Snowden John A, Badoglio Manuela, Labopin Myriam, Giebel Sebastian, McGrath Eoin, Marjanovic Zora, Burman Joachim, Moore John, Rovira Montserrat, Wulffraat Nico M, Kazmi Majid, Greco Raffaella, Snarski Emilian, Kozak Tomas, Kirgizov Kirill, Alexander Tobias, Bader Peter, Saccardi Riccardo, Farge Dominique
Department of Haematology, Sheffield Teaching Hospitals National Health Service Foundation Trust, Sheffield, United Kingdom.
European Society for Blood and Marrow Transplantation (EBMT) Paris Study Office and.
Blood Adv. 2017 Dec 20;1(27):2742-2755. doi: 10.1182/bloodadvances.2017010041. eCollection 2017 Dec 26.
Hematopoietic stem cell transplantation (HSCT) has evolved for >20 years as a specific treatment of patients with autoimmune disease (AD). Using European Society for Blood and Marrow Transplantation registry data, we summarized trends and identified factors influencing activity and outcomes in patients with AD undergoing first autologous HSCT (n = 1951; median age, 37 years [3-76]) and allogeneic HSCT (n = 105; median age, 12 years [<1-62]) in 247 centers in 40 countries from 1994 to 2015. Predominant countries of activity were Italy, Germany, Sweden, the United Kingdom, The Netherlands, Spain, France, and Australia. National activity correlated with the Human Development Index ( = .006). For autologous HSCT, outcomes varied significantly between diseases. There was chronological improvement in progression-free survival (PFS, < 10), relapse/progression ( < 10), and nonrelapse mortality ( = .01). Health care expenditure was associated with improved outcomes in systemic sclerosis and multiple sclerosis (MS). On multivariate analysis selecting adults for MS, systemic sclerosis, and Crohn disease, better PFS was associated with experience (≥23 transplants for AD, = .001), learning (time from first HSCT for AD ≥6 years, = .01), and Joint Accreditation Committee of the International Society for Cellular Therapy and European Society for Blood and Marrow Transplantation accreditation status ( = .02). Despite improved survival over time ( = .02), allogeneic HSCT use remained low and largely restricted to pediatric practice. Autologous HSCT has evolved into a treatment modality to be considered alongside other modern therapies in severe AD. Center experience, accreditation, interspecialty networking, and national socioeconomic factors are relevant for health service delivery of HSCT in AD.
造血干细胞移植（HSCT）作为自身免疫性疾病（AD）患者的一种特殊治疗方法已经发展了20多年。利用欧洲血液和骨髓移植学会登记数据，我们总结了1994年至2015年期间在40个国家的247个中心接受首次自体HSCT（n = 1951；中位年龄37岁[3 - 76岁]）和异基因HSCT（n = 105；中位年龄12岁[<1 - 62岁]）的AD患者的趋势，并确定了影响其治疗活性和结局的因素。主要开展该治疗的国家有意大利、德国、瑞典、英国、荷兰、西班牙、法国和澳大利亚。国家治疗活性与人类发展指数相关（r = 0.006）。对于自体HSCT，不同疾病的结局差异显著。无进展生存期（PFS，P < 0.01）、复发/进展（P < 0.01）和非复发死亡率（P = 0.01）随时间有改善。医疗保健支出与系统性硬化症和多发性硬化症（MS）的结局改善相关。在对MS、系统性硬化症和克罗恩病的成年患者进行多变量分析时，更好的PFS与经验（AD移植≥23次，P = 0.001）、学习（首次AD HSCT后时间≥6年，P = 0.01）以及国际细胞治疗学会和欧洲血液和骨髓移植学会联合认证委员会的认证状态（P = 0.02）相关。尽管随着时间推移生存率有所提高（P = 0.02），但异基因HSCT的使用仍然较少，且主要限于儿科实践。自体HSCT已发展成为一种在重度AD中可与其他现代疗法一并考虑的治疗方式。中心经验、认证、跨专业网络以及国家社会经济因素与AD中HSCT的医疗服务提供相关。
