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https://www.readbyqxmd.com/read/28516949/paroxysmal-nocturnal-haemoglobinuria
#1
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/28509134/two-cases-of-atypical-hemolytic-uremic-syndrome-ahus-and-eosinophilic-granulomatosis-with-polyangiitis-egpa-a-possible-relationship
#2
Mercedes Cao, Tamara Ferreiro, Bruna N Leite, Francisco Pita, Luis Bolaños, Francisco Valdés, Angel Alonso, Eduardo Vázquez, Juan Mosquera, María Trigás, Santiago Rodríguez
Atypical hemolytic uremic syndrome (aHUS) is a rare disease characterized by hemolysis, thrombocytopenia, and renal failure. It is related to genetic mutations of the alternative complement pathway and is difficult to differentiate from other prothrombotic microangiopathies. Eosinophilic granulomatosis with polyangiitis (EGPA) (Churg-Strauss syndrome, CSS) is a systemic ANCA-associated vasculitis and a hypereosinophilic disorder where eosinophils seem to induce cell apoptosis and necrosis and therefore, vasculitis...
March 1, 2017: CEN Case Reports
https://www.readbyqxmd.com/read/28508588/at-the-cross-section-of-thrombotic-microangiopathy-and-atypical-hemolytic-uremic-syndrome-a-narrative-review-of-differential-diagnostics-and-a-problematization-of-nomenclature
#3
REVIEW
Alexander Åkesson, Eva Zetterberg, Jenny Klintman
Complement-mediated atypical hemolytic uremic syndrome (aHUS) is a rare disease associated with high mortality and morbidity. Renal biopsies often indicate thrombotic microangiopathy (TMA). The condition is caused by an excessive activation of the alternative pathway leading to depositions of membrane attack complexes (MAC) on host cells. It may depend on mutations in complement components and regulatory proteins, or the formation of complement-specific antibodies. Mainly, an environmental trigger (e.g. infection) is needed for the excessive response to develop...
May 16, 2017: Therapeutic Apheresis and Dialysis
https://www.readbyqxmd.com/read/28502324/favorable-long-term-outcomes-of-isolated-liver%C3%A2-transplantation-in-a-child-with-atypical-hemolytic-uremic-syndrome-caused-by-a%C3%A2-novel%C3%A2-complement-factor-h-mutation%C3%A2
#4
Heeyeon Cho, Yeonhee Lee
Atypical hemolytic uremic syndrome (aHUS) is a rare syndrome characterized by microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney injury that is usually caused by complement dysregulation. Complement factor H (CFH) is a regulator of the complement system produced in the liver, and CFH gene mutations are the most frequent causes of aHUS. To date, the therapeutic options for aHUS with CFH mutations have consisted of plasma infusions, plasma exchange, kidney transplantation, isolated liver transplantation, or combined liver and kidney transplantation...
May 15, 2017: Clinical Nephrology
https://www.readbyqxmd.com/read/28499586/-gemcitabine-induced-thrombotic-microangiopathy-can-we%C3%A2-improve-screening-and%C3%A2-treatment
#5
Xavier Charmetant, Anne Jolivot, Thomas Fournier, Jean-Charles Puthet, Philippe Cassier, Sandrine Lemoine, Laurent Juillard
Thrombotic microangiopathy is a rare but severe complication of treatment with gemcitabine. Its prevalence increases because gemcitabine's indications are growing. We report four cases, which presented with common clinical and biological manifestations, i.e. high blood pressure, proteinuria and increasing plasmatic creatinine level. However, severity was not similar, hemodialysis was inconstant. There is no consensus on treatment for this condition. Stopping gemcitabine is essential. Treatment was dispensed considering the severity of the presentation: plasma exchange therapy of variable outcome, and eculizumab, which was efficient when used...
May 9, 2017: Néphrologie & Thérapeutique
https://www.readbyqxmd.com/read/28488841/catastrophic-antiphospholipid-syndrome-an-update
#6
Gerard Espinosa, Ignasi Rodriguez-Pinto, Ricard Cervera
Catastrophic antiphospholipid syndrome (CAPS) is a rare variant that accounts for 1% of patients with APS. Despite its low frequency, the mortality-related is very high ranging from 50% of patients in the first series to 37% in the most recent data. The current knowledge of this potential devastating entity comes from the International Registry of patients with CAPS, named CAPS Registry. Small vessel thrombosis, laboratory features of microangiopathic haemolytic anemia, and development of multisystem involvement in a very short period of time are the main characteristics of this syndrome...
May 8, 2017: Panminerva Medica
https://www.readbyqxmd.com/read/28487302/prophylactic-use-of-eculizumab-during-surgery-in-chronic-cold-agglutinin-disease
#7
Eirik Tjønnfjord, Øystein A Vengen, Sigbjørn Berentsen, Geir Erland Tjønnfjord
Primary chronic cold agglutinin disease (CAD) is an autoimmune haemolytic anaemia in which a specific bone marrow lymphoproliferative disorder causes production of cold agglutinins (CA). Binding of CA to erythrocyte surface antigens results in a predominantly extravascular haemolysis that is entirely complement dependent. Because of complement activation, exacerbations are common during febrile infections, trauma or major surgery. Involvement of the terminal complement pathway with C5-mediated intravascular haemolysis is probably not prominent in stable disease but is supposed to be of importance in exacerbations following acute phase reaction...
May 9, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28484166/molecular-genetics-biochemistry-and-biology-of-pnh
#8
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/28477014/anti-complement-component-5-antibody-targeting-mg4-domain-inhibits-choroidal-neovascularization
#9
Dong Hyun Jo, Jin Hyoung Kim, Wonjun Yang, Hyori Kim, Shinjae Chang, Dongjo Kim, Minseok Chang, Kihwang Lee, Junho Chung, Jeong Hun Kim
Age-related macular degeneration (AMD) is one of the main causes of visual impairment in adults. Visual deterioration is more prominent in neovascular AMD with choroidal neovascularization (CNV). Clinical and postmortem studies suggested that complement system activation might induce CNV. In this study, we demonstrated that an anti-mouse complement component 5 (C5) antibody targeting MG4 domain of β chain effectively inhibited CNV which was induced by laser photocoagulation in mice. The targeted epitope of this anti-C5 antibody was different from that of currently utilized anti-C5 antibody (eculizumab) in the MG7 domain in which a single nucleotide polymorphism (R885H/C) results in poor response to eculizumab...
April 19, 2017: Oncotarget
https://www.readbyqxmd.com/read/28461395/eculizumab-cessation-in-atypical-hemolytic-uremic-syndrome
#10
Samuel A Merrill, Zachary D Brittingham, Xuan Yuan, Alison R Moliterno, C John Sperati, Robert A Brodsky
No abstract text is available yet for this article.
May 1, 2017: Blood
https://www.readbyqxmd.com/read/28458317/long-term-eculizumab-treatment-contributes-to-recovery-from-end-stage-renal-disease-caused-by-atypical-hemolytic-uremic-syndrome
#11
Yuji Yamada, Ryohei Abe, Yutaka Okano, Yoshitaka Miyakawa
We experienced a favorable outcome in an adult case of atypical hemolytic uremic syndrome (aHUS) after long-term eculizumab treatment. A 38-year-old Japanese man with a history of central retinal vein occlusion was admitted to our hospital with progressive dyspnea. He was found to have non-immune hemolytic anemia, thrombocytopenia, and acute renal failure two weeks after an episode of the common cold. Plasma exchange was ineffective; therefore, we initiated eculizumab after we excluded other thrombotic microangiopathies...
2017: Internal Medicine
https://www.readbyqxmd.com/read/28455885/an-extremely-rare-splice-site-mutation-in-the-gene-encoding-complement-factor-i-in-a-patient-with-atypical-hemolytic-uremic-syndrome
#12
Tina S Ipe, Jooeun Lim, Meredith Anne Reyes, Mike Ero, Christopher Leveque, Bradley Lewis, Jamey Kain
BACKGROUND: Atypical hemolytic uremic syndrome (aHUS) is a rare disease characterized by thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney failure. The disease is difficult to diagnose due to its similarity with other hematologic disorders, such as thrombotic thrombocytopenic purpura (TTP). However, genetic mutations are found in 50-70% of patients with aHUS and can be useful in its diagnosis. STUDY DESIGN AND METHODS: A 40-year-old male presented to our hospital with acute kidney injury, evidenced by high creatinine levels (8...
April 28, 2017: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/28446488/eculizumab-in-a-child-with-atypical-haemolytic-uraemic-syndrome-and-haemophagocytic-lymphohistiocytosis-triggered-by-cytomegalovirus-infection
#13
Gloria M Fraga-Rodriguez, Sonia Brió-Sanagustin, Eulalia Turón-Viñas, Bradley P Dixon, Eduardo Carreras-González
We present the case of a 21-month-old girl with two rare and life-threatening conditions, atypical haemolytic uraemic syndrome (aHUS) and haemophagocytic lymphohistiocytosis (HLH), triggered by a cytomegalovirus (CMV) infection. Soon after admission, the girl became anuric and required continuous venovenous haemodiafiltration.Initial treatments included methylprednisolone, fibrinogen and plasma infusion (for HLH), plasmapheresis (for thrombotic microangiopathy), immunoglobulins (for inflammation), ganciclovir (for CMV infection) and the antibiotic cefotaxime...
April 26, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28439485/treatment-of-atypical-hemolytic-uremic-syndrome-in-the-era-of-eculizumab
#14
Rawaa Ebrahem, Salam Kadhem, Quoc Truong
Hemolytic-uremic syndrome (HUS) is the triad of microangiopathic hemolytic anemia (MAHA), thrombocytopenia, and acute kidney injury (AKI); the main cause of multi-organ failure is related to thrombotic microangiopathy (TMA). Atypical HUS (aHUS) is a disease of uncontrolled complement activation associated with a high mortality rate and most cases progress to end-stage renal disease. About 50% of patients with this syndrome carry mutations in genes that encode complement proteins. Also, aHUS constitutes an over-activation of the complement pathway which is either inherited, acquired, or both...
March 23, 2017: Curēus
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...
March 27, 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/28428030/complement-inhibition-with-eculizumab-for-thrombotic-microangiopathy-rescues-a-living-donor-kidney-transplant-in-a-patient-with-antiphospholipid-antibody-syndrome
#18
Praveen Ramakrishnan Geethakumari, Patrick Mille, Rakesh Gulati, Srikanth Nagalla
Antiphospholipid antibody syndrome (APS) is an enigmatic heterogeneous disorder despite several revelations in its pathobiology. Renal transplantation in patients with APS has been notoriously difficult due to the high risk of development of thrombotic microangiopathy (TMA), which is often refractory to conventional treatment modalities such as aggressive anticoagulation and plasmapheresis. We describe a case of a 58-year-old male with secondary APS undergoing living unrelated renal transplantation for end-stage renal disease from lupus nephritis...
March 10, 2017: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/28419995/hemolytic-uremic-syndrome-and-kidney-transplantation-a-case-series-and-review-of-the-literature
#19
Sabrina Milan Manani, Grazia Maria Virzì, Anna Giuliani, Anna Clementi, Alessandra Brocca, Daniela Dissegna, Francesca Martino, Emanuele Stefano Giovanni d'Amore, Claudio Ronco
BACKGROUND: Hemolytic uremic syndrome (HUS) can be triggered by Shiga toxin producing Escherichia coli (STEC) infection or it can be defined as atypical HUS (aHUS) if it is related to uncontrolled complement activation. aHUS is characterized by a high incidence of recurrence after kidney transplantation, and it can also occur de novo in transplant recipients. Eculizumab is used both to prevent and to treat aHUS following kidney transplantation. In this paper, we report our centre experience and we present 4 cases of HUS in patients who underwent kidney transplantation...
April 19, 2017: Nephron
https://www.readbyqxmd.com/read/28405075/acquired-thrombotic-thrombocytopenic-purpura-and-atypical-hemolytic-uremic-syndrome-successfully-treated-with-eculizumab
#20
Appalanaidu Sasapu, Michele Cottler-Fox, Pooja Motwani
Acquired idiopathic thrombotic thrombocytopenic purpura is a life-threatening disease with a mortality of up to 90%, if not promptly recognized and treated. We report a 64-year-old woman with this condition who presented with left-sided weakness and seizure-like activity preceded by headache and easy bruising. She did not achieve optimal response to plasma exchange, corticosteroids, rituximab, and vincristine. We initiated treatment with eculizumab, following which she had durable remission that continued for 30 months after discontinuation of the drug...
April 2017: Proceedings of the Baylor University Medical Center
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