Patients with sickle cell disease using disease-modifying therapies more, but not enough
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Key takeaways:
- Most patients with sickle cell disease are not using disease-modifying therapies.
- DMT use increased nearly 10% between 2014 and 2021 due to approval of three new medications.
Nearly three-quarters of individuals with sickle cell disease are not using disease-modifying therapies in their treatment, according to a study published in JAMA Network Open.
Usage increased over the past decade with the approval of new disease-modifying therapies (DMTs) — L-glutamine, crizanlizumab (Adakveo, Novartis) and voxelotor (Oxbryta, Global Blood Therapeutics) — to go along with hydroxyurea, but clinicians have not prescribed them optimally, researchers concluded.
“DMTs play an important role in the management of sickle cell disease, yet our findings show they remain underutilized,” Terri Victoria Newman, PharmaD, MS, assistant professor of pharmacy and therapeutics at University of Pittsburgh’s School of Pharmacy, told Healio.
“Ensuring timely, appropriate access to these therapies is essential to realizing the full value of these treatments in clinical practice,” she added. “Investigating the multi-level factors that contribute to underutilization can help more patients benefit from these important disease-modifying options.”
Background and methods
Sickle cell disease impacts about 100,000 people in the United States, and DMTs can prevent, reduce and improve complications, according to background data provided by the study’s investigators.
Hydroxyurea, the only DMT approved for sickle cell treatment prior to 2017, reduced vaso-occlusive crises (VOCs) by 44%, in addition to reductions in hospitalizations and death.
Newman and colleagues used Optum’s deidentified Clinformatics Data Mart Database to conduct a cross-sectional study examining “characteristics that may be associated with DMT use and to describe observed patterns of yearly DMT,” they wrote.
Researchers divided patients from Jan. 1, 2014, to Sept. 30, 2021, into four groups — inconsistent DMT users, incident DMT users, consistent DMT users and non-DMT users.
Inconsistent users filled at least one prescription in the 6-month pre-index period and none post-index. Incident users did the opposite. Consistent users had one fill in both indexes and nonusers had none.
Results and what’s next
In a sample of 5,022 patients, 73.9% did not use DMTs, 17.8% consistently used them, 5.5% used them incidentally and 2.9% used them inconsistently. Women made up most of the study population (58.3%) and patients aged 18-45 years old comprised the largest segment (41.4%).
“Nearly 75% of patients with sickle cell disease do not receive DMTs is highly representative of the unmet need in this population,” Newman said.
Inconsistent DMT users had the highest rates of acute chest syndrome, VOCs, splenic and pulmonary complications, and kidney disease. Non-DMT users had the lowest rates of acute chest syndrome and VOCs, but the highest for diabetes and hyperlipidemia. Results varied between the four groups for multiple diseases and complications.
In a second sample of 6,387 patients, total DMT use rose from 19.6% in 2014 to 28.3% in 2021.
“Further research springing from this article should aim to uncover barriers and facilitators to the use of DMTs to uncover the various patient, provider, and health system factors that affect DMT use,” Newman said. “Additional research should also focus on specific medication access barriers that hinder optimal use of DMTs.
“Additionally, there is a need for more robust real-world and clinical evidence demonstrating the clinical, economic, and safety outcomes of DMTs. This includes conducting long-term evaluations and using representative samples that reflect and capture various sickle cell disease types, phenotypic expressions, geographic locations, and more.”
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