New subcutaneous injection offers convenient long-term control for rare blood disorder PNH

A new subcutaneous injection treatment is offering a significant breakthrough for patients suffering from Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare acquired blood stem cell disorder. This innovative approach provides a more convenient and stable long-term control option, helping individuals regain a better quality of life.

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At that time, I sought medical attention due to severe dizziness, frequent nosebleeds, and unexplained bruising. Blood tests revealed very low hemoglobin and platelet counts, and further examination confirmed the diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH).

PNH affects red blood cells, white blood cells, and platelets. While its name suggests "nocturnal hemoglobinuria," only about a quarter of patients experience this typical symptom. Most commonly, patients present with fatigue, abdominal pain, lower back pain, headaches, or fever, all related to hemolysis or anemia. The condition arises from an acquired PIG-A gene mutation, which leaves red blood cells lacking protective proteins (CD55, CD59) on their surface, making them vulnerable to attack by the immune system's complement.

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In the past, lacking specific medication, I could only rely on frequent blood transfusions to maintain my strength, especially before and after my period. I even needed transfusions twice a month, which was a significant burden on my life. If I needed dental treatment, I had to arrange for platelet transfusions first.

For patients like Ms. Chou, the journey has been arduous. Previously, she relied on frequent blood transfusions, sometimes twice a month, to manage severe fatigue caused by chronically low hemoglobin levels (5-6 g/dL). This dependency significantly impacted her daily life, limiting her ability to work and enjoy simple activities like family travel. Even dental procedures required prior platelet transfusions.

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The most important goal of treating Paroxysmal Nocturnal Hemoglobinuria is to stabilize hemolysis, improve anemia symptoms, and reduce the risk of serious complications. This allows patients to return to normal daily life functions, maintain their routines, and reduce the frequency of blood transfusions.

While earlier medications offered some relief, they were time-consuming, often requiring over four hours for administration, including waiting times. The new subcutaneous injection, administered monthly, has been a game-changer. It maintains stable hemoglobin levels (9–10 g/dL) and significantly improves energy levels. This less time-consuming treatment, often completed in about half an hour, drastically enhances accessibility and convenience, especially for those living far from medical centers or with demanding work schedules. Clinical observations show many patients on this new treatment no longer require blood transfusions.

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The new subcutaneous injection options, with their sustained and stable drug efficacy, significantly save time and improve quality of life.