PDF(Pubmed)
Abstract：
An international consensus conference sponsored by the Pediatric Blood and Marrow Transplant consortium entitled \"Late Effects Screening and Recommendations Following Allogeneic Hematopoietic Cell Transplant for Immune Deficiency and Nonmalignant Hematologic Disease\" was held in Minneapolis, Minnesota on May 10, 2016 and May 11, 2016. The purpose of the conference was to address the unmet need for greater understanding of and the screening for long-term complications in the growing population of survivors of transplantation for nonmalignant disorders. The conference focused on transplantation for hemoglobinopathy, immune deficiency, and inherited bone marrow syndromes. A multidisciplinary group of experts in the disease areas and transplantation late effects presented the current state of understanding of how the underlying disease, pretransplantation therapies, and transplantation-related factors uniquely interact to influence the development of late toxicities. Recommendations were put forth by the group for the late effects screening of survivors of transplantation for these nonmalignant disorders. The findings and recommendations that came from this conference will be presented in a series of 6 additional manuscripts in the upcoming months. In this manuscript, we explore the need for screening practices specific to the survivors of transplantation for nonmalignant diseases and the methodologic challenges associated with the study of these patients.
摘要：
由儿科血液和骨髓移植联盟主办的题为“异基因造血细胞移植后免疫缺陷和非恶性血液病的后期效应筛查和建议”的国际共识会议在明尼阿波利斯举行，明尼苏达州于2016年5月10日和2016年5月11日。会议的目的是解决对越来越多的非恶性疾病移植幸存者中的长期并发症的理解和筛查的未满足需求。会议的重点是血红蛋白病的移植，免疫缺陷，和遗传性骨髓综合征.疾病领域和移植后期影响的多学科专家组介绍了对潜在疾病的了解现状，移植前治疗,和移植相关因素独特地相互作用以影响晚期毒性的发展。该小组提出了建议，以筛查这些非恶性疾病的移植幸存者的后期效果。这次会议的结果和建议将在接下来的几个月中发表在一系列6份额外的手稿中。在这份手稿中,我们探讨了对非恶性疾病移植幸存者进行特定筛查的必要性,以及与这些患者研究相关的方法学挑战.
