Living with Sickle Cell Disease: Treatment from Birth to Adulthood

Panelists:
Santonio Holmes, President, Ill and Long Foundation, NFL Wide Receiver, Super Bowl 43 MVP
Dr. Peter A. Lane, MD, Director, Sickle Cell Disease Program Childrens Healthcare of Atlanta
Dr. Julle Kanter, MD, Director, Lifespan Comprehensive Sickle Cell Center, Medical University
of South Carolina Childrens Hospital
Charles Adams, MSW, Social Worker, Division of Hematology, The Childrens Hospital of
Philadelphia
Dr. Allstair Abraham, MD, Attending Physician, Division of Blood and Marrow Transplantation,
Childrens National Health System.
On Thursday, June 23rd, the Congressional Sickle Cell Caucus hosted a briefing discussing the
challenges that patients with Sickle Cell Disease face and what programs, funding, and
legislation may help to alleviate some of these challenges.
Sickle Cell Disease:
Sickle cell disease (SCD) is the most common blood inherited disorder with approximately
100,000 Americans currently being affected by the disease and 2,000 new cases being diagnosed
annually. Sickle cell disease disproportionally effects communities of color with approximately
90% of cases being found in African-Americans and 10% in Latinos.
Sickle cell is a genetic disease that cause a malfunction in a persons red blood cells caused by the
presence of the abnormal hemoglobin S. The sickle hemoglobin gives the cells the appearance of
a sickle that the disease is named after. The abnormal shape of the cells causes low hemoglobin
levels which make carrying oxygen to the body difficult. Additionally, these blood cells also
have more difficulty moving through the blood stream. Complications associated with sickle cell
include severe pain, stroke, infection, and organ damage.
Sickle cell disease is generally treated through a combination of medication and blood
transfusions. While effective at reducing pain and possible complications of sickle cell disease,
these treatments are by no means cures for the disease. Currently, the only cure for sickle cell is a
bone marrow transplant from a compatible donor.
Living with Sickle Cell: Challenges and Proposed Solutions
Treatment options:
The complications and treatment options that are associated with SCD can make living stable
lives difficult for patients with sickle cell disease. SCD requires constant medical treatment from
a variety of medical professionals including emergency room visits and SCD specialists. But
most emergency room doctors are unable to treat SCD effectively. Additionally, the number of
SCD specialists are dangerously low. So while a patient may have insurance to cover the cost of
medical treatment, they do not have access to a doctor to treat them. In response to this problem,
Dr. Kanter stated that MUSC has been developing a caregiver program that would redistribute
SCD doctors in areas where they are needed so that patients could have access to a doctor

reasonably close to where they reside. In addition to these efforts, the Congressional Sickle Cell
Disease Caucus recently wrote a letter to the CMMI asking them to allocate additional funding to
state programs that improve outpatient care for those who have SCD.
Education and job opportunities:
Additionally, living with SCD may also be difficult for patients due to their frequent need for
long term hospital stays and medical appointments. This often makes it difficult for patients to
complete their education or maintain a job without reasonable accommodations. In response to
this problem, The Childrens Hospital of Philadelphia has implemented a program wherein which
patients with SCD (and other diseases that may be disruptive to daily life) are able to get jobs or
attend schools at partner institutions that are more understanding to their unique situations. These
new programs are crucial in allowing patients with SCD to live stable lives.
Cures:
Currently, the only cure for SCD is a bone marrow transplant from a compatible donor. However,
few patients are able to utilize this option. First, it is very rare for a patient to find a compatible
family donor who does not also have SCD. Even if a patient is able to find a donor, most doctors
will only agree to perform the procedure on children. This leaves little hope for adults who are
diagnosed with SCD. But, spurred by public outcry, more research is currently being conducted
into ways that bone marrow transplants could be performed in adults. The STRIDE Trial is a
program that is currently working to perform bone marrow transplants for adults who have an
eligible bone marrow donor. Dr. Abraham expressed the sentiment that he hopes that within
several years bone marrow transplants could become more common among adults diagnosed
with SCD.
In addition to improving current cures, Dr. Abrahams also spoke about the new research being
done to develop new cures for SCD. While only in its initial stage of development and research,
Dr. Abrahams is hopeful that gene therapy can be utilized to actually cure the defective genes in
SCD patients.