小剂量利妥昔单抗治疗难治性免疫性血小板减少症临床观察

目的：探讨应用利妥昔单抗治疗难治性免疫性血小板减少症（ ITP）的疗效。方法选择诊断为难治性ITP16例,应用小剂量利妥昔单抗,每周1次（100 mg）静脉滴注,连用4周。动态检测血常规。结果完全反应4例有效,7例,无效5例,总有效率68．75％。有效患者疗效持续时间5～28个月,2例复发,其余患者疗效维持较好。结论小剂量利妥昔单抗治疗难治性免疫性血小板减少症疗效确切,毒副作用较小。     

Intérêt du daratumumab dans l’amylose AL réfractaire chez une patiente de 96 ans

Systemic immunoglobulin light-chain (AL) amyloidosis is characterized by deposition of amyloid fibrils of light chains produced by clonal CD38 + plasma cells, resulting in organ dysfunction. Cardiac involvement has a major prognostic value. Antiplasma cell chemotherapy reduces the synthesis of immunoglobulin light chains (precursors of amyloid deposits). We describe a case of AL amyloidosis in a 95-year-old patient. Our patient responded poorly to treatment with rituximab, cyclophosphamide-bortezomib-dexamethasone, and rituximab-bendamustine. Finally, the anti-CD38 antibody daratumumab was associated with the best hematologic responsiveness without significant adverse effects. In conclusion, our case suggests that daratumumab is an effective and well-tolerated alternative to chemotherapy in the treatment af AL amyloidosis in very elderly patients.     

Long-term clinical outcome and survival predictors in patients with cirrhosis after 10-mm-covered transjugular intrahepatic portosystemic shunt

Background and aimsTransjugular intrahepatic portosystemic shunts (TIPS) are successfully used in the management of portal hypertension (PH)-related complications. Debate surrounds the diameter of the dilation. The aim was to analyse the outcomes of and complications deriving from TIPS in patients with cirrhosis and identify predictors of survival.MethodsThis was a retrospective single-centre study, which included patients with cirrhosis who had a TIPS procedure for PH from 2009 to October 2018. Demographic, clinical and radiological data were collected. The Kaplan–Meier method was used to measure survival and predictors of survival were identified with the Cox regression model.ResultsA total of 98 patients were included (78.6% male), mean age was 58.5 (SD±/−9.9) and the median MELD was 13.3 (IQR 9.5–16). The indications were refractory ascites (RA), variceal bleeding (VB) and hepatic hydrothorax (HH). Median survival was 72 months (RA 46.4, VB 68.5 and HH 64.7) and transplant-free survival was 26 months. Clinical and technical success rates were 70.5% and 92.9% respectively. Age (HR 1.05), clinical success (HR 0.33), sodium (HR 0.92), renal failure (HR 2.46) and albumin (HR 0.35) were predictors of survival. Hepatic encephalopathy occurred in 28.6% of patients and TIPS dysfunction occurred in 16.3%.ConclusionsTIPS with 10-mm PTFE-covered stent is an effective and safe treatment for PH-related complications in patients with cirrhosis. Age, renal failure, sodium, albumin and clinical success are independent predictors of long-term survival.     

The controversial role of dual sequential defibrillation in shockable cardiac arrest

Background

In the United States, over 350,000 cardiac arrests occur outside of the hospital and 209,000 occur in the hospital. Shockable rhythms such as ventricular fibrillation (VF) have a survival rate of 20–30% outside of the hospital setting. Dual Sequential Defibrillation (DSD) has demonstrated success in terminating VF that is refractory to multiple attempts using a single defibrillator.

Safety and efficacy of Ahmed glaucoma valve implantation in refractory glaucomas in Northern Indian eyes

Purpose

To evaluate the safety and efficacy of Ahmed glaucoma valve (AGV) implantation in refractory glaucoma in Northern Indian eyes.

Background

The success rate of trabeculectomy remains low in cases of refractory glaucoma even with the use of antifibrotics. Glaucoma drainage devices have proven to be more efficacious in reducing intraocular pressure (IOP) in these glaucomas.

Methods

Retrospective records of 55 consecutive patients who underwent AGV implantation at Dr. Shroff’s Charity Eye Hospital, New Delhi, India from January 2003 to December 2012 were reviewed. Pre-operative data included age, gender, eye laterality, specific diagnosis, number of anti-glaucoma medications, number of prior incisional surgeries, visual acuity and IOP on medical treatment. Postoperative data included visual acuity and IOP on day one, 1 week, 1 month, 3 months, 6 months, 1 year and yearly thereafter, number of anti-glaucoma medications, any complication or additional surgical intervention required. Success was defined as IOP >5 and <22 mmHg with or without treatment.

Results

Mean IOP decreased from 39.71 ± 8.99 pre-operatively to 17.52 ± 5.72 mmHg at last follow-up (p < 0.001) and number of medications reduced from 3.27 ± 0.84 to 1.25 ± 0.88 (p < 0.001). Visual acuity remained within one Snellen line or improved at last follow-up in 47 cases (85.4%). The cumulative probability of success was 85.45% at 1 year and 79.63% at 3 years. The incidence of post-operative complications was 25.45%.

Conclusion

AGV implantation has proven to be safe and is effective in controlling IOP in refractory glaucoma in Northern Indian eyes.     

新活素对老年顽固性心力衰竭患者无创血流动力学参数的影响

目的应用无创血流动力学监测观察新活素(重组人脑利钠肽)对老年顽固性心力衰竭患者血流动力学参数的影响。方法选取2012年1月至2013年10月收治的老年顽固性心力衰竭患者38例,随机分为试验组(新活素)和对照组。两组治疗前后分别应用Bio Z ICG无创血流动力学监测仪。选取10种血流动力学参数:心率(HR)、收缩压(SBP)、心输出量(CO)、心脏指数(CI)、每搏输出量(SV)、每搏指数(SI)、速度指数(VI)、加速指数(ACI)、预射血期(PEP)、收缩时间比率(STR)进行血流动力学定量评估。结果治疗后,试验组与对照组比较,心率减慢,输出量/心脏指数、每搏输出量、每搏指数明显提高(P<0.05),预射血期、收缩时间比率明显缩短(P<0.05),血浆脑利钠肽浓度(BNP)显著下降(P<0.05),收缩压、速度指数、加速指数差异无统计学意义。结论新活素治疗老年顽固性心力衰竭具有较好的血流动力学效应,而无创血流动力学监测可作为评估心衰治疗的量化指标。     

2-Chlorodeoxyadenosine with or without daunorubicin in relapsed or refractory acute myeloid leukemia

 2-Chlorodeoxyadenosine (2-CdA) is a purine analogue which has proved to be active in acute myeloid leukemia (AML), especially in children. In adults, results yielded by 2-CdA alone or with ara-C were less encouraging. Here we report on the efficacy of 2-CdA with or without daunorubicin (DNR) in 19 relapsing or refractory adult AML patients, with a median age of 57 years. 2-CdA was administered as a continuous infusion to all patients at a dose of 0.1 mg/kg per day for 7 days. For 14 patients, DNR was added at a dose of 50 mg/m² per day on days 5, 6, and 7. Antileukemic activity was observed in all the patients, but no single complete remission was achieved. One patient had a long-lasting partial response (response rate=5%). The remaining patients died of progressive AML (n=7), uncontrollable infection with persistent disease (n=10), and cerebral hemorrhage (n=1). Median survival from start of 2-CdA therapy was 56 days. Long-lasting neutropenia and transfusion-dependent thrombopenia were encountered in all 16 evaluable patients. Grade 4 hepatic toxicity occurred in one patient. Other side effects included nausea in six, mucositis in three, and mental disturbances in three patients. Compared with 2-CdA alone, the addition of DNR to 2-CdA changed neither the response rate nor the toxicities. In conclusion, our data do not support the use of 2-CdA ± DNR for relapsing or refractory adult AML patients, at least as used in the present regimen. Received: 21 July 1997 / Accepted: 18 November 1997     

Complete remission of refractory anemia following a single high dose of cyclophosphamide

 We describe a case of stable complete remission in a patient with refractory anemia complicated by severe autoimmune hemolytic anemia, achieved with a single high dose (4 g/m²) of cyclophosphamide (cyclo). Concomitantly, an effective mobilization of CD34-positive cells was induced. Other immunosuppressive approaches including high-dose methylprednisolone, high-dose immunoglobulin, and cyclosporine had been ineffective. This finding suggests that, in selected cases, an immunologic mechanism may mediate cytopenia in myelodysplastic syndromes (MDS). In addition, it demonstrates that successful mobilization of peripheral blood stem cells can be induced with high-dose cyclo in MDS. Received: September 14, 1998 / Accepted: October 9, 1998     

Treatment of refractory unstable angina in geographically isolated areas without cardiac surgery. Invasive versus conservative strategy (TRUCS study).

AIMS: We compared invasive (on-site coronary angioplasty or emergency air-ambulance transfer for bypass grafting surgery) vs conservative (persistent medical treatment) strategies in the management of refractory unstable angina in geographically isolated hospitals without cardiac surgical facilities. METHODS AND RESULTS: One hundred and forty eight randomized patients with refractory unstable angina were compared on an intention-to-treat basis. Outcomes (invasive vs conservative): (a) in hospital: stabilization (96% vs 43%, P=0.0001), non-fatal myocardial infarction (2.6% vs 4.2%, P=ns), death (1.3% vs 8.3%, P=0.046), combined outcome (3.9% vs 12.5%, P=0.053) and hospitalization (11.4+/-6.3 vs 12.4+/-8.0 days, P=ns). (b) 30-days follow-up: non-fatal myocardial infarction (2.6% vs 4.2%, P=ns), death (2.6% vs 11.1%, P=0.030) and combined outcome (5.3% vs 15.3%, P=0.031). (c) 12 month follow-up: non-fatal myocardial infarction (3. 9% vs 4.2%, P=ns), death (3.9% vs 12.5%, P=0.053), combined outcome (7.9% vs 16.7%, P=ns), re-admissions for unstable angina: (17.1% vs 23.6%, P=ns), late coronary angioplasty: (15.8% vs 11.1%, P=ns) and (d) late coronary bypass grafting: (7.9% vs 12.5%, P=ns). CONCLUSION: Invasive treatment of patients with refractory angina in remote areas without surgical back-up results in significant in-hospital stabilization and a reduction in major events in-hospital and at 30 days. Coronary angioplasty in stand-alone units and air-transfer of these patients seems safe.     

Ketogenic diet for refractory epilepsy with MEHMO syndrome: Caution for acute necrotizing pancreatitis

BackgroundThe MEHMO (mental retardation, epileptic seizures, hypogonadism and hypogenitalism, microcephaly, and obesity) syndrome, which is caused by a hemizygous variant in the EIF2S3 gene on chromosome Xp22, is associated with significant morbidity and mortality. Refractory epileptic seizures and glucose dysregulation are characteristic manifestations of the MEHMO syndrome, which can often diminish patients’ quality of life.CaseA 5-year-old boy was referred to our hospital because of profound intellectual disability, micropenis, cryptorchidism, central hypothyroidism, and microcephaly. He had neonatal hypoglycemia at birth and later experienced refractory epileptic seizures and developed obesity and insulin-dependent diabetes. A diagnosis of MEHMO syndrome was established on the basis of the patient’s clinical manifestations and de novo novel missense variant in the EIF2S3 gene (NM_001415.3:c.805 T > G) that was detected through whole-exome analysis. Although the patient’s refractory seizures and diabetes had been well controlled with a combination of ketogenic diet (KD) therapy and insulin therapy, acute fatal necrotizing pancreatitis occurred at the age of 68 months. Moreover, despite intensive care, his condition rapidly deteriorated to multiple organ failure and acute respiratory distress syndrome, resulting in death.ConclusionThe pathophysiology of glucose intolerance in MEHMO syndrome remains to be elucidated; however, recent studies have suggested that EIF2S3 gene variants could lead to glucose dysregulation and β-cell damage in the pancreas. We suspect that in the present case, KD therapy led to an abnormal load on the beta cells that were damaged owing to eIF2γ dysfunction. Therefore, the adverse effects of KD in patients with MEHMO syndrome should be considered.