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Paroxysmal nocturnal hemoglobinuria

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https://www.readbyqxmd.com/read/28918528/signaling-of-the-complement-cleavage-product-anaphylatoxin-c5a-through-c5ar-cd88-contributes-to-pharmacological-hematopoietic-stem-cell-mobilization
#1
Kamila Bujko, Sylwia Rzeszotek, Kai Hoehlig, Jun Yan, Axel Vater, Mariusz Z Ratajczak
Several mechanisms have been postulated for orchestrating the mobilization of hematopoietic stem/progenitor cells (HSPCs), and we previously proposed that activation of the complement cascade plays a crucial role in the initiation and execution of the egress of HSPCs from bone marrow (BM) into peripheral blood (PB). In support of this notion, we demonstrated that mice deficient in the mannan-binding lectin (MBL) pathway, which activates the proximal part of the complement cascade, as well as mice deficient in the fifth component of the complement cascade (C5), which is part of the distal part of the complement cascade, are poor mobilizers...
September 16, 2017: Stem Cell Reviews
https://www.readbyqxmd.com/read/28879212/safety-and-efficacy-of-the-complement-inhibitor-amy-101-in-a-natural-model-of-periodontitis-in-non-human-primates
#2
Tetsuhiro Kajikawa, Ruel A Briones, Ranillo R G Resuello, Joel V Tuplano, Edimara S Reis, Evlambia Hajishengallis, Cristina A G Garcia, Despina Yancopoulou, John D Lambris, George Hajishengallis
Periodontitis is a chronic inflammatory disease associated with overactivation of the complement system. Recent preclinical studies suggest that host-modulation therapies may contribute to effective treatment of human periodontitis, which may lead to loss of teeth and function if untreated. We previously showed that locally administered AMY-101 (Cp40), a peptidic inhibitor of the central complement component C3, can inhibit naturally occurring periodontitis in non-human primates (NHPs) when given once a week...
September 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28811010/distinct-clinical-characteristics-of-paroxysmal-nocturnal-hemoglobinuria-in-patients-in-southern-taiwan-a-multicenter-investigation
#3
Hui-Ching Wang, Ching-Yuan Kuo, I-Ting Liu, Tsai-Yun Chen, Yu-Hsiang Chang, Shyh-Jer Lin, Shih-Feng Cho, Yi-Chang Liu, Ta-Chih Liu, Sheng-Fung Lin, Chao-Sung Chang
Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare acquired disorder. The aim of this study was to investigate the demographics, clinical manifestations, and outcomes of PNH patients in southern Taiwan. Data on PNH patients diagnosed over a 30-year period (1985-2015) were retrospectively collected from four tertiary medical centers in southern Taiwan. Blood samples were collected for hematologic panel testing and flow cytometry detection of PNH clones. Radiologic studies were performed to assess the frequency of complications...
August 2017: Kaohsiung Journal of Medical Sciences
https://www.readbyqxmd.com/read/28808592/a-case-of-fulminant-meningococcemia-it-is-all-in-the-complement
#4
Kellie L Hawkins, Mariah Hoffman, Sonia Okuyama, Sarah E Rowan
Eculizumab is a novel monoclonal antibody that inhibits complement-mediated hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH). Complement deficiency is a well-known risk factor for meningococcal infection. We describe a case of a young patient with PNH treated with eculizumab who presented with a life-threatening case of nongroupable meningococcemia. As this new biologic agent becomes more widely prescribed, providers should be aware of the increased risk of meningococcemia. In addition to vaccination, providers may consider the use of oral penicillin for antibiotic prophylaxis against Neisseria meningitidis in these cases of functional complement deficiency...
2017: Case Reports in Infectious Diseases
https://www.readbyqxmd.com/read/28799485/monoclonal-antibodies-a-review
#5
Surjit Singh, Nitish Kumar, Pradeep Dwiwedi, Jaykaran Charan, Rimplejeet Kaur, Preeti Sidhu, Vinay Kumar Chugh
Over the last three decades, monoclonal antibodies (MAbs) have made a striking transformation from scientific tools to powerful human therapeutics. Muromonab CD3 a murine MAb, was first FDA approved therapeutic MAb for prevention of kidney transplant rejection. Since its approval in 1986, there has been decline in the further application and approvals until the late 1990s when the first chimeric Mab, Rituximab was approved for the treatment of low grade B cell lymphoma in 1997. With the approval by licensing authorities of chimeric, followed by humanized and then fully human monoclonal antibodies, rate of approval and monoclonal antibodies available in the market for the treatment of various diseases has increased dramatically...
August 9, 2017: Current Clinical Pharmacology
https://www.readbyqxmd.com/read/28796431/acute-kidney-injury-in-a-postpartum-woman-with-paroxysmal-nocturnal-hemoglobinuria-a-case-report-and-literature-review
#6
Masatoshi Nishimoto, Masaru Matsui, Hideo Tsushima, Kaori Tanabe, Miho Tagawa, Ken-Ich Samejima, Yasuhiro Akai, Yoshihiko Saito
Paroxysmal nocturnal hemoglobinuria is a rare clonal hematopoietic stem cell disorder characterized by intravascular hemolysis, hemoglobinuria, and inflammatory thrombotic state. Intravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH) can lead to acute and chronic renal injury through hemoglobin-mediated toxicity. A 32-year-old pregnant woman with myelodysplastic syndrome was admitted to our hospital with severe preeclampsia. Shortly after an urgent caesarean section, she became obtunded and showed signs of acute kidney injury (AKI) with anuria, severe intravascular hemolysis, and hypermagnesemia...
August 10, 2017: Hemodialysis International
https://www.readbyqxmd.com/read/28781057/multiple-lacunar-infarcts-in-paroxysmal-nocturnal-hemoglobinuria
#7
Alex Tiburtino Meira, Gabriel Sampaio Froehner, André Petean Trindade, Silméia Garcia Zanati Bazan, Gabriel Pereira Braga, Rodrigo Bazan
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disease characterized by clonal hematopoietic stem cell disorder, with increased mortality and morbidity. Venous thrombosis is the most common cause of mortality in PNH. The relationship between PNH and cerebrovascular disease is unclear; few cases are reported in the literature, most of them related to cerebral venous thrombosis; In PNH the involvement of intracranial and extracranial arterial sites is very rare. We report a case of a 49-year-old woman who has a medical history of diabetes mellitus, hypertension, and PNH and presented multiple lacunar strokes in a routine consultation with a hematologist...
August 3, 2017: Journal of Stroke and Cerebrovascular Diseases: the Official Journal of National Stroke Association
https://www.readbyqxmd.com/read/28766381/development-and-validation-of-an-enzyme-linked-immunosorbent-assay-to-measure-free-eculizumab-concentration-in-serum
#8
Christophe Passot, Céline Desvignes, David Ternant, Theodora Bejan-Angoulvant, Anne-Claire Duveau, Philippe Gatault, Gilles Paintaud
AIM: Eculizumab is a monoclonal antibody toward C5 fraction of the complement system. It is approved to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. To perform pharmacokinetic studies and therapeutic drug monitoring, a validated assay is required. MATERIALS & METHODS: An indirect ELISA with recombinant human C5 sensitized microtiter plates were developed. RESULTS: The assay allows the measurement of free eculizumab concentration in human serum...
August 2017: Bioanalysis
https://www.readbyqxmd.com/read/28761231/renal-manifestations-in-paroxysmal-nocturnal-hemoglobinuria
#9
R Ram, K P Adiraju, S Gudithi, K V Dakshinamurty
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired chronic disorder characterized by a triad of clinical features - hemolytic anemia, pancytopenia, and thrombosis. Not many reports of renal involvement in PNH are available in literature. We present a case series of PNH with renal involvement. We present the data of PNH patients who attended to Departments of General Medicine and Nephrology at a government-run tertiary care institute in South India. The diagnosis of PNH in these patients during initial phase, between 1998 and 2004 was based on sucrose lysis and Ham's test...
July 2017: Indian Journal of Nephrology
https://www.readbyqxmd.com/read/28761230/renal-biopsy-in-paroxysmal-nocturnal-hemoglobinuria-an-insight-into-the-spectrum-of-morphologic-changes
#10
V Puri, A Gandhi, S Sharma
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, potentially life-threatening disease of blood, characterized by complement-induced intravascular hemolytic anemia and thrombosis. PNH can sometimes present directly with renal manifestations, without showing any hematological manifestation. It, therefore, becomes essential for clinicians and pathologists to be aware of the spectrum of renal changes in PNH. The aim of this study was to document the morphologic changes observed in renal biopsies in patients with PNH...
July 2017: Indian Journal of Nephrology
https://www.readbyqxmd.com/read/28748287/presence-of-acute-and-chronic-renal-failure-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-results-of-a-retrospective-analysis-from-the-spanish-pnh-registry
#11
Ana Villegas, Ramiro Núñez, Anna Gaya, María Victoria Cuevas-Ruiz, José Miguel Bosch, Anna Carral, Beatriz Arrizabalaga, María Isabel Gómez-Roncero, Asunción Mora, Pilar Bravo, Esperanza Lavilla, Carmen Monteserín, Belén Hernández, Pilar Martínez-Barranco, Isidro Jarque, María Anunciación Urquía, Gloria García-Donas, Salut Brunet, Fernando Ataulfo González, Álvaro Urbano
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening blood disease. With the advent of eculizumab treatment, renal function has substantially improved, although no data from real-world clinical practice are available. An observational, retrospective, multicenter study was conducted in Spain on clinical data obtained from outpatient visits of patients with PNH (Spanish PNH Registry) who had experienced acute (ARF) or chronic (CRF) renal failure. Of the 128 patients registered (April 2014), 60 were diagnosed with classic PNH...
July 26, 2017: Annals of Hematology
https://www.readbyqxmd.com/read/28728504/the-diverse-expression-of-the-wt1-gene-in-patients-with-acquired-bone-marrow-failure-syndromes
#12
Yahong You, Jiali Huo, Shihong Lu, Yingqi Shao, Meili Ge, Jun Shi, Xingxin Li, Jinbo Huang, Zhendong Huang, Jing Zhang, Min Wang, Neng Nie, Yizhou Zheng
Acquired bone marrow failure syndromes (aBMFS) encompass a wide range of diseases. A study to investigate WT1 expression in BM was conducted in 387 patients with aBMFS in China. The WT1 level in patients with aplastic anemia (AA) was significantly lower than that in patients with paroxysmal nocturnal hemoglobinuria (PNH, p = .023) and myelodysplastic syndrome (MDS, p < .001). In addition, the WT1 level in patients with MDS significantly increased as the disease progressed to an advanced stage. Patients with hypoplastic MDS had a differentiated expression level of WT1 compared with that of NSAA (p < ...
July 21, 2017: Leukemia & Lymphoma
https://www.readbyqxmd.com/read/28698856/the-first-case-of-paroxysmal-nocturnal-hemoglobinuria-and-budd-chiari-syndrome-treated-with-complement-inhibitor-eculizumab-in-korea
#13
Hyerim Kim, In-Suk Kim, Su-Hee Cho, Hyun Ji Lee, Chulhun L Chang, Ki Tae Yoon
No abstract text is available yet for this article.
June 2017: Blood Research
https://www.readbyqxmd.com/read/28692147/persisting-hyperbilirubinemia-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-chronically-treated-with-eculizumab-the-role-of-hepatocanalicular-transporter-variants
#14
Ferras Alashkar, Susanne N Weber, Colin Vance, Dörte Herich-Terhürne, Ulrich Dührsen, Frank Lammert, Alexander Röth
BACKGROUND: Eculizumab-treated paroxysmal nocturnal hemoglobinuria (PNH) patients (pts) show a dramatic decrease in serum lactate dehydrogenase (LDH) activities and bilirubin concentrations. However, some pts remain hyperbilirubinemic, possibly indicating an inadequate response due to extravascular hemolysis. METHODS: Mutation analyses of hepatocanalicular transporter/nuclear receptor variants (ABCB4, ABCB11, ATP8B1, NR1H4) were performed in eight (five of eight males; mean age 38 years [range 26-68 years]) out of the 174 pts with PNH/-clone at our department due to a persistent increase in total bilirubin concentrations (median 3...
July 10, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/28675769/outcomes-of-allogeneic-stem-cell-transplantation-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-with-or-without-aplastic-anemia
#15
Sung-Eun Lee, Sung Soo Park, Young-Woo Jeon, Jae-Ho Yoon, Byung-Sik Cho, Ki-Sung Eom, Yoo-Jin Kim, Seok Lee, Chang-Ki Min, Hee-Je Kim, Seok-Goo Cho, Dong-Wook Kim, Woo-Sung Min, Jong Wook Lee
OBJECTIVE: The aim of this study was to evaluate the long-term outcomes of allogeneic stem cell transplantation (SCT) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with or without aplastic anemia (AA). METHOD: A total of 33 patients with PNH clones who underwent allogeneic SCT were analyzed. RESULTS: After a median follow-up of 57 months (range, 6.0-151.3), the 5-year estimated overall survival rate was 87.9±5.7%. Four patients died of transplant-related mortality (TRM)...
July 4, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/28646725/eculizumab-decreases-the-procoagulant-activity-of-extracellular-vesicles-in-paroxysmal-nocturnal-hemoglobinuria-a-pilot-prospective-longitudinal-clinical-study
#16
Adeline Wannez, Bérangère Devalet, Céline Bouvy, Julie Laloy, Benoit Bihin, Bernard Chatelain, Christian Chatelain, Jean-Michel Dogné, François Mullier
INTRODUCTION: Paroxysmal nocturnal hemoglobinuria (PNH) is a disease characterized by the susceptibility of blood cells to attack by the complement system, inducing extracellular vesicle (EV) production. Thromboembolism is the leading cause of death in this condition. Eculizumab, a humanized monoclonal antibody which inhibits the C5 protein of the complement, reduces the thrombotic risk in PNH. MATERIALS AND METHOD: We conducted a pilot, prospective, open-label, longitudinal clinical study with six PNH patients treated with eculizumab...
August 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28630122/eculizumab-treatment-and-impaired-opsonophagocytic-killing-of-meningococci-by-whole-blood-from-immunized-adults
#17
Monica Konar, Dan M Granoff
Eculizumab, a humanized anti-complement C5 monoclonal antibody (mAb) for treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome, blocks the terminal complement pathway required for serum bactericidal activity (SBA). Because treated patients are at >1000-fold increased risk of meningococcal disease, vaccination is recommended; whether vaccination can protect by opsonophagocytic activity in the absence of SBA is not known. Meningococci were added to anticoagulated blood from 12 healthy adults vaccinated with meningococcal serogroup B and serogroup A, C, W, Y vaccines...
August 17, 2017: Blood
https://www.readbyqxmd.com/read/28630090/t-cell-transcriptomes-from-paroxysmal-nocturnal-hemoglobinuria-patients-reveal-novel-signaling-pathways
#18
Kohei Hosokawa, Sachiko Kajigaya, Keyvan Keyvanfar, Wangmin Qiao, Yanling Xie, Danielle M Townsley, Xingmin Feng, Neal S Young
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder originating from hematopoietic stem cells and is a life-threating disease characterized by intravascular hemolysis, bone marrow (BM) failure, and venous thrombosis. The etiology of PNH is a somatic mutation in the phosphatidylinositol glycan class A gene (PIG-A) on the X chromosome, which blocks synthesis of the glycolipid moiety and causes deficiency in GPI-anchored proteins. PNH is closely related to aplastic anemia, in which T cells mediate destruction of BM...
July 15, 2017: Journal of Immunology: Official Journal of the American Association of Immunologists
https://www.readbyqxmd.com/read/28629435/eculizumab-treatment-stochastic-occurrence-of-c3-binding-to-individual-pnh-erythrocytes
#19
Michela Sica, Tommaso Rondelli, Patrizia Ricci, Maria De Angioletti, Antonio M Risitano, Rosario Notaro
BACKGROUND: C5 blockade by eculizumab prevents complement-mediated intravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH). However, C3-bound PNH red blood cells (RBCs), arising in almost all treated patients, may undergo extravascular hemolysis reducing clinical benefits. Despite the uniform deficiency of CD55 and of CD59, there are always two distinct populations of PNH RBCs, with (C3+) and without (C3-) C3 binding. METHODS: To investigate this paradox, the phenomenon has been modeled in vitro by incubating RBCs from eculizumab untreated PNH patients with compatible sera containing eculizumab, and by assessing the C3 binding after activation of complement alternative pathway...
June 19, 2017: Journal of Hematology & Oncology
https://www.readbyqxmd.com/read/28626543/cerebral-stroke-in-a-teenage-girl-with-paroxysmal-nocturnal-hemoglobinuria
#20
Francesco Gervasi, Lucia D'Amelio, Antonino Trizzino, Fabrizia Ferraro, Delia Russo, Giuseppe Santangelo, Francesca Cardella, Angela Trizzino, Floriana Di Marco, Piero Farruggia
We report a case of paroxysmal nocturnal hemoglobinuria (PNH) in a 14 year-old girl presenting a cerebral arterial thrombosis. The initial diagnosis was carential anemia due to menarche following identification of slight macrocytic anemia, leucopenia and mild thrombocytopenia at routine blood analysis. The child was eventually referred to a children's hospital after the onset of progressive fatigue, anorexia and paleness. Severe anemia (hemoglobin 6 g/dL) with negative Coombs test, mild leucopenia (white blood cells 4...
June 1, 2017: Hematology Reports
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