血小板生成素受体激动剂在血小板减少性疾病中应用的研究进展

血小板减少性疾病是常见的血液系统疾病,病因繁多,多见于原发免疫性血小板减少症(ITP)、骨髓衰竭性疾病、恶性血液病、慢性肝脏疾病,以及化疗引起的血小板减少症.目前,血小板生成素受体激动剂(TPORA)在各种血小板减少性疾病治疗中具有重要意义,可有效提高患者血小板计数,为血小板减少性疾病患者提供新的治疗方法.本文就TPORA在血小板减少性疾病中的临床应用和治疗安全性方面的研究进展进行综述.     

血小板生成素受体激动剂的临床应用及研究进展

血小板减少症为临床常见的疾病,可见于血液系统疾病与非血液系统疾病.随着对促血小板生成药物研究的深入,血小板生成素受体激动剂（TPORA）罗米司亭及艾曲泊帕的临床应用,可有效提高患者血小板计数,为治疗血小板减少症提供新的治疗方法.笔者拟就TPORA的作用机制、其在血液系统疾病及肝病相关的血小板减少症中的临床应用的进展进行综述.     

成年人原发免疫性血小板减少症的二线治疗方案选择

原发免疫性血小板减少症(ITP)为以血小板数目减少为特征的自身免疫性疾病.近年来,成年人ITP在发病机制、诊断、治疗等方面均获得重大进展,尤其是利妥昔单抗、血小板生成素(TPO)及血小板生成素受体激动剂(TPORA)在临床成年人ITP治疗中的广泛应用,使成年人ITP二线治疗方案的选择不再局限于脾切除术.笔者主要探讨成年人ITP患者二线治疗方案的选择,为采取一线治疗方案治疗失败的ITP患者提供个体化治疗方案.     

基于美国FAERS数据库的艾曲泊帕药品不良事件信号挖掘

目的：艾曲泊帕是一种小分子非肽类促血小板生成素受体激动剂(thrombopoietin receptor agonists,TPORA)。本研究对艾曲泊帕药品不良事件(adverse drug event,ADE)进行信号挖掘,总结相关ADE临床特征和用药风险,为临床安全用药提供参考。方法：基于美国药品食品监督管理局不良事件报告系统(FoodandDrug Administration Adverse Event Reporting System,FAERS)数据,运用比值失衡测量法中报告比值比法(reporting odds ratio,ROR)和比例报告比值法(proportional reporting ratio,PRR)对艾曲泊帕的ADE进行数据挖掘。结果：在FAERS数据库中共收集到以艾曲泊帕为首要怀疑药物的报告18 135份,共涉及ADE 48 640条(1份报告可能涉及多个ADE),阳性信号中以血小板计数降低、死亡、血小板计数升高等较为常见,网硬蛋白升高、骨髓网状纤维化、克隆进化等ADE信号强度较高,与艾曲泊帕的使用具有强相关。结论：临床在使用艾曲泊帕时,应密切关注患者...     

促血小板生成素在脐血移植中的应用

促血小板生成素(Thrombopoietin,TPO)是促进巨核细胞增殖分化最重要的细胞因子,通过与靶细胞表面的c-Mpl受体结合发挥作用,促进巨核细胞的增殖、分化、成熟以及血小板的产生.自1988年第1例脐血移植用于治疗范可尼贫血后,脐血移植以供者无风险、供、受者之间HLA配型相合程度要求较低、GVHD发生率低且程度...     

促血小板生成药物的研究现状及进展

血小板减少是临床常见的问题,血小板的生成主要依赖于血小板生成素的调节,其与特异性受体c-mpl结合后激活下游多条信号通路,包括STAT3/5,MAPK/RAs及P13K.新一代的血小板生成素模拟物克服了重组血小板生成素携带的免疫源性,其通过不同方式激活血小板生成素受体,并提高血小板数量,为治疗血小板减少的患者带来希望.此外白细胞介素及血小板源性生长因子也参与血小板的生成.单胺递质5-羟色胺是血小板内容物之一,已证实其是巨核细胞生长因子之一,通过与巨核细胞表面的5-HT2B受体结合而促进巨核细胞的增殖和分化.本文拟就促进血小板生成的细胞因子的作用机制和新的血小板生长因子的研究现状及进展作一综述.     

免疫性血小板减少症老年患者个体化治疗的研究进展

免疫性血小板减少症(ITP)是一种由体液免疫及细胞免疫异常共同介导的自身免疫性疾病,血小板破坏增多及生成减少共同参与其发病.既往普遍认为儿童及育龄期妇女为ITP的高发人群,但近年来相关文献报道,随着年龄的增长,ITP的发病率不断增高,年龄≥60岁的老年人已经成为新的ITP高发人群.由于ITP老年患者合并症多,对治疗反应及耐受性较年轻患者差,更易发生严重的出血症状,病死率增高.目前,针对ITP老年患者的经典治疗方法包括:糖皮质激素、静注人免疫球蛋白(IVIG)、脾切除术、免疫抑制剂等,但是约1/3的ITP老年患者接受治疗后,疾病仍迁延不愈.随着相关研究的深入,利妥昔单抗、血小板生成素受体激动剂(TPORA)罗米司亭与艾曲波帕、新型免疫抑制剂霉酚酸酯(MMF)等新药在治疗ITP老年患者中均显示出较好的疗效,然而根据ITP老年患者的具体情况对其实施个体化治疗变得越来越重要.笔者拟对ITP的个体化治疗方法及其最新研究进展,结合ITP老年患者这一特殊群体的具体情况进行综述.     

重组人血小板生成素治疗免疫性血小板减少症的效果分析及对炎症因子的调控作用研究

目的分析重组人血小板生成素治疗免疫性血小板减少症的效果及对炎症因子的调控作用。方法对2011年4月至2015年12月期间诊治的免疫性血小板减少症患者120例进行研究,以同期健康体检者100例为对照组。分析重组人血小板生成素的治疗效果及治疗前后患者血清中炎症因子水平的变化。结果与对照组相比,治疗前免疫性血小板减少症患者血小板、白细胞计数、血清白细胞介素1、白细胞介素2、白细胞介素6、白细胞介素10、肿瘤坏死因子α(TNF-α)、干扰素(IFN-γ)、内皮素及其受体、瘦素及其受体、基质金属蛋白酶(MMP)2和MMP9水平明显升高(P0.05);经重组人血小板生成素治疗后免疫性血小板减少症后患者血小板数明显升高(P0.05),而血红蛋白、白细胞计数和丙氨酸氨基转移酶(ALT)及总胆红素(TBil)水平无显著差异(P0.05);且治疗后血清白细胞介素1、白细胞介素2、白细胞介素6、白细胞介素10、TNF-α、IFN-γ、内皮素及其受体、瘦素及其受体、MMP2和MMP9水平明显降低(P0.05)。结论重组人血小板生成素治疗患者免疫性血小板减少症的效果显著,可能与其调控血清炎症反应蛋白有关。     

脐血移植后巨细胞病毒感染的预防及治疗

脐血移植(CBT)目前被广泛应用于治疗儿童及成年人的恶性血液病.因脐血干细胞的固有特点,CBT后巨细胞病毒(CMV)感染发生率较其他类型的造血干细胞移植有所升高,故CBT后CMV感染的防治尤为重要.本文就CBT后CMV感染的发生、预防及治疗等进行综述.     

利妥昔单抗在成年人原发性免疫性血小板减少症中的应用进展

原发性免疫性血小板减少症(ITP)是临床上常见的出血性疾病,以抗体介导的血小板破坏及血小板生成不良为特征.原发性ITP的初始治疗方案包括:糖皮质激素及静脉输注丙种球蛋白,而脾切除术、利妥昔单抗、血小板生成素(TPO)受体激动剂等常被用于治疗难治性及慢性原发性ITP患者.笔者拟就利妥昔单抗在成年人原发性ITP中的临床应用及进展进行综述.     

Umbilical cord blood transplantation]

Umbilical cord blood transplantation(CBT) from HLA-mismatched unrelated donors has been increasingly performed. One of the advantages of unrelated CBT is a low risk of severe acute graft-versus-host disease(aGVHD). The degree of HLA disparities is not strongly associated with the occurrence of severe aGVHD. The disadvantages of CBT include a delayed time of hematopoietic recovery and a high rate of graft failure, which are significantly associated with the lower number of infused cord blood cells. Despite a relatively high rate of early transplant-related mortality, cord blood cells from HLA-mismatched unrelated donors should be considered as an alternative hematopoietic stem source for both children and adults who have no suitable related and unrelated bone marrow donors. Further clinical and laboratory studies are needed to improve the outcome of CBT.     

Rational-emotive therapy vs general cognitive-behavior therapy in the treatment of low self-esteem and related emotional disturbances

The present study compared the relative effectiveness of “preferential” rational-emotive therapy (RET) and general cognitive-behavior therapy (CBT) in the treatment of low self-esteem and related emotional disturbances. Thirty-three subjects were randomly assigned to RET, CBT, and waiting-list control (WLC) groups. Therapy consisted of 8 weekly 1 1/2hour group sessions. At posttest, both the RET and CBT groups changed significantly more than the WLC group on all measures: self-esteem, depression, general and social-evaluative anxiety, anger, and rational thinking. On the self-esteem and self-efficacy measures, the CBT group changed significantly more than the RET group. At a six-month follow-up, both the RET and CBT groups maintained their gains, and there were no significant differences between groups on any measure.     

The use of cognitive behavioural therapy techniques for anxiety and depression in hospice patients: a feasibility study

Anxiety and depression are common in palliative care patients. Cognitive behavioural therapy (CBT) has been recommended as one possible treatment, and it has been suggested that non-mental health professionals may be able to treat milder disorders with CBT. The aim of this study was to assess whether the use of CBT techniques in hospice patients is an acceptable intervention, and whether a palliative care professional with short CBT training can usefully apply such techniques. A feasibility study of a brief CBT intervention following training was conducted. Qualitative analysis was carried out on patient feedback from semi-structured interviews. Hospital Anxiety and Depression Scale scores pre-and post-intervention were analysed statistically. Eleven patients completed a short course using CBT techniques. The intervention was acceptable for 10 patients. One patient found it unacceptable due to advanced disease, lethargy and severe depression. Anxiety or depression symptoms were helped in eight patients. This study showed that a palliative care professional with short training in CBT was usefully able to apply CBT techniques to hospice patients with mild-to-moderate anxiety or depression.     

Preliminary evaluation of the clinical implementation of cognitive-behavioral therapy for chronic pain management in pediatric sickle cell disease

ObjectivesEvaluate the implementation of cognitive-behavioral therapy (CBT) for chronic pain in a clinical setting by comparing youth with sickle cell disease (SCD) who initiated or did not initiate CBT.DesignYouth with SCD (ages 6–18; n = 101) referred for CBT for chronic pain were compared based on therapy attendance: Established Care; Early Termination; or Comparison (i.e., did not initiate CBT).SettingOutpatient pediatric psychology and comprehensive SCD clinics in 3 locations at a southeastern children’s hospital.InterventionsCBT delivery was standardized. Treatment plans were tailored to meet individualized needs.Main Outcome Measures: Healthcare utilization included pain-related inpatient admissions, total inpatient days, and emergency department reliance (EDR) at 12-months pre-post CBT. Patient-reported outcomes (PROs) included typical pain intensity, functional disability, and coping efficacy pre-post treatment.ResultsAdjusting for age, genotype, and hydroxyurea, early terminators of CBT had increased rates of admissions and hospital days over time relative to comparisons; those who established care had faster reduction in admissions and hospital days over time relative to comparisons. EDR decreased by 0.08 over time for Established Care and reduced by 0.01 for every 1 completed session. Patients who completed pre- and post-treatment PROs reported decreases in typical pain intensity, functional disability, and improved coping efficacy.ConclusionsEstablishing CBT care may support reductions in admissions for pain, length of stay, and EDR for youth with chronic SCD pain, which may be partially supported by patient-reported improvements in functioning, coping, and lower pain intensity following CBT. Enhancing clinical implementation of multidisciplinary treatments may optimize the health of these youth.     

Cortical bone trajectory screws in the treatment of lumbar degenerative disc disease in patients with osteoporosis

Lumbar degenerative disc disease (DDD) in the elderly population remains a global health problem, especially in patients with osteoporosis. Osteoporosis in the elderly can cause failure of internal fixation. Cortical bone trajectory (CBT) is an effective, safe and minimally invasive technique for the treatment of lumbar DDD in patients with osteoporosis. In this review, we analyzed the anatomy, biomechanics, and advantages of the CBT technique in lumbar DDD and revision surgery. Additionally, the clinical trials and case reports, indications, advancements and limitations of this technique were further discussed and reviewed. Finally, we concluded that the CBT technique can be a practical, effective and safe alternative to traditional pedicle screw fixation, especially in DDD patients with osteoporosis.     

Tailored cognitive-behavioral therapy in early rheumatoid arthritis for patients at risk: a randomized controlled trial

Recent developments in chronic pain research suggest that effectiveness of cognitive-behavioral therapy (CBT) may be optimized when applying early, customized treatments to patients at risk. For this purpose, a randomized, controlled trial with tailor-made treatment modules was conducted among patients with relatively early rheumatoid arthritis (RA disease duration of <8 years), who had been screened for psychosocial risk profiles. All participants received standard medical care from a rheumatologist and rheumatology nurse consultant. Patients in the CBT condition additionally received an individual CBT treatment with two out of four possible treatment modules. Choice of treatment modules was determined on the basis of patient priorities, which resulted in most frequent application of the fatigue module, followed by the negative mood, social relationships and pain and functional disability modules. Analyses of completers and of intention-to-treat revealed beneficial effects of CBT on physical, psychological and social functioning. Specifically, fatigue and depression were significantly reduced at post-treatment and at the 6-month follow-up in the CBT condition in comparison to the control condition, while perceived support increased at follow-up assessment. In addition, helplessness decreased at post-treatment and follow-up assessment, active coping with stress increased at post-treatment, and compliance with medication increased at follow-up assessment in the CBT condition in comparison to the control condition. Results indicate the effectiveness of tailor-made CBT for patients at risk in relatively early RA, and supply preliminary support for the idea that customizing treatments to patient characteristics may be a way to optimize CBT effectiveness in RA patients.     

脐血移植治疗遗传性疾病的研究进展与展望

目前,造血干细胞移植(HSCT)是唯一能治愈遗传性疾病的方法,而脐血作为造血干细胞的新来源,脐血移植(CBT)已被广泛应用于遗传性疾病的临床治疗.笔者回顾了CBT应用于多种类型遗传性疾病,如血红蛋白病、遗传代谢病、先天性免疫缺陷病(PID)及先天性骨髓衰竭性疾病的疗效,为遗传性疾病患者治疗方案的选择提供了理论依据.     

Biobehavioral Pain Profile in Individuals with Chronic Spine Pain

Pain in the spine is the most frequently described pain problem in primary care, afflicting at least 54 million Americans. When spinal pain becomes chronic, the prognosis for recovery is poor, often leading to disability and reduced quality of life. Clinical treatment is inadequate, often focusing on physical pathology alone. To improve treatment outcomes for chronic pain as recommended by current guidelines, the Biobehavioral Pain Profile (BPP), which includes six pain response subscales, was developed to guide cognitive behavioral therapy (CBT). The purpose of this study was to describe the BPP in 100 individuals with chronic spine pain and examine the associations between the BPP and important clinical outcomes, including chronic pain, disability, and quality of life. Participants reported a high level of pain, a low quality of life, and a high level of disability despite receiving treatment with opioids. Scores on BPP subscales including evaluating loss of control, past and current experience, physiologic responsivity, and thoughts of disease progression were elevated, indicating a need for CBT. Five of the six BPP subscales had a significant association with quality of life, chronic pain, and disability with the thought of disease progression being a strong factor for most of the clinical outcome variables. By identifying BPP, clinicians can provide appropriate treatments to improve individuals’ quality of life and prevent further disability. Further study using the BPP to guide CBT is needed.     

Nurse-led group cognitive behavioral therapy for major depressive disorder among adults in Japan: A preliminary single-group study

ObjectivesThe prevalence and burden of disease of depression necessitates effective and accessible treatment options worldwide. Since April 2016, Japanese national health insurance has covered nurse-administered cognitive behavioral therapy (CBT) for mood disorders. However, empirical support for nurse-led CBT for depression in Asian countries, especially in Japan, is still lacking. This preliminary study aimed to examine the feasibility and acceptability of nurse-led group CBT for Japanese patients with depression.MethodsIn this single-arm study, we evaluated the effects of a 6-week group CBT, led by trained nurses, on patients with major depression. The primary outcome was the Beck Depression Inventory-II (BDI-II). Assessments were conducted at the beginning and end of the intervention.ResultsOf 25 participants screened, 23 were eligible for the study (of these, three dropped out during the trial but were included in the analysis). Nurse-led group CBT led to significant improvements in the severity of depression (BDI-II, P < 0.001). The mean total BDI-II score improved from 23.1 (SD = 7.56) to 12.4 (SD = 8.57), and the pre-to post-effect size was large (Cohen's d = 1.33). After CBT, 45% of the participants were judged to be treatment responders, and 34% met the remission criteria.ConclusionsOur preliminary findings indicate that 6 weeks of nurse-led group CBT produced a favorable treatment outcome for individuals with major depression in a Japanese clinical setting. The results of this study might encourage more Asian nurses to provide CBT as a part of their nursing practice. Further controlled trials that address the limitations of this study are required.     

Umbilical cord blood transplantation

Umbilical cord blood transplantation (CBT) has been widely used as an alternative source of hematopoietic cell support for stem cell transplant patients. CBT offers several advantages over traditional stem cell sources, such as immediate availability, absence of risk for donors, lower risk of acute graft-versus-host disease, and a less stringent requirement for human leukocyte antigen matching. Recent studies suggest that CBT is a safe and effective strategy for adult patients lacking a suitable related or unrelated donor. However, delayed engraftment and delayed immune reconstitution are significant clinical problems. Novel strategies, such as the use of multiple donors, cotransplantation with accessory cells, ex vivo expansion of cord blood hematopoietic progenitor cells, graft manipulation to improve T-cell recovery, and pharmacologic interventions to restore early thymopoiesis, hold promise to enhance engraftment and immune reconstitution after CBT. These approaches may significantly increase the quality and availability of cord blood for transplantation.