Primary Periodic Paralysis (PPP) Diagnosis and Management

Every patient’s journey with Primary Periodic Paralysis PPP is different

Delayed and missed diagnoses

Some patients are fortunate to receive a diagnosis early on. However, for many patients the road to an answer can be long and trying. PPP often has nonspecific and episodic symptoms that mimic those of more common diseases, resulting in years of diagnostic delays and misdiagnoses.1

The average time from onset of symptoms to correct diagnosis is 26 years.*2
  • *Based on a survey of 66 self-selected patients over the age of 40 years with a clinical diagnosis of Primary Periodic Paralysis who sought support via the Internet.2

Dr. Jackson, a PPP expert, discusses the difficulty of diagnosing PPP

Establishing a PPP diagnostic protocol

Strongbridge Biopharma, as part of its commitment to helping patients, sponsored a roundtable discussion with expert clinicians in the field about PPP clinical challenges and best practices. The output of that discussion was a white paper including a detailed diagnostic algorithm for PPP, shown below, which could help you identify PPP in your patients so that they can learn to manage their condition, and receive treatment.

For more information on the diagnostic journey of PPP, read the white paper.

Although genetic testing can help confirm a suspected diagnosis, the absence of a genetic alteration does not preclude diagnosis.3

Management strategies for PPP

These strategies, including both lifestyle changes and medical approaches, may help your patients manage their PPP on a daily basis.

Lifestyle changes

Attacks may be minimized with:

  • Avoiding triggers
  • Mild exercise
  • Changes to diet

Medical approaches, both for prevention and management

Attacks may be managed by:

  • Prescribing medications such as KEVEYIS
  • OTC medications:
    • Oral potassium salts
    • Inhalation of salbutamol
  • Intravenous medications:
    • Calcium gluconate

Janine, a real KEVEYIS patient, discusses her dose adjustment

Management strategies will depend on the patient’s specific subtype of PPP.
Support resources are available to your patients with PPP. Learn more.

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Indication

KEVEYIS is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.

Important Safety Information

Contraindications
  • Hypersensitivity to dichlorphenamide or other sulfonamides
  • Concomitant use of KEVEYIS and high-dose aspirin
  • Severe pulmonary disease, limiting compensation to metabolic acidosis caused by KEVEYIS
  • Hepatic insufficiency: KEVEYIS may aggravate hepatic encephalopathy
Warnings and Precautions

Hypersensitivity and Other Life-Threatening Reactions

  • Fatalities associated with the administration of sulfonamides have occurred because of adverse reactions including Stevens-Johnson syndrome, toxic epidermal necrolysis, fulminant hepatic necrosis, agranulocytosis, aplastic anemia, and other blood dyscrasias.
  • Pulmonary involvement can occur in isolation or as part of a systemic reaction.
  • Discontinue KEVEYIS at the first appearance of skin rash or any sign of immune-mediated or other life-threatening adverse reaction.

Concomitant Use of Aspirin or Other Salicylates

  • Carbonic anhydrous inhibitors, including KEVEYIS, can cause metabolic acidosis, which can increase the risk of salicylate toxicity.
  • Anorexia, tachypnea, lethargy, and coma have been reported with concomitant use of dichlorphenamide and high-dose aspirin.
  • Concomitant use of KEVEYIS and high-dose aspirin is contraindicated. Use with caution and carefully monitor in patients receiving low-dose aspirin.

Hypokalemia

  • KEVEYIS increases potassium excretion and can cause hypokalemia.
  • The risk of hypokalemia is greater when KEVEYIS is used in patients with conditions associated with hypokalemia (e.g., adrenocortical excess, renal tubular acidosis type 1 and 2), and in patients receiving other drugs that may cause hypokalemia (e.g., loop diuretics, thiazide diuretics, laxatives, antifungals, penicillin, and theophylline).
  • Baseline and periodic measurements of serum potassium are recommended.
  • If hypokalemia develops or persists, consider reducing the dose or discontinuing KEVEYIS and correction of potassium levels.

Metabolic Acidosis

  • KEVEYIS can cause hyperchloremic non-anion gap metabolic acidosis.
  • Concomitant use of KEVEYIS with other drugs that cause metabolic acidosis may increase the severity of acidosis.
  • Concomitant use of KEVEYIS in compensated patients with respiratory acidosis, such as in advanced lung diseases, may lead to respiratory decompensation.
  • Baseline and periodic measurements of serum bicarbonate during KEVEYIS treatment are recommended.
  • If metabolic acidosis develops or persists, consider reducing the dose or discontinuing KEVEYIS.

Falls

  • KEVEYIS increases the risk of falls; risk is greater in the elderly and with higher doses.
  • Consider dose reduction or discontinuation of KEVEYIS in patients who experience falls while treated with KEVEYIS.
Pregnancy and Lactation

Use during pregnancy only if the potential benefit justifies the potential risk to the fetus. It is not known in humans whether dichlorphenamide is excreted in human milk; exercise caution when administered to a nursing woman.

Adverse Reactions

The most common adverse reactions seen in clinical trials (incidence ≥ 10% and greater than placebo) include paresthesias, cognitive disorder, dysgeusia, and confusional state.

References

  1. Charles G, Zheng C, Lehmann-Horn F, Jurkat-Rott K, Levitt J. Characterization of hyperkalemic periodic paralysis: a survey of genetically diagnosed individuals. J Neurol. 2013;260:2606-2613.
  2. Cavel-Greant D, Lehmann-Horn F, Jurkat-Rott K. The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients. Acta Myol. 2012;31:126-133.
  3. Statland JM, Fontaine B, Hanna MG, et al. Review of the diagnosis and treatment of periodic paralysis. Muscle Nerve. 2018;57:522-530.