Despite readily available treatments for paroxysmal nocturnal hemoglobinuria (PNH), only around one-quarter of patients with the condition receive PNH-specific treatments, and patients with PNH wait an average of almost 5 months after diagnosis to begin to receive treatment, an analysis of claims data reports.
“Better care management and the introduction of new treatment options are needed to address delays between diagnosis and treatment, and high rates of hospitalization and emergency department use among patients with PNH,” Anem Waheed, MD, MPH, and colleagues wrote in Clinical and Applied Thrombosis/Hemostasis.
“PNH is a rare, acquired hematological disease that typically arises in young adults,” Dr. Waheed explains. “PHN leads to hemolytic anemia along with other debilitating signs and symptoms and is commonly treated with complement inhibitors. Our findings highlight that patients with newly diagnosed PNH have high healthcare utilization and appear to have delays in treatment initiation. Further education of patients and physicians is needed to improve PNH morbidity in our patients.”
Dr. Waheed and team investigated treatment patterns, healthcare resource utilization, and costs for patients with newly diagnosed PNH in two large health insurance claims databases, MarketScan and Optum.
Patients in the study had a valid ICD-10 diagnosis code of PNH (D59.9) in any position from April 2016 to December 2020, were 18 years or older when diagnosed, and had at least 6 months of continuous enrollment prior to the diagnosis date and after the diagnosis date. The researchers included all patients in all analyses, and used all available claims data.
The average age of 271 participants was 46.6, and 57.9% were female. Overall, 66 (25.1%) received any PNH-specific pharmacologic treatment, and the average time from diagnosis to treatment was 4.7 months (Table).
Notably, once patients received PNH-specific treatment, they were likely to be adherent while on therapy (97% medication possession ratio). Overall, 39.9% of patients had an inpatient stay, and 50.9% had an emergency department visit.
The authors acknowledge limitations to the study, including the small sample size and the study population limited to patients insured by private health plans or Medicare Advantage, which may not represent the national population, such as people covered by Medicaid and those without insurance.
“Healthcare resource utilization [HCRU] and costs remain high. . . . Nearly 1 in 5 newly diagnosed [patients with PNH] still require at least 1 blood transfusion, signaling some unmet medical need in PNH,” the authors wrote. “Future research should re-examine treatment trends and HCRU with additional data sources, particularly as new treatments become available to PNH patients.
Studies Reveal Areas in Need of Improvement
The results surprised Dr. Waheed because PNH treatments have been readily available since 2007. “Rare diseases are challenging to study outside the highly regulated environment of clinical trials,” she notes. “Real-world studies help highlight and uncover areas we need to improve so we can improve the lives of our patients.”
Many questions remain unanswered, she adds. “Given that this is a real-world claims based analysis, we don’t know why a large proportion of patients with PNH were not on therapy. We also don’t know why there was a higher than expected discontinuation of therapy.”
The authors plan further related research. “Given the high healthcare utilization of patients with newly diagnosed PNH, we plan to study complications of PNH in the realworld setting,” they wrote. “The field of complement-mediated diseases is changing rapidly, and it’s a very exciting time to see how we can improve the lives of our patients.”
