Patient-centered outcomes: translating clinical efficacy into benefits on health-related quality of life.

Abstract:

BACKGROUND:Multiple sclerosis (MS) is a neurodegenerative disease associated with marked impairments in health-related quality of life (HRQoL). Although standard clinical end points such as the Expanded Disability Status Scale and annualized relapse rate remain useful in assessing MS activity and severity, these measures do not fully reflect the patient's experience of the disease. The impact of MS on employment status, social and family relationships, sexual satisfaction, pain, fatigue, enjoyment of life, vision, bladder/bowel control, cognition, and emotional well-being can be profound and may influence the patient's adherence to long-term treatment. Generic HRQoL instruments such as the Medical Outcome Survey Short Form-36 and the Functional Status Questionnaire initially were used in MS studies. However, MS-specific and hybrid instruments that possess greater sensitivity now have been developed. EXPERT CLINICAL OPINION:The effects of interferon-beta on HRQoL have been evaluated in several studies, and improvements on some dimensions of HRQoL, particularly in patients with mild disability, have been reported. Two large pivotal studies of natalizumab prospectively included HRQoL assessments as tertiary efficacy end points. The impact of natalizumab on HRQoL outcomes in patients with relapsing-remitting MS was evident at 2 years regardless of sustained disease progression or relapse status. FUTURE DIRECTIONS:Patient-centered outcomes are becoming increasingly important in evaluations of MS therapies. Therefore, inclusion of HRQoL assessments in pivotal clinical studies eventually should become standard practice. Increasing our understanding of minimally important clinical change in these measures will be an important step in helping researchers and clinicians interpret these results beyond statistical significance.

journal_name

Neurology

journal_title

Neurology

authors

Miller D,Rudick RA,Hutchinson M

doi

10.1212/WNL.0b013e3181dbb884

subject

Has Abstract

pub_date

2010-04-27 00:00:00

pages

S24-35

eissn

0028-3878

issn

1526-632X

pii

74/17_Supplement_3/S24

journal_volume

74 Suppl 3

pub_type

杂志文章
  • 11C-methionine PET for differential diagnosis of low-grade gliomas.

    abstract::Management of low-grade gliomas continues to be a challenging task, because CT and MRI do not always differentiate from nontumoral lesions. Furthermore, tumor extent and aggressiveness often remain unclear because of a lack of contrast enhancement. Previous studies indicated that large neutral amino acid tracers accum...

    journal_title:Neurology

    pub_type: 临床试验,杂志文章

    doi:10.1212/wnl.50.5.1316

    authors: Herholz K,Hölzer T,Bauer B,Schröder R,Voges J,Ernestus RI,Mendoza G,Weber-Luxenburger G,Löttgen J,Thiel A,Wienhard K,Heiss WD

    更新日期:1998-05-01 00:00:00

  • Temporal lobe central benzodiazepine binding in unilateral mesial temporal lobe epilepsy.

    abstract::PET-demonstrated decreases in [11C]flumazenil binding occur in anterior mesial temporal structures on the side of epileptogenesis in unilateral mesial temporal lobe epilepsy. We performed quantitative autoradiography on anterior mesial and lateral temporal specimens from 11 subjects with unilateral mesial temporal lob...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.45.5.934

    authors: Burdette DE,Sakurai SY,Henry TR,Ross DA,Pennell PB,Frey KA,Sackellares JC,Albin RL

    更新日期:1995-05-01 00:00:00

  • Long-term therapy with high doses of subcutaneous immunoglobulin in multifocal motor neuropathy.

    abstract:BACKGROUND:In multifocal motor neuropathy (MMN), high dose of IV immunoglobulin (IVIg) is the only established treatment. Subcutaneous self-infusion with immunoglobulin G (IgG) (SCIG) increases patient autonomy and treatment flexibility, and might be associated with fewer side effects due to lower peak IgG levels. In a...

    journal_title:Neurology

    pub_type: 临床试验,杂志文章

    doi:10.1212/WNL.0b013e3181f735ce

    authors: Harbo T,Andersen H,Jakobsen J

    更新日期:2010-10-12 00:00:00

  • Quantifying physical decline in juvenile neuronal ceroid lipofuscinosis (Batten disease).

    abstract:OBJECTIVE:To use the Unified Batten Disease Rating Scale (UBDRS) to measure the rate of decline in physical and functional capability domains in patients with juvenile neuronal ceroid lipofuscinosis (JNCL) or Batten disease, a neurodegenerative lysosomal storage disorder. We have evaluated the UBDRS in subjects with JN...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/WNL.0b013e318237f649

    authors: Kwon JM,Adams H,Rothberg PG,Augustine EF,Marshall FJ,Deblieck EA,Vierhile A,Beck CA,Newhouse NJ,Cialone J,Levy E,Ramirez-Montealegre D,Dure LS,Rose KR,Mink JW

    更新日期:2011-11-15 00:00:00

  • Controlled-release carbidopa/levodopa (Sinemet 50/200 CR4): clinical and pharmacokinetic studies.

    abstract::Several controlled-release carbidopa/levodopa preparations have been formulated to achieve a more stable and extended antiparkinsonian action. The most effective is Sinemet CR (Sinemet CR4), with an erodible polymer matrix that retards release of levodopa. In 19 parkinsonians with prominent dose-by-dose fluctuations, ...

    journal_title:Neurology

    pub_type: 临床试验,杂志文章

    doi:

    authors: LeWitt PA,Nelson MV,Berchou RC,Galloway MP,Kesaree N,Kareti D,Schlick P

    更新日期:1989-11-01 00:00:00

  • Preclinical cognitive decline and subsequent sleep disturbance in older women.

    abstract:OBJECTIVE:To determine whether longitudinal cognitive decline is associated with increased risk of sleep disturbance in older, nondemented, community-dwelling women. METHODS:We studied 2,474 women (mean age 68.9 years) who were part of a prospective study started in 1986; women with baseline or follow-up evidence of p...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/01.wnl.0000265814.69163.da

    authors: Yaffe K,Blackwell T,Barnes DE,Ancoli-Israel S,Stone KL,Study of Osteoporotic Fractures Group.

    更新日期:2007-07-17 00:00:00

  • Risk of natalizumab-associated PML in patients with MS is reduced with extended interval dosing.

    abstract:OBJECTIVE:To use the large dataset from the Tysabri Outreach: Unified Commitment to Health (TOUCH) program to compare progressive multifocal leukoencephalopathy (PML) risk with natalizumab extended interval dosing (EID) vs standard interval dosing (SID) in patients with multiple sclerosis (MS). METHODS:This retrospect...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/WNL.0000000000008243

    authors: Ryerson LZ,Foley J,Chang I,Kister I,Cutter G,Metzger RR,Goldberg JD,Li X,Riddle E,Smirnakis K,Kasliwal R,Ren Z,Hotermans C,Ho PR,Campbell N

    更新日期:2019-10-08 00:00:00

  • Neurophysiologic analysis of neuromuscular symptoms in UK Gulf War veterans: a controlled study.

    abstract:BACKGROUND:UK veterans who were deployed to the Gulf in 1990 to 1991 reported higher prevalence of neuromuscular symptoms. OBJECTIVE:To investigate whether these Gulf War-related symptoms were associated with objective evidence of neuromuscular dysfunction. METHODS:Forty-nine Gulf War veterans with more than four neu...

    journal_title:Neurology

    pub_type: 临床试验,杂志文章

    doi:10.1212/01.wnl.0000032755.27372.fc

    authors: Sharief MK,Priddin J,Delamont RS,Unwin C,Rose MR,David A,Wessely S

    更新日期:2002-11-26 00:00:00

  • The mechanism of action of glatiramer acetate treatment in multiple sclerosis.

    abstract:OBJECTIVE:Glatiramer acetate (formerly known as copolymer 1) is the major noninterferon immunomodulatory agent used in the treatment of relapsing-remitting multiple sclerosis. Its mechanism of action over the past 40 years has evolved with our understanding of the immune response. METHODS:We review the various mechani...

    journal_title:Neurology

    pub_type: 杂志文章,评审

    doi:10.1212/WNL.0b013e3181c97e39

    authors: Racke MK,Lovett-Racke AE,Karandikar NJ

    更新日期:2010-01-05 00:00:00

  • Cerebral vasocapacitance and TIAs.

    abstract::We report the vasocapacitance of the cerebral circulation, as determined by cerebral blood flow reactivity to induced hypercapnia using fluoromethane positron emission tomography, in 32 patients with unilateral anterior circulation transient ischemic attacks. A hemodynamic subset of eight patients, defined based on ex...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.39.1.25

    authors: Levine RL,Lagreze HL,Dobkin JA,Hanson JM,Satter MR,Rowe BR,Nickles RJ

    更新日期:1989-01-01 00:00:00

  • Neurofilament light and tau as blood biomarkers for sports-related concussion.

    abstract:OBJECTIVE:To compare neurofilament light (NfL) and tau as blood-based biomarkers for acute sports-related concussion (SRC) and determine whether their concentrations at different time points after the injury are associated with prolonged time to return to play (RTP). METHODS:A total of 288 professional hockey players ...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/WNL.0000000000005518

    authors: Shahim P,Tegner Y,Marklund N,Blennow K,Zetterberg H

    更新日期:2018-05-15 00:00:00

  • Blinded evaluation confirms long-term asymmetric effect of unilateral thalamotomy or subthalamotomy on tremor in Parkinson's disease.

    abstract::In the past, stereotactic surgery was a regular treatment for prominent unilateral tremor in Parkinson's disease (PD), but follow-up studies were usually short-term and always unblinded. We examined 17 PD patients in long-term follow-up (mean, 10.9 years after surgery) and used videotapes and the Unified Parkinson's D...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.42.7.1311

    authors: Diederich N,Goetz CG,Stebbins GT,Klawans HL,Nittner K,Koulosakis A,Sanker P,Sturm V

    更新日期:1992-07-01 00:00:00

  • Experimental CNS ischemia: serotonin antagonists reduce or prevent damage.

    abstract::We tested three serotonin antagonists--lysergic acid diethylamide (LSD), 2-bromolysergic acid diethylamide (BOL), and cinanserin--for effects on experimental spinal cord ischemia in the rabbit. Blood flow to spinal cord was obstructed. Drugs were administered before or after initiation of occlusion, and flow was later...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.34.4.469

    authors: Zivin JA,Venditto JA

    更新日期:1984-04-01 00:00:00

  • Myasthenia gravis with features of the myasthenic syndrome. An investigation with electrophysiologic methods including single-fiber electromyography.

    abstract::A 47-year-old woman had myasthenia gravis with only moderate weakness and a consistently poor response to anticholinesterase medication. She was investigated with routine electrophysiologic studies and single-fiber electromyography. The studies showed a limited effect of anticholinesterase medication. There was a decr...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.25.1.80

    authors: Schwartz MS,Stålberg E

    更新日期:1975-01-01 00:00:00

  • Prospective study of military service and mortality from ALS.

    abstract:BACKGROUND:Two recent studies suggest that the risk of ALS is increased among Gulf War veterans. It is not known whether military service outside of the Gulf War is associated with increased risk of ALS. METHODS:The authors prospectively assessed the relation between service in the military and ALS mortality among par...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/01.WNL.0000148649.17706.D9

    authors: Weisskopf MG,O'Reilly EJ,McCullough ML,Calle EE,Thun MJ,Cudkowicz M,Ascherio A

    更新日期:2005-01-11 00:00:00

  • Noninvasive diagnosis of extracranial cerebrovascular disease: oculoplethysmography-phonoangiography and directional Doppler ultrasonography.

    abstract::The ability of two noninvasive diagnostic methods--oculoplethysmography-carotid phonoangiography (OPG/CPA) and directional Doppler ultrasonography--to detect extracranial cerebrovascular disease was examined in 400 consecutive studies. Cerebral arteriography was performed in 74 patients. Transient ischemic attack was ...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.29.5.623

    authors: Ginsberg MD,Greenwood SA,Goldberg HI

    更新日期:1979-05-01 00:00:00

  • Risk factors in Alzheimer's disease: a clinicopathologic study.

    abstract::We investigated potential risk factors for Alzheimer's disease (AD) in a clinicopathologic study of 407 patients with definite AD, 100 non-Alzheimer dementia patients, and 50 normal subjects. The AD patients had more first-degree relatives with dementia than the non-AD dementia group (odds ratio of 1.85, 95% confidenc...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.42.4.770

    authors: Mendez MF,Underwood KL,Zander BA,Mastri AR,Sung JH,Frey WH 2nd

    更新日期:1992-04-01 00:00:00

  • IgG receptor IIa alleles determine susceptibility and severity of Guillain-Barré syndrome.

    abstract:OBJECTIVE:Guillain-Barré syndrome (GBS) is characterized by nerve infiltration of leukocytes and autoantibodies of the immunoglobulin G (IgG) isotype directed against nerve constituents. Leukocyte receptors for IgG (FcgammaR) constitute an important link between the humoral and cellular parts of the immune system and c...

    journal_title:Neurology

    pub_type: 临床试验,杂志文章

    doi:10.1212/wnl.54.8.1661

    authors: van der Pol WL,van den Berg LH,Scheepers RH,van der Bom JG,van Doorn PA,van Koningsveld R,van den Broek MC,Wokke JH,van de Winkel JG

    更新日期:2000-04-25 00:00:00

  • Practice parameter: evaluation of children and adolescents with recurrent headaches: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society.

    abstract:OBJECTIVE:The Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society develop practice parameters as strategies for patient management based on analysis of evidence. For this parameter, the authors reviewed available evidence on the evaluation of the...

    journal_title:Neurology

    pub_type: 指南,杂志文章,实务指引

    doi:10.1212/wnl.59.4.490

    authors: Lewis DW,Ashwal S,Dahl G,Dorbad D,Hirtz D,Prensky A,Jarjour I,Quality Standards Subcommittee of the American Academy of Neurology.,Practice Committee of the Child Neurology Society.

    更新日期:2002-08-27 00:00:00

  • A 1-year controlled trial of acetyl-l-carnitine in early-onset AD.

    abstract:OBJECTIVE:To determine the efficacy of acetyl-l-carnitine (ALCAR) on the rate of decline in early-onset AD patients. METHODS:A 1-year, multicenter, double-blind, placebo-controlled, randomized trial was conducted. Subjects were 45 to 65 years old, with a diagnosis of probable AD according to National Institute of Neur...

    journal_title:Neurology

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1212/wnl.55.6.805

    authors: Thal LJ,Calvani M,Amato A,Carta A

    更新日期:2000-09-26 00:00:00

  • The effects of acute and chronic ethanol administration on twitch and tetanic force production by rat gastrocnemius muscle in situ.

    abstract::The effects of acute ethanol injection on force production were measured in normal and chronic alcoholic rats. Acutely injected normal rats, acutely injected chronic alcoholic rats, and noninjected chronic alcoholic rats were compared with control groups: normal nonalcoholic rats and a dietetically deprived control gr...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.30.2.139

    authors: Martyn DA,Munsat TL

    更新日期:1980-02-01 00:00:00

  • Practice guideline update summary: Pharmacologic treatment for pediatric migraine prevention: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology and the American Headache Society.

    abstract:OBJECTIVE:To provide updated evidence-based recommendations for migraine prevention using pharmacologic treatment with or without cognitive behavioral therapy in the pediatric population. METHODS:The authors systematically reviewed literature from January 2003 to August 2017 and developed practice recommendations usin...

    journal_title:Neurology

    pub_type: 杂志文章,评审

    doi:10.1212/WNL.0000000000008105

    authors: Oskoui M,Pringsheim T,Billinghurst L,Potrebic S,Gersz EM,Gloss D,Holler-Managan Y,Leininger E,Licking N,Mack K,Powers SW,Sowell M,Victorio MC,Yonker M,Zanitsch H,Hershey AD

    更新日期:2019-09-10 00:00:00

  • Predictive role of heterozygous p.R4810K of RNF213 in the phenotype of Chinese moyamoya disease.

    abstract:OBJECTIVE:Precise genetic analyses were conducted with ring finger protein 213 (RNF213) in relation to a particular clinical phenotype in Chinese patients with moyamoya disease (MMD) to determine whether heterozygosity is responsible for the early-onset and severe form of this disease. METHODS:A case-control study for...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/WNL.0000000000008901

    authors: Wang Y,Zhang Z,Wei L,Zhang Q,Zou Z,Yang L,Li D,Shang M,Han C,Mambiya M,Bao X,Li Q,Hao F,Zhang K,Wang H,Liu S,Liu M,Zeng F,Nie F,Wang K,Liu W,Duan L

    更新日期:2020-02-18 00:00:00

  • Gamma-hydroxybutyrate in experimental myoclonus.

    abstract::In Swiss mice, muscimol (3 mg per kilogram, intraperitoneally) caused myoclonic jerks of the hindquarters. These jerks were strong, repetitive, more or less regular, and of high frequency (peak response, 76 per minute). Pretreatment with sodium gamma-hydroxybutyrate (50 to 200 mg per kilogram) caused a dose-dependent ...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.32.4.434

    authors: Menon MK

    更新日期:1982-04-01 00:00:00

  • Insulin resistance and risk for stroke.

    abstract:BACKGROUND AND PURPOSE:Resistance to insulin-mediated glucose uptake by peripheral tissues is a cardinal defect in type 2 diabetes mellitus. Insulin resistance is also common among nondiabetic individuals, and may be an important risk factor for stroke in both populations. The authors review the definition, epidemiolog...

    journal_title:Neurology

    pub_type: 杂志文章,评审

    doi:10.1212/wnl.59.6.809

    authors: Kernan WN,Inzucchi SE,Viscoli CM,Brass LM,Bravata DM,Horwitz RI

    更新日期:2002-09-24 00:00:00

  • Brugada syndrome in spinal and bulbar muscular atrophy.

    abstract:OBJECTIVE:The aim of this study was to clarify myocardial involvement and its clinical implications in subjects with spinal and bulbar muscular atrophy (SBMA), a neuromuscular disease affecting both neuronal and nonneuronal tissues. METHODS:Two independent cardiologists evaluated ECGs from a total of 144 consecutive s...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/WNL.0000000000000434

    authors: Araki A,Katsuno M,Suzuki K,Banno H,Suga N,Hashizume A,Mano T,Hijikata Y,Nakatsuji H,Watanabe H,Yamamoto M,Makiyama T,Ohno S,Fukuyama M,Morimoto S,Horie M,Sobue G

    更新日期:2014-05-20 00:00:00

  • Medial temporal lobe atrophy on MRI scans and the diagnosis of Alzheimer disease.

    abstract:BACKGROUND:Despite convenience, accessibility, and strong correlation to severity of Alzheimer disease (AD) pathology, medial temporal lobe atrophy (MTA) has not been used as a criterion in the diagnosis of prodromal and probable AD. METHODS:Using a newly validated visual rating system, mean MTA scores of three bilate...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/01.wnl.0000336925.79704.9f

    authors: Duara R,Loewenstein DA,Potter E,Appel J,Greig MT,Urs R,Shen Q,Raj A,Small B,Barker W,Schofield E,Wu Y,Potter H

    更新日期:2008-12-09 00:00:00

  • Dementia in Down's syndrome: cerebral glucose utilization, neuropsychological assessment, and neuropathology.

    abstract::We measured the cerebral metabolic rate for glucose (CMRglc) with positron emission tomography and [18F]2-fluoro-2-deoxy-D-glucose in a 47-year-old man with trisomy 21 Down's syndrome (DS) and autopsy-confirmed Alzheimer's disease. Dementia was evident from a confirmed history of cognitive decline, memory loss, and pe...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/wnl.38.6.938

    authors: Schapiro MB,Ball MJ,Grady CL,Haxby JV,Kaye JA,Rapoport SI

    更新日期:1988-06-01 00:00:00

  • Thymus changes in anti-MuSK-positive and -negative myasthenia gravis.

    abstract::Morphologic findings of thymuses from 32 anti-acetylcholine receptor (AChR)-negative myasthenia gravis patients, 12 with and 20 without antibodies against the muscle-specific kinase (MuSK), were compared with those from 30 AChR-positive subjects. In contrast with the high frequency of thymic hyperplastic changes in AC...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/01.WNL.0000150587.71497.B6

    authors: Lauriola L,Ranelletti F,Maggiano N,Guerriero M,Punzi C,Marsili F,Bartoccioni E,Evoli A

    更新日期:2005-02-08 00:00:00

  • Prevalence and significance of stroke symptoms among patients receiving maintenance dialysis.

    abstract:OBJECTIVE:The purpose of this cross-sectional study was to determine the prevalence and potential significance of stroke symptoms among end-stage renal disease (ESRD) patients without a prior diagnosis of stroke or TIA. METHODS:We enrolled 148 participants with ESRD from 5 clinics. Stroke symptoms and functional statu...

    journal_title:Neurology

    pub_type: 杂志文章

    doi:10.1212/WNL.0b013e31826845e9

    authors: Kurella Tamura M,Meyer JB,Saxena AB,Huh JW,Wadley VG,Schiller B

    更新日期:2012-09-04 00:00:00