scholarly journals Efficacy of Thalidomide Treatment in Children With Transfusion Dependent β-Thalassemia: A Retrospective Clinical Study

2021 ◽  
Vol 12 ◽  
Author(s):  
Xinyu Li ◽  
Shuting Hu ◽  
Yong Liu ◽  
Junjiu Huang ◽  
Weicong Hong ◽  
...  
Keyword(s):  
Evaluation Index ◽  
Safety And Efficacy ◽  
The Third ◽  
Retrospective Clinical Study ◽  
Transfusion Volume ◽  
Treatment Volume ◽  
Promising Treatment ◽  
Pre Treatment ◽  
Thalidomide Treatment ◽  
Youden’S Index

Background: Thalidomide has been reported as a promising treatment for reducing transfusion volume in adults with β-thalassemia. However, the evidence about the safety and efficacy of thalidomide on children with transfusion dependent β-thalassemia (TDT) is scarce.Methods: Seventy-seven children with TDT treated with thalidomide at least for 6 months were included and retrospectively analyzed. Oral dose was started at 2.5 mg·kg-1·d-1. Blood volume for maintenance of hemoglobin above 90 g·L-1 compared with pre-treatment volume is the evaluation index for response.Results: After the sixth month treatment, 51/77 (66.2%) maintained Hb over 90 g·L-1 without transfusion. Adverse events were reported in 48 (63.2%) patients. Age, sex, genotype category, dosage, and transfusion interval before thalidomide treatment were not correlated to treatment response. The AUC was 0.806 for the HbF at the third month of treatment in predicting probability of major responders at the sixth month treatment. Based on Youden’s index algorithm in the ROC curve, 47.298 g·L-1 was the optimal cut-off value of the HbF at the third month of treatment in predicting major responders at the sixth month treatment, with sensitivity of 67.5% and specificity of 93.3%.Conclusions: The dose of thalidomide between 2.5 mg·kg-1·d-1 and 3.6 mg·kg-1·d-1 is effective in TDT children. Severe side effects are uncommon. HbF concentration of 47.298 g·L-1 at the third month is recommended as the predictor for further major responders.

scholarly journals Efficacy of thalidomide treatment in children with transfusion dependent β-thalassemia: a retrospective clinical study

2021 ◽  
Author(s):  
Xin-Yu Li ◽  
Shu-Ting Hu ◽  
Yong Liu ◽  
Jun-Jiu Huang ◽  
Wei-Cong Hong ◽  
...  
Keyword(s):  
Evaluation Index ◽  
Safety And Efficacy ◽  
The Third ◽  
Retrospective Clinical Study ◽  
Transfusion Volume ◽  
Treatment Volume ◽  
Promising Treatment ◽  
Pre Treatment ◽  
Thalidomide Treatment ◽  
Youden’S Index

Background Thalidomide has been reported as a promising treatment for reducing transfusion volume in adults with β-thalassemia. However, the evidence about the safety and efficacy of thalidomide on children with transfusion dependent β-thalassemia (TDT) is scarce. Methods Seventy-seven children with TDT treated with thalidomide at least for 6 months were included and retrospectively analyzed. Oral dose was started at 2.5 mg•kg-1•d-1 Blood volume for maintenance of hemoglobin above 90 g•L-1 compared with pre-treatment volume is the evaluation index for response. Results After the sixth month treatment, 51/77 (66.2%) maintained Hb over 90 g•L-1 without transfusion. Adverse events were reported in 48 (63.2%) patients.Age, sex, genotype category, dosage and transfusion interval before thalidomide treatment were not correlated to treatment response. The AUC was 0.806 for the HbFat the third month of treatment in predicting probability of major responders at the sixth month treatment. Based on Youden’s index algorithm in the ROC curve, 47.298 g•L-1 was the optimal cut-off value of the HbFat the third month of treatment in predicting major responders at the sixth month treatment, with sensitivity of 67.5%, specificity of 93.3%. Conclusions The dose of thalidomide between 2.5 mg•kg-1•d-1to 3.6 mg•kg-1•d-1 is effective in TDT children. Severe side effects are uncommon. HbF concentration of 47.298 g•L-1 at the third month is recommended as the predictor for further major responders.

scholarly journals The role of trans obturator tape as a surgical procedure for female stress urinary incontinence and its impact on quality of life in Jammu region

2019 ◽  
Vol 8 (12) ◽  
pp. 4975
Author(s):  
Sapna Puri ◽  
Manisha Kohli
Keyword(s):  
Quality Of Life ◽  
Stress Incontinence ◽  
Symptomatic Relief ◽  
Female Stress Urinary Incontinence ◽  
Medical Sciences ◽  
King’S Health Questionnaire ◽  
Promising Treatment ◽  
Pre Treatment

Background: Stress incontinence is one of the most common but debilitating health issue among women. It has a detrimental effect on overall health and quality of life of women. Trans obturator tape (TOT) has emerged as a promising treatment modality. The aim of present study was to assess the usefulness of TOT in terms of change in quality of life of stress incontinence patients.Methods: The study was performed at Department of Obstetrics and Gynecology, Acharya Shri Chandler of Medical Sciences (ASCOMS), Jammu. A total of 50 symptomatic women were enrolled in the study and underwent TOT procedure using outside-in technique. The quality of life of women was assessed at enrolment and 12 months after the procedure using King’s Health Questionnaire (KHQ). Change in QOL was assessed using paired ‘t’-test.Results: After 12 months follow-up, a total of 27 (54%) patients were entirely symptom free. As compared to pre-treatment QOL assessment on KHQ for general health/incontinence impact, quality of life and symptom scores a % decline of 73.2%, 79.8% and 78.4% was observed. For all the three parts, the change in scores was significant statistically. No other complications and side effects were reported.Conclusions: TOT is a useful procedure which provided symptomatic relief as well as QOL enhancement.

scholarly journals Pegvisomant and Pasireotide LAR as second line therapy in acromegaly: clinical effectiveness and predictors of response

2021 ◽  
Vol 184 (2) ◽  
pp. 217-229
Author(s):  
Sabrina Chiloiro ◽  
Antonella Giampietro ◽  
Federica Mirra ◽  
Federico Donfrancesco ◽  
Tommaso Tartaglione ◽  
...  
Keyword(s):  
First Generation ◽  
Third Ventricle ◽  
Free Time ◽  
Second Line ◽  
Tumor Extension ◽  
The Third ◽  
Clinical Biomarkers ◽  
Predictors Of Response ◽  
Igf I ◽  
Pre Treatment

Background The treatment of acromegaly resistant to first-generation somatostatin receptor ligands (SRLs) is often difficult. Pegvisomant and Pasireotide LAR are mostly used in these subset of patients, as second line therapies. Choice of the type of second line therapies is difficult, since predictors of response are still unclear, impairing personalized therapy. We aimed to investigate predictors of response to Pegvisomant and Pasireotide LAR. Methods Seventy-four acromegaly patients entered this observational, cross-sectional and retrospective study if (i) resistant to high dose first-generation SRLs and (ii) treated with Pegvisomant and Pasireotide LAR for at least 12 consecutive months. Patients treated with radiotherapy in the previous 10 years were excluded. Results Fourty-one patients were treated with Pegvisomant and 33 with Pasireotide LAR. At the end of the study, acromegaly was controlled in 35 patients treated with Pegvisomant (85.4%) and in 23 treated with Pasireotide LAR (69.7%). In this cohort, a poor Pegvisomant response and a shorter progression free time were observed in cases with tumor extension to the third ventricle (P = 0.004, HR: 1.6, 95%CI: 1.2–4.6), with a Ki67-Li >4% (P = 0.004, HR: 3.49, 95%CI: 1.4–4.0) and with pre-treatment IGF-I >3.3×ULN (P=0.03, HR: 1.3, 95%CI: 1.1–6.0). A poor Pasireotide LAR response and a shorter progression free time were observed in cases with tumor extension to the third ventricle (P=0.025, HR: 1.6 95%CI: 1.4–3.4), pre-treatment IGF-I >2.3×ULN (P=0.049, HR: 2.4, 95%CI: 1.4–8.0), absent/low SST5 membranous expression (P=0.023 HR: 4.56 95%CI: 1.3–6.4) and in patients carried the d3-delated GHR isoform (P=0.005, HR: 11.37, 95%CI: 1.3–20.0). Conclusion Molecular and clinical biomarkers can be useful in predicting the responsiveness to Pegvisomant and Pasireotide LAR.

scholarly journals A179 CANADIAN INVOLVEMENT IN THE EVALUATION OF NOVEL THERAPY FOR DIABETIC GASTROPARESIS: OVERVIEW OF PLEDGE TRIALS

2020 ◽  
Vol 3 (Supplement_1) ◽  
pp. 45-47
Author(s):  
L W Liu ◽  
M Syrzycka ◽  
P Janiszewski ◽  
L Kemps ◽  
B Degeronimo
Keyword(s):  
Diabetic Gastroparesis ◽  
Extension Study ◽  
Open Label ◽  
Double Blind ◽  
Open Label Study ◽  
Label Study ◽  
Safety And Efficacy ◽  
Double Blind Placebo ◽  
The Third ◽  
Parallel Group

Abstract Background Diabetic gastroparesis(DG) is a serious, chronic complication of type 1 or 2 diabetes mellitus(DM) presenting with a delay in gastric emptying(GE). An estimated 3 million Canadians have been diagnosed with DM; up to 5% of these patients may develop DG. DG can result in poor glycemic control, recurrent nausea and vomiting, often resulting in hospitalization. To date, no effective treatments are available. A phase 2 study showed that relamorelin (RLM), a synthetic ghrelin agonist, was safe and effective in treating DG. Investigators across Canada are participating in a set of phase 3 international trials of RLM in the treatment of DG. Aims To report the Canadian involvement in the international effort to evaluate the safety and efficacy of RLM in the treatment of DG. PLEDGE is a set of 5 trials: two identical 12-week studies, a 46-week extension study, a 52-week exposure study, and an open-label extension study. Collectively, the data from these studies will help to evaluate the safety and efficacy of RLM, a novel treatment for Canadian patients living with DG. Methods Four global, multicenter, randomized, double-blind, placebo-controlled, parallel-group studies compare the efficacy of RLM with placebo in participants with DG using composite endpoints of nausea, abdominal pain, postprandial fullness, bloating. Participants are randomized to RLM 10μg or placebo subcutaneously (SC) twice daily groups. The open-label continuation of treatment will follow participants until RLM becomes commercially available to provide long-term safety information to support the safe use of RLM as a chronic treatment of DG. As seen in Figure 1, participants from the two 12-week studies will rollover into the third study that will continue for 46 weeks. The fourth study will enroll participants that were not randomized in the first two studies because their symptoms were less severe and will also accept new participants. Participants will be randomized 2:1 to RLM 10μg or placebo SC twice daily groups. Participants from the third and fourth studies have the option to enroll in the open-label study. Results Target enrollment is approx. 1800 participants for the 4 global, multicenter, randomized, double-blind, placebo-controlled, parallel-group studies and 1000 participants for the open label study. 700 sites are expected to participate globally; 15 Canadian sites in 6 provinces are participating. Conclusions Canadian centers are actively involved in the PLEDGE trials to help determine the efficacy and safety of RLM, a potential new treatment for DG. This publication increases awareness of the Canadian gastroenterology community, providing an option to refer interested patients to PLEDGE study centers. PLEDGE Studies (NCT03285308, NCT03426345, NCT03420781, NCT03383146, NCT03786380): Placebo-controlled, randomized RLM-MD-01/02/03/04 and open-label study 3071-305-020 to study the safety and efficacy of relamorelin for the treatment of diabetic gastroparesis Funding Agencies None

Safety and efficacy of a pre-treatment antiplatelet regimen of unruptured intracranial aneurysms: a single-center experience

2020 ◽  
Vol 62 (8) ◽  
pp. 1029-1041
Author(s):  
Anthony Peret ◽  
Benjamin Mine ◽  
Thomas Bonnet ◽  
Noémie Ligot ◽  
Jason Bouziotis ◽  
...  
Keyword(s):  
Intracranial Aneurysms ◽  
Single Center ◽  
Safety And Efficacy ◽  
Single Center Experience ◽  
Unruptured Intracranial Aneurysms ◽  
Pre Treatment

scholarly journals Size of cervical lesion in locally advanced carcinoma cervix and response to neoadjuvant chemotherapy

2019 ◽  
Vol 8 (11) ◽  
pp. 4254
Author(s):  
Neetu Ahirwar
Keyword(s):  
Neoadjuvant Chemotherapy ◽  
Tumour Size ◽  
Locally Advanced ◽  
Vascular Supply ◽  
Cervical Lesion ◽  
Carcinoma Cervix ◽  
Advanced Carcinoma ◽  
Response To Chemotherapy ◽  
Treatment Volume ◽  
Pre Treatment

Background: Recently neoadjuvant chemotherapy has started being considered for advanced stage of carcinoma cervix. Drug delivery to pelvic tumour is optimal with neoadjuvant chemotherapy since tumour vascular supply has not been damaged by any previous pelvic interference. Tumor size and parametrial involvement have been reported to be important predictor of NACT response. Objective of this study was to find out association between size of cervical lesion in locally advanced carcinoma cervix and response to neoadjuvant chemotherapy.Methods: The present prospective cohort study was carried out in the Department of Obstetrics and Gynaecology with the collaboration of Department of Radiotherapy, Chhatrapati Shahuji Maharaj Medical University Lucknow for a period of 1-year august 2010 to august 2011. 26 patients with histologically proven locally advanced carcinoma cervix were studied. In all cases Cisplatin 75 mg/m2 and paclitaxel 135 mg/m2 on day one was given at 14 days interval up to maximum of three courses. Evaluation of operability status was done two weeks after second course of chemotherapy. Those found operable were taken up for radical hysterectomy and rest were given 3rd course of chemotherapy. After two weeks of 3rd course again operability assessment was done and patient was taken up either for surgery or radiotherapy.Results: It was observed that out of 14 patients who had tumour size <4 cm, 9 (64.2%) responded completely (CR), 2 (14.2%) responded partially and 3 (21.4%) responded as SD while in 12 patients with tumour size >4 cm, 4 (33.3%) responded completely (CR) and rest 8 (66.6%) response was partial (PR).Conclusions: Response to chemotherapy was modified by pre-treatment volume of the tumour.

scholarly journals 1590. Updated Aminoglycoside (AG) MIC Breakpoints (BP) to Minimize Adverse Events and Improve Outcome: Impact on Susceptibility (S) Rates

2019 ◽  
Vol 6 (Supplement_2) ◽  
pp. S580-S580
Author(s):  
Michael A Pfaller ◽  
Robert K Flamm ◽  
Paul G Ambrose ◽  
David Andes ◽  
John S Bradley ◽  
...  
Keyword(s):  
The United States ◽  
Drug Combinations ◽  
In Vitro Susceptibility ◽  
Safety And Efficacy ◽  
Improve Outcome ◽  
The Third ◽  
Carbapenem Resistant ◽  
Vestibular Toxicity ◽  
The Impact

Abstract Background In 2016 USCAST, the National Advisory Committee (NAC) for the United States (US) to EUCAST, undertook the re-evaluation of the in vitro susceptibility (AST) test interpretive criteria (IC) for gentamicin (GM), tobramycin (TO) and amikacin (AK) against Enterobacteriaceae (ENT), P. aeruginosa (PSA) and S. aureus (SA) based on an analysis of contemporary microbiology and PK/PD data. In 2019 USCAST posted the third version (www.uscast.org) of AG IC document and CLSI and EUCAST has published AG IC in CLSI M100-S29 and EUCAST v 9.0 documents. USCAST ICs for S were generally lower than those proposed by CLSI for all organism/drug combinations. PK/PD emphasized high, extended interval dosing (5 renal function groups) to reduce nephro-vestibular toxicity and a stasis exposure endpoint. Here, we evaluate the impact on S rates for US AST data that these IC changes created. Methods Clinical isolates from 2010 to 2018 US SENTRY Program (reference broth microdilution AST) were analyzed for S based on current and previous IC values. AG results for GM, TO and AK were evaluated against 66,280 ENT, 13,959 PSA and 51,950 SA. Benchmark S data for meropenem, cefepime, piperacillin–tazobactam and new AG, plazomicin (PZM) were included as well as ESBL and carbapenem-resistant ENT (CRE; 805 isolates). Results S rates for ENT as determined by USCAST IC were reduced by 4.2/1.2/3.1% for AK/GM/TO (CLSI) and by 3.3% for AK (EUCAST); no S rate difference for GM and TO as determined by USCAST/EUCAST. For PSA, S decreased by 46.8/6.2% for AK/TO (EUCAST) and 51.6/6.2% (CLSI). S for SA vs. GM declined by only 0.2% (CLSI). No AG IC could be calculated/offered for Acinetobacter or GM X PSA or AM/TO X SA. Best S overall coverage X ESBL (99.2%) or CRE (97.2%) isolates was by PZM. Conclusion USCAST IC updates for AG lead to reduced values for some organism/drug combinations among ENT and PSA compared with those proposed elsewhere. The USCAST-recommended ICs were based on achieving AUC/MIC ratio target associated with net bacterial stasis. Given the assumption of AG combination therapy, stasis was considered a reasonable endpoint when evaluating AG ICs to improve both safety and efficacy. Some organism X drug exposures could not be calculated and lower IC for pneumonia isolates (GM, TO) was recommended. Disclosures All authors: No reported disclosures.

scholarly journals Antiretroviral Therapy with a Twice-Daily Regimen Containing 400 Milligrams of Indinavir and 100 Milligrams of Ritonavir in Human Immunodeficiency Virus Type 1-Infected Women during Pregnancy

2008 ◽  
Vol 52 (4) ◽  
pp. 1542-1544 ◽  
Author(s):  
Jade Ghosn ◽  
Ines De Montgolfier ◽  
Chantal Cornélie ◽  
Stéphanie Dominguez ◽  
Claire Pérot ◽  
...  
Keyword(s):  
Human Immunodeficiency Virus ◽  
Daily Regimen ◽  
Child Transmission ◽  
Third Trimester ◽  
Safety And Efficacy ◽  
Hiv Rna ◽  
The Third ◽  
Immunodeficiency Virus ◽  
Mother To Child

ABSTRACT We evaluated the safety and efficacy of a twice daily regimen containing 400 mg of indinavir and 100 mg of ritonavir in 32 human immunodeficiency virus (HIV)-infected women during pregnancy. The median indinavir trough concentration was 208 ng/ml during the third trimester. At delivery, 26 of 28 women on indinavir-ritonavir had HIV RNA levels of <200 copies/ml. No infant was HIV infected. These data are encouraging for the use of this combination for prevention of mother-to-child transmission of HIV.

Recent Studies on Coating of some Magnesium Alloys; Anodizing, Electroless Coating and Hot Press Cladding

2012 ◽  
Vol 533 ◽  
pp. 167-181
Author(s):  
Nahed El Mahallawy ◽  
Mohamed Harhash
Keyword(s):  
Corrosion Resistance ◽  
Magnesium Alloys ◽  
Deposition Time ◽  
Anodic Film ◽  
Hot Press ◽  
Bond Quality ◽  
The Third ◽  
Environmental Friendly ◽  
Pre Treatment ◽  
Electroless Ni

In the present study, coating of some magnesium alloys including AZ31 and AZ91 were studied using different techniques namely anodizing, electroless and hot press cladding. AZ91 alloy was coated using anodizing process using three types of environmental friendly electrolytes; the first based on sodium silicate, the second based on sodium hydroxide-boric acid-borax and the third on sodium silicate-potassium hydroxide-sodium carbonate-sodium tetra borate. Characterization of the anodizing layer was achieved by determination of surface morphology, microstructure, phase analysis, coat thickness, adhesion and corrosion resistance. It was found that the anodic film thickness increases with increasing the current density, anodizing voltage and deposition time until the deposition stops due to the formation of a thick anodic film. The range of the anodic film thickness is 28 42 µm.Optimization of the anodizing conditions - current density and deposition time was determined for each electrolyte. A corrosion efficiency ranging from 94% to 97% was reached; the highest value corresponding to the third electrolyte. Another study is the electroless Ni plating technique with zinc pre-treatment applied on several magnesium alloys and the effect of pre-treatment and post heat treatment on the coat characteristics. The surface morphology, surface roughness, thickness of the layer, EDX analysis, adhesion, hardness and corrosion resistance are covered in this part. The electroless layer thickness is about 6 µm. The results showed good bond quality of the coat maintaining good corrosion behaviour of electroless Ni-P based on potentiodynamic polarization tests in chloride solution where it was improved after heat treatment process. On the other hand, AZ31 was covered by a commercial pure aluminum sheet by hot pressing. The influence of the applied pressure, holding time and temperature on the bond characteristics was studied. The experimental investigation has revealed a good bond quality due to the effective mutual diffusion of Mg and Al. The phase analysis resulted in the formation of two equilibrium phases namely; Mg17Al12and Mg2Al3. The corrosion resistance of AZ31 is enhanced as a results of this process by 98.6%. Further points will be covered.Keywords: Magnesium alloys; Coating; Anodizing; Electroless; Hot press cladding; environmental friendly electrolytes; corrosion

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