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自体间充质基质细胞骨内应用治疗儿童和青少年类固醇或化疗后缺血性坏死的长期疗效。

Longtime Outcome After Intraosseous Application of Autologous Mesenchymal Stromal Cells in Pediatric Patients and Young Adults with Avascular Necrosis After Steroid or Chemotherapy.

机构信息

Department I-General Pediatrics, Hematology and Oncology, University Hospital Tuebingen-Children's Hospital Tuebingen, Tuebingen, Germany.

Department of Orthopedic Surgery, Städtisches Klinikum Dresden, Dresden, Germany.

出版信息

Stem Cells Dev. 2020 Jul 1;29(13):811-822. doi: 10.1089/scd.2020.0019. Epub 2020 May 12.

Abstract

Avascular necrosis (AVN) is a severe complication of immunosuppressant therapy or chemotherapy. A beneficial AVN therapy with core decompression (CD) and intraosseous infusion of mesenchymal stromal cells (MSCs) has been described in adult patients, but there are only few data on MSC applications in pediatric and young adult patients (PYAP). Between 2006 and 2015, 14 AVN lesions of 10 PYAP (6 females) with a median age of 16.9 years (range 8.5-25.8 years) received CD and intraosseous application of autologous MSCs. Data of these patients were analyzed regarding efficacy, safety, and feasibility of this procedure as AVN therapy and compared with a control group of 13 AVN lesions of 11 PYAP (5 females) with a median age of 17.9 years (range 13.5-27.5 years) who received CD only. During the follow-up analysis [MSC group: median 3.1 (1.6-5.8) years after CD; CD group: median 2.0 (1.5-8.5) years after CD], relative lesion sizes (as assessed by magnetic resonance imaging) compared with the initial lesion volume, were significantly lower ( < 0.05) in the MSC group (volume reduction to a median of 18.5%) when compared with the CD group (58.0%). One lesion in the MSC group comprised a complete remission. Size progression was not observed in either group. Clinical improvement (pain, mobility) was not significantly different between the two groups. None of the patients experienced treatment-related adverse effects. CD and additional MSC application was regarded safe, effective, feasible, and superior in reducing the lesion size when compared with CD only. Prospective, randomized clinical trials are needed to further evaluate these findings.

摘要

骨坏死(AVN)是免疫抑制剂治疗或化疗的严重并发症。已在成年患者中描述了经皮髓芯减压(CD)和骨髓间充质干细胞(MSCs)腔内输注的有益 AVN 治疗方法,但在儿科和年轻成年患者(PYAP)中应用 MSC 的数据很少。2006 年至 2015 年,10 名 PYAP(6 名女性)的 14 个 AVN 病变,中位年龄为 16.9 岁(范围 8.5-25.8 岁),接受了 CD 和自体 MSC 腔内应用。分析了这些患者的数据,以评估该方法作为 AVN 治疗的疗效、安全性和可行性,并与仅接受 CD 的 11 名 PYAP(5 名女性)的 13 个 AVN 病变的对照组(中位年龄 17.9 岁,范围 13.5-27.5 岁)进行比较。在随访分析中(MSC 组:CD 后中位 3.1 年[1.6-5.8 年];CD 组:CD 后中位 2.0 年[1.5-8.5 年]),与初始病变体积相比,相对病变大小(通过磁共振成像评估)在 MSC 组中明显较低( < 0.05)(体积减少至中位数 18.5%)与 CD 组(58.0%)相比。MSC 组中有 1 个病变完全缓解。两组均未观察到病变进展。两组之间的临床改善(疼痛、活动度)无明显差异。没有患者出现与治疗相关的不良事件。与仅 CD 相比,CD 加额外 MSC 应用被认为更安全、有效、可行,并能降低病变大小。需要前瞻性、随机临床试验来进一步评估这些发现。

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