Thrombolytic therapy is effective in paroxysmal nocturnal hemoglobinuria: a series of nine patients and a review of the literature

Araten, David J.; Notaro, Rosario; Thaler, Howard T.; Kernan, Nancy A.; Boulad, Farid; Castro‐Malaspina, Hugo; Small, Trudy N.; Scaradavou, Andromachi; Magnan, Heather; Prockop, Susan E.; Chaffee, Sara; Gonsky, Jason; Thertulien, Raymond; Tarquini, Roberto; Luzzatto, Lucio

doi:10.3324/haematol.2011.049767

Cited by 24 publications

(26 citation statements)

References 36 publications

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“…118 This suggests the principle mechanism of heparin action in this setting is through the interaction with C5b-9 and not through the potentiation of C1 inhibitor. There have been reports of an increased incidence of heparin-induced thrombocytopenia and consequent thrombosis, [125][126][127] thought to be explained by the increased platelet activation in PNH with induced release of platelet factor 4. If there is concern, theoretically, fondaparinux may be a safer formulation.…”

Section: Prevention Of Thrombosismentioning

confidence: 99%

Thrombosis in paroxysmal nocturnal hemoglobinuria

Hill

Kelly

Hillmen

2013

Blood

389

429

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The most frequent and feared complication of paroxysmal nocturnal hemoglobinuria (PNH) is thrombosis. Recent research has demonstrated that the complement and coagulation systems are closely integrated with each influencing the activity of the other to the extent that thrombin itself has recently been shown to activate the alternative pathway of complement. This may explain some of the complexity of the thrombosis in PNH. In this review, the recent changes in our understanding of the pathophysiology of thrombosis in PNH, as well as the treatment of thrombosis, will be discussed. 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. Complement inhibition with eculizumab has a dramatic effect in PNH and has a major impact in the prevention of thrombosis as well as its management in this disease.

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Section: Prevention Of Thrombosismentioning

confidence: 99%

Thrombosis in paroxysmal nocturnal hemoglobinuria

Hill

Kelly

Hillmen

2013

Blood

389

429

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“…Systemic thrombolytic therapy, or thrombolytic therapy delivered via canalization directly to the affected site, can be considered in patients with acute onset of Budd-Chiari syndrome. [48][49][50][51] If there is no contraindication, anticoagulation should continue indefinitely for a patient with PNH (even if that patient is treated with eculizumab) who has experienced a thromboembolic complication. For PNH patients on long-term anticoagulation, a vitamin K antagonist such as Coumadin has been used customarily.…”

Section: How I Treat Pnh Based On Disease Classificationmentioning

confidence: 99%

Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria

Parker

2016

Hematology

123

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Once suspected, the diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) is straightforward when flow cytometric analysis of the peripheral blood reveals a population of glycosyl phosphatidylinositol anchor protein-deficient cells. But PNH is clinically heterogeneous, with some patients having a disease process characterized by florid intravascular, complement-mediated hemolysis, whereas in others, bone marrow failure dominates the clinical picture with modest or even no evidence of hemolysis observed. The clinical heterogeneity is due to the close, though incompletely understood, relationship between PNH and immune-mediated bone marrow failure, and that PNH is an acquired, nonmalignant clonal disease of the hematopoietic stem cells. Bone marrow failure complicates management of PNH because compromised erythropoiesis contributes, to a greater or lesser degree, to the anemia; in addition, the extent to which the mutant stem cell clone expands in an individual patient determines the magnitude of the hemolytic component of the disease. An understanding of the unique pathobiology of PNH in relationship both to complement physiology and immune-mediated bone marrow failure provides the basis for a systematic approach to management.

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“…Budd-Chiari syndrome) to cerebro-vascular accidents. [8][9][10] Pulmonary hypertension in PNH can be either due to NO depleted state or due to widespread formation of thrombuses in pulmonary microvasculature. 11 In our patient however, the possibility of chronic pulmonary thromboembolism could not be excluded as CTPA was not done.…”

Section: Discussionmentioning

confidence: 99%

Paroxysmal Nocturnal Hemoglobinuria: A Chronic Rare Acquired Hemolytic Anemia in a Middle-aged Female with Thrombotic Complications and Multi-system Involvement

Bussell

Murphy²,

Nicks³

et al. 2017

J. Medicine

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Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired coombs negative hemolytic anemia, the hallmark of which is chronic intravascular hemolysis, thrombophilia and their sequelae owing to the deficiency of several surface proteins on blood cell membranes. In this case a middle-aged female presents with severe anemia requiring repeated blood transfusion and recurrent deep vein thrombosis (DVT). She had been hospitalized in multiple occasions in multiple centers without any satisfactory diagnosis. The constant laboratory features were that of hemolytic anemia, thrombocytopenia and a negative coombs test; although reticulocyte count had not been elevated. Lastly Blood flow cytometry revealed PNH clones. Although excellent improvement of the clinical features of PNH can be achieved by treatment with Eculizumab, we could not offer that to our patient due to its unavailability in Bangladesh. She was discharged with advice for Hematopoietic Stem Cell Transplantation (HSCT) after her symptoms improved with symptomatic management. High degree of suspicion and appropriate application of clinical knowledge is necessary to make such a diagnosis early in the disease process, for prompt initiation of treatment and thus minimizing complications.J MEDICINE July 2017; 18 (2) : 123-127

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Thrombolytic therapy is effective in paroxysmal nocturnal hemoglobinuria: a series of nine patients and a review of the literature

Cited by 24 publications

References 36 publications

Thrombosis in paroxysmal nocturnal hemoglobinuria

Thrombosis in paroxysmal nocturnal hemoglobinuria

Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria: A Chronic Rare Acquired Hemolytic Anemia in a Middle-aged Female with Thrombotic Complications and Multi-system Involvement

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