Prevention and Management of Thromboembolism in Patients with Paroxysmal Nocturnal Hemoglobinuria in Asia: A Narrative Review.

Authors: Ueda, Yasutaka¹ (AUTHOR) yueda@bldon.med.osaka-u.ac.jp, Chou, Wen-Chien² (AUTHOR), Goh, Yeow-Tee³ (AUTHOR), Rojnuckarin, Ponlapat^4,5 (AUTHOR), Kim, Jin Seok^5,6 (AUTHOR), Wong, Raymond Siu Ming^6,7 (AUTHOR), Lee Wong, Lily Lee^7,8 (AUTHOR), Jang, Jun Ho^8,9 (AUTHOR), Chiou, Tzeon-Jye^9,10 (AUTHOR), Kanakura, Yuzuru^10,11 (AUTHOR), Lee, Jong Wook^11,12 (AUTHOR)

Source: International Journal of Molecular Sciences. Mar2025, Vol. 26 Issue 6, p2504. 17p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *COMPLEMENT inhibition, *ASIANS, *LACTATE dehydrogenase, THROMBOEMBOLISM prevention

Geographic Terms: ASIA

Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan.

Authors: Mulherin, Brian^1,2 (AUTHOR) brian.mulherin@aoncology.com, Shenoy, Apeksha³ (AUTHOR) apeksha.shenoy@bostonsp.com, Arnett, Lily³ (AUTHOR) lily.arnett@bostonsp.com, Jiao, Weiqi³ (AUTHOR) weiqi.jiao@bostonsp.com, Guarinoni, Joseph⁴ (AUTHOR) jguarinoni@pantherxrare.com, Sarda, Sujata⁵ (AUTHOR) jinny.min@apellis.com, Min, Jinny⁵ (AUTHOR), Dingli, David⁶ (AUTHOR) dingli.david@mayo.edu

Source: Hematology Reports. Dec2024, Vol. 16 Issue 4, p669-681. 13p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *FATIGUE (Physiology), *CHRONIC diseases, *FUNCTIONAL assessment, *QUALITY of life

Expert consensus on the management of pharmacodynamic breakthrough-hemolysis in treated paroxysmal nocturnal hemoglobinuria.

Authors: Dingli, David¹ (AUTHOR), De Castro III, Carlos² (AUTHOR), Koprivnikar, Jamie³ (AUTHOR), Kulasekararaj, Austin⁴ (AUTHOR), Maciejewski, Jaroslaw⁵ (AUTHOR), Mulherin, Brian⁶ (AUTHOR), Panse, Jens⁷ (AUTHOR), Pullarkat, Vinod⁸ (AUTHOR), Röth, Alexander⁹ (AUTHOR), Shammo, Jamile¹⁰ (AUTHOR), Terriou, Louis¹¹ (AUTHOR), Weitz, Ilene¹² (AUTHOR), Yermilov, Irina¹³ (AUTHOR), Gibbs, Sarah¹³ (AUTHOR), Broder, Michael¹³ (AUTHOR) mbroder@pharllc.com, Beenhouwer, David¹³ (AUTHOR), Kuter, David¹⁴ (AUTHOR)

Source: Hematology. Dec2024, Vol. 29 Issue 1, p1-10. 10p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *BLOOD diseases, *BLOOD coagulation disorders, *COMPLEMENT inhibition, *APLASTIC anemia

Population Pharmacokinetic and Pharmacokinetic/Pharmacodynamic Analyses of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria: Pop PK and PK/PD Analyses of Pegcetacoplan in PNH: R. L. Crass et al.

Authors: Crass, Ryan L.^1,2 (AUTHOR) ryan.crass@apellis.com, Smith, Brandon¹ (AUTHOR) brandonismith9211@gmail.com, Adriaens, Sven¹ (AUTHOR) sven.adriaens@amadorbio.com, Chapel, Sunny¹ (AUTHOR) sunny@a2-ai.com, Langdon, Grant³ (AUTHOR) Grant.Langdon@sobi.com

Source: Drugs in R&D. Dec2024, Vol. 24 Issue 4, p563-573. 11p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *COMPLEMENT (Immunology), *COMPLEMENT inhibition, *ERYTHROCYTES, *BLOOD diseases

Monitoring and Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients with Aplastic Anemia in Asia: An Expert Consensus.

Authors: Wong, Raymond Siu Ming¹ (AUTHOR) raymondwong@cuhk.edu.hk, Ho Jang, Jun² (AUTHOR), Wong, Lily Lee Lee³ (AUTHOR), Kim, Jin Seok⁴ (AUTHOR), Rojnuckarin, Ponlapat^5,6 (AUTHOR) rojnuckarinp@gmail.com, Goh, Yeow-Tee⁷ (AUTHOR), Ueda, Yasutaka⁸ (AUTHOR) yueda@bldon.med.osaka-u.ac.jp, Chou, Wen-Chien⁹ (AUTHOR), Lee, Jong Wook¹⁰ (AUTHOR), Kanakura, Yuzuru¹¹ (AUTHOR), Chiou, Tzeon-Jye¹² (AUTHOR) 108178@w.tmu.edu.tw

Source: International Journal of Molecular Sciences. Nov2024, Vol. 25 Issue 22, p12160. 16p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *APLASTIC anemia, *LACTATE dehydrogenase, *FLOW cytometry, *PHYSICIANS

Geographic Terms: ASIA

Thrombosis in Paroxysmal Nocturnal Hemoglobinuria (PNH): From Pathogenesis to Treatment.

Authors: Kokoris, Styliani¹ (AUTHOR) stylianikok@gmail.com, Polyviou, Antri² (AUTHOR) polyviouandri@gmail.com, Evangelidis, Paschalis³ (AUTHOR) pascevan@auth.gr, Grouzi, Elisavet⁴ (AUTHOR) grouzielisavet@gmail.com, Valsami, Serena⁵ (AUTHOR) serenavalsami@yahoo.com, Tragiannidis, Konstantinos³ (AUTHOR) konstantinos.tragiannidis@gmail.com, Gialeraki, Argyri¹ (AUTHOR) agialer@med.uoi.gr, Tsakiris, Dimitrios A.⁶ (AUTHOR) dimitrios.tsakiris@unibas.ch, Gavriilaki, Eleni³ (AUTHOR) gavriiel@auth.gr

Source: International Journal of Molecular Sciences. Nov2024, Vol. 25 Issue 22, p12104. 24p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *LOW-molecular-weight heparin, *COMPLEMENT inhibition, *SYMPTOMS, *VENOUS thrombosis

Beyond Recycling Antibodies: Crovalimab's Molecular Design Enables Four-Weekly Subcutaneous Injections for PNH Treatment.

Authors: Sampei, Zenjiro^1,2 (AUTHOR) sampei.zenjiro97@chugai-pharm.co.jp, Haraya, Kenta^1,2 (AUTHOR), Gan, Siok Wan² (AUTHOR), Muraoka, Masaru^1,2 (AUTHOR), Hayasaka, Akira¹ (AUTHOR), Fukuzawa, Taku^1,2 (AUTHOR), Shida-Kawazoe, Meiri^1,2 (AUTHOR), Tsuboi, Yoshinori¹ (AUTHOR), Gotoh, Akihiko³ (AUTHOR), Obara, Naoshi⁴ (AUTHOR), Ueda, Yasutaka⁵ (AUTHOR)

Source: International Journal of Molecular Sciences. Nov2024, Vol. 25 Issue 21, p11679. 19p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *SUBCUTANEOUS injections, *SINGLE nucleotide polymorphisms, *INTRAVENOUS therapy, *IMMUNOTECHNOLOGY

Navigating the Complement Pathway to Optimize PNH Treatment with Pegcetacoplan and Other Currently Approved Complement Inhibitors.

Authors: Hillmen, Peter¹ (AUTHOR) peter.hillmen@apellis.com, Horneff, Regina² (AUTHOR), Yeh, Michael¹ (AUTHOR), Kolev, Martin¹ (AUTHOR), Deschatelets, Pascal¹ (AUTHOR)

Source: International Journal of Molecular Sciences. Sep2024, Vol. 25 Issue 17, p9477. 16p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *COMPLEMENT inhibition, *HEMATOPOIETIC stem cells, *ERYTHROCYTES, *CD55 antigen

Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria.

Authors: Donoghue, Jeremiah¹ (AUTHOR), Youngs, Matthew² (AUTHOR), Reeve, Alex² (AUTHOR), Vydyula, Krishna² (AUTHOR), Kunst, Natalia³ (AUTHOR), Trikha, Roochi⁴ (AUTHOR), Gallacher, Daniel¹ (AUTHOR) d.c.gallacher@bham.ac.uk

Source: PharmacoEconomics. May2025, Vol. 43 Issue 5, p499-508. 10p.

Subject Terms: *Cost effectiveness, *Economic models, *Decision making, *Empirical research, Paroxysmal hemoglobinuria, Technology assessment, Medical technology

Company/Entity: National Institute for Health & Care Excellence (Great Britain)

Three Years On: The Role of Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH) since Its Initial Approval.

Authors: Horneff, Regina¹ (AUTHOR) regina.horneff@sobi.com, Czech, Barbara¹ (AUTHOR), Yeh, Michael² (AUTHOR), Surova, Elena¹ (AUTHOR)

Source: International Journal of Molecular Sciences. Aug2024, Vol. 25 Issue 16, p8698. 16p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *HEMOLYTIC anemia, *COMPLEMENT inhibition, *LACTATE dehydrogenase, *HEMOLYSIS & hemolysins

Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin ≥10 g/dL) who had received eculizumab or were naive to complement inhibitors

Authors: Panse, Jens^1,2 (AUTHOR) jpanse@ukaachen.de, Daguindau, Nicolas³ (AUTHOR), Okuyama, Sonia⁴ (AUTHOR), Peffault de Latour, Régis⁵ (AUTHOR), Schafhausen, Philippe⁶ (AUTHOR), Straetmans, Nicole⁷ (AUTHOR), Al-Adhami, Mohammed⁸ (AUTHOR), Persson, Emmelie⁹ (AUTHOR), Wong, Raymond Siu Ming¹⁰ (AUTHOR)

Source: PLoS ONE. 7/29/2024, Vol. 19 Issue 7, p1-20. 20p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *COMPLEMENT inhibition, *FATIGUE (Physiology), *HEMOGLOBINS, *ECULIZUMAB, *FETAL hemoglobin, *ATRIAL flutter

Eculizumab Treatment of Massive Hemolysis Occurring in a Rare Co-Existence of Paroxysmal Nocturnal Hemoglobinuria and Myasthenia Gravis.

Authors: Bicskó, Ráhel Réka¹ (AUTHOR) bicsko.reka@med.unideb.hu, Illés, Árpád¹ (AUTHOR) lgergely@med.unideb.hu, Hevessy, Zsuzsanna² (AUTHOR) hevessy@med.unideb.hu, Ivády, Gergely² (AUTHOR) ivady@med.unideb.hu, Kerekes, György³ (AUTHOR) gkerekes@med.unideb.hu, Méhes, Gábor⁴ (AUTHOR) gabor.mehes@med.unideb.hu, Csépány, Tünde⁵ (AUTHOR) csepany@med.unideb.hu, Gergely, Lajos¹ (AUTHOR)

Source: Hematology Reports. Jun2024, Vol. 16 Issue 2, p255-259. 5p.

Subject Terms: *MYASTHENIA gravis, *PAROXYSMAL hemoglobinuria, *HEMOLYSIS & hemolysins, *RESPIRATORY acidosis, *ECULIZUMAB, *RESPIRATORY diseases

Normalization of Hemoglobin, Lactate Dehydrogenase, and Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan.

Authors: Mulherin, Brian P.^1,2 (AUTHOR) bmulhrn@yahoo.com, Yeh, Michael¹ (AUTHOR), Al-Adhami, Mohammed³ (AUTHOR), Dingli, David⁴ (AUTHOR)

Source: Drugs in R&D. Jun2024, Vol. 24 Issue 2, p169-177. 9p.

Subject Terms: *CLINICAL trials, *PAROXYSMAL hemoglobinuria, *LACTATE dehydrogenase, *FATIGUE (Physiology), *SOCIAL norms

Health-related quality of life and influencing factors of patients with paroxysmal nocturnal hemoglobinuria in China.

Authors: Yu, Huaxin¹ (AUTHOR), Duan, Shengnan¹ (AUTHOR), Wang, Pei² (AUTHOR), Fu, Rong³ (AUTHOR), Lv, Zixuan¹ (AUTHOR), Yu, Yuchi⁴ (AUTHOR), Miao, Pu⁴ (AUTHOR), Shi, Junwei⁴ (AUTHOR), Zhuang, Niekun⁴ (AUTHOR), Hu, Huiying⁴ (AUTHOR), Yuan, Ni^1,5 (AUTHOR) nier1209@163.com, Che, Sijia⁴ (AUTHOR) info@pnhchina.org

Source: Orphanet Journal of Rare Diseases. 5/3/2024, Vol. 19 Issue 1, p1-15. 15p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *QUALITY of life, *INCOME, *LOGISTIC regression analysis, *MUTUAL aid, *BLOOD coagulation factors, *ATRIAL flutter

Geographic Terms: CHINA

Autoimmune Hemolytic Anemias: Classifications, Pathophysiology, Diagnoses and Management.

Authors: Loriamini, Melika^1,2 (AUTHOR) melika.loriamini@mail.utoronto.ca, Cserti-Gazdewich, Christine^1,3,4 (AUTHOR) christine.cserti@uhn.ca, Branch, Donald R.^1,2,5 (AUTHOR) don.branch@utoronto.ca

Source: International Journal of Molecular Sciences. Apr2024, Vol. 25 Issue 8, p4296. 18p.

Subject Terms: *AUTOIMMUNE hemolytic anemia, *PAROXYSMAL hemoglobinuria, *PATHOLOGICAL physiology, *BLOOD groups, *DIAGNOSIS, *MONOCLONAL antibodies

The disease burden of paroxysmal nocturnal hemoglobinuria in Denmark: Epidemiology, survival, healthcare resource utilization, costs, treatment gaps, and labor market attachment.

Authors: Rich, Carly¹ (AUTHOR) carly.rich@sobi.com, Wilson, Koo¹ (AUTHOR), Olsen, Jens² (AUTHOR), Pedersen, Mikkel² (AUTHOR), Frederiksen, Henrik³ (AUTHOR)

Source: European Journal of Haematology. Mar2024, Vol. 112 Issue 3, p412-423. 12p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *LABOR market, *MEDICAL care, *EPIDEMIOLOGY, *TRANSFER payments, *MEDICAL care costs

Geographic Terms: DENMARK

Description of 18 PNH patients with prevalent GPI‐deficient monocytes. An Italian survey.

Authors: Lanza, F.¹ (AUTHOR) francesco.lanza@auslromagna.it, Massari, E.² (AUTHOR), Marconi, G.¹ (AUTHOR), Poletti, G.² (AUTHOR), Krampera, M.³ (AUTHOR), Lazzari, C.⁴ (AUTHOR), Di Martino, G.⁴ (AUTHOR), Raia, M.⁵ (AUTHOR), Scalia, G.⁵ (AUTHOR)

Source: British Journal of Haematology. Apr2025, p1. 5p. 1 Illustration.

Subject Terms: *FLOW cytometry, *PAROXYSMAL hemoglobinuria, *ERYTHROCYTES, *MEDICAL research, *DIAGNOSIS, *LEUKOCYTES, *PHENOTYPES

Geographic Terms: ITALY

First cases of danicopan use in adolescent patients with paroxysmal nocturnal haemoglobinuria.

Authors: Holt, Matthew^1,2 (AUTHOR) matthew.holt@nhs.net, Kelly, Richard^1,3 (AUTHOR), Hughes, Samantha⁴ (AUTHOR), Munir, Talha¹ (AUTHOR), Varghese, Abraham¹ (AUTHOR), Nagumantry, Sateesh⁵ (AUTHOR), Arnold, Louise¹ (AUTHOR), Muus, Petra¹ (AUTHOR), Griffin, Morag¹ (AUTHOR)

Source: British Journal of Haematology. Apr2025, p1. 4p. 1 Illustration.

Subject Terms: *PAROXYSMAL hemoglobinuria, *CHILD patients, *COMPLEMENT (Immunology), *CLINICAL trials, *APLASTIC anemia

Real‐world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study.

Authors: Metafuni, Elisabetta¹ (AUTHOR), Brioschi, Filippo Achille² (AUTHOR), Patriarca, Andrea³ (AUTHOR), Leoni, Claudia^4,5 (AUTHOR), Battipaglia, Giorgia⁶ (AUTHOR), Carlino, Daniela⁷ (AUTHOR), Condorelli, Annalisa⁸ (AUTHOR), Di Giacomo, Valeria⁹ (AUTHOR), Marktel, Sarah¹⁰ (AUTHOR), Marzilli, Maria Antonietta¹¹ (AUTHOR), Miglino, Maurizio¹² (AUTHOR), Oliva, Esther Natalie¹³ (AUTHOR), Sau, Antonella¹⁴ (AUTHOR), Ricco, Alessandra¹⁵ (AUTHOR), Sanpaolo, Grazia¹⁶ (AUTHOR), Trolese, Annarita¹⁷ (AUTHOR), Iori, Anna Paola¹⁸ (AUTHOR), Sica, Simona¹ (AUTHOR), Barcellini, Wilma⁴ (AUTHOR), Fattizzo, Bruno^4,5 (AUTHOR) bruno.fattizzo@unimi.it

Source: British Journal of Haematology. Apr2025, Vol. 206 Issue 4, p1246-1249. 4p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *HEMOGLOBINS, *MEDICAL research, *HEMATOLOGIC agents, *ADVERSE health care events, *EMPIRICAL research, *COMPLEMENT inhibition

Oral iptacopan for paroxysmal nocturnal haemoglobinuria—First real‐world experience.

Authors: Kelly, Richard J.¹ (AUTHOR) richardkelly@nhs.net, Kulasekararaj, Austin G.² (AUTHOR), Arnold, Louise M.¹ (AUTHOR), Barnfield, Catherine¹ (AUTHOR), Storey, Isla¹ (AUTHOR), Molyneux, Jessica¹ (AUTHOR), Youngs, Nora L.¹ (AUTHOR), Large, Joanna² (AUTHOR), Mitchell, Lindsay³ (AUTHOR), Nagumantry, Sateesh K.⁴ (AUTHOR), Gandhi, Shreyans² (AUTHOR), Trikha, Roochi² (AUTHOR), Varghese, Abraham¹ (AUTHOR), Munir, Talha¹ (AUTHOR), Muus, Petra¹ (AUTHOR), Griffin, Morag¹ (AUTHOR)

Source: British Journal of Haematology. Apr2025, Vol. 206 Issue 4, p1241-1245. 5p.

Subject Terms: *PAROXYSMAL hemoglobinuria, *ANEMIA, *PLATELET count, *DRUG side effects, *COMPLEMENT inhibition, *HEMOGLOBINS