Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia
Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia
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DOI：
10.3324/haematol.2014.109892
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发表时间：
2015-01-01
期刊：
影响因子：
10.1
通讯作者：
Yoshimi, Ayami
中科院分区：
文献类型：
--
作者：
Niemeyer, Charlotte M.;Loh, Mignon L.;Yoshimi, Ayami
青少年粒单核细胞白血病是一种罕见的小儿骨髓增生性疾病。虽然造血干细胞移植仍然是大多数患者的唯一治愈性治疗选择，但患有青少年粒单核细胞白血病的儿童越来越多地在I-II期临床试验中接受新药物作为移植前治疗或移植后复发的治疗。然而，目前缺乏对青少年粒单核细胞白血病患者治疗效果进行标准化评价的反应标准或结局定义。在此，我们提出了评价非移植治疗反应的标准和造血干细胞移植后缓解状态的定义。对于非移植治疗的评价，我们定义了6个临床变量（白色血细胞计数、血小板计数、外周血中的造血前体细胞和原始细胞、骨髓原始细胞百分比、脾脏大小和髓外疾病）和3个遗传变量（细胞遗传学、分子和嵌合体反应），用于描述每个个体病例中对治疗反应的异质性。我们希望这些标准将有助于青少年粒单核细胞白血病临床试验结果的比较。
Juvenile myelomonocytic leukemia is a rare myeloproliferative disease in young children. While hematopoietic stem cell transplantation remains the only curative therapeutic option for most patients, children with juvenile myelomonocytic leukemia increasingly receive novel agents in phase I-II clinical trials as pre-transplant therapy or therapy for relapse after transplantation. However, response criteria or definitions of outcome for standardized evaluation of treatment effect in patients with juvenile myelomonocytic leukemia are currently lacking. Here we propose criteria to evaluate the response to the non-transplant therapy and definitions of remission status after hematopoietic stem cell transplantation. For the evaluation of non-transplant therapy, we defined 6 clinical variables (white blood cell count, platelet count, hematopoietic precursors and blasts in peripheral blood, bone marrow blast percentage, spleen size and extramedullary disease) and 3 genetic variables (cytogenetic, molecular and chimerism response) which serve to describe the heterogeneous picture of response to therapy in each individual case. It is hoped that these criteria will facilitate the comparison of results between clinical trials in juvenile myelomonocytic leukemia.