Treatment with high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (aHSCT) is an intensive treatment option for patients with severe forms of systemic sclerosis (SSc). Even though associated with a high treatment related mortality, the results in this high-risk population are generally favourable. The knowledge on the potential mechanism of action of this therapy and how it can improve patients with SSc is crucial to better select the right patients for aHSCT. This is a monocentric retrospective study from Tübingen, Germany, including 32 patients who underwent aHSCT. Peripheral blood samples were analysed for different lymphocyte subsets at various timepoints before and after aHSCT. Patients were divided into responders and non-responders according to the modified Rodnan skin score and lung function test in the three years following aHSCT. Responders showed significantly lower levels of cluster of differentiation (CD)4 positive T cells in the first months after aHSCT (month 1 and 3), B cells (month 3 and 6 after aHSCT) and natural killer cells (month 1). Mantel-cox test showed a significant deviation of the probability curves, i.e. patients with lower CD4 + T cells and natural killer cells one month and B cells after 3 months after stem cell transplantation had a higher probability to belong to the responder group. Taken together, this study supports the theory that a profound CD4 + T cell and B cell lymphopenia is important for patients with SSc to achieve a sustained response after aHSCT.

中文翻译：

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患有系统性硬化症且 CD4 数量较低的患者在自体干细胞移植后获得了良好的结果

高剂量化疗后进行自体造血干细胞移植 (aHSCT) 是严重系统性硬化症 (SSc) 患者的强化治疗选择。尽管与治疗相关的高死亡率相关，但在这一高危人群中的结果通常是有利的。了解这种疗法的潜在作用机制以及它如何改善 SSc 患者对于更好地选择合适的 aHSCT 患者至关重要。这是一项来自德国蒂宾根的单中心回顾性研究，包括 32 名接受 aHSCT 的患者。在 aHSCT 之前和之后的不同时间点分析外周血样本的不同淋巴细胞亚群。根据aHSCT后三年内改良Rodnan皮肤评分和肺功能测试将患者分为有反应者和无反应者。应答者在 aHSCT 后的最初几个月（第 1 个月和第 3 个月）、B 细胞（aHSCT 后第 3 个月和第 6 个月）和自然杀伤细胞（第 1 个月）显示出分化簇 (CD)4 阳性 T 细胞水平显着降低。Mantel-cox检验显示概率曲线存在显着偏差，即干细胞移植后1个月CD4+T细胞和自然杀伤细胞较低、3个月后B细胞较低的患者属于应答组的概率较高。总而言之，这项研究支持这样的理论：CD4 + T 细胞和 B 细胞淋巴细胞严重减少对于 SSc 患者在 aHSCT 后实现持续缓解非常重要。