Shah Ankoor, Spierings J, van Laar J M, Sullivan Keith M
Division of Rheumatology, Duke University, Durham, NC, USA.
Department of Rheumatology and Clinical Immunology, University Medical Centre Utrecht, Utrecht, The Netherlands.
J Scleroderma Relat Disord. 2021 Jun;6(2):199-205. doi: 10.1177/2397198320985766. Epub 2021 Jan 14.
Systemic sclerosis is a chronic autoimmune disease with a poor prognosis, particularly when a patient has rapidly progressive skin or pulmonary involvement. Autologous hematopoietic stem cell transplant is an emerging treatment for this condition, that has been demonstrated to be more effective than immunosuppressants. Careful selection of patients has reduced the transplant-related mortality and maximized the likelihood of benefit. In this report, we present three cases of successful autologous hematopoietic stem cell transplant in patients who would not have met inclusion criteria for entrance into the completed hematopoietic stem cell transplant. After >18 months of follow-up, three patients had clinically significant benefit in terms of skin tightening and pulmonary function tests. Future studies of hematopoietic stem cell transplant in systemic sclerosis may aim to carefully liberalize inclusion criteria to include patients who may not have otherwise been treated while still maintaining an acceptable safety profile.
系统性硬化症是一种预后较差的慢性自身免疫性疾病,尤其是当患者出现快速进展的皮肤或肺部受累时。自体造血干细胞移植是针对这种疾病的一种新兴治疗方法,已被证明比免疫抑制剂更有效。对患者进行仔细筛选降低了移植相关死亡率,并使获益可能性最大化。在本报告中,我们介绍了3例成功接受自体造血干细胞移植的患者,这些患者原本不符合已完成的造血干细胞移植的纳入标准。经过超过18个月的随访,3例患者在皮肤紧致和肺功能测试方面均有显著临床获益。未来关于系统性硬化症造血干细胞移植的研究可能旨在谨慎放宽纳入标准,以纳入那些可能未接受其他治疗但仍保持可接受安全性的患者。