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更新于：2025-03-06
Mycophenolate Mofetil
吗替麦考酚酯
更新于：2025-03-06
概要
基本信息
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
872
项与 吗替麦考酚酯 相关的临床试验NCT06807606
Optimized CBT for Pts With High-risk Hematologic Malignancies Who Have Relapsed After First ASCT
NCT06568549
Reduced Immunosuppression in Older Renal Transplant Recipients With Trugraf® Monitoring (RIOT Trial): A Prospective, Randomized, Multicenter Trial.
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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25,726
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 ; Yorgun Altunbas, Melek ; Kasap, Nurhan ; 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 Allogeneic Hematopoietic Cell Transplantation for Patients with IL10RA Deficiency in Japan
Article
作者: Ishige, Takashi ; Sasahara, Yoji ; Goto, Kimitoshi ; Tomomasa, Dan ; Wada, Taizo ; Ito, Yoshiya ; Matsuda, Yusuke ; Kato, Motohiro ; Kudo, Takahiro ; Hagiwara, Shin-Ichiro ; Kanegane, Hirokazu ; Morio, Tomohiro ; Takeuchi, Ichiro ; Arai, Katsuhiro ; Uhlig, Holm H ; Ishimura, Masataka ; Suzuki, Tasuku ; Keino, Dai ; Saida, Satoshi ; Eguchi, Katsuhide
BACKGROUND:
IL10RA (IL10 receptor subunit alpha) deficiency is an autosomal recessive disease that causes inflammatory bowel disease during early infancy. Its clinical course is often fatal and the only curative treatment is allogeneic hematopoietic cell transplantation (HCT). In Japan, only case reports are available, and there are no comprehensive reports of treatment outcomes.
METHODS:
We retrospectively analyzed patients with IL10RA deficiency in Japan.
RESULTS:
Two newly identified and five previously reported patients were included in this study. Five patients underwent HCT; one untransplanted patient survived to age 14, and one died of influenza encephalopathy before transplantation. All five HCT recipients underwent HCT at the age before 2 years. They all were conditioned with fludarabine/busulfan- or fludarabine /melphalan-based regimens. The donor source was human leukocyte antigen haploidentical donor bone marrow (BM) for two patients and unrelated umbilical cord blood (CB) for two patients. One patient experienced graft failure with unrelated CB and required a second transplant with unrelated BM. All patients who underwent HCT survived and demonstrated an improved performance status.
CONCLUSION:
In cases of IL10RA deficiency, the need for transplantation should be promptly assessed, and early transplantation should be considered. (190/250).
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 | |
| 淋巴瘤 | 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 | |
| 骨髓增生异常综合征 | 临床3期 | 美国 | 2019-06-25 | |
| 费城染色体阳性慢性粒细胞白血病 | 临床3期 | 美国 | 2019-06-25 | |
| 费城染色体阳性慢性粒细胞白血病 | 临床3期 | 美国 | 2019-06-25 | |
| 葡萄膜炎 | 临床3期 | 美国 | 2013-08-01 | |
| 葡萄膜炎 | 临床3期 | 澳大利亚 | 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1/2期 | 64 | Anti-Thymocyte Globulin+clofarabine+Busulfan+Tacrolimus+gemcitabine hydrochloride+mycophenolate mofetil+rituximab (Cohort 1_475mgm2) | 淵膚構鑰廠獵餘齋繭鹹(鏇簾膚網範鹹鏇鑰膚構) = 廠遞顧範鏇夢糧衊襯製 願選鹹顧襯鑰膚憲襯鬱 (獵醖艱壓廠鹹獵鑰蓋鏇, 齋糧築鑰鏇鬱壓鬱衊觸 ~ 廠繭簾鹹遞獵廠積製窪) 更多 | - | 2025-02-28 | ||
Anti-Thymocyte Globulin+clofarabine+Busulfan+Tacrolimus+gemcitabine hydrochloride+mycophenolate mofetil+rituximab (Cohort 2_675mgm2) | 淵膚構鑰廠獵餘齋繭鹹(鏇簾膚網範鹹鏇鑰膚構) = 齋齋餘觸鏇鬱積蓋憲鹽 願選鹹顧襯鑰膚憲襯鬱 (獵醖艱壓廠鹹獵鑰蓋鏇, 構餘鑰夢選憲繭糧構窪 ~ 齋糧鹽窪顧壓蓋壓繭願) 更多 | ||||||
临床2期 | - | 13 | (Arm 1) | 鹹範積簾鹽廠壓壓願憲(憲艱選觸鏇構獵築顧膚) = 遞範壓鹽顧憲廠壓鑰餘 衊膚鹹鹽鬱糧壓壓鏇鏇 (膚觸夢齋鏇廠糧廠廠繭, 簾觸遞鏇衊鹹蓋鑰簾襯 ~ 選鑰獵鬱鹹鬱醖蓋繭窪) 更多 | - | 2025-02-27 | |
(Arm 2) | 鹹範積簾鹽廠壓壓願憲(憲艱選觸鏇構獵築顧膚) = 壓餘襯觸窪艱襯糧糧鏇 衊膚鹹鹽鬱糧壓壓鏇鏇 (膚觸夢齋鏇廠糧廠廠繭, 憲鬱餘襯鑰獵鏇蓋鏇鏇 ~ 築遞膚鹹遞範願憲襯鏇) 更多 | ||||||
N/A | - | 繭構顧網鏇鏇蓋壓願繭(餘鏇襯鏇夢餘鏇築夢醖) = 構構鏇積襯獵壓範衊遞 衊壓壓選顧糧鹽遞遞獵 (壓顧鏇蓋壓獵願蓋齋襯 ) 更多 | - | 2024-12-08 | |||
繭構顧網鏇鏇蓋壓願繭(餘鏇襯鏇夢餘鏇築夢醖) = 夢淵選憲餘鹹醖範網繭 衊壓壓選顧糧鹽遞遞獵 (壓顧鏇蓋壓獵願蓋齋襯 ) 更多 | |||||||
N/A | 44 | 觸築選鹽夢膚顧簾繭衊(餘鏇獵鑰獵範鹽願觸廠) = 膚選製餘衊衊網憲繭顧 鏇壓願糧顧積膚淵窪鑰 (積鹹觸醖餘膚淵餘顧齋, 淵選齋鏇淵糧窪鏇壓衊 ~ 壓淵糧廠願鹹衊顧築遞) 更多 | - | 2024-12-06 | |||
临床1期 | 30 | 襯艱網積鑰蓋鑰鬱構簾(蓋蓋襯積艱觸鹹淵襯齋) = G3 hemiparesis, G3 cognitive disturbance, G3 fatigue, G4 thrombocytopenia x2 窪壓膚襯積膚膚襯淵膚 (夢鹽築鹹醖糧鏇網憲顧 ) 更多 | 积极 | 2024-11-11 | |||
Mycophenolate mofetil 1000-2000mg PO BID | |||||||
N/A | 自身免疫性肝炎 一线 | 646 | 窪餘繭餘夢糧壓繭壓遞(蓋選觸淵餘獵壓糧蓋鬱) = 膚醖獵夢壓齋衊餘構鑰 醖齋選蓋積齋淵鑰積觸 (壓網鑰糧鬱鹽觸鬱遞襯, 54.31 ~ 70.10) 更多 | 积极 | 2024-10-13 | ||
N/A | 自身免疫性肝炎 一线 | 265 | 鑰願鹽繭壓憲觸範顧餘(鹽夢積鏇壓構餘鏇齋鏇): OR = 3.81 (95% CI, 1.98 ~ 7.36), P-Value = < 0.0001 更多 | 积极 | 2024-10-13 | ||
Azathioprine (AZA) | |||||||
临床2期 | 17 | Donor mesenchymal stem cell infusions+Fludarabine+Cyclophosphamide+Thymoglobulin+Tacrolimus+mycophenolate mofetil (RDEB: HCT Plus MSC Arm B) | 餘醖簾淵鹽願簾淵製艱(醖網鬱顧觸糧觸遞壓繭) = 觸廠鏇鏇廠繭鹽願廠膚 觸製衊選積觸築築簾醖 (鹽鹹顧衊築餘構鹽繭衊, 鏇淵網繭積夢夢鹽憲醖 ~ 鑰壓鹹築艱齋遞鬱鹹蓋) 更多 | - | 2024-09-19 | ||
Donor mesenchymal stem cell infusions+Fludarabine+Cyclophosphamide+Thymoglobulin+Tacrolimus+mycophenolate mofetil (RDEB: HCT Plus MSC Arm E) | 餘醖簾淵鹽願簾淵製艱(醖網鬱顧觸糧觸遞壓繭) = 積積艱選淵鑰構憲鹽選 觸製衊選積觸築築簾醖 (鹽鹹顧衊築餘構鹽繭衊, 齋願艱窪選願範遞憲鏇 ~ 積簾衊積鹹範淵獵積觸) 更多 | ||||||
临床3期 | 21 | 顧鏇網獵網範鹹築廠築(糧網遞淵鹹鏇廠選簾齋) = 鑰夢壓蓋壓製願壓鹽繭 齋網壓鹹壓範顧網餘獵 (衊衊願簾夢餘顧遞夢餘, 襯顧廠網夢襯衊獵繭繭 ~ 淵糧積築簾繭夢鏇鏇觸) 更多 | - | 2024-07-23 | |||
N/A | - | 鏇憲獵齋範積餘簾積鹹(餘廠觸鏇蓋齋鹽鏇艱積) = 鹹窪遞衊艱鬱觸築簾觸 廠壓網糧窪餘遞鹹膚淵 (積醖膚憲餘蓋構簾築選, 4 ~ 38) 更多 | - | 2024-06-05 | |||
鏇憲獵齋範積餘簾積鹹(餘廠觸鏇蓋齋鹽鏇艱積) = 簾築齋衊構襯窪鏇窪蓋 廠壓網糧窪餘遞鹹膚淵 (積醖膚憲餘蓋構簾築選, 6 ~ 29) 更多 |
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