For patients with paroxysmal nocturnal hemoglobinuria (PNH) who receive eculizumab, transfusion dependence is associated with greater health care resource utilization (HRU) and costs than transfusion independence is. This is according to the results of a new study published in the journal Advances in Therapy.

This retrospective study was based on an analysis of patients with PNH who had records for eculizumab infusions in the IBM® MarketScan® Research Databases from between April 1, 2014, and September 30, 2019. Patients were stratified for analyses according to whether they were transfusion dependent (TD) or had transfusion-free (TF) status. TD status was based on having received 1 or more blood transfusions in the 6 months after an eculizumab infusion. The researchers compared outcomes between the 2 cohorts based on treatment patterns, HRU, and costs over a follow-up period.

A total of 151 patients were identified, with 55 (36.4%) having TD status and 96 (63.6%) having TF status. The mean age for the TD cohort was 35.1 years (median 30.0 years), and for the TF cohort it was 37.6 years (median 39.5 years). Patients with TD status showed a higher mean Quan-Charlson comorbidity index score (mean score, 1.2) compared with patients who had TF status (mean score, 0.9). Aplastic anemia was also more common in the TD patients (63.6%) than in TF patients (42.7%).

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The mean observation period for each cohort was 1.6 years. Discontinuations of eculizumab were reported in 66% of TD patients and 58% of TF patients. The TD cohort had a median time to discontinuation of 0.5 years, compared with 0.9 years for the TF cohort.

All-cause hospitalizations were 2.95 times more common in the TD cohort than in the TF cohort (95% CI, 1.42-5.44; P =.002), and PNH-related hospitalizations were 2.99 times more common for TD patients (95% CI, 1.31-6.39; P =.008). Lengths of stays for all-cause and PNH-related hospitalizations were also significantly longer for patients in the TD cohort.

All-cause direct medical costs were higher for TD patients, with an adjusted cost difference between cohorts of $247,848 (95% CI, $87,350-$445,161; P =.004). All-cause hospitalization, PNH-related hospitalization, PNH-related comorbidity, and PNH-related symptom costs were also significantly higher with the TD cohort. PNH-related total medical costs were not significantly different between cohorts, however (P =.258).

Additionally, medical-related absenteeism was associated with greater costs for TD patients. On an all-cause basis the adjusted cost difference between cohorts for this metric was $4186 (95% CI, $2155-$6217; P <.001). This difference was mostly related to hospitalizations.

The study researchers concluded that in this study over one-third of patients with PNH receiving eculizumab remained in TD status and that this status was associated with substantial HRU and costs. “Taken together, these findings suggest that the current PNH standard of care may be insufficient,” the researchers wrote in their report.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Cheng WY, Sarda SP, Mody-Patel N, et al. Real-world healthcare resource utilization (HRU) and costs of patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving eculizumab in a US population. Adv Ther. 2021;38(8):4461-4479. doi:10.1007/s12325-021-01825-4