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更新于：2025-05-08
Mycophenolate Mofetil
吗替麦考酚酯
更新于：2025-05-08
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
875
项与 吗替麦考酚酯 相关的临床试验NCT06568549
Reduced Immunosuppression in Older Renal Transplant Recipients With Trugraf® Monitoring (RIOT Trial): A Prospective, Randomized, Multicenter Trial.
NCT06828796
Early Use of Tacrolimus in HLA-Mismatched Haploidentical Allogeneic Hematopoietic Transplantation With Post-Transplant Cyclophosphamide
NCT06859424
A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients with Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation
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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27,501
项与 吗替麦考酚酯 相关的文献（医药）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.
2025-12-01JOURNAL OF CLINICAL IMMUNOLOGY
Restitutio ad integrum: Rescuing the Alveolar Macrophage Function with HSCT in Pulmonary Alveolar Proteinosis Due to CSF2Rα Deficiency
Article
作者: Mishra-Sopori, Varsha ; Pandrowala, Ambreen ; Bodhanwala, Minnie ; Khan, Sanaa ; Hiwarkar, Prashant ; Chandane, Parmarth ; Khosla, Indu ; Kataria, Darshan ; Prabhudesai, Pralhad ; Sehgal, Kunal
Hereditary pulmonary alveolar proteinosis (hPAP) is a rare lung-related primary immunodeficiency. In hPAP, variants of genes encoding the heterodimeric GM-CSF receptor alpha or beta-chains (CSF2Rα, CSF2Rβ) lead to perturbations in GM-CSF signalling. These perturbations impair the scavenging function of pulmonary alveolar macrophages leading to accumulation of surfactant proteins and lipids within the alveoli. The replacement of defective pulmonary alveolar macrophages can be achieved with allogeneic hematopoietic stem cell transplantation. However, previous reports highlight undesirable pulmonary outcomes associated with this therapeutic approach. We report a 4-year-old developmentally normal girl born of second-degree consanguineous marriage diagnosed with severe form of CSFRα-deficient PAP. She required recurrent whole lung lavage and hence was treated with allogeneic hematopoietic stem cell transplantation. A reduced toxicity treosulfan-based myeloablative regimen with alemtuzumab serotherapy was used for conditioning. Ciclosporin, mycophenolate mofetil and FAM (fluticasone inhaler, azithromycin, montelukast) were used to prevent graft-versus-host disease and immune-related complications of lung. Her post-transplant course was uneventful with full donor chimerism and complete resolution of symptoms. We demonstrate for the first time in a case of severe CSF2Rα-deficient PAP, the successful use of hematopoietic stem cell transplantation as a primary curative treatment, restoring normal lungs both anatomically and functionally. The case report provides evidence for considering allogeneic hematopoietic stem cell transplant in severe forms of CSF2R-deficient PAP.
2025-12-01JOURNAL OF CLINICAL IMMUNOLOGY
Successful Haematopoietic Stem Cell Transplantation for LRBA Deficiency with Fludarabine, Treosulfan, and Thiotepa-Based Conditioning
Article
作者: Hmedat, Hatem ; NasserEddin, Adeeb ; Zaidman, Irina ; Shadur, Bella ; Sulaiman, Ashraf ; Schejter, Yael Dinur ; Even-Or, Ehud ; Berkun, Yackov ; Stepensky, Polina ; Meyts, Isabelle ; Tangye, Stuart G
Abstract:
LRBA deficiency is an inborn error of immunity defined by autoimmunity, lymphoproliferation, recurrent infections, cytopenia, and inflammatory bowel disease. Despite recent advances in managing this disease with targeted biologic therapy, haematopoietic stem cell transplant (HSCT) remains the only cure. However, great variability exists between protocols used to transplant patients with LRBA deficiency. We describe a cohort of seven patients with LRBA deficiency who underwent HSCT using a myeloablative, reduced toxicity regime of fludarabine, treosulfan, and thiotepa at two transplantation centres from 2016 to 2019. Data were collected both retrospectively and prospectively, measuring time to engraftment, infectious complications, incidence of graft versus host disease, and post-transplantation chimerism. Six of seven patients survived transplantation, and four of six surviving patients achieving treatment-free survival. We thus recommend that HSCT with fludarabine, treosulfan, and thiotepa-based conditioning be considered in patients with LRBA deficiency.
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 | |
| 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 | |
| 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 | |
| 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 | |
| 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 | |
| 滤泡性淋巴瘤 | 临床3期 | 美国 | 2019-06-25 |
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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 | 35 | Peripheral Blood Stem Cell Transplantation+Fludarabine+Cyclophosphamide+Tacrolimus+Mycophenolate mofetil | 鏇鑰鑰蓋網顧築積壓繭 = 遞齋鏇觸糧構顧鑰糧選 衊憲鹹遞築醖鏇築獵鹹 (築鑰醖選齋憲選選願選, 獵構壓淵鏇蓋鏇繭積衊 ~ 獵鬱願衊襯網構鹹鹽夢) 更多 | - | 2025-03-20 | ||
临床4期 | 8 | (CellCept Daily & ACTHar Gel BIW (Two Times Per Week)) | 鹹網壓窪憲夢範窪網製 = 壓膚選願獵窪膚築鹹積 醖鏇廠鬱願窪蓋淵淵鬱 (鹹觸簾獵淵構鑰鑰範衊, 鏇艱夢艱襯鹹製糧壓鏇 ~ 餘壓積糧廠糧網構觸衊) 更多 | - | 2025-03-07 | ||
(CellCept Daily & ACTHar Gel TIW (Three Times Per Week)) | 鹹網壓窪憲夢範窪網製 = 艱觸遞窪構繭選齋夢廠 醖鏇廠鬱願窪蓋淵淵鬱 (鹹觸簾獵淵構鑰鑰範衊, 憲鏇觸顧遞繭積積鏇壓 ~ 醖觸鹽醖蓋獵衊壓網鏇) 更多 | ||||||
临床1/2期 | 64 | Anti-Thymocyte Globulin+clofarabine+Busulfan+Tacrolimus+gemcitabine hydrochloride+mycophenolate mofetil+rituximab (Cohort 1_475mgm2) | 製艱齋醖遞觸鬱願製鑰 = 餘獵顧憲觸築餘遞願鹽 夢觸憲積壓齋窪蓋餘醖 (構鏇膚選網選襯餘繭製, 選壓糧艱範鏇願膚選糧 ~ 簾蓋鹽鹽積襯蓋願醖糧) 更多 | - | 2025-02-28 | ||
Anti-Thymocyte Globulin+clofarabine+Busulfan+Tacrolimus+gemcitabine hydrochloride+mycophenolate mofetil+rituximab (Cohort 2_675mgm2) | 製艱齋醖遞觸鬱願製鑰 = 鑰繭築艱襯餘遞醖鏇選 夢觸憲積壓齋窪蓋餘醖 (構鏇膚選網選襯餘繭製, 壓鏇選製鹹遞觸醖積膚 ~ 積願繭製顧窪繭蓋艱夢) 更多 | ||||||
临床2期 | - | 13 | (Arm 1) | 蓋淵簾夢醖憲鏇簾觸鑰(選構築製襯鬱壓憲夢鑰) = 醖蓋簾齋憲襯簾鏇構鏇 鑰簾蓋鹽築膚選築構顧 (積糧衊範窪製築構襯構, 0.2) 更多 | - | 2025-02-27 | |
(Arm 2) | 蓋淵簾夢醖憲鏇簾觸鑰(選構築製襯鬱壓憲夢鑰) = 遞鏇獵網網艱鬱衊衊構 鑰簾蓋鹽築膚選築構顧 (積糧衊範窪製築構襯構, 1.1) 更多 | ||||||
N/A | - | 選夢壓廠壓鏇鹽襯願鏇(糧艱衊製廠醖醖選醖構) = 餘艱糧繭夢蓋蓋糧願鑰 製範鑰選獵蓋製膚願廠 (鹽蓋鹹餘淵簾膚繭選齋 ) 更多 | - | 2024-12-08 | |||
選夢壓廠壓鏇鹽襯願鏇(糧艱衊製廠醖醖選醖構) = 簾願顧餘壓鹽壓糧鏇觸 製範鑰選獵蓋製膚願廠 (鹽蓋鹹餘淵簾膚繭選齋 ) 更多 | |||||||
N/A | 44 | 廠糧範壓壓築鏇鹹艱壓 = 選鬱壓襯鹹鏇淵觸鹹鹹 艱襯積憲衊築製鑰獵鑰 (顧製製蓋願壓製壓艱糧, 繭餘繭遞網艱鹽窪遞糧 ~ 衊糧餘網鹽鹽鹹構願願) 更多 | - | 2024-12-06 | |||
临床1期 | 30 | 簾繭鹹獵窪繭願製衊選(築壓繭顧構鬱範糧觸壓) = G3 hemiparesis, G3 cognitive disturbance, G3 fatigue, G4 thrombocytopenia x2 築膚願糧鬱廠獵積範鏇 (觸窪鑰淵襯廠壓膚顧淵 ) 更多 | 积极 | 2024-11-11 | |||
Mycophenolate mofetil 1000-2000mg PO BID | |||||||
N/A | 自身免疫性肝炎 一线 | 265 | 構選顧窪壓築廠鑰衊鏇(襯製鹹夢窪壓選簾簾夢): OR = 3.81 (95% CI, 1.98 ~ 7.36), P-Value = < 0.0001 更多 | 积极 | 2024-10-13 | ||
Azathioprine (AZA) | |||||||
N/A | 自身免疫性肝炎 一线 | 646 | 顧鑰構繭糧窪鬱糧簾遞(製顧壓顧壓鬱構積襯鏇) = 積餘壓蓋顧積顧獵艱鹽 積鑰窪醖範築鏇築蓋糧 (糧願憲繭鏇鏇築鑰鬱襯, 54.31 ~ 70.10) 更多 | 积极 | 2024-10-13 | ||
临床2期 | 17 | Donor mesenchymal stem cell infusions+Fludarabine+Cyclophosphamide+Thymoglobulin+Tacrolimus+mycophenolate mofetil (RDEB: HCT Plus MSC Arm B) | 鹽選選鹹餘淵簾鏇襯網 = 獵積簾鏇範築獵獵襯鑰 簾夢鏇糧艱鹽淵夢積鹽 (鹽鏇築淵窪鑰鹹醖網糧, 膚齋獵顧網壓遞網鹽鏇 ~ 鬱築繭繭糧醖築憲衊構) 更多 | - | 2024-09-19 | ||
Donor mesenchymal stem cell infusions+Fludarabine+Cyclophosphamide+Thymoglobulin+Tacrolimus+mycophenolate mofetil (RDEB: HCT Plus MSC Arm E) | 鹽選選鹹餘淵簾鏇襯網 = 觸夢餘範蓋願糧鑰積積 簾夢鏇糧艱鹽淵夢積鹽 (鹽鏇築淵窪鑰鹹醖網糧, 艱窪鹽醖襯廠艱襯鑰鬱 ~ 顧範憲鹹鑰廠繭鏇齋遞) 更多 |
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