Clinical research is booming! There are currently over 270,000 active studies on https://clinicaltrials.gov. As a research coordinator, I want to discuss the need for participating in research initiatives for Sickle Cell Disease (SCD), as well as what is involved.
As of today, there are about 500 studies devoted specifically to Sickle Cell Disease (SCD) which is a higher number than in the past. What does this mean for people with SCD?
This means that pharmaceutical companies are not only interested in SCD, but they are actively funding research to find new drugs to treat it. Currently, there are only a couple of drugs approved for use to help prevent most SCD complications. If you cannot use those medications then management of the disease can become more difficult. In order to generate more medication options for SCD, more individuals have to enroll in research studies.
It’s not as simple as approving a new drug for use. Research medications must be evaluated on how well they work in humans. Research is heavily regulated and monitored by organizations like the US Food and Drug Administration (FDA) and local Institutional Review Boards (IRBs), so every drug approved for human use, even Ibuprofen and cough syrup, has been tested on a human subject. Enrolling an individual in a research trial requires several steps. First, anyone who wants to enroll in a study has to fit the guidelines for who can participate, otherwise known as eligibility guidelines. Second, the informed consent form (ICF) must be reviewed. The ICF is a document that explains, in layman’s terms, the study procedures, risks/benefits, requirements to participate, and what is hoped to be gained from the study. These documents go into great detail to eliminate questions and concerns about what will take place during the study. The ICF is reviewed, page by page, before consent to participate is obtained. The most important thing to remember is that consent is voluntary, non-binding, and can be withdrawn at any time. After the patient provides consent, he/she must complete a screening process to validate the person’s eligibility and have multiple clinic visits for labs and routine monitoring.
In other words, research requires a lot of steps!
As a research coordinator, I love what clinical trials provide for medicine. Hopefully, this has sparked interest for others as well. For more information, contact Adrianna, IHTC Research Coordinator, at 317-871-0000.
What is Sickle Cell Disease?
Sickle cell disease is a disease of the hemoglobin. Hemoglobin is what is inside your red blood cells, carrying oxygen all over your body. Most people have what is called Hemoglobin A, which is shaped like a fist and keeps your red blood cells round and squishy. These red blood cells are able to move through your blood vessels easily.
People with sickle cell disease have Hemoglobin S. Hemoglobin S is shaped like a rectangle, and it stretches out your red blood cells (see image below). These stretched out cells (also called sickle cells) are rigid, sticky, and fragile. They get stuck in your blood vessels, and can damage your organs.
Some people have sickle cell trait. A person with sickle cell trait has Hemoglobin A and Hemoglobin S. Since they have Hemoglobin A, they usually don’t have health problems from the Hemoglobin S.
There are other kinds of hemoglobin besides Hemoglobin S and Hemoglobin A. Some people make Hemoglobin C or E, or another unusual type. These types can also be combined with Hemoglobin S to cause different types of sickle cell disease.
How do you get sickle cell disease?
Sickle cell disease is an inherited disease, which means you get it from your parents. In order for a person to have sickle cell disease, at least one of their parents must have sickle cell trait. Their other parent must have sickle cell trait or another hemoglobin trait.
A person with a hemoglobin trait makes two kinds of hemoglobin, but they can only pass down one kind to their child. If two parents both have sickle cell trait (Hemoglobin AS), they can each only pass down one kind of hemoglobin. If one parent passes down Hemoglobin A and the other passes down Hemoglobin S, their baby will have trait. If they both pass down Hemoglobin A, their baby will just make the usual hemoglobin. If they both pass down Hemoglobin S, their baby will have sickle cell disease (Hemoglobin SS).
People can get other types of sickle cell disease if one of their parents passes down Hemoglobin S, and the other parent passes down another unusual hemoglobin, like Hemoglobin C. This person would have Hemoglobin SC.
There are many different types of hemoglobin. It’s important to know what type you have, and what type your partner has.
On January 29, members of the Indiana Sickle Cell Advisory Committee and the Indiana Sickle Cell Consortium participated in Sickle Cell Advocacy Day at the State Capitol. Several IHTC staff members and patients attended. The focus of the day was to educate legislators on the need for funding for programs and services for adults in Indiana with Sickle Cell Disease. Currently, the state funds two programs at IHTC for patients: the Sickle SAFE Program, for ages 0-3, and the SCORE Program, for ages 3-21. IHTC staff members and other consortium members agree that the state needs to do more for adults. The day’s agenda included speeches from several stakeholders in the sickle cell community, including patients, parents of patients, medical professionals, and even Representative Greg Porter, who has long been a sickle cell champion. Several other legislators visited the event or sent their staff members to gather information. We have plans to make 2019 Advocacy Day even bigger and better! Keep watching this space for more information!
A person inherits genes that produce hemoglobin from his/her parents. It is important to identify people with hemoglobin trait so they will be aware of their risk of having children with sickle cell disease. If one parent has sickle cell trait and the other parent has normal hemoglobin, there is a 50 percent chance with each pregnancy that the child will be born with sickle cell trait. If both parents have sickle cell trait or another hemoglobin trait, there is a 25 percent chance with each pregnancy that the child will have sickle cell disease or another hemoglobin disease.
People with sickle cell trait usually do not have any disease symptoms. However, it is possible for a person with sickle cell trait to have complications of the disease under extreme conditions, such as:
I had some familiarity with Sickle Cell Disease (SCD) when my son was born in November 1996. I was told that he had Sickle Cell Trait (SCT), which he inherited from me. SCD became a part of my family December 25, 2002 with the birth of my twin daughters. One of my daughters had the trait and the other had the disease (HbSS). I didn’t know much about the disease at that time, but I would soon become inundated with information through independent learning and education by doctors and others in the sickle cell world.
At that office visit, we received the name of a sickle cell educator, a referral to the hematology clinic at Riley Hospital for Children, and an address/invite to the Martin Center’s sickle cell support group. Our family was armed and ready for battle! We did not know it at the time, but these resources would prove to be extremely valuable to us and ultimately part of our extended family. They were our front-line supporters as my daughter began to experience complications related to SCD, endure hospitalizations, and require extensive care and support.
My purpose for sharing this backdrop is to encourage anyone reading this article to make sure that there is proper coordination of care for any sickle cell patient in your life: your child, your patient, your friend, your student, or anyone else. This begins with having the right team on board to support the patient’s needs.
My daughter’s team is extensive, beginning with our biological family and spiritual support. Her medical team includes her primary care physician, dentist, and hematologists at the Indiana Hemophilia and Thrombosis Clinic (IHTC). She also has access to wrap-around care provisions at IHTC including Physical Therapy, Dental Hygienist, Phlebotomy, Dietary, and Social Services. Her hematologists collaborate with other service providers including pulmonary care at Riley Hospital, optometry care, and radiology for her annual Transcranial Doppler scans, just to name a few. IHTC is also the bridge between home and school, for educating and collaborating with school nursing staff and teachers to ensure our daughter has the best support possible.
As you can see, this team is extensive. The number one best advocate for a child with SCD is the parent or caregiver with whom the child resides. This advocate must always be alert to ensure the care team is aware and ready. Recently, my daughter’s dentist referred her to an endodontist to remove all four of her wisdom teeth. We consulted with the endodontist and scheduled the surgery. Immediately after the office visit, I reached out to three people at IHTC: her hematologist, her primary nursing contact, and our social/educational advocate to advise them of the upcoming surgery and understand their requirements from a sickle cell perspective.
I quickly learned that care coordination is critical. Her hematologist reached out to me immediately with questions and copied the dental hygienist on staff. They both followed up with me again, then reached out to the endodontist directly. Timing was great because my daughter had a clinic appointment coming up before the dental surgery. We were able to have an on-site consult with her IHTC care team, and receive resources/instructions for her post-op care. This resulted in a smooth process and recovery.
Don’t take anything for granted. Proper care coordination is critical to ensure that those with Sickle Cell Disease have the best available treatment, care, support, and possibility for effective recovery.
Jacquelyn Robinson, MSM, M.Ed., PHR, GPHR
Parent and advocate for Sickle Cell patients and families; founder of SCACURE Networks, Inc.
People with sickle cell disease (SCD) make hemoglobin S. This hemoglobin stretches out the red blood cells (also called “sickling”). Sickled red blood cells have trouble moving through the blood vessels and can cause pain and organ damage. Hydroxyurea helps people with SCD by increasing the amount of Hemoglobin F in the body. Hemoglobin F helps keep red blood cells from sickling, so it can prevent some problems caused by SCD.
Hydroxyurea has been studied for decades to see how it can help people with SCD. There are several benefits that have been discovered. These include:
Hydroxyurea has been used in patients with SCD for over 30 years. Research has shown that hydroxyurea is a safe and effective medication. People with SCD can start taking it as young as 9 months of age. If you have questions about hydroxyurea, please talk to your or your child’s hematologist.
Dr. Clark Kramer
September is National Sickle Cell Awareness Month. We honor and hope for all the #SickleCellWarriors who strive and survive and thrive every day.
Did you know the Partners Program hosts CME accredited sickle cell disease (SCD) modules, free to the public? The website, www.PartnersPRN.org, hosts three CME activities covering the issues facing adolescents and young adults (AYAs) with SCD and the phase of transitioning from a pediatric care model to an adult care model, a period associated with significantly increased risk for care discontinuity, acute complications, and death.
Authored by Dr. Emily Riehm Meier, Pediatric Hematologist and Sickle Cell Research Director at the Indiana Hemophilia and Thrombosis Center in Indianapolis, these CME activities detail the most important and modifiable barriers to better outcomes among AYAs with SCD: underutilization of hydroxyurea, lack of comprehensive care for these patients, and need for structured transition programs to shepherd patients through the transition period.
Would you like to gain more education about these important and timely topics - and earn free CMEs?
These CME activities offer a patient-centered and practical approach to managing the care and transition of AYAs with SCD with a vision toward extending life expectancy, improving quality of life, and increasing competency among care providers for persons with SCD.
Learn more by clicking the link. To complete an activity, you must be registered and logged in.
Since 1983, the United States government has recognized September as Sickle Cell Awareness Month. In the thirty-four years since, a great deal of progress has been made with regards to research and treatment. In fact, activity in these two combined areas has led to a doubling of the average life expectancy for Sickle Cell patients in the United States. That is obviously great news that we can all appreciate and salute. We are making great strides at an accelerated rate and with the advent of the new CRISPR technology (https://www.yourgenome.org/facts/what-is-crispr-cas9), we stand on the verge of having a cure that, one day, can be accessible to more patients than stem cell transplants. But that day is still in the future.
The Sickle Cell community applauds these advances, but we need to remain focused on those individuals that face day after day of unrelenting pain, missed life opportunities and burdensome financial challenges. We must remain fixated on the needs of patients and families who face lives of uncertainty and recurrent flirtations with despair. And we should remain determined to do everything in our power to mitigate and/or minimize the many compounding issues that affect the members of our Sickle Cell community. We are not alone in this and we remain grateful that, now more than ever before, Sickle Cell advocacy and activism is at an all-time high.
This brings us to an important point. For thirty-four years we have heard the words “Sickle Cell Awareness.” Awareness is only one part of the solution. Even so, it continues to amaze us that we still have so much awareness work to do. Too many people still don’t know much about Sickle Cell. A nursing student recently said, “I wish they would have spent more time on Sickle Cell. One class. That’s all we had.” This young woman was aware of Sickle Cell but she really didn’t know how to care for people who have it. This is just one example of why we need more than awareness to make a meaningful impact on this illness.
We need to combine increased awareness with increased action. Whether it is institutionalizing a way that ensures that healthcare providers undergo more intensive training on Sickle Cell care or creating increased opportunities for Sickle Cell patients to access higher levels of education and employment, we need to take action that can make a difference. Awareness can’t do that by itself. It takes action to make things change.
Gary Gibson; President/CEO
Martin Center Sickle Cell Initiative
How important is community connection? Are there benefits to feeling like you're part of a group? The Indiana Sickle Cell Consortium (ISCC) believes that a sense of community is important for everyone’s wellbeing, especially those with a chronic condition. Connecting with other people who can relate to the ups and downs of living with a chronic condition help ensure you don’t feel like you’re alone. That's why consortium members work to connect those affected by sickle cell to others with similar experiences.
Our most recent community outreach project involved ISCC booth representation at the INShape Indiana Black and Minority Health Fair from July 13th to 16th. At this event we had the opportunity to talk to over 1,000 people about sickle cell disease and the importance of getting tested for sickle cell trait. Several people with sickle cell disease volunteered to staff our booth, and this was a chance for them to talk about their experiences with health fair attendees.
What’s the next event in the pipeline? The 9th Annual Sickle Cell-abration on September 30, 2017! (Check out event details here: https://2017sicklecellabration.eventbrite.com). This event, which includes education about sickle cell disease, will also feature a fashion show. Fashion show models will all be people with sickle cell disease. The fashion show will be a chance for patients to share their experiences and meet other people who are living with or affected by sickle cell disease.
Feeling part of a larger community is important for those who are living with a chronic illness. The ISCC is striving to help people living with sickle cell create that community for themselves. If you're interested in volunteering or participating in any of our events contact the consortium at ISCC@ihtc.org.
Ellen Bloom, MPH, CHES; Sickle SAFE Program Coordinator
The Indiana Hemophilia & Thrombosis Center