Investigating the Morbidity of Glucocorticoid Use in Patients With Autoimmune Bullous Diseases (AIBDs)

Investigating the Nature and Prevalence of Glucocorticoid-related Morbidity in Patients With Autoimmune Bullous Disease (AIBD) Using the Glucocorticoid Toxicity Index (GTI)

This project utilised the validated glucocorticoid toxicity index (GTI) tool to assess the morbidity of glucocorticoid-use in patients with autoimmune bullous disease. In particular, the study investigated the nature and prevalence of glucocorticoid-induced myopathy.

Study Overview

• Status: Active, not recruiting
• Conditions: Glucocorticoids Toxicity; Autoimmune Bullous Dermatosis
• Intervention / Treatment: Drug: GlucoCorticoid

Detailed Description

This study is an multi-centre observational trial. Patients with AIBDs are recruited based on the criteria: an age of eighteen years or above; a new or established diagnosis of autoimmune bullous disease confirmed by clinical assessment, histopathology, immunofluorescence, and enzyme-linked immunoassay (ELISA) or biochip testing; new or current use of oral glucocorticoids for the treatment of autoimmune bullous disease (Group 1, active treatment group), patients with receipt of previous oral glucocorticoid use with no current use (Group 2, steroid-sparing control group) or patients who had ceased glucocorticoids during the study (Group 3, ceased steroid group) ; attendance of at least one baseline visit (V1) and one follow-up visit during the study period; and, the provision of capacitated and informed consent.

Patients are considered for enrolment based on inclusion criteria after attendance at a bullous disease clinic at a participating study site. At the baseline visit (V1), patients are comprehensively interviewed for demographic factors (age, gender), pre-existing medical conditions, and past and current medications. Patients attend regular follow-up visits at three-month intervals, to a maximum follow-up period of 15 months (V6). Treatment plans, including glucocorticoid dose, are determined by accredited medical professionals based on the clinical condition of the patient.

The Glucocorticoid Toxicity Index (GTI) tool (Figure 1) is used to evaluate glucocorticoid toxicity at all visits, including in dermatologic, neuropsychiatric, musculoskeletal, endocrine and metabolic domains.

Myopathy was measured and graded in accordance to the GTI.

Patients were divided into three groups: Group 1 (control) comprised of patients was comprised of who were not exposed to glucocorticoids during the study period, Group 2 comprised of patients whose glucocorticoid dose was ceased during the study period. Patients who continued on glucocorticoid treatment for the duration of the study period formed the third group.

• Study Type: Observational
• Enrollment (Estimated): 138

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2217
      • Premier Specialists

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with autoimmune bullous disease who regularly attend a bullous clinic as part of their regular care. Patients who have either used glucocorticoids in the past or are currently using glucocorticoids.

Description

Inclusion Criteria:

• An age of eighteen years or above.
• A new or established diagnosis of autoimmune bullous disease confirmed by clinical assessment, histopathology, immunofluorescence, and enzyme-linked immunoassay (ELISA) or biochip testing
• New or current use of oral glucocorticoids for the treatment of autoimmune bullous disease (Group 1, active treatment group), or patients with receipt of previous oral glucocorticoid use with no current use (Group 2, steroid-sparing control group)
• Attendance of at least one baseline visit (V1) and one follow-up visit during the study period; and, the provision of capacitated and informed consent.

Exclusion Criteria:

• Inability to consent.
• Under 18 years of age.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients with autoimmune bullous disease not currently receiving glucocorticoids; Patients with autoimmune bullous disease who have received glucocorticoids for their condition in the past, but are not currently on steroids.
Patients with autoimmune bullous disease receiving glucocorticoids for the duration of the study; Patients with autoimmune bullous disease currently receiving glucocorticoids for their condition, as independently assessed by an appropriately qualified medical professional/dermatologist, for the duration of the study period.	Drug: GlucoCorticoid; Glucocorticoid as calculated in prednisone oral equivalents.
Patients with autoimmune bullous disease ceasing glucocorticoids during the study period; Patients with autoimmune bullous disease who initially had glucocorticoid treatment at the first visit, and had ceased glucocorticoid use during the study period.	Drug: GlucoCorticoid; Glucocorticoid as calculated in prednisone oral equivalents.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Glucocorticoid Toxicity Score	Glucocorticoid toxicity score as calculated by the glucocorticoid toxicity index (GTI) application licenced by Steritas. The final glucocorticoid toxicity index (GTI) score is comprised of two sub-scores. The GTI-cumulative worsening score (GTI-CWS) captures any additive glucocorticoid toxicities, transient or persistent, that are not present at baseline is scored from 0 to +439. The GTI-aggregate improvement score (GTI-AIS) assesses end-point toxicity comparative to baseline and is scored from -317 to +410; a negative score represents an improvement in toxicity, whilst a positive score indicates worsening toxicity.	2 years

Collaborators and Investigators

• Sponsor: Premier Specialists, Australia
• Collaborators: Steritas
• Investigators: Study Chair: Dedee Murrell, MBBS, University of New South Wales

Study record dates

Study Major Dates

• Study Start (Actual): February 2, 2019
• Primary Completion (Estimated): September 1, 2023
• Study Completion (Estimated): September 1, 2025

Study Registration Dates

• First Submitted: August 30, 2022
• First Submitted That Met QC Criteria: August 31, 2022
• First Posted (Actual): September 1, 2022

Study Record Updates

• Last Update Posted (Estimated): August 31, 2023
• Last Update Submitted That Met QC Criteria: August 28, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Skin Diseases, Vesiculobullous; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Glucocorticoids
• Other Study ID Numbers: ABDF-GTI2021

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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