Department of General, Visceral and Transplantation Surgery, University of Heidelberg, Im Neuenheimer Feld 110, 69120 Heidelberg, Germany.
BMC Cancer. 2014 Mar 10;14:168. doi: 10.1186/1471-2407-14-168.
Pancreatic cancer is the third most common cancer related cause of death. Even in the 15% of patients who are eligible for surgical resection the outlook is dismal with less than 10% of patients surviving after 5 years. Allogeneic hematopoietic (allo-HSCT) stem cell transplantation is an established treatment capable of to providing cure in a variety of hematopoietic malignancies. Best results are achieved when the underlying neoplasm has been turned into a stage of minimal disease by chemotherapy. Allo-HSCT in advanced solid tumors including pancreatic cancer have been of limited success, however studies of allo-HSCT in solid tumors in minimal disease situations have never been performed. The aim of this trial is to provide evidence for the clinical value of allo-HSCT in pancreatic cancer put into a minimal disease status by effective surgical resection and standard adjuvant chemotherapy.
METHODS/DESIGN: The STEM PACE trial is a single center, phase II study to evaluate adjuvant allogeneic hematopoietic stem cell transplantation in pancreatic cancer after surgical resection. The study will evaluate as primary endpoint 2 year progression free survival and will generate first time state-of-the-art scientific clinical evidence if allo-HSCT is feasible and if it can provide long term disease control in patients with effectively resected pancreatic cancer. Screened eligible patients after surgical resection and standard adjuvant chemotherapy with HLA matched related stem cell donor can participate. Patients without a matched donor will be used as a historical control. Study patients will undergo standard conditioning for allo-HSCT followed by transplantation of allogeneic unmanipulated peripheral blood stem cells. The follow up of the patients will continue for 2 years. Secondary endpoints will be evaluated on 7 postintervention visits.
The principal question addressed in this trial is whether allo-HSCT can change the unfavourable natural course of this disease. The underlying hypothesis is that allo-HSCT has the capacity to provide long-term disease control to an extent otherwise not possible in pancreatic cancer, thereby substantially improving survival of affected patients.
This trial has been registered: ISRCTN47877138.
胰腺癌是第三大常见癌症相关死亡原因。即使在 15%有资格接受手术切除的患者中，预后也很糟糕，不到 10%的患者在 5 年后存活。异基因造血（allo-HSCT）干细胞移植是一种已确立的治疗方法，可在多种血液恶性肿瘤中提供治愈。当基础肿瘤通过化疗转化为疾病最小阶段时，可获得最佳效果。allo-HSCT 在晚期实体瘤（包括胰腺癌）中的效果有限，但在疾病最小化情况下对 allo-HSCT 在实体瘤中的研究从未进行过。本试验的目的是提供通过有效手术切除和标准辅助化疗将胰腺癌置于疾病最小化状态后进行 allo-HSCT 的临床价值的证据。
方法/设计：STEM PACE 试验是一项单中心、二期研究，旨在评估手术后胰腺切除术后辅助性异基因造血干细胞移植在胰腺癌中的应用。该研究将以 2 年无进展生存率为主要终点，并将首次提供 allo-HSCT 在有效切除的胰腺癌患者中是否可行以及是否能提供长期疾病控制的最先进的科学临床证据。可通过筛选出手术后和标准辅助化疗后具有 HLA 匹配相关干细胞供体的合格患者参与。无匹配供体的患者将作为历史对照。研究患者将接受 allo-HSCT 的标准预处理，然后移植未处理的异体外周血干细胞。患者的随访将持续 2 年。次要终点将在 7 次干预后访视中进行评估。
本试验主要解决的问题是 allo-HSCT 是否可以改变该疾病的不良自然病程。基本假设是 allo-HSCT 有能力提供长期疾病控制，在胰腺癌中这是不可能的，从而显著改善受影响患者的生存。
本试验已注册：ISRCTN47877138。
