Few Sickle Cell Disease Patients Are Adequately Treated for Pain

Despite the availability of medications designed to prevent vaso-occlusive events in persons with sickle cell disease (SCD), few who experience these chronic pain episodes have prescriptions for these drugs, researchers found.

Using data from an administrative claims database, less than 4% of patients with SCD who are eligible for these medications were found to have prescriptions for either L-glutamine (Endari), crizanlizumab (Adakveo), or voxelotor (Oxbryta), all of which have been FDA approved since 2017, reported Robert Cronin, MD, of the Ohio State University in Columbus, and colleagues.

Furthermore, less than one-third of SCD patients had prescriptions for hydroxyurea, a drug commonly used to reduce pain caused by the disease, they noted in Blood Advances.

“We know that the use of hydroxyurea has been disappointingly low, despite its ability to lower sickle cell disease complications and reduce mortality,” Cronin told MedPage Today. “We wanted to see whether these new medications proven to lower the number of pain episodes also have poor uptake. It is astonishing how rarely these new FDA-approved medications are being used.”

“We have new FDA-approved medications that are clinically proven to be efficacious at lowering the number of pain episodes in people with sickle cell disease, but less than 5% of the people who would benefit from these medications are getting them,” he added.

The researchers reported that for every 10-year increase in age, patients had decreased odds of using hydroxyurea, L-glutamine, and crizanlizumab, and women had increased odds of hydroxyurea use only (OR 1.27, 95% CI 1.21-1.32). Patients with a higher Charlson Comorbidity Index had increased odds of using all medications.

Moreover, “unlike other medications included in this study, not being in a super rural geographic location was associated with approximately three times higher odds (OR 2.93, 95% CI 1.16-7.42) of utilizing crizanlizumab,” they noted.

“This discrepancy is a failure of the healthcare system to deliver adequate care to this often-neglected population that faces significant healthcare disparities,” Cronin said. “We need to better understand the obstacles to accessing these medications and develop strategies and interventions to overcome them.”

He added that there are a number of possible explanations for the low utilization of these medications. “Briefly, provider or patient preference and treatment characteristics may limit access to them, lack of efficacy data on combination medications may limit access of providers prescribing, the COVID-19 pandemic may have interrupted access to in-person clinic appointments and supply chain disruptions, and the cost of these new treatments is high.”

“However, one that stuck out most was the access issue of crizanlizumab, which was three times less likely to be prescribed in super-rural areas. Crizanlizumab is an infusion that typically occurs at an infusion center, likely leading to access issues for the rural community,” he noted.

For this analysis, Cronin and colleagues used 2016-2020 data from the MarketScan claims database to identify 7,957 patients who had visited a healthcare provider at least three times and had confirmed SCD. Median age was 37 years, 61.2% were women, 16.0% were classified as super-rural (not living within a metropolitan statistical area that incorporates more than 80% of the U.S. population), and 57% had two or more pain episodes annually.

They assessed the use of hydroxyurea, L-glutamine, voxelotor, and crizanlizumab, both alone and concurrently, by age, sex, and geographic location.

Cronin and colleagues noted that use of administrative databases can result in data quality issues, including missing information.