Start PROCYSBI immediately after diagnosis of nephropathic cystinosis1

PROCYSBI® (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules should be initiated immediately following diagnosis of nephropathic cystinosis in patients aged ≥1 year.1

Early cystine-depleting therapy (CDT) treatment can delay or limit damage to the patient’s tissues and organs caused by rising cystine levels, but treatment cannot reverse damage that has already occurred.2-4

Determine dose by patient experience with cysteamine bitartrate1

Patients switching from immediate-release (IR) cysteamine bitartrate should start PROCYSBI by taking a total daily dose equal to their previous total daily dose.1

For cysteamine-naïve patients, PROCYSBI should be initiated with a dosage equal to one-sixth to one-fourth of the maintenance dose. The maintenance dosage after initial dose escalation is 1.3 g/m2 of body surface area (BSA) per day divided into 2 doses given every 12 hours.1

Find your patient’s dose

This tool is intended to provide dose calculations for your cysteamine-naïve patients. The recommended maintenance dosage is 1.3 g/m2 per day. Dose calculations should be rounded to the nearest available strengths of capsules or packets of oral granules.

Please see the Dosage and Administration section of the Full Prescribing Information for complete dosing instructions or request a representative for more information on calculating dosage.

Once your patient’s dosage is determined, please refer to the Monitoring Frequency Table below to determine monitoring frequency.

Starting PROCYSBI
Instructions

Enter the height and weight of your patient in desired units to calculate BSA.*

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Please enter the patient’s height.
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Please enter the patient’s weight. This calculator is not intended for weight above 51 kg/112 lb. Please reach out to a Horizon Representative for assistance.
Calculated BSA:

Dosage Results

Starting PROCYSBI Dosage in Cysteamine-Naïve Patients Every 12 Hours, as a Fraction of the Maintenance Dosage1

1/6 of dosage mg

1/4 of dosage mg

Maintenance Dosage Every 12 Hours1§

mg

The results may be printed or emailed once calculated. Note the email address provided will not be registered for educational or promotional emails and is used solely for delivering the custom dosing results.

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Print Results

*BSA can be calculated by multiple equations; the Mosteller formula is used on this website.5
†Rounded to the nearest whole number.
‡Rounded to the nearest tenth.

§Higher dosages may be required to achieve target therapeutic white blood cell (WBC) cystine concentration. The maximum dosage of PROCYSBI is 1.95 g/m2 of BSA per day.

If you have questions or would like to learn more about appropriate dosing and administration, you may connect with a representative.

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Dosing Flashcard

For dosing calculations by patient weight, download the dosing flashcard.

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Starting low and slowly increasing the dose may reduce the risk of some adverse events1

  • Patients aged 1 year to <6 years: Increase the dosage in 10% increments to the maintenance dosage while monitoring WBC cystine concentrations. Allow a minimum of 2 weeks between dosage adjustments.
  • Patients aged ≥6 years: Gradually increase the dosage over 4 to 6 weeks until the maintenance dosage is achieved.

Administration options

PROCYSBI offers flexibility in administration, with 3 different ways your patients can take it.1

  • Two sizes of PROCYSBI® (cysteamine bitartrate) delayed-release capsules icon

    1 - Swallowed whole

    PROCYSBI capsules can be swallowed whole with fruit juice (except grapefruit juice) or water. Do not crush or chew the capsules.1

  • PROCYSBI® (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules microbeads mixed with food or liquid icon

    2 - Opened and mixed with select foods or liquids

    For patients who cannot tolerate PROCYSBI on an empty stomach or have difficulty swallowing, capsules or packets should be opened and the microbeads mixed with select foods or liquids. The microbeads may be mixed in about ½ cup (4 oz.) of applesauce, berry jelly, or fruit juice (except grapefruit juice). The mixture should be consumed within 30 minutes of mixing and should not be saved for later use.1

  • Gastrostomy tube icon

    3 - Through a G-tube

    For individuals with a G-tube that is size 14 French or larger, PROCYSBI capsules or packets should be opened and the microbeads mixed with about ½ cup (4 oz.) of strained applesauce with no chunks. The mixture should be administered within 30 minutes of mixing and should be not saved for later use.1

Stomach icon

Stomach acidity affects PROCYSBI’s release and absorption

To maintain the right stomach acid levels for continuous cystine control, patients should1:

Take PROCYSBI at least 1 hour before or after they take medicines that contain bicarbonate or carbonate.

AND icon.

Not eat for at least 2 hours before and 30 minutes after taking PROCYSBI.

OR icon.

Take PROCYSBI with no more than ½ cup (4 oz) of food up to 1 hour before or after they take it, if they cannot tolerate PROCYSBI on an empty stomach.

References: 1. PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules [prescribing information] Horizon. 2. Nesterova G, Gahl WA. Cystinosis: the evolution of a treatable disease. Pediatr Nephrol. 2013;28(1):51-59. 3. Gahl WA, Balog JZ, Kleta R. Nephropathic cystinosis in adults: natural history and effects of oral cysteamine therapy. Ann Intern Med. 2007;147(4):242-250. 4. 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. 5. Mosteller RD. Simplified calculation of body-surface area. N Engl J Med. 1987;317(17):1098.

Ongoing management of cystinosis

Continuous cystine control is critical to limiting the progression of cystinosis. Even brief interruptions in cysteamine dosing allow a rapid return to toxic levels of cystine.1

In a study, patients who 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 those with a shorter timeframe between doses and in excess of the target level.1

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

Graph showing significantly higher cystine levels in people who delayed taking their next 6-hour dose by 3 hours (at hour 9)

Graph showing significantly higher cystine levels in people who delayed taking their next 6-hour dose by 3 hours (at hour 9)

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

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Patients switching from IR cysteamine to PROCYSBI

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Cysteamine-naïve patients aged 1 year to <6 years

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Cysteamine-naïve patients ≥6 years
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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)
Support and resources icon Support and resources icon

Cysteamine-naïve patients aged 1 year 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)
Support and resources iconSupport and resources icon

Cysteamine-naïve 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)

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

There are 2 WBC cystine level tests available, and they are not interchangeable.4,5 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
Monitoring Timing2
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] Horizon. 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.

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 Intracranial 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.

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 Intracranial 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.