Compliance with Therapy

Enzyme replacement therapy provides the body with a supply of enzyme to reduce the amount of substrate being stored and to prevent re-accumulation. In order to continue to benefit from the treatment, symptomatic patients must continue to receive intravenous infusions. 

Because Gaucher disease is both chronic and progressive, poor adherence can be a major reason for not achieving the full benefits of long term enzyme replacement therapy. 1,2 A suboptimal response to treatment can cause medical complications and may also lead to increased need for health care resources compared to adherent patients.2

According to the World Health Organization, poor adherence to treatment of chronic diseases is a world wide problem of striking magnitude. In its guide “Adherence to Long-Term Therapies: Evidence for Action”, the WHO reports that in the developed world alone, adherence to long-term therapies averages only 50%.3

While patients play a role in maintaining adherence, there is evidence that health care providers greatly influence the behavior of patients. Practitioners who identify patients with poor adherence can often improve adherence by emphasizing the value of the prescribed therapy. Patients who chronically do not maintain their adherence to treatment may need more personalized interventions to help them adhere to their therapy.4

Barriers to Adherence

There can be many barriers to adherence for Gaucher disease patients. Research has shown that patients are less likely to be adherent when taking medication primarily to prevent future consequences. This is because the benefits of treatment may be delayed, while the costs are more immediate (financial, inconvenience, time, discomfort).5

Non-adherence can also be related to the patient’s belief that his disease is not serious and that treatment is not necessary. The patient’s belief that he is not vulnerable to long-term negative outcomes may lead him to conclude that treatment is not necessary for an asymptomatic condition.5

Factors Influencing Adherence

Studies show that there is a significant connection between medication adherence and these factors:

  1. The quality of the interaction between the patient and the health care provider.  Patients’ trust in their physician has been shown to predict patient adherence to a medical regimen.6
  2. The patient’s motivation, a sense of normality, attitude, energy, and experience of results, or a feeling of a threat to well being. 7
  3. Support from family, friends, nurses and physicians.7

Strategies to Support Patient Adherence

Patients who adhere to treatment see themselves as working to maintain their health in close collaboration with healthcare professionals.7 The patient’s active commitment to care is important.5,6

It is crucial that patients are involved in their treatment planning and that their needs are considered. Patients feel more autonomous when their physician encourages questions and listens to their requests.5

According to a study by Leonard Barry in the Annals of Family Medicine, the following approaches can help support patient adherence:6

  1. Trust: Building a positive physician-patient, patient-nurse/counselor relationship is vital to establishing trust. Trust and commitment have a positive influence on patients’ health behaviors. Both interpersonal knowledge and support of the patient’s autonomy convey respect and build trust. Health care providers evoke patients’ trust and convey respect by:
    1. Listening carefully to their patients.
    2. Developing whole-person knowledge about them
    3. Explaining issues clearly
    4. Demonstrating compassion
    5. Treating patients as partners in their own care
  2. Education: Information should be given verbally and in written format. Patient education must be personalized to individual needs in order to be most effective and have a lasting impact on the patient.
  3. Understanding:  Learn about the patient’s health beliefs and find out what is meaningful to him. Ask the patient what they think and know about the treatment of their LSD. 
  4. Communication: Ensure that the healthcare team is available and accessible to the patient. Studies support the conventional wisdom that patients respond positively to open dialogue and information rather than closed ended instruction.24 Discussion helps to establish a two-way flow of information between the healthcare professional and the patient.  And it is essential that the patient be involved in his treatment planning.
  5. Evaluation: Assess adherence at each clinic visit. You can support the patient’s efforts by asking about adherence in a non-judgmental way. Non-judgmental conversation will often allow the patient to acknowledge any problems with adherence.

Poor adherence to medication regimens is common, often contributing to adverse outcomes and increased health care costs.4 A collaborative approach to care can augment the patient’s medication adherence, and the physicians who have strong patient relationships appear to have great influence on their patient’s health behaviors.4,6

Indication & Usage

Cerezyme® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:

  1. anemia
  2. thrombocytopenia
  3. bone disease
  4. hepatomegaly or splenomegaly

Important Safety Information

Approximately 15% of patients have developed IgG antibodies to Cerezyme during the first year of therapy. Approximately 46% of patients with detectable IgG antibodies experienced symptoms of hypersensitivity, and these patients have a higher risk of hypersensitivity. It is suggested that patients be monitored periodically for IgG antibody formation during the first year of treatment.

Hypersensitivity has also been observed in patients without detectable IgG antibodies. Symptoms suggestive of hypersensitivity have been noted in approximately 6.6% of all patients, and anaphylactoid reactions in less than 1%. Treatment with Cerezyme should be approached with caution in patients who have exhibited hypersensitivity symptoms such as pruritus, flushing, urticarial, angioedema, chest discomfort, dyspnea, coughing, cyanosis, and hypotension. Pre-treatment with antihistamines and/or corticosteroids and a reduced rate of infusion may allow continued treatment in most patients.

In less than 1% of patients, pulmonary hypertension and pneumonia have been observed during treatment with Cerezyme. These are known complications of Gaucher disease regardless of treatment. Patients with respiratory symptoms in the absence of fever should be evaluated for the presence of pulmonary hypertension.

Approximately 13.8% of patients have experienced adverse events related to treatment with Cerezyme. Some of these are injection site reactions such as discomfort, pruritus, burning, swelling or sterile abscess at the site at the site of venipuncture. Additional adverse reactions that have been reported include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Transient peripheral edema has also been reported for this therapeutic class of drug.

To report suspected adverse reactions, contact Sanofi Genzyme at 800-745-4447, option 2

Please see Full Prescribing Information (PDF).


  1. Anderson H, Charrow J, Kaplan P, et al. Individualization of long-term enzyme replacement therapy for Gaucher disease. Genet Med.2005; 7(2):105–110.
  2. Pastores GM, Weinreb NJ, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol 2004:41(suppl 5);4-14.
  3. Accessed 12-15-10.
  4. Osterberd, L, Blaschke, T. Drug therapy: adherence to medication. New England Journal of Mediciine.2005; 353:5:487-97.
  5. Cook, F. et al. Telephone counseling to improve osteoporosis treatment adherence: an effectiveness study in community practice settings. American Journal of Medical Quality. 2007; 22: 445-455.
  6. Berry, et al. Patients’ commitment to their primary physician and why it matters. Annals of Family Medicine. 2008;6:1:6-13.
  7. Kyngis, H. Compliance of adolescents with chronic disease. Journal of Clinical Nursing. 2000; 9:549-556.