scholarly journals The Efficacy of Immediate Diet for Reducing Local Adverse Events of Inhaled Corticosteroid: A Pilot Study

2012 ◽  
Vol 73 (2) ◽  
pp. 93
Author(s):  
Myoung Kyu Lee ◽  
Won Yeon Lee ◽  
Suk Joong Yong ◽  
Kye Chul Shin ◽  
Chong Whan Kim ◽  
...  
Keyword(s):  
Pilot Study ◽  
Adverse Events ◽  
Inhaled Corticosteroid

Safety of Vinflunine in Patients with Advanced Urothelial Carcinoma Refractory to Platinum-based Chemotherapy: A Prospective Pilot Study

2019 ◽  
Vol 14 (1) ◽  
pp. 31-36
Author(s):  
Raafat Abdel-Malek ◽  
Kyrillus S. Shohdy ◽  
Noha Abbas ◽  
Mohamed Ismail ◽  
Emad Hamada ◽  
...  
Keyword(s):  
Pilot Study ◽  
Adverse Events ◽  
Urothelial Carcinoma ◽  
Transitional Cell ◽  
Chemotherapeutic Agents ◽  
Serious Adverse Events ◽  
Platinum Based Chemotherapy ◽  
Number Of Patients ◽  
Advanced Urothelial Carcinoma ◽  
Grade 3

Background: Several single chemotherapeutic agents have been evaluated as the second-line treatment of advanced urothelial carcinoma. Despite encouraging efficacy outcomes, toxicity has often led to dose modifications or discontinuation. We aimed to assess the safety of vinflunine in a particular population of advanced transitional cell carcinoma of urothelium (TCCU), that were exposed to the previous toxicity of chemotherapy. Methods: This is an open-label, prospective, single-center pilot study to evaluate the response rate and safety profile of vinflunine in patients with advanced TCCU. It was planned to enroll 25 evaluable patients. Eligible patients are those with progressive disease after first-line platinum-based regimen for advanced or metastatic disease. Results: The study was prematurely closed due to two sudden deaths that were judged by the review board as treatment-related. Only ten patients were evaluated and received at least one cycle of vinflunine. All but one were male and seven underwent radical surgery. Eight had a distant metastasis (mainly lung and/or liver). Disease control rate was 40%, four patients had a partial response with median duration of response of 3.5 months. The median overall survival was 3.2 months (95% CI:1.67- 4.73). There were three serious adverse events namely two sudden deaths and one grade 4 thrombocytopenia. Nine grade 3/4 adverse events occurred. The most common all-grade adverse events were fatigue (50%), constipation (40%) and vomiting (40%). Moreover, grade 3 fatigue occurred in 30% of patients. Only one patient, who achieved PR for 5 months, was fit to receive further cytotoxic chemotherapy. Conclusion: The activity of vinflunine in advanced urothelial carcinoma came at the expense of its safety. The use of vinflunine has to be limited to the selected group of patients. However, this is a single institute experience in a limited number of patients.

scholarly journals The Use of the Oxygenated AirdriveTM Machine Perfusion System in Kidney Graft Preservation: A Clinical Pilot Study

2020 ◽  
Vol 61 (6) ◽  
pp. 153-162
Author(s):  
Julia H.E. Houtzager ◽  
Sebastiaan David Hemelrijk ◽  
Ivo C.J.H. Post ◽  
Mirza M Idu ◽  
Frederike J. Bemelman ◽  
...  
Keyword(s):  
Kidney Transplantation ◽  
Pilot Study ◽  
Adverse Events ◽  
Deceased Donor ◽  
Kidney Graft ◽  
Machine Perfusion ◽  
End Points ◽  
Viable Solution ◽  
Technical Failures

Background: The shortage of donor kidneys has led to the use of marginal donors, e.g., those whose kidneys are donated after circulatory death. Preservation of the graft by hypothermic machine perfusion (HMP) provides a viable solution to reduce warm ischemic damage. This pilot study was undertaken to assess the feasibility and patient safety of the AirdriveTM HMP system in clinical kidney transplantation. Methods: Five deceased-donor kidneys were preserved using the oxygenated Airdrive HMP system between arrival at the recipient center (Amsterdam UMC) and implantation in the patient. The main study end-points were adverse effects due to the use of Airdrive HMP. Secondary end-points were clinical outcomes and perfusion parameters. All events occurring during the transplantation procedure or within 1 month of follow-up were monitored. Results: Five patients were included in this pilot study. No technical failures were observed during the preservation period using the Airdrive HMP. Mean perfusion parameters were: duration 8.5 h (3–15 h), pressure 25 mm Hg (18–25 mm Hg), flow 49.77 mL/min (19–58 mL/min), resistance 0.57 mm Hg/min/mL (0.34–1.3 mm Hg/min/mL), and temperature 8.2 °C (2–13°C). Mean cold ischemia time (CIT) was 20.2 h (11–29.5 h). No adverse events or technical failures were observed during preservation and transplantation or during the 1-month follow-up. Conclusions: This pilot study showed the feasibility of the use of the Airdrive HMP system with no adverse events in clinical kidney transplantation.

scholarly journals Paliperidone ER in the Treatment of Borderline Personality Disorder: A Pilot Study of Efficacy and Tolerability

2011 ◽  
Vol 2011 ◽  
pp. 1-7 ◽  
Author(s):  
Silvio Bellino ◽  
Paola Bozzatello ◽  
Camilla Rinaldi ◽  
Filippo Bogetto
Keyword(s):  
Borderline Personality Disorder ◽  
Pilot Study ◽  
Personality Disorder ◽  
Adverse Events ◽  
Mechanism Of Action ◽  
Severity Index ◽  
Borderline Personality ◽  
Controlled Trials ◽  
Efficacy And Safety ◽  
Dsm Iv

Antipsychotics are recommended for the treatment of impulsive dyscontrol and cognitive perceptual symptoms of borderline personality disorder (BPD). Three reports supported the efficacy of oral risperidone on BPD psychopathology. Paliperidone ER is the metabolite of risperidone with a similar mechanism of action, and its osmotic release reduces plasmatic fluctuations and antidopaminergic effects. The aim of this study is to evaluate efficacy and safety of paliperidone ER in BPD patients. 18 outpatients with a DSM-IV-TR diagnosis of BPD were treated for 12 weeks with paliperidone ER (3–6 mg/day). They were assessed at baseline, week 4, and week 12, using the CGI-Severity item, the BPRS, the HDRS, the HARS, the SOFAS, the BPD Severity Index (BPDSI), and the Barratt Impulsiveness Scale (BIS-11). Adverse events were evaluated with the DOTES. Paliperidone ER was shown to be effective and well tolerated in reducing severity of global symptomatology and specific BPD symptoms, such as impulsive dyscontrol, anger, and cognitive-perceptual disturbances. Results need to be replicated in controlled trials.

scholarly journals Percutaneous administration of allogeneic bone-forming cells for the treatment of delayed unions of fractures: a pilot study

2021 ◽  
Vol 12 (1) ◽  
Author(s):  
Marc Jayankura ◽  
Arndt Peter Schulz ◽  
Olivier Delahaut ◽  
Richard Witvrouw ◽  
Lothar Seefried ◽  
...  
Keyword(s):  
Pilot Study ◽  
Adverse Events ◽  
Phase I ◽  
Bone Fractures ◽  
Fracture Site ◽  
Post Treatment ◽  
Long Bone ◽  
Allogeneic Bone ◽  
Hla Antibodies ◽  
Long Bone Fractures

Abstract Background Overall, 5–10% of fractures result in delayed unions or non-unions, causing major disabilities and a huge socioeconomic burden. Since rescue surgery with autologous bone grafts can cause additional challenges, alternative treatment options have been developed to stimulate a deficient healing process. This study assessed the technical feasibility, safety and preliminary efficacy of local percutaneous implantation of allogeneic bone-forming cells in delayed unions of long bone fractures. Methods In this phase I/IIA open-label pilot trial, 22 adult patients with non-infected delayed unions of long bone fractures, which failed to consolidate after 3 to 7 months, received a percutaneous implantation of allogeneic bone-forming cells derived from bone marrow mesenchymal stem cells (ALLOB; Bone Therapeutics) into the fracture site (50 × 106 to 100 × 106 cells). Patients were monitored for adverse events and need for rescue surgery for 30 months. Fracture healing was monitored by Tomographic Union Score (TUS) and modified Radiographic Union Score. The health status was evaluated using the Global Disease Evaluation (GDE) score and pain at palpation using a visual analogue scale. The presence of reactive anti-human leukocyte antigen (HLA) antibodies was evaluated. Results During the 6-month follow-up, three serious treatment-emergent adverse events were reported in two patients, of which two were considered as possibly treatment-related. None of the 21 patients in the per-protocol efficacy population needed rescue surgery within 6 months, but 2/21 (9.5%) patients had rescue surgery within 30 months post-treatment. At 6 months post-treatment, an improvement of at least 2 points in TUS was reached in 76.2% of patients, the GDE score improved by a mean of 48%, and pain at palpation at the fracture site was reduced by an average of 61% compared to baseline. The proportion of blood samples containing donor-specific anti-HLA antibodies increased from 8/22 (36.4%) before treatment to 13/22 (59.1%) at 6 months post-treatment, but no treatment-mediated allogeneic immune reactions were observed. Conclusion This pilot study showed that the percutaneous implantation of allogeneic bone-forming cells was technically feasible and well tolerated in patients with delayed unions of long bone fractures. Preliminary efficacy evidence is supporting the further development of this treatment. Trial registration NCT02020590. Registered on 25 December 2013. ALLOB-DU1, A pilot Phase I/IIa, multicentre, open proof-of-concept study on the efficacy and safetyof allogeneic osteoblastic cells (ALLOB®) implantation in non-infected delayed-union fractures.

scholarly journals Pilot study in patients with congenital low-flow vascular malformation treated with low dose sirolimus

2020 ◽  
Author(s):  
Veroniek Harbers ◽  
Gerard Rongen ◽  
van der Carine Vleuten ◽  
Bas Verhoeven ◽  
de Peter Laat ◽  
...  
Keyword(s):  
Pilot Study ◽  
Adverse Events ◽  
Vascular Malformation ◽  
Low Dose ◽  
Liver Toxicity ◽  
Vascular Malformations ◽  
Low Flow ◽  
Bone Marrow Toxicity ◽  
Serious Adverse Events ◽  
Grade Iii

Abstract Background Patients with congenital low-flow vascular malformations (capillary (CM), lymphatic (LM), venous (VM) or combined) may have an impaired quality of life (QoL), due to their symptoms, which include pain, swelling, bleeding, thrombosis, and functional impairment. Unfortunately, current treatment methods are challenging and not always successful. Previous studies have shown that the mTOR-inhibitor sirolimus is an effective treatment for these patients. Target levels of 10–15 ng/ml were well tolerated; however, grade three adverse events were observed (ranged 20–40%). Methods A pilot study was performed using a Challenge–Dechallenge–Rechallenge (CDR) design to determine the pharmacodynamics of low target levels of sirolimus (target levels 4–10 ng/ml) in respect of efficacy and adverse events in patients with disabling low-flow vascular malformations without treatment alternatives. The patients received sirolimus over a three-to-six-month period (Challenge), followed by the withdrawal of sirolimus (Dechallenge). If the complaints returned, sirolimus was reintroduced during a twelve month period (Rechallenge). Efficacy was determined on pain (end point of the pilot study) and other symptoms related to the vascular malformation; and adverse events were determined in all phases of the study. Results An improvement in symptoms was seen in 92% (n = 11/12) of patients during the Challenge phase. In the Rechallenge phase, a positive response rate of 78% was found (n = 7/9). These response rates are comparable to those found in the literature despite low target levels of sirolimus. However, less serious adverse events were observed with low dose sirolimus, especially bone marrow toxicity and grade III liver toxicity. Conclusions This pilot using low dose sirolimus showed high efficacy in patients with therapy resistant and disabling low-flow malformation, with a lower incidence of serious adverse events (especially bone marrow toxicity and grade III liver toxicity). This is extremely relevant to patients with low-flow vascular malformation, as current clinical protocols tend to advise lifelong treatment. Trial registration The pilot study was part of a phase III study. Trial registration: EudraCT number: 2016-002157-38 and ClinicalTrials.gov Identifier: NCT03987152, registered 06/14/2019 - Retrospectively registered, https://clinicaltrials.gov/ct2/show/NCT03987152?term=sirolimus&cond=Vascular+Malformations&cntry=NL&draw=2&rank=1

scholarly journals A pilot study of novel duodenal covered self-expandable metal stent fixation

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Yasuki Hori ◽  
Kazuki Hayashi ◽  
Itaru Naitoh ◽  
Katsuyuki Miyabe ◽  
Makoto Natsume ◽  
...  
Keyword(s):  
Pilot Study ◽  
Adverse Events ◽  
Gastric Outlet Obstruction ◽  
Clinical Success ◽  
Performance Status ◽  
Outlet Obstruction ◽  
Poor Performance ◽  
Stent Migration ◽  
Poor Performance Status ◽  
Sems Placement

AbstractMigration of duodenal covered self-expandable metal stents (C-SEMSs) is the main cause of stent dysfunction in patients with malignant gastric outlet obstruction (mGOO). Because endoscopic SEMS placement is frequently selected in patients with poor performance status, we concurrently focused on the safety of the treatment. This pilot study included 15 consecutive patients with mGOO who underwent duodenal partially covered SEMS (PC-SEMS) placement with fixation using an over-the-scope-clip (OTSC). Technical feasibility, clinical success for oral intake estimated by the Gastric Outlet Obstruction Scoring System (GOOSS) score, and adverse events including stent migration were retrospectively assessed. All procedures were successful, and clinical success was achieved in 86.7% (13/15). Mean GOOSS scores were improved from 0.07 to 2.53 after the procedure (P < 0.001). Median survival time was 84 days, and all patients were followed up until death. Stent migration occurred in one case (6.7%) at day 17, which was successfully treated by removal of the migrated PC-SEMS using an enteroscope. For fixation using an OTSC, additional time required for the procedure was 8.9 ± 4.1 min and we did not observe OTSC-associated adverse events. Poor performance status was associated with clinical success (P = 0.03), but we could provide the treatment safely and reduce mGOO symptoms even in patients with poor performance status. In conclusion, duodenal PC-SEMS fixation using an OTSC is feasible for preventing stent migration in patients with mGOO including those with poor performance status.

Sign in / Sign up

Export Citation Format

Share Document