People with sickle cell disease seldom receive newer pain medications
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Key findings:
- Only 31.5% of patients with vaso-occlusive pain received prescriptions for hydroxyurea, followed by l-glutamine (3.2%) and crizanlizumab (2.3%).
- Women had increased odds of hydroxyurea use (OR = 1.27; 95% CI, 1.21-1.32).
Fewer than 4% of eligible individuals with sickle cell disease received prescriptions for newer FDA-approved pain medications, according to study results published in Blood Advances.
Further studies are needed to identify obstacles and improve patients’ access to these drugs, researchers wrote.
Rationale and methodology
“We are in a new era in the treatment of sickle cell disease with multiple new medications that have been FDA approved since 2017,” Robert M. Cronin, MD, MS, associate professor in the department of internal medicine and biomedical informatics at The Ohio State University College of Medicine, told Healio. “While clinical trials have demonstrated the efficacy of these new medications for reducing acute vaso-occlusive pain episodes, we don’t know what their usage is in the real world.”
Uptake of hydroxyurea, the only FDA-approved treatment for sickle cell disease until 2017, has been disappointingly low despite its ability to reduce disease-related complications and mortality, Cronin added.
“We therefore wanted to determine whether these new medications that can lower the number of pain episodes also have poor uptake, and to test our hypothesis that there are differences in usage of these new medications based on demographics and geographic location,” he said.
The study included 7,957 individuals (median age, 37 years; 61.2% women; 16% super-rural) identified in the IBM Watson MarketScan administrative billing databases between 2016 and 2020.
All individuals had three encounters with the health care system for a sickle cell disease diagnosis and a minimum of 1 year of continuous enrollment during the study period.
The use of hydroxyurea, l-glutamine (Endari, Emmaus Medical), voxelotor (Oxbryta, Global Blood Therapeutics) and crizanlizumab (Adakveo, Novartis) — alone or concurrently — served as primary outcomes.
Findings
Overall, 57% of the study population experienced two or more pain episodes annually.
Yet, only 31.5% of those with vaso-occlusive pain received prescriptions for hydroxyurea, followed by l-glutamine (3.2%) and crizanlizumab (2.3%). Moreover, only 2.9% of individuals with lower hemoglobin levels received prescriptions for voxelotor.
A similarly low proportion of patients used combination therapy to alleviate vaso-occlusive pain, and a mere 0.3% used combinations of newer therapies.
“It was astonishing how rarely these new FDA-approved medications are being used,” Cronin said. “We also saw disparities in usage of these medications based on age and geographic location.”
For every 10-year increase in age, patients had decreased odds of hydroxyurea, l-glutamine and crizanlizumab use. However, researchers observed increased odds of hydroxyurea use among women (OR = 1.27; 95% CI, 1.21-1.32).
Those who did not reside in a super-rural geographic location, defined as outside of a metropolitan statistical area that incorporates over 80% of the U.S. population, had nearly three times higher odds of crizanlizumab use (OR = 2.93; 95% CI, 1.16-7.42).
“Crizanlizumab is an infusion that typically occurs at an infusion center, likely leading to access issues for the rural community,” Cronin said.
Implications
The findings highlight the need for physicians to familiarize themselves with the new medications and discuss them with their patients with sickle cell disease, Cronin told Healio.
“Physicians should also educate and empower their sickle cell population with information about these medications so they can better manage their disease and advocate for themselves,” he said. “Because certain groups have more challenges getting these medications, physicians should help patients with sickle cell disease access these new medications.”
Further research is needed to address the knowledge gap of which combination treatment, if any, reduces vaso-occlusive pain episodes compared with hydroxyurea therapy alone and evaluate the cost-effectiveness of combination therapy, Cronin added.
“It is also imperative that we identify the obstacles and improve access to these new FDA-approved medications for sickle cell disease in the real world,” he said.
For more information:
Robert M. Cronin, MD, MS, can be reached at robert.cronin@osumc.edu.
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