Eissa Hesham M, Lu Lu, Baassiri Malek, Bhakta Nickhill, Ehrhardt Matthew J, Triplett Brandon M, Green Daniel M, Mulrooney Daniel A, Robison Leslie L, Hudson Melissa M, Ness Kirsten K
Department of Oncology, St. Jude Children's Research Hospital, Memphis, TN.
Department of Pediatrics, University of Minnesota, Minneapolis, MN; and.
Blood Adv. 2017 Nov 7;1(24):2243-2246. doi: 10.1182/bloodadvances.2017010280. eCollection 2017 Nov 14.
Outcomes of hematopoietic stem cell transplantation (HSCT) have markedly improved over the past 2 decades, underscoring a need to better understand the long-term health effects of this intensive treatment modality. We describe the burden of chronic medical conditions and frail health among St. Jude Lifetime Cohort Study participants treated for childhood hematologic malignancies with HSCT (n = 112) or with conventional therapy (n = 1106). Chronic conditions and frail health were ascertained clinically and classified according to a modified version of the Common Terminology Criteria for Adverse Events (version 4.03) and the Fried Frailty Criteria. Seventy-nine transplants were allogeneic (41 from a sibling donor, 34 unrelated, and 4 others from related donor). Twenty-five allogeneic donor recipients had a history of chronic graft-versus-host disease. Compared to those treated with conventional therapy, a higher percentage of HSCT survivors had severe, disabling, or life threatening (grade 3-4) chronic conditions (81.3% vs 69.2%, = .007). By age 25 years, HSCT survivors experienced 148 grade 3-4 events/100 compared to 60 among conventional therapy survivors ( < .001). Percentages of survivors with second neoplasms (17.0% vs 7.9%, = .003), grade 3-4 cardiovascular (19.6% vs 10.2%, = .004) and pulmonary (16.1% vs 4.6%, < .001) conditions, and frail health (7.1% vs 1.6%, < .001) were higher after HSCT than conventional therapy. These results underscore the need for clinical follow-up and provide data to guide the development of prevention and/or intervention strategies for this vulnerable population.
在过去20年中，造血干细胞移植（HSCT）的疗效有了显著改善，这突出表明需要更好地了解这种强化治疗方式对长期健康的影响。我们描述了圣犹大终身队列研究中接受HSCT（n = 112）或传统疗法（n = 1106）治疗儿童血液系统恶性肿瘤的参与者的慢性疾病负担和健康脆弱状况。慢性疾病和健康脆弱状况通过临床确定，并根据不良事件通用术语标准（第4.03版）的修订版和弗里德虚弱标准进行分类。79例移植为同种异体移植（41例来自同胞供体，34例无关供体，4例来自相关供体）。25例同种异体供体接受者有慢性移植物抗宿主病病史。与接受传统疗法的患者相比，HSCT幸存者中患有严重、致残或危及生命（3 - 4级）慢性疾病的比例更高（81.3%对69.2%，P = 0.007）。到25岁时，HSCT幸存者每100人经历148次3 - 4级事件，而传统疗法幸存者为60次（P < 0.001）。HSCT后，二次肿瘤幸存者的比例（17.0%对7.9%，P = 0.003）、3 - 4级心血管疾病（19.6%对10.2%，P = 0.004）和肺部疾病（16.1%对4.6%，P < 0.001）以及健康脆弱状况（7.1%对1.6%，P < 0.001）均高于传统疗法。这些结果强调了临床随访的必要性，并为指导这一脆弱人群的预防和/或干预策略的制定提供了数据。
