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Compliance is key to achieve sustained results with NAGLAZYME® (galsulfase) therapy1,2

Enzyme replacement therapy (ERT) is lifelong. In order for the patient to benefit from the treatment, NAGLAZYME® (galsulfase) must be infused regularly, on a weekly basis. If therapy stops, glycosaminoglycans will again build up, and symptoms may return. It is important to help patients and families understand that results vary and some improvements occur over a long period of time.

Phase 3 data and long-term survey data show that compliance with weekly infusions results in sustained improvement in endurance, pulmonary function, and urinary glycosaminoglycan levels.1,2

Clinicians can aid in compliance by helping patients and caregivers to manage key deterrents, such as IV pain as well as logistical issues.

Improving compliance with NAGLAZYME treatment

Compliance with NAGLAZYME therapy is key to successful MPS VI treatment. It is therefore critical that clinicians proactively address issues that may affect compliance over the long term.3 Here are some key issues and ways to address them:

  • Patients with MPS VI may have difficulty making infusion appointments due to work and school obligations or frequent illness or hospitalization
    • Be as flexible as possible in scheduling or rescheduling appointments
  • Painful IV insertion can be a cause for anxiety, which could reduce compliance
    • Consider the use of topical anesthetics and other strategies to reduce the pain of IV insertion

BioMarin RareConnections can help you in supporting patients and parents with reimbursement difficulties and other logistical challenges. Learn more.

Intravenous (IV) access and pain control

Venipuncture is a painful procedure, and a cause of considerable anxiety in children. IV access in patients with MPS VI can present a special challenge due to:

  • Thickened, rough skin4
  • Kinked veins
  • Stiff joints (which can make it difficult for patients to straighten their arms)5

Strategies to avoid IV challenges

Pain control and reduction of anxiety with IV access should be addressed at the initiation of therapy. Methods to reduce discomfort include6:

  • Topical anesthetics
  • Warming the limb prior to venipuncture
  • Advising the patient and parents to maintain good hydration before infusion

Take the sting out of needles

Download a brochure for patients and caregivers to aid in easing or preventing venipuncture pain. It is recommended that the clinician consult a child life specialist prior to the first infusion and again as needed.

Download Needle Fear Brochure

Patient education and support

Caring for patients with chronic conditions poses specific challenges. While many patients and families are well informed about MPS VI, they may still have a range of questions and concerns. Therefore, it’s important to:

  • Educate patients and their families regarding their treatment options
  • Encourage families to speak to others who have had experience with MPS VI and enzyme replacement therapy

Be a patient advocate

Patients with MPS VI often face pain and debility, leading to emotional and psychological problems.7 To help patients, clinicians are encouraged to provide strong psychosocial support by8:

  • Engaging in individual counseling
  • Encouraging interaction with a multidisciplinary team of providers
  • Promoting support groups and communication with other patients and caregivers8,9

Some patients may be able to receive their NAGLAZYME infusions at home »

References:

  1. Harmatz P, Giugliani R, Schwartz IVD et al; MPS VI Study Group. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab. 2008;94(4):469-475. doi:10.1016/j.ymgme.2008.04.001.
  2. Giugliani R, Lampe C, Guffon N, et al. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux–Lamy syndrome)—10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014;164A(8):1953-1964. doi:10.1002/ajmg.a.36584.
  3. NAGLAZYME [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2019.
  4. Tran et al., Pediatr Dermatol. 2016 Nov;33(6):594-601.
  5. Valayannopoulos et al. Orphanet Journal of Rare Diseases 2010, 5:5.
  6. WHO guidelines on drawing blood: best practices in phlebotomy. Accessed: https://www.euro.who.int/__data/assets/pdf_file/0005/268790/WHO-guidelines-on-drawing-blood-best-practices-in-phlebotomy-Eng.pdf
  7. Holley UA. Social isolation: a practical guide for nurses assisting clients with chronic illness. Rehabil Nurs. 2007;32(2):51-56.
  8. Wilcox WR. Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care. J Pediatr. 2004;144(5 suppl):S3-S14.
  9. Lindeke LL, Leonard BJ, Presler B, Garwick A. Family-centered care coordination for children with special needs across multiple settings. J Pediatr Health Care. 2002;16(6):290-297.

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. These reactions have occurred during and up to 24 hours after completion of the NAGLAZYME infusion. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions, including anaphylaxis.

Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue NAGLAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. In patients who have experienced anaphylaxis or other severe allergic reactions during infusion with NAGLAZYME, caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.

Immune-Mediated Reactions
As with other enzyme replacement therapies, immune-mediated reactions, including membranous glomerulonephritis have been observed. In clinical trials, nearly all patients developed antibodies as a result of treatment with NAGLAZYME; however, no consistent predictive relationship between total antibody titer, neutralizing or IgE antibodies, and infusion-associated reactions, urinary glycosaminoglycan (GAG) levels, or endurance measures has been found.

Risk of Acute Cardiorespiratory Failure
Caution should be exercised when administering NAGLAZYME to patients susceptible to fluid volume overload because congestive heart failure may result. Consider a decreased total infusion volume and infusion rate when administering NAGLAZYME to these patients.

Acute Respiratory Complications Associated with Administration
Consideration to delay NAGLAZYME infusion should be given when treating patients who present with an acute febrile or respiratory illness. Sleep apnea is common in MPS VI patients and antihistamine pretreatment may increase the risk of apneic episodes. Evaluation of airway patency should be considered prior to the initiation of treatment. Patients using supplemental oxygen or continuous positive airway pressure (CPAP) during sleep should have these treatments readily available during infusion in the event of an infusion reaction, or extreme drowsiness/sleep induced by antihistamine use.

Infusion Reactions
Pretreatment with antihistamines with or without antipyretics is recommended prior to the start of infusion to reduce the risk of infusion reactions. If infusion reactions occur, decreasing the infusion rate, temporarily stopping the infusion, or administering additional antihistamines and/or antipyretics is recommended.

Spinal or Cervical Cord Compression
Spinal/cervical cord compression is a known and serious complication that is expected to occur during the natural course of MPS VI. Signs and symptoms of spinal/cervical cord compression include back pain, paralysis of limbs below the level of compression, and urinary or fecal incontinence. Patients should be evaluated for spinal/cervical cord compression prior to initiation of NAGLAZYME to establish a baseline and risk profile. Patients treated with NAGLAZYME should be regularly monitored for the development or progression of spinal/cervical cord compression and be given appropriate clinical care.

Adverse Reactions
During infusion, serious adverse reactions included laryngeal edema, apnea, pyrexia, urticaria, respiratory distress, angioedema, and anaphylactoid reaction; severe adverse reactions included urticaria, chest pain, rash, abdominal pain, dyspnea, apnea, laryngeal edema, and conjunctivitis. The most common adverse events (≥10%) observed in clinical trials in patients treated with NAGLAZYME were rash, pain, urticaria, pyrexia, pruritus, chills, headache, nausea, vomiting, abdominal pain and dyspnea. The most common adverse reactions requiring interventions are infusion-related reactions.

 

To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-888-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information with Boxed Warning for risk of anaphylaxis or visit www.Naglazyme.com.

INDICATION
NAGLAZYME® (galsulfase) is indicated for patients with mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.