Therapy related CMML: a case report and review of the literature

Therapy related chronic myelomonocytic leukemia (t-CMML) is rare. We report a 23-year-old female who developed acute fulminant hepatic failure after drug overdose. She underwent ABO incompatible orthotopic liver transplant. She received cyclophosphamide along with other immunosuppressants. Seven years later, she was diagnosed with t-CMML-2 with 45XX,-7 karyotype. She received 4 cycles of azacitidine and proceeded with allogeneic bone marrow transplant. This is the first a case of t-CMML reported in a liver transplant recipient. In this article, we also summarize all reported cases of t-CMML, and we review therapy related MDS in recipients of solid organ transplant.     

Patients’ motivations for participating in cardiovascular clinical trials: a local perspective

Objective

To investigate patients’ motivations for participating in cardiovascular clinical trials.

Methods

Patients attending TREAD Research, located within Tygerberg Hospital, Parow, Western Cape, between January 2005 and May 2006 were approached to participate in the study. Consenting patients were given a validated questionnaire to complete in their home language. All questionnaires were anonymous and 250 consecutive patients completed the questionnaire. They provided basic demographic data and rated their response to 18 statements concerning factors that may or may not have influenced their decision to participate in a clinical trial.

Results

The mean (± SD) age of subjects was 56.3 ± 10.9 years. A large percentage of the respondents were unemployed (66.5%). Access to medical care was a motivation for the majority of patients (90.5%). Ninety-six per cent of patients appreciated the regular follow up they received as trial participants; 90% of patients entered the trial to receive medication, which they could otherwise not afford. A substantial 98% of patients participated to learn more about their disease. Almost all (99%) wanted to further the scientific understanding of their condition. A reassuring 94% of subjects felt that they were not pressurised into the study; 80% of patients disagreed that participation in clinical trials was an easy way to obtain money.

Conclusions

Access to medical care and making a contribution to scientific knowledge are two of the most common motivations for participation in cardiovascular clinical trials. The role of remuneration is relatively unimportant.     

青霉胺联合硫酸锌治疗肝豆状核变性42例

1 临床资料肝豆状核变性(Wilson disease,WD)[1].42(男20,女22)例,年龄5～13(平均8)岁,其中27例以乏力、纳差、黄疸为首发症状,检查发现肝脏肿大,血清胆红素及ALT升高,4例以关节痛、手足不自主运动为首发症状,检查发现脑电图异常,3例首发症状类似佝偻病,血钙、血磷无变化,X线检查可见骨质疏松、关节间隙变窄;2例首发症状为血尿,尿常规检查发现红细胞及蛋白,6例首发症状为精神障碍,表情呆板,颅脑CT显示基底节低密度灶,脑脊液检查未见异常.入院时23例诊断明确,19例出现误诊[2],所有患儿诊断明确后,随机用青霉胺治疗9例,常用量: 600～1500 mg/d,分3次于饭前长期po[2],其临床症状消失,各项检查正常的平均时间为62 d,其中1例已出现肾病综合征症状;用硫酸锌治疗14例,常用量: 50 g/L硫酸锌2～4 mL,每天3次于饭前1 h服用.     

脊柱后路内窥镜治疗极外侧椎间盘突出症6例

0引言极外侧型腰椎间盘突出症是指突出物位于侧隐窝外侧,椎间孔及椎间孔外的腰椎间盘突出.国外报道其发生率为10.3%～11.7%,而国内报道的发生率明显偏低,约为1.24%[1].     

Efficacy and Cardiovascular Safety of Roxadustat for Treatment of Anemia in Patients with Non–Dialysis-Dependent CKD: Pooled Results of Three Randomized Clinical Trials

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Cytotoxic and antibacterial activities of endophytic fungi isolated from plants at the National Park, Pahang, Malaysia

Background  

Endophytes, microorganisms which reside in plant tissues, have potential in producing novel metabolites for exploitation in medicine. Cytotoxic and antibacterial activities of a total of 300 endophytic fungi were investigated.     

Physiological Stress in Native Southern Brook Trout During Episodic Stream Acidification in the Great Smoky Mountains National Park

Episodic stream acidification from atmospheric deposition is suspected to detrimentally impact native southern brook trout (Salvelinus fontinalis) in Great Smoky Mountains National Park (GRSM) headwater streams. To test the hypothesis that episodes of stream acidification cause physiological distress to native trout, caged fish at three sites were exposed to acid episodes during in situ bioassays conducted in June 2006 and March 2007. Stream pH decreased (>0.7 pH units) and total dissolved aluminum (Al_TD) increased (>175 μg/L) at all three sites during acid episodes in both bioassays. Whole-body sodium concentrations were significantly reduced (10–20%) following the acid episodes, when preceding 24-h mean pH values of 4.88, 5.09, and 4.87 and corresponding 24-h time-weighted average Al_TD concentrations of 210, 202, and 202 μg/L were observed. Lower whole-body sodium concentrations were correlated with elevated H⁺ and Al_TD concentrations. Loss of sodium ions in native southern brook trout was consistent with physiological distress resulting from acid exposure reported in salmonids in other investigations. Further research is necessary to conclude whether acid episodes are responsible for extirpation of brook trout from headwater streams in the GRSM.     

Effects of NXY-059 in experimental stroke: an individual animal meta-analysis

Background and purpose:

Disodium 2,4-disulphophenyl-N-tert-butylnitrone (NXY-059) was neuroprotective in experimental stroke models but ineffective in a large clinical trial. This first-ever individual animal meta-analysis was used to assess the preclinical studies.

Experimental approach:

Studies were obtained from AstraZeneca and PubMed searches. Data for each animal were obtained from the lead author of each study and/or AstraZeneca. Published summary data were used if individual data were not available. Infarct volume and motor impairment were standardized to reflect different species and scales. Standardized mean difference (SMD), coefficients from multilevel models and 95% confidence intervals (95% CI) are presented.

Key results:

Fifteen studies (26 conditions, 12 laboratories) involving rats (544), mice (9) and marmosets (32) were identified (NXY-059: 332, control: 253) with individual data for 442 animals. Four studies were unpublished. Studies variably used randomization (40%), blinding of surgeon (53%) and outcome assessor (67%). NXY-059 reduced total (SMD −1.17, 95% CI −1.50 to −0.84), cortical (SMD −2.17, 95% CI −2.99 to −1.34) and subcortical (−1.43, 95% CI −2.20 to −0.86) lesion volume; efficacy was seen in transient, permanent and thrombotic ischaemia, up to 180 min post occlusion. NXY-059 reduced motor impairment (SMD −1.66, 95% CI −2.18 to −1.14) and neglect. Evidence for performance, attrition and publication bias was present.

Conclusions and implications:

NXY-059 was neuroprotective in experimental stroke although bias may have resulted in efficacy being overestimated. Efficacy in young, healthy, male animals is a poor predictor of clinical outcome. We suggest the use of preclinical meta-analysis before initiation of future clinical trials.