Multiple myeloma: Role of autologous transplantation

Autologous stem cell transplantation (ASCT) has been the mainstay of multiple myeloma (MM) treatment for approximately 30 years. Although the continuous introduction of novel agents in the armamentarium against MM has questioned its value, ASCT remains a backbone treatment for fit MM patients. However, there is no unanimous approach for several aspects including the positioning of ASCT in the therapeutic algorithm either upfront or following the first relapse, the need for single or tandem ASCT, as well as the role of ASCT as salvage therapy. Furthermore, the anti-CD38 monoclonal antibodies along with the next generation proteasome inhibitors and immunomodulatory drugs provide a platform for optimizing the induction and consolidation/maintenance regimens. In this review, we present current data pertaining to all aspects of ASCT in MM, whereas we highlight the open issues that should be addressed in the design of future clinical trials in the field.     

Maintenance Therapy for Cutaneous T-cell Lymphoma After Total Skin Electron Irradiation: Evidence for Improved Overall Survival With Ultraviolet Therapy

BackgroundTreatment of cutaneous T-cell lymphoma (CTCL) with total skin electron beam (TSEB) therapy has been associated with deep responses but short progression-free intervals. Maintenance therapy might prolong the response duration; however, limited data assessing the outcomes with maintenance therapy after TSEB are available. We evaluated the effect of maintenance therapy on the outcomes for patients with CTCL receiving TSEB therapy.Materials and MethodsWe conducted a single-center retrospective analysis of 101 patients with CTCL who had received TSEB therapy from 1998 to 2018 at the Winship Cancer Institute of Emory University and compared the overall survival (OS) and progression-free survival (PFS) for patients had received maintenance therapy, including retinoids, interferon, ultraviolet therapy, nitrogen mustard, and extracorporeal photopheresis compared with those who had not.ResultsWe found that pooled maintenance therapies improved PFS (hazard ratio [HR], 0.60; P = .026) but not OS (median HR, 0.73; P = .264). The median PFS and OS was 7.2 months versus 9.6 months and 2.4 years versus 4.2 years for the no maintenance and maintenance groups, respectively. On exploratory analysis of the individual regimens, ultraviolet therapy was associated with improved OS (HR, 0.21; P = .034) and PFS (HR, 0.26; P = .002) compared with no maintenance.ConclusionAmong the patients with CTCL who had received TSEB therapy, maintenance therapy improved PFS for all patients, and ultraviolet-based maintenance improved both PFS and OS in a subset of patients.     

Effect of Prophylactic Post-transplant Ponatinib Administration on Outcomes in Patients With Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia

BackgroundThe objective of the present retrospective study was to evaluate the effect of ponatinib administration as maintenance therapy on the outcomes after allogeneic hematopoietic stem cell transplantation in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia.Patients and MethodsWe retrospectively analyzed the data from 34 consecutive patients treated at our institution from January 2008 to June 2019. We had administered post-transplant tyrosine kinase inhibitors preemptively before December 2017. Thereafter, we had initiated the prophylactic use of post-transplant ponatinib. The initial ponatinib dose was 15 mg/d. Ponatinib plasma trough levels were measured using the liquid chromatography-tandem mass spectrometry method 8 days after the first administration and subsequently.ResultsNine patients received ponatinib maintenance. The 2-year overall survival and leukemia-free survival in the ponatinib maintenance group tended to be better than that in the non–ponatinib group (100% vs. 70.5%, P = .10; and 100% vs. 50.8%, P = .02, respectively). In the first 7 of the 9 consecutive patients, the median plasma concentration after ponatinib administration (15 mg/d) was 15.6 ng/mL (range, 4.8-23.3 ng/mL). Although the treatment schedule for 1 patient was altered because of adverse effects (elevation of serum amylase and neutropenia), ponatinib administration was continued for all the patients, except for 1 patient with molecular relapse. One patient developed a transient elevation of serum lipase. No patient presented with any arterial occlusive events.ConclusionOur results have indicated that the strategy of ponatinib maintenance after allogeneic hematopoietic stem cell transplantation is safe, efficacious, and promising.     

成年血液透析患者矿物质及骨代谢紊乱调查

目的 调查江苏省昆山市第一人民医院维持性血液透析（maintenance hemodialysis,MHD）患者的慢性肾脏病矿物质及骨代谢异常（chronic kidney disease-mineral and bone disorders,CKD-MBD）情况,比较分析老年和非老年患者CKD-MBD的特点,为临床治疗提供依据.方法 调查234例MHD患者的透析龄、透前肌酐、血小板、白蛋白、血色素、血钙、血磷、ALP及iPTH的水平及临床资料,与指南比较分析老年组和非老年组血钙、血磷、ALP及iPTH的特点.结果 我院MHD患者非老年组患病率高于老年组（P＜0.05）;透析龄、干体重、透前肌酐、白蛋白水平等指标两组间差异无统计学意义（P＞0.05）;血清钙、磷、iPTH达标率分别为83.76％、19.66％、40.17％.两组比较,老年组血清钙、磷、iPTH、ALP均不同程度的低于非老年组,差异有统计学意义（P＜0.05）.结论 老年组MHD患者CKD-MBD指标低于非老年组,提示两组患者CKD-MBD发生机制存在差异,老年患者容易合并低转运骨病（akinesis bone disease,ABD）.     

雌激素对维持性血透妇女患者骨质疏松的影响

探讨雌激素水平在维持性血透(MHD)妇女骨质疏松发生中的作用.选择年龄18～45岁的妇女120例,其中MHD(≥3个月)妇女60例为对照组,MHD(≥3个月)且罹患骨质疏松60例妇女为观察组.两组研究对象均检测血浆雌二醇、肿瘤坏死因子α(TNF-α)、甲状旁腺素(PTH)及血钙水平,同时用定量CT法(QCT)测定骨密度.观察组雌二醇水平低于对照组(P<0.05),TNF-α水平观察组高于对照组(P<0.05),PTH和血钙水平两组间无显著差异(P=0.567和P=0.588);直线相关分析示,观察组雌二醇与骨密度呈正相关(r=0.865,P<0.01);观察组经多元线性回归分析示,雌二醇、血钙浓度与骨密度值呈正相关、TNF-α、PTH与骨密度呈负相关(F=140.32,P<0.01),且雌二醇对骨密度的影响较大(t=5.386,P<0.01).血浆低雌激素水平是MHD妇女骨质疏松发生的一个主要因素,且可以通过调节TNF-α水平而促进骨质疏松的发生.     

慢性肾功能衰竭维持性血液透析患者死亡原因临床分析

目的 :通过对复旦大学附属中山医院近 10年住院期间死亡的尿毒症维持性血透患者的死亡原因进行回顾性分析 ,以期为临床提供一些可防治措施。方法 :选择该院 1990年 1月～ 12月在住院期间死亡的维持性血透患者共 99例 ,将 1999年 1月～ 12月在该院接受 1年以上维持性血透的存活的 2 32例尿毒症患者作为对照进行临床分析。结果 :因心脏原因死亡 4 0例 ,占4 0 .5 % ;脑血管意外 2 9例 ,占 2 9.3% ;感染 15例 ,占 15 .1%。死亡组患者的平均年龄显著高于对照组 (P <0 .0 5 ,平均血透维持时间显著短于对照组 (P <0 .0 0 1) ,血透期间体重的增长率和左心室质量 (LVM)均显著高于对照组 ,而血清白蛋白浓度和红细胞压积则显著低于对照组。因心力衰竭死亡患者其透析期间体重增长率和LVM均显著高于总死亡组。因脑溢血死亡患者的收缩血压显著高于对照组 (P <0 .0 5 )。因感染死亡的患者其血清白蛋白浓度和红细胞压积均显著低于总死亡组。结论 :心力衰竭、脑溢血和肺部感染是本院尿毒症维持性血液透析患者最常见的 3种死亡原因。高龄 (6 5岁以上 )、高血压、低白蛋白血症、严重贫血和左心室肥厚是慢性肾功能衰竭维持性血液透析患者死亡的主要危险因素     

不安腿综合征对血液透析患者生活质量及睡眠质量影响的研究

目的了解北京大学第一医院肾内科血液透析中心不安腿综合征(RLS)的患病率,对其严重程度进行量化评分,并初步探讨RLS的影响因素及其严重程度对睡眠质量和生活质量的影响。方法以我院108例因慢性肾功能不全进行维持性血液透析治疗的患者为研究对象。不安腿综合征的诊断采用国际不安腿综合征研究小组制定的不安腿诊断标准量表。以匹兹堡睡眠质量指数(PSQI)和简明健康状况调查表(SF-36量表)分别进行生活质量和睡眠质量评估。记录患者的透析方案、生化指标及治疗用药等资料,探讨RLS的影响因素。随机抽取一组不伴RLS患者为对照组,进一步评估RLS对睡眠质量和生活质量的影响。结果①我院血液透析中心RLS患者共14例,男性7例,女性7例,其患病率为13%。②RLS患者与不伴RLS患者在年龄、性别、透析龄、每周透析时间,尿素清除率,透析前血肌酐、尿素氮、血红蛋白、红细胞压积、铁蛋白、转铁蛋白饱和度、钙、磷、钙磷乘积、全段甲状旁腺激素(iPTH)等生化指标,以及是否使用铁剂、钙离子拮抗剂等治疗方面均未发现差异。③RLS组PSQI为(10.86±5.48),不伴RLS组PSQI为(4.93±4.73),两者间差异具有显著性(P=0.005)。但RLS严重程度仅与主观睡眠质量具有相关性(P<0.05),与SF-36分值呈负相关(r=-0.661,P=0.01)。结论我中心维持性血液透析患者中RLS患病率为13%。RLS患者的睡眠较差,主要表现在主观睡眠质量、睡眠时间、习惯睡眠效应、日间功能等方面。同时维持性血液透析RLS患者的RLS严重程度影响到患病者的生活质量。     

维持性血液透析患者动静脉内瘘血栓形成影响因素分析

目的探讨维持性血液透析患者动静脉内瘘血栓形成的影响因素。方法观察首次动静脉造瘘的患者176例,每半年测定血清白蛋白（ALB）、C反应蛋白（CRP）、尿素氮（BUN）、β2-微球蛋白（β2-MG）、全段甲状旁腺激素（iPTH）以及血浆纤维蛋白质（Fib）和血红蛋白（Hb）等项目1次。对其中122例最终因血栓形成导致内瘘失功的患者进行相关因素与动静脉内瘘寿命之间COX回归分析。结果动静脉内瘘血栓形成与原发病为糖尿病、ALB、CRP、Fib存在回归关系。结论糖尿病、CRP、Fib是动静脉内瘘血栓形成的危险因素,ALB是动静脉内瘘的保护因素。     

不同营养评估方法与维持性血液透析患者死亡关系的比较

目的 本研究的目的是研究不同的营养评估方法与维持性血液透析患者的预后的关系. 方法 对我院血液透析中心的83名患者进行营养评估,包括主观综合性营养评估（subjective global assessment,SGA）、营养不良-炎症评分（malnutrition inflammation score,MIS）和微型营养评定简表（mini nutrition assessment short form,MNA-SF）.同时收集基本资料、辅助检查结果.随访48月（40±13月）,采用Kaplan-Meier以及Cox回归分析比较不同营养评估方法与患者全因死亡风险的差别. 结果 Kaplan-Meier分析提示：SGA＜25分患者的死亡风险较SGA≥25分患者高（P＜0.05）,MIS＞10分组比MIS≤10分死亡风险高（P＞0.05）.以MNA-SF分组,MNA-SF≥11分死亡风险低于NA-SF＜11分（P＞0.05）.多因素Cox回归分析提示,SGA及MIS不同分组仍然与维持性血液透析患者不同的全因死亡风险有关（P＜0.05）. 结论 不同营养评估方法对于维持性血液透析患者长期生存的预测能力不同,SGA法以及MIS法所评价的营养状况与维持性血液透析患者长期的全因死亡风险有较好相关性.     

品质管理圈活动在减少维持性透析患者急诊透析次数中的应用

目的探讨品质管理圈（品管圈）活动在减少维持性透析患者急诊透析次数中的应用效果。方法2012年12月起血液透析中心8名护理人员和1名医生自愿组成1个品管圈,通过评价选定＂减少维持性透析患者急诊透析次数＂为活动主题。通过问卷调查、访谈、比较研究等方法对维持性透析患者发生急诊透析情况进行分析,制定相关的对策并实施。比较改善前（2013年2月3日-5月3日）与改善后（2013年7月2日-10月2日）急诊透析情况。结果通过品管圈活动,维持性透析患者急诊透析次数由改善前的平均11.7例次/月减少至改善后的5.3例次/月,超出了预期目标。同时圈员在解决问题能力、责任心、沟通协调能力、自信心、积极性以及品管圈手法应用各方面都得到了不同程度的提高。结论品管圈活动不仅可以减少维持性透析患者急诊透析的次数,同时也可提高圈员间的团队凝聚力、和谐度及质量管理能力。