Information about Pulmozyme use in pediatric patients 

Safety data in cystic fibrosis (CF) patients under 5 years of age

While clinical trial data are limited in pediatric patients younger than 5 years of age, the use of Pulmozyme should be considered for pediatric CF patients who may experience potential benefit in:

  • Pulmonary function
  • Relative risk of respiratory tract infection

Adverse events in patients under 5 years of age

  • In patients under 5 years of age, only the safety profile of Pulmozyme has been studied.
  • In a clinical study, Pulmozyme 2.5 mg by inhalation once daily was administered over 2 weeks to 98 patients with CF (65 patients aged 3 months to < 5 years, 33 patients aged 5 to ≤ 10 years). 25
    • The PARI BABY™ reusable nebulizer (which uses a face mask instead of a mouthpiece) was utilized in patients unable to demonstrate the ability to inhale or exhale orally throughout the entire treatment period (54/65, 83% of the younger; and 2/33, 6% of the older patients).
    • This study demonstrated that nebulized medication can be delivered to the airway, is present in comparable amounts, and produces similar side effects in younger children compared with older children. 25
Adverse reactions in pediatric patients 1
  Younger age group (n=65) Older age group (n=33)
Cough 45% (29) 30% (10)
Moderate to severe cough 37% (24) 18% (6)
Rhinitis 35% (23) 27% (9)
Rash 6% (4) 0% (0)
  • Reports of cough (including moderate to severe cough), rhinitis, and rash were more frequent in the younger age group compared with the older age group; other events were of mild to moderate severity.
  • The nature of adverse events was similar to that seen in larger trials of Pulmozyme.

 

Trials or studies demonstrating efficacy in Pulmozyme in patients younger than 5 years of age have not been conducted.
Data in studies conducted in the adult setting cannot be extrapolated to patients younger than 5 years of age.

Important Safety Information

Pulmozyme is contraindicated in patients with known hypersensitivity to dornase alfa, Chinese Hamster Ovary cell products, or any component of the product.

The most common adverse reactions associated with the use of Pulmozyme include: voice alteration, pharyngitis, rash, laryngitis, chest pain, conjunctivitis, rhinitis, decrease in FVC of ≥ 10%, fever, dyspepsia, and dyspnea. There have been no reports of anaphylaxis attributed to the administration of Pulmozyme. Mild to moderate urticaria and mild skin rash have been observed and have been transient.

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

For further information, please see the Pulmozyme full Prescribing Information.

Indication

Pulmozyme (dornase alfa) is indicated for daily administration in conjunction with standard therapies for the management of cystic fibrosis (CF) patients to improve pulmonary function.

In CF patients with an FVC ≥ 40% of predicted, daily administration of Pulmozyme has also been shown to reduce the risk of respiratory tract infections requiring parenteral antibiotics.

Pediatric Use

The safety and effectiveness of Pulmozyme have been established in pediatric patients 5 years of age and older. The safety of Pulmozyme, 2.5 mg by inhalation, was studied with 2 weeks of daily administration in 65 patients with cystic fibrosis aged 3 months to < 5 years. While clinical trial data are limited in pediatric patients younger than 5 years of age, the use of Pulmozyme should be considered for pediatric CF patients who may experience potential benefit in pulmonary function or who may be at risk of respiratory tract infection.

The safety of Pulmozyme, 2.5 mg by inhalation, was studied with 2 weeks of daily administration in 98 pediatric patients with cystic fibrosis 3 months to 10 years of age (65 aged 3 months to < 5 years, 33 aged 5 to ≤ 10 years). The PARI BABY™ reusable nebulizer (which uses a facemask instead of a mouthpiece) was utilized in patients unable to demonstrate the ability to inhale or exhale orally throughout the entire treatment period (54/65, 83% of the younger; and 2/33, 6% of the older patients). Overall, the nature of adverse reactions was similar to that seen in the placebo-controlled trials in older patients. The number of patients reporting cough was higher in the younger age group as compared to the older age group (29/65, 45%; compared to 10/33, 30%) as was the number reporting moderate to severe cough (24/65, 37%; compared to 6/33, 18%). The number of patients reporting rhinitis was higher in the younger age group as compared to the older age group (23/65, 35%; compared to 9/33, 27%) as was the number reporting rash (4/65, 6% as compared to 0/33, 0%).