Help and Hope for Sickle Cell Disease

Sickle Cell Disease (SCD) is an inherited blood disorder, meaning it’s passed down from the parents to their children. Two parents who are carriers of the sickle cell gene have a 25% (1 in 4) chance of having a child with sickle cell disease.

It is a rare blood disorder, affecting about 100,000 people in the U.S., but more children of African American heritage—1 in about every 365 babies—are born with Sickle Cell Disease. In people with Sickle Cell Disease, red blood cells are shaped like a half-moon or a bow rather than the smooth and round donut shape of a normal red blood cell. These sickled cells can block the flow of blood to various organs, causing bouts of severe pain—called “pain crisis”—as well as some serious complications, including eye problems, heart failure, kidney disease, iron overload causing liver damage and even strokes at a very young age. The most common cause of death is from severe infection also known as sepsis, or acute chest syndrome if not caught and treated on time, says Tonjeh Bah, MD, Medical Director of MetroHealth’s Adult Sickle Cell Program.

MetroHealth hematologists manage Sickle Cell Disease using:

• Disease Modifying Therapy which are medicines to prevent cells from sickling and to prevent complications, pain crises and infections. An example is Hydroxyurea.
• Partial or total Blood exchange transfusions to lower the percentage of sickle cells in the blood.
• Outpatient infusion clinic visits are available for adults with Sickle Cell Disease so they can receive IV fluids and pain medicines to manage acute pain crises.
• Targeted Emergency Department protocols where every sickle cell patient who has established care with Metro Health has an emergency room care plan that serves as a guide to the ED doctor when they come in for pain crisis.
• Our Hospital in the Home (HiTH) Program is a unique pilot initiative that allows adult patients who have been admitted for 1-2 days for sickle cell pain crisis to continue their care in the comfort of their home for up to 5 days with a visiting nurse coming to their home 1-2 times a day to administer pain meds or fluids as needed until crisis are controlled by their home oral pain regimen.

There is reason to feel positive. For some patients with Sickle Cell Disease, a permanent cure may be possible. One option is a stem cell transplant, which is when a patient with sickle disease receives healthy cells from a compatible donor that helps the bone marrow make healthy red blood cells.

A newer option is gene therapy, where doctors collect stem cells from the patients, treat them with a drug that alters their genetic makeup, and puts them back into the sickle cell patient’s body—so that they can make healthy red blood cells. Two such therapies were approved by the U.S. Food and Drug Administration in December 2023.

The future for those with Sickle Cell Disease is better than ever, says Dr. Bah. “In the past 7- 10 years, we’ve had at least six new medications to help reduce the amount of pain crisis. With our growing Bone marrow transplant program and promising gene therapy lab, Metro Health will be able to offer these to our sickle cell patients soon. Gene therapy and stem cell transplant have given us hope for a cure.”