Economic Burden of Health Care Utilization for Paroxysmal Nocturnal Hemoglobinuria

Eculizumab is the current standard of care for the treatment of paroxysmal nocturnal hemoglobinuria (PNH); however, some patients continue to experience ongoing hemolysis and anemia, resulting in red blood cell transfusion dependence, unmet clinical needs, and economic burden. A real-world study presented at the 2020 ASH Annual Meeting observed an economic burden among patients with PNH treated with eculizumab, particularly among those dependent on blood transfusions.

The study included 151 patients aged ≥12 years from the IBM® MarketScan® Research Databases with two or more claims for eculizumab infusion between April 1, 2014, and Sept. 30, 2019. The index date was the first observed claim for eculizumab infusion with three or more months of continuous eligibility prior (baseline period). Patients were then stratified into transfusion-dependent (n=55; 36%; defined as one or more claims for blood transfusion within six months of any eculizumab infusion) and transfusion-free (n=96; 64%) cohorts.

Overall mean patient age was 36.7 years (range, 12-74), and 56% were female. At baseline, patients in the transfusion-dependent cohort received more blood transfusions (71.0% vs. 18.0%) and required greater use of corticosteroid therapy (46% vs. 32%).

During a mean overall observation period of 19 months, patients received a median of eight eculizumab infusions (range, 3-30) during the maintenance phase, and 61% of patients discontinued eculizumab. Median time to treatment discontinuation was 254 days.

Mean number of blood transfusions among transfusion-dependent patients was 8.5 (range, 1-54). During the observation period, patients in the transfusion-dependent cohort had 2.95 times more all-cause hospitalizations (159 vs. 66 hospitalizations) and 4.58 times more hospitalization days (1,490 vs. 377 days) compared with the transfusion-free cohort (P<0.05 for all). PNH-related hospitalizations were also more common in the transfusion-dependent group (87 vs. 26 hospitalizations; P=0.008), as well as hospitalization days (975 vs. 142 days; P<0.001).

“These findings suggest that the current PNH standard of care may be insufficient for transfusion-dependent patients,” the researchers concluded. “New therapies are needed to reduce the considerable burden of patients with PNH.”


Cheng WY, Sarda SP, Mody-Patel N, et al. Real-World Treatment Patterns and Healthcare Resource Utilization (HRU) of Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population. Abstract 3415. Presented at the 62nd American Society of Hematology Annual Meeting & Exposition, Dec. 2-11, 2020.