Primary Children's Medical Center, University of Utah School of Medicine, Salt Lake City, UT 84132, USA.
Biol Blood Marrow Transplant. 2010 Sep;16(9):1212-21. doi: 10.1016/j.bbmt.2009.12.536. Epub 2010 Jan 14.
Advances in pediatric bone marrow transplantation (BMT) are slowed by the small number of patients with a given disease who undergo transplantation, a lack of sufficient infrastructure to run early-phase oncology protocols and studies of rare nonmalignant disorders, and challenges associated with funding multi-institutional trials. Leadership of the Pediatric Blood and Marrow Transplant Consortium (PBMTC), a large pediatric BMT clinical trials network representing 77 active and 45 affiliated centers worldwide, met in April 2009 to develop strategic plans to address these issues. Key barriers, including infrastructure development and funding, along with scientific initiatives in malignant and nonmalignant disorders, cellular therapeutics, graft-versus-host disease, and supportive care were discussed. The PBMTC's agenda for approaching these issues will result in infrastructure and trials specific to pediatrics that will run through the PBMTC or its partners, the Blood and Marrow Transplant Clinical Trials Network and the Children's Oncology Group.
儿科骨髓移植（BMT）的进展受到限制，因为进行移植的特定疾病患者数量较少，缺乏足够的基础设施来运行早期肿瘤学方案和研究罕见的非恶性疾病，以及与资助多机构试验相关的挑战。代表全球 77 个活跃中心和 45 个附属中心的儿科血液和骨髓移植联盟（PBMTC）的领导层于 2009 年 4 月开会制定了战略计划，以解决这些问题。讨论了关键障碍，包括基础设施发展和资金，以及恶性和非恶性疾病、细胞治疗、移植物抗宿主病和支持性护理方面的科学计划。PBMTC 解决这些问题的议程将导致针对儿科的特定基础设施和试验，这些试验将通过 PBMTC 或其合作伙伴——血液和骨髓移植临床试验网络和儿童肿瘤学组来运行。
