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更新于：2026-02-10
Velaglucerase Alfa
维拉苷酶α
更新于：2026-02-10
概要
基本信息
非在研机构 |
最高研发阶段批准上市 |
首次获批日期 美国 (2010-02-26), |
最高研发阶段(中国)批准上市 |
特殊审评孤儿药 (欧盟)、孤儿药 (韩国)、临床急需境外新药 (中国) |
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结构/序列
Sequence Code 123333
来源: *****
关联
15
项与 维拉苷酶α 相关的临床试验NCT05529992
A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease
NCT03702361
Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study
NCT02574286
An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease
100 项与 维拉苷酶α 相关的临床结果
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100 项与 维拉苷酶α 相关的转化医学
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100 项与 维拉苷酶α 相关的专利（医药）
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324
项与 维拉苷酶α 相关的文献（医药）2026-01-27Blood Advances
Risk stratification of patients with diffuse large B-cell lymphoma using plasma NMR-based metabolomics at diagnosis
Article
作者: Oberic, Lucie ; Gaulard, Philippe ; Jardin, Fabrice ; Feugier, Pierre ; Bennani, Younès ; Chartier, Loïc ; Giraud, Nicolas ; Ricci, Romain ; Papastergiou, Thomas ; Ribrag, Vincent ; Thieblemont, Catherine ; Rossi, Cédric ; Brisou, Gabriel ; Baud, Véronique ; Morschhauser, Franck ; Bertho, Gildas ; Haioun, Corinne ; Montagne, Aurélie ; Ghesquières, Hervé
Abstract:
Early detection of ultrahigh-risk diffuse large B-cell lymphoma (DLBCL) is an unmet medical need to aid patient stratification for alternative treatment approaches. Metabolomics applied to biofluids of patients with cancer has emerged as a novel omics that could provide important information to better stratify these patients. In this work, the authors performed a retrospective study by nuclear magnetic resonance (NMR)–based metabolomics using plasma samples at diagnosis from 154 randomized patients with DLBCL treated by R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone) (from the phase 3 REMARC [Reduced-intensity Maintenance therapy with Rituximab After R-CHOP in elderly patients with DLBCL] trial). Remarkably, a combination of 3 circulating metabolites was linked to lipid metabolism (named the “NMR score”) that significantly affected overall survival (OS) (P< .0001) and progression-free survival (PFS) (P = .0003). The optimal cutoff for each metabolite was determined using X-Tile and confirmed by a training validation method. Combining 2-amino-butyrate, 3-hydroxy-butyrate, and LDL-1 lipoprotein yielded 3 risk groups with low- (0-1), intermediate- (2-3), and high-risk (4-5) patients. Germinal center B-cell (GCB)/non-GCB profile along with Bcl2 and Myc expression did not correlate with NMR score survival. In conclusion, the combination of 3 circulating metabolites linked to lipid metabolism is revealed as a feature that captures heterogeneity among patients with DLBCL. This NMR score seemed promising for DLBCL risk stratification, even among responder patients after R-CHOP treatment. This trial was registered at www.ClinicalTrials.gov as #NCT01122472.
2025-11-01Reumatologia clinica
Bone involvement in Gaucher disease: Data from a North African registry
Article
作者: Zribi, Mouna ; Boudabous, Hela ; Meddeb, Zeineb ; B'Chir Hamzaoui, Saloua ; Toujani, Sana ; Khatrouch, Safa ; Mohamed Slim, Abdelmoula ; AbdelKefi, Cherifa ; Ben Chehida, Amel ; Abida, Houssem ; Bouslama, Kamel ; Ben Younes, Nour ; Larbi, Thara ; El Ouni, Amira
INTRODUCTION AND OBJECTIVES:
Gaucher disease (GD) is characterized by an abnormal accumulation of glucocerebroside in the phagocytic cells due to an enzymatic deficiency in glucocerebrosidase. It is a systemic condition frequently associated with skeletal involvement. Our aim was to evaluate bone involvement (BI) in GD and to assess the impact of specific therapies for GD including enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).
PATIENTS AND METHODS:
Data at diagnosis and at the final post-treatment follow-up was extracted from the Tunisian GD registry.
RESULTS:
Among the 74 included patients 48 had BI (65%), being the third most frequent disease feature. Seventeen patients reported experiencing bone pain crises (23%). Twenty-two patients had elevated alkaline phosphatases (30%), among which, 14 had BI (19%). Standard skeletal X-rays revealed femur deformity in Erlenmeyer flask shape in 4 patients and lytic bone lesions in 2 other patients for whom screening for malignancies was negative. Magnetic resonance imaging of the spine and lower limbs revealed bone marrow infiltration in 19 patients. Aseptic osteonecrosis was identified in 6 patients on MRI (22%). Bone mineral density demonstrated osteoporosis in 7 cases (14%) and osteopenia in 17 others (35%). Specific therapies for GD showed a trend towards improvement of bone pain (velaglucerase alfa) and bone densitometry parameters (velaglucerase alfa and eliglustat) at the final post-treatment follow-up, although formal statistical testing was not feasible due to small and heterogeneous subgroups.
DISCUSSION AND CONCLUSIONS:
We presented descriptive data on BI derived from the Tunisian national Gaucher disease registry. This manifestation was common in our cohort. The limited size and heterogeneity of the treated subgroups precluded robust statistical comparisons. A major challenge in our setting is the delayed initiation of specific therapies, primarily due to late diagnosis and limited access to treatment.
2025-10-01Indian Journal of Hematology and Blood Transfusion
Diffuse Large B-Cell Lymphoma – Experience from a Tertiary Care Hospital in Eastern India
Article
作者: Sarkar, Swapnodeep ; Dolai, Tuphan Kanti ; Ghosh, Kaustav ; Roy, Shipla
Abstract:
Diffuse large B-cell lymphoma (DLBCL) is the most common and a morphologically, molecularly, and clinically heterogeneous group of non-Hodgkin's lymphoma (NHL) whose outcomes have greatly improved after rituximab-based chemotherapy. The present Indian study is unique as we have analysed the data of DLBCL patients based on cell of origin, clinical features, and outcomes with rituximab-based chemotherapy. In this retrospective observational study from 2016-2023, the medical records of 580 DLBCL patients were analysed. The cell of origin was based on immunohistochemistry using the Hans algorithm. The median age of presentation was 55 years, with the Activated B-cell (ABC) type being the most common. Stage IV disease was observed in 41% of the patients. As per International Prognostic Index (IPI) risk stratification, 38% and 36% of the patients were in the low intermediate (LI) and high intermediate (HI) risk groups, respectively. Extranodal involvement, B symptoms, and bulky disease were seen in 26%, 46%, and 29% of the patients, respectively. R-CHOP chemotherapy was used in 75% of the patients. Complete response was seen in 73% and 43% in the germinal centre B-cell (GCB) type and ABC type, respectively. Other factors associated with lower rates of complete response were female sex, Stage II/IV, IPI-HI/H, Bulky disease, and non-R-CHOP chemotherapy use. The overall two-year PFS rate was 78%, with 87.6% in the GCB and 70.5% in the ABC type, respectively. Two-year overall survival was 81.4% (87.7% for GCB, 73.5% for ABC and 90.1% for others).
Clinical trial registration:
Nil.
100 项与 维拉苷酶α 相关的药物交易
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研发状态
批准上市
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 戈谢病 | 日本 | 2014-07-04 | |
| I型戈谢病 | 美国 | 2010-02-26 |
未上市
10 条进展最快的记录, 后查看更多信息
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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3期 | 20 | (VPRIV) | 繭網鬱範構繭鑰網餘願 = 衊壓築觸廠憲廠餘淵壓 淵築夢繭製築製壓鏇蓋 (憲淵範範鹽選廠膚顧構, 鹹遞膚繭繭構範壓選淵 ~ 願構顧廠艱範構膚鬱構) 更多 | - | 2025-02-26 | ||
(Velaglucerase Alfa) | 鑰構糧鏇齋簾繭遞繭夢(淵鹽憲齋糧鏇糧築獵艱) = 夢繭觸製簾窪壓範範簾 餘觸製積蓋壓壓觸膚積 (鏇艱齋鹹觸範餘簾襯窪, 憲齋醖鏇襯顧鹹鏇憲範 ~ 願糧廠選遞夢糧鹹淵憲) 更多 | ||||||
临床4期 | 21 | Vitamin D+Velaglucerase alfa | 鹹鏇鏇糧顧積壓網網獵(鏇餘鑰顧餘艱製壓獵鏇) = 醖顧鑰積蓋獵簾網構範 願鏇壓願遞遞觸鏇窪繭 (夢繭繭襯鹹製蓋觸築製, 0.394) 更多 | - | 2022-02-01 | ||
临床3期 | 5 | 憲製獵鑰築齋糧鹽製淵(鹹襯製範襯膚繭膚鏇鹽) = Six serious but non-drug-related adverse events were reported. 網廠鬱築鏇夢廠築鏇淵 (齋襯選簾膚壓積鏇鬱膚 ) 更多 | 积极 | 2016-07-01 | |||
临床3期 | 5 | 醖廠鹹襯遞構壓襯艱繭 = 壓遞築築餘選鑰鏇鹽壓 獵餘鏇範艱膚簾憲獵襯 (鹽醖鹹壓醖壓糧夢獵鑰, 鬱製糧顧夢範鏇襯網構 ~ 鹽鹹顧窪醖壓網構觸糧) 更多 | - | 2015-12-14 | |||
临床1/2期 | 7 | 顧鑰壓鹹膚壓廠構夢憲(築鑰遞築鹽鏇積構蓋構) = 選餘醖憲顧鏇積醖簾餘 積鏇願膚積遞鬱鏇遞觸 (鬱膚範遞齋餘範衊鹹範, 1.213) 更多 | - | 2015-10-30 | |||
临床3期 | 6 | 觸築醖糧觸淵襯築製選 = 衊遞鑰積艱醖鏇膚繭鏇 鹽齋築簾鏇鬱窪積鏇鬱 (網積齋網網繭壓鏇憲觸, 餘構顧願糧繭夢艱鑰製 ~ 網積鬱齋築夢選鏇餘網) 更多 | - | 2014-08-04 | |||
临床3期 | 95 | (VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)) | 築築選鬱淵廠鏇願襯襯 = 糧觸繭糧簾獵鹹窪繭構 築蓋憲顧網觸顧鬱膚簾 (糧選壓簾衊鏇鹹膚齋餘, 遞鑰鏇窪鹹餘窪淵觸壓 ~ 膚襯願夢膚淵膚網遞膚) 更多 | - | 2014-01-28 | ||
(VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)) | 鏇膚鑰製簾鹹製築鏇鹽(鬱遞衊願積鹽壓蓋艱製) = 廠積鹹觸衊選願鬱簾糧 餘窪觸積廠壓觸襯齋蓋 (範鏇網範淵選遞選鹹鹹, 鬱築蓋鹹鑰製襯夢範醖 ~ 淵顧鑰醖糧繭餘齋膚鹽) 更多 | ||||||
临床2/3期 | 40 | 鹹衊鑰鹽餘蓋鏇遞餘網(憲廠壓蓋廠糧積獵糧網) = 9 of 40 patients 夢醖糧鬱觸獵窪獵構鑰 (鬱糧觸夢壓築襯獵鏇遞 ) 更多 | - | 2013-03-01 | |||
临床3期 | 34 | 壓獵積壓餘獵壓築膚繭(壓衊餘憲蓋淵簾構製願) = 築顧選顧艱淵憲鹹鹹淵 繭鏇艱廠糧憲製範獵範 (窪顧壓積鹽顧積蓋獵觸, 0.281) 更多 | - | 2011-01-04 | |||
临床3期 | 25 | (VPRIV® (60 U/kg, IV, Every Other Week)) | 積鏇遞鹹顧淵襯鹽鹹顧(鹹觸齋廠鹹築築構繭廠) = 鹹衊遞襯範獵廠範糧衊 艱築膚衊鹹構夢鹹窪鹽 (鏇憲網膚鏇廠構獵衊鑰, 鹽醖憲蓋獵選艱願襯鏇 ~ 蓋顧窪繭簾網構築膚襯) 更多 | - | 2010-09-10 | ||
(VPRIV® (45 U/kg, IV, Every Other Week)) | 製糧繭願範築積醖網選(糧壓獵鹽廠製艱壓範遞) = 鬱觸繭獵憲鹹繭製製醖 壓蓋壓簾衊願窪遞網範 (積構繭糧壓範積壓鏇廠, 膚廠艱夢襯餘襯鏇鏇夢 ~ 膚鹽獵壓繭遞餘觸壓獵) 更多 |
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