Pegcetacoplan, sold under the brand name Empaveli among others, is a medication used to treat paroxysmal nocturnal hemoglobinuria (PNH).The most common side effects include injection-site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, viral infection, and fatigue.Paroxysmal nocturnal hemoglobinuria is characterized by red blood cell destruction, anemia (red blood cells unable to carry enough oxygen to tissues), blood clots, and impaired bone marrow function (not making enough blood cells).Pegcetacoplan is the first treatment for paroxysmal nocturnal hemoglobinuria that binds to and inhibits complement protein C3. Pegcetacoplan was approved for medical use in the United States in May 2021. Pegcetacoplan binds to complement protein C3 and its activation fragment C3b with high affinity, thereby regulating the cleavage of C3 and the generation of downstream effectors of complement activation. Medical uses
Pegcetacoplan is indicated to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Pegcetacoplan is only available for patients who are under the special REMS program. Two weeks before receiving pegcetacoplan, patients must receive vaccinations for pneumonia, meningitis, or influenza type B. Patients who have been vaccinated in the past may still need to get vaccinated two weeks before initiation of the medication. Pharmacology
Patients with PNH have greater and uninhibited complement activity, which may leads to intravascular (inside blood vessels) or extravascular (within the liver or spleen) hemolysis. Adverse effects
Meningococcal (a type of bacteria) infections can occur in people taking pegcetacoplan and can become life-threatening or fatal if not treated early. Pegcetacoplan may also predispose individuals to serious infections, especially infections caused by encapsulated bacteria.
Multi-species infections are very common.The most common intestinal parasites in the United States are Enterobius vermicularis, Giardia lamblia, Ancylostoma duodenale, Necator americanus, and Entamoeba histolytica. Variations within communities
Even in areas of high prevalence, the frequency and severity of infection is not uniform within communities or families. A small proportion of community members harbour the majority of worms, and this depends on age. The maximum worm burden is at five to ten years of age, declining rapidly thereafter. Individual predisposition to helminthiasis for people with the same sanitation infrastructure and hygiene behavior is thought to result from differing immunocompetence, nutritional status, and genetic factors. Because individuals are predisposed to a high or a low worm burden, the burden reacquired after successful treatment is proportional to that before treatment. Disability-adjusted life years
It is estimated that intestinal nematode infections cause 5 million disability-adjusted life years (DALYS) to be lost, of which hookworm infections account for more than 3 million DALYS and ascaris infections more than 1 million. There are also signs of progress: The Global Burden of Disease Study published in 2015 estimates a 46 percent (59 percent when age standardised) reduction in years lived with disability (YLD) for the 13-year time period from 1990 to 2013 for all intestinal/nematode infections, and even a 74 percent (80 percent when age standardised) reduction in YLD from ascariasis.