Efficacy and safety of autologous peripheral blood stem cell transplantation for Philadelphia chromosome-positive acute lymphoblastic leukemia: A study protocol for a multicenter exploratory prospective study (Auto-Ph17 study).
Efficacy and safety of autologous peripheral blood stem cell transplantation for Philadelphia chromosome-positive acute lymphoblastic leukemia: A study protocol for a multicenter exploratory prospective study (Auto-Ph17 study).
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自体外周血干细胞移植对费城染色体阳性急性淋巴细胞白血病的功效和安全性：多中心探索性前瞻性研究的研究方案（自动-PH17研究）。
DOI：
10.1097/md.0000000000009568
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发表时间：
2017-12
期刊：
影响因子：
1.6
通讯作者：
Kiyoi H
中科院分区：
文献类型：
--
作者：
Nishiwaki S;Sugiura I;Miyata Y;Saito S;Sawa M;Nishida T;Miyamura K;Kuwatsuka Y;Kohno A;Yuge M;Kasai M;Iida H;Kurahashi S;Osaki M;Goto T;Terakura S;Murata M;Nishikawa H;Kiyoi H
费城染色体阳性急性淋巴细胞白血病（Ph + ALL）的预后已显着改善，因为引入酪氨酸激酶抑制剂（TKI）。 虽然异基因造血细胞移植（allo-HCT）是一种主要的治疗选择，但自体外周血干细胞移植（auto-PBSCT）的作用已被重新考虑，特别是在早期分子缓解的患者中。这是一项多中心探索性研究，研究对象为年龄在55 - 70岁之间、在3个化疗周期内达到完全分子学缓解的Ph+ALL患者。 目标样本量为5例，注册期为2年。主要终点是移植后第100天的死亡率，次要终点是生存率、复发率、非复发死亡率和不良事件。本研究分为3个阶段：外周血干细胞采集、移植和维持。使用环磷酰胺（CPM）、多柔比星、长春新碱（VCR）和泼尼松龙（PSL）的组合进行化学动员。作为准备方案，使用LEED方案，其由美法仑、CPM、依托泊苷和地塞米松组成。使用VCR、PSL和达沙替尼的组合进行12个周期的维持治疗。分析自体外周血干细胞移植前后BCR-ABL嵌合基因和T细胞亚群的微小残留病（MRD）与复发的关系。该方案得到了名古屋大学医院和所有参与医院的机构审查委员会的批准。根据赫尔辛基宣言，在登记前获得所有患者的书面知情同意书。研究结果将通过同行评审期刊上的出版物传播。试验注册号UMIN 000026445。
The prognosis of Philadelphia chromosome positive acute lymphoblastic leukemia (Ph + ALL) has been dramatically improved since the introduction of tyrosine kinase inhibitors (TKIs). Although allogeneic hematopoietic cell transplantation (allo-HCT) is a major treatment option, the role of autologous peripheral blood stem cell transplantation (auto-PBSCT) has been reconsidered, especially in patients who achieved early molecular remission. This is a multicenter exploratory study for Ph + ALL patients aged between 55 and 70 years who achieved complete molecular remission within 3 cycles of chemotherapy. The target sample size is 5, and the registration period is 2 years. The primary endpoint is Day100- mortality after transplantation, and the secondary endpoints are survival, relapse rate, nonrelapse mortality, and adverse events. This study is divided into 3 phases: peripheral blood stem cell harvest, transplantation, and maintenance. Chemomobilization is performed using a combination of cyclophosphamide (CPM), doxorubicin, vincristine (VCR), and prednisolone (PSL). As a preparative regimen, the LEED regimen is used, which consists of melphalan, CPM, etoposide, and dexamethasone. Twelve cycles of maintenance therapy using a combination of VCR, PSL, and dasatinib are performed. In association with relapse, the minimal residual disease (MRD) of BCR-ABL chimeric gene and T-cell subsets are analyzed both before and after auto-PBSCT. The protocol was approved by the institutional review board of Nagoya University Hospital and all the participating hospitals. Written informed consent was obtained from all patients before registration, in accordance with the Declaration of Helsinki. Results of the study will be disseminated via publications in peer-reviewed journals. Trial registration number UMIN000026445.