Pulmozyme治疗囊性纤维化效果如何？

Author: Medicalhalo

Release time: 2025-10-19 11:44:20

Effective in treating cystic fibrosis. Cystic fibrosis is an autosomal recessive multisystem progressive disease that causes mucus in various organs to become thicker. The disease primarily affects a patient's upper and lower respiratory tract, causing long-term lung infection and limiting the ability to breathe over time.

Pulmozyme is a mucus-thinning inhaled medication used to manage and treat cystic fibrosis. It has been shown to reduce respiratory infections in selected patients with forced vital capacity (FVC) greater than 40% of predicted value.

cystic fibrosis

Cystic fibrosis is a genetic disease defined by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Loss or reduced function of the CFTR protein is associated with multiple organ dysfunction and shortened life expectancy. About 85.5% of people in the United States with cystic fibrosis have the gene variant F508del. Manifestations of cystic fibrosis in patients with the F508del gene variant begin in infancy and manifest as steatorrhea, poor weight gain, and respiratory symptoms (cough, wheezing).

As people with cystic fibrosis age, chronic respiratory bacterial infections can lead to loss of lung function and bronchiectasis. As newborn screening becomes more widespread in several countries, including the United States, many people with cystic fibrosis are asymptomatic at the time of diagnosis.

Pulmozyme to treat cystic fibrosis

Pulmozyme is currently used as a mucolytic agent to treat the lung disease cystic fibrosis (a major cause of morbidity and mortality). It reduces the viscosity of mucus in the lungs and promotes the removal of secretions.

Study results show that Pulmozyme can improve lung function and reduce the exacerbation of respiratory symptoms in patients with cystic fibrosis. In a randomized, double-blind, placebo-controlled trial involving 968 patients with cystic fibrosis, once-daily and twice-daily administration of Pulmozyme reduced the age-adjusted risk of respiratory exacerbation by 28% (P=0.04) and 37% (P<0.01), respectively. In another six-month randomized controlled trial, patients treated with Pulmozyme experienced fewer lung exacerbations. Meanwhile, a 1-year randomized controlled trial showed that Pulmozyme improved lung function in patients with cystic fibrosis compared with placebo.

In addition, 15 trials compared Pulmozyme with placebo or no Pulmozyme (2447 participants), two compared daily Pulmozyme with hypertonic saline (32 participants), and one study compared daily Pulmozyme with hypertonic saline and alternate-day Pulmozyme (48 participants). One study compared Pulmozyme with mannitol and a combination of the two drugs (38 participants). Pulmozyme compared with placebo or no treatment at one month, three months, six months and two years. In trials of up to two years, Pulmozyme may have reduced the number of lung exacerbations (moderate-quality evidence).

Another trial (43 children) showed that Pulmozyme may lead to greater improvements in FEV1 compared with hypertonic saline (low-quality evidence), and one trial (23 participants) reported little or no difference in lung function between Pulmozyme and mannitol or Pulmozyme and Pulmozyme plus mannitol (low-quality evidence). One trial (23 participants) found that Pulmozyme improved quality of life compared with Pulmozyme plus mannitol (low-quality evidence).

Pulmozyme

Adverse reactions of Pulmozyme include voice changes, pharyngitis, rash, laryngitis, chest pain, conjunctivitis, rhinitis, etc. Some of these symptoms may affect the patient's treatment compliance and quality of life.

Adverse reaction measures

1. Voice changes: For voice changes, you can try to relieve them by resting or reducing the dose. For pharyngitis, patients should drink more water to keep their throat moist, relieve throat pain and discomfort, and avoid ingesting irritating foods and drinks, such as chili peppers, alcohol, etc. If symptoms persist or worsen, you should stop using Pulmozyme immediately and consult a doctor for diagnosis and treatment.

2. Skin rash: During medication, pay attention to keeping the skin clean, hygienic, and dry, and avoid scratching with your hands to avoid secondary infection. Some anti-allergic drugs, such as chlorpheniramine, loratadine, etc., can also be used under the guidance of a doctor. If the rash is severe, systemic drugs, such as hormones, may be needed.

Summary

Therefore, for patients with cystic fibrosis, treatment with Pulmozyme requires comprehensive consideration of its efficacy and safety. Patients should strictly follow the doctor's instructions when using this drug, and pay attention to their own clinical manifestations and adverse reactions. If they feel unwell, they should seek medical treatment in time.