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管理和预防儿童及青少年和青年白血病患者的低纤维蛋白原血症:来自MD安德森癌症中心的见解

Managing and Preventing Hypofibrinogenemia in Pediatric and AYA Leukemia Patients: Insights From MD Anderson Cancer Center.

作者信息

He Jiasen, Munir Faryal, Ayerbe Camila, Dickson Samantha, Catueno Samanta, Cuglievan Branko, Gibson Amber, McCall David, Nunez Cesar, Roth Michael, Tewari Priti, Garcia Miriam B, Rodriguez Nidra, Cortes Jose

机构信息

Department of Pediatrics, Pediatric Hematology-Oncology, The University of Texas MD Anderson Cancer Center.

McGovern Medical School at UT Health Science Center.

出版信息

J Pediatr Hematol Oncol. 2025 Jul 1;47(5):235-241. doi: 10.1097/MPH.0000000000003042. Epub 2025 Apr 24.

DOI:10.1097/MPH.0000000000003042
PMID:40306291
Abstract

BACKGROUND

Cryoprecipitate is often used to prevent and treat complications associated with low fibrinogen levels in pediatric leukemia patients. Cryoprecipitate, rich in fibrinogen, is administered to augment fibrinogen levels and mitigate the risk of bleeding in these patients. The use of cryoprecipitate is often strategic, involving both prophylactic measures and interventions in response to bleeding events. This approach plays a crucial role in the comprehensive care of pediatric leukemia patients, particularly during periods of heightened vulnerability to bleeding complications. However, data regarding the use of cryoprecipitate in children with leukemia are lacking.

METHODS

We conducted a retrospective chart review of children, adolescents, and young adults with leukemia who received cryoprecipitate at the University of Texas MD Anderson Cancer Center from 2020 to 2022. We gathered baseline clinical and demographic data, cryoprecipitate usage details, and fibrinogen levels. Paired-samples t tests were used to compare fibrinogen levels before and after cryoprecipitate infusion.

RESULTS

We identified 36 patients who received cryoprecipitate, 1 to 25 years of age, 67% of whom (24/36) were male. In this cohort, 27/36 (75%) were recently exposed to asparaginase, 2/36 (6%) had a history of venous thromboembolism, and 6/36 (17%) had a history of major bleeding. Cryoprecipitate was used to treat active bleeding (11/36, 31%), bleeding prevention (22/36, 61%), and preoperatively (3/36, 8%). Patients frequently required transfusions of other blood products. Common comorbidities in patients receiving cryoprecipitate included disseminated intravascular coagulation (10/36, 28%) and sepsis (10/36, 28%). The median baseline fibrinogen level across the entire study population was 85.5 mg/dL (IQR: 59.8 to 113). The median dose of cryoprecipitate infused was 6.6 mL/kg (IQR: 3.09 to 14.6), and the median postinfusion peak fibrinogen level was 185 mg/dL (IQR: 155 to 292) ( P <0.001). No major direct treatment-related adverse events were reported.

CONCLUSION

Cryoprecipitate is commonly administered to children with leukemia, effectively raising fibrinogen levels with minimal associated side effects. However, further research, ideally through randomized trials, is needed to assess its true clinical benefits in this population. Currently, there is a notable lack of pediatric-focused randomized transfusion medicine trials. Integrating these studies into ongoing oncologic trials could be a practical and valuable approach that warrants careful consideration and planning.

摘要

背景

冷沉淀常用于预防和治疗小儿白血病患者因纤维蛋白原水平低而引发的并发症。富含纤维蛋白原的冷沉淀被用于提高这些患者的纤维蛋白原水平,并降低出血风险。冷沉淀的使用通常具有策略性,包括预防措施以及针对出血事件的干预措施。这种方法在小儿白血病患者的综合护理中起着关键作用,尤其是在出血并发症易感性增加的时期。然而,关于白血病患儿使用冷沉淀的数据尚缺乏。

方法

我们对2020年至2022年在德克萨斯大学MD安德森癌症中心接受冷沉淀治疗的儿童、青少年和青年白血病患者进行了回顾性病历审查。我们收集了基线临床和人口统计学数据、冷沉淀使用细节以及纤维蛋白原水平。采用配对样本t检验比较冷沉淀输注前后的纤维蛋白原水平。

结果

我们确定了36例接受冷沉淀治疗的患者,年龄在1至25岁之间,其中67%(24/36)为男性。在这个队列中,27/36(75%)最近接触过天冬酰胺酶,2/36(6%)有静脉血栓栓塞病史,6/36(17%)有大出血病史。冷沉淀用于治疗活动性出血(11/36,31%)、预防出血(22/36,61%)和术前使用(3/36,8%)。患者经常需要输注其他血液制品。接受冷沉淀治疗的患者常见的合并症包括弥散性血管内凝血(10/36,28%)和脓毒症(10/36,28%)。整个研究人群的基线纤维蛋白原水平中位数为85.5mg/dL(四分位间距:59.8至113)。冷沉淀的输注中位剂量为6.6mL/kg(四分位间距:3.09至14.6),输注后纤维蛋白原水平的峰值中位数为185mg/dL(四分位间距:155至292)(P<0.001)。未报告重大的直接治疗相关不良事件。

结论

冷沉淀常用于白血病患儿,能有效提高纤维蛋白原水平,且副作用最小。然而,需要进一步研究,理想情况下通过随机试验,来评估其在该人群中的真正临床益处。目前,明显缺乏以儿童为重点的随机输血医学试验。将这些研究纳入正在进行的肿瘤学试验可能是一种切实可行且有价值的方法,值得仔细考虑和规划。

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