Paroxysmal Nocturnal Hemoglobinuria in the Context of a Myeloproliferative Neoplasm: A Case Report and Review of the Literature.
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TLDR
In this paper, a 62-year-old man developed a highly hemolytic PNH more than 10 years after the diagnosis of myeloproliferative neoplasms (MPNs), which are also burdened by increased thrombotic tendency.Abstract:
Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolytic anemia and thrombosis and is notoriously associated with aplastic anemia and myelodysplastic syndromes. Rarer associations include myeloproliferative neoplasms (MPNs), which are also burdened by increased thrombotic tendency. The therapeutic management of this rare combination has not been defined so far. Here, we describe a 62-year-old man who developed a highly hemolytic PNH more than 10 years after the diagnosis of MPN. The patient started eculizumab, obtaining good control of intravascular hemolysis but without amelioration of transfusion-dependent anemia. Moreover, we performed a review of the literature regarding the clinical and pathogenetic significance of the association of PNH and MPN. The prevalence of PNH clones in MPN patients is about 10%, mostly in association with JAK2V617F-positive myelofibrosis. Thrombotic events were a common clinical presentation (35% of subjects), sometimes refractory to combined treatment with cytoreductive agents, anticoagulants, and complement inhibitors. The latter showed only partial effectiveness in controlling hemolytic anemia and, due to the paucity of data, should be taken in consideration after a careful risk/benefit evaluation in this peculiar setting.read more
Citations
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Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations
Imre Bodó,Ismail Amine,A. Boban,Horia Bumbea,A. D. Kulagin,Elena Lukina,Agnieszka Piekarska,Irena Preloznik Zupan,Juraj Sokol,Jerzy Windyga,Jaroslav Cermak +10 more
TL;DR: In this paper , the authors identified specific populations of patients who may benefit from switching to proximal C3 from terminal C5 inhibition, based on a Delphi-like process by a group of expert PNH specialists across Central Europe.
Journal ArticleDOI
Thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH): Real world data of a Greek nationwide multicenter retrospective study
S. Chatzileontiadou,Evdoxia Hatjiharissi,Maria A. Angelopoulou,J. Asimakopoulos,N. Loutsidi,Thomas Chatzikonstantinou,Panagiotis Zikos,Anthoula Bouchla,Zoe Bezirgiannidou,E. Kouvata,C. Frouzaki,Panteleimon A Chaloudis,Damianos Sotiropoulos,Vassiliki Douka,Antonia Sirigou,Eudokia Mandala,M. Psyllaki,Helen A. Papadaki,Theodoros Marinakis,Nora Viniou,Stela Kokkori,Flora N. Kontopidou,A. Skepetari,George Vassilopoulos,Ioannis Kotsianidis,V. B. Rathi Pappa,C Lalayanni,Ioannis Baltadakis,Sossana Delimpassi,Maria Pagoni,Maria Papaioannou +30 more
TL;DR: The most common sites of thrombosis were intra-abdominal veins in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) as discussed by the authors , with thromboembolic events being the most common cause of death.
References
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Paroxysmal nocturnal hemoglobinuria.
TL;DR: Bone marrow transplantation remains the only cure for PNH but should be reserved for patients with suboptimal response to eculizumab, a first-in-class monoclonal antibody that inhibits terminal complement.
Journal ArticleDOI
Thrombosis in paroxysmal nocturnal hemoglobinuria
TL;DR: Mechanisms explored include platelet activation, toxicity of free hemoglobin, nitric oxide depletion, absence of other glycosylphosphatidylinositol-linked proteins such as urokinase-type plasminogen activator receptor and endothelial dysfunction and complement inhibition with eculizumab.
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Myeloproliferative neoplasms and thrombosis
TL;DR: Novel disease-related determinants such as leukocytosis and JAK2V617F mutational status and/or mutational burden are now under active investigation and will improve the management of thrombosis.