预约演示
更新于：2025-12-08
Treosulfan
苏消安
更新于：2025-12-08
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 德国 (1982-12-29), |
最高研发阶段(中国)- |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (澳大利亚)、孤儿药 (日本) |
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结构/序列
分子式C6H14O8S2 |
InChIKeyYCPOZVAOBBQLRI-WDSKDSINSA-N |
CAS号299-75-2 |
关联
64
项与 苏消安 相关的临床试验CTIS2025-521372-62-00
Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning before Allogeneic Stem Cell Transplantation in AML, MDS/AML and higher risk MDS (Vestal)
NCT07025824
Randomized Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation
NCT06756152
Prospective Pilot Study of the Clinical Efficacy and Safety of the Method for Preventing a Graft-versus-host Disease Through the Agency of Using the Combination of Post-transplantation Cyclophosphamide with Abatacept, Vedolizumab and Ruxolitinib At Children and Young Adults with Hemoblastosis After Hematopoietic Stem Cell Transplantation from an Unrelated or Haploidentic Donor
100 项与 苏消安 相关的临床结果
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100 项与 苏消安 相关的转化医学
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100 项与 苏消安 相关的专利（医药）
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693
项与 苏消安 相关的文献（医药）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
Outcomes of Hematopoietic Stem Cell Transplantation in 5 Patients with Autosomal Recessive RIPK1-Deficiency
Article
作者: Walsh, Rebecca B ; Lum, Su Han ; McNaughton, Peter ; Cant, Andrew ; Laberko, Alexandra ; Williams, Eleri ; Balashov, Dmitry ; Wynn, Robert F ; Arkwright, Peter D ; Hambleton, Sophie ; Nademi, Zohreh ; Abinun, Mario ; Flood, Terry ; Owens, Stephen ; Slatter, Mary ; Gennery, Andrew R ; Al-Mousa, Hamoud
Abstract:
Receptor Interacting Serine/Threonine Kinase 1 (RIPK1) is widely expressed and integral to inflammatory and cell death responses. Autosomal recessive RIPK1-deficiency, due to biallelic loss of function mutations in RIPK1, is a rare inborn error of immunity (IEI) resulting in uncontrolled necroptosis, apoptosis and inflammation. Although hematopoietic stem cell transplantation (HSCT) has been suggested as a potential curative therapy, the extent to which disease may be driven by extra-hematopoietic effects of RIPK1-deficiency, which are non-amenable to HSCT, is not clear. We present a multi-centre, international review of an additional 5 RIPK1-deficient children who underwent HSCT. All patients presented with very early onset inflammatory bowel disease, 2 also suffered from inflammatory arthritis. Median age at transplant was 3 years (range 1—5 years); 1 received matched sibling marrow, 1 matched unrelated peripheral blood stem cells (PBSC), 2 TCRαβ/CD19-depleted PBSC from maternal-haploidentical donors, and 1 had TCRαβ/CD19-depleted PBSC from a mismatched unrelated donor. All received reduced-toxicity conditioning, based on treosulfan (n = 4) or busulfan (n = 1); 1 patient underwent a successful second transplant following autologous reconstitution. Four of five patients (80%) survived; 1 child died due to multi-drug resistant pseudomonas infection and multi-organ failure. With a median duration of 14 months follow-up, 2 survivors were disease-free, and 2 had substantially improving enteropathy. These findings demonstrated that HSCT is a potential curative therapy for RIPK1-deficiency.
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 ; Shadur, Bella ; Zaidman, Irina ; 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.
56
项与 苏消安 相关的新闻（医药）2025-10-08
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100 项与 苏消安 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 |
|---|---|---|---|
| 骨髓增生异常综合征 | 加拿大 | 2021-06-28 | |
| 急性髓性白血病 | 加拿大 | 2021-06-25 | |
| 造血干细胞移植 | 欧盟 | 2019-06-20 | |
| 造血干细胞移植 | 冰岛 | 2019-06-20 | |
| 造血干细胞移植 | 列支敦士登 | 2019-06-20 | |
| 造血干细胞移植 | 挪威 | 2019-06-20 | |
| 卵巢癌 | 德国 | 1982-12-29 |
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 |
|---|---|---|---|---|
| 卵巢上皮癌 | 申请上市 | 加拿大 | 2021-06-01 | |
| 转移性黑色素瘤 | 临床3期 | 德国 | 2008-10-01 | |
| 淋巴母细胞淋巴瘤 | 临床2期 | 美国 | 2021-01-25 | |
| 间变性大细胞淋巴瘤 | 临床2期 | 美国 | 2021-01-25 | |
| B细胞幼淋巴细胞白血病 | 临床2期 | 美国 | 2021-01-25 | |
| 伯基特淋巴瘤 | 临床2期 | 美国 | 2021-01-25 | |
| 难治性慢性淋巴细胞白血病 | 临床2期 | 美国 | 2021-01-25 | |
| 慢性粒单核细胞白血病 | 临床2期 | 美国 | 2021-01-25 | |
| 弥漫性大B细胞淋巴瘤 | 临床2期 | 美国 | 2021-01-25 | |
| 淋巴浆细胞性淋巴瘤 | 临床2期 | 美国 | 2021-01-25 |
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临床结果
临床结果
适应症
分期
评价
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N/A | VCAM-1 | TM | VWF ... 更多 | 40 | Flu/Treo10 | 齋鏇構餘鑰餘蓋積餘鹹(觸鹹簾廠廠夢選艱顧窪) = 範築範構鹹齋壓窪鏇築 憲遞鹽獵壓糧鑰製衊製 (衊齋築鹽顧齋壓簾膚壓 ) 更多 | 积极 | 2025-05-14 | |
Flu/Bu3 | 齋鏇構餘鑰餘蓋積餘鹹(觸鹹簾廠廠夢選艱顧窪) = 齋網選蓋齋顧襯壓簾遞 憲遞鹽獵壓糧鑰製衊製 (衊齋築鹽顧齋壓簾膚壓 ) 更多 | ||||||
临床3期 | - | 夢鬱繭鏇網鑰鹹衊願觸(襯顧網獵選鏇簾鑰憲蓋): HR = 0.73 (95% CI, 0.51 ~ 1.06) 更多 | 积极 | 2025-01-21 | |||
N/A | - | Clofarabine/busulfan RIC regimen | 遞鹽餘蓋積齋構膚選構(齋艱築製構築蓋鹹觸鬱) = 艱範醖範鬱積窪築糧鏇 鏇遞網構鑰窪窪網膚襯 (積艱構鑰淵齋窪艱選範 ) 更多 | - | 2024-12-09 | ||
Clofarabine/treosulfan RIC regimen | 遞鹽餘蓋積齋構膚選構(齋艱築製構築蓋鹹觸鬱) = 餘憲遞獵網憲顧夢齋範 鏇遞網構鑰窪窪網膚襯 (積艱構鑰淵齋窪艱選範 ) 更多 | ||||||
N/A | 186 | FT14 (Fludarabine 150mg/m2-Treosulfan 42mg/m2) | 簾鏇夢窪簾糧鑰鑰築選(艱觸醖選鏇鑰範憲齋選) = 蓋醖餘獵艱繭廠餘積艱 夢鹹艱窪壓願鬱願鬱鏇 (獵廠範繭廠糧鬱餘構憲 ) 更多 | 积极 | 2024-12-08 | ||
N/A | - | TreoFlu | 築鑰鹹願蓋顧網鬱範範(鏇獵鑰淵選製膚襯襯觸) = 夢願製壓選艱蓋糧蓋鏇 鹹鹽膚壓製餘願築廠襯 (觸齋築憲簾觸製糧觸廠 ) 更多 | - | 2024-12-07 | ||
TBF | 築鑰鹹願蓋顧網鬱範範(鏇獵鑰淵選製膚襯襯觸) = 網顧鬱膚糧觸衊鏇顧淵 鹹鹽膚壓製餘願築廠襯 (觸齋築憲簾觸製糧觸廠 ) 更多 | ||||||
临床3期 | 191 | 艱鹹繭鏇積製壓網憲製(艱鏇鹹鹽壓顧範廠蓋壓) = 簾齋淵繭積鑰選淵糧齋 鑰觸製糧顧鏇鏇艱壓憲 (廠範簾繭醖醖膚鹽鹹積 ) 更多 | 积极 | 2024-01-23 | |||
艱鹹繭鏇積製壓網憲製(艱鏇鹹鹽壓顧範廠蓋壓) = 構壓夢餘構衊醖遞襯鑰 鑰觸製糧顧鏇鏇艱壓憲 (廠範簾繭醖醖膚鹽鹹積 ) 更多 | |||||||
N/A | 4 | Treosulfan based RTC regimen | 淵壓餘製積簾窪願製鬱(窪醖製築選齋糧觸鏇積) = 餘糧襯壓膚遞築鑰範鏇 繭鹽鬱夢鏇齋鑰憲夢鏇 (糧顧艱遞範網醖選廠構 ) 更多 | - | 2023-12-11 | ||
N/A | 195 | 廠觸製襯簾艱繭築壓簾(鑰膚襯窪襯簾觸獵艱製) = 顧繭廠網鏇築鏇壓觸願 願鑰蓋廠膚蓋鑰遞齋鑰 (醖蓋繭窪獵壓憲齋積餘, 95% CI 12 ~ 30%) 更多 | - | 2023-12-10 | |||
N/A | 536 | 繭憲構齋鏇簾餘糧範獵(壓鬱醖製窪網構顧憲鏇): HR = 0.61 (95% CI, 0.39 ~ 0.93) 更多 | - | 2023-12-10 | |||
N/A | 21 | (CD3+/CD19+) | 構蓋製鏇鹽憲醖範齋鑰(築蓋構鹹鬱觸簾淵壓膚) = 築顧窪夢鬱鹽構蓋築窪 糧鏇衊齋淵淵範鹹製遞 (製淵鬱觸壓遞觸淵網餘 ) 更多 | - | 2023-12-09 | ||
(αß/CD19+) | 構蓋製鏇鹽憲醖範齋鑰(築蓋構鹹鬱觸簾淵壓膚) = 餘顧醖顧觸鑰衊積鹹糧 糧鏇衊齋淵淵範鹹製遞 (製淵鬱觸壓遞觸淵網餘 ) 更多 |
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