{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T09:13:14Z","timestamp":1788513194940,"version":"build-2803163510"},"reference-count":82,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2026,5,25]],"date-time":"2026-05-25T00:00:00Z","timestamp":1779667200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,5,25]],"date-time":"2026-05-25T00:00:00Z","timestamp":1779667200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aims<\/jats:title>\n                    <jats:p>Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra\u2010rare, acquired, non\u2010malignant haematological disorder that, if left untreated, can lead to significant morbidity. This systematic literature review (SLR) summarized real\u2010world evidence (RWE) for pegcetacoplan, a complement 3\/3b inhibitor (C3i) available since 2021.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      The SLR (PROSPERO\u2010CRD420251043506) followed 2020 PRISMA guidelines and included RW studies of pegcetacoplan (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009&gt;\u20091 pts.; English; to April 2025) in adults (age\u2009\u2265\u200918\u2009years) with PNH.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Of 409 identified records, 39 qualified, representing 12 distinct studies. Six studies (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20094\u201339) reported median haemoglobin (Hb) with baseline 8.1\u20139.6\u2009g\/dL. Ending median Hb and maximum pegcetacoplan durations were: 12.0\u2009g\/dL at 12\u2009months, 11.1\u201312.1\u2009g\/dL at 6\u2009months (3 studies), and 11.1\u2009g\/dL at 3\u2009months (1 study). In 4 other studies (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200948\u201370), ending mean Hb (maximum pegcetacoplan duration) was: 11.3\u2009g\/dL (7.2\u2009months), 11.5\u2009g\/dL (6.6\u2009months), 11.5\u2009g\/dL (5.9\u2009months), and 11.58\u2009g\/dL (3\u2009months). Six studies reported reduced absolute reticulocyte count (ARC;\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20094\u201339) from baseline median 155\u2013301\u2009\u00d7\u200910\n                      <jats:sup>9<\/jats:sup>\n                      \/L to median 56\u2013106\u2009\u00d7\u200910\n                      <jats:sup>9<\/jats:sup>\n                      \/L from 14\u2009days of pegcetacoplan, maintained to maximum pegcetacoplan of 1\u201312\u2009months. Three studies reported lactate dehydrogenase (LDH;\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20094\u201362); all showed reductions from baseline median 543.0 to 161.7\u2009U\/L after 3\u2009months, and baseline median 299.5\u2013316.0\u2009U\/L to 193.5\u2013187.0\u2009U\/L after 6\u2009months of pegcetacoplan. In complement 5 inhibitor\u2010na\u00efve, LDH reduced from 977.8 to 358.9\u2009U\/L after 8.4\u2009months, and 503.6 to 292.5\u2009U\/L in C5i\u2010experienced after 7.2\u2009months. Three studies (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20094\u201363) reported reduced LDH from above the upper limit of normal levels. Six studies (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200923\u201370) reported reduced red blood cell transfusions (RBCt) after maximum pegcetacoplan durations of up to 12\u2009months, and median durations of 3.0 and 10.2\u2009months. Functional Assessment of Chronic Illness Therapy (FACIT)\u2010Fatigue scale scores in 1 study increased from baseline (mean 28.4) to 38.6 at 3\u2009months, 36.3 at 6\u2009months, and 34.9 at 9\u2009months of pegcetacoplan treatment. Two studies reported FACIT\u2010Fatigue scores of 34.6\u201340.1 with pegcetacoplan for \u2265\u20091\u2009month. EQ\u20105D mean utility scores (0.85\u20130.94) in 2 studies were comparable to population normative values. On the Short\u2010Form 36 Health Survey, mental and physical component scores were slightly lower than US normative values.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>RWE indicates pegcetacoplan is associated with improved haematological outcomes, reduced RBCt dependence and fatigue, and enhanced HRQoL. These findings from real\u2010world studies with diverse cohorts support generalizability and are broadly comparable with clinical trial evidence.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ejh.70228","type":"journal-article","created":{"date-parts":[[2026,5,26]],"date-time":"2026-05-26T02:05:22Z","timestamp":1779761122000},"page":"799-813","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Pegcetacoplan Delivers Real\u2010World Therapeutic Benefits and Reduces Disease Burden for Patients With Paroxysmal Nocturnal Haemoglobinuria: A Systematic Literature Review of Pegcetacoplan Real\u2010World Clinical and Patient\u2010Reported Outcomes"],"prefix":"10.1111","volume":"117","author":[{"given":"Juan Carlos Vallejo","family":"Llamas","sequence":"first","affiliation":[{"name":"University Clinical Hospital of Salamanca  Salamanca 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