Pozelimab, a human monoclonal immunoglobulin for the treatment of CHAPLE disease
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Manmeet Kaur
Abstract
The complement is a crucial factor of the innate immune system. However, its activation can lead to various diseases, so it needs to be controlled. In mammals, surface-bound complement regulatory proteins safeguard cells from uncontrolled complement-mediated lysis. One of the human complement regulators is CD55, also known as the decay-accelerating factor (DAF), a single-chain, type I cell surface protein anchored to glycosylphosphatidylinositol (GPI). The genetic loss of the complement regulatory protein CD55 leads to a fatal illness known as CHAPLE disease. The complement and innate immunity become hyperactive in this disease, causing angiopathic thrombosis and protein-losing enteropathy. Patients with CHAPLE disease experience abdominal pain, nausea, vomiting, diarrhea, loss of appetite, weight loss, impaired growth, and swelling. This genetic condition has no known cure, and managing its symptoms can be challenging. Pozelimab, a human monoclonal immunoglobulin IgG4 antibody, is a drug that targets the terminal complement protein C5. The drug has a high affinity for both wild-type and variant human C5. Pozelimab has received designations such as fast track, orphan drug, and rare pediatric disease, making it a significant medical breakthrough. It is currently the only available treatment for this disease. In this review, we have summarized the preclinical and clinical data on pozelimab.
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Research ethics: Not applicable; as it is review article, no patient consent and IEC approval was needed. Article content is totally ours (Authors).
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Informed consent: Not applicable; as it is review article, no patient consent OR informed consent form was required.
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Author contributions: The authors have accepted responsibility for the entire content of this manuscript and approved its submission. (1) Dr Manmeet Kaur – Idea, Writing the article, review. (2) Dr Saurav Misra – Idea, Concept, Writing the article, final editing, review, Submission.
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Competing interests: None of the authors have competing interest. The authors states no conflict of interest.
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Research funding: Not applicable, None.
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Data availability: Not applicable; since it is a review article, there is no patient data, and all the information I have used is duly cited and available online/public domain.
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Artikel in diesem Heft
- Frontmatter
- Editorials
- Doctor patient relationship in AI era: trying to decipher the problem
- “Adiponcosis interplay: adipose tissue, microenvironment and prostate cancer”
- Minireview
- Interplay between male gonadal function and overall male health
- Reviews
- How should we differentiate hypoglycaemia in non-diabetic patients?
- Pozelimab, a human monoclonal immunoglobulin for the treatment of CHAPLE disease
- Cannabis effectiveness on immunologic potency of pulmonary contagion
- Exploring the impact of vitamin D on tendon health: a comprehensive review
- The underlying causes, treatment options of gut microbiota and food habits in type 2 diabetes mellitus: a narrative review
- Original Articles
- Long-term functional outcomes and predictors of efficacy in thulium laser enucleation of the prostate (ThuLEP) for benign prostatic hyperplasia (BPH): a retrospective observational study
- Investigating Majhool date (Phoenix dactylifera) consumption effects on fasting blood glucose in animals and humans
- A novel variant in the FLNB gene associated with spondylocarpotarsal synostosis syndrome
- Exploring pathogenic pathways in carpal tunnel syndrome: sterile inflammation and oxidative stress
