ReviewExpert review of hematology2014
Complement in paroxysmal nocturnal hemoglobinuria: exploiting our current knowledge to improve the treatment landscape.
Review in Expert review of hematology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03316521 (Safety, Tolerability, Pharmacokinetics), which is not on this map. Cited by 27 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of a Single Ascending Dose (SAD) and a Multiple Dose (MD) of the Complement Inhibitor AMY-101. A Prospective, Single-center, Open-label, First-In-Human (FIH) Clinical Study in Healthy Male Volunteers
Who cites it
27 citing papers in PubMed, 45 citations in OpenAlex.
- Antibody-Dependent and Antibody-Independent Hemolysis in Sickle Cell Disease.Antibodies (Basel, Switzerland) · 2026Review
- The Silent Culprit: Paroxysmal Nocturnal Hemoglobinuria Masquerading as Cryptogenic Stroke.Cureus · 2025Article
- Complement-targeted therapeutics: Are we there yet, or just getting started?European journal of immunology · 2024Review
- A guide to complement biology, pathology and therapeutic opportunity.Nature reviews. Immunology · 2024Review
- Inflammation in Fabry disease: stages, molecular pathways, and therapeutic implications.Frontiers in cardiovascular medicine · 2024Review
- The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA.Journal of comparative effectiveness research · 2023Article
- Insight into mode-of-action and structural determinants of the compstatin family of clinical complement inhibitors.Nature communications · 2022Article
- Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria.Annals of hematology · 2022Article
- Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria.Therapeutic advances in hematology · 2022Review
- Article
- Halting targeted and collateral damage to red blood cells by the complement system.Seminars in immunopathology · 2021Review
- Clinical promise of next-generation complement therapeutics.Nature reviews. Drug discovery · 2019Review
- Expanding Complement Therapeutics for the Treatment of Paroxysmal Nocturnal Hemoglobinuria.Seminars in hematology · 2018Review
- The MFHR1 Fusion Protein Is a Novel Synthetic Multitarget Complement Inhibitor with Therapeutic Potential.Journal of the American Society of Nephrology : JASN · 2018Article
- Complement Component 3 Negatively Regulates Antibody Response by Modulation of Red Blood Cell Antigen.Frontiers in immunology · 2018Article
- Complement C3-Targeted Therapy: Replacing Long-Held Assertions with Evidence-Based Discovery.Trends in immunology · 2017Article
- Method development and validation for the quantitation of the complement inhibitor Cp40 in human and cynomolgus monkey plasma by UPLC-ESI-MS.Journal of chromatography. B, Analytical technologies in the biomedical and life sciences · 2017Article
- Buried Hydrogen Bond Interactions Contribute to the High Potency of Complement Factor D Inhibitors.ACS medicinal chemistry letters · 2016Article
- Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria.Hematology. American Society of Hematology. Education Program · 2016Review
- From orphan drugs to adopted therapies: Advancing C3-targeted intervention to the clinical stage.Immunobiology · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 2 countries.
Funding
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder associated with an acquired deficiency in glycophosphatidylinositol-anchor biosynthesis that renders erythrocytes susceptible to complement attack. Intravascular hemolysis via the membrane attack complex is a clinical hallmark of the disease, and C5 blockade is currently the only approved treatment for PNH. However, residual anemia is an emerging observation for many PNH patients receiving anti-C5 treatment. A range of complement-targeted therapeutic approaches, encompassing surface-directed inhibition of C3 convertases, blockade of membrane attack complex assembly or C3 interception using peptidic inhibitors, has yielded promising results and offers leverage for even more effective treatment of PNH. This article discusses recent advances in this rapidly evolving field, integrating critical perspectives from preclinical PNH models and diverse complement modulation strategies with genetic insights and therapy response profiles. It also evaluates the relative efficacy, limitations and benefits afforded by C3 or C5 inhibition in the context of PNH therapeutics.
Indexed as
Identifiers
What Socratic holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.