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Treatment Adherence

Ongoing management of cystinosis

Continuous cystine control is critical to limiting the progression of cystinosis. Even a brief interruption in cysteamine administration can result in a rapid return to toxic levels of cystine.1

In a study, when patients had a longer interval between their dose of cystine-depleting therapy (CDT) by 3 hours (9 hours vs 6 hours) had white blood cell (WBC) cystine levels that were 65% higher than when they had a shorter timeframe.1

Comparison of WBC cystine content with immediate-release (IR) cysteamine administered at 9 hours vs 6 hours after nighttime dose1

cystine-concentration-with-delayed-dose-graph
  • Study Design

    Comparison of morning leukocyte content with IR CDT when administered every 6 hours (8 AM, 2 PM, 8 PM, 2 AM) vs 4 times a day with a 9-hour night pause (8 AM, 1 PM, 6 PM, 11 PM) in a cohort of 22 Dutch patients (mean age 14.7 ± 9.7 years) with nephropathic cystinosis.5

WBC cystine levels should be regularly measured to help ensure optimal PROCYSBI dosing2

To reach the correct maintenance dose, WBC cystine levels should be monitored according to the schedules in the table below. Once patients reach their maintenance dose, cystine levels should be measured at least twice a year for both children and adults.2

Monitoring Frequency2

  • Patients switching from IR cysteamine to PROCYSBI
    • 2 weeks after PROCYSBI initiation
    • Once therapeutic target is achieved, monitor quarterly for 6 months
    • Then twice yearly (at minimum)
  • Cysteamine-naive patients aged 1 to <6 years
    • 2 weeks after PROCYSBI initiation
    • Then monitor until the target WBC cystine concentration is achieved
    • Then monthly for 3 months
    • Then quarterly for 1 year
    • Then twice yearly (at minimum)
  • Cysteamine naive
    patients 6 years
    • Obtain measurement after reaching the maintenance PROCYSBI dosage
    • Then monthly for 3 months
    • Then quarterly for 1 year
    • Then twice yearly (at minimum)

  • Patients switching from IR cysteamine to PROCYSBI

  • Cysteamine-naïve patients aged 1 to <6 years

  • Cysteamine-naïve patients ≥6 years

  • 2 weeks after PROCYSBI initiation
  • Once therapeutic target is achieved, monitor quarterly for 6 months
  • Then twice yearly (at minimum)
  • 2 weeks after PROCYSBI initiation
  • Then monitor until the target WBC cystine concentration is achieved
  • Then monthly for 3 months
  • Then quarterly for 1 year
  • Then twice yearly (at minimum)
  • Obtain measurement after reaching the maintenance PROCYSBI dosage
  • Then monthly for 3 months
  • Then quarterly for 1 year
  • Then twice yearly (at minimum)

Maintenance doses may require adjustment to achieve the target WBC cystine levels.2

If the WBC cystine concentration is greater than the target level, consider the following before dose adjustment2:

  • Adherence to medication and dosing interval
  • The timing between the last dose and the blood draw for the laboratory measurement
  • The timing of PROCYSBI administration in relation to food or other administration instructions

Regular WBC cystine level testing is an important element in the effective treatment of cystinosis2-4

Two WBC cystine level tests are available but cannot be used interchangeably.4,5 The same type of test should be used consistently to achieve comparable results. Instruct patients to get a trough blood test and to take PROCYSBI immediately after. It’s recommended to use the same test type each time in order to compare results over time.2 Be sure to recommend that your patients obtain their blood draw just before they take their next dose of PROCYSBI.2,4

Type of Test Testing Institution Test-Specific Target Cystine Level5
Granulocytes University of California San Diego Less than 1.9 nmol ½ cystine/mg protein
Mixed leukocytes Baylor Genetics Less than 1.0 nmol ½ cystine/mg protein

Timing of WBC Monitoring2

Obtain WBC sample 12 hours after dosing with PROCYSBI. It is important to accurately record the time of the last dose, the actual dose, and the time the blood sample was taken.

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  • References

    1. Levtchenko EN, van Dael CM, de Graaf-Hess AC, et al. Strict cysteamine dose regimen is required to prevent nocturnal cystine accumulation in cystinosis. Pediatr Nephrol. 2006;21(1):110-113. 2. PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules [prescribing information] Amgen. 3. Elmonem MA, Veys KR, Soliman NA, van Dyck M, van den Heuvel LP, Levtchenko E. Cystinosis: a review. Orphanet J Rare Dis. 2016;11(47):1-17. 4. Langman CB, Barshop BA, Deschênes G, et al. Controversies and research agenda in nephropathic cystinosis: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference. Kidney Int. 2016;89(6):1192-1203. 5. Gertsman I, Johnson WS, Nishikawa C, Gangoiti JA, Holmes B, Barshop BA. Diagnosis and monitoring of cystinosis using immunomagnetically purified granulocytes. Clin Chem. 2016;62(5):766-772.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with serious hypersensitivity reaction, including anaphylaxis to penicillamine or cysteamine.

WARNINGS AND PRECAUTIONS

  • Ehlers-Danlos-like Syndrome: Skin and bone lesions that resemble clinical findings for Ehlers-Danlos-like syndrome have been reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts. Monitor patients for development of skin or bone lesions and reduce PROCYSBI dosing if patients develop these lesions.
  • Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. Discontinue use if severe skin rash occurs.
  • Gastrointestinal (GI) Ulcers and Bleeding: GI ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate. Monitor for GI symptoms and consider decreasing the dose if severe symptoms occur.
  • Fibrosing Colonopathy: Fibrosing colonopathy has been reported with postmarketing use of PROCYSBI. Evaluate patients with severe, persistent, and/or worsening abdominal symptoms for fibrosing colonopathy. If the diagnosis is confirmed, permanently discontinue PROCYSBI and switch to immediate-release cysteamine bitartrate capsules.
  • Central Nervous System (CNS) Symptoms: CNS symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine. Monitor for CNS symptoms; interrupt or reduce the dose for severe symptoms or those that persist or progress.
  • Leukopenia and/or Elevated Alkaline Phosphatase Levels: Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels. Monitor white blood cell counts and alkaline phosphatase levels; decrease or discontinue the dose until values revert to normal.
  • Benign lntracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate-release cysteamine bitartrate treatment. Monitor for signs and symptoms of PTC; interrupt or reduce the dose for signs/symptoms that persist, or discontinue if diagnosis is confirmed.

ADVERSE REACTIONS

The most common adverse reactions reported in PROCYSBI clinical trials (≥ 5%): were:

  • Patients 2 years of age and older previously treated with cysteamine: vomiting, nausea, abdominal pain, headache, conjunctivitis, influenza, gastroenteritis, nasopharyngitis, dehydration, ear infection, upper respiratory tract infection, fatigue, arthralgia, cough, and pain in extremity.
  • Patients 1 year of age and older naïve to cysteamine treatment: vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, headache.

DRUG INTERACTIONS

  • Drugs that increase gastric pH may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration. Monitor WBC cystine concentration with concomitant use.
  • Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, as well as the effectiveness and safety of PROCYSBI.
  • PROCYSBI can be administered with electrolyte (except bicarbonate) and mineral replacements necessary for management of Fanconi Syndrome as well as vitamin D and thyroid hormone.

USE IN SPECIFIC POPULATIONS

  • Lactation: Because of the potential risk for serious adverse reactions in breastfed children from cysteamine, breastfeeding is not recommended during treatment with PROCYSBI.

Please see Full Prescribing Information.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with serious hypersensitivity reaction, including anaphylaxis to penicillamine or cysteamine.

WARNINGS AND PRECAUTIONS

  • Ehlers-Danlos-like Syndrome: Skin and bone lesions that resemble clinical findings for Ehlers-Danlos-like syndrome have been reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts. Monitor patients for development of skin or bone lesions and reduce PROCYSBI dosing if patients develop these lesions.
  • Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. Discontinue use if severe skin rash occurs.
  • Gastrointestinal (GI) Ulcers and Bleeding: GI ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate. Monitor for GI symptoms and consider decreasing the dose if severe symptoms occur.
  • Fibrosing Colonopathy: Fibrosing colonopathy has been reported with postmarketing use of PROCYSBI. Evaluate patients with severe, persistent, and/or worsening abdominal symptoms for fibrosing colonopathy. If the diagnosis is confirmed, permanently discontinue PROCYSBI and switch to immediate-release cysteamine bitartrate capsules.
  • Central Nervous System (CNS) Symptoms: CNS symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine. Monitor for CNS symptoms; interrupt or reduce the dose for severe symptoms or those that persist or progress.
  • Leukopenia and/or Elevated Alkaline Phosphatase Levels: Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels. Monitor white blood cell counts and alkaline phosphatase levels; decrease or discontinue the dose until values revert to normal.
  • Benign lntracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate-release cysteamine bitartrate treatment. Monitor for signs and symptoms of PTC; interrupt or reduce the dose for signs/symptoms that persist, or discontinue if diagnosis is confirmed.

ADVERSE REACTIONS

The most common adverse reactions reported in PROCYSBI clinical trials (≥ 5%): were:

  • Patients 2 years of age and older previously treated with cysteamine: vomiting, nausea, abdominal pain, headache, conjunctivitis, influenza, gastroenteritis, nasopharyngitis, dehydration, ear infection, upper respiratory tract infection, fatigue, arthralgia, cough, and pain in extremity.
  • Patients 1 year of age and older naïve to cysteamine treatment: vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, headache.

DRUG INTERACTIONS

  • Drugs that increase gastric pH may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration. Monitor WBC cystine concentration with concomitant use.
  • Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, as well as the effectiveness and safety of PROCYSBI.
  • PROCYSBI can be administered with electrolyte (except bicarbonate) and mineral replacements necessary for management of Fanconi Syndrome as well as vitamin D and thyroid hormone.

USE IN SPECIFIC POPULATIONS

  • Lactation: Because of the potential risk for serious adverse reactions in breastfed children from cysteamine, breastfeeding is not recommended during treatment with PROCYSBI.

Please see Full Prescribing Information.