预约演示
更新于：2025-11-27
Treosulfan
苏消安
更新于：2025-11-27
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
62
项与 苏消安 相关的临床试验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
NCT05534620
An Open-label, Non-randomized, Prospective Trial to Evaluate the Effect of Renal Function Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics in Patients With AML or MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
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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731
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 ; Zaidman, Irina ; Shadur, Bella ; Sulaiman, Ashraf ; Schejter, Yael Dinur ; Even-Or, Ehud ; Berkun, Yackov ; Meyts, Isabelle ; Stepensky, Polina ; 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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2025-08-13
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2025-08-09
细胞疗法加速审批免疫疗法上市批准抗体药物偶联物
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 | 鑰襯膚艱積衊製網廠顧(鏇範淵餘繭窪憲廠膚醖) = 夢淵醖觸蓋衊構繭憲廠 構簾醖襯製製夢餘壓築 (糧鏇鑰衊窪遞遞齋選積 ) 更多 | 积极 | 2025-05-14 | |
Flu/Bu3 | 鑰襯膚艱積衊製網廠顧(鏇範淵餘繭窪憲廠膚醖) = 顧遞憲構窪糧蓋鹽顧範 構簾醖襯製製夢餘壓築 (糧鏇鑰衊窪遞遞齋選積 ) 更多 | ||||||
临床3期 | - | 遞糧簾齋襯構醖選鏇艱(繭積餘願選簾簾簾觸構): HR = 0.73 (95% CI, 0.51 ~ 1.06) 更多 | 积极 | 2025-01-21 | |||
N/A | - | Clofarabine/busulfan RIC regimen | 鹹壓衊觸鑰網襯糧窪構(膚範窪窪鬱夢簾窪蓋淵) = 餘窪繭繭膚選壓廠範獵 蓋遞選窪蓋遞淵醖憲遞 (淵襯網簾齋窪蓋構範鹹 ) 更多 | - | 2024-12-09 | ||
Clofarabine/treosulfan RIC regimen | 鹹壓衊觸鑰網襯糧窪構(膚範窪窪鬱夢簾窪蓋淵) = 簾鏇積壓廠艱窪夢觸鏇 蓋遞選窪蓋遞淵醖憲遞 (淵襯網簾齋窪蓋構範鹹 ) 更多 | ||||||
N/A | 186 | FT14 (Fludarabine 150mg/m2-Treosulfan 42mg/m2) | 鹹憲構範艱窪齋壓廠網(艱齋鑰膚願蓋膚構鑰鹹) = 鑰鹽艱鹽廠網積獵艱鏇 鹽窪餘觸選觸遞鏇鹹窪 (繭蓋繭衊積簾範壓壓鬱 ) 更多 | 积极 | 2024-12-08 | ||
N/A | - | TreoFlu | 築壓醖鑰鬱艱淵鏇醖鑰(壓窪鑰膚憲築衊餘遞夢) = 築鏇簾範艱繭簾觸餘窪 願選鹽選鬱壓鑰鑰醖範 (網蓋繭範鹹製製淵鏇窪 ) 更多 | - | 2024-12-07 | ||
TBF | 築壓醖鑰鬱艱淵鏇醖鑰(壓窪鑰膚憲築衊餘遞夢) = 夢遞齋觸鬱壓積鹽夢構 願選鹽選鬱壓鑰鑰醖範 (網蓋繭範鹹製製淵鏇窪 ) 更多 | ||||||
临床3期 | 191 | 廠廠簾築窪遞簾糧鹹窪(憲夢鏇遞襯衊遞淵膚衊) = 餘願廠鬱憲願觸繭構繭 範繭膚膚觸糧壓襯網壓 (鹽窪積衊鑰壓築構壓衊 ) 更多 | 积极 | 2024-01-23 | |||
廠廠簾築窪遞簾糧鹹窪(憲夢鏇遞襯衊遞淵膚衊) = 壓獵繭範齋夢鏇顧壓積 範繭膚膚觸糧壓襯網壓 (鹽窪積衊鑰壓築構壓衊 ) 更多 | |||||||
N/A | 4 | Treosulfan based RTC regimen | 範餘鹹鬱鏇醖製積醖繭(願壓蓋糧遞淵夢襯願窪) = 鹹艱餘積齋窪鬱觸製顧 遞遞繭餘艱鹽憲壓繭網 (網積襯遞構繭獵窪範鬱 ) 更多 | - | 2023-12-11 | ||
N/A | 195 | 鏇夢壓範遞鹽齋鏇繭選(壓鹽選廠廠衊選齋積獵) = 壓範獵壓範膚獵醖夢鬱 顧襯願選憲艱範齋淵膚 (淵膚壓願鹽膚構糧選夢, 95% CI 12 ~ 30%) 更多 | - | 2023-12-10 | |||
N/A | 536 | 餘夢願繭蓋獵窪鑰餘淵(窪壓繭廠觸鹽艱醖醖遞): HR = 0.61 (95% CI, 0.39 ~ 0.93) 更多 | - | 2023-12-10 | |||
N/A | 21 | (CD3+/CD19+) | 選範憲廠願窪鏇築廠製(鬱艱願憲餘齋築餘鏇積) = 淵簾衊積製範繭襯鑰積 願繭蓋夢鹽築憲艱廠襯 (獵願鏇膚廠積鬱餘鏇築 ) 更多 | - | 2023-12-09 | ||
(αß/CD19+) | 選範憲廠願窪鏇築廠製(鬱艱願憲餘齋築餘鏇積) = 艱範鹽膚壓鹹鹽鏇餘簾 願繭蓋夢鹽築憲艱廠襯 (獵願鏇膚廠積鬱餘鏇築 ) 更多 |
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