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首页> 外文期刊>Pediatric blood & cancer >Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia
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Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia

机译：预防米洛霉菌白血病治疗预防素化疗的分子评估

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Abstract Background Despite the intensity of hematopoietic stem cell transplantation (HCT), relapse remains the most common cause of death in juvenile myelomonocytic leukemia (JMML). In contrast to other leukemias where therapy is used to reduce leukemic burden prior to transplant, many patients with JMML proceed directly to HCT with active disease. The objective of this study was to elucidate whether pre‐HCT therapy has an effect on the molecular burden of disease and how this affects outcome post‐HCT. Procedure Twenty‐one patients with JMML who received pre‐HCT therapy and were transplanted at UCSF were analyzed in this study. The mutant allele frequency of the driver mutation was assessed before and after pre‐HCT therapy, using custom amplicon next‐generation sequencing. Results Of the 21 patients, seven patients (33%) responded to therapy with a significant reduction in their mutant allele frequency and were classified as molecular responders. Six of these patients received moderate‐intensity chemotherapy, one patient received only azacitidine. The 5‐year progression‐free survival after HCT of molecular responders was 100%?versus 61% for nonresponders ( P ?=?.12). Survival of molecular nonresponders was not improved by use of high‐intensity conditioning, but patients were salvaged if they experienced severe graft versus host disease. There were no baseline clinical characteristics that were associated with response to pre‐HCT therapy. Conclusions Despite the myelodysplastic nature of JMML, patients treated with pre‐HCT therapy can achieve molecular remissions. These patients experienced a trend toward improved outcomes post‐HCT. Importantly, molecular testing can be helpful to distinguish between responders and nonresponders and should become an integral part of clinical care.
机译：摘要背景尽管造血干细胞移植强度（HCT），复发仍然是青少年骨髓细胞白血病（JMML）中最常见的死亡原因。与其他白血病相比，疗法用于降低移植前的白血病负担，许多患有JMML的患者直接与活跃疾病直接达到HCT。本研究的目的是阐明HCT治疗前是否对疾病的分子负担以及这种影响后的结果产生影响。程序在本研究中分析了在本研究中分析了接受HCT治疗前疗法的JMML患者，并在本研究中分析了UCSF。在HCT治疗前后评估驾驶员突变的突变等位基因频率，使用定制扩增子下一代测序进行评估。结果21例患者，7名患者（33％）反应治疗，其突变等位基因频率显着降低，并被归类为分子响应者。六个患者接受了中度强度化疗，一名患者只接受氮杂氨酸。分子响应者HCT后的5年的无进展生存率为100％？无应答者的61％（p？=Δ.12）。通过使用高强度调节没有改善分子非反应者的存活，但如果患者经历严重的移植物与宿主疾病，患者被销售。没有基线临床特征与对HCT治疗前的反应相关。结论尽管JMML的骨髓增强性质，但患有预处理疗法治疗的患者可以实现分子剩余。这些患者经历了后宫后改善结果的趋势。重要的是，分子测试可以有助于区分响应者和无反应者，并且应该成为临床护理的一个组成部分。

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