Read by QxMD icon Read

Compassionate use

Kasper Raus
BACKGROUND: When a new intervention or drug is developed, this has to pass through various phases of clinical testing before it achieves market approval, which can take many years. This raises an issue for drugs which could benefit terminally ill patients. These patients might set their hopes on the experimental drug but are unable to wait since they are likely to pass away before the drug is available. As a means of nevertheless getting access to experimental drug, many seriously ill and terminally ill patients are therefore very willing to participate in randomised controlled trials...
October 18, 2016: BMC Medical Ethics
Shanna Burke, Peter Maramaldi
OBJECTIVE: This study examined the complexity and myriad clinical manifestations and expressions of velocardiofacial syndrome (VCFS). It aimed to determine if VCFS invariably met the three disability criteria for the Compassionate Allowance List (CAL) program administered by the Social Security Administration (SSA). METHODS: A systematic evaluative review of the literature found in 10 academic databases was completed. Inclusion criteria of the search terms yielded 1,383 initial manuscripts...
October 12, 2016: Fetal and Pediatric Pathology
Andrzej Górski, Ryszard Międzybrodzki, Beata Weber-Dąbrowska, Wojciech Fortuna, Sławomir Letkiewicz, Paweł Rogóż, Ewa Jończyk-Matysiak, Krystyna Dąbrowska, Joanna Majewska, Jan Borysowski
Antimicrobial resistance is considered to be one of the greatest challenges of medicine and our civilization. Lack of progress in developing new anti-bacterial agents has greatly revived interest in using phage therapy to combat antibiotic-resistant infections. Although a number of clinical trials are underway and more are planned, the realistic perspective of registration of phage preparations and their entering the health market and significantly contributing to the current antimicrobial crisis is rather remote...
2016: Frontiers in Microbiology
John Coutsouvelis, Sharon Avery, Michael Dooley, Carl Kirkpatrick, Andrew Spencer
Sinusoidal obstruction syndrome, previously known as veno-occlusive disease (VOD/SOS), is a complication in patients undergoing haemopoietic stem cell transplantation (HSCT). Severe VOD/SOS, including progression to multi-organ failure, has resulted in a mortality of greater than 80%. Defibrotide's varying pharmacological actions, particularly on endothelial cells, make it is a useful agent to consider for prophylaxis and treatment of VOD/SOS. Barriers to its routine use include the high acquisition cost and the fact that neither the oral or parenteral formulations are licensed products in many countries at this time...
September 30, 2016: Cancer Treatment Reviews
Jocelyn Bessette Gorlin, Cynthia Peden McAlpine, Ann Garwick, Elizabeth Wieling
This research examined the experiences of families living with a child with severe autism. There is limited literature on the experiences of families when a child has severe autism as distinct from milder autism and includes the voices of multiple family members. Van Manen's phenomenological approach was used for data collection and analysis. This approach allowed for the use of innovative data sources, including unstructured individual and family interviews, observations, and family lifelines (a pictorial, temporal picture with comments of the families lives)...
October 6, 2016: Journal of Pediatric Nursing
D A Schmerler, A J Espay
Although currently lacking a sensitive and specific electrophysiologic battery test, functional (psychogenic) dystonia can sometimes be diagnosed with clinically definite certainty using available criteria. Certain regional phenotypes have been recognized as distinctive, such as unilateral lip and jaw deviation, laterocollis with ipsilateral shoulder elevation and contralateral shoulder depression, fixed wrist and finger flexion with relative sparing of the thumb and index fingers, and fixed foot plantar flexion and inversion...
2017: Handbook of Clinical Neurology
Pranav Loyalka, Angelo Nascimbene, Michael Schechter, Marija Petrovic, Ajay Sundara Raman, Igor D Gregoric, Biswajit Kar
INTRODUCTION: Transcatheter aortic valve implantation (TAVI) in patients with degenerated bioprosthetic aortic valve has been successfully performed as an alternative to surgery. We describe our initial experience of valve-in-valve TAVI in five patients, using new generation Edwards Sapien 3 transcatheter heart valves implanted into degenerated 19 mm bioprosthetic valves. 20-mm Edwards S3 valves were offered for compassionate use. All patients had significant aortic valve stenosis. METHODS AND RESULTS: The main vascular access was achieved and pre-closed with two Proglide closure devices in one patient and Prostar closure devices in four patients...
October 3, 2016: Catheterization and Cardiovascular Interventions
Christine Léaute-Labrèze, Olivia Boccara, Caroline Degrugillier-Chopinet, Juliette Mazereeuw-Hautier, Sorilla Prey, Geneviève Lebbé, Stéphanie Gautier, Valérie Ortis, Martine Lafon, Agnès Montagne, Alain Delarue, Jean-Jacques Voisard
BACKGROUND AND OBJECTIVES: Given the widespread use of propranolol in infantile hemangioma (IH) it was considered essential to perform a systematic review of its safety. The objectives of this review were to evaluate the safety profile of oral propranolol in the treatment of IH. METHODS: We searched Embase and Medline databases (2007-July 2014) and unpublished data from the manufacturer of Hemangiol/Hemangeol (marketed pediatric formulation of oral propranolol; Pierre Fabre Dermatologie, Lavaur, France)...
September 29, 2016: Pediatrics
Gretchen Sisson, Katrina Kimport
CONTEXT: Popular entertainment may reflect and produce-as well as potentially contest-stigma regarding abortion provision. Knowledge of how providers are portrayed on-screen is needed to improve understanding of how depictions may contribute to the stigmatization of real providers. METHODS: All abortion provision plotlines on American television from 2005 to 2014 were identified through Internet searches. Plotlines were assessed in their entirety and coded for genre, abortion provision space, provider characteristics, method and efficacy of provision, and occurrence of violence...
September 29, 2016: Perspectives on Sexual and Reproductive Health
Maebh Barry, Cathy Quinn, Carmel Bradshaw, Maria Noonan, Marie Brett, Sandra Atkinson, Christina New
OBJECTIVE: To explore the influence of the Amulet artwork and exhibition on midwifery students' perceptions of caring for parents experiencing perinatal death. DESIGN: A descriptive qualitative design involving face-to-face semi-structured interviews following institutional ethical approval. SETTING: A regional Maternity Hospital in Ireland which hosted the National Artwork and Exhibition exploring the hidden world of infant death. PARTICIPANTS: A purposive sample of six consenting post registration midwifery students who had attended the Amulet artwork and exhibition...
September 16, 2016: Nurse Education Today
Martin Sievert, Igor Zwir, Kevin M Cloninger, Nigel Lester, Sandor Rozsa, C Robert Cloninger
BACKGROUND: Multiple factors influence the decision to enter a career in medicine and choose a specialty. Previous studies have looked at personality differences in medicine but often were unable to describe the heterogeneity that exists within each specialty. Our study used a person-centered approach to characterize the complex relations between the personality profiles of resident physicians and their choice of specialty. METHODS: 169 resident physicians at a large Midwestern US training hospital completed the Temperament and Character Inventory (TCI) and the Satisfaction with Life Scale (SWLS)...
2016: PeerJ
Lucia Grumetto, Antonio Del Prete, Giovanni Ortosecco, Antonella Borrelli, Salvatore Del Prete, Aldo Mancini
Background. We report a case of bilateral posterior subcapsular cataracts (PSCs) in a 24-year-old man with an allergic conjunctivitis history caused by a long-term therapy with glucocorticoids. Case Presentation. The patient showed a visual acuity of 9/10 for both eyes. He followed a therapy with ketotifen and bilastine for four years. During the last six months before our evaluation, he was treated with chloramphenicol and betamethasone, interrupted for onset of cataracts and increased intraocular pressure...
2016: Case Reports in Ophthalmological Medicine
Jina Huh, Rebecca Marmor, Xiaoqian Jiang
BACKGROUND: Online health community (OHC) moderators help facilitate conversations and provide information to members. However, the necessity of the moderator in helping members achieve goals by providing the support they need remains unclear, with some prior research suggesting that moderation is unnecessary or even harmful for close-knit OHCs. Similarly, members' perceptions of moderator roles are underexplored. Starting January of 2013, WebMD moderators stopped working for WebMD communities...
2016: Journal of Medical Internet Research
Jean Carragher, Kevin Gormley
AIM: A discussion of the concepts of leadership and emotional intelligence in nursing and midwifery education and practice. BACKGROUND: The need for emotionally intelligent leadership in the health professions is acknowledged internationally throughout the nursing and midwifery literature. The concepts of emotional intelligence and emotional-social intelligence have emerged as important factors for effective leadership in the health care professions and require further exploration and discussion...
September 8, 2016: Journal of Advanced Nursing
Tania M Welzel, Jörg Petersen, Kerstin Herzer, Peter Ferenci, Michael Gschwantler, Heiner Wedemeyer, Thomas Berg, Ulrich Spengler, Ola Weiland, Marc van der Valk, Jürgen Rockstroh, Markus Peck-Radosavljevic, Yue Zhao, Maria Jesus Jimenez-Exposito, Stefan Zeuzem
OBJECTIVE: We assessed the effectiveness and safety of daclatasvir (DCV) plus sofosbuvir (SOF), with or without ribavirin (RBV), in a large real-world cohort, including patients with advanced liver disease. DESIGN: Adults with chronic HCV infection at high risk of decompensation or death within 12 months and with no available treatment options were treated in a European compassionate use programme. The recommended regimen was DCV 60 mg plus SOF 400 mg for 24 weeks; RBV addition or shorter duration was allowed at physicians' discretion...
September 7, 2016: Gut
Garet P Lahvis
Natural selection favors individuals to act in their own interests, implying that wild animals experience a competitive psychology. Animals in the wild also express helping behaviors, presumably at their own expense and suggestive of a more compassionate psychology. This apparent paradox can be partially explained by ultimate mechanisms that include kin selection, reciprocity, and multilevel selection, yet some theorists argue such ultimate explanations may not be sufficient and that an additional "stake in others" is necessary for altruism's evolution...
September 7, 2016: Current Topics in Behavioral Neurosciences
J Vockley, J Charrow, J Ganesh, M Eswara, G A Diaz, E McCracken, R Conway, G M Enns, J Starr, R Wang, J E Abdenur, J Sanchez-de-Toledo, D L Marsden
Long-chain fatty acid oxidation disorders (LC-FAOD) can cause cardiac hypertrophy and cardiomyopathy, often presenting in infancy, typically leading to death or heart transplant despite ongoing treatment. Previous data on triheptanoin treatment of cardiomyopathy in LC-FAOD suggested a clinical benefit on heart function during acute failure. An additional series of LC-FAOD patients with critical emergencies associated with cardiomyopathy was treated with triheptanoin under emergency treatment or compassionate use protocols...
August 27, 2016: Molecular Genetics and Metabolism
Ferdinando De Vita, Jole Ventriglia, Antonio Febbraro, Maria Maddalena Laterza, Alessio Fabozzi, Beatrice Savastano, Angelica Petrillo, Anna Diana, Guido Giordano, Teresa Troiani, Giovanni Conzo, Gennaro Galizia, Fortunato Ciardiello, Michele Orditura
BACKGROUND: Pancreatic adenocarcinoma is an aggressive disease with poor prognosis. In a randomized phase III trial, combination of Nab-paclitaxel (Nab-P) plus gemcitabine showed superior activity and efficacy in first-line treatment compared with gemcitabine alone. METHODS: Nab-P is not dispensed in Italy; however, we obtained this drug from our Ethics Committee for compassionate use. The aim of this study was to evaluate the efficacy and safety profile of this Nab-P and gemcitabine combination in a cohort of patients treated outside clinical trials...
2016: BMC Cancer
A E Martin, J A D'Agostino, M Passarella, S A Lorch
OBJECTIVE: Nurses provide parental support and education in the neonatal intensive care unit (NICU), but it is unknown if satisfaction and expectations about nursing care differ between racial groups. STUDY DESIGN: A prospective cohort was constructed of families with a premature infant presenting to primary care between 1 January 2010 and 1 January 2013 (N=249, 52% white, 42% black). Responses to questions about satisfaction with the NICU were analyzed in ATLAS...
September 1, 2016: Journal of Perinatology: Official Journal of the California Perinatal Association
Philippe Calain
The West African Ebola epidemic has set in motion a collective endeavour to conduct accelerated clinical trials, testing unproven but potentially lifesaving interventions in the course of a major public health crisis. This unprecedented effort was supported by the recommendations of an ad hoc ethics panel convened in August 2014 by the WHO. By considering why and on what conditions the exceptional circumstances of the Ebola epidemic justified the use of unproven interventions, the panel's recommendations have challenged conventional thinking about therapeutic development and clinical research ethics...
August 29, 2016: Journal of Medical Ethics
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"