This trial is evaluating whether Mycophenolate mofetil (MMF) will improve 2 primary outcomes and 3 secondary outcomes in patients with Syndrome. Measurement will happen over the course of +1, +2 and +3 years post HSCT.
This trial requires 37 total participants across 2 different treatment groups
This trial involves 2 different treatments. Mycophenolate Mofetil (MMF) is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
"The concept of syndromes is an important one and is crucial for understanding how different medical conditions can be associated. This article provides a definition of the term syndrome and discusses how the term relates to other medical conditions and approaches." - Anonymous Online Contributor
"There is no standard treatment for this group of conditions. Specific treatment for each condition will vary depending on the specific presentation and the severity of the symptoms. In all cases, the goal of therapy is to treat symptoms that are not as part of the syndrome. The clinician will have to assess the individual needs of the person with the syndrome." - Anonymous Online Contributor
"The prevalence of syndrome is low in US population. However, it is not uncommon to see a patient in each day. We need to understand the cause, diagnosis and treatment of syndrome, and to discover the unknown genes that may influence the disease. Clinical trial is one strategy for finding a cure for syndrome." - Anonymous Online Contributor
"Syndromes are medical symptoms combined and united to form a specific syndrome or group of related syndromes. The diagnostic feature of a syndrome is the presence of an identifiable, consistent set of signs and symptoms that characterize the syndrome rather than the diagnosis of any particular disease. For example, most people with RHD have the specific condition, sickle cell disease, but only some people know they have the disease. The syndrome of RHD includes the condition of sickle cell disease, but also includes many other conditions, such as malaria, which may also result in the disorder of sickle cell disease." - Anonymous Online Contributor
"The criteria for the diagnosis of syndrome are quite vague. However we suggested that the characteristics of the disorder may not always be seen and that there are many signs of syndrome, which can explain the presentation of this syndrome." - Anonymous Online Contributor
"Symptom resolution for some individuals may be an indication a syndrome may be partially, but not completely, curable. Patients who have incomplete, fluctuating symptom resolution in response to conventional therapy for the disease may develop an intolerance to the drugs or may suffer adverse reactions to the drug(s). Symptom improvement in response to conventional treatment of the person's condition does not guarantee, by itself, a cure for the person's syndrome." - Anonymous Online Contributor
"Syndromes are best understood under the paradigm that we need to understand the underlying etiological mechanisms of a syndrome in order to be able to describe it, understand the clinical consequences of the syndrome, and to develop appropriate treatment strategies. In the case of the syndrome of late-onset depression, the underlying mechanism of the depression could be related to problems in sleep, appetite, or the hypothalamic-pituitary-adrenal axis. These may also be part of the mechanism of bipolar disorder. In contrast, it seems that major depression is related to changes in the neurotransmitter system." - Anonymous Online Contributor
"This review supports the recent notion that a patient-specific individualised plan should be developed in the context of the patient's medical circumstances. The use of sirolimus may benefit patients with an increased risk of toxicity (e.g. elderly, transplant recipients, diabetic) in a population where the potential for increased efficacy is equally important." - Anonymous Online Contributor
"We calculated the age that a disorder becomes symptomatic to be 5 years. In a recent study, findings provide a benchmark for disease-onset in children and adolescents. We hypothesize an association between disease-onset and a gene by environment interaction." - Anonymous Online Contributor
"Mild GI distress and dermatologic toxicity occurred in most patients receiving sirolimus. Skin reactions, particularly erythema and itching, were the most common, and were more frequent with higher blood levels of sirolimus. Sirolimus was also associated with exacerbation of existing diabetes." - Anonymous Online Contributor
"Combining mTORin with other treatment modalities seems to be more effective than mTORin alone. Although other drugs may be used alternatively by some patients, it may be beneficial to consider mTORin in combination with other treatments. The data from this review may be of relevance in the discussion about which treatments might be offered to patients with SLE." - Anonymous Online Contributor
"After 4 years of sirolimus the most common adverse events (in more than 1/2 of patients) were leukopenia (<35 x 10(6) leukocytes per microL) and/or thrombocytopenia (<100 x 10(6) platelets per microL). The most serious adverse events (in less than 1/2 of patients) were: pneumonitis, opportunistic infection/bacterial (in 0.6% patients), leukomorphia, myocardial infarction (MI) and/or congestive heart failure (CHF) (0.4%)." - Anonymous Online Contributor