Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives

Oleg A. Shchelochkov, Klara Dickinson, Bruce F. Scharschmidt, Brendan Lee, Miguel Marino, Cynthia Le Mons

Research output: Contribution to journalArticle

11 Citations (Scopus)

Abstract

Patients and families living with metabolic disorders face challenging dietary and drug treatment regimens. On the hypothesis that poor palatability, volume and frequency of drug/formula administration contribute to treatment non-adherence and hyperammonemic episodes, a survey was conducted of patient, caregiver (CG) and physician perspectives on treatments used in urea cycle disorders (UCD). Methods A paper and online survey assessed experience with UCD medications, medical foods and dietary supplements. Results 25 physicians, 52 adult patients and 114 CG responded. In 2009, the most common UCD-specific intervention reported by patients included sodium phenylbutyrate (60%), followed by L-citrulline (46%), amino acid medical foods (15%), L-arginine preparations (18%), and sodium benzoate (8%). Only 36% of patients reported experiencing no hyperammonemic episodes in the last 2 years. The most commonly reported cause of hyperammonemic episodes was infection or other acute illnesses, followed by dietary indiscretion, side effects of medications, and drug non-adherence. Most patients, caregivers and physicians (> 75%) ranked nitrogen-scavenging medications, L-citrulline, L-arginine, and medical foods as “effective” or “very effective.” Non-adherence was common (e.g. 18% of patients admitted to missing sodium phenylbutyrate “at least once a week” and “at least one a day”). Barriers to adherence included taste of medications, frequency of drug administration, number of pills, difficulty swallowing pills, side effects, forgetting to take medications, and high cost. Strategies to mitigate the gastrointestinal side effects of medications included the use of gastric tubes and acid reflux medications. Physicians indicated that 25% and 33% of pediatric and adult patients, respectively, were given less than the recommended dose of sodium phenylbutyrate due to concerns of tolerance, administration, and cost. Conclusions Despite positive views of their effectiveness, respondents found medications, medical foods and dietary supplements difficult to take and viewed adherence as inadequate, thus contributing to hyperammonemic episodes.

Original languageEnglish (US)
Pages (from-to)43-47
Number of pages5
JournalMolecular Genetics and Metabolism Reports
Volume8
DOIs
StatePublished - Sep 1 2016

Fingerprint

Inborn Urea Cycle Disorder
Caregivers
Dietary Supplements
Pharmaceutical Preparations
Physicians
Citrulline
Therapeutics
Arginine
Sodium Benzoate
Costs and Cost Analysis
Food
Medication Adherence
Deglutition
Gastroesophageal Reflux
Drug-Related Side Effects and Adverse Reactions
Nitrogen
Pediatrics
Amino Acids

ASJC Scopus subject areas

  • Endocrinology
  • Genetics
  • Molecular Biology

Cite this

Barriers to drug adherence in the treatment of urea cycle disorders : Assessment of patient, caregiver and provider perspectives. / Shchelochkov, Oleg A.; Dickinson, Klara; Scharschmidt, Bruce F.; Lee, Brendan; Marino, Miguel; Le Mons, Cynthia.

In: Molecular Genetics and Metabolism Reports, Vol. 8, 01.09.2016, p. 43-47.

Research output: Contribution to journalArticle

Shchelochkov, Oleg A. ; Dickinson, Klara ; Scharschmidt, Bruce F. ; Lee, Brendan ; Marino, Miguel ; Le Mons, Cynthia. / Barriers to drug adherence in the treatment of urea cycle disorders : Assessment of patient, caregiver and provider perspectives. In: Molecular Genetics and Metabolism Reports. 2016 ; Vol. 8. pp. 43-47.
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abstract = "Patients and families living with metabolic disorders face challenging dietary and drug treatment regimens. On the hypothesis that poor palatability, volume and frequency of drug/formula administration contribute to treatment non-adherence and hyperammonemic episodes, a survey was conducted of patient, caregiver (CG) and physician perspectives on treatments used in urea cycle disorders (UCD). Methods A paper and online survey assessed experience with UCD medications, medical foods and dietary supplements. Results 25 physicians, 52 adult patients and 114 CG responded. In 2009, the most common UCD-specific intervention reported by patients included sodium phenylbutyrate (60{\%}), followed by L-citrulline (46{\%}), amino acid medical foods (15{\%}), L-arginine preparations (18{\%}), and sodium benzoate (8{\%}). Only 36{\%} of patients reported experiencing no hyperammonemic episodes in the last 2 years. The most commonly reported cause of hyperammonemic episodes was infection or other acute illnesses, followed by dietary indiscretion, side effects of medications, and drug non-adherence. Most patients, caregivers and physicians (> 75{\%}) ranked nitrogen-scavenging medications, L-citrulline, L-arginine, and medical foods as “effective” or “very effective.” Non-adherence was common (e.g. 18{\%} of patients admitted to missing sodium phenylbutyrate “at least once a week” and “at least one a day”). Barriers to adherence included taste of medications, frequency of drug administration, number of pills, difficulty swallowing pills, side effects, forgetting to take medications, and high cost. Strategies to mitigate the gastrointestinal side effects of medications included the use of gastric tubes and acid reflux medications. Physicians indicated that 25{\%} and 33{\%} of pediatric and adult patients, respectively, were given less than the recommended dose of sodium phenylbutyrate due to concerns of tolerance, administration, and cost. Conclusions Despite positive views of their effectiveness, respondents found medications, medical foods and dietary supplements difficult to take and viewed adherence as inadequate, thus contributing to hyperammonemic episodes.",
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