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Lymphangioleiomyomatosis (LAM), a destructive lung disease that primarily affects women, is caused by loss-of-function mutations in TSC1 or TSC2, leading to hyperactivation of mechanistic/mammalian Target of Rapamycin 1 (mTORC1). Rapamycin (sirolimus) treatment suppresses mTORC1, but also induces autophagy, which promotes the survival of TSC2-deficient cells. Based on the hypothesis that simultaneous inhibition of mTORC1 and autophagy would limit the availability of critical nutrients and inhibit LAM cells, we conducted a Phase I clinical trial of sirolimus and hydroxychloroquine for LAM. Here, we report the analyses of plasma metabolomic profiles from the clinical trial.
This article was published in the following journal.
Hydroxychloroquine (HCQ) benefits metabolic and cardiovascular outcomes in patients with rheumatoid arthritis (RA), but its efficacy appears to be moderate as compared to placebo. The aim of our study...
To investigate fundus autofluorescence lifetime features in patients with hydroxychloroquine (HCQ) retinopathy, and to identify early markers of retinal alterations in patients due to HCQ.
Sirolimus is increasingly investigated as a new targeted therapy in pediatric populations. To date, population pharmacokinetic (PK) studies have identified several factors that explain in part the lar...
Blue rubber bleb nevus syndrome (BRBNS) is an extremely rare venous malformation that often appears as multiple hemangioma-like lesions in the skin and gastrointestinal tract. Sirolimus plays a key ro...
Few studies support treating morphea (localized scleroderma) with hydroxychloroquine.
RESULT is a phase II dose-escalating, open-label, safety and efficacy study to determine if there is a potential benefit of resveratrol in combination with sirolimus in patients with lymph...
Lymphangioleiomyomatosis (LAM) is a rare lung disease that is caused by genetic mutations. It results in the uncontrolled growth and proliferation of an unusual type of smooth muscle cell....
The goal of this clinical research study is to find the highest tolerable dose of sirolimus or vorinostat that can be given in combination with hydroxychloroquine to patients with advanced...
The MIDAS study aims to follow women with LAM who are currently taking, have previously failed or been intolerant of, or are considering treatment with mTOR inhibitors sirolimus or everoli...
Background: Lymphangioleiomyomatosis (LAM) is a rare, progressive disease. It usually affects women in the prime of their lives. It typically results in lung destruction. Studies have sho...
A chemotherapeutic agent that acts against erythrocytic forms of malarial parasites. Hydroxychloroquine appears to concentrate in food vacuoles of affected protozoa. It inhibits plasmodial heme polymerase. (From Gilman et al., Goodman and Gilman's The Pharmacological Basis of Therapeutics, 9th ed, p970)
A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to IMMUNOPHILINS. Sirolimus is a potent immunosuppressant and possesses both antifungal and antineoplastic properties.
A derivative of sirolimus and an inhibitor of TOR SERINE-THREONINE KINASES. It is used to prevent GRAFT REJECTION in heart and kidney transplant patients by blocking cell proliferation signals. It is also an ANTINEOPLASTIC AGENT.
A hematopoietic growth factor which promotes proliferation and maturation of neutrophil granulocytes. Clinically it is effective in decreasing the incidence of febrile neutropenia in patients with non-myeloid malignancies receiving myelosuppressive therapy or in reducing the duration of neutropenia and neutropenia-related clinical sequelae in patients with non-myeloid malignancies undergoing myeloblastive chemotherapy followed by BMT. It has also been used in AIDS patients with CMV retinitis being treated with GANCICLOVIR. (Gelman CR, Rumack BH & Hess AJ (eds): DRUGDEX(R) System. MICROMEDEX, Inc., Englewood, Colorado (Edition expires 11/30/95))
Number of patients who need to be treated in order to prevent one additional bad outcome. It is the inverse of Absolute Risk Reduction.
Pulmonary relating to or associated with the lungs eg Asthma, chronic bronchitis, emphysema, COPD, Cystic Fibrosis, Influenza, Lung Cancer, Pneumonia, Pulmonary Arterial Hypertension, Sleep Disorders etc Follow and track Lung Cancer News ...
Women's Health - key topics include breast cancer, pregnancy, menopause, stroke Follow and track Women's Health News on BioPortfolio: Women's Health News RSS Women'...
Within medicine, nutrition (the study of food and the effect of its components on the body) has many different roles. Appropriate nutrition can help prevent certain diseases, or treat others. In critically ill patients, artificial feeding by tubes need t...