A hemoglobinúria paroxística noturna (HPN) é uma doença clonal adquirida de células-tronco hematopoiéticas caracterizada por anemia hemolítica corpuscular, insuficiência da medula óssea e eventos trombóticos frequentes.
Introdução
O que você precisa saber de cara
A hemoglobinúria paroxística noturna (HPN) é uma doença clonal adquirida de células-tronco hematopoiéticas caracterizada por anemia hemolítica corpuscular, insuficiência da medula óssea e eventos trombóticos frequentes.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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Entender a doença
Do básico ao detalhe, leia no seu ritmo
Preparando trilha educativa...
Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 25 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 49 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
Triagem neonatal (Teste do Pezinho)
A triagem neonatal permite diagnóstico precoce e início imediato do tratamento.
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
2 genes identificados com associação a esta condição. Padrão de herança: Not applicable.
Component of the glycosylphosphatidylinositol-anchor (GPI-anchor) transamidase (GPI-T) complex that catalyzes the formation of the linkage between a proprotein and a GPI-anchor and participates in GPI anchored protein biosynthesis (PubMed:11483512, PubMed:12582175, PubMed:28327575, PubMed:34576938, PubMed:35165458, PubMed:35551457, PubMed:36970549, PubMed:37684232). May play a crucial role in GPI-T complex assembly in the luminal layer (PubMed:35165458, PubMed:35551457). Binds GPI-anchor (PubMed
Endoplasmic reticulum membrane
Multiple congenital anomalies-hypotonia-seizures syndrome 3
An autosomal recessive syndrome characterized by distinct facial features, intellectual disability, hypotonia and seizures, in combination with abnormal skeletal, endocrine, and ophthalmologic findings including impaired vision, as well as abnormal motility of the eyes.
Catalytic subunit of the glycosylphosphatidylinositol-N-acetylglucosaminyltransferase (GPI-GnT) complex that catalyzes the transfer of N-acetylglucosamine from UDP-N-acetylglucosamine to phosphatidylinositol and participates in the first step of GPI biosynthesis
Rough endoplasmic reticulum membrane
Paroxysmal nocturnal hemoglobinuria 1
A disorder characterized by hemolytic anemia with hemoglobinuria, thromboses in large vessels, and a deficiency in hematopoiesis. Red blood cell breakdown with release of hemoglobin into the urine is manifested most prominently by dark-colored urine in the morning.
Medicamentos aprovados (FDA)
3 medicamentos encontrados nos registros da FDA americana.
Variantes genéticas (ClinVar)
375 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 44 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
2 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Hemoglobinúria paroxística noturna
Selecione um estado ou use sua localização para ver resultados.
Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.
Pesquisa ativa
Ensaios clínicos abertos e novidades científicas recentes
Ensaios em destaque
🟢 Recrutando agora
16 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.
Outros ensaios clínicos
182 ensaios clínicos encontrados, 32 ativos.
Publicações mais relevantes
Mostrando amostra de 200 publicações de um total de 1.216
Optimizing Care in Patients with Paroxysmal Nocturnal Hemoglobinuria: Managing Suboptimal Response and Uncontrolled Disease.
The treatment of patients with PNH has been revolutionized by terminal complement C5 inhibitors, which control intravascular hemolysis and thrombosis, reduce morbidity and mortality, and improve life expectancy to that approaching people without PNH. In recent years, approval of proximal inhibitors provides clinicians and patients with additional treatment options such that patients who have residual anemia, ongoing symptoms affecting quality of life, or are intolerant to terminal C5 inhibition now have options to optimize treatment. Here, we provide five questions to guide clinicians involved in the care of patients with PNH in assessing treatment response on terminal inhibitors and identifying patients who might benefit from therapy adjustments. We also provide insights into additional treatment options.
Ravulizumab for Treatment of Paroxysmal Nocturnal Hemoglobinuria During Pregnancy.
Ravulizumab, a humanized, long-acting monoclonal antibody against complement factor C5, is a widely utilized treatment for paroxysmal nocturnal hemoglobinuria (PNH). As pregnant women with PNH are at increased risk for complications and the safety and efficacy of ravulizumab in pregnancy is not known, we performed an international multicenter retrospective analysis of 16 PNH patients with 19 pregnancies managed with ravulizumab and compared outcomes to 8 earlier pregnancies in the same patients treated with eculizumab. Of the eight eculizumab pregnancies, three resulted in miscarriages and one in early preterm delivery for threatened fetal demise and massive fetal growth retardation. All 19 pregnancies on ravulizumab resulted in birth of live infants with a median gestational age of 267 days (interquartile range (IQR) 259-275) and median birth weight of 3115grams (IQR 2458-3349 grams). Cord blood testing in two pregnancies receiving intensified ravulizumab dosing revealed detectable ravulizumab levels consistent with transplacental transfer. After a median follow-up of 16.2 months (IQR 4.4-40.1), no developmental abnormalities or severe infectious complications were observed in the children. This retrospective analysis provides evidence for the safety and effectiveness of ravulizumab in managing PNH during pregnancy and breastfeeding with favorable maternal and fetal outcomes.
Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, complement-mediated hematologic disease. We present the results of a phase 2, open-label clinical trial of vemircopan monotherapy in patients with PNH to explore the efficacy and safety of inhibiting the complement alternative pathway via factor D. Adults (aged ≥18 years) with PNH who were treatment-naïve and switched from eculizumab with hemoglobin (Hgb) of <10 g/dL or rolled over from danicopan monotherapy (ACH471-103 trial; ClinicalTrials.gov identifier: NCT03181633) were enrolled. The trial comprised a 60-day screening period, 12-week treatment period, and 148-week long-term extension. Participants received vemircopan 120 mg twice daily, with potential escalation to 180 mg twice daily. The primary endpoint was change in Hgb from baseline to week 12. Safety endpoints included treatment-emergent adverse events (TEAEs) and serious AEs. Twenty-nine participants were enrolled (treatment-naïve, n = 12; eculizumab-switch, n = 11; and danicopan-rollover, n = 6); all completed the 12-week treatment period, and 1 danicopan-rollover participant completed the long-term extension. Clinically meaningful improvements from baseline to week 12 in Hgb (change of ≥2 g/dL) were reported in the treatment-naïve (mean, 3.6 [standard deviation [SD], 1.5]) and eculizumab-switch (mean, 3.3 [SD, 2.0]) cohorts and maintained through the end of the trial. Eighty-two breakthrough intravascular hemolysis (BT-IVH) events in 25 participants were reported. Twenty-eight participants reported TEAEs, and 14 reported serious AEs; most were unrelated to vemircopan. No deaths occurred. This trial evaluating vemircopan monotherapy met its primary efficacy endpoint; however, concerns regarding BT-IVH risk and suboptimal, inconsistent control of IVH emerged, leading to early trial termination. This trial was registered at www.clinicaltrials.gov as #NCT04170023, and EudraCT as #2019-003830-17.
Telomere content and genomics of myeloid neoplasia by whole-genome sequencing.
Telomere length shortening has been associated with genomic instability and acquisition of molecular lesions, but these processes have not been systematically studied across large cohorts of myeloid neoplasia (MN). As proof of concept for a novel, cross-validated whole-genome sequencing-based method of telomere content (TC) determination combined with mutations, transcriptomics, and functional assays, we studied TC in correlation with specific molecular features of a large cohort (N = 1804) of patients with MN, including acute myeloid leukemia (AML) and myelodysplastic syndrome. When compared with healthy participants and patients with nonclonal diseases such as persistent polyclonal B-cell lymphocytosis, both MN and nonmalignant controls with clonal disease, such as paroxysmal nocturnal hemoglobinuria and aplastic anemia, exhibited decreased TC. Furthermore, we show that TC is lowered in adult MN abrogating correlation with age with considerable TC diversification among certain morphologic and molecular subtypes. For instance, AML harbored the lowest TC. Furthermore, MN originating from a more mature cell of origin (eg, acute promyelocytic leukemia) or characterized by hyperproliferative driver mutations (eg, RAS pathway genes) had lower TC, possibly indicating a loss of telomere maintenance capacity. In contrast, compared with other mutations, MN subtypes arising in a context of profound genetic alterations, such as TP53 mutations and complex karyotype, exhibited a relatively higher/preserved TC. This phenomenon did not involve alternative lengthening processes but was rather consistent with an increased TC due to preserved activity of the telomerase complex. Our results describe a common and genotype-specific telomeric makeup of a large cohort of patients with MN providing a molecular benchmark for future therapeutic targeting of the telomere machinery.
First report of perioperative iptacopan interruption in paroxysmal nocturnal hemoglobinuria without breakthrough hemolysis.
Publicações recentes
Epidemiology and care management of Paroxysmal Nocturnal Hemoglobinuria (PNH) in a real-world setting in France: Description from the French National Hospitalization Database.
Complement blockade: Therapeutic promises and remaining challenges in clinical practice.
🥇 Meta-análiseAcute Pancreatitis as a Therapeutic Complication in Case of Paroxysmal Nocturnal Hemoglobinuria.
Unprovoked Venous Thromboembolism in a Patient with a Small PNH Clone: A Therapeutic Dilemma.
Whole blood thrombin generation before and after eculizumab in a patient with hemolytic crisis due to paroxysmal nocturnal hemoglobinuria.
📚 EuropePMC2.205 artigos no totalmostrando 197
Case report: persistent anemia after eculizumab in paroxysmal nocturnal hemoglobinuria: non-dominantly active intravascular hemolysis.
Hematology (Amsterdam, Netherlands)Hemoglobinuria-associated acute kidney injury in hemolytic uremic syndrome without renal thrombotic microangiopathy.
BMC nephrologyIndirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment.
Journal of comparative effectiveness researchDecrease in PNH-type Blood Cells after the Cessation of Thrombopoietin Receptor Agonist in a Case of AA-PNH Syndrome.
Internal medicine (Tokyo, Japan)[Efficacy and safety of romiplostim N01 for refractory aplastic anemia: a retrospective single-center study].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhiThrombosis in paroxysmal nocturnal hemoglobinuria in the complement inhibitor era: mechanisms, risk stratification, and clinical management.
International journal of hematologyRenal hemosiderosis in paroxysmal nocturnal hemoglobinuria.
Kidney internationalAnalysis of adverse drug reactions associated with ravulizumab: a retrospective pharmacovigilance study utilizing the FAERS database.
Frontiers in immunologyAllogeneic Hematopoietic Cell Transplantation Using Post-Transplant Cyclophosphamide in Patients With Large Paroxysmal Nocturnal Hemoglobinuria Clones.
European journal of haematologyA Prospective 1-Year Study of Renal Recovery in Pigment Nephropathy: Insights Beyond the Acute Phase.
The Journal of the Association of Physicians of IndiaParoxysmal Nocturnal Hemoglobinuria as a Rare Cause of Chronic Asymptomatic Hemolysis: A Case Report.
CureusThe Challenges Associated with the Diagnosis and Treatment for Aplastic Anemia Concurrent with Lymphoplasmacytic Lymphoma/Waldenström's Macroglobulinemia.
Internal medicine (Tokyo, Japan)Optimizing Care in Patients with Paroxysmal Nocturnal Hemoglobinuria: Managing Suboptimal Response and Uncontrolled Disease.
Journal of blood medicineReal-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry.
American journal of hematologyHematologic disorders in pregnancy: the role of the complement system.
Expert review of hematologyZilucoplan is effective for ravulizumab-refractory generalized myasthenia gravis with the C5 p.Arg885His variant: a case report.
Neuromuscular disorders : NMDPopulation PK-PD Modeling of Danicopan Add-On Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria Treated With Ravulizumab or Eculizumab.
CPT: pharmacometrics & systems pharmacologyClinical Profile, Humanistic and Economic Burden of Paroxysmal Nocturnal Hemoglobinuria in Patients Treated With C5 Inhibitors.
EJHaemCOEXISTENCE OF APLASTIC ANEMIA AND PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: DIAGNOSTIC CHALLENGES AND THERAPEUTIC STRATEGIES - CASE REPORT.
Georgian medical newsDisease burden and treatment patterns of paroxysmal nocturnal hemoglobinuria in Japan: a real-world survey.
International journal of hematology[How I diagnose and treat paroxysmal nocturnal hemoglobinuria].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhiParoxysmal Nocturnal Hemoglobinuria in a Young Adult Woman: A Representative Case of Recurrent Intravascular Hemolysis.
CureusSwitching patients with PNH from pegcetacoplan to iptacopan: a case series.
Hematology (Amsterdam, Netherlands)Clonal expansion mechanisms in paroxysmal nocturnal hemoglobinuria.
International journal of hematologyComplement Biomarkers for Monitoring Last-Generation Complement Inhibitors in Paroxysmal Nocturnal Hemoglobinuria Patients.
American journal of hematologyRavulizumab for Treatment of Paroxysmal Nocturnal Hemoglobinuria During Pregnancy.
Blood advancesImprovement in anemia and symptoms after switching from crovalimab to iptacopan in paroxysmal nocturnal hemoglobinuria.
Hematology (Amsterdam, Netherlands)Integrative miRNA-mRNA Network and Molecular Dynamics-Based Identification of Therapeutic Candidates for Paroxysmal Nocturnal Hemoglobinuria.
Pharmaceuticals (Basel, Switzerland)Psychometric performance of the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue questionnaire among adults with paroxysmal nocturnal hemoglobinuria.
Journal of patient-reported outcomesTailoring Eculizumab Treatment: Evaluation of Model-Informed Precision Dosing for Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome.
Therapeutic drug monitoringReal life use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria: evidence from the REACTION observational study.
Annals of hematologyAcute kidney injury as a harbinger of paroxysmal nocturnal hemoglobinuria in an unsuspected case.
Indian journal of pathology & microbiology[Advancing treatment goals for paroxysmal nocturnal hemoglobinuria to align with quality of life improvements in the era of anti-complement therapy].
[Rinsho ketsueki] The Japanese journal of clinical hematologyCost per responder analysis of iptacopan versus eculizumab and ravulizumab in treatment of paroxysmal nocturnal hemoglobinuria: implications for decision-making.
Journal of medical economicsTherapeutic Antibodies in Hematology: Advances in Malignant and Non-Malignant Disorders.
CellsDiscovery of (3R,4R)-15: An Advanced Factor B Inhibitor Entering Phase 3 for Complement-Mediated Diseases.
Journal of medicinal chemistry[Targeting complement in atypical hemolytic and uremic syndrome: development and current status of eculizumab and ravulizumab].
Nephrologie & therapeutiqueLyme disease as a rare trigger for autoimmune hemolytic anemia.
BMC infectious diseasesLaboratory findings in patients treated with complement factor C3 inhibitor pegcetacoplan.
Clinical chemistry and laboratory medicineDisease-modifying treatments in paroxysmal nocturnal hemoglobinuria: a systematic review of economic evaluations.
Cost effectiveness and resource allocation : C/EParoxysmal Nocturnal Hemoglobinuria-Driven Hepatic Vein Thrombosis: A Case of Budd-Chiari Syndrome in Disguise.
CureusParoxysmal nocturnal hemoglobinuria presenting with acute kidney injury in an 18-year-old male: a case report.
Korean journal of family medicineWhole genome sequencing confirms reactivation of Tuberculosis five years after initial infection despite adequate preventative therapy in a patient with profound immunosuppression.
QJM : monthly journal of the Association of Physicians[Current Perspectives on Paroxysmal Nocturnal Hemoglobinuria (PNH)].
Revista medica de ChilePathophysiology of Primary Budd-Chiari Syndrome: A Narrative Review.
CureusSuccessful Birth After First-Trimester Ravulizumab Exposure in a Patient With Paroxysmal Nocturnal Hemoglobinuria: A Case Report.
EJHaemFirst report of perioperative iptacopan interruption in paroxysmal nocturnal hemoglobinuria without breakthrough hemolysis.
Hematology, transfusion and cell therapyC3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria.
Frontiers in immunology[The night has many faces: When schistocytes step out of line].
PraxisFree GPIs and Comparison of GPI Structures Among Species.
International journal of molecular sciencesA rare case of littoral cell angioma with paroxysmal nocturnal hemoglobinuria.
MedicineThrombin generation in PNH patients treated sequentially with Eculizumab and Ravulizumab: a paired analysis.
Journal of thrombosis and thrombolysisThe varieties of therapeutic experience: navigating treatment options for patients with PNH.
Hematology. American Society of Hematology. Education ProgramRotational Thromboelastometry (ROTEM)-Assisted Anaesthetic Management of a Parturient With Paroxysmal Nocturnal Haemoglobinuria and Aplastic Anaemia for Caesarean Section: A Case Report.
CureusComplement factor D is a drug target for metabolic-associated fatty liver disease.
Molecular immunologyMyeloproliferative neoplasms with clinically relevant paroxysmal nocturnal hemoglobinuria: clinical correlations and outcomes.
HaematologicaEfficacy and Safety of the C5 Inhibitor Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Meta-Analysis.
European journal of haematologyPredicting secondary myeloid neoplasms in acquired aplastic anemia using machine learning models.
Blood neoplasiaDiffuse Large B-Cell Lymphoma Combined With Paroxysmal Nocturnal Hemoglobinuria: A Rare Case Report.
Clinical case reportsParoxysmal Nocturnal Hemoglobinuria in Pregnancy Treated With Pegcetacoplan: Case Report and Pharmacokinetic Analysis.
EJHaemAdvances in Complement Inhibition Therapies for Paroxysmal Nocturnal Hemoglobinuria and Autoimmune Hemolytic Disorders.
Journal of blood medicineA case of allogeneic peripheral blood stem cell in a paroxysmal nocturnal hemoglobinuria patient from a COVID-positive donor: Managing the unpreventable.
Asian journal of transfusion scienceCommon features of rare disease patients in the emergency department: a systematised literature review.
Orphanet journal of rare diseasesHepatic Sinusoidal Obstruction Syndrome Secondary to Aplastic Anemia/Paroxysmal Nocturnal Hemoglobinuria Syndrome: A Rare Case.
Diagnostics (Basel, Switzerland)Mapping Health State Utility from Disease-Specific Measures in Spinal Muscular Atrophy and Paroxysmal Nocturnal Hemoglobinuria.
PharmacoEconomicsLong-term outcomes of αβ T-cell/CD19 B-cell-depleted peripheral blood stem cell transplantation from unrelated donors in pediatric and adolescent patients with severe aplastic anemia: a single-center study.
CytotherapyParoxysmal Nocturnal Hemoglobinuria.
Mayo Clinic proceedingsComplement-targeting therapies in hemolytic diseases.
Current opinion in immunologyConsensus of the Hematology Society of Taiwan on the management of paroxysmal nocturnal hemoglobinuria (PNH).
Journal of the Formosan Medical Association = Taiwan yi zhiImproved Iron Overload with Pegcetacoplan in Eculizumab-Experienced Patients with Paroxysmal Nocturnal Hemoglobinuria.
International journal of molecular sciences[Significance of the 50% hemolytic complement in hemolysis assessment and efficacy of eculizumab in patients with paroxysmal nocturnal hemoglobinuria].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhiIptacopan in C5 blockade refractory atypical hemolytic uremic syndrome with associated Castleman's disease: case report.
BMC nephrologyParoxysmal Nocturnal Hemoglobinuria in Patients with Systemic Lupus Erythematosus: Diagnostic Challenges and Improvement with C5 Inhibitor Treatment.
Internal medicine (Tokyo, Japan)Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.
Blood advancesCase report: Spontaneous remission of severe aplastic anemia mediated by mutant hematopoietic stem cells evading T-cell attack.
Frontiers in immunologyIdentification of plasma protein biomarkers for ankylosing spondylitis by integrating proteome with genome.
Clinical rheumatologyProgress in the use of biological therapies to treat paroxysmal nocturnal hemoglobinuria: focus on patient profiling.
Expert opinion on biological therapyRavulizumab exposure in early pregnancy.
Annals of hematologyRepair of Congenital Cardiac Disease Requiring Bypass for a Pediatric Patient With Paroxysmal Nocturnal Hemoglobinuria.
JACC. Case reportsCorrelation of clone size with clinical and lab presentation in Paroxysmal nocturnal hemoglobinuria (PNH): A single center experience.
Pakistan journal of medical sciencesParoxysmal nocturnal hemoglobinuria revealed by atypical thromboses: A case of cerebral, hepatic, and portal vein involvement leading to cirrhosis.
Radiology case reportsFirst reported case of Moraxella lacunata bacteremia following pharyngitis in an adult with paroxysmal nocturnal hemoglobinemia receiving ravulizumab: A case report.
IDCases[Challenges in optimizing targeted complement inhibitor therapy for paroxysmal nocturnal hemoglobinuria in an expanding treatment landscape].
[Rinsho ketsueki] The Japanese journal of clinical hematologyParoxysmal Nocturnal Hemoglobinuria: Unraveling Its Molecular Pathogenesis and Advancing Targeted Therapeutic Strategies.
Diseases (Basel, Switzerland)Biosynthesis of GPI anchored proteins, its deficiencies and treatment.
Journal of human geneticsStructure and function of therapeutic antibodies approved by the US FDA in 2024.
Antibody therapeuticsTelomere content and genomics of myeloid neoplasia by whole-genome sequencing.
BloodManaging transient immune complex reactions in patients with paroxysmal nocturnal hemoglobinuria: clinical observations from the COMMODORE 1 and 2 studies.
Therapeutic advances in hematologySuccessful switch from pegcetacoplan to iptacopan after repeated severe breakthrough hemolysis events - case report.
Hematology (Amsterdam, Netherlands)[Diagnosis of non-autoimmune hemolysis in the adult].
La Revue de medecine interneParoxysmal Nocturnal Hemoglobinuria Associated Cutaneous Thrombosis: A Case Report.
Indian dermatology online journalParoxysmal Nocturnal Hemoglobinuria with Large Clones in Non-Hypoplastic Myelodysplastic Syndrome: Report of Two Cases.
Acta haematologicaEnigmatic Roles of Complement Anaphylatoxin Signaling in Health and Disease.
Immune network[New treatment strategies for paroxysmal nocturnal hemoglobinuria: drug selection in the era of novel complement inhibitors].
[Rinsho ketsueki] The Japanese journal of clinical hematologyClinical characteristics and management of paroxysmal nocturnal hemoglobinuria in the Middle East: a narrative review.
Clinical and experimental medicineViral infections and related fatal adverse events associated with complement inhibitors for PNH: a real-world pharmacovigilance analysis in FAERS.
Frontiers in pharmacologyPNH clones prevalence study in ph-negative myeloproliferative neoplasms: a multicenter Italian study.
Annals of hematologyCase Report: Iptacopan in a paroxysmal nocturnal hemoglobinuria patient with severe renal insufficiency.
Frontiers in medicineReal-World Prevalence and Outcomes of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors in the US: A Retrospective Claims Database Analysis.
Journal of health economics and outcomes researchSuccessful Control of Chemotherapy-Induced Breakthrough Hemolysis With Ravulizumab in a Patient With Paroxysmal Nocturnal Hemoglobinuria During Carboplatin-Pemetrexed Treatment for Lung Adenocarcinoma.
CureusParoxysmal nocturnal hemoglobinuria masquerading as hemolytic uremic syndrome: a Case Report.
Frontiers in medicineDanicopan: complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria.
Trends in pharmacological sciencesCharacterization of Breakthrough Hemolysis in Patients With Paroxysmal Nocturnal Hemoglobinuria: An International Multicenter Experience.
American journal of hematologyDisseminated gonococcal infection developing two days after initial eculizumab administration in a patient with neuromyelitis optica spectrum disorder: A case report and literature review.
Journal of infection and chemotherapy : official journal of the Japan Society of ChemotherapyThe Onerous task of managing paroxysmal nocturnal hemoglobinuria in a Low resource setting: a case report. A hematologist's experience.
African health sciencesTargeting the Roots of Kidney Disease: Systematic Review of the Therapies Targeting the Complement System.
Medicina (Kaunas, Lithuania)Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan.
Advances in therapySafety, Efficacy, and Patient-Reported Outcomes From a Phase 2 Randomized Trial of Pozelimab and Cemdisiran Combination in Patients With Paroxysmal Nocturnal Hemoglobinuria.
EJHaemPatient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan.
The patientA decade of acquired aplastic anemia: insights from a Central South African Center.
The Pan African medical journalTreatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey.
Annals of hematologyCrovalimab: A Novel Approach in the Management of Paroxysmal Nocturnal Hemoglobinuria.
Health science reportsThe incidence of paroxysmal nocturnal hemoglobinuria cell clones in the Nordic countries.
Annals of hematologyLong-term effectiveness and safety of ravulizumab in paroxysmal nocturnal hemoglobinuria: a plain language summary.
Journal of comparative effectiveness researchHematopoietic stem cell transplantation is effective in achieving long-term survival for post-aplastic anemia myeloid neoplasms: the EBMT Severe Aplastic Anemia Report.
Haematologica[Pharmacological characteristics and clinical study results of danicopan (Voydeya® tablets)].
Nihon yakurigaku zasshi. Folia pharmacologica JaponicaNavigating the paroxysmal nocturnal hemoglobinuria (PNH) landscape.
Clinical advances in hematology & oncology : H&OWhole blood thrombin generation hypercoagulable profile in a patient with hemolytic crisis due to paroxysmal nocturnal hemoglobinuria: a case report.
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosisThe Silent Culprit: Paroxysmal Nocturnal Hemoglobinuria Masquerading as Cryptogenic Stroke.
CureusFirst-in-Class Clinically Investigated Oral Factor D Inhibitors for the Treatment of Complement-Mediated Diseases.
Pharmaceutical researchModel-Informed Precision Dosing of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria.
Clinical pharmacokineticsThe Silent Masquerader: Paroxysmal Nocturnal Hemoglobinuria Revealed.
The Journal of the Association of Physicians of India[Recent advances in the treatment of paroxystic nocturnal hemoglobinuria].
La Revue du praticienRed Blood Cells are Critical for Hemostasis and Thrombosis.
Seminars in thrombosis and hemostasisTwo Cases of Crovalimab-Induced Platelet Recovery in Bone Marrow Failure-Associated Paroxysmal Nocturnal Hemoglobinuria (PNH).
CureusPatient-reported outcomes in patients with paroxysmal nocturnal hemoglobinuria treated with crovalimab and approved C5 inhibitors in the phase III COMMODORE 2 and 1 studies.
Annals of hematologyRBPJ/PAF1 Signaling Axis Promotes Cloning Expansion in Paroxysmal Nocturnal Hemoglobinuria Through NOTCH Signaling.
International journal of laboratory hematologyDirect oral anticoagulants as secondary prophylaxis of venous thromboembolism in paroxysmal nocturnal hemoglobinuria: an Italian monocentric experience.
Journal of thrombosis and thrombolysis[A case of paroxysmal nocturnal hemoglobinuria treated with eculizumab in unintended pregnancy].
Zhonghua nei ke za zhiEfficient production of functional proaerolysin in E. coli.
Protein expression and purification[Correlation between complement deposition levels on blood cells and clinical biomarkers in patients with paroxysmal nocturnal hemoglobinuria].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhiEffectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor-Naive Patients With Paroxysmal Nocturnal Haemoglobinuria.
EJHaemReal-World Evidence of the Disease Burden and Economic Impact of Paroxysmal Nocturnal Hemoglobinuria in Italy.
Journal of clinical medicineDesign, Synthesis, and Biological Evaluation of a Novel Long-Acting Human Complement C3 Inhibitor Synthesized via the PASylation-Lipidation Modular (PLM) Platform.
Bioconjugate chemistry[Limitations and challenges of glucocorticoids in the treatment of paroxysmal nocturnal hemoglobinuria].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhiAdvancements in complement inhibition for PNH and primary complement-mediated thrombotic microangiopathy.
Blood advances[Clinical and Laboratory Characteristics of Cold Agglutinin Disease Patients with Positive Results of Acidified-Serum Lysis Test].
Zhongguo shi yan xue ye xue za zhiComparison of monoclonal antibody to CD59 for the diagnosis of paroxysmal nocturnal hemoglobinuria by flow cytometry.
Cytometry. Part B, Clinical cytometryComplement dysregulation at lymphatics.
The Journal of allergy and clinical immunologyWhat are the most important quality of life domains for patients with aplastic anemia and paroxysmal nocturnal hemoglobinuria?
Annals of hematologyCurrent view on the etiopathogenesis of aplastic anemia.
The Korean journal of physiology & pharmacology : official journal of the Korean Physiological Society and the Korean Society of PharmacologyAnchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria.
European journal of haematologyDose Adjustments of Pegcetacoplan in a Patient With Paroxysmal Nocturnal Hemoglobinuria Undergoing Surgery: A Case Report.
CureusCharacterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment.
Blood advancesThe Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment.
Turkish journal of haematology : official journal of Turkish Society of HaematologyFlow Cytometric Bone Marrow Evaluation in Suspected Myelodysplastic Neoplasms.
Current protocolsRed blood cells undergo lytic programmed cell death involving NLRP3.
Cell[Efficacy of iptacopan monotherapy for suboptimal response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria].
Terapevticheskii arkhivBreakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice.
BloodThe efficacy of immunosuppressive therapy with or without thrombopoietin receptor agonist in elderly patients with severe aplastic anemia.
Annals of hematologyWarren Alpert Medical School of Brown University: Clinicopathologic Conference: September 20th, 2024. A Woman in her 20s with Abdominal Pain, Anemia and Thrombocytopenia.
Journal of Brown hospital medicine[Unrelated bone marrow transplantation for acute myeloid leukemia evolved from paroxysmal nocturnal hemoglobinuria].
[Rinsho ketsueki] The Japanese journal of clinical hematologyShort-term recurrent coronary artery thrombosis with acute myocardial infarction in a patient with aplastic anemia-paroxysmal nocturnal hemoglobinuria syndrome: a case report.
Frontiers in cardiovascular medicinePrevention and Management of Thromboembolism in Patients with Paroxysmal Nocturnal Hemoglobinuria in Asia: A Narrative Review.
International journal of molecular sciencesEPYSQLI (SB12; Biosimilar to Reference Eculizumab) in Asian and Non-Asian Patients With Paroxysmal Nocturnal Hemoglobinuria: Subgroup Analysis of a Global Phase III Randomized Controlled Trial.
EJHaemCrovalimab: a new era in paroxysmal nocturnal hemoglobinuria management.
Annals of medicine and surgery (2012)FDA approval of crovalimab: a milestone in paroxysmal nocturnal hemoglobinuria treatment.
Annals of medicine and surgery (2012)Initial management of patients with acquired aplastic anemia in the United States: results from a large national claims database.
Annals of hematologyPathogenesis of paroxysmal nocturnal hemoglobinuria.
BloodSomatic mutations in Brazilian patients with paroxysmal nocturnal hemoglobinuria: a comprehensive analysis.
Frontiers in medicine[Advances in complement inhibition therapy for paroxysmal nocturnal hemoglobinuria].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhiA Rare Cause of Recurrent Abdominal Pain.
GastroenterologyPegcetacoplan for Breakthrough Hemolysis in an Adolescent With Classical Paroxysmal Nocturnal Hemoglobinuria on Eculizumab.
Pediatric blood & cancerFactor B as a therapeutic target for the treatment of complement-mediated diseases.
Frontiers in immunologyMeningococcal Sepsis in Patient with Paroxysmal Nocturnal Hemoglobinuria during Pegcetacoplan Therapy.
Emerging infectious diseasesRavulizumab and other complement inhibitors for the treatment of autoimmune disorders.
Multiple sclerosis and related disordersThe Path to Accessible Care: Development and Impact of Eculizumab Biosimilars for Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome.
BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapyDevelopment and application of UHPLC-MS/MS method to quantify eculizumab in PNH patients.
Journal of pharmaceutical and biomedical analysisDiscovery of a second, distinct development pattern of leukemic conversion from paroxysmal nocturnal hemoglobinuria.
International journal of hematologyThrombosis at Unusual Sites: Focus on Myeloproliferative Neoplasms and Paroxysmal Nocturnal Hemoglobinuria.
HamostaseologieDirect Oral Anticoagulants and Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Update on Evidence.
CureusModulating the complement system through epitope-specific inhibition by complement C3 inhibitors.
The Journal of biological chemistryUnraveling the Mysteries: Paroxysmal Nocturnal Hemoglobinuria and Its Unexpected Link to Stroke in Young Adult.
The Journal of the Association of Physicians of IndiaEculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety - a single-center study.
Hematology (Amsterdam, Netherlands)Adverse drug events (ADEs) risk signal mining related to eculizumab based on the FARES database.
Frontiers in pharmacologyRavulizumab demonstrates long-term efficacy, safety and favorable patient survival in patients with paroxysmal nocturnal hemoglobinuria.
Annals of hematologyCurrent status and perspectives of hematopoietic cell transplantation in patients with paroxysmal nocturnal hemoglobinuria.
Frontiers in immunologyMore Than a Haematoma: A Case of Aplastic Anemia.
CureusPatient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies.
Blood advances[Interpretation of the guidelines for diagnosing and treating paroxysmal nocturnal hemoglobinuria in China (2024)].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi[Clinical observation of allogeneic hematopoietic stem cell transplantation for treating five cases of classic paroxysmal nocturnal hemoglobinuria].
Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi[Paroxysmal Nocturnal Hemoglobinuria: A Rare but Treatable Cause of Acute Kidney Injury. Case Report].
Revista medica de ChileProximal complement inhibitors in paroxysmal nocturnal hemoglobinuria: an abundance of options in a rare disease.
Expert review of hematologyCurrent landscape of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitors and regulators.
Therapeutic advances in hematologyLong-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH.
BloodConcurrent Loss of CD16 in Granulocytes and CD14 in Mature Monocytes in a Patient With Pancytopenia: A Diagnostic Clue for Paroxysmal Nocturnal Hemoglobinuria.
International journal of laboratory hematologyExpert consensus on the management of pharmacodynamic breakthrough-hemolysis in treated paroxysmal nocturnal hemoglobinuria.
Hematology (Amsterdam, Netherlands)Pegcetacoplan: the first and only C3-targeted therapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria.
Expert review of hematologyThe Importance of Controlling Terminal Complement Activity and Intravascular Hemolysis in Paroxysmal Nocturnal Hemoglobinuria (PNH).
American journal of hematologyAdvancements in PNH treatment: crovalimab's clinical efficacy.
Annals of medicine and surgery (2012)An Unusual Case of Relapsing and Remitting Acute Kidney Injury.
Indian journal of nephrologyCrovalimab in the paroxysmal nocturnal hemoglobinuria treatment landscape.
ImmunotherapyPopulation Pharmacokinetic and Pharmacokinetic/Pharmacodynamic Analyses of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria.
Drugs in R&DTerminal complement inhibition and control of hemolysis in patients with paroxysmal nocturnal hemoglobinuria who switched from high-dose eculizumab to ravulizumab: a phase IV, single-arm clinical trial.
HaematologicaMonitoring and Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients with Aplastic Anemia in Asia: An Expert Consensus.
International journal of molecular sciencesThrombosis in Paroxysmal Nocturnal Hemoglobinuria (PNH): From Pathogenesis to Treatment.
International journal of molecular sciencesFluorescent Aerolysin (FLAER) Binding Is Abnormally Low in the Clonal Precursors of Acute Leukemias, with Binding Particularly Low or Absent in Acute Promyelocytic Leukemia.
International journal of molecular sciencesAssociações
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Referências e fontes
Bases de dados externas citadas neste artigo
Publicações científicas
Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.
- Optimizing Care in Patients with Paroxysmal Nocturnal Hemoglobinuria: Managing Suboptimal Response and Uncontrolled Disease.
- Ravulizumab for Treatment of Paroxysmal Nocturnal Hemoglobinuria During Pregnancy.
- Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.
- Telomere content and genomics of myeloid neoplasia by whole-genome sequencing.
- First report of perioperative iptacopan interruption in paroxysmal nocturnal hemoglobinuria without breakthrough hemolysis.
- Epidemiology and care management of Paroxysmal Nocturnal Hemoglobinuria (PNH) in a real-world setting in France: Description from the French National Hospitalization Database.
- Complement blockade: Therapeutic promises and remaining challenges in clinical practice.
- Acute Pancreatitis as a Therapeutic Complication in Case of Paroxysmal Nocturnal Hemoglobinuria.
- Unprovoked Venous Thromboembolism in a Patient with a Small PNH Clone: A Therapeutic Dilemma.
- Whole blood thrombin generation before and after eculizumab in a patient with hemolytic crisis due to paroxysmal nocturnal hemoglobinuria.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:447(Orphanet)
- MONDO:0100244(MONDO)
- Hemoglobinuria Paroxistica Noturna(PCDT · Ministério da Saúde)
- GARD:7337(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Artigo Wikipedia(Wikipedia)
- Q1479494(Wikidata)
Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar
