Elna Saah, MD: Unraveling the Current Landscape in Sickle Cell Disease

Sickle cell disease (SCD) impacts approximately 70,000 to 1000,000 people in the United States, making it the most common form of an inherited blood disorder. The condition is marked by the production of abnormal hemoglobin from birth, with complications of the disease leading to severe pain and other health complications, including infection, acute chest syndrome, and stroke.

In a new interview with HCPLive Hematology, Elna Saah, MD, a hematologist at the Emory University School of Medicine, described the public health concerns stemming from the condition and the importance of screening newborns to make a diagnosis immediately after birth.

“It’s a real big problem, a public health issue because it is progressive and complications continue to evolve as patients get older and older,” Saah told HCPLive. “In adults, there’s a lot of multiple organ involvement, which reduces the lifespan, causes earlier mortality and morbidity, and burdens both the patient and the healthcare system.”

Saah pointed to the variety, and potential disparity, in care resources between pediatric and adult populations with SCD. Children with SCD are typically treated by a general pediatric hematologist-oncologist, not necessarily an SCD specialist.

The care team also includes psychosocial support, including a social worker, psychologist, and teachers, to help manage trauma from the pain, as well as extensive support from advanced practice providers and nurse practitioners. As children reach adulthood, a transition is prepared for them to move from the pediatric side of care to adult providers.

However, according to Saah, the resources for SCD treatment are much more sparse and not as generous in adult care.

“We have fewer and fewer adult hematologists or hematologist oncologists who are willing and able and have the bandwidth to take on SCD patients,” Saah told HCPLive. “We also do not have a lot of supportive resources, including social workers and psychologists or neuropsychologists, to support this progressive disease and the patients end up with chronic pain.”

Saah also touched on the care landscape from the 1970s to the modern day, from early trial data indicating the proficiency of hydroxyurea to recent US Food and Drug Administration (FDA) approvals, including L-glutamine, Crizanlizumab, and Voxelotor. Other recent FDA approvals touched on included two gene therapies for SCD, including exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel) for children aged ≥12 years.

Saah also elaborated on recent data pointing to the unintentional negative effect of the 2016 Centers for Disease Control and Prevention on opioid prescriptions in patients with SCD. The release of the guidelines inadvertently curbed opioid prescribing for patients with SCD and those patients tended to seek hospitalization more frequently for their pain.

“I think we should take these results a bit more guarded, it is difficult to broadly analyze and extrapolate these Medicaid data on any individual patient,” Saah told HCPLive. “While we have seen that to be true in other patients, it behooves us to look for alternative support for long-term opioid and non-opioid prescribing in patients with chronic pain. It is unfortunate some patients did have opioids when they needed to have been withheld from them or delayed.”

Watch the full interview with Saah in the above video.

References

1. _{What is sickle cell disease? Centers for Disease Control and Prevention. July 6, 2023. Accessed April 18, 2024. https://www.cdc.gov/ncbddd/sicklecell/facts.html.}
2. _{Sickle cell disease. Hematology.org. Accessed April 18, 2024. https://www.hematology.org/education/patients/anemia/sickle-cell-disease.}
3. _{Treatment. National Heart Lung and Blood Institute. Accessed April 18, 2024. https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment}
4. _{Iapoce C. CDC opioid guidelines linked to negative consequences in sickle cell disease. HCP Live. March 12, 2024. Accessed April 18, 2024. https://www.hcplive.com/view/cdc-opioid-guidelines-linked-negative-consequences-sickle-cell-disease.}