Active clinical trials for "Anemia, Aplastic"

To date, allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes, haemoglobinopathies, bone marrow aplasia, severe combined immunodeficiency). Despite the major therapeutic progress made over the last 50 years, particularly in terms of supportive care, post-transplant morbidity and mortality remains high. Infectious complications, whose incidence varies between 30 and 60%, are the first cause of mortality in the immediate post-transplant period. In order to protect the patient from the occurrence of severe infectious episodes, aHSCTmust be performed in a highly protected environment (positive pressure chambers). This has implications for the experience and impact of hospitalization on the patient and family. This is particularly true in paediatrics, whether in children, adolescents or young adults, where it is not only the patient's quality of life that is at stake, but also their emotional and psychomotor development. In these patients, prolonged hospitalization (at least 6 weeks) in a sterile room will be responsible for physical deconditioning accompanied by a decrease in muscle mass, itself concomitant with undernutrition, and an increase in sedentary lifestyle. This prolonged hospitalisation in a sterile room, associated with myeloablative treatments, is therefore the cause of social isolation of patients, but it is also often synonymous with physical inactivity leading to a rapid decrease in physical condition, quality of life and an increase in fatigue. Today, the benefits of physical activity (PA) during and after cancer treatment have been widely demonstrated. The objective is to evaluate the feasibility of an adapted physical activity program during the isolation phase for achieving aHSCT in children, adolescents and young adults. This is a prospective, interventional, monocentric cohort study conducted at the Institute of Paediatric Haematology and Oncology in Lyon. The intervention will take place during the isolation phase and consists of an adapted physical activity (APA) program defined at inclusion, integrating supervised sessions with an APA teacher, as well as autonomous sessions. The program is individualized according to age, aerobic capacity, and PA preferences. Sessions are also tailored to the biological, psychological, and social parameters of patients. The total duration of the intervention is 3 months. To date, no PA studies have been performed in patients under 21 years of age requiring aGCSH during the sterile isolation phase. EVAADE will therefore be the first study in this population to offer an innovative procedure with a connected device.

Background: - Some treatments for cancer or other diseases can lead to infertility in women. These treatments include chemotherapy, some stem cell transplants, and pelvic radiotherapy. They are called gonadotoxic therapies. Women can now have their eggs frozen before they have these treatments. This may allow them to get pregnant later. Researchers want to learn more about this technology and processes. Objectives: - To provide egg freezing for women having gonadotoxic therapies at NIH. To learn more about the effects of these therapies. Eligibility: - Women at least 18 years old who are past puberty and before menopause. They must be scheduled to have gonadotoxic therapies. Design: Participants will be screened with medical history and blood and hormone tests. They will also have a physical exam and transvaginal ultrasound. Ovary stimulation: participants will have medications injected under the skin. These increase the chance of fertility. This phase will take about 8 20 days. Participants will have blood drawn and transvaginal ultrasound daily or every other day. Some participants will also have blood thinner injected daily. Egg retrieval: participants will check in to the hospital. Eggs will be removed with a needle during a short surgery. Participants will be awake but sedated. Participants may stay overnight in the hospital. They will return every 1 3 days for 1 3 weeks for blood tests. Mature eggs will be frozen after egg retrieval and immature eggs (which cannot be fertilized for clinical use) will be used for research. Participants can use their eggs in the future at outside, private fertility clinics to try to become pregnant. If the eggs are stored for more than 5 years, participants must pay for storage.

Background: Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for SAA. One is allogeneic hematopoietic stem cell transplant (HSCT). The other is immunosuppressive treatment (IST). In most cases, HSCT or IST works. But for some people, clonal evolution occurs after IST. One of the most common forms of clonal evolution is chromosome 7 abnormalities. These have a poor prognosis. HSCT can be used to treat them. Researchers do not know why clonal evolution happens. They want to look at data from past studies to learn more. Objective: To compare the data of people with SAA who developed chromosome 7 abnormalities between those who ultimately received HSCT versus those who received chemotherapy alone or supportive care. Eligibility: Adults and children with SAA who were enrolled on NHLBI protocol 12-H-0150, 06-H-0034, 03-H-0249, 03-H-0193, 00-H-0032, or 90-H-0146 Design: This study uses data from past studies. The participants in those studies have allowed their data to be used in future research. Researchers will review participants medical records. They will collect clinical data, such as notes, test results, and imaging scans. They will also collect the research data gathered as part of the original study. Researchers will enter the data into an in-house database. It is password protected. All data will be kept in secure network drives or in sites that comply with NIH security rules. Other studies may be added in the future.

RATIONALE: Gathering information about how often methemoglobinemia occurs in young patients receiving dapsone for hematologic cancer or aplastic anemia may help doctors learn more about the disease and plan the best treatment. PURPOSE: This research study is looking at methemoglobinemia in young patients with hematologic cancer or aplastic anemia treated with dapsone.

This study will collect samples of blood, stool, bone marrow, or other tissues from patients with hepatitis-associated aplastic anemia to investigate a possible association between exposure to viruses and the development of aplastic anemia in these patients. Cells from the samples obtained may be grown in the laboratory for future studies. Patients samples may be used to: Study abnormalities that occur in hepatitis-associated aplastic anemia; Test for various viruses; Test immune function; Measure factors related to the patients disease or diseases they may be at risk for; Evaluate the effectiveness of current therapies, refine treatment approaches, and identify potential new therapies; Identify possible measures for disease prevention; Identify possible genetic factors associated with hepatitis-associated aplastic anemia. Patients 2 years of age and older with severe aplastic anemia that developed within 6 months of an episode of hepatitis may be eligible for this study. Participants will complete questionnaires and provide tissue samples as described below. Questionnaires All patients (or another respondent for the patient) will fill out a questionnaire including demographic information (age, gender, race, ethnic group, education level, state of residence), current symptoms, medications, medical history, and history of possible exposures to toxins or viruses. A second questionnaire, which includes questions related to mental health, sexual behavior, alcohol and drug use, is optional for participants age 21 and older. These questionnaires are designed to uncover features of hepatitis-associated aplastic anemia and possibly reveal a common cause of the disease. Sample Collections Blood- will be collected at the time of the patient s initial evaluation or upon enrollment into the study and possibly periodically during the study. Blood will be drawn through a needle in an arm vein. Bone marrow- may be collected as part of the patient s standard medical care or specifically for research purposes of this study. For this procedure, the skin over the hipbone and the outer surface of the bone itself are numbed with an injection of a local anesthesia. Then, a larger needle is inserted into the hipbone and marrow is drawn into a syringe. Marrow cells are suctioned two to six times during the 15-minute procedure. Stool- will be provided by the patient. Liver- tissue may be biopsied as part of the patient s general medical care or for NIH patients, as part of their enrollment in a treatment protocol.

RATIONALE: Infection prophylaxis and management may help prevent cytomegalovirus (CMV) infection caused by a stem cell transplant. PURPOSE:This clinical trial studies infection prophylaxis and management in treating cytomegalovirus infection in patients with hematologic malignancies previously treated with donor stem cell transplant.

This study will use neuropsychological tests to look at nervous system side effects of Cyclosporine (CsA) in patients with aplastic anemia. CsA is used as part of an immunosuppressive regimen in treating severe aplastic anemia. The drug can produce nervous system side effects, such as tremor and, less commonly, insomnia, anxiety, headache, confusion or seizures. This study will look at effects of CsA on intellectual ability, depression, anxiety, attention, concentration, memory, perception, coordination, and thought processing in patients Patients 15 years of age or older who have severe aplastic anemia may be eligible for this study if they: are co-enrolled in a Clinical Center protocol in which they will receive CsA have not taken CsA for 6 months before enrolling in this study Participants undergo neuropsychological testing. In addition, they provide blood samples and their clinical data are reviewed for things that may influence the interpretation of findings from the testing, such as results of blood tests, types of medications taken, number of transfusions required, etc. The procedures are as follows: Before first dose of cyclosporine: Patients are asked about prior problems with their nervous system, prior treatment for their aplastic anemia (including transfusions), prior infections, and current medications. They then complete the following sets of tests: Battery 1: A set of three tests that measure intellectual ability, level of depression (if any) and level of anxiety (if any). Battery 2: A set of seven tests that measure changes in the central nervous system and how these changes affect attention, concentration, memory, perception, coordination, and thought processing. Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol. 6 months and 12 months after starting cyclosporine Patients are asked about treatment for their aplastic anemia (including transfusions), infections, and changes in medications that have occurred since they started taking cyclosporine. They then repeat the set of tests in Battery 2. Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol.

To quantify the role of drugs and other factors in the etiology of agranulocytosis and aplastic anemia.

To investigate the efficacy of posaconazole as prophylaxis antifungal agent in aplastic anemia / hypoplastic myelodysplastic syndrome (AA/hMDS) patients undergoing antithymocyte globulin (ATG) treatment

Background: Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for most people with SAA. One is allogeneic hematopoietic stem cell transplant (HSCT). The other is immunosuppressive treatment (IST). For people who are treated with IST, relapse can occur. If this happens, they can have HSCT or be re-treated with IST. The two most common IST regimes used for relapsed SAA are rabbit ATG (rATG) and alemtuzumab. Both rATG and alemtuzumab have similar response rates and survival rates. There is not much long-term data on people who need repeat IST treatment due to relapse. Researchers want to look at data from past studies to learn more. Objective: To compare the data of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST treatment. Eligibility: Adults and children with SAA who were enrolled on NHLBI protocol 12-H-0150, 06-H-0034, 05-H-0242, 03-H-0249, 03-H-0193, 00-H-0032, or 90-H-0146 Design: This study uses data from past studies. The participants in those studies have allowed their data to be used in future research. Researchers will review participants medical records. They will collect clinical data, such as notes, test results, and imaging scans. They will also collect the research data gathered as part of the original study. Researchers will enter the data into an in-house database. It is password protected. All data will be kept in secure network drives or in secure sites. Other studies may be added in the future....