Department of Hematology and BMT, Fortis Memorial Research Institute, Gurgaon, Haryana, India.
Neurol India. 2020 Mar-Apr;68(2):454-457. doi: 10.4103/0028-3886.284385.
Autologous Stem Cell Transplant (ASCT) provides long periods of progression-free-survival in multiple sclerosis (MS). This is an observational study to demonstrate the safety of ASCT in MS patients at a transplant center in North India using a lymphoablative regimen.
MS patients > 18 years referred by a neurologist or who came of their own volition were evaluated. Kurtzke Expanded Disability Status Scale (EDSS) score was calculated and those with a score of >7 were excluded. Informed written consent was taken. Mobilization was done with G-CSF with prednisolone to prevent disease flare-up. A minimum of 2 × 10 CD34 cells/kg was collected. Conditioning regimen consisted of rabbit ATG and cyclophosphamide. Rituximab 375 mg/m was given to prevent EBV reactivation and disease relapse. Antibiotic prophylaxis was given with levofloxacin, fluconazole, and valacyclovir. Any persistent change in EDSS scores ≥0.5 was considered significant.
Twenty patients were included. Seven patients had positive urine cultures prior to transplant and were treated before starting any chemotherapy. Majority patients were women (13/20). All patients developed febrile neutropenia, which was managed as per department policy. There was no mortality. Subjective symptoms improved in all patients. EDSS score improved in 6/19 patients (5/6 with RRMS) with no disease progression in any patient at a median follow-up duration of 242 days.
ASCT can be done safely for patients with relatively high EDSS scores with additional precautions for screening for infections. RRMS patients with the active disease show most improvement. SPMS patients may not show significant improvement in the short term.
自体干细胞移植（ASCT）可使多发性硬化症（MS）患者获得较长时间的无进展生存期。本研究为观察性研究，旨在展示在印度北部的一家移植中心，使用淋巴清除性方案对多发性硬化症患者进行 ASCT 的安全性。
我们对由神经科医生转诊或自愿就诊的年龄＞18 岁的 MS 患者进行评估。计算 Kurtzke 扩展残疾状况量表（EDSS）评分，评分＞7 分的患者被排除。所有患者均签署了知情同意书。使用 G-CSF 和泼尼松龙进行动员，以防止疾病复发。采集至少 2×10 CD34 细胞/kg。预处理方案包括兔抗胸腺细胞球蛋白和环磷酰胺。为了预防 EBV 再激活和疾病复发，给予利妥昔单抗 375mg/m。使用左氧氟沙星、氟康唑和伐昔洛韦进行抗生素预防。EDSS 评分持续升高≥0.5 被认为有意义。
共纳入 20 例患者。移植前 7 例患者的尿液培养阳性，在开始任何化疗前进行了治疗。大多数患者为女性（13/20）。所有患者均出现发热性中性粒细胞减少症，根据科室政策进行了管理。无死亡病例。所有患者的主观症状均改善。19 例患者中有 6 例（RRMS 患者 5 例）的 EDSS 评分改善，在中位随访 242 天内，没有患者疾病进展。
对于 EDSS 评分相对较高的患者，在进行 ASCT 时可以安全进行，并且需要额外注意筛查感染。有活动性疾病的 RRMS 患者改善最明显。SPMS 患者在短期内可能不会有明显改善。
