treatment-free remission

免治疗缓解
  • 文章类型: Journal Article
    维奈托克和低甲基化药物的联合治疗显着改善了不适合接受强化化疗的患者的预后。最近发表的对VIALE-A试验的探索性分析报告说,达到缓解的患者中有高达51%的患者存活超过2年。这些数据以及来自现实生活中的数据,导致质疑在长期幸存者中继续治疗多长时间是合适的。因此,最近的回顾性研究表明,在部分患者中暂停治疗,同时维持长期反应的可行性。此外,这些研究表明,再治疗可能会导致近三分之一的患者第二次缓解。我们报告了一例接受维奈托克和氮杂胞苷抢救治疗的患者,由于严重的血液学毒性,在母细胞清除后停止了几个周期。尽管暂停,他保持了持续近一年的持续反应,并在第二次复发时使用相同的组合成功治疗。
    Combined therapy with venetoclax and hypomethylating agents has significantly improved the outcome of unfit patients ineligible for intensive chemotherapy. A recently published exploratory analysis of the VIALE-A trial reported that up to 51% of patients achieving remission survived more than 2 years. These data along with those from reallife settings, lead to questioning how long it is appropriate to continue treatment in long-term survivors. Accordingly, recent retrospective studies suggested the feasibility of suspending therapy in selected patients while maintaining prolonged responses. Also, these studies showed that retreatment may induce a second remission in almost a third of patients. We report the case of a patient who received salvage therapy with venetoclax and azacytidine, that was discontinued few cycles after blasts clearance because of severe hematological toxicity. Despite suspension, he maintained a sustained response lasting almost one year and was successfully retreated with the same combination when a second relapse occurred.
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  • 文章类型: Case Reports
    1型糖尿病是一种慢性疾病,其特征是由于胰岛素缺乏引起的代谢异常和高血糖。由于胰腺β细胞的自身免疫破坏,胰岛素产生迅速下降。1型糖尿病的部分缓解(蜜月期)在新诊断的1型糖尿病的儿童和年轻人中很常见。存在β细胞功能的暂时恢复,使得很少或不需要外源性胰岛素。在需要静脉内胰岛素和随后的皮下胰岛素治疗的紧急入院后不久停止胰岛素治疗可能对患者和医疗保健提供者都是可怕的。在此期间,受影响的患者需要教育和支持。本报告描述了一名28岁男子的病例,该男子向急诊科就诊,具有1型糖尿病和糖尿病酮症酸中毒的特征。他接受了静脉输液和静脉胰岛素治疗,并采用皮下胰岛素方案出院。尽管对几种类型的胰岛细胞自身抗体检测呈阳性,患者能够在诊断后3个月内停止胰岛素治疗.患者保持自我启动的低碳水化合物饮食,有规律的减重锻炼,和正常的葡萄糖水平,而不需要胰岛素治疗。1型糖尿病的蜜月期,隐匿性自身免疫性糖尿病,和酮症倾向的2型糖尿病被讨论为重要的鉴别诊断。
    Type 1 diabetes is a chronic disease characterized by abnormal metabolism and hyperglycemia due to insulin deficiency. There is a rapid decline in insulin production due to autoimmune destruction of the pancreatic beta cells. Partial remission (honeymoon phase) of type 1 diabetes is common in children and young adults with newly diagnosed type 1 diabetes. There is temporary restoration of beta cell function such that little or no exogenous insulin is required. Stopping insulin therapy soon after an emergency admission requiring intravenous insulin and subsequent subcutaneous insulin therapy can be frightening for both patient and healthcare provider. Affected patients require education and support during this period. This report describes a case of a 28-year-old man who presented to the emergency department with features of type 1 diabetes and diabetic ketoacidosis. He was treated with intravenous fluids and intravenous insulin and discharged on a subcutaneous insulin regimen. Despite testing positive for several types of islet cell autoantibodies, the patient was able to stop insulin therapy within three months of diagnosis. The patient maintained a self-initiated low-carbohydrate diet, regular weight-reducing exercise, and normal glucose levels without the need for insulin therapy. The honeymoon phase of type 1 diabetes, latent autoimmune diabetes, and ketosis-prone type 2 diabetes are discussed as important differential diagnoses.
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  • 文章类型: Case Reports
    BCR-ABL1酪氨酸激酶抑制剂达沙替尼在慢性粒细胞白血病(CML)治疗中有效。达沙替尼的主要已知不良反应包括胸腔积液和肺动脉高压(PAH);然而,潜在机制尚不清楚.该病例报告描述了由心脏病专家和血液学家之间的多学科合作决定的两步达沙替尼剂量减少,以管理导致无治疗缓解(TFR)的PAH。这表明了该领域的一项重要改进。在这里,从2014年5月起,1名43岁的CML女性患者接受了每日100mg达沙替尼作为一线治疗.在开始服用达沙替尼之前,她的心电图和经胸超声心动图(TTE)没有明显异常。她在2015年6月出现了腿部水肿,TTE显示出较高的跨囊压力梯度值。基于这些发现,我们诊断为达沙替尼导致的PAH和右侧心力衰竭.然而,由于已确认分子反应(MR4.5)(国际规模:BCR-ABL1IS≤0.0032%)持续,在与心脏病学家进行了彻底讨论后,血液学家决定将达沙替尼剂量减少至70mg.剂量减少后,PAH立即改善;然而,在2017年再次观察到,第二次剂量减少至50mg后有所改善.此外,开始心血管药物治疗.PAH在2018年再次恶化,持续的MR4.5。因此,我们决定停用达沙替尼,因为MR4.5已持续超过4年.达沙替尼停药后,PAH再次改善,MR4.0(BCR-ABL1IS≤0.01%)水平已持续数年。此后,没有观察到PAH的明显恶化。我们介绍了一例可逆性达沙替尼诱导的PAH。通过几次剂量减少,可以成功治疗复发性PAH。并实现了TFR。这部分是由于血液学家和心脏病学家之间的有效合作。如果需要,在停用达沙替尼之前,可以考虑减少剂量作为治疗策略.
    The BCR-ABL1 tyrosine kinase inhibitor dasatinib is effective in chronic myeloid leukemia (CML) treatment. The major known adverse effects of dasatinib include pleural effusion and pulmonary arterial hypertension (PAH); however, the underlying mechanisms remain unclear. This case report describes a two-step dasatinib dose reduction decided by multi-disciplinary collaboration between cardiologists and hematologists for the management of PAH that led to treatment-free remission (TFR), suggesting an important improvement in the field. Herein, a 43-year-old woman with CML was administered 100 mg of dasatinib daily as a first-line therapy from May 2014. There were no evident abnormalities on her electrocardiogram and transthoracic echocardiography (TTE) charts before she started taking dasatinib. She developed leg edema in June 2015, and the TTE showed a high transtricuspid pressure gradient value. Based on these findings, we diagnosed PAH and right-sided heart failure due to dasatinib. However, since it was confirmed that the molecular response (MR4.5) (International Scale: BCR-ABL1IS ≤ 0.0032%) was sustained, the hematologist decided to reduce the dasatinib dose to 70 mg after thorough deliberations with the cardiologists. After the dose reduction, the PAH improved immediately; however, it was observed again in 2017, which improved with a second dose reduction to 50 mg. Additionally, cardiovascular drug therapy was initiated. The PAH was exacerbated again in 2018 with sustained MR4.5. Hence, we decided to discontinue dasatinib as the MR4.5 had been sustained over 4 years. After the discontinuation of dasatinib, PAH improved again, and near MR4.0 (BCR-ABL1IS ≤ 0.01%) level has been sustained for several years now. Thereafter, no apparent deterioration in PAH was observed. We present a case of reversible dasatinib-induced PAH. Successful management of recurrent PAH was possible with several dose reductions, and TFR was achieved. This was partly due to effective collaboration between the hematologists and cardiologists. If needed, dose reduction as a treatment strategy may be considered before discontinuing dasatinib.
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