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ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center - Research & Reviews

SOLIRIS® (ECULIZUMAB)
https://alexion.com/Products/Soliris

Soliris® is a first-in-class terminal complement inhibitor discovered, developed, and commercialized by Alexion. Soliris® works by inhibiting terminal complement, a part of the immune system that, when activated in an uncontrolled manner, plays a role in serious diseases like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (...

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PN...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6368201
Blood Kulasekararaj AG, Hill A et. al.

Dec 4th, 2018 - Ravulizumab, a new complement component C5 inhibitor administered every 8 weeks, was noninferior to eculizumab administered every 2 weeks in complement-inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria (PNH). This study assessed noninferiority of ravulizumab to eculizumab in clinically stable PNH patients during previous eculizumab therapy. In this phase 3, open-label, multicent...

Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inh...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6367644
Blood Lee JW, Sicre de Fontbrune F et. al.

Dec 4th, 2018 - Ravulizumab (ALXN1210), a new complement C5 inhibitor, provides immediate, complete, and sustained C5 inhibition. This phase 3, open-label study assessed the noninferiority of ravulizumab to eculizumab in complement inhibitor-naive adults with paroxysmal nocturnal hemoglobinuria (PNH). Patients with lactate dehydrogenase (LDH) ≥1.5 times the upper limit of normal and at least 1 PNH symptom were...

Detection of paroxysmal nocturnal hemoglobinuria clones to exclude inherited bone marro...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4161035
European Journal of Haematology; DeZern AE, Symons HJ et. al.

Mar 12th, 2014 - Inherited bone marrow failure syndromes (IBMFS) and acquired aplastic anemia (AA) are life-threatening marrow failure disorders. These entities can be difficult to distinguish because they present similarly. Correct diagnosis is imperative for proper therapy. This is a retrospective, single-center study of patients <40 yr of age, evaluated for bone marrow failure, and assayed for the presence o...

Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemog...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2857185
Haematologica Hill A, Rother RP et. al.

Feb 10th, 2010 - Paroxysmal nocturnal hemoglobinuria is an acquired hemolytic anemia characterized by intravascular hemolysis which has been demonstrated to be effectively controlled with eculizumab. However, lactate dehydrogenase levels remain slightly elevated and haptoglobin levels remain low in some patients suggesting residual low-level hemolysis. This may be due to C3-mediated clearance of paroxysmal noct...

Spectrum of renal involvement in paroxysmal nocturnal hemoglobinuria: report of three c...
https://doi.org/10.1007/s11255-008-9356-5
International Urology and Nephrology; Nair RK, Khaira A et. al.

Mar 27th, 2008 - Renal involvement in paroxysmal nocturnal hemoglobinuria (PNH) is usually clinically not apparent but, in cases with clinical involvement, varies from reversible acute dysfunction to chronic irreversible damage. We report two cases of acute kidney injury and one case of chronic kidney disease due to PNH. In all three cases a diagnosis of PNH was made after detection of renal dysfunction. The re...

Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal...
https://doi.org/10.1002/pbc.25068
Pediatric Blood & Cancer; Reiss UM, Schwartz J et. al.

Apr 29th, 2014 - Paroxysmal nocturnal hemoglobinuria (PNH) is rare in children, but represents a similarly serious and chronic condition as in adults. Children with PNH frequently experience complications of chronic hemolysis, recurrent thrombosis, marrow failure, serious infections, abdominal pain, chronic fatigue, and decreased quality of life with reduced survival. The terminal complement inhibitor eculizuma...

Polymorphism of the complement receptor 1 gene correlates with the hematologic response...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3912955
Haematologica Rondelli T, Risitano AM et. al.

Sep 16th, 2013 - Complement blockade by eculizumab is clinically effective in hemolytic paroxysmal nocturnal hemoglobinuria. However, the response is variable and some patients remain dependent on red blood cell transfusions. In 72 patients with hemolytic paroxysmal nocturnal hemoglobinuria on eculizumab we tested the hypothesis that response may depend on genetic polymorphisms of complement-related genes. We f...

Strategy for bone marrow transplantation in eculizumab-treated paroxysmal nocturnal hem...
https://doi.org/10.1007/s12185-011-0931-7
International Journal of Hematology; Taniguchi K, Okada M et. al.

Sep 19th, 2011 - Although the recent introduction of eculizumab has had a significant impact on the management of paroxysmal nocturnal hemoglobinuria (PNH), bone marrow transplantation (BMT) remains the only therapeutic option for patients who develop severe aplasia in the clinical course of PNH. However, information regarding BMT for eculizumab-treated PNH patients is scarce, and two major points-the optimal d...

Standardizing Leucocyte PNH clone detection: An international study.
https://doi.org/10.1002/cytob.21174
Cytometry. Part B, Clinical Cytometry; Fletcher M, Sutherland DR et. al.

Mar 24th, 2014 - Background: Consensus and Practical Guidelines for robust high-sensitivity detection of glycophosphatidylinostitol (GPI)-deficient structures on Red Blood Cells (RBCs) and White Blood Cells (WBCs) in Paroxysmal Nocturnal Hemoglobinuria (PNH) were recently published. Methods: UK NEQAS LI issued 3 stabilized samples manufactured to contain no PNH cells (normal), approximately 0.1% and 8% PNH leuc...

Fanconi syndrome and CKD in a patient with paroxysmal nocturnal hemoglobinuria and hemo...
https://doi.org/10.1053/j.ajkd.2009.07.022
American Journal of Kidney Diseases : the Official Journa... Hsiao PJ, Wang SC et. al.

Oct 16th, 2009 - Fanconi syndrome and chronic kidney disease associated with paroxysmal nocturnal hemoglobinuria is rarely reported. We describe a 51-year-old woman with glomerular filtration rate decrease and hypokalemia, glucosuria, and proteinuria during a 4-year period. Paroxysmal nocturnal hemoglobinuria was diagnosed 17 years earlier, and she has received multiple blood transfusions because of hemolytic e...

Natural history of paroxysmal nocturnal hemoglobinuria clones in patients presenting as...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3120909
European Journal of Haematology; Pu JJ, Mukhina G et. al.

Mar 30th, 2011 - To investigate the natural history of paroxysmal nocturnal hemoglobinuria (PNH) clones in patients with acquired aplastic anemia (AA). Twenty-seven patients with AA and a detectable PNH clone were monitored for a median of 5.7 years (range 1.5-11.5 years). Twenty-two patients received high-dose cyclophosphamide (HiCy) therapy. The erythrocyte and granulocyte PNH clone sizes were measured using ...

Mechanisms and clinical implications of thrombosis in paroxysmal nocturnal hemoglobinuria.
https://doi.org/10.1111/j.1538-7836.2011.04562.x
Journal of Thrombosis and Haemostasis : JTH; Van Bijnen ST, Van Heerde WL et. al.

Nov 14th, 2011 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disease characterized by a clone of blood cells lacking glycosyl phosphatidylinositol (GPI)-anchored proteins at the cell membrane. Deficiency of the GPI-anchored complement inhibitors CD55 and CD59 on erythrocytes leads to intravascular hemolysis upon complement activation. Apart from hemolysis, another prominent feature is a highly ...

Genetic variants in C5 and poor response to eculizumab.
https://doi.org/10.1056/NEJMoa1311084
The New England Journal of Medicine; Nishimura J, Yamamoto M et. al.

Feb 13th, 2014 - Eculizumab is a humanized monoclonal antibody that targets complement protein C5 and inhibits terminal complement-mediated hemolysis associated with paroxysmal nocturnal hemoglobinuria (PNH). The molecular basis for the poor response to eculizumab in a small population of Japanese patients is unclear. We assessed the sequences of the gene encoding C5 in patients with PNH who had either a good o...

Structural Basis for Eculizumab-Mediated Inhibition of the Complement Terminal Pathway.
https://doi.org/10.4049/jimmunol.1600280
Journal of Immunology (Baltimore, Md. : 1950); Schatz-Jakobsen JA, Zhang Y et. al.

May 19th, 2016 - Eculizumab is a humanized mAb approved for treatment of patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Eculizumab binds complement component C5 and prevents its cleavage by C5 convertases, inhibiting release of both the proinflammatory metabolite C5a and formation of the membrane attack complex via C5b. In this study, we present the crystal structure o...

Paroxysmal nocturnal hemoglobinuria.
https://doi.org/10.1097/MOH.0b013e328351c348
Current Opinion in Hematology; Parker CJ

Mar 7th, 2012 - The aim is to report on recent observations related to the natural history of paroxysmal nocturnal hemoglobinuria (PNH) and to review new therapeutic strategies for controlling the hemolysis of PNH. This review focuses on studies designed to characterize the long-term outcome of patients with PNH treated with eculizumab and to define the relationship between PNH and bone marrow failure syndrome...

The small population of PIG-A mutant cells in myelodysplastic syndromes do not arise fr...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3409821
Haematologica Pu JJ, Hu R et. al.

Feb 8th, 2012 - Patients with paroxysmal nocturnal hemoglobinuria harbor clonal glycosylphosphatidylinositol-anchor deficient cells arising from a multipotent hematopoietic stem cell acquiring a PIG-A mutation. Many patients with aplastic anemia and myelodysplastic syndromes also harbor small populations of glycosylphosphatidylinositol-anchor deficient cells. Patients with aplastic anemia often evolve into par...

Paroxysmal nocturnal hemoglobinuria and pregnancy before the eculizumab era: the French...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3166097
Haematologica de Guibert S, Peffault de Latour R et. al.

May 24th, 2011 - Pregnancy in women with paroxysmal nocturnal hemoglobinuria is rare, with few reports on maternal and fetal mortality rates. A specific questionnaire designed to solicit data on pregnancies in women with paroxysmal nocturnal hemoglobinuria was sent to all members of the French Society of Hematology in January 2008. We identified 27 pregnancies in 22 women at 10 French Society of Hematology cent...

Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3487438
Haematologica Peffault de Latour R, Schrezenmeier H et. al.

Jun 12th, 2012 - In the era of eculizumab, identifying patients with paroxysmal nocturnal hemoglobinuria who may benefit from allogeneic stem cell transplantation is challenging. We describe the characteristics and overall survival of 211 patients transplanted for paroxysmal nocturnal hemoglobinuria in 83 EBMT centers from 1978 to 2007. Next, we conducted a comparison with a cohort of 402 non-transplanted patie...

Managing a pregnant patient with paroxysmal nocturnal hemoglobinuria in the era of ecul...
https://doi.org/10.1016/j.leukres.2009.10.025
Leukemia Research; Danilov AV, Brodsky RA et. al.

Dec 3rd, 2009 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal stem cell disorder, which affects women of child-bearing age. PNH is associated with thrombotic complications, which are the main causes of morbidity and mortality. Management of a pregnant woman with PNH remains a challenge due to high incidence of thrombotic complications and the difficulty of differentiating a PNH crisis from the com...