Research map: Complement inhibitor therapy should be available to patients with paroxysmal nocturnal hemoglobinuria throughout the world

Papers in this map

1. The Complement Inhibitor Eculizumab in Paroxysmal Nocturnal Hemoglobinuria · Peter Hillmen · 2006 · 1307 citations · Cited by this paper
2. Recent advances in the pathophysiology and treatment of GVHD · 2026 · Related
3. Effect of Eculizumab on Hemolysis and Transfusion Requirements in Patients with Paroxysmal Nocturnal Hemoglobinuria · Peter Hillmen · 2004 · 642 citations · Cited by this paper
4. Immunological links between aplastic anemia and paroxysmal nocturnal hemoglobinuria · 2026 · Related
5. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria · Peter Hillmen · 2021 · 405 citations · Cited by this paper
6. Current status and challenges of TCR-T cell therapy for AML/MDS · 2026 · Related
7. Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab · Antonio Maria Risitano · 2009 · 361 citations · Cited by this paper
8. T-cell fitness, metabolic tumor volume, and serum LDH levels are associated with response to axicabtagene ciloleucel · 2026 · Related
9. The Role of Public-Sector Research in the Discovery of Drugs and Vaccines · Ashley J. Stevens · 2011 · 245 citations · Cited by this paper
10. NPM1-rearranged AML: clinical features, molecular pathogenesis, and therapeutic perspectives · 2026 · Related
11. Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization · Anita V. Hill · 2010 · 196 citations · Cited by this paper
12. Postmarketing surveillance study of asciminib in patients with resistant/intolerant chronic myeloid leukemia in Japan · 2026 · Related
13. Outrageous prices of orphan drugs: a call for collaboration · Lucio Luzzatto · 2018 · 162 citations · Cited by this paper
14. Genomic insights transform diagnosis, prognosis, and therapy in BCR::ABL1-negative myeloproliferative neoplasms · 2026 · Related
15. Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria · Régis Peffault de Latour · 2024 · 150 citations · Cited by this paper
16. Defibrotide for late-onset sinusoidal obstruction syndrome following umbilical cord blood transplantation: a single-center retrospective study · 2026 · Related
17. Bone marrow transplants for paroxysmal nocturnal haemoglobinuria · Radovan M. Saso · 1999 · 136 citations · Cited by this paper
18. Zanubrutinib for MYD88-negative Waldenström’s macroglobulinemia with massive splenomegaly · 2026 · Related
19. Epidemiology of Rare Anaemias in Europe · Béatrice Gulbis · 2010 · 116 citations · Cited by this paper
20. Darbepoetin alfa for the treatment of anemia in myelodysplastic syndromes: a post-marketing surveillance study in Japan · 2026 · Related
21. Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition · Rosario Notaro · 2022 · 112 citations · Cited by this paper
22. Management of Paroxysmal Nocturnal Haemoglobinuria: a personal view · Lucio Luzzatto · 2011 · 102 citations · Cited by this paper
23. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial · Jong Wook Lee · 2023 · 93 citations · Cited by this paper
24. Advancing therapeutic complement inhibition in hematologic diseases: PNH and beyond · Eleni Gavriilaki · 2021 · 82 citations · Cited by this paper
25. FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis · Thomas Michaeli · 2023 · 54 citations · Cited by this paper
26. Cost of Lung Cancer: A Systematic Review · Mahmood Yousefi · 2022 · 39 citations · Cited by this paper
27. Cost of treating metastatic colorectal cancer: a systematic review · Nazim Bhimani · 2022 · 35 citations · Cited by this paper
28. Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet · Lucio Luzzatto · 2022 · 31 citations · Cited by this paper
29. Navigating the Complement Pathway to Optimize PNH Treatment with Pegcetacoplan and Other Currently Approved Complement Inhibitors · Peter Hillmen · 2024 · 19 citations · Cited by this paper
30. Emergent treatments for β-thalassemia and orphan drug legislations · Enrico Costa · 2022 · 15 citations · Cited by this paper
31. Blood diseases in Africa: Redressing unjust disparities is an urgent unmet need · Julie Makani · 2022 · 14 citations · Cited by this paper
32. The complement model disease paroxysmal nocturnal hemoglobinuria · Christoph Q. Schmidt · 2024 · 14 citations · Cited by this paper
33. Unmanipulated haploidentical donor and matched unrelated donor hematopoietic stem cell transplantation in patients with paroxysmal nocturnal hemoglobinuria: a single-center study · Yue Lü · 2021 · 9 citations · Cited by this paper
34. Comparative clinical efficacy and safety of biosimilar ABP 959 and eculizumab reference product in patients with paroxysmal nocturnal hemoglobinuria · Austin Gladston Kulasekararaj · 2024 · 9 citations · Cited by this paper
35. Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or Without Aplastic Anemia: Multicenter Turkish Experience · Fergün Yılmaz · 2021 · 7 citations · Cited by this paper
36. Results of Long-Term Therapy with a Biosimilar of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria · Alexander Kulagin · 2024 · 6 citations · Cited by this paper
37. The Global Fund and the future of global health · The Lancet · 2025 · 5 citations · Cited by this paper
38. Paroxysmal nocturnal haemoglobinuria · Lucio Luzzatto · 2010 · 4 citations · Cited by this paper
39. Management of paroxysmal nocturnal hemoglobinuria (PNH) · Lucio Luzzatto · 2022 · 4 citations · Cited by this paper

Source: OpenAlex (CC0)