Real-World Healthcare Resource Utilization (HRU) and Costs of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population

Abstract

Introduction: To evaluate the economic burden and treatment patterns of patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with eculizumab, a C5 inhibitor, who were defined as blood transfusion-dependent (TD) versus blood transfusion-free (TF) in the US population. Methods: Patients aged at least 12 years with at least two claims for eculizumab infusion (first claim was the index date) were identified from the IBM Ò MarketScan Ò Research Databases (April 1, 2014-September 30, 2019. The overall PNH eculizumab user cohort was stratified into the TD cohort (i.e., at least one claim for blood transfusion within 6 months following any eculizumab infusion, including on the infusion date) or the TF cohort (i.e., all non-TD patients). Treatment patterns, healthcare resource utilization (HRU), and costs were evaluated and compared during follow-up (i.e., index date to end of enrollment or data availability). Results: Of 151 patients in the overall cohort (mean age 36.7 years; 55.6% female), 55 were TD (mean age 35.1 years; 67.3% female) and 96 were TF (mean age 37.6 years; 49.0% female). A total of 61% of patients (TD, 66%; TF, 58%) discontinued eculizumab, with TD patients having a shorter median time to discontinuation (TD, 0.5 years; TF, 0.9 years). TD patients had more all-cause hospitalizations than TF patients (p \ 0.05). TD patients incurred higher all-cause direct medical costs (adjusted cost difference = $247,848) and medical-related absenteeism costs (adjusted cost difference = $4186) than TF patients (all p \ 0.05), largely driven by hospitalizations. Similar trends were observed for PNH-related HRU and costs. Conclusions: The economic burden of patients with PNH treated with eculizumab is greater among those dependent on blood transfusions.

The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors in France, Germany and the United Kingdom: Patient‐reported insights on symptoms and quality of life

“…The reported ranges for FACIT‐Fatigue and EORTC QLQ‐C30 scores were worse (greater impairment) than those of the general population, and nearly all patients reported impaired work productivity and activity. As trends were generally similar across all four subgroups, these data generally align with and expand upon previous studies suggesting persistent unmet need associated with C5i therapy 2,5,6,16,22,24,28 …”

“…To our knowledge, this is the first cross-sectional, multinational study of European patient-reported clinical and humanistic outcomes among As trends were generally similar across all four subgroups, these data generally align with and expand upon previous studies suggesting persistent unmet need associated with C5i therapy. 2,5,6,16,22,24,28 Overall, patient characteristics were generally consistent with baseline findings for the International PNH Registry population (at 2011 and 2017), as well as populations from retrospective observational studies and a counterpart survey study conducted in the United States. 2,5,6,28,37 Specifically, our observed overall Hb levels align with the most recent international registry data (median 10.0 vs. 9.8 g/dL) 2 ; median Hb levels for the eculizumab subgroup were also generally consistent with a real-world study of UK patients treated for at least 13 months (10.0 vs. 10.9 g/dL).…”

“…24 However, the proportion of women in the current study is somewhat higher than the range reported in the literature (66.2% vs. 53.0%-55.7%). 2,5,6,37 Notably, the companion US study population had an even higher proportion of women (73.0%). 28 This discrepancy may be attributable to recruitment through PAGs, which have predominantly female members.…”

“…The literature on clinical and humanistic outcomes associated with C5i therapies for PNH has described a high and persistent disease burden in routine practice 2,5,6,16,22,24,28 . However, evidence on the impact of persistent suboptimal haemoglobin levels and associated symptoms from the patient perspective remains scarce, especially outside US patient populations.…”

Real-World Eculizumab Dosing Patterns Among Patients with Paroxysmal Nocturnal Hemoglobinuria in a US Population

“…Further evidence regarding the HRU (eg, hospitalizations, outpatient visits) and direct medical costs among eculizumab-treated PNH patients is also warranted. 28 In particular, there is a need for comparative assessments of the economic and clinical outcomes among eculizumab-treated patients who received a regularly scheduled dose relative to those with deviations from label-recommended dosing, including those who required dose escalation.…”