Cystic fibrosis (CF) Patient Profiles: Scenarios for prescribing with Pulmozyme 

How would you manage the challenges presented in these CF patients?

Monica R, age 5, starting school

  • Parents actively manage care

Medical History

  • Initial treatment included vest therapy BID and pancreatic enzyme replacement
  • Occasionally displayed nonproductive cough, usually with onset of a cold

Current presentation

  • Intermittent cough becoming more frequent and productive over last 2 weeks
  • Slight loss of appetite and weight during the last month
  • FEV1 normal at 95% of predicted
  • Chest CT scan shows air trapping, mucus impaction, bronchial wall thickening

How might Pulmozyme help your younger patients fight CF?

Exacerbations* can occur at a young age. Studies have shown lung damage may already be present by the time patients present CF symptoms.

*An exacerbation is defined as a respiratory tract infection requiring parenteral antibiotics. 1

About 1 in 4 patients have experienced 1 or more exacerbations by age 10(19) About 1 in 4 patients have experienced 1 or more exacerbations by age 10(19)
By the time patients present symptoms 50% to 70% of CF patients have computed tomography (CT)-defined bronchiectasis(20,21) By the time patients present symptoms 50% to 70% of CF patients have computed tomography (CT)-defined bronchiectasis(20,21)

Amy T, age 22, young professional

  • Determined to reduce her risk of exacerbations*
  • Prefers to schedule and manage her own treatment at home

Recent medical history

  • Treated with vest therapy, inhaled antibiotics, and dornase alfa QD
  • Recently prescribed a CFTR modulator
  • Experienced just 3 exacerbations in college and maintained FEV1 between 75% and 80% of predicted
  • Hospitalized twice in the last 8 months with Pseudomonas aeruginosa despite compliance and regular visits to her pulmonologist. Pulmozyme dose increased to BID each time, but reverted to QD 2 weeks after hospital discharge

Current presentation

  • Low-grade fever
  • Increasing cough and shortness of breath interfering with work and therapy over the last month
  • FEV1 decreased to 70% of predicted

*An exacerbation is defined as a respiratory tract infection requiring parenteral antibiotics. 1

If once-daily dosing isn’t enough

Keep appropriate patients fighting with Pulmozyme BID dosing

Patients > 21 years of age may benefit from twice-daily (BID) dosing to reduce exacerbation risk.

Pulmozyme BID reduced the relative risk of exacerbations among patients ≥5 years of age with FVC ≥40% of predicted. 1

Incidence of first exacerbation* in patients > 21 years of age (n=384) 1
Placebo 44%
2.5 mg QD 48%
2.5 mg BID 39%
In patients with FVC >= 40% of predicted, Pulmozyme works to reduce risk of exacerbation even if patients don't feel its effects In patients with FVC >= 40% of predicted, Pulmozyme works to reduce risk of exacerbation even if patients don't feel its effects

Consider BID dosing for your CF patients over age 21

Matthew D, age 30, busy professional

  • Has trouble with treatment adherence

Recent medical history

  • Infertility issues and history of asthma
  • Chest CT showed bronchiectasis
  • Treated with Pulmozyme QD, hypertonic saline QD, vest physiotherapy BID, and inhaled antibiotics BID

Current presentation

  • Experienced 4 exacerbations* over the last 2 years, leading to hospitalization
  • Treated with Pulmozyme BID in the hospital and remained on it even after FEV1 was stabilized
  • FEV1 was at 58% when he was hospitalized and is now back up to or near 70%
In patients with FVC >= 40% of predicted, Pulmozyme works to reduce risk of exacerbation even if patients don't feel its effects In patients with FVC >= 40% of predicted, Pulmozyme works to reduce risk of exacerbation even if patients don't feel its effects

*An exacerbation is defined as a respiratory tract infection requiring parenteral antibiotics. 1

The fight against CF can continue into adulthood

Patients between 15 and 30 years of age are more likely to experience at least 1 exacerbation 19

Pulmonary Exacerbation by Age Group Chart Pulmonary Exacerbation by Age Group Chart

Pulmonary exacerbations are defined as treatment in the hospital and/or at home with a course of IV antibiotics. 19
The light gray bars represent the total number of patients with CF, by age, according to the 2014 Cystic Fibrosis Foundation Patient Registry report. 19

 

Signs and symptoms that may indicate the onset of pulmonary exacerbations include 22:

  • Change in lung function
  • Increased cough
  • Change in sputum (volume or consistency)
  • Loss of appetite or weight
  • Fever
Frequent exacerbations are associated with a faster rate of lung function decline(23) Frequent exacerbations are associated with a faster rate of lung function decline(23)

Consider Pulmozyme BID for your older patients

Communicating the importance of adhering to treatment

As new treatments are added to CF treatment plans, it is important to remind patients about the role their current treatments play in the management of CF. Because many patients are on Pulmozyme, reminding them how Pulmozyme works and the distinct role it plays in fighting CF may help keep them adherent.

For more information for patients about staying on track with their CF treatment. You can also review a list of topics to discuss throughout treatment. 

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%).