This trial is evaluating whether Inhaled Treprostinil will improve 2 primary outcomes and 5 secondary outcomes in patients with Hypertension, Pulmonary. Measurement will happen over the course of Baseline, Week 16.
This trial requires 10 total participants across 2 different treatment groups
This trial involves 2 different treatments. Inhaled Treprostinil is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
Immunosuppression and corticosteroids are the standard therapy for sarcoidosis. Immunosuppression is used either as a single agent or in combination with other medications. Corticosteroids (and sometimes an immunosuppressive drug such as cyclosporine or azathioprine) are used as monotherapy and in combination with immunosuppressive therapy. There are no randomized controlled trials for the treatment of sarcoidosis.
Sarcoidosis can be cured. This is possible if the treatment is started early in the process and continued for many years. Sarcoidosis can be effectively treated and managed with good quality of life as good as that obtained in unaffected relatives.
Sarcoidosis is probably diagnosed at an early stage. A high index of suspicion is necessary when patients present with tuberculosis, pulmonary edema, respiratory disturbances, unexplained fever lasting for longer than 2 weeks, unexplained weight loss, adenopathy, or pulmonary function impairment. An occupational history of tuberculosis, exposure to hard sand, and abnormal chest X-ray have a high sensitivity for detecting sarcoidosis. The chest X-ray must be reviewed and a history taken by the doctor who makes the diagnosis. The most common symptom of sarcoidosis is a lung examination in which lung nodules may be present and in some cases, they may be bilateral.
Exposure to certain environmental substances, such as mycobacterium, might be a factor influencing susceptibility to Sarcoidosis. A more in-depth knowledge of the pathogenesis of Sarcoidosis is expected to allow a better understanding of the complex etiology of the disease.
Sarcoidosis is a disease of unknown cause with multiple etiologies; the most common form in Caucasian adults is systemic sclerosis, secondary infection and exposure to inhaled xenobiotics being the most frequent predisposing factors. Sarcoidosis can present with various non-specific clinical manifestations including pulmonary nodules and hilar adenopathy in which the condition can also be confused with tuberculosis. The diagnosis is often confirmed by pulmonary biopsy. Non-specific findings are frequently encountered in sarcoidosis and should not lead to diagnosis of the disease.
Although sarcoidosis is fairly common—it occurs in approximately one person in every 1,600—the number of people with the condition who ever receive medical attention for sarcoidosis is low. If sarcoidosis was to be treated on the basis of current practices and if physicians and patients were more appropriately warned about the limitations of available treatments, many people with sarcoidosis would be misdiagnosed. The use of a high throughput screening approach to sarcoidosis, however, is warranted.
The majority of patients taking inhaled treprostinil, reported no additional benefits for other treatment options. A minority reported worsening of their symptoms. These preliminary data suggest the use of inhaled treprostinil is not always combined with other therapy; the efficacy of treprostinil on pulmonary disease remains questionable, however, and warrants further evaluation.
The study demonstrates that treprostinil was effective in treating and improving symptoms of severe respiratory failure and hypoxia. The drug is well tolerated and warrants further investigation as a treatment for other forms of severe respiratory disease.
Results from a recent paper indicate that sarcoidosis is associated with a high prevalence of familial inheritance. A large multicenter study is needed to confirm these findings and determine the clinical relevance of sarcoidosis in a multi-ethnic population.
Inhaled treprostinil is a novel drug which may have a significant impact on the management of pulmonary arterial hypertension and other cardiovascular diseases associated with sarcoidosis.
Symptoms may be present as early as age 15 years or as late as 44 years. In the US, sarcoidosis is most common between the ages of 34-41. It occurs more commonly in women in all age groups: women are 4 to 6 times more likely to get sarcoidosis compared to men and in adults, women are more likely to get the disease. Older patients are more likely to present with a lower lung function and radiographic disease. There must be a biological explanation for this association and why women are so predisposed to sarcoidosis answer: answer: Sarcoidosis in Chinese, especially female, occurs earlier in life and has a late development interval.
This randomized clinical trial demonstrated superiority of inhaled treprostinil over placebo. We conclude that this novel prostanoid treatment should not merely be regarded as a salvage therapy but as a new therapeutic approach, with the potential for improving pulmonary function.