World Sickle Cell Day 2017

Sickle Cell Disease (SCD) is a rare blood disorder affecting the normal production of hemoglobin. Two copies of the SCD mutation gene HbS result in sickle cell (SS) disease. The disease leads to production of abnormal hemoglobin in the shape of a sickle. This also leads to ‘sickling’ or sticking together of cells blocking the flow of blood in blood vessels. This condition is extremely painful and can be fatal if adequate treatment is not given on time. Currently, the only treatment available for SCD is regular blood transfusions, hydroxy urea tablets and anti-coagulant therapies. The only known cure is bone marrow transplant with a 6/6 HLA match. Gene therapy clinical trials have been very successful so far for SCD with one patient being completely cured (case study published in the New England Journal of Medicine, 2017). Gene therapy involves the replacement of the faulty gene with the normal globin gene producing normal hemoglobin.

World Sickle Cell Day 2017

World Sickle Cell Awareness Day is marked on 19^th June each year. In 2008, the General Assembly of the United Nations adopted a resolution to consider sickle cell disease (SCD) as a serious public health concern. SCD is one of the most prevalent genetic disorders across the globe. It is mostly concentrated in the four continents of Africa, Asia, North and South America. It is also present in Southern Europe in Italy and Greece. Apathy and lack of prevention and control programs have led to a serious rise in prevalence and incidence of SCD. According to the journal PLOS Medicine, around 30% of the globe will be affected by SCD with the highest numbers in Sub-Saharan Africa and India.

The major health organizations globally, including the World Health Organization (WHO) and the United Nations (UN), have recognized the need to tackle SCD on a war footing. It is essential to put in place robust prevention and control programs including counseling, antenatal testing and prenatal screening to prevent further live births.

World Sickle Cell Day 2017 is being celebrated across the globe in partnership with patient organizations, advocacy cells, hospitals, health care providers and well-wishers. This day is particularly significant as it aims to make the public aware of the public health concern of a rapidly increasing genetic blood disorder. It is essential that the governments of all nations take note of this concern, and put in place programs to enable timely diagnostics, treatment, prevention and control.

Concerns in SCD also involve the availability and access to safe and tested blood. Blood-borne viruses like HIV, HBV and HCV are a real threat to patients with SCD and it is imperative for all patients to access quality blood. In many countries, even access to basic transfusion facilities is not available, and this needs to be addressed by respective governments. Other facilities including genetic counseling, availability of drugs and other therapeutic procedures, lab tests and psycho-social support are essential for a person with SCD to thrive in life.

World Sickle Cell Day is a proactive platform for patients and families to express their concerns to the government and policy makers. It also raises awareness among the public who may be encouraged to go in for carrier screening to avoid further incidences of SCD. Fund-raisers, walkathons, street plays and other events are usually held to create a public campaign for SCD.

Future Hopes

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While gene therapy holds out positive hopes for the future, until it actually arrives it is important for patients to comply with current treatment and stay healthy. Gene therapy is a final curative and can drastically alter the quality of lives of patients, eliminating the need for blood transfusions and other therapies. Clinical trials across various phases are still on, in North America and Europe, with initial results indicating great promise. It may be another 5-8 years before the treatment reaches people. Till then, it is essential for patients to stay positive and optimistic and take care of their health to qualify for gene therapy treatment.

References:

1. 3rd World Sickle Cell Day - (http://www.unesco.org/new/en/unesco/events/all-events/?tx_browser_pi1%5BshowUid%5D=4087)
2. World Sickle Cell Day 2017 - (http://www.moh.gov.sa/en/HealthAwareness/healthDay/2015/Pages/HealthDay-2015-06-19.aspx)
3. Sickle cell disease - (https://ghr.nlm.nih.gov/condition/sickle-cell-disease)
4. Sickle Cell  Foundation of Georgia - (http://sicklecellga.org/worldday/)
5. World Sickle Cell Day 2017 - (http://www.conqueringthecurve.org/events/worldsicklecellday2017)
6. Support Group Meeting - (http://www.cscfkids.org/)
7. Did Gene Therapy Cure Sickle Cell Disease? - (http://www.acsh.org/news/2017/03/07/did-gene-therapy-cure-sickle-cell-disease-10950)
8. Sickle Cell Anemia Is On The Rise Worldwide - (http://www.npr.org/sections/health-shots/2013/07/17/202628070/sickle-cell-anemia-is-on-the-rise-worldwide)
9. Hoban, Megan D., Stuart H. Orkin, and Daniel E. Bauer. "Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease." Blood (2016): blood-2015.

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Source-Medindia