Joint Rheumatology Program, First Department of Propaedeutic and Internal Medicine, Medical School, National and Kapodistrian University of Athens, Greece.
Clin Exp Rheumatol. 2021 Jul-Aug;39 Suppl 131(4):29-33. doi: 10.55563/clinexprheumatol/dhn3mb. Epub 2020 Nov 10.
Autologous haematopoietic stem cell transplantation (HSCT) has exhibited superior efficacy compared to conventional immunosuppressives in rapidly progressive diffuse systemic sclerosis (SSc) patients, albeit still of limited availability. We examined disease outcomes of conventionally-treated real-world inception patients eligible for HSCT, according to HSCT criteria used in the ASTIS and SCOT randomised trials, and compared them to the outcomes of participants in these trials.
Overall and event-free survival rates in our inception cohort were analysed at 4.5 and 7 years after HSCT criteria fulfilment and compared to those reported in HSCT and control arms of ASTIS and SCOT.
Forty-five of our 142 inception cohort patients fulfilled HSCT criteria within 4 years from disease onset and had comparable baseline characteristics to SCOT/ASTIS patients. Four patients underwent HSCT. The remaining 41 were treated with conventional DMARDs: cyclophosphamide (n=24), mycophenolate mofetil (n=17), rituximab (n=2), tocilizumab (n=3), methotrexate (n=6) or combinations and their 10-year survival was 56% vs. 76% in those with diffuse SSc not fulfilling HSCT criteria. Their survival rates at the time endpoints of SCOT and ASTIS (4.5 and 7 years, respectively) were comparable to the conventionally-treated SCOT/ASTIS control groups. Extrapolating from SCOT/ASTIS results, if all our patients had undergone HSCT promptly, their overall and event-free survival rates could have increased from 73/51% to 83/72% at 4.5 years, and from 63/39% to 76/72% at 7 years, respectively.
Wider availability and physician's early acknowledgement and referral of eligible patients for HSCT could significantly improve disease outcomes of rapidly progressive diffuse SSc patients.
与传统免疫抑制剂相比，自体造血干细胞移植（HSCT）在快速进展性弥漫性系统性硬化症（SSc）患者中显示出更好的疗效，但可用性仍然有限。根据 ASTIS 和 SCOT 随机试验中使用的 HSCT 标准，我们检查了符合 HSCT 条件的常规治疗真实世界起始患者的疾病结局，并将其与这些试验中的参与者的结局进行了比较。
在符合 HSCT 标准后 4.5 年和 7 年，分析我们的起始队列中患者的总生存率和无事件生存率，并与 ASTIS 和 SCOT 的 HSCT 和对照组报告的结果进行比较。
在疾病发病后 4 年内，我们的 142 名起始队列患者中有 45 名符合 HSCT 标准，与 SCOT/ASTIS 患者具有可比的基线特征。4 名患者接受了 HSCT。其余 41 名患者接受了常规 DMARD 治疗：环磷酰胺（n=24）、霉酚酸酯（n=17）、利妥昔单抗（n=2）、托珠单抗（n=3）、甲氨蝶呤（n=6）或联合治疗，他们的 10 年生存率为 56%，而不符合 HSCT 标准的弥漫性 SSc 患者为 76%。他们在 SCOT 和 ASTIS 的时间终点（分别为 4.5 年和 7 年）的生存率与常规治疗的 SCOT/ASTIS 对照组相当。根据 SCOT/ASTIS 的结果推断，如果我们所有的患者都能及时接受 HSCT，他们的总生存率和无事件生存率可能会从 4.5 年时的 73/51%增加到 83/72%，从 63/39%增加到 76/72%。
更广泛地提供和医生早期认识和转介符合条件的患者进行 HSCT，可以显著改善快速进展性弥漫性 SSc 患者的疾病结局。
