scholarly journals THALIDOMIDE FOR PATIENTS WITH THALASSEMIA INTERMEDIA: A RETROSPECTIVE MULTICENTER CLINICAL STUDY

2020 ◽  
Vol 12 (1) ◽  
pp. e2020021
Author(s):  
Kun Yang ◽  
Yi Wu ◽  
Yali Zhou ◽  
Tianhong Zhou ◽  
Li Wang ◽  
...  

Objective: This study focused on the efficacy and safety of thalidomide for patients with thalassemia intermedia (TI) in a multicenter trial. Methods:Clinical and laboratory data of 62 patients subjected to thalidomide therapy in four centers were retrospectively analyzed. We evaluated the efficacy and safety of thalidomide in the short-term (three months) and long-term follow-up (12 and 24 months). Response to thalidomide was defined as follows: Main Responder (MaR) showing an increase in Hb level of >2.0 g/dl or removal from blood transfusion and Minor Responder (MiR) achieving elevated hemoglobin (Hb) level of 1.0-2.0 g/dl or ≥50% reduction in blood transfusion frequency. Results:The overall response rate (ORR) of 62 patients with TI was 93.5% (58/62), with MaR and MiR rates accounting for 62.9% (39/62) and 30.6% (19/62) in short-term follow-up and 66.1% (41/62) and 27.4% (17/62) in long-term follow-up, respectively. The clinical response during long-term follow-up was maintained and the Hb level remained stable during the observation period. The response was still observed in patients with dose reduction despite a slight decrease in Hb level. However, Hb decreased rapidly to the baseline level after drug discontinuation. No effect of thalidomide on spleen size in nonsplenectomized patients was evident. Minimal side-effects were documented throughout, except peripheral neurotoxicity in one patient. Nevertheless, the mean serum ferritin (SF) level was significantly increased after treatment. Conclusion: Thalidomide had significant therapeutic effects on patients with TI, and the response was sustained with acceptable short-term and long-term adverse reactions. While these preliminary results support the potential long-term efficacy and safety of thalidomide as a therapeutic agent for TI, several issues need to be addressed before its application in the clinic.

2018 ◽  
Vol 47 (12) ◽  
pp. 3019-3029 ◽  
Author(s):  
Jayson Lian ◽  
Amin Mohamadi ◽  
Jimmy J. Chan ◽  
Phillip Hanna ◽  
David Hemmati ◽  
...  

Background: Numerous treatment options have been proposed for enthesopathy of the extensor carpi radialis brevis (eECRB). Purpose: To (1) compare the efficacy and safety of nonsurgical treatment options for eECRB described in randomized placebo-controlled trials at short-term, midterm, and long-term follow-up and (2) evaluate outcomes in patients receiving placebo. Study Design: Systematic review and meta-analysis. Methods: Following PRISMA guidelines, 4 electronic databases were searched for randomized placebo-controlled trials for eECRB. Studies reporting visual analog scale (VAS) for pain scores and/or grip strength were included. Random- or fixed-effects meta-analysis was employed to compare treatments with at least 2 eligible studies using the standardized mean difference and odds ratio. The study protocol was registered at PROSPERO (ID: CRD42018075009). Results: Thirty-six randomized placebo-controlled trials, evaluating 11 different treatment modalities, with a total of 2746 patients were included. At short-term follow-up, only local corticosteroid injection improved pain; however, it was associated with pain worse than placebo at long-term follow-up. At midterm follow-up, laser therapy and local botulinum toxin injection improved pain. At long-term follow-up, extracorporeal shock wave therapy provided pain relief. With regard to grip strength, only laser therapy showed better outcomes in comparison with placebo. While there was no difference among various treatments in the odds ratio of an adverse event, they all increased adverse events compared with placebo. In placebo-receiving patients, a sharp increase in the percentage of patients reporting mild pain or less was observed from 2% at short-term follow-up to 92% at midterm follow-up. Conclusion: Most patients experienced pain resolution after receiving placebo within 4 weeks of follow-up. At best, all treatments provided only small pain relief while increasing the odds of adverse events. Therefore, if clinicians are inclined to provide a treatment for particular patients, they may consider a pain relief regimen for the first 4 weeks of symptom duration. Patient-specific factors should be considered when deciding on treatment or watchful waiting.


2019 ◽  
Vol 19 (1) ◽  
Author(s):  
Yue Gu ◽  
Xueqin Yang ◽  
Hang Liang ◽  
Deli Li

Abstract Background Statin has been more and more widely used in chronic liver disease, however, existed studies have attained contradictory results. According to the present study, we aimed to test the efficacy and safety of statin via a meta-analysis. Methods Different databases were searched for full-text publication based on inclusion and exclusion criteria. For data-pooling, fixed-effect model was applied if heterogeneity wasn’t detected. Otherwise, random-effect model was adopted. Heterogeneity was detected by I squire (I2) test. All results of analysis were illustrated as forest plots. Publication bias was assessed using the Begg’s adjusted rank correlation test. Standard mean difference (SMD) was calculated in continuous variables. Pooled hazard ratio or odds ratio was calculated in catergorical variables. Results Seventeen clinical studies were finally included. Hepatic portal hemodynamic parameters were improved in statin users for a short-term response. For a long-term follow-up, statin treatment surprisingly decreased mortality rate (HR = 0.782, 95% CI: 0.718–0.846, I2 > 50%) and lower the occurrence of hepatocellular carcinoma (HR = 0.75, 95% CI: 0.64–0.86, I2 > 50%) in liver cirrhosis. Statin seemed not to decrease the risk of esophageal variceal bleeding and spontaneous bacterial peritonitis. However, statin was proved to decrease the risk of hepatic encephalopathy and ascites. Incidence of drug related adverse events didn’t increase in statin users. Dose-dependent effects of statin on hepatocellular carcinoma development, decompensated cirrhosis events occurrence, and liver cirrhosis progression. Conclusion Statin influenced parameters of hepatic portal vessel pressure in short-term treatment. Prognosis of liver cirrhosis benefited from statin treatment in long term follow-up. The efficacy and safety of statin in liver cirrhosis treatment is confirmed. To date, similar study is hardly seen before.


VASA ◽  
2019 ◽  
Vol 48 (4) ◽  
pp. 321-329
Author(s):  
Mariya Kronlage ◽  
Erwin Blessing ◽  
Oliver J. Müller ◽  
Britta Heilmeier ◽  
Hugo A. Katus ◽  
...  

Summary. Background: To assess the impact of short- vs. long-term anticoagulation in addition to standard dual antiplatelet therapy (DAPT) upon endovascular treatment of (sub)acute thrombembolic occlusions of the lower extremity. Patient and methods: Retrospective analysis was conducted on 202 patients with a thrombembolic occlusion of lower extremities, followed by crirical limb ischemia that received endovascular treatment including thrombolysis, mechanical thrombectomy, or a combination of both between 2006 and 2015 at a single center. Following antithrombotic regimes were compared: 1) dual antiplatelet therapy, DAPT for 4 weeks (aspirin 100 mg/d and clopidogrel 75 mg/d) upon intervention, followed by a lifelong single antiplatelet therapy; 2) DAPT plus short term anticoagulation for 4 weeks, followed by a lifelong single antiplatelet therapy; 3) DAPT plus long term anticoagulation for > 4 weeks, followed by a lifelong anticoagulation. Results: Endovascular treatment was associated with high immediate revascularization (> 98 %), as well as overall and amputation-free survival rates (> 85 %), independent from the chosen anticoagulation regime in a two-year follow up, p > 0.05. Anticoagulation in addition to standard antiplatelet therapy had no significant effect on patency or freedom from target lesion revascularization (TLR) 24 months upon index procedure for both thrombotic and embolic occlusions. Severe bleeding complications occurred more often in the long-term anticoagulation group (9.3 % vs. 5.6 % (short-term group) and 6.5 % (DAPT group), p > 0.05). Conclusions: Our observational study demonstrates that the choice of an antithrombotic regime had no impact on the long-term follow-up after endovascular treatment of acute thrombembolic limb ischemia whereas prolonged anticoagulation was associated with a nominal increase in severe bleeding complications.


2020 ◽  
pp. bjsports-2020-102525
Author(s):  
Stefanos Karanasios ◽  
Vasileios Korakakis ◽  
Rod Whiteley ◽  
Ioannis Vasilogeorgis ◽  
Sarah Woodbridge ◽  
...  

ObjectiveTo evaluate the effectiveness of exercise compared with other conservative interventions in the management of lateral elbow tendinopathy (LET) on pain and function.DesignSystematic review and meta-analysis.MethodsWe used the Cochrane risk-of-bias tool 2 for randomised controlled trials (RCTs) to assess risk of bias and the Grading of Recommendations Assessment, Development and Evaluation methodology to grade the certainty of evidence. Self-perceived improvement, pain intensity, pain-free grip strength (PFGS) and elbow disability were used as primary outcome measures.Eligibility criteriaRCTs assessing the effectiveness of exercise alone or as an additive intervention compared with passive interventions, wait-and-see or injections in patients with LET.Results30 RCTs (2123 participants, 5 comparator interventions) were identified. Exercise outperformed (low certainty) corticosteroid injections in all outcomes at all time points except short-term pain reduction. Clinically significant differences were found in PFGS at short-term (mean difference (MD): 12.15, (95% CI) 1.69 to 22.6), mid-term (MD: 22.45, 95% CI 3.63 to 41.3) and long-term follow-up (MD: 18, 95% CI 11.17 to 24.84). Statistically significant differences (very low certainty) for exercise compared with wait-and-see were found only in self-perceived improvement at short-term, pain reduction and elbow disability at short-term and long-term follow-up. Substantial heterogeneity in descriptions of equipment, load, duration and frequency of exercise programmes were evident.ConclusionsLow and very low certainty evidence suggests exercise is effective compared with passive interventions with or without invasive treatment in LET, but the effect is small.PROSPERO registration numberCRD42018082703.


1987 ◽  
Vol 27 (4) ◽  
pp. 283-287 ◽  
Author(s):  
Prasad R. Palakurthy ◽  
Claudio Maldonado ◽  
Gurbachan Sohi ◽  
Nancy C. Flowers

2005 ◽  
Vol 12 (4) ◽  
pp. 344
Author(s):  
Young-Woo Kim ◽  
Ho-Guen Chang ◽  
Kyu-Nam Seo ◽  
Kee-Byung Lee ◽  
Hyung-Su Kim

2018 ◽  
Vol 80 (02) ◽  
pp. 081-087
Author(s):  
Nicola Bongartz ◽  
Christian Blume ◽  
Hans Clusmann ◽  
Christian Müller ◽  
Matthias Geiger

Background To evaluate whether decompression in lumbar spinal stenosis without fusion leads to sufficient improvement of back pain and leg pain and whether re-decompression alone is sufficient for recurrent lumbar spinal stenosis for patients without signs of instability. Material and Methods A successive series of 102 patients with lumbar spinal stenosis (with and without previous lumbar surgery) were treated with decompression alone during a 3-year period. Data on pre- and postoperative back pain and leg pain (numerical rating scale [NRS] scale) were retrospectively collected from questionnaires with a return rate of 65% (n = 66). The complete cohort as well as patients with first-time surgery and re-decompression were analyzed separately. Patients were dichotomized to short-term follow-up (< 100 weeks) and long-term follow-up (> 100 weeks) postsurgery. Results Overall, both back pain (NRS 4.59 postoperative versus 7.89 preoperative; p < 0.0001) and leg pain (NRS 4.09 versus 6.75; p < 0.0001) improved postoperatively. The short-term follow-up subgroup (50%, n = 33) showed a significant reduction in back pain (NRS 4.0 versus 6.88; p < 0.0001) and leg pain (NRS 2.49 versus 6.91: p < 0.0001). Similar results could be observed for the long-term follow-up subgroup (50%, n = 33) with significantly less back pain (NRS 3.94 versus 7.0; p < 0.0001) and leg pain (visual analog scale 3.14 versus 5.39; p < 0.002) postoperatively. Patients with previous decompression surgery benefit significantly regarding back pain (NRS 4.82 versus 7.65; p < 0.0024), especially in the long-term follow-up subgroup (NRS 4.75 versus 7.67; p < 0.0148). There was also a clear trend in favor of leg pain in patients with previous surgery; however, it was not significant. Conclusions Decompression of lumbar spinal stenosis without fusion led to a significant and similar reduction of back pain and leg pain in a short-term and a long-term follow-up group. Patients without previous surgery benefited significantly better, whereas patients with previous decompression benefited regarding back pain, especially for long-term follow-up with a clear trend in favor of leg pain.


2019 ◽  
Vol 101-B (7_Supple_C) ◽  
pp. 55-60 ◽  
Author(s):  
E. K. Laende ◽  
C. G. Richardson ◽  
M. J. Dunbar

Aims Early implant migration measured with radiostereometric analysis (RSA) has been proposed as a useful predictor of long-term fixation of tibial components in total knee arthroplasty. Evaluation of actual long-term fixation is of interest for cemented components, as well as for cementless fixation, which may offer long-term advantages once osseointegration has occurred. The objective of this study was to compare the long-term migration with one- and two-year migration to evaluate the predictive ability of short-term migration data and to compare migration and inducible displacement between cemented and cementless (porous metal monoblock) components at least ten years postoperatively. Patients and Methods Patients who had participated in RSA migration studies with two-year follow-up were recruited to return for a long-term follow-up, at least ten years from surgery. Two cemented tibial designs from two manufacturers and one porous metal monoblock cementless tibial design were studied. At the long-term follow-up, patients had supine RSA examinations to determine migration and loaded examinations (single leg stance) to determine inducible displacement. In total, 79 patients (54 female) returned, with mean time since surgery of 12 years (10 to 14). There were 58 cemented and 21 cementless tibial components. Results Migration at one year and two years was significantly correlated with long-term migration (p < 0.001). Median migration at the long-term follow-up was 0.6 mm (maximum total point motion; interquartile range (IQR) 0.4 to 0.9) for the cemented group and 0.6 mm (IQR 0.3 to 1.1) for the cementless group with no difference between groups (p = 0.99). Inducible displacement was significantly lower for the cementless components (p < 0.001). Conclusion Long-term migration was strongly correlated with two-year migration. Although long-term migration was not different for cemented or cementless tibial components, inducible displacement at the long-term visit was significantly lower for these cementless components, suggesting superior fixation. These findings support the predictive value of short-term migration in determining long-term fixation. Cite this article: Bone Joint J 2019;101-B(7 Supple C):55–60


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