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CPX-351 for Higher Risk Myelodysplastic Syndrome: Cytarabine and Daunorubicin in Disguise?

Petra MuusTop Scholar,Theo de WitteTop Scholar

The Lancet Haematology(2023)

Leeds Teaching Hosp NHS Trust

Cited 0|Views4
Abstract
Higher-risk myelodysplastic syndrome is a heterogenous disease with a poor prognosis that occurs in older people. 1 Greenberg PL Myelodysplastic syndromes, thy name is heterogeneity. Br J Haematol. 2023; 201: 381-382 Crossref PubMed Scopus (0) Google Scholar Current treatment regimens usually result in a transient and often less than complete response. Only allogeneic haematopoietic stem-cell transplantation (HSCT) can potentially cure a select group of relatively young and fit patients. These patients usually receive remission induction therapy before allogeneic HSCT, but in the absence of well designed, prospective randomised clinical trials, its value remains uncertain. 2 de Witte T Bowen D Robin M et al. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood. 2017; 129: 1753-1762 Crossref PubMed Scopus (220) Google Scholar CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 studyCPX-351 appears to be active and safe in patients with higher-risk myelodysplastic syndrome and chronic myelomonocytic leukaemia, allowing bridging to allogenic HSCT in most patients. Full-Text PDF
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Hematopoietic Cell Transplantation
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要点】:研究探讨了CPX-351在治疗高风险髓样发育不良综合征(MDS)中的疗效和安全性,发现其能有效桥接至同种异体造血干细胞移植(HSCT)。

方法】:采用多中心、单臂、二期临床研究方法。

实验】:在多中心研究中对CPX-351进行了测试,具体数据集名称未提及,结果显示CPX-351在大多数患者中安全有效。