高强度聚焦超声治疗不能手术胰腺癌的1年生存分析：中国和保加利亚的多中心临床研究

目的:通过多中心临床研究分析高强度聚焦超声治疗不能手术胰腺癌的1年生存情况。方法:2013年1月到2014年1月,中国和保加利亚的不能手术的胰腺癌患者32例,男/女=20/12,年龄41~81(59.8±9.1)岁。胰腺癌病灶最大径20~60(39.3±9.6)mm,远处转移/无转移的患者18/14例。接受HIFU和/或化疗。记录术后并发症,疼痛变化和生存情况。使用Kaplan-Meier法计算总生存率和中位生存时间,比较化疗与否、是否发生远处转移的患者生存率有无差异(Log-Rank检验)。结果:术后1月,皮肤浅Ⅱ°烧伤者2例,经保守处理后1~2周愈合。伴有胰腺癌相关性疼痛症状的患者30例与术前比较,减轻的24例(80.0%),不变的5例(16.7%),加重的1例(3.3%)。所有患者的1年生存率为38.4%,中位生存时间为12个月。接受辅助化疗的患者1年生存率为57.4%,中位生存时间12月;未接受辅助化疗的患者1年生存率为20.8%,中位生存时间为6月;二者间比较有显著性差异。伴有远处转移的患者1年生存率为0%,中位生存时间为7月;无远处转移的患者1年生存率为49.2%,中位生存时间为12月;二者间比较无统计学差异。结论:中国和保加利亚的不能手术胰腺癌患者均能安全完成HIFU治疗,1年生存率和中位生存时间优于其它非手术治疗手段。辅助化疗能增加生存受益,远处转移是预后不良因素。     

Fanconi's anemia treated by allogeneic marrow transplantation

Eight patients with Fanconi's anemia were given cyclophosphamide alone (seven patients) or combined with procarbazine and antithymocyte globulin (one patient) followed by marrow grafts from HLA-identical siblings. All patients had engraftment. Seven developed acute and three chronic graft-versus-host disease (GVHD). Three patients died with GVHD and infectious complications (days 19, 56, and 82) and one with an intracerebral hemorrhage (day 540). Four patients are surviving 647- 3435 days after grafting, two are well, and two have chronic GVHD that is improving. These results show that Fanconi's anemia can be treated successfully by allogeneic marrow transplantation.     

Chronic graft-versus-host disease in 52 patients: adverse natural course and successful treatment with combination immunosuppression

Fifty-two of 175 (30%) survivors of allogeneic marrow transplantation developed chronic graft-versus-hose diseases (GVHD). Five with limited chronic GVHD had an indolent clinical course with involvement of only the skin and liver. Forty-seven with extensive chronic GVHD had an unfavorable multiorgan disorder that resembled several autoimmune diseases. Thirteen patients with extensive disease (group I) were not treated and only 2 survive with Karnofsky scores >- 70%. Mortality resulted from infections and morbidity from sica syndrome, pulmonary and hepatic insufficiency, scleroderma-like skin disease, and contractures. Another 13 (group II) received a median of 8 mo prednisone and/or a brief course of antithymocyte globulin, and 3 survive without disability. The other 21 (group III) were treated with a combination of prednisone (1.0 mg/kg/q.o.d.) and either cyclophosphamide, procarbazine, or azathioprine (all 1.5 mg/kg/day) for a median of 13 mo. Combination therapy was well tolerated with only modest myelotoxicity. Fifteen in group III had a good and 4 a fair response to treatment while 2 with no response died. Azathioprine and prednisone was the most effective regimen. All therapy has been discontinued in 12 group III patients: GVHD returned in 5 (including 2 who died in spite of retreatment) while 7 remain free of GVHD for a median of 11 (range 6-30) mo observation. Only I group III survivor is disabled and 16 of the original 21 are alive 2-4 yr after transplant with Karnofsky scores of 70%-100%. Thus, combination immmunosuppression appears to favorably affect and, in some cases, premanently arrest the adverse natural course of extensive chronic GVHD.     

Clustering of mutations in the biotin-binding region of holocarboxylase synthetase in biotin-responsive multiple carboxylase deficiency

Holocarboxylase synthetase (HCS) catalyses the biotinylation of the four biotin-dependent carboxylases found in humans. A deficiency in HCS results in biotin-responsive multiple carboxylase deficiency (MCD). We have identified six different point mutations in the HCS gene in nine patients with MCD. Two of the mutations are frequent among the MCD patients analyzed. Four of the mutations cluster in the putative biotin- binding domain as deduced from the corresponding Escherichia coli enzyme and consistent with an explanation for biotin-responsiveness based on altered affinity for biotin. The two others may define an additional domain involved in biotin-binding or biotin-mediated stabilization of the protein.      

Dysgonic fermenter-2: a clinico-epidemiologic review

In the literature to date, there have been 44 confirmed cases of infection with the Dysgonic Fermenter-2 (DF-2) bacterium. DF-2 infections appear to demonstrate a strong association with dog bites (or recent exposure to dogs) and have a predilection for patients with defective host defenses although immunocompetent individuals are also susceptible. Recently, the first two cases of documented DF-2 infection following cat bite have been reported. Of the cases reported, 42 of the 44 blood cultures grew DF-2. In one of the two cases where blood culture failed to grow DF-2, the bacterium was isolated at the time of operation from an infected myxoma of the tricuspid valve. In the other case, the organism was isolated from the eyelid margin of a case of angular blepharitis. Peripheral blood smears also afford an effective and practical clinical tool for early diagnosis; 9 of 10 patients for whom smears were done tested positive. This paper reviews the epidemiologic, microbiological, and clinical features of this relatively new illness and also offers general guidelines to physicians for clinical management. Health professionals, especially those providing care for high risk groups, should be alerted to this potentially fatal infection.