Paroxysmal nocturnal haemoHemolysisIntravascularCases of paroxysmal cold hemoglobiHematologyPegcetacoplanEMPAVELIPure red celHemolytic anemiaAplasticAHUSClonalThrombosis2017OccurClinicalAdult patientsNMOSDEfficacyAntibodyInhibitorTransfusion independenceRenal failureSymptomsThrombocytopeniaFebrileMyelodysplastic syndromesOutcomesRecurrentBloodSplenomegalyCommonly detectedTreatmentUrineDiagnosisExtravascularSolirisVaccinationPediatric patientsDiseaseCongenitalInhibitorsFindings
Paroxysmal nocturnal haemo7
- where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain (also known as APL-2) for the treatment of paroxysmal nocturnal haemoglobinuria. (europa.eu)
- What is paroxysmal nocturnal haemoglobinuria? (europa.eu)
- Paroxysmal nocturnal haemoglobinuria (PNH) is a condition in which there is excessive breakdown of red blood cells (haemolysis), leading to the release into the urine of a large amount of haemoglobin (the protein found in red blood cells that carries oxygen around the body). (europa.eu)
- Paroxysmal nocturnal haemoglobinuria phenotype cells and leucocyte subset telomere length in childhood acquired aplastic anaemia. (bvsalud.org)
- The significance of paroxysmal nocturnal haemoglobinuria (PNH(pos) ) cells and leucocyte subset telomere lengths in paediatric aplastic anaemia (AA) is unknown. (bvsalud.org)
- The European Commission has approved Alexion Pharmaceuticals' Ultomiris (ravulizumab) for the treatment of some adults with the ultra-rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH). (pharmatimes.com)
- Soliris is a medicine used to treat adults and children with paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS). (europa.eu)
Hemolysis18
- In the open-label Phase II study (NCT03439839), LNP023 demonstrated improvements in hematological response and biomarkers of disease activity in patients with active hemolysis despite treatment with eculizumab. (novartis.com)
- Patients taking LNP023 as an add-on to eculizumab saw significant reductions in their lactate dehydrogenase levels, a biomarker of intravascular hemolysis, and marked improvement in their hemoglobin (Hb) levels. (novartis.com)
- Importantly, all patients on LNP023 monotherapy retained their Hb levels with no changes in biomarkers of disease activity and no signs or symptoms of breakthrough hemolysis. (novartis.com)
- This condition was first described in 1854 as an abrupt onset of systemic manifestations, including severe anemia and hemoglobinuria, occurring upon exposure to cold temperatures and resulting from massive intravascular hemolysis. (medscape.com)
- But PNH is clinically heterogeneous, with some patients having a disease process characterized by florid intravascular, complement-mediated hemolysis , whereas in others, bone marrow failure dominates the clinical picture with modest or even no evidence of hemolysis observed. (aamds.org)
- In paroxysmal nocturnal hemoglobinuria (PNH), various symptoms due to intravascular hemolysis exert a negative impact on patients' quality of life (QOL). (elsevierpure.com)
- Paroxysmal nocturnal hemoglobinuria patients who have not received eculizumab treatment due to mild hemolysis may benefit from eculizumab treatment. (elsevierpure.com)
- In patients with paroxysmal nocturnal hemoglobinuria (PNH), the membrane-attack complex (MAC) formed on red blood cells (RBCs) causes hemolysis due to the patient's own activated complement system by an infection, inflammation, or surgical stress. (biomedcentral.com)
- BUSINESS WIRE )-- A prespecified interim analysis of the ALPHA Phase III trial evaluating danicopan (ALXN2040), an investigational, oral factor D inhibitor, as an add-on to C5 inhibitor therapy ULTOMIRIS ® (ravulizumab-cwvz) or SOLIRIS ® (eculizumab) showed positive high-level results in patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH). (biospace.com)
- So this is going to be chronic and present in almost every patient, although there are rare cases where they may present with say, thrombosis and less hemolysis. (reachmd.com)
- PNH is a very rare, life-threatening and debilitating disease characterized by complement-mediated hemolysis with or without hemoglobinuria. (empr.com)
- This series of containers holds urine of a patient with paroxysmal nocturnal hemoglobinuria, showing the episodic nature of the dark urine (hemoglobinuria) during intravascular hemolysis, usually occurring at night. (medscape.com)
- Overt hemolysis is often evident, and patients can have hemoglobinuria and acrocyanosis. (ispub.com)
- The patient presented with evidence of hemolysis. (ispub.com)
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder characterized by intravascular hemolysis and hemoglobinuria. (msdmanuals.com)
- Paroxysmal nocturnal hemoglobinuria is suspected in patients who have typical symptoms of anemia (eg, pallor, fatigue, dizziness, possible hypotension) or unexplained normocytic anemia with intravascular hemolysis, particularly if leukopenia or thrombocytopenia and/or thrombotic events are present. (msdmanuals.com)
- The diagnosis of loxoscelism was based upon the description of the spider by the patient himself and by the typical clinical findings which included necrosis at the site of the bite with a scarlet fever-like skin rash, intravascular hemolysis, hemoglobinuria, coagulation disturbances and acute renal failure. (scielo.br)
- Extravascular hemolysis and complement consumption in Paroxysmal Nocturnal Hemoglobinuria patients undergoing eculizumab treatment. (cdc.gov)
Intravascular1
- Reports of widespread thromboses and disseminated intravascular coagulation (DIC) in patients with coronavirus disease 19 (COVID-19) have been rapidly increasing in number. (nature.com)
Cases of paroxysmal cold hemoglobi2
- Surgery is not indicated in cases of paroxysmal cold hemoglobinuria, other than to aid in the diagnosis of underlying infections or neoplasms. (medscape.com)
- Although most cases of paroxysmal cold hemoglobinuria occur as an acute event in children younger than 5 years, recurrent episodes have been reported. (medscape.com)
Hematology6
- Patients with paroxysmal nocturnal hemoglobinuria (PNH) undergoing treatment with eculizumab or ravulizumab have a high illness burden and relatively low quality of life (QoL), suggesting a need for improved treatments, according to research published in Annals of Hematology . (hematologyadvisor.com)
- Professor Jong-Wook Lee, MD, PhD, Department of Hematology at Seoul St. Mary's Hospital of The Catholic University of Korea, and investigator in the ALPHA trial, said: "C5 inhibitors are a proven treatment option for patients living with PNH, yet a small percentage may continue to experience anemia and burden of transfusion due to clinically significant EVH, however it is not life-threatening. (biospace.com)
- He is in charge of the reference center for Aplastic Anemia and Nocturnal Paroxysmal Hemoglobinuria (PNH) and the French network on Hematology-immunology rare diseases (MaRIH). (eurobloodnet.eu)
- The majority of patients with PNH currently receiving treatment with eculizumab have continuing anemia," said Peter Hillmen, MB, ChB, PhD, Professor of Experimental Hematology at the University of Leeds and an investigator in the PEGASUS study. (empr.com)
- The Benign Hematology Program gives patients and their families a variety of educational options tailored to individual learning styles. (froedtert.com)
- Apellis will be presenting long-term data reinforcing the robust efficacy and safety profile of EMPAVELI in patients with PNH at the 2022 American Society of Hematology (ASH) Annual Meeting being held December 10-13, 2022, in New Orleans, Louisiana. (biospace.com)
Pegcetacoplan11
- Empaveli (pegcetacoplan) is a complement inhibitor used to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH). (rxlist.com)
- Apellis Pharmaceuticals announced positive topline results from the phase 3 PEGASUS trial of pegcetacoplan (APL-2) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). (empr.com)
- In PEGASUS, the efficacy and safety of pegcetacoplan was evaluated in 80 adult patients with PNH. (empr.com)
- Patients were randomized to receive either pegcetacoplan 1080mg subcutaneously twice weekly (n=41) or eculizumab (n=39) for 16 weeks. (empr.com)
- 0001). Patients in the pegcetacoplan and eculizumab treatment arms had an adjusted mean hemoglobin change of 2.4g/dL and -1.5g/dL, respectively, from a baseline of 8.7g/dL at week 16. (empr.com)
- The PEGASUS results show that pegcetacoplan has the potential to become a new standard of care for patients with PNH. (empr.com)
- Patients with paroxysmal nocturnal hemoglobinuria can now self-administer pegcetacoplan subcutaneously following the FDA's approval of an injector. (cancernetwork.com)
- Empaveli (pegcetacoplan) (MW, 44 kDa) is used to treat paroxysmal nocturnal hemoglobinuria . (medscape.com)
- Apellis continues to enroll patients in the Phase 3 VALIANT study of systemic pegcetacoplan for IC-MPGN and C3G. (biospace.com)
- In October 2022, Sobi announced the dosing of the first patient in the Phase 3 CASCADE study of systemic pegcetacoplan for CAD. (biospace.com)
- Sobi continues to enroll patients in its Phase 2 study evaluating the efficacy and safety of systemic pegcetacoplan in patients with HSCT-TMA. (biospace.com)
EMPAVELI4
- Safety and effectiveness of Empaveli in pediatric patients have not been established. (rxlist.com)
- Female patients of reproductive potential are advised to use effective contraception during treatment with Empaveli and for 40 days after the last dose. (rxlist.com)
- Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. (rxlist.com)
- Vaccinate patients against encapsulated bacteria as recommended at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection. (rxlist.com)
Pure red cel1
- Frequent STAT3 mutations in CD8 T cells from patients with pure red cell aplasia. (cdc.gov)
Hemolytic anemia8
- Paroxysmal cold hemoglobinuria (PCH) is a form of autoimmune hemolytic anemia (AIHA) that, while rare, is nevertheless one of the most common causes of acute AIHA in young children. (medscape.com)
- Paroxysmal cold hemoglobinuria (PCH) has the distinction of being the first, albeit rarest, type of autoimmune hemolytic anemia (AIHA) to be identified. (medscape.com)
- [ 12 ] Furthermore, because the D-L antibody does not necessarily occur with a specific cold exposure event, nor is it recurrent in nature, renaming paroxysmal cold hemoglobinuria as Donath-Landsteiner hemolytic anemia has been proposed. (medscape.com)
- The antibody thermal activity range of paroxysmal cold hemoglobinuria is simlar to that of cold hemagglutinin disease (CHD), the more common cold variant of autoimmune hemolytic anemia. (medscape.com)
- One study in patients with hemolytic anemia found that increasing the dose from 2mg/kg to 4mg/kg to 8mg/kg led to increased drug effect from 2 days, to 7-14 days, to 11-21 days. (clevelandclinic.org)
- Conditions in this category include warm autoimmune hemolytic anemia, cold agglutinin syndrome , mixed- or combined-type autoimmune hemolytic anemia, and paroxysmal cold hemoglobinuria . (medscape.com)
- In a patient with mixed-type autoimmune hemolytic anemia, the direct antiglobulin test commonly detects both IgG and C3. (medscape.com)
- Paroxysmal cold hemoglobinuria shares similar antibody thermal activity range as cold hemagglutinin disease (CHD), the more common cold variant of autoimmune hemolytic anemia. (medscape.com)
Aplastic6
- In the overall cohort, the mean age was 46.8 years, the mean age at diagnosis was 37.5 years, 73% of patients were female sex, and 33.6% of patients had aplastic anemia or severe aplastic anemia. (hematologyadvisor.com)
- So you're looking at this patient, and you say they might have aplastic anemia, so you're a little nervous about that. (reachmd.com)
- Aplastic anemia is suspected in patients, particularly young patients, with pancytopenia. (merckmanuals.com)
- Will aplastic anaemia patients be able to have the coronavirus vaccine? (theaat.org.uk)
- We analyzed the clinical course and outcome in 50 patients (27 males, 23 females) suffering from aplastic anemia (AA), treated in our department between 1987 and 2007. (springermedizin.at)
- Effectiveness of immunosuppressive therapy in older patients with aplastic anemia. (springermedizin.at)
AHUS5
- Eculizumab had previously been FDA approved for treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor antibody positive generalized myasthenia gravis (gMG). (clevelandclinic.org)
- Ultomiris is also indicated for the treatment of adults and pediatric patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. (pharmaceutical-technology.com)
- It develops medicines to treat conditions such as atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), hypophosphatasia (HPP), lysosomal acid lipase de?ciency (LAL-D), neurofibromatosis type 1 plexiform neurofibromas (NF1 PN), neuromyelitis optica spectrum disorder (NMOSD) and paroxysmal nocturnal hemoglobinuria (PNH). (pharmaceutical-technology.com)
- In patients with PNH, aHUS, refractory gMG and NMOSD, the complement proteins are over-active and damage the patients' own cells. (europa.eu)
- Eculizumab is most commonly prescribed for treatment of 2 rare blood disorders: atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH). (cdc.gov)
Clonal5
- Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal hematopoietic stem cell (HSC) disease. (medscape.com)
- Clonal Hematopoiesis of Indeterminate Potential (CHIP) in Patients with Coronary Artery Disease and in Centenarians. (hematology.org)
- Paroxysmal nocturnal hemoglobinuria is a clonal disorder caused by an acquired mutation in the PIGA gene in hematopoietic stem cells. (msdmanuals.com)
- Paroxysmal nocturnal hemoglobinuria or PNH is a rare benign clonal acquired hematopoietic stem-cell (HSC) disorder that results from somatic mutation of the X- linked phosphatidylinositol glycan class A gene known as the PIGA gene. (aacc.org)
- Abnormal expression and mutation of the RBPJ gene may be involved in CD59 clonal proliferation in paroxysmal nocturnal hemoglobinuria. (cdc.gov)
Thrombosis4
- Aside from anemia and thrombosis, PNH is also linked with fatigue, dyspnea, abdominal pain, chest pain , hemoglobinuria, renal insufficiency, and bone marrow failure, each of which presents individual risks for patient health and QoL. (hematologyadvisor.com)
- Thrombosis is the leading cause of death and affects 40% of PNH patients. (aacc.org)
- The preliminary research in paroxysmal nocturnal hemoglobinuria with thrombosis]. (cdc.gov)
- Polymorphism of the ABO gene associate with thrombosis risk in patients with paroxysmal nocturnal hemoglobinuria. (cdc.gov)
20171
- To learn more about the increased risk of meningococcal disease in patients receiving eculizumab and prevention strategies, read the July 7, 2017 MMWR . (cdc.gov)
Occur2
- Currently, episodes of paroxysmal cold hemoglobinuria usually occur after a viral infection and are abrupt in onset and transitory. (medscape.com)
- This may occur at different times of the day and vary from patient to patient. (medscape.com)
Clinical12
- For this cross-sectional survey-based study, researchers evaluated the clinical and economic burden of PNH among patients receiving C5 inhibition therapy. (hematologyadvisor.com)
- To determine clinical factors related with improvements in QOL in PNH patients treated, we analyzed changes in QOL scales in PNH patients treated with eculizumab based on data collected from post-marketing surveillance in Japan. (elsevierpure.com)
- Here, we extended these observations with results from an open-label, non-placebo-controlled, 52-week, phase 3 clinical safety and efficacy study evaluating eculizumab in a broader PNH patient population. (ashpublications.org)
- One limitation of the study centered around the inability to validate diagnostics of cachexia because of the absence of clinical data in several of the patients from the 12 central tumor types from the study. (cancernetwork.com)
- In the remaining 7 patients, ethanol embolization was decided as the primary treatment method after full review of findings from clinical and imaging examinations of the patients. (ajnr.org)
- The decision allows use of the drug - the first and only long-acting C5 complement inhibitor administered every eight weeks - in patients with haemolysis with clinical symptoms indicative of high disease activity, and also for those who are clinically stable after having been treated with Soliris (eculizumab) for at least the past six months. (pharmatimes.com)
- Steroids are commonly employed, but these agents have not been shown to shorten the clinical course of paroxysmal cold hemoglobinuria. (medscape.com)
- No obvious correlations between clinical parameters, including age, karyotype, existence of paroxysmal nocturnal hemoglobinuria clones, pretreatment blood counts, progenitor cell counts, and the response to immunosuppressive therapy (IST), were found. (springermedizin.at)
- Comparative study on baseline clinical characteristics of Asian versus non-Asian patients with paroxysmal nocturnal hemoglobinuria. (cdc.gov)
- With the exception of 1 patient and potentially hazardous procedure, clinical effi cacy in some falciparum who had become infected while in was initially considered for 2 of our malaraia-endemic areas preclude its Myanmar, all patients acquired falci- patients but was deemed unnecessary usefulness ( 4 ). (cdc.gov)
- Some immunosuppressive therapies or conditions may mask or modulate the clinical manifestations (e.g., the patient may be afebrile). (cdc.gov)
- Methodology: We conducted a descriptive cross-sectional study of in- and out-patients from whose clinical samples' bacteria were isolated at the bacteriology unit of the laboratory. (bvsalud.org)
Adult patients3
- The approval was based on findings from a single-institution phase 2 trial (NCT02649972) conducted by Memorial Sloan Kettering Cancer Center (MSKCC) and Genentech in a population of adult patients with histiocytic disorders. (cancernetwork.com)
- Adbry (tralokinumab) (molecular weight [MW], 147 kilodaltons), is indicated for the treatment of moderate to severe atopic dermatitis in adult patients whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. (medscape.com)
- Ultomiris is indicated for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy (IVIg) or plasmapheresis. (pharmaceutical-technology.com)
NMOSD2
- While the PREVENT trial did not pharmacodynamics of eculizumab in APQ4 antibody positive NMOSD patients, pharmacodynamics of eculizumab has been studied in other disease states. (clevelandclinic.org)
- Ultromiris is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD), paroxysmal nocturnal hemoglobinuria (PNH), in adults and pediatric patients one month of age and older. (pharmaceutical-technology.com)
Efficacy2
- The efficacy of transfusion therapy for patients with PNH has been documented, but no studies have focused on the perioperative use of salvaged autologous blood in patients with PNH. (biomedcentral.com)
- Now PNH patients can experience greater freedom in their lives with significantly fewer infusions per year without any compromise on efficacy or safety. (pharmatimes.com)
Antibody3
- Eculizumab was first studied in 14 AQP4 antibody positive neuromyelitis optica patients in an open-label trial. (clevelandclinic.org)
- Headache, nausea, and dizziness were the most common side effects reported in the open label trial of eculizumab in APQ4 antibody positive neuromyelitis optica patients. (clevelandclinic.org)
- It is used in patients who have an antibody called AQP4 and whose disease is relapsing (where the patient has attacks [relapses] between periods with no symptoms). (europa.eu)
Inhibitor4
- Despite disease occurring among vaccinated individuals, clinicians should continue vaccinating patients who receive a complement inhibitor. (cdc.gov)
- The terminal complement inhibitor eculizumab was recently shown to be effective and well tolerated in patients with paroxysmal nocturnal hemoglobinuria (PNH). (ashpublications.org)
- Oral MEK1/2 inhibitor cobimetinib (Cotellic) has received approval by the FDA for the treatment of patients with histiocytic neoplasms including Erdheim-Chester disease, Rosai-Dorfman disease, and Langerhans cell histiocytosis. (cancernetwork.com)
- Data from a pivotal Phase III study showed that Ultomiris was non-inferior to Soliris (eculizumab) in complement inhibitor treatment-naïve patients with PNH, based on the co-primary endpoints of transfusion avoidance and normalisation of lactate dehydrogenase (LDH) levels. (pharmatimes.com)
Transfusion independence1
- Forty-nine patients (51%) achieved transfusion independence for the entire 52-week period. (ashpublications.org)
Renal failure2
Symptoms8
- Clinicians should have a high index of suspicion for meningococcal disease in patients receiving complement inhibitors with symptoms of meningitis or meningococcemia. (cdc.gov)
- C5 inhibition is a mainstay in this patient population, and many patients receive either eculizumab or ravulizumab, which may help to alleviate disease symptoms. (hematologyadvisor.com)
- When you look at patients with PNH in general, it takes a long time for these patients to receive the diagnosis, and they suffer from symptoms for a long time before that. (reachmd.com)
- 40 percent of the patients usually receive their diagnosis within 12 months, and the rest of them, it might take them around five years to get the diagnosis, and they usually see around five, approximately five physicians during that journey because of symptoms that they have to be told that they have PNH. (reachmd.com)
- The National Blood Clot Alliance provides patient education and professional training on the signs, symptoms and prevention of blood clots and clotting disorders. (stoptheclot.org)
- Other methods such as blood transfusions and treatment with blood-thinning compounds to prevent clotting were used in some patients to improve symptoms. (europa.eu)
- There is a close temporal relationship observed between viral or bacterial agents and the development of paroxysmal cold hemoglobinuria within 2-3 weeks of upper respiratory or gastrointestinal symptoms. (medscape.com)
- Should maintain a high index of suspicion for meningococcal disease in patients taking eculizumab who present with any symptoms consistent with either meningitis or meningococcemia, even if the patient's symptoms initially appear mild, and irrespective of the patient's meningococcal vaccine or antimicrobial prophylaxis status. (cdc.gov)
Thrombocytopenia2
- Indicated for thrombocytopenia associated with chronic idiopathic thrombocytopenic purpura in patients experiencing inadequate response to corticosteroids, immunoglobulins, or splenectomy. (medscape.com)
- During the meeting several analyses will be presented in patients with haemophilia A, paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenia (ITP), relapsed or refractory diffuse large b-cell lymphoma, myelofibrosis, and haemophagocytic lymphohistiocytosis. (centralillinoisproud.com)
Febrile1
- World Health Organization (WHO) as a in Omdurman are workers in the govern- febrile patient with falciparum malaria with ment and informal sectors. (who.int)
Myelodysplastic syndromes1
- Randomized Open-Label Phase II Study of Decitabine in Patients With Low- or Intermediate-Risk Myelodysplastic Syndromes. (aamds.org)
Outcomes3
- The purpose of our study was to present our initial experience of ethanol embolization in a series of 17 patients with auricular AVMs and assess the interim therapeutic outcomes of this method. (ajnr.org)
- The European Transplant Allied Healthcare Professionals (ETAHP) reaches out to allied healthcare professionals throughout Europe in order to ensure the best care possible for all transplant patients, with the aim to optimize patient outcomes. (esot.org)
- Our aim is to improve outcomes for the patients. (esot.org)
Recurrent1
- Taylor CJ, Neilson JR, Chandra D, Ibrahim Z. Recurrent paroxysmal cold haemoglobinuria in a 3-year-old child: a case report. (medscape.com)
Blood19
- Basel, August 29, 2020 - Novartis today announced new Phase II data for LNP023, an investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH), presented at the virtually held 2020 European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting. (novartis.com)
- Once suspected, the diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) is straightforward when flow cytometric analysis of the peripheral blood reveals a population of glycosyl phosphatidylinositol anchor protein-deficient cells. (aamds.org)
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood characterized by destruction of red blood cells by the complement system, a part of the body's innate immune system. (wikipedia.org)
- Washed RBCs lacking white blood cells and complement components were used in the past, but the practice of washing the RBCs was deemed unnecessary and packed RBCs are usable without any problem for patients with PNH [ 2 ]. (biomedcentral.com)
- So there's a substantial proportion of these patients who can present with a weird blood clot. (reachmd.com)
- Dr. Scadden discusses which blood cancer patients are eligible for stem cell transplantation, as well as the latest advances in the field. (cancernetwork.com)
- Depending on the technique and the reagents used, a positive direct antiglobulin test has been reported in 1:1000 to 1:14,000 blood donors and 1%-15% of hospital patients. (medscape.com)
- LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care. (lls.org)
- Patients may also experience problems related to blood clotting. (europa.eu)
- Bone marrow transplantation to replace the defective red blood cells was another therapy available to patients. (europa.eu)
- In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body's immune system, causes the destruction of red blood cells, which can lead to progressive anaemia, fatigue, dark urine, and shortness of breath. (pharmatimes.com)
- In PNH patients these two complement regulatory proteins are absent or partially expressed on red blood cells. (aacc.org)
- The medicine can only be obtained with a prescription and must be given under the supervision of a doctor who has experience in the management of patients with kidney disorders and disorders affecting the nervous system or the blood. (europa.eu)
- In some patients who receive plasma exchange (the removal, treatment and return of their own blood plasma, the liquid part of the blood) or infusion of plasma, additional doses of Soliris are required. (europa.eu)
- In patients with PNH, the breakdown of red blood cells leads to a reduction in haemoglobin levels. (europa.eu)
- Treatment with Soliris over 26 weeks led to stable haemoglobin levels in 49% of the patients (21 out of 43), without the need for transfusions of red blood cells. (europa.eu)
- Six out of seven patients did not need any transfusion of red blood cells, and haemoglobin levels improved during 12 weeks of treatment with Soliris. (europa.eu)
- A registry study of patients with PNH who had never had a blood transfusion looked at the blood levels of the enzyme lactate dehydrogenase (LDH). (europa.eu)
- It is deficient in red blood cells from patients with paroxysmal nocturnal hemoglobinuria (PNH). (lu.se)
Splenomegaly2
- A minority of patients have splenomegaly (2). (ispub.com)
- gross hemoglobinuria and splenomegaly are common. (msdmanuals.com)
Commonly detected1
- Severe hemoglobinuria is commonly detected during the acute event, resulting in a red-brown discoloration to the urine. (medscape.com)
Treatment22
- All patients required RBC transfusions prior to starting LNP023 treatment. (novartis.com)
- Although all patients receiving eculizumab, and the majority (95.4%) of patients receiving ravulizumab, had been on treatment for at least 3 months, more than 80% of patients in both groups had levels of hemoglobin consistent with anemia. (hematologyadvisor.com)
- ravulizumab, 22.6%) were also reported among patients receiving treatment for at least 1 year. (hematologyadvisor.com)
- Patients with PNH had below average scores on both the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30). (hematologyadvisor.com)
- Overall, our results suggest that patients with PNH receiving therapy with [eculizumab] or [ravulizumab] maintain a substantial burden of illness, indicating that there is a need for novel therapeutics in PNH treatment," the authors wrote. (hematologyadvisor.com)
- These data show that danicopan has the potential to resolve clinically significant EVH while allowing patients to remain on standard of care treatment with ULTOMIRIS or SOLIRIS. (biospace.com)
- Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder. (rarediseases.org)
- So these are all the things that we see on the objective side in these patients, both the diagnosis and then throughout their treatment course. (reachmd.com)
- Estimates suggest about 80% of advanced-stage cancer patients will develop cachexia, a "multifactorial syndrome characterized by muscle wasting, leading to a significant weight loss that impacts patient's quality of life, tolerance to treatment, response to therapy, and survival. (cancernetwork.com)
- Patients with histiocytic neoplasms can now receive treatment with cobimetinib following its approval by the FDA. (cancernetwork.com)
- There has always been an unmet need for patients with histiocytosis, and we are thrilled that with this approval, these patients will now have access to a viable treatment option. (cancernetwork.com)
- Nelson J. Chao, MD, MBA, and his colleagues discussed the use of ruxolitinib as treatment for patients with graft-vs-host disease. (cancernetwork.com)
- Bylvay (odevixibat) (MW unknown) is indicated for the treatment of pruritus in patients aged 3 months and older. (medscape.com)
- The study group consisted of 17 patients (11 male, 6 female) with a mean age of 25.4 years (age range, 3-47 years) at the time of treatment. (ajnr.org)
- ECTTA is the forum for experience exchange on treatment of patients with end-stage heart and lung failure. (esot.org)
- The mainstay of treatment for paroxysmal cold hemoglobinuria is supportive care and the avoidance of cold exposure. (medscape.com)
- A hematologist-oncologist and an infectious diseases expert may be helpful for the proper diagnosis and treatment of paroxysmal cold hemoglobinuria. (medscape.com)
- Treatment should also be stopped in patients with refractory gMG who do not respond to Soliris after 12 weeks. (europa.eu)
- Eight patients (16 %) did not respond to treatment, and one died shortly after transplantation. (springermedizin.at)
- Together, both IST and stem cell transplantation are reasonable treatment options for patients with AA. (springermedizin.at)
- because of the rapid improvement af- a water-soluble artemisinin derivate Four patients were Norwegian tour- ter artesunate treatment. (cdc.gov)
- ia Prevention in UK Travelers advocate None of the patients needed exchange pdf) and may be obtained from IDIS artesunate as the fi rst-line treatment for transfusions. (cdc.gov)
Urine1
- Hemoglobinuria test is a urine test that checks for hemoglobin in the urine. (medlineplus.gov)
Diagnosis1
- The other thing that we see is a lot of times once you establish a diagnosis, you'll find that these patients will have flares. (reachmd.com)
Extravascular1
- However, unlike cold hemagglutinin disease, in which the IgM-complement interaction results in the cells' removal (via extravascular phagocytosis), paroxysmal cold hemoglobinuria occurs upon completion of complement lysis within the vascular circulation. (medscape.com)
Soliris5
- McNamara LA, Topaz N, Wang X, Hariri S, Fox L, MacNeil J. High risk for invasive meningococcal disease among patients receiving eculizumab (Soliris) despite receipt of meningococcal vaccine . (cdc.gov)
- Danicopan plus ULTOMIRIS or SOLIRIS demonstrated superiority compared to placebo plus ULTOMIRIS or SOLIRIS for this specific patient population, with statistically significant and clinically meaningful improvements in hemoglobin levels, transfusion avoidance and FACIT Fatigue scores from baseline. (biospace.com)
- Soliris is given as an infusion (drip) into a vein and the recommended dose depends on what it is used for, and for patients under 18 years of age, on their bodyweight. (europa.eu)
- Soliris should be given for life unless the patient develops serious side effects. (europa.eu)
- In a study in 7 children with PNH who were treated with at least one transfusion in the previous two years, all patients received Soliris. (europa.eu)
Vaccination3
- Recent data show that some patients receiving eculizumab who were up to date with meningococcal vaccination still developed meningococcal disease. (cdc.gov)
- The Food and Drug Administration (FDA)-approved prescribing information for eculizumab includes a black box warning for increased risk of meningococcal disease, and the Advisory Committee on Immunization Practices (ACIP) recommends meningococcal vaccination for all patients receiving eculizumab. (cdc.gov)
- Should continue meningococcal vaccination of all patients who receive eculizumab. (cdc.gov)
Pediatric patients1
- In one study, pediatric patients 6 to 58 months old were given rifampin suspended in simple syrup or as dry powder mixed with applesauce at a dose of 10 mg/kg body weight. (nih.gov)
Disease14
- The goal of this activity is for learners to be better able to discuss the burden of suboptimal disease control and how to involve patients in the therapeutic decision-making process. (medscape.org)
- in addition, the extent to which the mutant stem cell clone expands in an individual patient determines the magnitude of the hemolytic component of the disease. (aamds.org)
- Approach to the patient with renal disease. (medlineplus.gov)
- We are proud of our continued innovation to advance new ways of targeting the complement cascade to help address the needs of patients living with this debilitating disease. (biospace.com)
- adults and children 1 month of age and older with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). (biospace.com)
- We feature top medical specialists, member advocacy organizations, rare disease patients and Centers of Excellence. (rarediseases.org)
- I remember a young patient I had during the first period of time when I was training who had a mesenteric vein clot and had to actually get a small bowel resection and then transplant because of this disease. (reachmd.com)
- One picture is coming into better focus, however, which suggests that an immune-triggered, complement-mediated thrombotic microangiopathy (TMA) is surprisingly common in patients with coronavirus disease 19 (COVID-19). (nature.com)
- Cold agglutinin disease has also been detected in patients with polycythemia vera. (ispub.com)
- The basis for cold agglutinin disease in the patient was not established. (ispub.com)
- The LLS mission: Cure leukemia, lymphoma, Hodgkin disease and myeloma, and improve the quality of life of patients and their families. (lls.org)
- Chronic kidney disease is a major cause of death occurs in 67% of patients due to toxicity of free hemoglobin and iron with extensive hemoglobin deposition. (aacc.org)
- Patients with the chronic form of the disease must wear proper clothes and garments to protect the extremities from becoming chilled. (medscape.com)
- Recent data show that some patients receiving eculizumab who were vaccinated with the recommended meningococcal vaccines still developed meningococcal disease, most often from nongroupable Neisseria meningitidis , which rarely causes invasive disease in healthy individuals. (cdc.gov)
Congenital2
- In the latter half of the 19th century, the most common cause of paroxysmal cold hemoglobinuria was congenital or adult tertiary-stage syphilis. (medscape.com)
- Prothrombotic mechanisms in patients with congenital p.Cys89Tyr mutation in CD59. (cdc.gov)
Inhibitors1
- The warning includes a recommendation to administer meningococcal vaccines to patients receiving complement inhibitors. (cdc.gov)