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Effective treatments
are available to help relieve the symptoms
and complications of sickle cell anemia, but
in most cases there’s no cure. Some
researchers believe that bone marrow
transplants may offer a cure in a small
number of cases. Researchers are looking for
new treatments for sickle cell anemia,
including gene therapy and safer and more
effective bone marrow transplants.
People who have sickle
cell anemia need regular medical care. Some
doctors and clinics specialize in treating
people with the condition. Doctors
specializing in sickle cell anemia are often
hematologists (doctors who treat people with
blood disorders)—or pediatric hematologists
(if they also treat children).
Goals of Treatment
The goals of treating
sickle cell anemia are to relieve pain;
prevent infections, eye damage, and strokes;
and control complications if they occur. The
treatments include medicine, blood
transfusions, and specific treatment for
complications.
Treating Pain
Mild painful crises can
be managed with treatments such as
over-the-counter medicine and heating pads.
However, severe pain may need to be treated
in a hospital. Painful crises are the
leading cause of emergency room visits and
hospitalizations of people with sickle cell
anemia.
The usual treatments
for acute (short-term) pain crises are
pain-killing medicines and fluids, given
either by mouth or through a vein, to
prevent dehydration (a condition in which
your body doesn’t have enough fluids). The
pain-killing medicines most often used are
acetaminophen, nonsteroidal
anti-inflammatory drugs (NSAIDs), and
narcotics such as meperidine, morphine,
oxycodone, and others.
The treatment of
patients who have mild-to-moderate pain
usually begins with NSAIDs or acetaminophen.
If pain continues, a narcotic may be added.
Moderate-to-severe pain is often treated
with narcotics. The narcotic may be used
alone or together with NSAIDs or
acetaminophen. Narcotic abuse and addiction
are pain management issues that must be
considered in any pain control plan.
A medicine called
hydroxyurea may be given to adults and older
adolescents with severe sickle cell anemia
to reduce their number of painful crises.
This medicine is used only to prevent these
crises, not to treat them when they occur.
Given daily, hydroxyurea can reduce the
frequency of painful crises and of acute
chest syndrome. People taking the medicine
also may need fewer blood transfusions.
People taking
hydroxyurea must be watched carefully
because the medicine can cause serious side
effects, including an increased risk of
dangerous infections. Some evidence suggests
that long-term use of hydroxyurea can cause
tumors or leukemia. Because of these risks,
the medicine is usually only used in adults
and older teenagers with severe sickle cell
anemia. Although hydroxyurea is being tested
in infants and children at this time, it
won’t be approved for use in children until
its long-term effects can be more closely
studied.
Preventing Infections
Infection is a major
complication of sickle cell anemia. In fact,
pneumonia is the leading cause of death in
children with the condition. Other
infections common in people with sickle cell
anemia include meningitis, influenza, and
hepatitis. If a child with sickle cell
anemia shows early signs of an infection,
such as fever, seek treatment right away.
To prevent infections
in babies and young children, treatments
include:
Daily doses of
penicillin. Treatment may begin as early as
2 months of age and continue until the child
is at least 5 years old.
Vaccinations for
pneumonia, meningitis, influenza, and
hepatitis.
A yearly flu shot.
Adults who have sickle
cell anemia also should have flu shots every
year and be vaccinated for pneumonia.
Preventing Eye Damage
Sickle cell anemia can
damage the blood vessels in the eyes.
Parents should ask their child’s doctor
about regular checkups with an eye doctor
who specializes in diseases of the retina.
The retina is a thin layer of tissue inside
the back of the eye. Adults with sickle cell
anemia also should have regular checkups
with an eye doctor.
Preventing Strokes
Stroke prevention and
treatment is now possible for children and
adults who have sickle cell anemia. Starting
at age 2, children with sickle cell anemia
often receive regular ultrasound scans of
the head (this is called transcranial
Doppler ultrasound). These scans are used to
monitor blood flow in the brain.
The scans allow doctors
to find out which children are at high risk
for a stroke and treat them with regular
blood transfusions. Routine blood
transfusions have been found to greatly
reduce the number of strokes in children.
Blood Transfusions
Blood transfusions are
used to treat worsening anemia and sickle
cell complications. A sudden worsening of
anemia due to an infection or enlargement of
the spleen is a common reason for a blood
transfusion. Some, but not all, patients
need transfusions to prevent
life-threatening events such as stroke or
pneumonia.
Regular blood
transfusions do have side effects, and
patients must be carefully watched. Side
effects can include a dangerous buildup of
iron in the blood (which must be treated) as
well as an increased risk of infection from
the transfused blood.
Treating Other
Complications
Acute chest syndrome is
a severe and life-threatening complication
in children and adults who have sickle cell
anemia. Treatment usually requires
hospitalization and may include oxygen,
blood transfusions, antibiotics, pain
medicine, and monitoring the body’s fluids.
Leg ulcers can be
painful, and patients may be given strong
pain medicines. Ulcers can be treated with
cleansing solutions and medicated creams or
ointments. Skin grafts may be needed if the
condition continues. Bed rest and keeping
the legs raised to reduce swelling are
helpful, although not always possible.
Gallbladder surgery may
be needed if the presence of gallstones
leads to gallbladder disease.
Priapism can be treated
with fluids or surgery.
Regular Health Care for
Children
Children with sickle
cell anemia should get regular health care,
just like children without the condition.
They need to have their growth checked and
to get the usual shots that all children
receive.
It’s recommended that
before age 2, children with sickle cell
anemia see the doctor often (sometimes every
2 or 3 months). After age 2, children may
need to see the doctor less often, but
usually at least every 6 months. These
visits are a time for parents to talk with
their child’s doctor and ask questions about
the child’s care. Parents should ask about
eye checkups and the need for an ultrasound
scan of the brain.
Until age 5, daily
penicillin is given to most children who
have sickle cell anemia. Many patients are
prescribed a vitamin called folic acid (folate)
to help prevent some of the complications of
sickle cell anemia.
New Treatments
Today, research on
sickle cell anemia is looking at bone marrow
transplants, gene therapy, and new
medicines. The hope is that these studies
will provide better treatments for sickle
cell anemia. Researchers also are looking
for a way to predict the severity of the
condition.
Bone Marrow Transplant
Bone marrow transplant
can be a very effective treatment for sickle
cell anemia, but because of its risks, only
some patients can or should have this
procedure.
The bone marrow
transplant procedure is risky and could
result in serious side effects and even
death. It’s usually used only for younger
patients with severe sickle cell anemia, but
the decision is made on a case-by-case
basis.
Bone marrow used for a
transplant must come from a closely matched
donor, usually a close family member, who
doesn’t have sickle cell anemia.
Gene Therapy
Gene therapy is being
studied as a possible treatment for sickle
cell anemia. Researchers are looking to see
whether a normal gene can be planted in the
bone marrow of a person with sickle cell
anemia, and thus cause the body to produce
normal red blood cells. Researchers also are
studying the possibility of treatment to
“turn off” the sickle cell gene or “turn on”
a gene that makes red blood cells behave
normally.
New Medicines
New medicines being
studied are:
Butyric acid. This is a
food additive that may increase normal
hemoglobin in the blood.
Clotrimazole. This is
used now to treat fungus infections. This
medicine helps prevent the loss of water
from a red blood cell and can keep the cell
from turning into a sickle cell.
Nitric oxide. This may
make sickle cells less sticky and keep blood
vessels open. People with sickle cell anemia
have low levels of nitric acid in their
blood. |
