Rousso Sharon Z, Shamriz Oded, Zilkha Amir, Braun Jacques, Averbuch Diana, Or Reuven, Weintraub Michael, Revel-Vilk Shoshana, Stepensky Polina
*Department of Pediatrics, Hadassah-Hebrew University Medical Center, Mount Scopus Departments of ‡Pediatrics ¶Bone Marrow Transplantation and Cancer Immunotherapy #Pediatric Hematology-Oncology and Bone Marrow Transplantation §Pediatric Intensive Care Unit ∥Pediatric Infectious Diseases Unit, Hadassah-Hebrew University Medical Center, Ein Kerem, Jerusalem, Israel †Division of Nephrology, the Hospital for Sick Children, University of Toronto, Ontario, Canada.
J Pediatr Hematol Oncol. 2015 Jul;37(5):e295-300. doi: 10.1097/MPH.0000000000000352.
Hematopoietic stem cell transplantation (HSCT) remains the leading treatment for the majority of severe primary immune deficiency (PID). This study aims to analyze changes in outcome over time. We conducted a retrospective analysis of HSCT in children with PID in a tertiary medical center over the period of 1983 to 2012. We identified 93 children with PID with a median follow-up of 3.6 years (range, 29 d to 21.2 y) after HSCT. The 2-year survival rates after HSCT for children with severe combined immune deficiency, hemophagocytic lymphohistiocytosis/lymphoproliferative disease, Wiskott-Aldrich syndrome, granulocyte defect, and undefined PID were 65.7%±6.8%, 80%±10.3%, 83.3%±15.2%, 75%±12.5%, and 25%±21.7%, respectively. Survival was associated with year of HSCT and matching. The hazard ratio (HR) (95% CI) for HSCT done in 1983 to 1999 compared with 2000 to 2012 and for matched (related and unrelated) compared with mismatched donor were 2.14 (0.99 to 4.653) and 3.07 (1.46 to 6.4), respectively. Survival was not associated with age, sex of the recipient, underlying PID, conditioning regimen, and presence of acute graft-versus-host disease. After adjustment to the underlying PID, donor and use of fludarabine-based conditioning, the HR (95% CI) for HSCT from the year 2000 was 4.69 (range, 1.4 to 15.45). Advances in HSCT over time have improved the survival of children with PID.
造血干细胞移植（HSCT）仍然是大多数严重原发性免疫缺陷病（PID）的主要治疗方法。本研究旨在分析随着时间推移治疗结果的变化。我们对1983年至2012年期间一家三级医疗中心的PID患儿进行了HSCT回顾性分析。我们确定了93例PID患儿，HSCT后中位随访时间为3.6年（范围：29天至21.2年）。严重联合免疫缺陷、噬血细胞性淋巴组织细胞增生症/淋巴细胞增殖性疾病、威斯科特-奥尔德里奇综合征、粒细胞缺陷和未明确的PID患儿HSCT后的2年生存率分别为65.7%±6.8%、80%±10.3%、83.3%±15.2%、75%±12.5%和25%±21.7%。生存率与HSCT年份和配型有关。1983年至1999年进行的HSCT与2000年至2012年进行的HSCT相比，以及匹配（相关和无关）供体与不匹配供体相比的风险比（HR）（95%CI）分别为2.14（0.99至4.653）和3.07（1.46至6.4）。生存率与受者年龄、性别、潜在PID、预处理方案以及急性移植物抗宿主病的存在无关。在对潜在PID、供体和基于氟达拉滨的预处理的使用进行调整后，2000年以来HSCT的HR（95%CI）为4.69（范围：1.4至15.45）。随着时间的推移，HSCT的进展提高了PID患儿的生存率。
