years since they did. A person should follow these guidelines even if he or she is still taking penicillin.
                
Influenza. All people with SCD should receive an influenza shot every year at the start of flu season. This should begin at 6 months of age. Only the inactivated vaccine, which comes as a shot, should be used in people with SCD.
                
Meningococcus. A child with SCD should receive this vaccine (Menactra or Menveo) at 2, 4, 6, and 1215 months of age. The child should receive a booster vaccine 3 years after this series of shots, then every 5 years after that.
                
Screening Tests and Evaluations
                
Height, Weight, Blood Pressure, and Oxygen Saturation
                
Doctors will monitor height and weight to be sure that a child is growing properly and that a person with SCD is maintaining a healthy weight.
                
Doctors will also track a persons blood pressure. When a person with SCD has high blood pressure, it needs to be treated promptly because it can increase the risk of stroke.
                
Oxygen saturation testing provides information about how much oxygen the blood is carrying.
                
Blood and Urine Testing
                
People with SCD need to have frequent lab tests.
                
Blood tests help to establish a persons baseline for problems like anemia. Blood testing also helps to show whether a person has organ damage, so that it can be treated early.
                
Urine testing can help to detect early kidney problems or infections.
                
Transcranial Doppler (TCD) Ultrasound Screening
                
Children who have hemoglobin SS or hemoglobin S0 thalassemia and are between the ages of 2 and 16 should have TCD testing once a year.
                
This study can find out whether a child is at higher risk for stroke. When the test is abnormal, regular blood transfusions can decrease the chances of having a stroke.
                
The child is awake during the TCD exam. The test does not hurt at all. The TCD machine uses sound waves to measure blood flow like the ultrasound machine used to examine pregnant women.
                
Eye Examinations
                
An eye doctor, or ophthalmologist, should examine a persons eyes every 12 years from the age of 10 onwards.
                
These exams can detect if there are SCD-related problems of the eye. Regular exams can help doctors find and treat problems early to prevent loss of vision. A person should see his or her doctor right away for any sudden change in vision.
                
Pulmonary Hypertension
                
Doctors have different approaches to screening for pulmonary hypertension. This is because studies have not given clear information as to when and how a person should receive the screening. People with SCD and their caretakers should discuss with their doctor whether screening makes sense for them.
                
Cognitive Screening
                
People with sickle cell disease can develop cognitive (thinking) problems that may be hard to notice early in life.
                
Sometimes these problems are caused by silent strokes that can only be seen with magnetic resonance imaging (MRI) of the brain.
                
People with SCD should tell their doctors or nurses if they have thinking problems, such as difficulties learning in school, slowed decision making, or trouble organizing their thoughts.
                
People can be referred for cognitive testing. This testing can identify areas in which a person could use extra help.
                
Children with SCD who have thinking problems may qualify for an Individualized Education Program, or IEP. An IEP is a plan that helps students to reach their educational goals. Adults may be able to enroll in vocational rehabilitation programs that can help them with job training.
                
Education and Guidance
                
Doctors and other providers will talk with people who have SCD and their caretakers about complications and also review information at every visit.
                
Because there are a lot of things to discuss, new topics are often introduced as a child or adult reaches an age when that subject is important to know about.
                
Doctors and nurses know that there is a lot of information to learn, and they dont expect people to know everything after one discussion. People with SCD and their families should not be afraid to ask questions.
                
Topics that are usually covered include:
                
Hours that medical staff are available and contact information to use when people with SCD or caretakers have questions
                
A plan for what to do and where to get care if a person has a fever, pain, or other signs of SCD complications that need immediate attention
                
How SCD is inherited and the risk of having a child with SCD
                
The importance of regular medical visits, screening tests, and evaluations
                
How to recognize and manage pain
                
How to palpate (feel) a childs spleen. Because of the risk of splenic sequestration crisis, caretakers should learn how to palpate a childs spleen. They should try to feel for the spleen daily and more frequently when the child is ill. If they feel that the spleen is bigger than usual, they should call the care provider. 
                
Transitioning Care
                
When children with SCD become adolescents or young adults, they often need to transition from a pediatric care team to an adult care team. This period has been shown to be associated with increased hospital admissions and medical problems. There seem to be many reasons for this.
                
Some of the increased risk is directly related to the disease. As people with SCD get older, they often develop more organ damage and more disabilities.
                
The shift in care usually occurs at the same time that adolescents are undergoing many changes in their emotional, social, and academic lives. The transition to more independent self-management may be difficult, and following treatment plans may become less likely.
                
When compared with pediatrics, there are often fewer adult SCD programs available in a given region. This makes it more difficult for a person with SCD to find appropriate doctors, particularly those with whom they feel comfortable.
                
To improve use of regular medical care by people with SCD and to reduce age-related complications, many SCD teams have developed special programs that the make transition easier. Such programs should involve the pediatric and the adult care teams. They should also start early and continue over several years.
                
Managing Some Complications of SCD
                
Acute Pain
                
Each person with SCD should have a home treatment regimen that is best suited to their needs. The providers on the SCD team usually help a person develop a written, tailored care plan. If possible, the person with SCD should carry this plan with them when they go to the emergency room.
                
When an acute crisis is just starting, most doctors will advise the person to drink lots of fluids and to take a non-steroidal anti-inflammatory (NSAID) pain medication, such as ibuprofen. When a person has kidney problems, acetaminophen is often preferred.
                
If pain persists, many people will find that they need a stronger medicine.
                
Combining additional interventions, such as massage, relaxation methods, or a heating pad, may also help.
                
If a person with SCD cannot control the pain at home, he or she should go to an SCD day hospital/outpatient unit or an emergency room to receive additional, stronger medicines and intravenous (IV) fluids.
                
Some people may be able to return home once their pain is under better control. In this case, the doctor may prescribe additional pain medicines for a short course of therapy.
                
People often need to be admitted to the hospital to fully control an acute pain crisis.
                
When taken daily, hydroxyurea has been found to decrease the number and severity of pain episodes.
                
Chronic Pain
                
Sometimes chronic pain results from a complication, such as a leg ulcer or aseptic necrosis of the hip. In this case, doctors try to treat the complication causing the pain.
                
While chronic pain is common in adults with SCD, the cause is often poorly understood. Taking pain medicines daily may help to decrease the pain. Some examples of these medicines include:
                
NSAID drugs, such as ibuprofen
                
Duloxetine
                
Gabapentin
                
Amitriptyline
                
Strong pain medicines, such as opiates
                
Other approaches, such as massage, heat, or acupuncture may be helpful in some cases. Chronic pain often comes with feelings of depression and anxiety. Supportive counseling and, sometimes, antidepressant medicines may help. (See coping and emotional issues.)
                
Severe Anemia
                
People should see their doctors or go to a hospital right away if they develop anemia symptoms from a splenic sequestration crisis or an aplastic crisis. These conditions can be life-threatening, and the person will need careful monitoring and treatment in the hospital. A person also usually needs a blood transfusion.
                
People with SCD and symptoms of severe anemia from other causes should also see a doctor right away.
                
Infections
                
Fever is a medical emergency in SCD. All caretakers of infants and children with SCD should take their child to their doctor or go to a hospital right away when their child has a fever. Adults with SCD should also seek care for fever or other signs of infection.
                
All children and adults who have SCD and a fever (over 38.50 C or 101.30 F) must be seen by a doctor and treated with antibiotics right away.
                
Some people will need to be hospitalized, while others may receive care and follow-up as an outpatient.
                
Acute Chest Syndrome
                
People with SCD and symptoms of acute chest syndrome should see their doctor or go to a hospital right away.
                
They will need to be admitted to the hospital where they should receive antibiotics and close monitoring. They may need oxygen therapy and a blood transfusion.
                
When taken daily, the medicine hydroxyurea has been found to decrease the number and severity of acute chest events.
                
Clinical Stroke
                
People with SCD who have symptoms of stroke should be brought to the hospital right away by an ambulance. If a person is having symptoms of stroke, someone should call 9-1-1.
                
Symptoms of stroke may include:
                
Weakness of an arm or leg on one side of the body
                
Trouble speaking, walking, or understanding
                
Loss of balance
                
Severe headache
                
If imaging studies reveal that the person has had an acute stroke, he or she may need an exchange transfusion. This procedure involves slowly removing an amount of the persons blood and replacing it with blood from a donor who does not have SCD or sickle cell trait. Afterward, the person may need to receive monthly transfusions or other treatments to help to prevent another stroke.
                
Silent Stroke and Cognitive Problems
                
Children and adults with SCD and cognitive problems may be able to get useful help based upon the results of their testing. For instance, children may qualify for an IEP. Adults may be able to enroll in vocational, or job, training programs.
                
Priapism
                
Sometimes, a person may be able to relieve priapism by:
                
Emptying the bladder by urinating
                
Taking medicine
                
Increasing fluid intake
                
Doing light exercise
                
If a person has an episode that lasts for 4 hours or more, he should go to the hospital to see a hematologist and urologist.
                
Pregnancy
                
Pregnant women with SCD are at greater risk for problems. They should always see an obstetrician, or OB, who has experience with SCD and high-risk pregnancies and deliveries.
                
The obstetrician should work with a hematologist or primary medical doctor who is well informed about SCD and its complications.
                
Pregnant women with SCD need more frequent medical visits so that their doctors can follow them closely. The doctor may prescribe certain vitamins and will be careful to prescribe pain medicines that are safe for the baby.
                
A pregnant woman with SCD may need to have one or more blood transfusions during her pregnancy to treat complications, such as worsening anemia or an increased number of pain or acute chest syndrome events.
                
Hydroxyurea
                
What Is Hydroxyurea?
                
Hydroxyurea is an oral medicine that has been shown to reduce or prevent several SCD complications.
                
This medicine was studied in patients with SCD because it was known to increase the amount of fetal hemoglobin (hemoglobin F) in the blood. Increased hemoglobin F provides some protection against the effects of hemoglobin S.
                
Hydroxyurea was later found to have several other benefits for a person with SCD, such as decreasing inflammation.
                
Use in adults. Many studies of adults with hemoglobin SS or hemoglobin S thalassemia showed that hydroxyurea reduced the number of episodes of pain crises and acute chest syndrome. It also improved anemia and decreased the need for transfusions and hospital admissions.
                
Use in children. Studies in children with severe hemoglobin SS or S thalassemia showed that hydroxyurea reduced the number of vaso-occlusive crises and hospitalizations. A study of very young children (between the ages of 9 and 18 months) with hemoglobin SS or hemoglobin S thalassemia also showed that hydroxyurea decreased the number of episodes of pain and dactylitis.
                
Who Should Use Hydroxyurea?
                
Since hydroxyurea can decrease several complications of SCD, most experts recommend that children and adults with hemoglobin SS or S0 thalassemia who have frequent painful episodes, recurrent chest crises, or severe anemia take hydroxyurea daily.
                
Some experts offer hydroxyurea to all infants over 9 months of age and young children with hemoglobin SS or S0 thalassemia, even if they do not have severe clinical problems, to prevent or reduce the chance of complications. There is no information about how safe or effective hydroxyurea is in children under 9 months of age.
                
Some experts will prescribe hydroxyurea to people with other types of SCD who have severe, recurrent pain. There is little information available about how effective hydroxyurea is for these types of SCD.
                
In all situations, people with SCD should discuss with their doctors whether or not hydroxyurea is an appropriate medication for them.
                
Pregnant women should not use hydroxyurea.
                
How Is Hydroxyurea Taken?
                
To work properly, hydroxyurea should be taken by mouth daily at the prescribed dose. When a person does not take it regularly, it will not work as well, or it wont work at all.
                
A person with SCD who is taking hydroxyurea needs careful monitoring. This is particularly true in the early weeks of taking the medicine. Monitoring includes regular blood testing and dose adjustments.
                
What Are the Risks of Hydroxyurea?
                
Hydroxyurea can cause the bloods white cell count or platelet count to drop. In rare cases, it can worsen anemia. These side effects usually go away quickly if a person stops taking the medication. When a person restarts it, a doctor usually prescribes a lower dose.
                
Other short-term side effects are less common.
                
It is still unclear whether hydroxyurea can cause problems later in life in people with SCD who take it for many years. Studies so far suggest that it does not put people at a higher risk of cancer and does not affect growth in children. But further studies are needed.
                
Red Blood Cell Transfusions
                
Doctors may use acute and chronic red blood cell transfusions to treat and prevent certain SCD complications. The red blood cells in a transfusion have normal hemoglobin in them.
                
A transfusion helps to raise the number of red blood cells and provides normal red blood cells that are more flexible than red blood cells with sickle hemoglobin. These cells live longer in the circulation. Red blood cell transfusions decrease vaso-occlusion (blockage in the blood vessel) and improve oxygen delivery to the tissues and organs.
                
Acute Transfusion in SCD
                
Doctors use blood transfusions in SCD for complications that cause severe anemia. They may also use them when a person has an acute stroke, in many cases of acute chest crises, and in multi-organ failure.
                
A person with SCD usually receives blood transfusions before surgery to prevent SCD-related complications afterwards.
                
Chronic Transfusion
                
Doctors recommend regular or ongoing blood transfusions for people who have had an acute stroke, since transfusions decrease the chances of having another stroke.
                
Doctors also recommend chronic blood transfusions for children who have abnormal TCD ultrasound results because transfusions can reduce the chance of having a first stroke.
                
Some doctors use this approach to treat complications that do not improve with hydroxyurea. They may also use transfusions in people who have too many side effects from hydroxyurea.
                
What Are the Risks of Transfusion Therapy?
                
Possible complications include:
                
Hemolysis
                
Iron overload, particularly in people receiving chronic transfusions (can severely impair heart and lung function)
                
Infection
                
Alloimmunization (can make it hard to find a matching unit of blood for a future transfusion)
                
All blood banks and hospital personnel have adopted practices to reduce the risk of transfusion problems.
                
People with SCD who receive transfusions should be monitored for and immunized against hepatitis. They should also receive regular screenings for iron overload. If a person has iron overload, the doctor will give chelation therapy, a medicine to reduce the amount of iron in the body and the problems that iron overload causes.
                

                
Hematopoietic Stem Cell Transplantation
                
At the present time, hematopoietic stem cell transplantation (HSCT) is the only cure for SCD. People with SCD and their families should ask their doctor about this procedure.
                
What Are Stem Cells?
                
Stem cells are special cells that can divide over and over again. After they divide, these cells can go on to become blood red cells, white cells, or platelets.
                
A person with SCD has stem cells that make red blood cells that can sickle. People without SCD have stem cells that make red cells that usually wont sickle.
                
What Stem Cells Are Used in HSCT?
                
In HSCT, stem cells are taken from the bone marrow or blood of a person who does not have sickle cell disease (the donor). The donor, however, may have sickle cell trait.
                
The donor is often the persons sister or brother. This is because the safest and most successful transplants use stem cells that are matched for special proteins called HLA antigens. Since these antigens are inherited from parents, a sister or brother is the most likely person to have the same antigens as the person with SCD.
                
What Happens During HSCT?
                
First, stem cells are taken from the donor. After this, the person with SCD (the recipient) is treated with drugs that destroy or reduce his or her own bone marrow stem cells.
                
The donor stem cells are then injected into the persons vein. The injected cells will make a home in the recipients bone marrow, gradually replacing the recipients cells. The new stem cells will make red cells that do not sickle.
                
Which People Receive HSCT?
                
At the present time, most SCD transplants are performed in children who have had complications such as strokes, acute chest crises, and recurring pain crises. These transplants usually use a matched donor.
                
Because only about 1 in 10 children with SCD has a matched donor without SCD in their families, the number of people with SCD who get transplants is low.
                
HSCT is more risky in adults, and that is why most transplants are done in children.
                
There are several medical centers that are researching new SCD HSCT techniques in children and adults who dont have a matched donor in the family or are older than most recipients. Hopefully, more people with SCD will be able to receive a transplant in the future, using these new methods.
                
What Are the Risks?
                
HSCT is successful in about 85 percent of children when the donor is related and HLA matched. Even with this high success rate, HSCT still has risks.
                
Complications can include severe infections, seizures, and other clinical problems. About 5 percent of people have died. Sometimes transplanted cells attack the recipients organs (graft versus host disease).
                
Medicines are given to prevent many of the complications, but they still can happen.