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28
Jul

sickle-cell-disease

Sickle Cell Disease

Sickle Cell Disease

Sickle cell disease is the name for a group of conditions that affect red blood cells. The most serious of these conditions is called sickle cell anaemia.Healthy red blood cells are disc shaped, but in sickle cell disease these cells have a crescent or “sickle” shape. Sickle shaped cells are prone to clumping and can block tiny block blood vessels. 

This can cause pain crises, infection, chronic progressive organ damage and stroke.[1,8]

Sickle cells do not carry oxygen around the body as efficiently as healthy cells. This can result in anaemia, tiredness and shortage of breath.[1]

Sickle cell disease is an inherited condition. If both parents carry the sickle cell gene (known as sickle cell trait), there’s a 25% chance that each child they have will be born with sickle cell disease.[1]

In 2010 an estimated 305,800 babies were born with sickle cell disease worldwide.[4] People of African, Caribbean, Middle Eastern, Eastern Mediterranean and Asian origin are most commonly affected.

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. In the developing world the majority of children born with the condition will not survive childhood.[6]

Sickle Cell Disease in the UK

  • Sickle cell disease is the most common inherited blood disorder in the UK[5]
  • An estimated 250,000 people in the UK have sickle cell trait[2]
  • Approximately 15,000 people in the UK have sickle cell disease[3]
  • In the UK those with African and Caribbean heritage are most commonly affected[1]
  • The life expectancy for sickle cell disease sufferers in high income countries is 40 – 60 years of age [1]

 Sickle Cell Disease and Cord Blood

The pain crises caused by sickle cell are managed with paracetamol and ibuprofen. Infections are treated with antibiotics. Sickle cell anaemia sufferers may require blood transfusions, sometimes at 3-4 week intervals.[1]

The only cure for sickle cell disease is a hematopoietic stem cell transplant.  Haematopoietic stem cells are blood stem cells, and can be found in bone marrow, peripheral blood stem cells, or umbilical cord blood.

Stem cell transplants are increasingly considered for children and young adults with sickle cell anaemia. When the stem cell donor is a matched sibling, the results are excellent: less than 5% mortality, 5-10% incidence of graft rejection, and close to 90% SCD-free survival.

After a successful stem cell transplant, SCD patients become symptom-free and can expect to live a very good quality of life. There are possible complications of stem cell transplant, however: reproductive organ damage, sterility, and perhaps a chronic immune reaction between the cells of the donor and the patient. This is called graft-versus-host disease and occurs in about 10-20% of transplant patients today.[6]

Independent Advice

Dr. Naynesh KamaniDr. Naynesh Kamani is Director of the Center for Cellular Therapies at AABB. He 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.

 

“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. Since SCD is recessively inherited, there is a 25% chance that the unborn baby may also have SCD, and a 25% chance that he/she will be a tissue-type match to the SCD patient.

Because not all patients have matched sibling donors, researchers have resorted to transplanting SCD patients with unrelated donors found on registries like Be The Match or Be The Cure. These results are not as good as related donors, because of increased complications due to tissue type incompatibility between the donor and recipient. Newer approaches to transplantation are now being explored in several clinical trials for children and adults with SCD who lack matched sibling donors.

SCD patients and parents of children with SCD should know about the ability of stem cell transplants to cure this affliction.

They need to be educated and empowered to make decisions regarding therapy in concert with their healthcare providers. I would argue that even though the majority of patients with SCD may not be candidates for a stem cell transplant, it is important to inform patients and their health care providers of this potential option soon after diagnosis so that families can be educated about all potential therapeutic options.” [6]

References

  1. http://www.nhs.uk/conditions/Sickle-cell-anaemia/Pages/Introduction.aspx
  2. http://www.parliament.uk/business/publications/written-questions-answers-statements/written-question/Commons/2014-12-01/216527/
  3. http://www.dmu.ac.uk/research/research-faculties-and-institutes/health-and-life-sciences/sickle-cell-education/what-is-sickle-cell.aspx
  4. http://www.medicinenet.com/script/main/art.asp?articlekey=171632
  5. http://www.pharmaceutical-journal.com/learning/learning-article/sickle-cell-disease-symptoms-complications-and-management/20069268.article
  6. https://parentsguidecordblood.org/en/news/cord-blood-transplants-sickle-cell-disease 

The information in this article is for information purposes only. It is not intended to replace the advice of a medical expert. If you have any concerns about your health you should always discuss them with your doctor.

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