Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria

Hill, Anita; Hillmen, Peter; Richards, Stephen J.; Elebute, Dupe; Marsh, Judith; Chan, Jason R.; Mojcik, Christopher F.; Rother, Russell P.

doi:10.1182/blood-2005-02-0564

Cited by 197 publications

(156 citation statements)

References 25 publications

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“…6 Eculizumab was evaluated in 195 patients with PNH in clinical studies. 2,[7][8][9] By inhibiting terminal complement activation, eculizumab dramatically reduced intravascular hemolysis, as measured by a reduction in levels of lactate dehydrogenase (LDH), leading to improvements in anemia, fatigue, and quality of life as well as reductions in blood transfusions and thrombosis. Interestingly, while LDH was reduced from approximately ten times the upper limit of the normal range to near normal values with eculizumab treatment, levels remained slightly elevated in some patients.…”

Section: Introductionmentioning

confidence: 99%

Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization

Hill¹,

Rother²,

Arnold³

et al. 2010

Haematologica

168

135

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Section: Introductionmentioning

confidence: 99%

Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization

Hill¹,

Rother²,

Arnold³

et al. 2010

Haematologica

168

135

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“…PNH testing is becoming increasingly important for disease monitoring in terms of progression, regression, remission, or response to therapy with humanized monoclonal antibody (MoAb) against the C5 complement protein (11)(12)(13). The implementation of high sensitivity assays enables additional screening for minor PNH clones (<1.0%) and/or cells with PNH phenotype (<0.1%) in patients with aplastic anemia, myelodysplastic syndrome, and other bone marrow failures (14)(15)(16)(17).…”

mentioning

confidence: 99%

Intra‐ and interlaboratory variability of paroxysmal nocturnal hemoglobinuria testing by flow cytometry following the 2012 Practical Guidelines for high‐sensitivity paroxysmal nocturnal hemoglobinuria testing

Marinov

Kohoutová

Tkáčová

et al. 2013

Cytometry Part B Clinical

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Background: Sutherland et al. recently published the Practical Guidelines for high-sensitivity detection of paroxysmal nocturnal hemoglobinuria (PNH) clones by flow cytometry (FCM), containing concise protocols for PNH testing.Methods: Using this approach, we studied the intra-and interlaboratory variability observed in a multicenter study in which fresh blood samples containing three clinically relevant PNH clone sizes within the granulocytic, monocytic, and red blood cell (RBC) populations were shipped to each participating center.Results : Conclusion: Our results demonstrate very good intra-and interlaboratory performance characteristics for both precision and reproducibility analyses and excellent correlation and agreement between centers for all target PNH clone sizes. Our data confirm the reliability and robustness of the recently published Practical Guidelines approach for high sensitivity PNH testing by flow cytometry and suggest that such an approach represents an excellent basis for standardization of PNH testing by flow cytometry. V C 2013 International Clinical Cytometry Society

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“…Nonmyeloablative conditioning regimens are being investigated in PNH [34] and could potentially expand the population of older patients eligible for HSCT. Eculizumab, a humanized monoclonal antibody against complement C5, has been shown to reduce hemolysis in clinical trials [35,36] and may be a promising future therapy for patients with PNH. …”

Section: Discussionmentioning

confidence: 99%

A patient with paroxysmal nocturnal hemoglobinuria, T cell large granular lymphocyte clonal expansion, and monoclonal gammopathy of undetermined significance

Fukumoto¹,

Gotlib

2006

Am. J. Hematol.

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Paroxysmal nocturnal hemoglobinuria (PNH) has been described in association separately with T cell large granular lymphocyte (LGL) clonal expansions and plasma cell dyscrasias. We describe a patient with anemia related to hemolytic PNH, with concurrent T cell LGL oligoclonal expansion and IgG k monoclonal gammopathy of undetermined significance. Peripheral blood flow cytometry revealed decreased expression of CD55 and CD59 on erythrocytes and decreased expression of CD55 and CD66 on neutrophils. An LGL population was present in the peripheral blood and was characterized as oligoclonal by polymerase chain reaction-based analysis of the T cell receptor c-chain variable region. Serum protein electrophoresis with immunofixation showed a low level IgG k monoclonal protein.We describe the diagnostic evaluation of this patient and provide a brief review of the reported associations among PNH, LGL clonal expansion, and monoclonal gammopathy.

show abstract

Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria

Cited by 197 publications

References 25 publications

Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization

Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization

Intra‐ and interlaboratory variability of paroxysmal nocturnal hemoglobinuria testing by flow cytometry following the 2012 Practical Guidelines for high‐sensitivity paroxysmal nocturnal hemoglobinuria testing

A patient with paroxysmal nocturnal hemoglobinuria, T cell large granular lymphocyte clonal expansion, and monoclonal gammopathy of undetermined significance

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