Department of Clinical Oncology, Faculty of Medicine, Beni-Suef University, Beni-Suef, Egypt; Department of Pediatric Oncology, Children's Cancer Hospital Egypt, Cairo, Egypt.
Department of Pediatric Oncology, Children's Cancer Hospital Egypt, Cairo, Egypt; Department of Pediatric Oncology, National Cancer Institute, Cairo University, Cairo, Egypt.
Clin Lymphoma Myeloma Leuk. 2020 Aug;20(8):e529-e541. doi: 10.1016/j.clml.2020.04.011. Epub 2020 Apr 21.
The presence of FMS-like tyrosine kinase 3 (FLT3) internal tandem duplication (ITD) mutation in pediatric acute myeloid leukemia (AML) is associated with high rates of induction failure and worse survival. Its presence places the patient into a high-risk group. We aimed to describe the outcome of pediatric AML with FLT3-ITD mutation.
We performed a retrospective analysis of cases of AML from July 2007 till July 2017 at Children's Cancer Hospital Egypt.
Seventy-one patients had FLT3 gene mutation out of 687 patients with AML. Sixty-five patients had FLT3 gene mutation with allelic ratio > 0.4; 43 (66.1%) of 65 patients experienced complete remission (CR). Of the 43 patients, 16 patients maintained CR, 18 patients relapsed after first CR, 8 patients died, and 1 patient was lost to follow-up. Patients with relapsing disease died after salvage chemotherapy, except for one patient, who was alive after second CR. Allogeneic bone marrow transplantation (allo-BMT) was performed for 9 (13.8%) of 65 patients in first CR, of whom 8 were alive and in CR, and 1 patient experienced disease relapse and died. Seven patients (10.7%) were alive without allo-BMT. Three years' overall and event-free survival for patients with FLT3-ITD mutation with high allelic ratio was 26.9% and 22.8%, respectively. Three years' overall and event-free survival for patients treated with allo-BMT was 77.8% and 78.8%, respectively, versus patients treated without allo-BMT, 16.3% and 12.8%, respectively.
FLT3-ITD mutation in pediatric AML was associated with poor treatment outcomes, and the survival of relapsing patients was extremely poor. Allo-BMT in first remission was the best treatment option. Alternative donor transplants and FLT3 inhibitors are needed to improve outcome in developing countries.
儿科急性髓系白血病（AML）中存在 FMS 样酪氨酸激酶 3（FLT3）内部串联重复（ITD）突变与诱导失败率高和生存率差有关。其存在将患者置于高危组。我们旨在描述具有 FLT3-ITD 突变的儿科 AML 的结果。
我们对 2007 年 7 月至 2017 年 7 月在埃及儿童癌症医院的 AML 病例进行了回顾性分析。
在 687 例 AML 患者中，有 71 例 FLT3 基因突变。65 例患者 FLT3 基因突变等位基因比> 0.4；43（66.1%）例患者达到完全缓解（CR）。在 43 例患者中，16 例患者维持 CR，18 例患者在首次 CR 后复发，8 例患者死亡，1 例患者失访。复发疾病患者在挽救化疗后死亡，除 1 例患者在第二次 CR 后存活外。65 例首次 CR 患者中，9 例（13.8%）接受同种异体骨髓移植（allo-BMT），其中 8 例存活且处于 CR 期，1 例患者出现疾病复发并死亡。7 例（10.7%）患者未接受 allo-BMT 仍存活。FLT3-ITD 突变高等位基因比患者的 3 年总生存率和无事件生存率分别为 26.9%和 22.8%。allo-BMT 治疗患者的 3 年总生存率和无事件生存率分别为 77.8%和 78.8%，而未接受 allo-BMT 治疗患者的 3 年总生存率和无事件生存率分别为 16.3%和 12.8%。
儿科 AML 中的 FLT3-ITD 突变与不良治疗结果相关，复发患者的生存率极差。首次缓解时进行 allo-BMT 是最佳治疗选择。在发展中国家，需要进行异基因移植和 FLT3 抑制剂的治疗以改善预后。
