Results 11 to 20 of about 117,553 (304)

Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin) [PDF]

medRxiv, 2022
Introduction: β-thalassemia is caused by autosomal mutations in the β-globin gene, which induce the absence or low-level synthesis of β-globin in erythroid cells.
Cristina Zuccato, Lucia Carmela Cosenza, Matteo Zurlo, Jessica Gasparello, Chiara Papi, Elisabetta D’Aversa, Giulia Breveglieri, Ilaria Lampronti, Alessia Finotti, Monica Borgatti, Chiara Scapoli, Alice Stievano, Monica Fortini, Eric Ramazzotti, Nicola Marchetti, Marco Prosdocimi, Maria Rita Gamberini, Roberto Gambari   +17 more
openalex   +2 more sources

Preliminary results of the European multicentric phase III trial regarding sirolimus in slow-flow vascular malformations

JCI Insight, 2023
BACKGROUND Slow-flow vascular malformations frequently harbor activating mutations in the PI3K/AKT/mTOR cascade. Phase II trials pinpointed sirolimus effectiveness as a drug therapy.
Emmanuel Seront, An Van Damme, Catherine Legrand, A. Bisdorff-Bresson, Philippe Orcel, Thomas Funck-Brentano, M. Sevestre, A. Dompmartin, Isabelle Quéré, P. Brouillard, N. Revencu, Martina De Bortoli, Frank Hammer, Philippe Clapuyt, D. Dumitriu, M. Vikkula, L. Boon   +16 more
semanticscholar   +1 more source

Sirolimus suppresses circulating fibrocytes in idiopathic pulmonary fibrosis in a randomized controlled crossover trial

JCI Insight, 2023
BACKGROUND Fibrocytes are BM-derived circulating cells that traffic to the injured lungs and contribute to fibrogenesis. The mTOR inhibitor, sirolimus, inhibits fibrocyte CXCR4 expression, reducing fibrocyte traffic and attenuating lung fibrosis in ...
Diana C. Gomez Manjarres, Dierdre B. Axell-House, D. Patel, John Odackal, Victor Yu, M. Burdick, B. Mehrad   +6 more
semanticscholar   +1 more source

Sirolimus plus prednisolone vs sirolimus monotherapy for kaposiform hemangioendothelioma: a randomized clinical trial.

Blood, 2022
The Kasabach-Merritt phenomenon (KMP) in kaposiform hemangioendothelioma (KHE) is characterized by life-threatening thrombocytopenia and consumptive coagulopathy. This study compared the efficacy and safety of sirolimus plus prednisolone versus sirolimus
Yi Ji, Siyuan Chen, Jiangyuan Zhou, Kaiying Yang, Xuepeng Zhang, B. Xiang, T. Qiu, Xue Gong, Zixin Zhang, Yuru Lan, F. Hu, Feiteng Kong, Qingxia Qiu, Yongbo Zhang   +13 more
semanticscholar   +1 more source

nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors

Journal of Clinical Oncology, 2021
PURPOSE Malignant perivascular epithelioid cell tumor (PEComa) is a rare aggressive sarcoma, with no approved treatment. To our knowledge, this phase II, single-arm, registration trial is the first prospective clinical trial in this disease ...
A. Wagner, V. Ravi, R. Riedel, K. Ganjoo, B. V. Van Tine, R. Chugh, L. Cranmer, E. Gordon, J. Hornick, H. Du, B. Grigorian, A. Schmid, S. Hou, K. Harris, D. Kwiatkowski, N. Desai, M. Dickson   +16 more
semanticscholar   +1 more source

Efficacy of sirolimus in children with lymphatic malformations of the head and neck

European Archives of Oto-Rhino-Laryngology, 2022
Children with extensive lymphatic malformations of the head and neck often suffer from functional impairment and aesthetic deformity which significantly affect the quality of life and may be life-threatening. Treatment with sirolimus has the potential to
S. Wiegand, A. Dietz, G. Wichmann
semanticscholar   +1 more source

Sirolimus (Rapamycin) for Slow-Flow Malformations in Children: The Observational-Phase Randomized Clinical PERFORMUS Trial.

JAMA dermatology, 2021
Importance Sirolimus is increasingly being used to treat various vascular anomalies, although evidence of its efficacy is lacking. Objective To assess the efficacy and safety of sirolimus for children with slow-flow vascular malformations to better ...
A. Maruani, E. Tavernier, O. Boccara, J. Mazereeuw-Hautier, S. Leducq, D. Bessis, L. Guibaud, Pierre Vabres, V. Carmignac, S. Mallet, S. Barbarot, C. Chiaverini, C. Droitcourt, A. Bursztejn, C. Lengellé, J. Woillard, D. Herbreteau, A. Le Touze, A. Joly, C. Léauté-Labrèze, J. Powell, H. Bourgoin, V. Gissot, B. Giraudeau, B. Morel   +24 more
semanticscholar   +1 more source

Sirolimus for epileptic seizures associated with focal cortical dysplasia type II

Annals of Clinical and Translational Neurology, 2022
To determine whether sirolimus, a mechanistic target of rapamycin (mTOR) inhibitor, reduces epileptic seizures associated with focal cortical dysplasia (FCD) type II.
Mitsuhiro Kato, A. Kada, H. Shiraishi, J. Tohyama, E. Nakagawa, Yukitoshi Takahashi, T. Akiyama, A. Kakita, N. Miyake, A. Fujita, A. Saito, Y. Inoue   +11 more
semanticscholar   +1 more source

mTOR Inhibition with Sirolimus in Multiple System Atrophy: A Randomized, Double‐Blind, Placebo‐Controlled Futility Trial and 1‐Year Biomarker Longitudinal Analysis

Movement Disorders, 2022
Multiple system atrophy (MSA) is a fatal neurodegenerative disease characterized by the aggregation of α‐synuclein in glia and neurons. Sirolimus (rapamycin) is an mTOR inhibitor that promotes α‐synuclein autophagy and reduces its associated ...
J. Palma, Jose Martinez, Patricio Millar Vernetti, Thong C Ma, Miguel A Perez, J. Zhong, Yingzhi Qian, Suman Dutta, Katherine N. Maina, Ibrar Siddique, G. Bitan, B. Ades-aron, T. Shepherd, U. Kang, H. Kaufmann   +14 more
semanticscholar   +1 more source

Sirolimus as a second-line treatment for Graves’ orbitopathy

Journal of Endocrinological Investigation, 2022
A beneficial effect of sirolimus in Graves’ orbitopathy (GO) was reported, suggesting a possible use in clinical practice. We conducted an observational, single-centre, no-profit, clinical study to investigate the efficacy of sirolimus as a second-line ...
G. Lanzolla, M. N. Maglionico, Simone Comi, F. Menconi, P. Piaggi, C. Posarelli, M. Figus, C. Marcocci, M. Marinò   +8 more
semanticscholar   +1 more source