Abstract:
OBJECTIVE:In May 2018, the US Food and Drug Administration approved pegvaliase-pqpz (Palynziq*), the first enzyme substitution therapy for the treatment of phenylketonuria (PKU). This article provides an overview of the mechanism of action, pharmacokinetic properties, clinical efficacy, and the safety and tolerability profile of pegvaliase. METHODS:Relevant information was identified through a comprehensive literature search of several databases using the keywords pegvaliase, rAvPAL-PEG, and phenylketonuria. Additional information was gathered from the pegvaliase package insert, posters presented at scientific meetings, and materials provided from the manufacturer, BioMarin. RESULTS:Pegvaliase is effective in decreasing blood phenylalanine levels, and is associated with a manageable side-effect profile. Phase III clinical trial data demonstrated that 60.7% of patients were able to achieve blood phenylalanine levels less than the guideline recommended 360 µmol/L at 24 months. Brief sub-studies also showed the improvement in inattention symptoms in patients treated with pegvaliase, compared to placebo. CONCLUSION:Pegvaliase is a promising new treatment option for adults living with PKU. Further studies are warranted to determine long-term safety and clinical efficacy in sub-populations.
journal_name
Curr Med Res Opinjournal_title
Current medical research and opinionauthors
Mahan KC,Gandhi MA,Anand Sdoi
10.1080/03007995.2018.1528215subject
Has Abstractpub_date
2019-04-01 00:00:00pages
647-651issue
4eissn
0300-7995issn
1473-4877journal_volume
35pub_type
杂志文章,评审abstract:BACKGROUND:The implementation of international pediatric asthma guidelines hinges on the distinction between intermittent and persistent phenotypes and the prescription of recommended phenotype-specific pharmacotherapy. OBJECTIVES:To ascertain key factors associated with specialist-confirmed phenotype and document phy...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1185/03007995.2012.716031
更新日期:2012-09-01 00:00:00
abstract::The most common side-effect of statins, mainly during dose titration, is liver toxicity, In these cases, sufficient control of low density lipoprotein cholesterol (LDL-C) in patients with heterozygous familial hypercholesterolaemia (HFH) becomes problematical. In patients with intolerance to resins as well, especially...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:
更新日期:2001-01-01 00:00:00
abstract::In a double-blind study of 102 patients with a mean age of 79 years, zopiclone was compared to flunitrazepam. The patients rated their sleep in a diary. There was no statistically significant difference between the relatively low dose of 5 mg zopiclone and 1 mg flunitrazepam for eleven out of the twelve variables meas...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,多中心研究,随机对照试验
doi:10.1185/03007999509110492
更新日期:1995-01-01 00:00:00
abstract::A study was carried out in 80 mild to moderately depressed patients seen in general practice to assess the relative effectiveness and tolerance of a single night-time dose compared with the usual divided daily dosage regimen of a chlordiazepoxide (5 mg)/amitriptyline (12.5 mg) combination. Patients were assigned at ra...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1185/03007998009109490
更新日期:1980-01-01 00:00:00
abstract:BACKGROUND:It has been postulated that intraocular pressure, an important glaucoma risk factor, correlates positively with arterial blood pressure (blood pressure). However, results of experimental and clinical studies are often contradictory. It is hypothesized that, in some hypertensive patients, disturbances in intr...
journal_title:Current medical research and opinion
pub_type: 杂志文章,评审
doi:10.1080/03007995.2017.1415203
更新日期:2018-03-01 00:00:00
abstract::Data from phase III clinical trials suggest that high dose chemotherapy (HDC) is currently not indicated for any stage of breast cancer. Therefore HDC should only be considered within the context of clinical trials. Furthermore, there is no significant evidence to support the routine use of taxanes in women with metas...
journal_title:Current medical research and opinion
pub_type:
doi:
更新日期:2001-01-01 00:00:00
abstract::An open, controlled study was carried out in 30 in-patients with low arterial pressure syndrome to compare the effectiveness and tolerance of dimetophrine and midodrine in restoring blood pressure to normal levels and relieving symptoms. Patients were allocated at random to receive treatment for 15 days with either 10...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1185/03007998409109590
更新日期:1984-01-01 00:00:00
abstract:OBJECTIVES:To compare the efficacy and safety of ciprofloxacin 0.3%/dexamethasone 0.1% (CIP/DEX) otic suspension with that of neomycin 0.35%/polymyxin B 10,000 IU/mL/hydrocortisone 1.0% (N/P/H) otic suspension in patients with acute otitis externa (AOE). STUDY DESIGN:Randomized, observer-masked, parallel-group, multic...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,多中心研究,随机对照试验
doi:10.1185/030079902125004312
更新日期:2004-08-01 00:00:00
abstract:OBJECTIVE:To compare acute normovolemic hemodilution versus low central venous pressure strategy versus conventional fluid management in reducing intraoperative estimated blood loss, hematocrit drop and need for blood transfusion in patients undergoing radical retropubic prostatectomy under general anesthesia. RESEARC...
journal_title:Current medical research and opinion
pub_type: 杂志文章,随机对照试验
doi:10.1185/03007995.2013.877436
更新日期:2014-05-01 00:00:00
abstract:OBJECTIVE:The purpose of this study was to evaluate the safety and clinical efficacy of alcaftadine 0.25% ophthalmic solution, a new topical anti-allergic agent for the prevention of the signs and symptoms of allergic conjunctivitis induced by conjunctival allergen challenge (CAC). STUDY DESIGN AND METHODS:This two-ar...
journal_title:Current medical research and opinion
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1185/03007995.2010.548797
更新日期:2011-03-01 00:00:00
abstract::Fifty-two patients suffering from presumed viral pharyngeal infection or tonsillitis were treated with either benzydamine or placebo oral rinse as a gargle at 3-hourly intervals in a randomized double-blind study. Patients on the active preparation experienced faster resolution of pain and dysphagia and at 7 days 88% ...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1185/03007998209112381
更新日期:1982-01-01 00:00:00
abstract:OBJECTIVE:To provide a commentary on recent studies with the new IOP-lowering fixed-combination product brinzolamide 1%/timolol 0.5%. METHODS:Medline was searched for brinzolamide/timolol fixed-combination clinical literature (up to May 18, 2010) and the current comprehensive brinzolamide/timolol literature was review...
journal_title:Current medical research and opinion
pub_type: 杂志文章,评审
doi:10.1185/03007995.2010.517718
更新日期:2010-11-01 00:00:00
abstract:OBJECTIVE:To identify determinants of non-compliance with bisphosphonates in women with postmenopausal osteoporosis. By considering the year of the introduction of weekly bisphosphonates important additional information is obtained. METHODS:New female users of daily or weekly alendronate or risedronate between 1999 an...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1185/030079908x297358
更新日期:2008-05-01 00:00:00
abstract::During the past decades several epidemiological studies have provided a 'portrait' of the potential candidate for cardiovascular disease, introducing many emerging life-style risk factors such as obesity, unhealthy diet, exposure to passive smoking, lack of exercise, psychosocial stress, and depression, as well as ele...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1185/030079902125000525
更新日期:2002-01-01 00:00:00
abstract::A double-blind, parallel-group study was carried out to compare the efficacy and tolerability of a controlled-release tablet formulation of trazodone with the standard trazodone tablet. Three hundred and forty-seven general practice patients with depressive symptoms were recruited into the trial. Patients were randoml...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,多中心研究,随机对照试验
doi:10.1185/03007999009111497
更新日期:1990-01-01 00:00:00
abstract:OBJECTIVE:There are contrasting positions concerning the benefit-risk ratio of acetaminophen use for osteoarthritis (OA)-related pain. To clarify the effectiveness of acetaminophen or acetaminophen-codeine combinations according to their regimen of use, we evaluated whether being a regular user (adherent) of these medi...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1080/03007995.2017.1308920
更新日期:2017-06-01 00:00:00
abstract:PURPOSE:To evaluate effectiveness and safety information of transdermal fentanyl (TDF) (Duragesic/Durogesic) and sustained-release oral morphine (SRM) in cancer pain (CP) and chronic non-cancer pain (NCP), a pooled analysis was conducted on datasets of published, open label, uncontrolled (no comparator group) and rando...
journal_title:Current medical research and opinion
pub_type: 杂志文章,meta分析
doi:10.1185/030079904X2114
更新日期:2004-09-01 00:00:00
abstract::A national survey of psychotherapeutic drug use was carried out in the U.S.A. in 1979. Data were obtained from a probability sample (n = 3161) of adults in the general population, using a clinically-oriented interview lasting an average of 90 minutes. The data provide estimates of the prevalence of using anxiolytic an...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1185/03007998409109542
更新日期:1984-01-01 00:00:00
abstract:OBJECTIVE:Subcutaneous immunotherapy (SCIT) for allergic rhinitis (AR) has been shown to control symptoms for up to several years following treatment discontinuation, but the effect of SCIT on healthcare costs for commercially insured patients is unknown. The objective of this study was to compare healthcare costs and ...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1080/03007995.2017.1359517
更新日期:2017-11-01 00:00:00
abstract:OBJECTIVE:We recently reported the efficacy of rofecoxib in two randomized controlled trials in chronic low back pain (CLBP). The objectives of this report are to present data regarding the onset of efficacy of rofecoxib from these trials and propose methods for reporting onset. RESEARCH DESIGN AND METHODS:Patients we...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1185/030079904125003160
更新日期:2004-05-01 00:00:00
abstract::The manifestation of pain in any individual patient may result from a variety of underlying mechanisms that also may vary from one disease state to another. Global measures of pain intensity and relief are inadequate for characterizing specific pain qualities or identifying the unique effects of pain treatments on dif...
journal_title:Current medical research and opinion
pub_type: 社论
doi:10.1185/030079904X12924
更新日期:2004-01-01 00:00:00
abstract:OBJECTIVE:Heart failure (HF) is a major health problem facing the US and studies suggest that the incidence of this condition will rise significantly over the next 10 years. Limiting the incidence or duration of HF hospitalizations would, therefore, have a major impact on healthcare costs. The purpose of this review is...
journal_title:Current medical research and opinion
pub_type: 杂志文章,评审
doi:10.1185/030079906X80233
更新日期:2006-01-01 00:00:00
abstract::A clinical evaluation was carried out in 19 patients with lichen ruber planus to assess the effectiveness of 0.05% betamethasone-17,21-dipropionate ointment in the treatment of patients whose lesions had developed resistance to prolonged treatment with 0.1% betamethasone-17-valerate ointment. The new steroid ointment ...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1185/03007997609109305
更新日期:1976-01-01 00:00:00
abstract:OBJECTIVE:Painful physical symptoms are common in patients with major depressive disorder (MDD) and can negatively affect patient outcomes. Duloxetine has demonstrated efficacy in treating MDD and other certain painful conditions; this study specifically evaluated patients with both MDD and MDD-associated pain. METHOD...
journal_title:Current medical research and opinion
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1185/03007995.2011.609540
更新日期:2011-10-01 00:00:00
abstract:OBJECTIVE:Vildagliptin is a potent and selective dipeptidyl peptidase-IV (DPP-4) inhibitor that improves glycemic control in patients with type 2 diabetes by increasing alpha and beta-cell responsiveness to glucose. This study assessed the effect of multiple doses of vildagliptin 100 mg once daily on warfarin pharmacok...
journal_title:Current medical research and opinion
pub_type: 杂志文章,随机对照试验
doi:10.1185/030079907x188008
更新日期:2007-05-01 00:00:00
abstract:OBJECTIVE:Modern immunology has been extremely successful in elucidating many features of the immune system, but not in stemming pandemics of non-infectious, immune-related disease associated with industrialized populations. These pandemics involve a broad range of allergic, autoimmune, and inflammatory diseases, poten...
journal_title:Current medical research and opinion
pub_type: 杂志文章,评审
doi:10.1185/03007995.2012.695731
更新日期:2012-07-01 00:00:00
abstract:OBJECTIVE:The CCP signature test (Prolaris) quantifies a patient's risk of disease progression and prostate cancer specific mortality using a gene-expression-based cell cycle progression (CCP) score. This study evaluated the potential clinical utility of the CCP test in a US-based clinical setting. METHODS:Urologists ...
journal_title:Current medical research and opinion
pub_type: 杂志文章
doi:10.1185/03007995.2013.873398
更新日期:2014-04-01 00:00:00
abstract::A double-blind trial was carried out in 60 elderly patients with osteoarthritis of the knee or hip to compare the efficacy and tolerability of treatment with tiaprofenic acid and indomethacin. Patients received either 600 mg tiaprofenic acid or 100 mg indomethacin daily for 3 months. The results of assessments made ev...
journal_title:Current medical research and opinion
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1185/03007998309112387
更新日期:1983-01-01 00:00:00
abstract:OBJECTIVE:Neurogenic bladder refers to morphofunctional alterations of the bladder-sphincter complex secondary to central or peripheral neurological lesions. Discal etiology can be suggested by clinical observation in patients complaining of classical lower back pain, but not excluded even without musculoskeletal pain....
journal_title:Current medical research and opinion
pub_type: 杂志文章,评审
doi:10.1185/03007995.2013.807788
更新日期:2013-08-01 00:00:00
abstract::Background: In routine clinical practice, the implantation of a drug-eluting stent (DES) versus a bare metal stent (BMS) for percutaneous coronary intervention (PCI) has been guided by criteria for appropriate use. The cost-effectiveness (CE) of adopting these guidelines, however, is not clear, and was investigated fr...
journal_title:Current medical research and opinion
pub_type: 杂志文章,多中心研究
doi:10.1080/03007995.2019.1708288
更新日期:2020-03-01 00:00:00