There are many awareness months which we observe throughout the year. The purpose of these campaigns — often tied to a particular disease or condition — are to increase the general public’s knowledge and perhaps create some understanding for those affected.
A little background:
Sickle Cell Awareness Month was first officially recognized by the federal government in 1983. (Proclamation 5102 on September 21, 1983 by President Ronald Reagan)
It is a life-long condition which affects many people in Nevada. One of our sub-speciality clinics is the Sickle Cell Treatment Center of Nevada which treats patients with sickle cell disease (SCD) who are 18 years old and younger, as well as some established adult patients.
General estimates conclude about 100,000 people in the United States are affected by sickle cell disease, although the Centers for Disease Control and Prevention says exact numbers are unknown.
What is it?
The term sickle cell disease (SCD) describes a group of inherited red blood cell disorders. People with SCD have abnormal hemoglobin, called hemoglobin S or sickle hemoglobin in their red blood cells.
Overview of the condition:
(courtesy: U.S. Department of Health & Human Services)
Cells in tissues need a steady supply of oxygen to work well. Normally, hemoglobin in red blood cells takes up oxygen in the lungs and carries it to all the tissues of the body. Red blood cells that contain normal hemoglobin are disc shaped (like a doughnut without a hole). This shape allows the cells to be flexible so that they can move through large and small blood vessels to deliver oxygen. Sickle hemoglobin is not like normal hemoglobin. It can form stiff rods within the red cell, changing it into a crescent, or sickle shape. Sickle-shaped cells are not flexible and can stick to vessel walls, causing a blockage that slows or stops the flow of blood. When this happens, oxygen can’t reach nearby tissues.
Who may be affected by SCD?
(courtesy U.S. Dept. of Health & Human Services)
The disease is more common in certain ethnic groups, including:
• People of African descent (among whom 1 in 12 carries a sickle cell gene)
• Hispanic Americans from Central and South America
• People of Middle Eastern, Asian, Indian and Mediterranean descent
We wanted to reach out to Dr. Rashid to find a little more about sickle cell disease and pose the following questions. Dr. Rashid is Director of the Sickle Cell Treatment Center, one of the sub-specialty clinics of the Children’s Specialty Center of Nevada.
Cure 4 The Kids: As a physician treating patients with sickle cell disease, what is the most problematic symptom for those affected?
Dr. Rashid: The most common problematic symptom of patients with sickle cell disease is pain. Pain occurs when the sickled blood cells are not able to move as freely in the blood vessels as they are sticky and start to clump together. This decreases the blood flow and causes pain. While some patients may go years without a painful episode, others may experience pain once or twice a month or more. A pain episode is treated with oral and/or intravenous or injectable pain medications, increased oral fluid intake or intravenous fluids and treatment of the trigger that caused the painful episode. Triggers include stress, fever, low oxygen levels (high altitudes), chilly temperatures, overdoing it, dehydration and acidosis (change in the blood chemistry).
The challenge is in the ability to measure the degree of pain and the know the best treatment to use. Pain is very subjective to the sufferer and unlike many other medical conditions, there is no objective way to measure the degree of pain that a person experiences. For physicians who do not understand sickle cell disease and who are not familiar with the patient, this has brought up a lot of misunderstanding and mismanagement of the pain. Especially in this current environment of ‘Opiate Epidemic”, there is great hesitance and increased regulation in prescribing opioids. This can increase the likelihood that a lot of patients suffering from pain are not getting the treatment that they need. The medical community must do better in addressing these issues.
Cure 4 The Kids: As I understand it, it’s been a while since there were new treatments approved for those with sickle cell disease. In July, the FDA approved something new called Endari. Is this a big deal for patients? Does this benefit all patients or just those with specific health issues?
Dr Rashid: Before we discuss the medication known as Endari, I just want to point out that currently we do have a medication that has had more than twenty years of data showing safety and efficacy in reducing the morbidity and mortality caused by sickle cell disease. This medication is Hydroxyurea. However, as sickle cell disease is a chronic condition, and that Hydroxyurea alone may not be the ‘Magic Drug”, we are very excited to have other options to offer our patients.
Endari is a brand name for L-glutamine powder which is used as an oral solution to treat sickle cell disease. It is the second FDA approved treatment for sickle cell disease and is approved for those aged 5 and older. Glutamine is an amino acid in our body that helps with many processes including energy and protein production. During a critical illness or when the body is stressed, the levels of glutamine decrease which can affect the body in several ways. By increasing the body’s stores of glutamine, it can increase the red blood cell integrity, increase energy and prevent red blood cell breakdown. It has also been shown to inhibit the sickling of red blood cells and decrease red blood cells from sticking. Research has also demonstrated fewer hospital visits per year. Since glutamine levels in those with sickle cell disease tend to be lower, it seems like a promising treatment. It can be given in conjunction with Hydroxyurea, the first FDA approved treatment for sickle cell disease. The most common side effects were constipation, nausea, headache, cough, pain in the extremities, back pain, chest pain and abdominal pain.
By the way, our center was part of the clinical trial for this medication with a few of our patient contributed directly to the success of the study. I just want to take this opportunity to thank them all!
Cure 4 The Kids: Anything else you think we should know about SCD?
Dr Rashid: Although sickle cell disease is a serious health condition, those with the disease can lead fulfilling lives. It is important to be followed by a hematologist or a primary care provider who understands and is knowledgeable about the management of sickle cell disease.
It is also important to continue to spread awareness of the disease because despite extensive efforts, there continues to be misconceptions spread that negatively affect the care and lifestyles of those with sickle cell disease. It is important to increase the number of providers that are well educated about sickle cell disease care, and to increase access to the best care for children and adults with sickle cell disease, no matter where they live or seek care in the region. We also need to raise awareness about the challenges that people with sickle cell disease among policy makers, the general community and healthcare providers.
To achieve these goals, the Children’s Specialty Center of Nevada Sickle Cell Treatment Center is a partner in the Pacific Sickle Cell Regional Collaborative which is funded through a grant from the U.S. Department of Health and Human Services, health Resources and Services Administration, Maternal and Child Health Bureau, Sickle Cell Disease Treatment and Demonstration Program.
We also wanted to get a patient’s perspective on sickle cell disease. We asked Beatrice to answer a few questions for us. She was diagnosed with sickle cell disease at the age of two, she is now 20 years old.
Cure 4 The Kids: Can you help us understand how sickle cell disease affects your everyday life? Or does it?
Beatrice: If you get sick, it can prevent you from doing things: working, going to school, hanging out with friends, etc. You always have to be cautious about swimming, extreme heat or extreme cold. You have to take precautions that others don’t have to take into account. You have to keep hydrated and be self-aware and take care of yourself.
Cure 4 The Kids: What do you want people to know about this disease?
Beatrice: There is not enough awareness of it. It doesn’t just affect African Americans. It is not contagious. It is not scary to live with as long as you have the right treatment.
Cure 4 The Kids: Do you feel the disease is understood by co-workers, or other students, or others in general?
Beatrice: It’s not understood. They don’t understand that you can get pain from blood sickling and can be out of school or work for weeks.