Deferiprone Versus Sequential Deferiprone-Deferoxamine Treatment in Thalassemia Major: A Five Years Multicenter Randomized Clinical Trial under the Auspices of the Society for the Study of Thalassemia and Hemoglobinopathies (SoSTE).

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 575-575 ◽  
Author(s):  
Aurelio Maggio ◽  
Marcello Capra ◽  
Liana Cuccia ◽  
Francesco Gagliardotto ◽  
Carmelo Magnano ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Side Effects ◽  
Serum Ferritin ◽  
Statistical Significance ◽  
Thalassemia Major ◽  
Randomised Clinical Trial ◽  
Short Term ◽  
Long Term Study ◽  
One Year

Abstract Three short-term randomised clinical trials suggested not difference of Deferiprone (L1) vs Deferoxamine (DFO) in term of iron overload efficacy in thalassemia major (TM) patients. To assess whether L1 (75mg/Kg) alone was comparable to a sequential treatment using L1 (75mg/Kg) for 4 days and DFO (50mg/Kg) for 3 days, we carried ahead a large long-term randomised clinical trial. One-hundred and forty consecutive patients with TM and serum ferritin between 1,500 and 3,000 ng/ml were randomly assigned to L1 (n°69) or sequential L1-DFO (n°71) and treated for 5 years. The main measure of efficacy was the reduction of serum ferritin levels. Secondary outcomes were liver and heart iron contents assessed by T2* magnetic resonance. After one year-treatment the mean serum feritin reduction was −105 ± 90.4 in L1 and −409 ± 64.2 in sequential L1-DFO treatment (p <0.01), respectively. The greater mean serum ferritin reduction of sequential L1- DFO treatment was also confirmed all over the study (2° year L1 106 ± 713, L1-DFO -321 ± 92 (p <0.01); 3° year L1 137 ± 137, L1-DFO -292 ± 117 (p <0.05); 4° year L1 216 ± 200, L1-DFO-230 ± 170 (p <0.01); 5° year L1 336 ± 244, L1-DFO -598 ± 203 (p <0.01)). After one-year treatment this sequential group showed greater efficacy in term of serum ferritin levels reduction (−409 ± 64.2) in comparison with the DFO alone arm (−232 ± 619) of a previous randomised multicenter clinical trial in which a comparable cohort of patients were studied (p<0.05). Reversible leukocytopenia was shown in 8 (11.5%) L1 and in 7 (9.8%) sequential L1-DFO treated patients. No agranulocythosis was reported on sequential L1-DFO treated patients during the 5 years study. Hypertransaminasemia developed in 13 (18.8%) L1 and in 5 (7%) sequential L1-DFO treated patients. No other major side effects have been reported. Discontinuation of treatment was necessary in 55.6% L1 and in 57.7% sequential L1-DFO treated patients (chi2 0.03, p=0.86). The failures of treatment were less in sequential L1-DFO arm (n°2) in comparison to L1 alone arm (n°8), although this difference not so far reached the statistical significance (chi2 3.4, p=0.06). These findings suggest that sequential L1-DFO treatment in a long-term study is more effective than L1 alone with milder and reversible side effects. Moreover, its efficacy is also higher in comparison with DFO alone at short-term evaluation. Fig. 1 VARIATIONS OF THE FERRITIN LEVELS DURING FIVE YEARS TREATMENT BETWEEN THE TWO ARMS OF THE TRIAL Fig. 1. VARIATIONS OF THE FERRITIN LEVELS DURING FIVE YEARS TREATMENT BETWEEN THE TWO ARMS OF THE TRIAL

Efficacy and safety of percutaneous administration of dihydrotestosterone in children of different genetic backgrounds with micropenis

2017 ◽  
Vol 30 (12) ◽  
Author(s):  
Dan Xu ◽  
Liangsheng Lu ◽  
Li Xi ◽  
Ruoqian Cheng ◽  
Zhou Pei ◽  
...  
Keyword(s):  
Androgen Receptor ◽  
Side Effects ◽  
Low Doses ◽  
Efficacy And Safety ◽  
Short Term ◽  
Term Study ◽  
Long Term Study ◽  
Percutaneous Administration ◽  
Open Prospective Study

AbstractBackground:Exogenous androgen supplement is an optional treatment for micropenis; however, its use in childhood is controversial due to potential side effects.Methods:Twenty-three children (mean age: 4.07±3.4 years) with micropenis of unknown causes harboring the 46,XY karyotype were recruited in an open prospective study. Androgen receptor (Results:Two patients were found withConclusions:Short term and local application of DHT at low doses in patients with micropenis could accelerate penile growth effectively without evident side effects; however, precautions still need be taken due to the paucity of long term study and the lack of ideal DHT dosage.

Short-term vision gains at 12 weeks correlate with long-term vision gains at 2 years: results from the BEVORDEX randomised clinical trial of bevacizumab versus dexamethasone implants for diabetic macular oedema

2017 ◽  
Vol 102 (4) ◽  
pp. 479-482 ◽  
Author(s):  
Hemal Mehta ◽  
Samantha Fraser-Bell ◽  
Vuong Nguyen ◽  
Lyndell L Lim ◽  
Mark C Gillies
Keyword(s):  
Clinical Trial ◽  
Macular Oedema ◽  
Diabetic Macular Oedema ◽  
Randomised Clinical Trial ◽  
Macular Thickness ◽  
Central Macular Thickness ◽  
Short Term ◽  
Treatment Allocation ◽  
Visual Outcomes

AimTo determine whether early vision gains predict long-term visual outcomes in the BEVORDEX randomised clinical trial of bevacizumab or dexamethasone implants for diabetic macular oedema.MethodsPost hoc analysis of 68 study eyes (77%) that completed 2 years follow-up of the BEVORDEX multicentre randomised clinical trial set in Australia (ClinicalTrials.gov identifier: NCT01298076). Study eyes from both groups were combined and stratified by visual acuity (VA) change in the first 12 weeks in to three groups: (a) suboptimal gain: <5 letters gain (includes VA loss), (b) moderate gain: 5–9 letters gain, (c) pronounced gain: ≥10 letters gain. This was correlated with VA outcome at 104 weeks taking into account treatment allocation and baseline lens status.ResultsThe change in VA in the first 12 weeks was significantly correlated with VA change at 104 weeks (p<0.001). This was independent of treatment allocation (p=0.353) and lens status at baseline (p=0.593). The change in central macular thickness at 12 weeks did not correlate with VA gain at 104 weeks (p=0.847).ConclusionShort-term visual gain at 12 weeks was strongly correlated with long-term vision improvement independent of treatment allocation or baseline lens status. Early improvement in central macular thickness was not predictive of long-term visual outcomes.Trial registration numberNCT01298076, Post-results.

scholarly journals Kontrowersje wokół długoterminowych badań Gillesa-Erica Séraliniego nad bezpieczeństwem zdrowotnym kukurydzy GMO

2014 ◽  
Vol 12 (3) ◽  
pp. 33-54
Author(s):  
Katarzyna Lisowska ◽  
Alexander Cortez
Keyword(s):  
Safety Evaluation ◽  
Negative Effects ◽  
Short Term ◽  
Term Study ◽  
Experimental Animals ◽  
Long Term Study ◽  
Chemical Toxicology ◽  
The Subject ◽  
One Year

At the end of the year 2012, Food and Chemical Toxicology published a long-term study by Seralini et al., describing the safety evaluation of genetically modified NK603 maize and Roundup herbicide. Contrary to previous, short-term studies, this experiment revealed some negative effects of these substances on the health of experimental animals. GM feeds and Roundup generate revenue worth millions of dollars. This may be the reason why Seralini’s paper has became the subject of much heated criticism, mainly from parties linked to business and agro-biotechnology. After one year of debate, the editors of Food Chem Toxicol. decided to retract the paper, an unprecedented event given that the published article was peer-reviewed and there was no evidence of plagiarism or fraud. Here, we stress the results of Seralini’s study, discuss the methodological hints of that work and cite the commentaries on the whole situation.

scholarly journals Comparison of Peritoneal Closure Versus Non-closure After Non-infected Elective Laparotomy with a Midline Incision: A Randomized Clinical Trial

2021 ◽  
Vol In Press (In Press) ◽  
Author(s):  
Arash Mohammadi Tofigh ◽  
Mohammad Hossein Jafarzadeh
Keyword(s):  
Clinical Trial ◽  
Incisional Hernia ◽  
Medical Center ◽  
Midline Incision ◽  
Short Term ◽  
Peritoneal Closure ◽  
Group Randomized Trial ◽  
One Year ◽  
Closure Group

Background: There is no consensus regarding closure or non-closure of peritoneum in laparotomy, and this topic remains a controversy among surgeons. Objectives: This clinical trial aimed to compare short-term and long-term benefits of peritoneal closure with non-closure in an academic medical center. Methods: In this double-blinded two-arm parallel-group randomized trial, 124 patients undergoing laparotomy with midline incision were assessed from March 2019 to September 2019 at Imam Hossein Medical Center, Tehran, Iran. We used the Rand function of the Excel software to randomly assign 62 patients to the peritoneal closure group and 62 patients to the non-closure group. The patients were evaluated for short-term complications including wound-related fever, infection, need for analgesics, pain in the first 2, 6, 24, and 48 hours postoperatively according to the Visual Analogue scale (VAS), duration of hospitalization, as well as long-term complications including incisional hernia and intraperitoneal adhesion one year after the surgery. Statistical analysis was carried out with SPSS version 22 software. Results: The non-closure peritoneum group had a lower rate of wound-related fever, infection, and analgesic need than the peritoneal closure group, but these differences were not statistically significant (P = 0.488, P = 0.455, and P = 0.062, respectively). The adhesion rate and incisional hernia incidence one year after the surgery were not significantly different between the two groups (P = 0.363, P = 0.586). Pain intensity was significantly lower in the non-closure group than in the closure group in the first two, six, and 24 hours (P = 0.008, P = 0.004, and P = 0.047, respectively) but not significantly at 48 hours (P = 0.146). Conclusions: Peritoneum closure after non-emergency, non-infected laparotomy increases the postoperative pain while it has no benefit for long-term complications like incisional hernia or intra-peritoneal adhesion.

Clinical Outcomes, Structure, and Function Improve With Both Heavy and Moderate Loads in the Treatment of Patellar Tendinopathy: A Randomized Clinical Trial

2021 ◽  
Vol 49 (4) ◽  
pp. 982-993
Author(s):  
Anne-Sofie Agergaard ◽  
Rene B. Svensson ◽  
Nikolaj M. Malmgaard-Clausen ◽  
Christian Couppé ◽  
Mikkel H. Hjortshoej ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Outcome ◽  
Randomized Clinical Trial ◽  
Clinical Outcomes ◽  
Rating Scale ◽  
Power Doppler ◽  
Patellar Tendinopathy ◽  
Short Term ◽  
Level Of Evidence

Background: Loading interventions have become a predominant treatment strategy for tendinopathy, and positive clinical outcomes and tendon tissue responses may depend on the exercise dose and load magnitude. Purpose/Hypothesis: The purpose was to investigate if the load magnitude influenced the effect of a 12-week loading intervention for patellar tendinopathy in the short term (12 weeks) and long term (52 weeks). We hypothesized that a greater load magnitude of 90% of 1 repetition maximum (RM) would yield a more positive clinical outcome, tendon structure, and tendon function compared with a lower load magnitude of 55% of 1 RM when the total exercise volume was kept equal in both groups. Study Design: Randomized clinical trial; Level of evidence, 1. Methods: A total of 44 adult participants with chronic patellar tendinopathy were included and randomized to undergo moderate slow resistance (MSR group; 55% of 1 RM) or heavy slow resistance (HSR group; 90% of 1 RM). Function and symptoms (Victorian Institute of Sport Assessment–Patella questionnaire [VISA-P]), tendon pain during activity (numeric rating scale [NRS]), and ultrasound findings (tendon vascularization and swelling) were assessed before the intervention, at 6 and 12 weeks during the intervention, and at 52 weeks from baseline. Tendon function (functional tests) and tendon structure (ultrasound and magnetic resonance imaging) were investigated before and after the intervention period. Results: The HSR and MSR interventions both yielded significant clinical improvements in the VISA-P score (mean ± SEM) (HSR: 0 weeks, 58.8 ± 4.3; 12 weeks, 70.5 ± 4.4; 52 weeks, 79.7 ± 4.6) (MSR: 0 weeks, 59.9 ± 2.5; 12 weeks, 72.5 ± 2.9; 52 weeks, 82.6 ± 2.5), NRS score for running, NRS score for squats, NRS score for preferred sport, single-leg decline squat, and patient satisfaction after 12 weeks, and these were maintained after 52 weeks. HSR loading was not superior to MSR loading for any of the measured clinical outcomes. Similarly, there were no differences in functional (strength and jumping ability) or structural (tendon thickness, power Doppler area, and cross-sectional area) improvements between the groups undergoing HSR and MSR loading. Conclusion: There was no superior effect of exercising with a high load magnitude (HSR) compared with a moderate load magnitude (MSR) for the clinical outcome, tendon structure, or tendon function in the treatment of patellar tendinopathy in the short term. Both HSR and MSR showed equally good, continued improvements in outcomes in the long term but did not reach normal values for healthy tendons. Registration: NCT03096067 (ClinicalTrials.gov identifier)

scholarly journals Family Burden and Coping among Caregivers of Schizophrenia

2017 ◽  
Vol 4 (4) ◽  
Author(s):  
S. Geetha ◽  
Dr. M. V. Sudhakaran
Keyword(s):  
Human Population ◽  
Statistical Significance ◽  
Subjective Burden ◽  
Short Term ◽  
Care Giving ◽  
Primary Caretakers ◽  
Schizophrenic Patients ◽  
Coping Scale ◽  
And Coping

Schizophrenia is a strict mental disorder affecting about human population. Being chronic and often incapacitating, it extracts tremendous cost from patients, caregivers and society. Caregivers of patients with schizophrenia face stress and emotional hardship and are frequently forced to assume lifelong care-taking roles. Subjective burden refers to the caregivers’ short term and long term reactions to the patient’s symptoms and behaviors, and the care giving task resulting from it. Perceived distress and interpersonal strain are examples. It refers to the extent to which the care giver feels he or she is burdened. This study aims to conducted for analysis the burden and coping among caregivers of schizophrenia. This study conducted with 30 Schizophrenic patients and 30 primary caretakers of the patients, totally 60 samples were studied. The result shows that there is association between burden assessment schedules of caregiver with that of caregivers coping scale. It revealed statistical significance. Low coping score seen in caregiver who had high burden score. Lower burden score seen in caregivers who had high coping level.

scholarly journals Long-Term Outcome of Deferasirox Chelation in Patients with Thalassemia Major

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3620-3620
Author(s):  
Sule Unal ◽  
Neslihan Kalkan ◽  
Mualla Cetin ◽  
Fatma Gumruk
Keyword(s):  
Serum Creatinine ◽  
Sample Size ◽  
Serum Ferritin ◽  
Thalassemia Major ◽  
Iron Chelator ◽  
The Other ◽  
Single Center ◽  
Other Hand

Abstract Introduction: Iron overload is one of themajor complicationsof transfusion treatment in patient with thalassemia major. Deferasirox is a once-daily orally active iron chelator and long-term efficacy and safety data are being published. Herein we report the long-term follow-up data of thalassemia major patients in a single center. Methods: Of the 67 patients with thalassemia major who were under follow-up in a single center, 42 who were on deferasirox chelation for at least three years were included in the study. Patients' initial serum ferritin, ALT, creatinine, cardiac T2* and hepatic T2* values were recorded at the time of deferasirox initiation and at last visit. Deferasirox was not initiated as an iron chelator to none of the patients with a cardiac T2* value below 8 ms. All of the patients had creatinine clearance above 40 ml/minute and had serum creatinine levels within age appropriate normals at deferasirox initiation. None of the patients received any other chelations during the follow-up period. Results: Mean age of the patients were 16±9.4 years (2-33.4 years) at initiation of deferasirox and 22 (52%) were females. Eighteen (43%) of the patients were splenectomized. Median follow-up time of deferasirox chelation was 7.9 years (3-10). The median deferasirox doses at initiation of chelation and at last visit were 20.5 mg/kg/day and 30.7 mg/kg/day (7-40), respectively. Serum ferritin levels decreased significantly with deferasirox chelation (median 1969 ng/ml (516-5404) vs 1113 ng/ml (339-4003), p<0,001). We did not find statistically significant difference between the inital cardiac T2* values and the values at the last visit (median 25 .3 ms((8.7-42) vs 32 ms (6.6-42), p=0.607), despite a dramatic increase. On the other hand, hepatic T2* values did not significantly change compared to initial values, as well (median 3.7 ms (1-13.6) vs 3.3 (1-16), p=0.865). However of the patients who had cardiac T2* value between 10-20 ms, 67% was found to have T2* value above 20 ms by the end of the follow-up duration. On the other hand 53% of the patients with hepatic T2* value below 3.5 ms, had T2* values above 3.5 ms by the end of the follow-up, indicating improvement in iron stores. None of the patients exibited an adverse event that requires cessation of the drug totally, but patients exibited transient hypertransaminasemia that required transient cessation and/or dose decrement. The changes in serum ALT and serum creatinine levels at the initiation and at last visit were not significant. Conclusions: This is a a study that includes patients with a relatively long duration of follow-up. Although the cardiac T2* values improved by the end of the follow-up, this change was not found statistically significant. This can be attributed to the sample size and in a larger sample size, the change might be found significant. Additionally, the patients included in the study were composed of not only naive patients to chelation but also of the patients who were imcomplant to previous chelation and who were highly iron loaded before initiation of deferasirox. Disclosures No relevant conflicts of interest to declare.

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