{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T06:07:59Z","timestamp":1790402879904,"version":"4.1.0"},"reference-count":20,"publisher":"The Journal of Health Economics and Outcomes Research","issue":"2","license":[{"start":{"date-parts":[[2025,8,15]],"date-time":"2025-08-15T00:00:00Z","timestamp":1755216000000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["jheor.org"],"crossmark-restriction":true},"short-container-title":[],"abstract":"\n Background<\/jats:title>\n Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder with C5 inhibitors (C5i), eculizumab and ravulizumab, being part of current treatment options.<\/jats:p>\n <\/jats:sec>\n \n Objectives<\/jats:title>\n To estimate the 5-year prevalence of PNH and describe the healthcare resource utilization and direct healthcare costs associated with C5i among commercially insured patients with PNH treated with C5i in the US.<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n The 5-year prevalence of adults with PNH in IQVIA PharMetrics\u00ae Plus was estimated (2018-2022). A retrospective cohort study (2011-2022) was also conducted in adults with PNH treated with C5i and \u22653 months of continuous health plan coverage following the first claim for C5i (index date). PNH-related health resource utilization and direct healthcare costs were assessed from index date until earliest of treatment discontinuation\/end of data\/end of continuous health plan coverage (follow-up period).<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n The 5-year prevalence of PNH was 2.4 per 100\u202f000 persons in commercial claims. A total of 371 patients treated with C5i (median age: 40 years; female: 55.3%; eculizumab: 53.9%; ravulizumab: 46.1%) were followed for a mean\u2009\u00b1\u2009SD [median] of 19.3\u2009\u00b1\u200916.9 [14.7] months. Annual incidence rates of PNH-related blood transfusion and breakthrough hemolysis (BTH) among patients treated with C5i were 1.2 (eculizumab: 1.3; ravulizumab: 1.0) and 4.5 (eculizumab: 5.2; ravulizumab: 3.3) per person per year (PPPY), respectively. In patients treated with eculizumab and ravulizumab, respectively, PNH-related blood transfusion was required by 46.2% and 11.9% of patients in the first 6 months post-index, and over the follow-up period, transfusion avoidance was observed in 46.2% and 78.2% of patients. The 6- and 12-month rates of PNH-related thrombosis were 8.0% and 10.6% for eculizumab and 6.1% and 11.6% for ravulizumab, respectively. Among patients treated with C5i, estimated annual total PNH-related costs PPPY were $660\u202f533 (eculizumab: $697\u202f459; ravulizumab: $612\u202f522) for the first year and $633\u202f984 (eculizumab: $691\u202f022; ravulizumab: $570\u202f832) for subsequent years, with treatment costs accounting for 94.3% to 94.6% of total costs.<\/jats:p>\n <\/jats:sec>\n \n Discussion<\/jats:title>\n Despite treatment with C5i, patients with PNH still exhibited BTH, required blood transfusions, and experienced thrombosis.<\/jats:p>\n <\/jats:sec>\n \n Conclusion<\/jats:title>\n This study highlights the unmet need for more effective PNH treatments to address the economic and clinical burden associated with PNH and improve disease control among patients.<\/jats:p>\n <\/jats:sec>","DOI":"10.36469\/001c.142049","type":"journal-article","created":{"date-parts":[[2025,8,15]],"date-time":"2025-08-15T18:55:23Z","timestamp":1755284123000},"update-policy":"https:\/\/doi.org\/10.36469\/00001","source":"Crossref","is-referenced-by-count":1,"title":["Real-World Prevalence and Outcomes of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors in the US: A Retrospective Claims Database Analysis"],"prefix":"10.36469","volume":"12","author":[{"given":"Srinivas K","family":"Tantravahi","sequence":"first","affiliation":[{"name":"Huntsman Cancer Institute, University of Utah, Salt Lake City, Utah"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominick","family":"Latremouille-Viau","sequence":"additional","affiliation":[{"name":"Groupe d\u2019analyse, Lt\u00e9e, Montr\u00e9al, QC, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raj","family":"Desai","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Soyon","family":"Lee","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corp., East Hanover, New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jincy","family":"Paulose","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corp., East Hanover, New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anumaxine","family":"Geevarghese","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corp., East Hanover, New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annie","family":"Gu\u00e9rin","sequence":"additional","affiliation":[{"name":"Groupe d\u2019analyse, Lt\u00e9e, Montr\u00e9al, Qu\u00e9bec, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shravanthi","family":"Seshasayee","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohin","family":"Chanpura","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corp., East Hanover, New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Glorian","family":"Yen","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corp., East Hanover, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"22273","published-online":{"date-parts":[[2025,8,15]]},"reference":[{"key":"ref1","article-title":"Voydeya approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular haemolysis in adults with the rare disease PNH"},{"key":"ref2","article-title":"Danicopan product label"},{"key":"ref3","article-title":"FDA approves new treatment for adults with serious rare blood disease"},{"key":"ref4","article-title":"Pegcetacoplan product label"},{"key":"ref5","doi-asserted-by":"publisher","first-page":"10760296231213073","DOI":"10.1177\/10760296231213073","article-title":"Treatment patterns and healthcare resource utilization of patients with paroxysmal nocturnal hemoglobinuria: a retrospective claims data analysis","volume":"30","author":"D. Clayton","year":"2024","journal-title":"Clin Appl Thromb Hemost"},{"issue":"8","key":"ref6","doi-asserted-by":"publisher","first-page":"941","DOI":"10.18553\/jmcp.2023.29.8.941","article-title":"Descriptive, real-world treatment patterns, resource use, and total cost of care among eculizumab- and ravulizumab-treated members with paroxysmal nocturnal hemoglobinuria","volume":"29","author":"K. C. Broderick","year":"2023","journal-title":"J Manag Care Spec Pharm"},{"issue":"1","key":"ref7","doi-asserted-by":"publisher","first-page":"230","DOI":"10.3324\/haematol.2019.236877","article-title":"Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria","volume":"106","author":"R. A. Brodsky","year":"2021","journal-title":"Haematologica"},{"issue":"1","key":"ref8","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1080\/16078454.2020.1807226","article-title":"Cost burden of breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria receiving ravulizumab versus eculizumab","volume":"25","author":"I. Tomazos","year":"2020","journal-title":"Hematology"},{"issue":"8","key":"ref9","doi-asserted-by":"publisher","first-page":"4461","DOI":"10.1007\/s12325-021-01825-4","article-title":"Real-world healthcare resource utilization (HRU) and costs of patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving eculizumab in a US population","volume":"38","author":"W. Y. Cheng","year":"2021","journal-title":"Adv Ther"},{"issue":"6","key":"ref10","doi-asserted-by":"publisher","first-page":"530","DOI":"10.1182\/blood-2018-09-876136","article-title":"Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study","volume":"133","author":"J. W. Lee","year":"2019","journal-title":"Blood"},{"issue":"12-b suppl","key":"ref11","doi-asserted-by":"publisher","first-page":"S3","DOI":"10.18553\/jmcp.2020.26.12-b.s3","article-title":"Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology","volume":"26","author":"M. Bektas","year":"2020","journal-title":"J Manag Care Spec Pharm"},{"issue":"6","key":"ref12","doi-asserted-by":"publisher","first-page":"540","DOI":"10.1182\/blood-2018-09-876805","article-title":"Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor\u2013experienced adult patients with PNH: the 302 study","volume":"133","author":"A.G. Kulasekararaj","year":"2019","journal-title":"Blood"},{"issue":"suppl 4","key":"ref13","doi-asserted-by":"publisher","first-page":"S20","DOI":"10.1002\/ajh.26832","article-title":"Paroxysmal nocturnal hemoglobinuria: where we stand","volume":"98","author":"J. Panse","year":"2023","journal-title":"Am J Hematol"},{"issue":"suppl_1","key":"ref14","doi-asserted-by":"publisher","first-page":"3407","DOI":"10.1182\/blood-2019-125867","article-title":"Epidemiology of PNH and real-world treatment patterns following an incident PNH diagnosis in the US","volume":"134","author":"J. J. Jalbert","year":"2019","journal-title":"Blood"},{"key":"ref15","article-title":"Drug approval package: Soliris (eculizumab) injectable I.V."},{"key":"ref16","article-title":"Eculizumab product label"},{"key":"ref17","article-title":"FDA approves ravulizumab-cwvz for paroxysmal nocturnal hemoglobinuria"},{"key":"ref18","article-title":"Ravulizumab product label"},{"key":"ref19","article-title":"Novartis receives FDA approval for Fabhalta (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH"},{"key":"ref20","article-title":"Iptacopan product label"}],"container-title":["Journal of Health Economics and Outcomes Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jheor.org\/article\/142049-real-world-prevalence-and-outcomes-of-patients-with-paroxysmal-nocturnal-hemoglobinuria-treated-with-c5-inhibitors-in-the-us-a-retrospective-claims-d","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,21]],"date-time":"2026-05-21T19:07:18Z","timestamp":1779390438000},"score":1,"resource":{"primary":{"URL":"https:\/\/jheor.org\/article\/142049-real-world-prevalence-and-outcomes-of-patients-with-paroxysmal-nocturnal-hemoglobinuria-treated-with-c5-inhibitors-in-the-us-a-retrospective-claims-d"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,8,15]]},"references-count":20,"journal-issue":{"issue":"2","published-online":{"date-parts":[[2025]]}},"URL":"https:\/\/doi.org\/10.36469\/001c.142049","relation":{},"ISSN":["2327-2236"],"issn-type":[{"value":"2327-2236","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,8,15]]}}}