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https://www.readbyqxmd.com/read/28630122/eculizumab-blocks-vaccine-induced-opsonophagocytic-killing-of-meningococci-by-whole-blood-from-immunized-adults
#1
Monica Konar, Dan M Granoff
Eculizumab, a humanized anti-complement C5 monoclonal antibody 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, but 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...
June 19, 2017: Blood
https://www.readbyqxmd.com/read/28630090/t-cell-transcriptomes-from-paroxysmal-nocturnal-hemoglobinuria-patients-reveal-novel-signaling-pathways
#2
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...
June 19, 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
#3
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
#4
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
https://www.readbyqxmd.com/read/28621538/discovery-of-highly-potent-and-selective-small-molecule-reversible-factor-d-inhibitors-demonstrating-alternative-complement-pathway-inhibition-in-vivo
#5
Edwige Lorthiois, Karen Anderson, Anna Vulpetti, Olivier Rogel, Frederic Cumin, Nils Ostermann, Stefan Steinbacher, Aengus Mac Sweeney, Omar Delgado, Sha-Mei Liao, Stefan Randl, Simon Rüdisser, Solene Dussauge, Kamal Fettis, Laurence Kieffer, Andrea de Ekernez, Louis Yang, Constanze Hartwieg, Upendra A Argikar, Laura R LaBonte, Ronald Newton, Viral Kansara, Stefanie Flohr, Ulrich Hommel, Bruce Jaffee, Jürgen Maibaum
The highly specific S1 serine protease Factor D (FD) plays a central role in the amplification of the complement alternative pathway (AP) of the innate immune system. Genetic associations in humans have implicated AP activation in age-related macular degeneration (AMD), and AP dysfunction predisposes individuals to disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The combination of structure-based hit identification and subsequent optimization of the center (S)-proline-based lead 7 has led to the discovery of non-covalent reversible and selective human Factor D (FD) inhibitors with drug-like properties...
June 16, 2017: Journal of Medicinal Chemistry
https://www.readbyqxmd.com/read/28608499/clinical-benefit-of-eculizumab-in-patients-with-no-transfusion-history-in-the-international-paroxysmal-nocturnal-haemoglobinuria-registry
#6
A M Almeida, C Bedrosian, A Cole, P Muus, H Schrezenmeier, J Szer, W F Rosse
BACKGROUND: Eculizumab reduces intravascular haemolysis and improves disease symptoms in patients with paroxysmal nocturnal haemoglobinuria (PNH). AIMS: To characterize, in a real-world setting, effect of eculizumab in patients with haemolytic PNH (LDH ≥1.5 ULN) and no history of red blood cell transfusion, including those with high disease activity (HDA). METHODS: Three populations from the International PNH Registry were studied: 1) non-transfused, untreated, 2) non-transfused, eculizumab-treated, 3) transfused, eculizumab-treated (≥1 transfusions in 6 months prior to eculizumab initiation)...
June 13, 2017: Internal Medicine Journal
https://www.readbyqxmd.com/read/28589020/countdown-to-2015-an-analysis-of-donor-funding-for-prenatal-and-neonatal-health-2003-2013
#7
Catherine Pitt, Christopher Grollman, Melisa Martínez-Álvarez, Leonardo Arregoces, Joy E Lawn, Josephine Borghi
BACKGROUND: In 2015, 5.3 million babies died in the third trimester of pregnancy and first month following birth. Progress in reducing neonatal mortality and stillbirth rates has lagged behind the substantial progress in reducing postneonatal and maternal mortality rates. The benefits to prenatal and neonatal health (PNH) from maternal and child health investments cannot be assumed. METHODS: We analysed donor funding for PNH over the period 2003-2013. We used an exhaustive key term search followed by manual review and classification to identify official development assistance and private grant (ODA+) disbursement records in the Countdown to 2015 ODA+ Database...
March 2017: BMJ Global Health
https://www.readbyqxmd.com/read/28578116/the-timed-up-and-go-test-as-a-diagnostic-criterion-in-normal-pressure-hydrocephalus
#8
Gabriel André Silva Mendes, Matheus Fernandes de Oliveira, Fernando Campos Gomes Pinto
INTRODUCTION: Normal Pressure Hydrocephalus (NPH) is a disease characterized by gait disturbance, urinary incontinence and dementia. Our objectives were to define an average value of the test for the population, check the specificity and test sensitivity as evaluation criteria and diagnostic testing and correlate with other already used more frequently. METHODS: A study conducted at the Neurosurgery Division of the Hospital do Servidor Público Estadual de São Paulo in which a group of 30 patients with PNH was submitted to the Mini Mental State Test Examination, Time Up and Go (TUG) and Japanese scale for NPH, prior to the Tap Test 3 hours and 72 hours after the Tap Test...
May 31, 2017: World Neurosurgery
https://www.readbyqxmd.com/read/28574201/standardized-high-sensitivity-flow-cytometry-testing-for-paroxysmal-nocturnal-hemoglobinuria-in-children-with-acquired-bone-marrow-failure-disorders-a-single-center-u-s-study
#9
Rachel E Donohue, Andrea N Marcogliese, Ghadir S Sasa, M Tarek Elghetany, Alka A Redkar, Alison A Bertuch, Choladda V Curry
BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematopoietic stem cell disorder that has not been well documented in children, particularly those with acquired bone marrow failure disorders (ABMFD) - acquired aplastic anemia (AAA) and myelodysplastic syndrome (MDS). Therefore, we sought to determine the prevalence of PNH populations in children with ABMFD. METHODS: PNH testing was performed in children with an ABMFD diagnosis using high sensitivity (≥0...
June 2, 2017: Cytometry. Part B, Clinical Cytometry
https://www.readbyqxmd.com/read/28560190/botulinum-as-a-toxin-for-treating-post-herpetic-neuralgia
#10
Xu-Dong Ding, Jing Zhong, Yan-Ping Liu, Hua-Xian Chen
BACKGROUND: We aimed to study the curative effects of botulinum A toxin (BTX-A) on the treatment of post-herpetic neuralgia (PNH). METHODS: We enrolled 58 PNH patients and treated them with hypodermic injection of BTX-A in Xiangyang No.1 People's Hospital, Hubei University of Medicine, Hubei, China. We measured and compared the Visual Analog Score (VAS), Neuropathy Pain Scale (NPS), Quality of Life Scale (SF-36) score, PNH seizure severity degree, seizure duration, frequency of attack and the use of painkillers before and after treatment...
May 2017: Iranian Journal of Public Health
https://www.readbyqxmd.com/read/28559287/occult-myeloproliferative-neoplasms-not-so-occult-any-more
#11
Dhauna Karam, Veena Iyer, Bharat Agrawal
Non-cirrhotic, non-malignant portal vein thrombosis (PVT) is commonly secondary to inherited or acquired prothrombotic states. However, even after extensive workup, 25% of patients with PVT have no apparent prothrombotic aetiology identified (idiopathic PVT). Inherited conditions include factor V Leiden, PT mutation and protein C/S/AT deficiency. Acquired conditions include APS, PNH and BCR-ABL 1-negative myeloproliferative neoplasms (MPN). BCR-ABL-1 negative MPNs are the most frequent underlying prothrombotic risk factor for PVT (15%-30%)...
May 30, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28516949/paroxysmal-nocturnal-haemoglobinuria
#12
REVIEW
Anita Hill, Amy E DeZern, Taroh Kinoshita, Robert A Brodsky
Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic stem cell (HSC) disease that presents with haemolytic anaemia, thrombosis and smooth muscle dystonias, as well as bone marrow failure in some cases. PNH is caused by somatic mutations in PIGA (which encodes phosphatidylinositol N-acetylglucosaminyltransferase subunit A) in one or more HSC clones. The gene product of PIGA is required for the biosynthesis of glycosylphosphatidylinositol (GPI) anchors; thus, PIGA mutations lead to a deficiency of GPI-anchored proteins, such as complement decay-accelerating factor (also known as CD55) and CD59 glycoprotein (CD59), which are both complement inhibitors...
May 18, 2017: Nature Reviews. Disease Primers
https://www.readbyqxmd.com/read/28484166/molecular-genetics-biochemistry-and-biology-of-pnh
#13
Taroh Kinoshita
Paroxysmal nocturnal hemoglobinuria (PNH) manifests by clonal expansion of mutant hematopoietic stem cells (HSCs) bearing a somatic mutation in the X-linked PIGA gene. PIGA mutations cause defective biosynthesis of GPI and cell surface deficiency of GPI-anchored proteins such as DAF and CD59, leading to intravascular hemolysis and thrombosis. These two major symptoms of PNH can be controlled by eculizumab, an anti-C5 monoclonal antibody. Bone marrow failure, the third major symptom of PNH, is autoimmune-mediated and contributes to the clonal expansion of GPI-defective HSCs by selectively attacking GPI-positive wild-type HSCs...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28446324/-research-advance-of-microparticles-in-hypercoagulation-of-hemolytic-anemia-review
#14
Xie Su, Peng Cheng, Dong-Hong Deng
Microparticles (MP) are small membrane vesicles released from many different cell types in response to cellular activation or apoptosis, which have the procoagulant effect. Hemolytic anemia(HA) is a type of anemia that have a short life expectancy of red blood cells due to the destruction which exceed the hematopoietic compensatory capacity of bone marrow. Sickle cell anemia(SCD), thalassemia and paroxysmal nocturnal hemoglobinuria(PNH) are all characterized by hypercoagulation and thromboembolism (TE). Research shows that MP can promote the formation of hypercoagulative state which in turn increases the risk of thromboembolism in HA...
April 2017: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28439081/long-lasting-neutralization-of-c5-by-sky59-a-novel-recycling-antibody-is-a-potential-therapy-for-complement-mediated-diseases
#15
Taku Fukuzawa, Zenjiro Sampei, Kenta Haraya, Yoshinao Ruike, Meiri Shida-Kawazoe, Yuichiro Shimizu, Siok Wan Gan, Machiko Irie, Yoshinori Tsuboi, Hitoshi Tai, Tetsushi Sakiyama, Akihisa Sakamoto, Shinya Ishii, Atsuhiko Maeda, Yuki Iwayanagi, Norihito Shibahara, Mitsuko Shibuya, Genki Nakamura, Takeru Nambu, Akira Hayasaka, Futa Mimoto, Yuu Okura, Yuji Hori, Kiyoshi Habu, Manabu Wada, Takaaki Miura, Tatsuhiko Tachibana, Kiyofumi Honda, Hiroyuki Tsunoda, Takehisa Kitazawa, Yoshiki Kawabe, Tomoyuki Igawa, Kunihiro Hattori, Junichi Nezu
Dysregulation of the complement system is linked to the pathogenesis of a variety of hematological disorders. Eculizumab, an anti-complement C5 monoclonal antibody, is the current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). However, because of high levels of C5 in plasma, eculizumab has to be administered biweekly by intravenous infusion. By applying recycling technology through pH-dependent binding to C5, we generated a novel humanized antibody against C5, SKY59, which has long-lasting neutralization of C5...
April 24, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28437723/clinical-course-and-disease-burden-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-by-hemolytic-status
#16
Mustafa N Yenerel, Petra Muus, Amanda Wilson, Jeff Szer
Disease characteristics of patients enrolled in the International PNH Registry were assessed during two follow-up periods based on hemolytic status while untreated with eculizumab: Non-hemolytic cohort: follow-up time defined as time from disease start until last reported untreated lactate dehydrogenase (LDH) value <1.5×upper limit normal (ULN); Hemolytic cohort: follow-up time defined as time from LDH ≥1.5×ULN at or post-disease start, to most recent untreated follow-up. A total of 1012 patients met criteria for the Non-hemolytic cohort and 1565 patients for the Hemolytic cohort; median (min, max) years of follow-up were 2...
June 2017: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28435653/paroxysmal-nocturnal-hemoglobinuria-in-the-differential-diagnosis-of-thrombocytopenia
#17
Fusun Gediz, Bahriye Kadriye Payzin, Ozlem Zekiye Cakmak, Yusuf Uzum, Damla Ernur, Fahri Sahin
Paroxysmal nocturnal hemoglobinuria (PNH) is a disease which diagnosis may be delayed due to variable clinical findings. We describe herein a case of PNH in a 21 year old woman who admitted with complaints of chronic weakness, intermittent spontaneous ecchymoses, and an intermittent abdominal pain. On laboratory tests thrombocytopenia and iron deficiency anemia without any clinical findings were found. Flow cytometric evaluations showed a PNH clone of 15% for erythrocytes, 64% for monocytes, and 60% for granulocytes...
February 23, 2017: Hematology Reports
https://www.readbyqxmd.com/read/28432724/fluorescent-aerolysin-flaer-based-paroxysmal-nocturnal-hemoglobinuria-pnh-screening-a-single-center-experience-from-india
#18
K Rahman, R Gupta, G Yadav, N Husein, M K Singh, S Nityanand
BACKGROUND: Fluorescent aerolysin (FLAER) has been recommended as an important part of antibody panel used for flow cytometric detection of paroxysmal nocturnal hemoglobinuria (PNH) clone. This study was aimed to observe the frequency of PNH-positive clones and their sizes in patients screened for various indications. METHOD: A retrospective analysis of 624 patients screened over a period of 30 months. Frequency and size of clone sizes noted, and laboratory parameters were compared among different groups of patient being screened...
June 2017: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/28418828/a-comparison-of-blood-and-cerebrospinal-fluid-cytokines-il-1%C3%AE-il-6-il-18-tnf-%C3%AE-in-neonates-with-perinatal-hypoxia
#19
Darinka Šumanović-Glamuzina, Filip Čulo, Melanie Ivana Čulo, Paško Konjevoda, Marjana Jerković-Raguž
Perinatal hypoxia-ischemia is a specific and important pathological event in neonatal care practice. The data on relationship between the concentrations of cytokines in blood and cerebrospinal fluid (CSF) and perinatal brain injury are scarce. The aim of this study is to evaluate changes in interleukin (IL-1β, IL-6, and IL-18) and tumor necrosis factor alpha (TNF-α) levels in newborns with perinatal hypoxia (PNH). CSF and serum samples of 35 term and near-term (35-40 weeks) newborns with PNH, at the age of 3-96 hours, were analyzed using enzyme-linked immunosorbent assay...
April 18, 2017: Bosnian Journal of Basic Medical Sciences
https://www.readbyqxmd.com/read/28411558/sporadic-periventricular-nodular-heterotopia-classification-phenotype-and-correlation-with-filamin-a-mutations
#20
Wenyu Liu, Bo Yan, Dongmei An, Jiahe Xiao, Fayun Hu, Dong Zhou
OBJECTIVE: The purpose of this study was to better delineate the clinical spectrum of periventricular nodular heterotopia (PNH) in a large patient population after long term follow up. Specifically, this study aimed to relate PNH subtypes to clinical or epileptic outcomes, epileptic discharges and underlying Filamin A (FLNA) mutations by analyzing anatomical features. METHODS: The study included 100 patients with radiologically confirmed nodular heterotopia. Patients' FLNA gene sequences and medical records were analyzed...
April 4, 2017: Epilepsy Research
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