Centro de Hematología y Medicina Interna, Clínica Ruiz, Puebla, México; Universidad Popular Autónoma del Estado de Puebla. Puebla, México.
Centro de Hematología y Medicina Interna, Clínica Ruiz, Puebla, México; Universidad Anáhuac Puebla. Puebla, México.
Mult Scler Relat Disord. 2023 Jul;75:104744. doi: 10.1016/j.msard.2023.104744. Epub 2023 May 5.
Multiple sclerosis (MS) is a disabling disease that affects young adults. Treatments for MS have increased exponentially in number, efficacy and risk. Autologous hematopoietic stem cell transplantation (aHSCT) can change the natural history of the disease. To analyze if aHSCT should be done early in the course of the disease or after failing of other therapies, we have studied the long-term results of aHSCT in a cohort of persons with MS who were given, or not, immunosuppressive drugs before the transplant.
Patients with MS referred to our center for aHSCT between June 2015 and January 2023 were prospectively entered in the study. All phenotypes of MS were included (relapsing remitting, primary progressive and secondary progressive). The follow up was assessed with the patient reported EDSS score in an online form; only patients followed by three or more years were included in the analysis. Patients were divided into two groups: Given or not disease modifying treatments (DMT) before the aHSCT.
1132 subjects were prospectively enrolled. 74 patients were followed for more than 36 months, and the subsequent analysis was done in this cohort. The response rate (RR = improvement + stabilization) at 12, 24 and 36 mo was 84%, 84% and 58% respectively for patients not receiving prior DMT and 72%, 90% and 67% for patients receiving DMT. In the whole group, the EDSS score dropped from a mean of 5.5 to 4.5 at 12 mo, to 5.0 at 24 mo and to 5.5 at 36 mo, after the aHSCT. The EDSS score was on average worsening in patients before the aHSCT, but the transplant stabilized the EDSS score at 3 years in patients with prior exposure to DMT, whereas in persons not given DMT, the transplant resulted in a significant decrease (p = .01) of the EDSS score. This indicates a positive response in all patients given aHSCT, but significantly better in those not exposed to DMT before the graft.
The response to aHSCT was better for persons not exposed to immunosuppressive DMT before the transplant, thus suggesting that aHSCT should be done early in the course of the disease and probably before the treatment with DMT. Additional studies are needed to further analyze the impact of the use of DMT therapies before the aHSCT in MS, as well as the timing of the procedure.
多发性硬化症（MS）是一种影响年轻人的致残性疾病。MS 的治疗方法在数量、疗效和风险方面呈指数级增长。自体造血干细胞移植（aHSCT）可以改变疾病的自然病程。为了分析 aHSCT 应该在疾病早期进行，还是在其他治疗方法失败后进行，我们研究了一组在移植前接受或未接受免疫抑制药物治疗的 MS 患者的 aHSCT 长期结果。
2015 年 6 月至 2023 年 1 月期间，我们前瞻性地将 MS 患者纳入本研究。纳入了所有 MS 表型（复发缓解型、原发性进展型和继发性进展型）。通过在线表格评估患者报告的 EDSS 评分进行随访；仅纳入随访时间超过 3 年的患者进行分析。患者分为两组：在 aHSCT 前是否接受疾病修正治疗（DMT）。
前瞻性纳入了 1132 名患者。74 名患者随访时间超过 36 个月，随后对该队列进行了分析。未接受 DMT 的患者在 12、24 和 36 个月时的反应率（RR=改善+稳定）分别为 84%、84%和 58%，而接受 DMT 的患者分别为 72%、90%和 67%。在整个组中，aHSCT 后 12 个月 EDSS 评分从平均 5.5 降至 4.5，24 个月降至 5.0，36 个月降至 5.5。在 aHSCT 前，患者的 EDSS 评分平均恶化，但在有 DMT 暴露史的患者中，移植稳定了 3 年的 EDSS 评分，而在未接受 DMT 的患者中，移植导致 EDSS 评分显著下降（p=0.01）。这表明所有接受 aHSCT 的患者均有反应，但未接受 DMT 治疗的患者反应更好。
未接受移植前免疫抑制 DMT 治疗的患者对 aHSCT 的反应更好，因此提示 aHSCT 应在疾病早期进行，可能在 DMT 治疗之前进行。需要进一步的研究来进一步分析 MS 患者在 aHSCT 前使用 DMT 治疗的影响，以及手术的时机。
