View clinical trials related to Hemosiderosis.
Filter by:Patients with hemochromatosis or Thalassemia develop progressive tissue and organs damages secondary to iron overload. Iron overload can result both from transfusional hemosiderosis and excess gastrointestinal iron absorption. Iron deposition in the heart, liver, and multiple endocrine glands results in severe damage to these organs, with variable degrees of endocrine and organ failure. Although patients with iron overload often present endocrine disorders, the pathogenetic mechanisms underlying endocrinopathies are not completely clear. In particular it is not elucidated if the spectrum of endocrinopathies could change with advancing age. All endocrinological comorbidities can develop from a primary damage of the target gland, from pituitary secondary failure or from both. The aim of this study is to investigate the prevalence of endocrinological diseases in adult patients with iron overload due to β-thalassemia or hemochromatosis and their impact on well-being and quality of life. The study design is a prospective cross-sectional clinical study. All subjects enrolled will be evaluated for the endocrine diseases. The study protocol will include data collection from family and patients' history of diseases, physical examination, hormonal assessment for all endocrine axes and instrumental examinations. The results will provide evidence on the prevalence of endocrine diseases in patients with iron overload and will add information to characterize the type and the degree of endocrine deficiencies, and on the pathogenic mechanisms involved, in order to individualize diagnostic and therapeutic approaches.
The goal of this clinical trial is to compare Leflunomide in Idiopathic pulmonary hemosiderosis. The main questions it aims to answer are: - The efficacy and safety of Leflunomide in Idiopathic pulmonary hemosiderosis - The mechanism of leflunomide in treating Idiopathic pulmonary hemosiderosis Participants will be treated with leflunomide plus stroid. A comparison group: Researchers will compare the control group treated with stroid to see if the efficacy of Leflunomide would be better than control group.
Subjects were tested for hemoglobin, ferritin, serum iron, transferrin saturation and reticulocyte count during routine prenatal examination at 24-26 weeks of gestation, and blood samples were taken for serum hepcidin detection in the laboratory and the values were recorded. Those who met the criteria were included in the study group, signed the informed consent form and randomized into groups, and were given different drug administration schemes (150mg orally every day, 300mg orally every day, 150mg orally every other day, 300mg orally every other day, intravenous). At the same time, each subject was given anemia diet education, and all subjects were given folic acid 400ug/d and vitamin C 0.5g/d orally during the treatment period. If the subjects were in the oral iron group, the same time of oral iron was determined as 20 o'clock ± 1 hour in the evening, and the oral iron was not taken with other drugs; If the subject is in the intravenous medication group, the medication is scheduled to be administered at a uniform time of 8 o'clock ± 1 hour in the morning. The above subjects were followed up. Hemoglobin, ferritin, serum iron, transferrin saturation and reticulocyte count were performed at 30-32 and 37 weeks of pregnancy and delivery, and blood samples were taken for serum hepcidin detection in the laboratory and the values were recorded. The adverse reactions were investigated with a questionnaire at the last prenatal examination before delivery. After full term delivery, the patient fills in the delivery information and enters it into the database. Finally, the data statistician and the above personnel used the blind method for statistical analysis and reached a conclusion.
Patients in maintenance treatment for HFE hemochromatosis since at least one year will be included in a two year study period and randomized in two groups experimental and control group. Because proton pump inhibitors are widely used as chronic medication, and because they can significantly modify iron absorption, patients will be stratified according to the use of proton pump inhibitors and gender. A first bloodletting will be performed at inclusion with the same volume as usually performed by the patients. Results of the biological test performed at this visit will guide the time schedule and volume of the next bloodletting according to randomization group (patients treated with bloodletting according to current guidelines "ferritin alone" versus patients treated with bloodletting according to "transferrin saturation and serum ferritin").Blood count and iron metabolism parameters will be performed at each bloodletting and follow-up visits. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit. Volume and schedule for bloodlettings will be determined according to guidelines specifically designed for this study to assure harmonization of treatment management, and centrally validated through the recording of the biological tests in the electronic Case Report Form which will provide the investigator with the volume and schedule of the next bloodletting. There will be two ways of treatment modification: either change of schedule or volume of bloodletting. Patients will undergo follow-up visit every six months with clinical examination, questionnaires at J0, M12 and M24 (SF-36; AIMS2-SF, WOMAC, EQ-5D-5L), and biological test. For health economics analysis, data will be obtained thanks to a dedicated extraction from SNDS database SNDS database will allow to gather hospital stays, visits, and other healthcare-related costs as well as vital status (date (month/year) of death) and cause of death. A de-identified copy of the clinical database, restricted to the relevant variables, will be sent for semideterministic matching purpose with SNDS extraction using four key variables: gender, same date (month/year) of birth, same date (day/month/year) of visit for bloodletting; pending, of course, regulatory authorization.
Detect the PHOX2B Mutation-confirmed congenital central hypoventilation syndrome