更新于：2024-12-12
Forodesine Hydrochloride
呋咯地辛
更新于：2024-12-12
概要
基本信息
药物类型 小分子化药 |
别名 (1S)-1,4-dideoxy-4-imino-(9-deazahypoxanthin-9-yl)-D-ribitol、1,4-Dideoxy-4-aza-1-(S)-(9-deazahypoxanthin-9-yl)-D-ribitol、FDS + [10] |
靶点 |
作用机制 PNP抑制剂(嘌呤核苷磷酸化酶抑制剂)、免疫抑制剂 |
在研适应症 |
在研机构 |
最高研发阶段批准上市 |
首次获批日期 日本 (2017-03-31), |
最高研发阶段(中国)- |
特殊审评孤儿药 (欧盟)、孤儿药 (日本) |
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结构
分子式C11H15ClN4O4 |
InChIKeyWEIAMZKHBCLFOG-QPAIBFMUSA-N |
CAS号284490-13-7 |
关联
20
项与 呋咯地辛 相关的临床试验Phase I/II Clinical Study of Forodesine in Japanese Recurrent/Refractory Peripheral T-cell Lymphoma Patients
Phase 1/2 clinical study of forodesine in Japanese recurrent/refractory peripheral T-cell lymphoma
Phase 1/2 clinical study of forodesine in Japanese recurrent/refractory peripheral T-cell lymphoma
100 项与 呋咯地辛 相关的临床结果
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100 项与 呋咯地辛 相关的转化医学
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100 项与 呋咯地辛 相关的专利（医药）
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209
项与 呋咯地辛 相关的文献（医药）2024-12-01ANNALS OF NEUROLOGY
Guanylate Kinase 1 Deficiency: A Novel and Potentially Treatable Mitochondrial DNA Depletion/Deletions Disease
Article
作者: Marti, Ramon ; Meneri, Megi ; Pesini, Alba ; Tort, Frederic ; Sciacco, Monica ; Hirano, Michio ; Milisenda, Jose Cesar ; Koganti, Lahari ; Tanji, Kurenai ; Tadesse, Saba ; Wang, Tian ; Matas‐Garcia, Ana ; Ramon, Javier ; Ugarteburu, Olatz ; Saneto, Russell P. ; Mansukhani, Mahesh ; Gu, Yue ; Shintaku, Jonathan ; Comi, Giacomo Pietro ; Hidalgo‐Gutierrez, Agustin ; Horwitz, Marshall S. ; Ribes, Antonia ; Garrabou, Gloria ; Gort, Laura ; Barriocanal‐Casado, Eliana ; Dorschner, Michael O. ; Ronchi, Dario ; Wang, Shuang
Objective:
Mitochondrial DNA (mtDNA) depletion/deletions syndrome (MDDS) comprises a group of diseases caused by primary autosomal defects of mtDNA maintenance. Our objective was to study the etiology of MDDS in 4 patients who lack pathogenic variants in known genetic causes.
Methods:
Whole exome sequencing of the probands was performed to identify pathogenic variants. We validated the mitochondrial defect by analyzing mtDNA, mitochondrial dNTP pools, respiratory chain activities, and GUK1 activity. To confirm pathogenicity of GUK1 deficiency, we expressed 2 GUK1 isoforms in patient cells.
Results:
We identified biallelic GUK1 pathogenic variants in all 4 probands who presented with ptosis, ophthalmoparesis, and myopathic proximal limb weakness, as well as variable hepatopathy and altered T‐lymphocyte profiles. Muscle biopsies from all probands showed mtDNA depletion, deletions, or both, as well as reduced activities of mitochondrial respiratory chain enzymes. GUK1 encodes guanylate kinase, originally identified as a cytosolic enzyme. Long and short isoforms of GUK1 exist. We observed that the long isoform is intramitochondrial and the short is cytosolic. In probands’ fibroblasts, we noted decreased GUK1 activity causing unbalanced mitochondrial dNTP pools and mtDNA depletion in both replicating and quiescent fibroblasts indicating that GUK1 deficiency impairs de novo and salvage nucleotide pathways. Proband fibroblasts treated with deoxyguanosine and/or forodesine, a purine phosphatase inhibitor, ameliorated mtDNA depletion, indicating potential pharmacological therapies.
Interpretation:
Primary GUK1 deficiency is a new and potentially treatable cause of MDDS. The cytosolic isoform of GUK1 may contribute to the T‐lymphocyte abnormality, which has not been observed in other MDDS disorders. ANN NEUROL 2024;96:1209–1224
2024-11-01Journal of neurointerventional surgery
Two year follow-up of distal unruptured intracranial aneurysms treated with a surface modified flow diverter under prasugrel monotherapy
Article
作者: Freitas, Rafael Kiyuze ; Abud, Daniel Giansante ; Machado, João Paulo ; Nakiri, Guilherme Seizem ; Monsignore, Lucas Moretti ; Abud, Thiago Giansante ; Colli, Benedicto Oscar ; de Oliveira, Ricardo Santos ; de Castro-Afonso, Luis Henrique
Background:
A new generation of modified surface flow diverters (FDs) and monotherapy using new antiplatelets may reduce both ischemic and hemorrhagic complications during the treatment of intracranial aneurysms. Previous preliminary safety analysis of distal unruptured intracranial aneurysms treated with the FD p48 MW HPC (phenox-Wallaby, Bochum, Germany) under antiplatelet monotherapy with prasugrel showed promising results. However, the long term outcomes of distal intracranial aneurysms treated with FDs under antiplatelet monotherapy are not known.
Methods:
This was a single center, prospective, pivotal, open single arm study. The primary (safety) endpoint was absence of any new neurological deficits after treatment until the 24 month follow-up. The primary (efficacy) endpoint was the incidence of complete aneurysm occlusion 24 months after treatment. The secondary (efficacy) endpoints were any incidence of aneurysm dome reduction 24 months after treatment.
Results:
21 patients harboring 27 distal aneurysms of the anterior circulation were included. No patient had neurologic deficits in the time from treatment to the 24 month follow-up. Complete aneurysm occlusion occurred in 20 (74%) of 27 aneurysms at the 24 month follow-up. Four aneurysms (14.8%) had dome reduction, and three aneurysms (11.1%) remained unchanged.
Conclusions:
In this pilot trial, treatment of distal unruptured intracranial aneurysms with an FD under monotherapy with prasugrel, followed by monotherapy with aspirin, appeared to be safe and effective. Randomized studies with long term follow-up are needed to confirm these results.
2024-11-01World Neurosurgery
Telescoping Flow Diverters for the Treatment of Brain Aneurysms: Indications and Outcome
Article
作者: Teichner, Eric M. ; Rosenwasser, Robert H. ; El Naamani, Kareem ; Winiker, Sarah ; Rosenwasser, Robert H ; Tjoumakaris, Stavropoula I ; Schmidt, Richard F. ; Jabbour, Pascal M ; Ghanem, Marc ; Schmidt, Richard F ; Jabbour, Pascal M. ; Momin, Arbaz A. ; Lan, Matthews ; Gooch, Michael R ; Roy, Joanna M. ; Ahmed, Meah T. ; Tjoumakaris, Stavropoula I. ; Ghosh, Ritam ; Roy, Joanna M ; Ahmed, Meah T ; Teichner, Eric M ; Momin, Arbaz A ; Gooch, Michael R. ; Musmar, Basel ; Zarzour, Hekmat
OBJECTIVE:
Few studies have reported the impact of telescoping flow diverters (FDs) in intracranial aneurysm treatment. Our study compared aneurysms treated using telescoping FDs to those treated with a single FD and identified predictors of telescoping.
METHODS:
This was a single-center retrospective review of a prospectively maintained database of aneurysms treated with FDs between 2011 and 2023. All patients who were treated with FDs for intracranial aneurysms were included in the study.
RESULTS:
The study comprised 750 patients with 750 aneurysms treated using 871 FDs. The study cohort was divided into 85 patients requiring telescoping FDs and 655 who did not. Rates of hemorrhage (7.1% vs. 1.8%, P < 0.001), symptomatic stroke (5.9% vs. 2.6, P < 0.001), and asymptomatic stroke (1.2% vs. 0.8%, P < 0.001) were significantly higher in the telescoping cohorts. At final follow-up, the rate of nonocclusion (9.8% vs. 5.1%, P = 0.029) and the rate of complete occlusion (88.5% vs. 81.1%, P = 0.029) were significantly higher in the telescoping cohort. On multivariate analysis, fusiform morphology (odds ratio [OR]: 2.4, 95% confidence interval [CI] 1.0-5.0, P = 0.03), increasing aneurysm height (OR: 1.0, 95% CI 1.0-1.1, P= 0.034), and the use of the Pipeline Embolization Device FD (OR: 2.4, 95% CI 1.3-4.4, P = 0.005) were independent predictors of telescoping.
CONCLUSIONS:
Aneurysms with fusiform morphology, increasing aneurysm height and those that underwent flow diversion using Pipeline Embolization Device had higher odds for telescoping. Significantly higher rates of angiographic occlusion with the use of telescoping FD add to the literature on its efficacy in treating aneurysms of varying morphology.
1
项与 呋咯地辛 相关的新闻（医药）2023-02-01
·生物谷
细胞疗法免疫疗法
100 项与 呋咯地辛 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 外周T细胞淋巴瘤 | 日本 | 2017-03-31 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 急性淋巴细胞白血病 | 临床2期 | 奥地利 | 2009-03-01 | |
| 急性淋巴细胞白血病 | 临床2期 | 捷克 | 2009-03-01 | |
| 急性淋巴细胞白血病 | 临床2期 | 法国 | 2009-03-01 | |
| 急性淋巴细胞白血病 | 临床2期 | 德国 | 2009-03-01 | |
| 急性淋巴细胞白血病 | 临床2期 | 意大利 | 2009-03-01 | |
| 急性淋巴细胞白血病 | 临床2期 | 英国 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 奥地利 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 捷克 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 法国 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 德国 | 2009-03-01 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 外周T细胞淋巴瘤 二线 | 48 | 範蓋鬱鑰願淵獵蓋鹽鹽(網構製繭網壓顧鏇鏇範) = 鏇淵鑰鏇壓蓋夢鹽願艱 艱艱獵壓夢範築簾窪觸 (積窪窪醖鬱鹽窪衊醖積, 14 ~ 38) 更多 | 积极 | 2019-01-01 | ||
临床1/2期 | 44 | 窪構構網鬱鹹壓醖製願(顧醖鹹顧襯遞積築衊蓋) = 選積廠鏇廠鹹憲壓醖壓 遞窪顧艱淵簾範襯網鏇 (顧鹽積壓簾選遞淵製築, 12.0 ~ 35.3) 更多 | 积极 | 2016-05-20 | |||
临床2期 | 144 | 鹽簾網鹽簾築窪衊窪網(壓鹹鹹廠願顧獵鏇鏇積) = 夢窪憲鏇獵壓鹽壓窪艱 選鏇壓繭選衊餘淵簾廠 (構鑰築製鹽衊選願繭鏇 ) 更多 | - | 2014-09-01 | |||
临床2期 | 8 | Oral forodesine 200 mg/d | 鏇糧艱廠齋遞積選鹽餘(鬱顧繭衊壓廠艱鏇願鬱) = 選廠醖鹽鹽膚網襯鑰蓋 糧夢範製遞淵繭壓衊積 (膚襯製顧繭選齋願顧範 ) 更多 | - | 2010-08-12 | ||
N/A | 64 | 鹽襯鹽積鹽範鏇獵夢醖(願襯蓋製獵遞鬱繭簾簾) = 製壓憲鑰獵積遞鬱鏇蓋 齋餘鑰製艱壓遞製艱膚 (鹹鑰淵選夢範醖鑰餘夢 ) 更多 | - | 2009-05-20 |
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