TRANSLATIONAL MEDICINE

taking discoveries to patients’ benefit

Progress Report until 25th Nov 2019

Clinical scientific supervisor: Emese Mezősi Educational supervisor: János Garai Scientific methodology supervisor: Andrea Párniczky, Zsolt Szakács

Vera Izsák 12-month fellowship program

Overview

1. Network meta-analysis of Cystic fibrosis - related diabetes screening methods Project started: October 2019

2. Registry: Graves’-Basedow disease Project started: Sept 2019

Screening methods for Cystic Fibrosis-related diabetes - a network meta-analysis

Senior invetigator: Andrea Parniczky

Junior investigator(s): Vera Izsák

Introduction

Cystic fibrosis (CF)

• Multisystem

• Autosomal recessive disease

• CFTR gene mutation

• Advances in treatment

increased survival rate

• Common comorbidites

• Particular form of diabetes

• Diminished lung function,

Poor nutritional status,

Increased mortality,

• Early management - improved

outcomes.

• Gold standard – Oral glucose

tolerance test (OGTT)

• CF ≥10 years of age

Cystic fibrosis related

diabetes (CFRD)

Evaluate the current diagnostic methods according to their diagnostic metrics.

Goal

Clinical Question

Population Patients with cystic fibrosis

Index test

Outcome

OGTT

Diagnostic metrics

Reference standard

Other screening methods: • 50-g/75-g glucose challenge test, with 60-minute glucose level

• 50 g glucose challenge with 120 min glucose value • Fasting plasma glucose

• Random blood glucose levels

• HbA1c

• Serial capillary blood glucose profiling

• Fructosamine • Urine glucose tests

• Continuous glucose monitoring system

Search

• Databases:

Pubmed, Embase, Scopus, Web of Science, Cohrane library

• Search key:

(„cystic fibrosis” OR cftr OR mucoviscidosis ) AND (diabet* OR prediabet* OR „impaired glucose tolerance” OR IGT OR „impaired fasting glucose” OR IFG OR indeterminate glucose tolerance OR INDET)

• Filters: no filter

• Date of search: 09.10.2019

Selection

10840

• Number of records in each database:

• Pubmed: 1934; Embase: 3177; Scopus: 3510 Web of Science: 2069 Cohrane:157;

5686 • Records after removing duplicates/database overlaps

787 • Records after screening by title

598 • Records after screening by abstract

Future plans

PROSPERO

registration

Statistical

analysis

Publication

Graves’ - Basedow disease registry

Clinical supervisor: Emese Mezősi Junior investigator(s): Vera Izsák

Introduction

Autoimmune disease

Most common cause of

hyperthyroidism

Prevalence of 1–1.5%.

Mortality

Primary

’ ’

Goals

Monitor

Prevalence

Incidence

Children and Adult

Describe

Predictive value of

symptoms and laboratory

tests

Prognostic factors

Assess

Effectiveness of the

applied treament on

symptoms and quality

of life

Nationwide International

Secondary

Timeline & Forms

Admission to

hospital

Medical history, symptoms

Diagnosis Medication

Follow – up visit Therapy

Follow-up and

Therapy strategy

FORM C

FORM A FORM B

Symptoms

Laboratory results

Radiological imaging

Details from the medical

history Family history

Relevant medications

FORM E

FORM D

Any significant changes:

In symptoms

Laboratory results

In medical history

In treatment In medication

Outcome

Radioiodine

therapy Surgery

Antithyroid drug

Response to

treatment

Adverse events

Response to

treatment

Adverse events

Preoperative

Intraoperative

Postoperative

Histopathology

Future plans

Enrollment

of patients

Ethical permission

in progress

Data

analysis

Summary

1. Netwok meta-analysis: data collection is in progress Expected submission: January 2020

2. Registry: protocol has been written Expected submission: January 2020

TRANSLATIONAL MEDICINE

taking discoveries to patients’ benefit

Thank you for your attention!