Dogan Simsek

Comparison of the efficacy of once- and twice-daily colchicine dosage in pediatric patients with familial Mediterranean fever – a randomized controlled noninferiority trial

BackgroundIn this study, we examined the efficacy and safety of a once-daily dosage schema of colchicine compared with a twice-daily dosage schema in pediatric patients with familial Mediterranean fever (FMF).MethodsIn this 24-week, multicenter, randomized controlled noninferiority trial, pediatric patients newly diagnosed with FMF carrying a homozygous or compound heterozygous mutation and not receiving any treatment were included. Patients were randomly assigned using a block randomization method to receive treatment with a once- or twice-daily dosage. Clinical and laboratory characteristics and medication side effects were recorded and compared between groups. The study was carried out in compliance with Good Clinical Practice and the Consolidated Standards for Reporting of Trials (CONSORT) statement.ResultsA total of 92 patients were selected, and 79 patients completed the study. There were 42 patients in the once-daily dosage group and 37 in the twice-daily dosage group. The results indicated that the once-daily dosage was not inferior to the twice-daily dosage regarding decrease in attack frequency and duration as well as improvement in clinical findings and Mor severity scores. Alterations in laboratory findings indicating inflammation, such as erythrocyte sedimentation rate, C-reactive protein, and serum amyloid A, were similar in both groups. The rates of drug side effects were similar between the once- and twice-daily dosage groups, implying comparable safety of colchicine, with the exception of diarrhea, which was slightly higher in the once-daily dosage group.ConclusionsUsing colchicine with either a once- or twice-daily dosage provides similar clinical and laboratory improvements. Considering both efficacy and safety, colchicine can be prescribed with a once-daily dosage.Trial Registration IDClinicalTrials.gov identifier NCT02602028. Registered 5 November 2015.

Brief Report: Deficiency of Complement 1r Subcomponent in Early-Onset Systemic Lupus Erythematosus: The Role of Disease-Modifying Alleles in a Monogenic Disease: ROLE OF C1r DEFICIENCY IN EARLY-ONSET SLE

To identify a genetic cause of early‐onset systemic lupus erythematosus (SLE) in a large consanguineous family from Turkey and to study the mechanisms of the disease.

Current Research in Outcome Measures for Pediatric Rheumatic and Autoinflammatory Diseases

A rational management of children and adolescents with rheumatic and autoinflammatory diseases requires the regular assessment of the level of disease activity and of child health and well-being through the use of well-validated outcome measures. Ideally, such instruments should be simple and feasible and easily applicable in standard clinical practice. In recent years, a number of novel outcome measures have been developed and validated for use in pediatric patients with rheumatic and autoinflammatory illnesses. Furthermore, there has been an increased focus on the appraisal of child and parent perception of the disease impact. The new tools have markedly enlarged the spectrum of disorders and health domains that can be assessed in a standardized way. This progress will help to enhance the reliability of research studies and clinical trials. The aim of the present review is to provide an update of the recent advances in this field of research.

Deficiency of Complement 1r Subcomponent in Early‐Onset Systemic Lupus Erythematosus: The Role of Disease‐Modifying Alleles in a Monogenic Disease

To identify a genetic cause of early‐onset systemic lupus erythematosus (SLE) in a large consanguineous family from Turkey and to study the mechanisms of the disease.

Validity and reliability of medication adherence scale in FMF (adult version)

MASIF (Medication Adherence Scale in FMF) is an instrument designed to measure adherence to treatment in children with Familial Mediterranean Fever (FMF). We have developed this scale for children with FMF and found valid and reliable.

The Turkish Version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR)

The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Turkish language. The reading comprehension of the questionnaire was tested in 10 JIA parents and patients. Each participating centre was asked to collect demographic, clinical data and the JAMAR in 100 consecutive JIA patients or all consecutive patients seen in a 6-month period and to administer the JAMAR to 100 healthy children and their parents. The statistical validation phase explored descriptive statistics and the psychometric issues of the JAMAR: the 3 Likert assumptions, floor/ceiling effects, internal consistency, Cronbach’s alpha, interscale correlations, test–retest reliability, and construct validity (convergent and discriminant validity). A total of 466 JIA patients (13.7% systemic, 40.6% oligoarticular, 22.5% RF negative poly-arthritis, and 23.2% other categories) and 93 healthy children were enrolled in four centres. The JAMAR components discriminated well-healthy subjects from JIA patients. All JAMAR components revealed good psychometric performances. In conclusion, the Turkish version of the JAMAR is a valid tool for the assessment of children with JIA and is suitable for use both in routine clinical practice and clinical research.

Deficiency of Complement 1r subcomponent in early-onset SLE: Role for disease-modifying alleles in a monogenic disease

To identify a genetic cause of early‐onset systemic lupus erythematosus (SLE) in a large consanguineous family from Turkey and to study the mechanisms of the disease.

Validity and reliability of medication adherence scale in FMF

Objective The optimal level of adherence necessary to achieve acceptable disease and quality-of-life outcomes for patients is not known. In order to identify these optimal levels, we need reliable and valid measures of adherence. Medication Adherence Scale in FMF (MASIF) is an instrument designed to measure adherence to treatment in children with Familial Mediterranean Fever (FMF). We have developed this scale for children with FMF and found valid and reliable. In this study, it was aimed to assess the validity and reliability of this adherence scale for medical treatment in adult FMF patients.

AB1118 Validity and Reliability of Medication Adherence Scale in FMF (Adult Version)

Background The optimal level of adherence necessary to achieve acceptable disease and quality-of-life outcomes for patients is not known. In order to identify these optimal levels, we need reliable and valid measures of adherence. Medication Adherence Scale in FMF (MASIF) is an instrument designed to measure adherence to treatment in children with FMF. We have developed this scale for children with FMF and found valid and reliable (1). Objectives It was aimed to assess the validity and reliability of this adherence scale for medical treatment in adult FMF patients. Methods This study is multicentre and 14 centers participated to the study. Patients with FMF using medication at least for 6 months and accepted to participate constituted the sample of the study. Besides “Medication Adherence Scale in FMF Patients (MASIF)”, “Data collection forms about the sociodemographic and medical information (demographic, clinical and laboratory findings) of patients”, was used as data collection instruments. Morisky medication adherence scale was used as a gold standard in order to evaluate the criterion validity of MASIF. We assessed the validity of the adult version of the MASIF using the OMERACT filter. Results The median age of the patients (n=133) was 28.60 years (min.18.12-max.71.34) and 52.6% of them were female. For internal consistency, Cronbachs alpha was 0,764 for MASIF adult version. Also, there was a positive and significant correlation between test and retest score (t=0.971; p=0.340). For the “criterion validity” the correlation with Morisky and MASIF was evaluated (r=0.530, p=0.000) and for the “structure” validity, factor analyzes and Kaiser-Meyer-Olkin tests were performed. After these tests, MASIF was found as a valid and reliable instrument. MASIF consists of 18 items and 4 sub-dimensions: knowledge about the medication, adherence to the treatment, barriers to drug use, factors that may increase compliance. The total score ranged from 18 to 90. A high score showed a good adherence to treatment. The cut-off point was determined as 55 points. A point over 55 was accepted as “good medication adherence” and a point less than 55 was considered as “bad medication adherence” Conclusions Approximately 10-15% of patients with FMF are non-responders but it was claimed that in fact they are non-compliers that causes these patients receive unnecessary biologic agent treatment procedures, which are expensive and have some serious adverse effects. This scale will provide assessment and follow up of adherence to treatment patients and determine whether the patient is non-responders or non-compliers. It may help to determine the non-compliance and prevent unnecessary and expensive biologic agents. Disclosure of Interest None declared