One of the greatest success stories of cord blood transplantation to date is its unrivalled ability to treat sickle cell disease (SCD).
“Expectant mothers who have a child with SCD should be counselled and informed about the importance of saving their baby’s cord blood for potential transplant of the child with SCD… SCD patients and parents of children with SCD should know about the ability of stem cell transplants to cure this affliction.” Dr. Naynesh Kamani, AABB
SCD is the most common inherited blood disorder in the UK, and a life-limiting illness that causes the sufferer terrible pain crises, eventually leading to organ damage and stroke.
- An estimated 250,000 people in the UK have sickle cell trait
- Approximately 15,000 people in the UK have sickle cell disease
What is Sickle Cell Disease?
Sickle cell disease is a term which describes a group of inherited conditions that affect the shape of red blood cells. The most serious and prevalent of these conditions is known as sickle cell anaemia. Typical, healthy red blood cells are a circular, disc shape; whereas, red blood cells in sickle cell disease, have a crescent or “sickle” shape. The sickle shape of these cells make them prone to clumping, leading to blockages in tiny blood vessels. This can result in pain crises, infection, chronic progressive organ damage and eventually, stroke.
Due to their abnormal shape, sickle cells cannot effectively transport oxygen around the body in comparison to healthy red blood cells. Consequently, the sufferer may experience anaemia, tiredness and shortage of breath.
Who is Affected by Sickle Cell Disease?
SCD is transmitted genetically, meaning that it is an inherited condition. If both parents are carriers of the sickle cell gene but do not display symptoms of SCD (known as sickle cell trait carriers), there’s a 25% chance that each child they have will be born with SCD.
In 2010, an estimated 305,800 babies were born with SCD worldwide. In the UK, those with African and Caribbean heritage are most commonly affected. The life expectancy for sickle cell disease sufferers in high-income countries is 40 – 60 years of age, but the prognosis for sickle cell sufferers varies enormously, and depends on access to quality medical care. In the US, 90% of children born with SCD will survive to adulthood; but in the developing world, the majority of children born with the condition will not survive childhood.
How is Sickle Cell Disease Treated?
The pain crises which arise from sickle-shaped cell clumping can be alleviated to a certain extent with paracetamol and ibuprofen. Symptoms of infection are treated with antibiotics. Sickle cell anaemia sufferers may also mange symptoms by receiving blood transfusions, sometimes at 3-4 week intervals – this therapy is sometimes referred to as chronic transfusion therapy.
The only cure for SCD is through a haemopoietic stem cell transplant, following myeloablative conditioning. Haemopoietic stem cells are blood forming cells, and are located in bone marrow and umbilical cord blood.
Stem cell transplants are most effective in children and young adults with sickle cell anaemia. When the stem cell donor is an immunologically compatible sibling, the outcomes are extremely positive: less than 5% mortality, 5-10% incidence of graft rejection, and close to 90% SCD-free survival. Following successful stem cell transplants, SCD patients are symptom-free and their quality of life is greatly improved.
Sickle Cell Disease and Cord Blood
Dr. Naynesh Kamani is Director of the Center for Cellular Therapies at AABB and specialises in pediatric immunology and bone marrow transplantation. He has worked in the stem cell transplant programs at Children’s Hospital of Philadelphia, Miami Children’s Hospital, and Santa Rosa Children’s Hospital in San Antonio, and directed the stem cell transplant program at the Children’s National Medical Center in Washington, D.C. According to Dr. Kamani, banked cord blood from a matched family member offers the most effective treatment for SCD:
“Expectant mothers who have a child with SCD should be counselled and informed about the importance of saving their baby’s cord blood for potential transplant of the child with SCD. Because not all patients have matched sibling donors, researchers have resorted to transplanting SCD patients with unrelated donors found on registries. These results are not as good as related donors, because of increased complications due to tissue type incompatibility between the donor and recipient. SCD patients and parents of children with SCD should know about the ability of stem cell transplants to cure this affliction.”
CellPlan is the world’s first insurer to specialise in cord blood stem cell care. Founded in collaboration with leading stem cell scientists, CellPlan is committed to protecting families from the costs of stem cell therapies. Our plan includes access to medical experts as well as covering the costs of treatment, medicines, travel and accommodation.
Cord blood stem cells are used to treat over 80 diseases of the blood and immune systems, including leukaemia and sickle cell disease. Cord blood transfusions are now also helping children with cerebral palsy and autism and seem set to save and enhance the lives of patients suffering from a wider range of conditions with time.
Umbilical cord blood is collected shortly after the birth of your child and can be processed and stored by your chosen cord blood bank. CellPlan works with the most accredited banks in the world, to ensure that families receive the best possible care. You can search for a cord blood bank here and apply for CellPlan protection here.