William Amarquaye, PharmD

September has been designated as National Sickle Cell Awareness Month. Established by Congress in 1983, this month is dedicated to the continued fostering of education surrounding sickle cell disease (SCD) management, research, and development, as well as the public perception.

This particular condition is a group of inherited blood disorders arising out of a genetic mutation. The hallmark shape of the red blood cell (RBC) is a sickle shape. Because of a deformity that happens in the RBC, oxygen is not able to get to where it needs to go in our bodies.

In this condition, RBCs tend to polymerize or clump together in blood vessels and a vaso-occlusive crisis (VOC) can occur. Patients with SCD often present to the emergency department (ED) with a sickle cell crisis, which can cause immense and excruciating amounts of pain for the patient.

The CDC estimates that approximately 100,000 Americans are affected by SCD.

 In the United States this condition primarily affects African Americans and Hispanic Americans, with SCD occurring in 1 in every 365 African American births and 1 in every 16,300 Hispanic American births.

The importance of this month can be highlighted by the lack of funding, treatment options over the years, and health disparities that patients with SCD often encounter. The year 2010 marked the 100

 anniversary of the first research paper on SCD in Western medical literature.

 Since that time, only a single hand is needed to determine how many drug treatments have been approved for the management of SCD.

Currently, there are only 5 FDA-approved therapies used to manage SCD. Stem cell transplantation, Hydroxyurea, Endari, Oxbryta, and Adakveo. Each of these treatment options are used to manage different aspects in sickle cell pathophysiology.

Increasing fetal hemoglobin, replacing stem cells in bone marrow, making RBCs more flexible, and preventing hemoglobin from sticking together each provide a way to prolong life for patients with SCD.

This past year, we saw the approval of 2 new medications for SCD (Oxybryta and Adakveo).

With the importance of finding more drug treatment options for SCD, it will be imperative to continue advocating for more quality funding for research and development. The topic of funding in SCD has also been discussed due to major disparities that have been found.

 revealed some of the major disparities in this disease state. The study noted that SCD is 3 times as prevalent as cystic fibrosis (CF), but there is more funding from the National Institutes of Health (NIH) per person with CF.

Additionally, CF trials are receiving more industry funding and philanthropic expenditures were greater per person for CF compared with SCD.

 Another major point of education during Sickle Cell Awareness Month that any patient with SCD can attest to is access to quality pain management, especially when presenting in the ED.

As previously mentioned, excruciating and intense pain is characteristic of SCD. Having a family member myself who has SCD, I know exactly what it feels like to directly see the agonizing and harrowing pain that exudes from the patient.

Each patient who presents to the ED with a sickle cell crisis should be seen and taken care of urgently and in a timely manner. SCD disease state guidelines outline specifically when a patient should be seen and taken care of promptly.

The 2014 NIH guidelines state that for children or adults with SCD and a VOC, analgesic therapy is to be rapidly initiated within 30 minutes of triage.

Even though the guidelines make it clear that a VOC is to have priority when presented in the ED, that is not the reality for many patients with SCD, unfortunately.

Haywood et al. showed that patients with SCD experienced longer wait times to see a physician upon arrival to the ED, even though they presented with higher levels of pain and were assigned higher priority triage as well.

Sickle Cell Awareness Month is a time for action and reflection as we navigate many parts of our health care system that can be more conducive for patients with SCD. Each aspect that was discussed will be vital for making sure that SCD gets the attention that it rightly deserves.

1. Hartsell, Malerie.National Sickle Cell Awareness Month. Live Healthy S.C. DHEC's Health and Environment Blog. 

https://blog.scdhec.gov/2017/09/18/national-sickle-cell-awareness-month-2/

. Updated October 21, 2019. Accessed September 17, 2020.

3. NIH recognizes sickle cell disease awareness month. National Institutes of Health 

https://www.nih.gov/news-events/news-releases/nih-recognizes-sickle-cell-disease-awarenessmonth#:~:text=September%20is%20National%20Sickle%20Cell%20Disease%20Awareness%20Month.,the%20condition%20now%20known%20as%20sickle%20cell%20disease

. September 15, 2010. Accessed September 17, 2020

4. Approved Treatments. Sickle Cell Disease News. 

https://sicklecellanemianews.com/approved-treatments/

. Updated December 5, 2019. Accessed September 17, 2020

5. Farooq F, Mogayzel PJ, Lanzkron S, Haywood C, Strouse JJ. Comparison of US federal and foundation funding of research for sickle cell disease and cystic fibrosis and factors associated with research productivity. 

 2020; 3(3):e201737. Doi 10.1001/jamanetworkopen.2020.1737. Accessed September 17, 2020.

6. National Heart, Lung, and Blood Institute. Evidence-based Management of Sickle Cell Disease: Expert Panel Report, 2014. 

http://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines

7. Haywood C Jr, Tanabe P, Naik R, Beach MC, Lanzkron S. The impact of race and disease on sickle cell patient wait times in the emergency department. Am J Emerg Med. 2013;31(4):651-656. doi:10.1016/j.ajem.2012.11.005. Accessed September 17, 2020.

Articles

September has been designated as National Sickle Cell Awareness Month for the continued fostering of education surrounding sickle cell disease management.