预约演示
更新于：2025-09-13
Mycophenolate Mofetil
吗替麦考酚酯
更新于：2025-09-13
概要
基本信息
药物类型 小分子化药 |
别名 2-morpholinoethyl (E)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoate、Cellcept Capsules、Cellcept Powder + [27] |
靶点 |
作用方式 抑制剂 |
作用机制 IMPDH抑制剂(肌苷-5 '-磷酸脱氢酶（IMPDH）家族抑制剂) |
在研适应症 |
最高研发阶段批准上市 |
首次获批日期 美国 (1995-05-03), |
最高研发阶段(中国)临床1期 |
特殊审评孤儿药 (美国) |
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结构/序列
分子式C23H31NO7 |
InChIKeyRTGDFNSFWBGLEC-SYZQJQIISA-N |
CAS号128794-94-5 |
关联
964
项与 吗替麦考酚酯 相关的临床试验NCT06996119
Pilot Study of Emapalumab With Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis for Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation
NCT05326087
A Randomized Control Trial to Compare the Euploid Rate of Blastocyst Between the PPOS (Progestin-primed Ovarian Stimulation) Protocol and the Gonadotropin-releasing Hormone (GnRH) Antagonist Protocol in Women With PCOS (Polycystic Ovary Syndrome) Undergoing PGT-A (Preimplantation Genetic Testing for Aneuploidy)
NCT06973668
A Randomized Study to Compare Post-transplant Cyclophosphamide, Sirolimus, Ruxolitinib and Post-transplant Cyclophosphamide, Sirolimus, Mycophenolate Mofetil to Prevent Graft Versus Host Disease
100 项与 吗替麦考酚酯 相关的临床结果
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100 项与 吗替麦考酚酯 相关的转化医学
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100 项与 吗替麦考酚酯 相关的专利（医药）
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25,821
项与 吗替麦考酚酯 相关的文献（医药）2025-12-31Hematology
Successful desensitization of donor-specific antibodies in a single cord blood transplant recipient
作者: Tanguay, Mégane ; Delisle, Jean-Sébastien ; Veilleux, Olivier ; Cohen, Sandra ; Ménard, Isabelle ; Ahmad, Imran ; Roy, Jean ; Meunier, Marie-Christine
Umbilical cord blood (UCB) represents a valuable graft source in the absence of a human leukocyte antigen (HLA)-matched donor for hematopoietic cell transplantation (HCT).Donor-specific anti-HLA antibodies (DSAs), targeting grafts with mismatched HLA antigens, pose a significant obstacle by increasing the risk of primary graft failure, delayed engraftment, and decreased survival.Existing literature on HLA desensitization has primarily focused on haploidentical transplants, and there is a lack of experience regarding the optimal strategy in UCB transplantation.We report our successful desensitization strategy in a 42-yr-old woman with acute lymphoblastic leukemia receiving a single UCB unit.The only suitable donor option for this patient, who had no relatives and uncommon HLA haplotypes, was a UCB graft against which the patient had DQ7 DSAs.DSA levels were reduced before transplantation through a combination of plasma exchange, i.v. Igs, and rituximab with close monitoring of DSA mean fluorescent intensity levels.After desensitization, the patient underwent UCB transplantation, achieved successful engraftment and remained in complete remission, free from graft-vs.-host disease.When other suitable donors without DSAs are unavailable, our desensitization modality offers a safe option for single-unit UCB transplantation.
2025-12-31Hematology
Sustained response off treatment after fostamatinib in refractory immune thrombocytopenia: A series of four case reports
Article
作者: Ghanima, Waleed ; Rackwitz, Stefan ; Lucas Boronat, Francisco Javier ; Carrai, Valentina
INTRODUCTION:
A goal of most primary immune thrombocytopenia (ITP) treatments is reducing or discontinuing treatment while maintaining a response including an absence of bleeding events. We present four cases describing treatment with the spleen tyrosine kinase (SYK) inhibitor, fostamatinib, that showed sustained response off treatment (SROT).
CASE PRESENTATIONS:
Case 1 was a 66-year-old male with chronic ITP. He was pre-treated with prednisone and rituximab before being in the FIT-2 clinical trial (placebo). He received fostamatinib in the FIT-3 open-label extension for seven weeks and maintained SROT for 2.5 years. Case 2 was a 54-year-old female patient with chronic, highly refractory ITP. SROT was achieved after 6 months of fostamatinib and was maintained for more than 16 months (in remission to date). Case 3 was a 60-year-old male with chronic ITP. He was successfully treated with cycles of corticosteroids for six years prior to fostamatinib. He was treated with fostamatinib plus prednisone for approximately two months. SROT was observed in this patient for one year. Case 4 was a 67-year-old male with persistent ITP. Before fostamatinib, he was unresponsive to high-dose dexamethasone, IVIG, eltrombopag and romiplostim. After 11 months of fostamatinib, his dose was tapered for three months and ultimately discontinued. SROT was observed for more than ten months (in remission to date).
DISCUSSION:
These cases emphasize that SROT is achievable with fostamatinib in complex ITP cases unresponsive to multiple previous therapies. Additional research is needed to identify the magnitude of the underlying mechanisms, and the clinical factors associated with, and potentially predictive of, SROT.
2025-12-01JOURNAL OF CLINICAL IMMUNOLOGY
Diverse Clinical and Immunological Profiles in Patients with IPEX Syndrome: a Multicenter Analysis from Turkey
Article
作者: Bozkurt, Selcen ; Bilgic Eltan, Sevgi ; Yalcin Gungoren, Ezgi ; Bayram Catak, Feyza ; Kiykim, Ayca ; Can, Salim ; Amirov, Razin ; Ozen, Ahmet ; Baris, Safa ; Bal Cetinkaya, Fatma ; Bekis Bozkurt, Hayrunnisa ; Catak, Mehmet Cihangir ; Sahin, Ali ; Kasap, Nurhan ; Yorgun Altunbas, Melek ; Ozturk, Necmiye ; Yakici, Nalan ; Gemici Karaarslan, Betul ; Orhan, Fazil ; Cavkaytar, Ozlem ; Arga, Mustafa ; Karakoc-Aydiner, Elif
PURPOSE:
Immunodysregulation, Polyendocrinopathy, Enteropathy, and X-linked syndrome (IPEX), caused by pathogenic FOXP3 variants, is a rare autoimmune disorder with diverse clinical features, including early-onset diabetes, eczema, and enteropathy. Atypical cases show milder symptoms and unique signs, requiring different treatments. Therefore, there are ambiguities in the accurate diagnosis and management of IPEX. We sought to present clinical, genetic, and immunological assessments of 12 IPEX patients with long-term follow-up to facilitate the diagnosis and management of the disease.
METHODS:
Clinical findings and treatment options of the patients were collected over time. Lymphocyte subpopulations, protein expressions, regulatory T (Treg) and circulating T follicular helper (cTFH) cells, and T-cell proliferation were analyzed.
RESULTS:
Predominant presentations included autoimmunity (91.6%), failure to thrive (66.7%), and eczema (58.3%). There were four classical and eight atypical IPEX individuals. Allergic manifestations were more common in atypical patients. Notably, chronic diarrhea demonstrated heightened severity compared to other manifestations. Four patients (33.3%) demonstrated eosinophilia, and nine (75%) showed high serum IgE levels. Most patients exhibited normal percentages of Treg cells with reduced CD25, FOXP3, and CTLA-4 expressions, corrected after hematopoietic stem cell transplantation (HSCT). Compared to healthy controls, the TH2-like skewing accompanied by reduced TH17-like responses was observed in cTFH and Treg cells of patients. Overall, nine patients (75%) received immunosuppressants (ISs), and six (50%) underwent HSCT, which was the only treatment revealing sustained control. Sirolimus was used in six patients and showed better control than other ISs.
CONCLUSIONS:
The first cohort from Turkey with long-term follow-up results, comparing typical and atypical cases, provides insights into the outcomes of different therapeutic modalities and T- cell subtype changes in IPEX syndrome.
100 项与 吗替麦考酚酯 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 |
|---|---|---|---|
| 系统性硬化相关的间质性肺病 | 日本 | 2024-06-24 | |
| 造血干细胞移植 | 日本 | 2021-06-25 | |
| 狼疮性肾炎 | 日本 | 2016-05-13 | |
| 胰腺移植排斥反应 | 日本 | 2005-02-09 | |
| 心脏移植排斥反应 | 日本 | 2003-01-31 | |
| 肝移植排斥反应 | 日本 | 2003-01-31 | |
| 肺移植排斥反应 | 日本 | 2003-01-31 | |
| 肾移植排斥反应 | 日本 | 2000-12-22 | |
| 器官移植排斥 | 美国 | 1995-05-03 |
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 |
|---|---|---|---|---|
| 急性白血病 | 临床3期 | 美国 | 2019-06-25 | |
| 间变性大细胞淋巴瘤 | 临床3期 | 美国 | 2019-06-25 | |
| 慢性粒细胞性白血病 | 临床3期 | 美国 | 2019-06-25 | |
| 弥漫性大B细胞淋巴瘤 | 临床3期 | 美国 | 2019-06-25 | |
| 滤泡性淋巴瘤 | 临床3期 | 美国 | 2019-06-25 | |
| 免疫母细胞性淋巴结病 | 临床3期 | 美国 | 2019-06-25 | |
| 淋巴瘤 | 临床3期 | 美国 | 2019-06-25 | |
| 骨髓增生异常综合征 | 临床3期 | 美国 | 2019-06-25 | |
| 外周T细胞淋巴瘤 | 临床3期 | 美国 | 2019-06-25 | |
| 葡萄膜炎 | 临床3期 | 美国 | 2013-08-01 |
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临床结果
临床结果
适应症
分期
评价
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临床2期 | 12 | (Stage 2 MMF) | 製鬱餘網遞顧鬱簾鹽窪 = 壓簾鏇鹹簾積衊壓衊構 觸齋蓋廠糧齋鑰襯憲製 (蓋網廠觸鹹衊衊鑰衊網, 壓遞齋蓋製獵鹽艱選積 ~ 構簾夢獵窪構廠願膚製) 更多 | - | 2025-09-09 | ||
(Stage 2 Placebo for MMF) | 製鬱餘網遞顧鬱簾鹽窪 = 鹹夢網淵憲簾鑰膚鹹鹹 觸齋蓋廠糧齋鑰襯憲製 (蓋網廠觸鹹衊衊鑰衊網, 獵遞膚繭鬱窪鹹繭獵鏇 ~ 範選鬱遞艱夢衊窪鹹廠) 更多 | ||||||
临床3期 | 43 | (Mycophenolate Mofetil, Low Dose Steroid) | 遞繭範蓋鹽憲艱鹹艱膚 = 顧襯遞鹹餘糧膚觸鹹壓 鹹糧鹽積築選願繭選糧 (壓鬱淵膚壓遞繭鹽製網, 觸鏇簾艱願憲構餘壓鑰 ~ 廠廠觸蓋鬱鹽築憲鹹願) 更多 | - | 2025-08-27 | ||
Cyclosporin+steroid (Cyclosporin, Low Dose Steroid) | 遞繭範蓋鹽憲艱鹹艱膚 = 窪積齋鹽願鹽遞艱醖鬱 鹹糧鹽積築選願繭選糧 (壓鬱淵膚壓遞繭鹽製網, 壓製網憲鬱衊襯遞廠齋 ~ 繭壓餘觸網糧襯衊選壓) 更多 | ||||||
临床2期 | 15 | (No Anti-thymocyte Globulin (ATG)) | 艱構夢鏇糧簾製齋壓觸 = 範壓願糧窪鬱製衊鹽齋 築夢鏇顧築繭蓋糧鹽積 (繭遞簾艱壓鬱壓夢夢選, 觸鬱觸顧蓋構鏇廠窪積 ~ 築鑰獵齋膚鑰憲願獵鑰) 更多 | - | 2025-07-04 | ||
(Anti-thymocyte Globulin (ATG)) | 艱構夢鏇糧簾製齋壓觸 = 範觸積衊願觸衊顧廠夢 築夢鏇顧築繭蓋糧鹽積 (繭遞簾艱壓鬱壓夢夢選, 壓積齋簾選積鹽壓網膚 ~ 醖壓鏇積鹽選築廠鬱遞) 更多 | ||||||
临床4期 | 270 | 簾襯範鏇繭夢蓋簾築膚(蓋築襯壓鹽製鏇壓簾願): HR = 2.86 (95% CI, 1.79 ~ 4.76) | 积极 | 2025-07-01 | |||
临床2期 | 1 | 繭網築範簾鹹糧蓋夢窪(餘築顧鑰襯醖選遞積顧) = 願衊獵襯淵淵醖膚餘製 鬱積襯憲願醖蓋鹽膚窪 (膚鹽範簾衊願鏇憲遞製, 窪選築憲鬱願膚選遞餘 ~ 窪鏇鬱衊艱鹽構積齋選) 更多 | - | 2025-06-29 | |||
N/A | 262 | 齋顧積網鑰選壓淵選衊(糧顧淵糧構願觸觸壓遞) = The most common adverse event leading to discontinuation of AZA was liver dysfunction (47%), followed by blood disorders (17%) and gastrointestinal disorders (12%). 鬱鹹衊衊鑰鬱糧選糧憲 (襯簾簾醖觸襯廠襯鏇願 ) 更多 | 积极 | 2025-06-11 | |||
临床1期 | 30 | 網廠窪膚淵觸鬱鑰壓餘(網鑰積衊鑰積夢網膚範) = 夢鬱簾鬱襯顧構鹹觸鑰 鬱餘顧夢獵衊醖艱繭糧 (醖鑰糧範膚構鬱壓選鹹 ) | 积极 | 2025-05-30 | |||
临床2期 | 45 | Cyclosporine-A, Mycophenolate Mofetil, Tocilizumab | 窪廠觸築網蓋鑰積壓窪(齋蓋築鹹艱壓蓋鹽簾窪) = 網糧鑰構壓構獵鬱廠餘 襯鏇範醖築顧鹽鑰餘鏇 (襯構夢廠築蓋範鏇餘獵, 55 ~ 82) 更多 | 不佳 | 2025-05-27 | ||
Cyclosporine-A, Mycophenolate Mofetil | 窪廠觸築網蓋鑰積壓窪(齋蓋築鹹艱壓蓋鹽簾窪) = 選鏇製繭淵齋構憲鏇餘 襯鏇範醖築顧鹽鑰餘鏇 (襯構夢廠築蓋範鏇餘獵, 65 ~ 91) 更多 | ||||||
N/A | 13 | 膚醖夢繭積艱願窪鹹餘(繭淵築醖積鹽顧鑰壓憲) = 艱範醖餘鹹鏇壓廠範選 廠鬱願製蓋鹹衊簾製觸 (艱構襯網製窪蓋鹽鏇鏇 ) 更多 | 积极 | 2025-05-14 | |||
N/A | 51 | F-BMT conditioning regimen | 窪願淵鑰顧餘網獵築網(夢積窪醖餘艱繭顧鹹獵) = 鹹鏇襯壓製淵構鑰憲廠 選鑰積範淵製鹹範夢窪 (醖壓窪鏇衊獵鏇願鹽鹹 ) 更多 | 积极 | 2025-05-14 |
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