Reproductive Capacity and Association to Iron Burden and Chelation Patterns in Thalassemia Major Patients
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 59
- 试验地点
- 2
- 主要终点
- Laboratory studies for pituitary and gonadal function and for iron level measures
研究概览
简要总结
The improved long-term survival of thalassemia major (TM) patients has resulted in increased focus on the ability to preserve fertility. While the association of iron toxicity with vital organ dysfunction, heart and liver, has been extensively investigated, the correlation of reproductive capacity and extent of iron overload is not well understood. Despite remarkable progress in methodology for prediction of reproductive status and intervention for preserving fertility, implementation in thalassemia is lacking.
The investigators hypothesize that iron toxicity to the anterior pituitary occurring in the process of transfusional iron loading is directly associated with a decline in gonadal function. The investigators expect pituitary MRI measurements of iron deposition as well as markers of oxidative damage to correlate with the functional studies of pituitary-gonadal axis performed in this study. This cross sectional study will examine the relation of pituitary iron deposition and pituitary volume; serum iron and oxidative stress measures, liver iron concentration (LIC), cardiac iron and chelation adequacy with pituitary and gonadal reproductive hormone levels (and spermatogenesis in adult male patients), in order to better define the association of iron burden and chelation patterns with fertility potential, in thalassemia patients with iron overload. The study will assess whether the current chelation treatment regimens, in particular during the pubertal developmental age, are adequate for preserving fertility and could lead to improved chelation routines for preventing the high prevalence of compromised fertility. In addition, by utilizing state-of-the-art markers for fertility status, findings from this study may improve current methods for screening for hypogonadism and reproductive potential and allow earlier intervention.
The investigators propose to examine 26-30 patients, 12 years and older, with measures of fertility potential, and correlate them to their current iron burden parameters and to the cumulative iron effect as indicated by past iron overload patterns and chelation history.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Transfusion-dependent* females and males with thalassemia (any genotype) who are 12 to 45 years of age.
- •History of at least 5 years of chronic transfusion (defined as ≥ 8 transfusions/year) (Age of initiation of transfusions does not matter)
- •Any pubertal stage.
- •Liver iron evaluated by SQUID, MRI or liver biopsy within 12 months prior to enrollment in the study.
- •Need to be able to stop hormonal therapy for 3 weeks (males) and one month (females) prior to study enrollment.
排除标准
- •Pregnant or lactating during study enrollment
- •Unable to obtain liver iron concentration within 12 months prior or 6 months after study entry.
结局指标
主要结局
Laboratory studies for pituitary and gonadal function and for iron level measures
时间窗: Up to 12 months
Each patient's pituitary iron and pituitary volume will be correlated with each hormone levels: LH, FSH, estrogen and AMH (Females), testosterone Inhibin B and (males). We will then evaluate potential associations using Pearson correlations. Univariate analyses on all variables and it's relation with iron, will also be conducted.
次要结局
- Retrospective analysis of annual LIC and of type and dose of iron chelation agent in the parallel time period(Up to 12 months)