Turner And Klinefelter Treatment Target Study. Improving Endocrine Treatment by Linking Biochemical Parameters to Morbidity and Quality of Life in Patients With Turner and Klinefelter Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 370
- 试验地点
- 1
- 主要终点
- The relationship between thyroid hormone status* and QoL as measured by the EQ-5D-5L in patients with TS.
研究概览
简要总结
Rationale: Health related Quality of life (HRQoL) is impaired in patients with Turner and Klinefelter syndrome (TS and KS). It is unknown what the optimal endocrine treatment target values are that maximize HRQoL in patients with these syndromes. Therefore the relation between HRQoL and biochemical parameters will be studied in large cohorts of patients with TS and KS. This information will give essential insight that will help to improve endocrine treatment and HRQoL in these patients.
Research objectives: To explore the relationship between biochemical parameters and HRQoL in patients with TS and KS.
Hypothesis: Biochemical parameters are related to HRQoL in patients with TS and KS.
Study design: Cross-sectional, observational, multicentre study Study population: Patients with KS or TS, 18 years or older Methods and procedures: To measure fatigue the Checklist Individual Strength (CIS-20) will be used, for QoL the 5-level EQ-5D (EQ-5D-L5) will be used and for stress the Perceived Stress Scale (PSS) and hair cortisol levels. For patients with KS the anxiety scale from the Liebowitz social anxiety scale (LSAS) will be used to measure social anxiety. To measure the long-term exposure to testosterone in KS patients, testosterone concentrations in hair will be measured. For patients with KS, all questions from the questionnaires will be discussed orally during a visit to the outpatients clinic. One extra tube of blood and a strand of hair will be collected during routine blood withdrawal. All other variables are already part of the standard patient care and are available in patient records. For patients with TS all information including the questionnaires and laboratory values is already available and will be collected from clinical records.
Main study parameters/endpoints: The relationship between different hormonal parameters and HRQoL as measured by questionnaires. The main hormonal parameter that will be investigated in KS is testosterone in hair. For patients with Turner syndrome, free thyroxine (FT4), thyroid stimulating hormone (TSH) and liver enzymes, which have already been collected, will be investigated. The relationships between the EQ-5D-L5 score and testosterone in hair (in patients with KS) and thyroid hormone status (in patients with TS) are the primary outcomes.
详细描述
Rationale: Health related Quality of life (HRQoL) is impaired in patients with Turner and Klinefelter syndrome (TS and KS). It is unknown what the optimal endocrine treatment target values are that maximize HRQoL in patients with these syndromes. Therefore the relation between HRQoL and biochemical parameters will be studied in large cohorts of patients with TS and KS. This information will give essential insight that will help to improve endocrine treatment and HRQoL in these patients.
Research objectives: To explore the relationship between biochemical parameters and HRQoL in patients with TS and KS.
Hypothesis: Biochemical parameters are related to HRQoL in patients with TS and KS.
Study design: Cross-sectional, observational, multicentre study
Study population: Patients with KS or TS, 18 years or older Methods and procedures: To measure fatigue the Checklist Individual Strength (CIS-20) will be used, for QoL the 5-level EQ-5D (EQ-5D-L5) will be used and for stress the Perceived Stress Scale (PSS) and hair cortisol levels. For patients with KS the anxiety scale from the Liebowitz social anxiety scale (LSAS) will be used to measure social anxiety. To measure the long-term exposure to testosterone in KS patients, testosterone concentrations in hair will be measured. For patients with KS, all questions from the questionnaires will be discussed orally during a visit to the outpatients clinic. One extra tube of blood and a strand of hair will be collected during routine blood withdrawal. All other variables are already part of the standard patient care and are available in patient records. For patients with TS all information including the questionnaires and laboratory values is already available and will be collected from clinical records.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Klinefelter or Turner syndrome as confirmed by genetic testing
- •Sufficient knowledge of the Dutch language to complete the questionnaires
- •At least 18 years old
排除标准
- •KS: Patients not under treatment in the EMC, AMC or VUmc or no planned visits during the study period
- •TS: No laboratory values or no questionnaires available in patient records
- •Severe psychiatric or neurologic disorders or other reasons for inability to complete the questionnaires as assessed by the treating physician.
- •Failure to obtain informed consent
结局指标
主要结局
The relationship between thyroid hormone status* and QoL as measured by the EQ-5D-5L in patients with TS.
时间窗: At study entry (cross-sectional study). There is just one visit per patient. The total study has an approximately duration of 3-4 years.
thyroid hormone status is a variable that consists of the following 6 categories: * Overt hyperthyroidism (FT4\>25pmol/L and TSH\<0,4mU/L) * Overt hypothyroidism (FT4 \<11 pmol/L and TSH \>4,3 mU/L) * Subclinical hypothyroidism with TSH \<10mU/L (FT4 11-25 pmol/L and TSH 4,3-10mU/L) * Subclinical hypothyroidism with TSH \>10mU/L (FT4 11-25 pmol/L and TSH \> 10mU/L) * Subclinical hyperthyroidism (FT4 11-25 pmol/L and TSH \<0,4 mU/L) * Euthyroidism (FT4 11-25 pmol/L and TSH 0,4- 4,3 mU/L) The range of the EQ-5D-5L is from 0 to 100. A higher score means a better outcome.
The relationship between testosterone concentrations in hair and QoL as measured by the EQ-5D-5L in patients with KS.
时间窗: At study entry (cross-sectional study). There is just one visit per patient. The total study has an approximately duration of 3-4 years.
The range of the EQ-5D-5L is from 0 to 100. A higher score means a better outcome.
次要结局
未报告次要终点
研究者
dr. Laura C. G. de Graaff-Herder
Principal investigator
Erasmus Medical Center