AIM: Evaluation of efficacy and safety of ipilimumab in patients with advanced, refractory melanoma enrolled into a national ipilimumab Expanded Access Program. PATIENTS AND METHODS: Adult patients with advanced/metastatic refractory melanoma were eligible for study inclusion. Ipilimumab was administered up to a total of four doses. RESULTS: One hundred and ninety-six patients were analyzed. Full ipilimumab induction was administered to 66.8% of patients. Median overall survival (OS) in the entire cohort was 7.5 months. Median OS for patients after four doses of ipilimumab was significantly longer than for patients with fewer doses (12.3 months vs. 2.0 months respectively; p<0.001). Median OS for patients with objective tumor response was 42.3 months. Normal baseline serum lactate dehydrogenase (LDH) and C-reactive protein (CRP) levels, and the number of affected organs correlated with improved OS. CONCLUSION: The number of affected organs and combination of baseline LDH and CRP levels could potentially serve as predictors for both treatment response and OS.
- MeSH
- Safety MeSH
- C-Reactive Protein metabolism MeSH
- Time Factors MeSH
- Drug Resistance, Neoplasm drug effects MeSH
- Adult MeSH
- L-Lactate Dehydrogenase metabolism MeSH
- Middle Aged MeSH
- Humans MeSH
- Neoplasm Recurrence, Local drug therapy mortality pathology MeSH
- Melanoma drug therapy mortality pathology MeSH
- Neoplasm Metastasis MeSH
- Survival Rate MeSH
- Young Adult MeSH
- Antibodies, Monoclonal therapeutic use MeSH
- Follow-Up Studies MeSH
- Prognosis MeSH
- Antineoplastic Agents therapeutic use MeSH
- Aged, 80 and over MeSH
- Aged MeSH
- Neoplasm Staging MeSH
- Salvage Therapy * MeSH
- Check Tag
- Adult MeSH
- Middle Aged MeSH
- Humans MeSH
- Young Adult MeSH
- Male MeSH
- Aged, 80 and over MeSH
- Aged MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Research Support, Non-U.S. Gov't MeSH
BACKGROUND: Everolimus, a mammalian target of rapamycin (mTOR) inhibitor, has been shown to be effective and safe in the treatment of subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC). The Everolimus For Fast Expanded aCcess in TSC SEGA (EFFECTS) study was designed to provide everolimus access to patients with SEGA associated with TSC and to mainly assess the safety and also efficacy of everolimus in a real-world setting. METHODS: EFFECTS was a phase 3b, open-label, noncomparative, multicenter, expanded access study. Eligible patients were ≥ 3 years of age, with a definite diagnosis of TSC, and with at least one SEGA lesion identified by MRI or CT scan. Patients received once daily everolimus (dose adjusted to attain a trough level of 5-15 ng/mL). Safety evaluation was the primary objective and included collection of adverse events (AEs) and serious AEs, with their severity and relationship to everolimus. Efficacy evaluation, which was the secondary objective, was based on the best overall response as per medical judgment. RESULTS: Of the 120 patients enrolled, 100 (83.3%) completed the study. Median age of patients was 11 years (range, 1-47). Median daily dose of everolimus was 5.82 mg (range, 2.0-11.8). Median duration of exposure was 56.5 weeks (range, 0.3-130). The overall incidence of AEs was 74.2%. Aphthous stomatitis (18 [15.0%]), pyrexia (18 [15.0%]), bronchitis (11 [9.2%]), and stomatitis (10 [8.3%]) were the most common AEs reported. Overall, 25 patients had grade 3 AEs; most frequent was stomatitis (4 [3.3%]). Grade 4 AEs were reported in three (2.5%) patients. A total of 62 (51.7%) patients had suspected drug-related AEs, of which 15 (12.5%) were of grade 3 or 4. In eight (6.7%) patients, AEs led to drug discontinuation. With regard to efficacy, 81 (67.5%) patients had a partial response, 35 (29.2%) had a stable disease, and one (0.8%) had progressive disease. The response was unknown in three (2.5%) patients. CONCLUSION: This study confirms the acceptable safety profile of everolimus in patients with SEGA associated with TSC in a real-world setting. The results further support the efficacy of everolimus in the treatment of SEGA associated with TSC. (EudraCT: 2010-022583-13).
- MeSH
- Stomatitis, Aphthous chemically induced MeSH
- Astrocytoma drug therapy MeSH
- Safety MeSH
- Bronchitis chemically induced MeSH
- Child MeSH
- Adult MeSH
- Everolimus adverse effects therapeutic use MeSH
- Fever chemically induced MeSH
- Remission Induction MeSH
- Infant MeSH
- Middle Aged MeSH
- Humans MeSH
- Adolescent MeSH
- Young Adult MeSH
- Brain Neoplasms drug therapy MeSH
- Child, Preschool MeSH
- Disease Progression MeSH
- Antineoplastic Agents adverse effects therapeutic use MeSH
- Stomatitis chemically induced MeSH
- Tuberous Sclerosis drug therapy MeSH
- Treatment Outcome MeSH
- Check Tag
- Child MeSH
- Adult MeSH
- Infant MeSH
- Middle Aged MeSH
- Humans MeSH
- Adolescent MeSH
- Young Adult MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial, Phase III MeSH
- Multicenter Study MeSH
- Research Support, Non-U.S. Gov't MeSH
BACKGROUND: Gastrointestinal stromal tumors (GISTs) are mesenchymal tumors most often caused by activating mutations of the KIT gene. KIT tyrosine kinase inhibitors provide targeted therapy for the underlying genetic mutation, and adjuvant therapy is indicated for patients who are at significant risk of relapse following GIST resection. This is a report of the safety of imatinib in patients with GIST in the adjuvant setting in an expanded access program. METHODS: In this multicenter, open-label, single-arm trial, safety was assessed based on the frequency of adverse events (AEs). RESULTS: Three hundred patients were treated and analyzed; 40 patients discontinued treatment. Median overall exposure during the program was 181 days (range 9-420); most patients (260/300 treated) completed the study. Six patients had disease recurrence, 4 of whom discontinued. In line with previously published reports, the most frequent AEs were nausea, diarrhea, and periorbital edema. The AEs were mild to moderate in most cases (76%). CONCLUSIONS: These findings are in agreement with the known safety profile of imatinib and confirm the safety of imatinib at 400 mg/day in the adjuvant setting. The incidence of severe AEs was low.
- MeSH
- Chemotherapy, Adjuvant MeSH
- Survival Analysis MeSH
- Adult MeSH
- Gastrointestinal Neoplasms drug therapy surgery MeSH
- Gastrointestinal Stromal Tumors drug therapy surgery MeSH
- Imatinib Mesylate administration & dosage adverse effects MeSH
- Protein Kinase Inhibitors administration & dosage adverse effects MeSH
- Middle Aged MeSH
- Humans MeSH
- Young Adult MeSH
- Aged, 80 and over MeSH
- Aged MeSH
- Treatment Outcome MeSH
- Check Tag
- Adult MeSH
- Middle Aged MeSH
- Humans MeSH
- Young Adult MeSH
- Male MeSH
- Aged, 80 and over MeSH
- Aged MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial MeSH
- Multicenter Study MeSH
In this position article, DESCARTES (Developing Education Science and Care for Renal Transplantation in European States) board members describe the current strategies aimed at expanding living and deceased donor kidney pools. The article focuses on the recent progress in desensitization and kidney paired exchange programmes and on the expanded criteria for the use of donor kidneys and organs from donors after circulatory death. It also highlights differences in policies and practices across different regions with special regard to European Union countries. Living donor kidney paired exchange, the deceased donor Acceptable Mismatch Programme and kidneys from donors after circulatory death are probably the most promising innovations for expanding kidney transplantation in Europe over the coming decade. To maximize success, an effort is needed to standardize transplant strategies, policies and legislation across European countries.
American journal of cardiology, ISSN 0002-9149 vol. 94, no. 9, suppl., November 2004
48 s. : tab., grafy ; 28 cm
- MeSH
- Angiostatins metabolism pharmacology MeSH
- Pharmaceutical Services organization & administration MeSH
- Hypercholesterolemia epidemiology prevention & control MeSH
- Cardiovascular Diseases epidemiology prevention & control MeSH
- Nonprescription Drugs MeSH
- Primary Prevention trends MeSH
- Managed Care Programs MeSH
- Publication type
- Collected Work MeSH
- Conspectus
- Patologie. Klinická medicína
- NML Fields
- angiologie
- kardiologie
- vnitřní lékařství
SUMMARY: The New E-Resource for Drug Discovery (NERDD) is a quickly expanding web portal focused on the provision of peer-reviewed in silico tools for drug discovery. NERDD currently hosts tools for predicting the sites of metabolism (FAME) and metabolites (GLORY) of small organic molecules, for flagging compounds that are likely to interfere with biological assays (Hit Dexter), and for identifying natural products and natural product derivatives in large compound collections (NP-Scout). Several additional models and components are currently in development. AVAILABILITY AND IMPLEMENTATION: The NERDD web server is available at https://nerdd.zbh.uni-hamburg.de. Most tools are also available as software packages for local installation.
INTRODUCTION: Limited access to healthcare services leads to lower vaccination rates in marginalized Roma communities (MRCs). This study aimed to explore health system barriers to HPV vaccination faced by people from MRCs from multiple perspectives. METHODS: The qualitative study was conducted in Slovakia in 2021/22 with 43 community members and health professionals. Data were analyzed using a combination of content analysis and consensual qualitative research. RESULTS: A substantial barrier to vaccination is limited coverage of vaccination expenses for certain age categories by health insurance. Moreover, Slovakia faces a significant shortage of healthcare personnel, leading to work overload and a lack of capacity and motivation to address HPV vaccination. Impaired relationships between health care providers and people from MRCs lead to the avoidance of healthcare services, which contributes to insufficient delivery of information and a lack of awareness regarding HPV-related diseases and vaccination. CONCLUSION: Strengthening the capacities of health care providers, expanding the age group covered by health insurance and providing tailored information to people from MRCs are necessary prerequisites to increase the availability of HPV vaccination and enable people to make informed decisions about HPV vaccination.
- MeSH
- Health Services Accessibility * MeSH
- Papillomavirus Infections * prevention & control MeSH
- Humans MeSH
- Roma * MeSH
- Vaccination MeSH
- Papillomavirus Vaccines * administration & dosage MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Geographicals
- Slovakia MeSH
BACKGROUND: Remote care and telehealth have the potential to expand healthcare access, and the COVID-19 pandemic has called for alternative solutions to conventional face-to-face follow-up and monitoring. However, guidance is needed on the integration of telehealth into clinical care of people with rheumatic and musculoskeletal diseases (RMD). OBJECTIVE: To develop EULAR points to consider (PtC) for the development, prioritisation and implementation of telehealth for people with RMD. METHODS: A multidisciplinary EULAR task force (TF) of 30 members from 14 European countries was established, and the EULAR standardised operating procedures for development of PtC were followed. A systematic literature review was conducted to support the TF in formulating the PtC. The level of agreement among the TF was established by anonymous online voting. RESULTS: Four overarching principles and nine PtC were formulated. The use of telehealth should be tailored to patient's needs and preferences. The healthcare team should have adequate equipment and training and have telecommunication skills. Telehealth can be used in screening for RMD as preassessment in the referral process, for disease monitoring and regulation of medication dosages and in some non-pharmacological interventions. People with RMD should be offered training in using telehealth, and barriers should be resolved whenever possible.The level of agreement to each statement ranged from 8.5 to 9.8/10. CONCLUSION: The PtC have identified areas where telehealth could improve quality of care and increase healthcare access. Knowing about drivers and barriers of telehealth is a prerequisite to successfully establish remote care approaches in rheumatologic clinical practice.
... dying from AIDS-related causes 18 -- 2.1.3 but the trends vary by region 18 -- 2.1.4 As treatment expands ... ... up treatment: the Treatment 2.0 initiative 90 -- 5.2.1 Optimize drug regimens 91 -- 5.2.2 Provide access ... ... communities 96 -- 5.3 Antiretroviral therapy 96 -- 5.3.1 Global, regional and country progress in access ... ... to antiretroviral therapy 96 -- 5.3.2 Access to antiretroviral therapy among women and children 102 ... ... Conclusions: achieving and sustaining Universal Access 171 -- A time of opportunities 171 -- Innovation ...
viii, 224 stran : ilustrace, tabulky ; 30 cm
- MeSH
- Global Health MeSH
- Epidemics prevention & control MeSH
- HIV Infections prevention & control MeSH
- Communicable Disease Control MeSH
- National Health Programs MeSH
- Publication type
- News MeSH
- Conspectus
- Veřejné zdraví a hygiena
- NML Fields
- veřejné zdravotnictví
- infekční lékařství
- NML Publication type
- publikace WHO
