TY - JOUR
T1 - Cost burden of breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria receiving ravulizumab versus eculizumab
AU - Tomazos, Ioannis
AU - Sierra, J. Rafael
AU - Johnston, Karissa M.
AU - Cheung, Antoinette
AU - Brodsky, Robert A.
AU - Weitz, Ilene C.
N1 - Funding Information:
The authors thank all the patients and investigators who participated in and contributed to this study. Medical writing and editorial support were provided by ApotheCom (Yardley, PA, USA) and was funded by Alexion Pharmaceuticals, Inc. (Boston, MA, USA). The content of this article, the ultimate data interpretation, and the decision to submit it for publication in Hematology were made by the authors. Editorial support/critical review was provided by Gabriela Marcheva, PharmD, at Alexion Pharmaceuticals, Inc.
Funding Information:
This study was funded by Alexion Pharmaceuticals, Inc. The authors thank all the patients and investigators who participated in and contributed to this study. Medical writing and editorial support were provided by ApotheCom (Yardley, PA, USA) and was funded by Alexion Pharmaceuticals, Inc. (Boston, MA, USA). The content of this article, the ultimate data interpretation, and the decision to submit it for publication in Hematology were made by the authors. Editorial support/critical review was provided by Gabriela Marcheva, PharmD, at Alexion Pharmaceuticals, Inc.
Publisher Copyright:
© 2020 Alexion Pharmaceuticals, Inc. Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2020/1/1
Y1 - 2020/1/1
N2 - Objectives: Although complement inhibition is highly effective, patients with paroxysmal nocturnal hemoglobinuria (PNH) may experience intravascular breakthrough hemolysis (BTH). Underlying causes may include elevated free C5, pregnancy, or non-pregnancy complement-activating conditions (e.g. infections). This study compared BTH-related resource utilization and costs in PNH patients treated with eculizumab versus ravulizumab. Methods: A cost model was developed using data from a targeted literature review and a survey of experienced clinicians. Costs associated with BTH episodes were calculated by cause and weighted by the proportion attributed to each cause and the cost of treating each episode. The model captured direct medical costs in 2018 US dollars. Annual BTH-related healthcare resource utilization was also calculated. Results: BTH episodes in the literature were commonly associated with elevated lactate dehydrogenase and aspartate aminotransferase, hemoglobinuria, transfusion needs, and/or recurrence of PNH symptoms. The majority of BTH management costs in eculizumab-treated patients related to changing from the approved dosing regimen following an episode of BTH, rather than acute management. No ongoing dosing changes were expected for ravulizumab-treated patients following episodes of BTH, substantially reducing its ongoing management costs. Resource utilization was greater for eculizumab-treated patients than ravulizumab-treated patients due to higher incidence of BTH, and risk of elevated free C5-related BTH. Total incremental cost was substantially lower for ravulizumab- vs eculizumab-treated patients ($407 vs $9379); results were consistent when pregnant women were not included ($386 vs $3472). Conclusion: Overall resource use and costs for BTH are estimated to be lower for PNH patients receiving ravulizumab compared with eculizumab.
AB - Objectives: Although complement inhibition is highly effective, patients with paroxysmal nocturnal hemoglobinuria (PNH) may experience intravascular breakthrough hemolysis (BTH). Underlying causes may include elevated free C5, pregnancy, or non-pregnancy complement-activating conditions (e.g. infections). This study compared BTH-related resource utilization and costs in PNH patients treated with eculizumab versus ravulizumab. Methods: A cost model was developed using data from a targeted literature review and a survey of experienced clinicians. Costs associated with BTH episodes were calculated by cause and weighted by the proportion attributed to each cause and the cost of treating each episode. The model captured direct medical costs in 2018 US dollars. Annual BTH-related healthcare resource utilization was also calculated. Results: BTH episodes in the literature were commonly associated with elevated lactate dehydrogenase and aspartate aminotransferase, hemoglobinuria, transfusion needs, and/or recurrence of PNH symptoms. The majority of BTH management costs in eculizumab-treated patients related to changing from the approved dosing regimen following an episode of BTH, rather than acute management. No ongoing dosing changes were expected for ravulizumab-treated patients following episodes of BTH, substantially reducing its ongoing management costs. Resource utilization was greater for eculizumab-treated patients than ravulizumab-treated patients due to higher incidence of BTH, and risk of elevated free C5-related BTH. Total incremental cost was substantially lower for ravulizumab- vs eculizumab-treated patients ($407 vs $9379); results were consistent when pregnant women were not included ($386 vs $3472). Conclusion: Overall resource use and costs for BTH are estimated to be lower for PNH patients receiving ravulizumab compared with eculizumab.
KW - Breakthrough hemolysis
KW - anti-C5 monoclonal antibody
KW - complement inhibitor
KW - eculizumab
KW - healthcare resource utilization
KW - medical costs
KW - paroxysmal nocturnal hemoglobinuria
KW - ravulizumab
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UR - http://www.scopus.com/inward/citedby.url?scp=85090002656&partnerID=8YFLogxK
U2 - 10.1080/16078454.2020.1807226
DO - 10.1080/16078454.2020.1807226
M3 - Article
C2 - 32856539
AN - SCOPUS:85090002656
SN - 1024-5332
VL - 25
SP - 327
EP - 334
JO - Hematology
JF - Hematology
IS - 1
ER -