The Struggle That Is Phenylketonuria : What Do The Patients and Caregivers Suffer From

Objective: To assess the stress levels and life hardships of phenylketonuria patients and their parents.

Study Overview

• Status: Completed
• Conditions: Caregiver Burden; Life Stress; Psychological Well-Being
• Intervention / Treatment: Other: parenting stress index, Zarit burden scale, Rosenberg self esteem scale, stait trait anxiety inventory, struggles and difficulties questionnaire

Detailed Description

Study Design: Between January 2020 - June 2020, a total of 156 phenylketonuria patients and their parents who arrived for their regular examinations were included. Parents were asked to fill the parenting stress index, Zarit burden scale and the strengths and difficulties questionnaire and children over the age of eleven were asked to fill the Rosenberg self-esteem scale, the stait-trait anxiety inventory and strengths and difficulties questionnaire.

• Study Type: Observational
• Enrollment (Actual): 156

Contacts and Locations

Study Locations

• Turkey
  • Sihhiye
    • Ankara, Sihhiye, Turkey, 06100
      • Hacettepe University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Probability Sample

Study Population

This study was carried out at Hacettepe University, İhsan Doğramacı Children's Hospital, Department of Metabolism, between January 2020 and June 2020. It was planned to include patients who were followed up with the diagnosis of phenylketonuria with diet or Sapropterin hydrochloride therapy, who didn't have BH4 metabolism disorder or any other health problem. Pregnant patients were also excluded

Description

Inclusion Criteria:

• Phenylketonuria patients

Exclusion Criteria:

• BH4 metabolism disorde
• Other chronic illness
• Pregnancy

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rosenberg Self-Esteem Scale	Rosenberg Self-Esteem Scale was developed by Morris Rosenberg in 1960. It evaluates self-esteem and consists of 10 items in total. In the Likert-type scale, which includes positively and negatively charged items, 0-1 points are considered as high self-esteem, 2-4 points as medium self-esteem, and 5-6 points as low self-esteem.	12 minutes
Stait trait anxiety inventory	State-Trait Anxiety Inventory is an inventory that measures state and trait anxiety levels of children. It has been developed by Spielberges et. al in 1983. It consists of two questionnaires with a total of 40 questions, 20 that assesses the anxiety level stately, that is, how the child feels at that moment, and 20 that assesses how he feels constantly, that is, in general. The scale is in 3-point Likert type and is evaluated with 1-3 points according to symptom severityThe lowest score that can be obtained from the scales is 20, and the highest score is 60, higher scores indicate higher anxiety.	45 minutes
Strenghts and difficulties questionnaire	Strengths and Difficulties Questionnaire was developed by Goodman in 1997 to assess children's social, emotional and behavioral difficulties. There is a parent form for ages 4-17 and a child form for ages 11-17. It consists of 25 questions in total	30 minutes
Zarit burden scale	Zarit Burden Scale was developed by Zarit et al. in 1980. It is a 5-point Likert-type scale consisting of 22 questions that aims to measure the distress experienced by caregivers. Answers are scored between 0-4. A minimum of 0 and a maximum of 88 points can be obtained from the scale. The high score indicates the height of the distress experienced.	25 minutes
Parenting stress index	Parenting Stress Index aims to measure the stress experienced by parents in their relationships with their children. It is a 4-point Likert-type scale consisting of 16 questions in total. A minimum of 16 and a maximum of 64 points can be obtained from the scale. A high score from the scale indicates that parental stress is high.	20 minutes

Collaborators and Investigators

• Sponsor: Hacettepe University
• Investigators: Study Director: Serap Sivri, Prof, Hacettepe University

Study record dates

Study Major Dates

• Study Start (Actual): May 27, 2020
• Primary Completion (Actual): September 1, 2020
• Study Completion (Actual): September 1, 2020

Study Registration Dates

• First Submitted: February 10, 2024
• First Submitted That Met QC Criteria: February 19, 2024
• First Posted (Actual): February 28, 2024

Study Record Updates

• Last Update Posted (Actual): February 28, 2024
• Last Update Submitted That Met QC Criteria: February 19, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Behavioral Symptoms; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Stress, Psychological; Phenylketonurias; Caregiver Burden
• Other Study ID Numbers: GO20/296

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No