This calls for the development of effective tools (i.e., symptom questionnaires) to measure the impact of PNH on quality of life, the perceived benefits of treatment, and patient preferences. Improving quality of life is an emerging objective in the current management of PNH. Preliminary results appear very promising, and these agents may revolutionize the field, significantly improving the quality of life of patients with PNH.Īs mentioned earlier, quality of life is affected both by the symptoms of the disease itself as well as by treatment-related issues, such as the need for frequent healthcare visits. These include agents that will have a longer half-life, allowing for more time between treatments agents that can be delivered subcutaneously or taken by mouth and agents that work by targeting a different part of the complement cascade. Thankfully, there are several novel therapies for PNH that are being studied. Therefore, there was a need for newer medications that could improve the quality of life for patients with PNH. In addition, some patients still have some symptoms on this medication. This can interfere with working and leisure activities, and can represent a substantial burden for patients. The medication is administered by infusion, which means that patients have to go to an infusion center every 2 weeks to receive it. While the C5 inhibitor improved the quality of life of patients with PNH, there were still a few drawbacks associated with therapy. Treatment with this C5 inhibitor reduced the need for transfusions, reduced the occurrence of blood clots, and improved anemia and fatigue. In 2007, the first biologic agent was approved for PNH: a C5 inhibitor. Traditionally, patients with PNH were treated with blood transfusions, to replace the destroyed red blood cells. Increased risk of complications, including breakthrough hemolysis and blood clots, are also a major concern. The chronic nature of the disease, with different treatment requirements, also contributes negatively to quality of life. ![]() PNH, a rare condition, is characterized by chronic destruction of red blood cells (hemolysis), resulting in anemia and fatigue, which can significantly affect patients’ quality of life. ![]() Brodsky, MD, Director, Division of Hematology, Johns Hopkins Kimmel Cancer Center, Baltimore Some of these drugs are oral, but they have short half-lives, so missed doses due to other medical issues or noncompliance could be dangerous…Ultimately, we’ll likely need randomized trials comparing these new agents head to head with long-acting C5 inhibitors, and not just patients with a suboptimal response to C5 inhibition." - Robert A. ![]()
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