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  1. Azarisman MS, Fauzi MA, Faizal MP, Azami Z, Roslina AM, Roslan H
    Postgrad Med J, 2007 Jul;83(981):492-7.
    PMID: 17621621
    BACKGROUND: This study was proposed to develop a composite of outcome measures using forced expiratory volume percentage of predicted, exercise capacity and quality of life scores for assessment of chronic obstructive pulmonary disease (COPD) severity.
    MATERIALS AND METHODS: Eighty-six patients with COPD were enrolled into a prospective, observational study at the respiratory outpatient clinic, National University Hospital Malaysia (Hospital Universiti Kebangsaan Malaysia--HUKM), Kuala Lumpur.
    RESULTS: Our study found modest correlation between the forced expiratory volume in 1 s (FEV(1)), 6 min walk distance and the SGRQ scores with mean (SD) values of 0.97 (0.56) litres/s, 322 (87) m and 43.7 (23.6)%, respectively. K-Means cluster analysis identified four distinct clusters which reached statistical significance which was refined to develop a new cumulative staging system. The SAFE Index score correlated with the number of exacerbations in 2 years (r = 0.497, p<0.001).
    CONCLUSION: We have developed the SGRQ, Air-Flow limitation and Exercise tolerance Index (SAFE Index) for the stratification of severity in COPD. This index incorporates the SGRQ score, the FEV(1) % predicted and the 6 min walk distance. The SAFE Index is moderately correlated with the number of disease exacerbations.
    Study site: Respiratory clinic, Pusat Perubatan Universiti Kebangsaan Malaysia (PPUKM), Kuala Lumpur, Malaysia
    Matched MeSH terms: Quality of Life
  2. Mariappan P, Chong WL
    BJU Int, 2006 Dec;98(6):1264-8.
    PMID: 17034498
    OBJECTIVE: To determine, in a population-based survey, the prevalence of lower urinary tract symptoms (LUTS), erectile dysfunction (ED) and incontinence in community-dwelling men in multiethnic Malaysia, as currently available Western demographic data might not be applicable in the Asian population.
    SUBJECTS AND METHODS: A cross-sectional population-based survey was carried out in the State of Penang, Malaysia, with a target population of men aged > or = 40 years. Using a multistage study design, random systematic sampling was used to represent the target population, who were weighted based on ethnicity and rural-urban ratios so as to represent the general population distribution. Trained field-workers conducted direct interviews and administered the American Urological Association Symptom Index (AUA-SI), the International Index of Erectile Function (IIEF-5) questionnaire and questions on incontinence based on the International Continence Society 2002 definition.
    RESULTS: In all, 418 men aged > or = 40 years were interviewed, of whom 353 completed the AUA-SI questionnaire (84.5% response rate). The prevalence of mild, moderate and severe LUTS was 80.6%, 6% and 0.3%, respectively. The prevalent symptoms were frequency and nocturia. There was moderate and severe ED in 45.9% of men, whereas incontinence was reported by 8.2%. The AUA-SI correlated strongly with age (R = 0.291, P < 0.001), IIEF-5 (R = - 0.265, P < 0.001) and diabetes mellitus.
    CONCLUSION: The prevalence and severity of LUTS, ED and incontinence increased with age in this multiethnic Asian population, in which ED correlated strongly with LUTS. Compared to the Western population, the prevalence of LUTS was significantly lower, while the prevalence of ED and incontinence were comparable.
    Matched MeSH terms: Quality of Life
  3. Sazlina SG, Browning CJ, Yasin S
    BMJ Open, 2012;2(6).
    PMID: 23161092 DOI: 10.1136/bmjopen-2012-002119
    INTRODUCTION: Like many countries Malaysia is facing an increase in the number of people with type 2 diabetes mellitus diabetes (T2DM) and modifiable lifestyle factors such as sedentary behaviour are important drivers of this increase. The level of physical activity is low among elderly Malay people. In Malaysia, strategies to promote physical activity in elderly Malay people with T2DM are not well documented in the research literature. This paper discusses an intervention to increase physical activity in elderly Malay people with T2DM. The aim of our study was to evaluate the effectiveness of personalised feedback alone and in combination with peer support in promoting and maintaining physical activity in comparison with usual care.
    METHODS AND ANALYSIS: A three-arm randomised controlled trial will be conducted among sedentary Malay adults aged 60 years and above with T2DM attending an urban primary healthcare clinic in Malaysia. The participants will be randomised into three groups for a 12-week intervention with a follow-up at 24 and 36 weeks to assess adherence. The primary outcome of this study is pedometer-determined physical activity. Glycaemic and blood pressure control, body composition, cardiorespiratory fitness, balance, lipid profile, health-related quality of life, psychological well-being, social support and self-efficacy for exercise are the secondary measures. Linear mixed models will be used to determine the effect of the intervention over time and between groups. ETHICAL AND DISSEMINATION: The Monash University Human Research Ethics Committee and the Malaysian Ministry of Health's Medical Research Ethics Committee approved this protocol. The findings of this study will be presented at international conferences and published in peer-reviewed journals.
    TRIAL REGISTRATION: This study protocol has been registered with the Malaysian National Medical Research Registry and with the Current Controlled Trial Ltd (http://www.controlled-trials.com/ISRCTN71447000/).
    Matched MeSH terms: Quality of Life
  4. Biswal BM, Sulaiman SA, Ismail HC, Zakaria H, Musa KI
    Integr Cancer Ther, 2013 Jul;12(4):312-22.
    PMID: 23142798 DOI: 10.1177/1534735412464551
    Hypothesis. Withania somnifera is an herb with antioxidant, anti-inflammatory, anticancer, antistress, and adaptogenic properties. Previous studies have shown its antistress effects in animals. Traditional Indian medicine has used it for centuries to alleviate fatigue and improve general well-being.
    Matched MeSH terms: Quality of Life
  5. Cheah PY, Liong ML, Yuen KH, Teh CL, Khor T, Yang JR, et al.
    Urology, 2004 Nov;64(5):881-6.
    PMID: 15533470 DOI: 10.1016/j.urology.2004.06.041
    OBJECTIVES: To evaluate the initial, long-term, and durable response rates to terazosin, placebo, or other therapies in patients with chronic prostatitis/chronic pelvic pain syndrome.
    METHODS: A total of 100 subjects, aged 20 to 50 years, who met the National Institutes of Health criteria for chronic prostatitis/chronic pelvic pain syndrome and had not previously been treated with alpha-blockers, were entered in a 14-week, double-blind comparison of terazosin or placebo therapy. Nonresponders and responders with subsequent relapse were treated with terazosin or other medications (open label). The criterion for response was a score of 0 to 2 on the National Institutes of Health Chronic Prostatitis Symptom Index quality-of-life item. The initial response was evaluated at week 14, and the long-term response was evaluated after a median of 38 weeks (range 34 to 42), regardless of any additional treatment. A durable response was defined as an initial response without additional treatment.
    RESULTS: Of the 43 patients in the terazosin group, 24 (56%) had an initial response compared with 14 (33%) of 43 subjects in the placebo group (P = 0.03). Long-term responses were noted in 23 (56%) of 41 assessable subjects treated with terazosin initially compared with 12 (32%) of 38 assessable subjects treated with placebo (P = 0.03). Of the nonresponders and initial responders with relapse, 7 (41%) of 17 subjects responded to terazosin compared with 7 (21%) of 34 given other treatment (P = 0.12). Durable responses occurred in 18 (44%) of the 41 assessable patients treated initially with terazosin and in 6 (16%) of 38 treated initially with placebo (P = 0.01).
    CONCLUSIONS: Patients treated with terazosin were more likely to have initial, long-term, and durable responses than those treated with placebo.
    Matched MeSH terms: Quality of Life
  6. Quek KF, Razack AH, Chua CB, Low WY, Loh CS
    Int J Urol, 2004 Oct;11(10):848-55.
    PMID: 15479289
    The present study aimed to evaluate the effects of treating lower urinary tract symptoms (LUTS) on anxiety, depression and psychiatric morbidity following one year of follow-up.
    Matched MeSH terms: Quality of Life
  7. Tan FL, Loh SY, Su TT, Veloo VW, Ng LL
    Asian Pac J Cancer Prev, 2012;13(11):5791-7.
    PMID: 23317258
    INTRODUCTION: Return-to-work (RTW) can be a problematic occupational issue with detrimental impact on the quality of life of previously-employed breast cancer survivors. This study explored barriers and facilitators encountered during the RTW process in the area of cancer survivorship.

    MATERIALS AND METHODS: Six focus groups were conducted using a semi-structured interview guide on 40 informants (employed multiethnic survivors). Survivors were stratified into three groups for successfully RTW, and another three groups of survivors who were unable to return to work. Each of the three groups was ethnically homogeneous. Thematic analysis using a constant comparative approach was aided by in vivo software.

    RESULTS: Participants shared numerous barriers and facilitators which directly or interactively affect RTW. Key barriers were physical-psychological after-effects of treatment, fear of potential environment hazards, high physical job demand, intrusive negative thoughts and overprotective family. Key facilitators were social support, employer support, and regard for financial independence. Across ethnic groups, the main facilitators were financial-independence (for Chinese), and socialisation opportunity (for Malay). A key barrier was after-effects of treatment, expressed across all ethnic groups.

    CONCLUSIONS: Numerous barriers were identified in the non-RTW survivors. Health professionals and especially occupational therapists should be consulted to assist the increasing survivors by providing occupational rehabilitation to enhance RTW amongst employed survivors. Future research to identify prognostic factors can guide clinical efforts to restore cancer survivors to their desired level/type of occupational functioning for productivity and wellbeing.

    Matched MeSH terms: Quality of Life
  8. Wey MC, Loh S, Doss JG, Abu Bakar AK, Kisely S
    Aust N Z J Psychiatry, 2016 Jul;50(7):685-94.
    PMID: 26560842 DOI: 10.1177/0004867415615947
    OBJECTIVE: People with chronic schizophrenia have high rates of physical ill-health such as heart disease. However, there has been less attention to the issue of poor oral health including dental caries (tooth decay) and periodontal (gum) disease, although both have consequences for quality of life and systemic physical health. We therefore measured tooth decay and gum disease in Malaysians with schizophrenia.

    METHODS: We recruited long-stay inpatients with schizophrenia from June to October 2014. Four dental specialists assessed oral health using the decayed-missing-filled teeth index, the Community Periodontal Index of Treatment Needs and the Debris Index of the Simplified Oral Hygiene Index. Results were compared with the 2010 Oral Health survey of the general Malaysian population.

    RESULTS: A total of 543 patients participated (66.7% males, 33.3% females; mean age = 54.8 years [standard deviation = 16.0]) with a mean illness duration of 18.4 years (standard deviation = 17.1). The mean decayed-missing-filled teeth was 20.5 (standard deviation = 9.9), almost double that of the general population (11.7). Higher decayed-missing-filled teeth scores were associated with both older age (p 

    Matched MeSH terms: Quality of Life
  9. Aljumah K, Hassali MA
    BMC Psychiatry, 2015;15:219.
    PMID: 26376830 DOI: 10.1186/s12888-015-0605-8
    Adherence to antidepressant treatment is essential for the effective management of patients with major depressive disorder. Adherence to medication is a dynamic decision-making process, and pharmacists play an important role in improving adherence to antidepressant treatment in different settings within the healthcare system. The aim of this study was to assess whether pharmacist interventions based on shared decision making improved adherence and patient-related outcomes.
    Matched MeSH terms: Quality of Life
  10. Syed Nong Chek SR, Robinson S, Mohd Affandi A, Baharum N
    Int J Dermatol, 2016 Oct;55(10):1092-5.
    PMID: 27061170 DOI: 10.1111/ijd.13307
    BACKGROUND: Psoriasis involving the face is visible and can cause considerable emotional distress to patients. Its presence may also confer a poorer prognosis for the patient. This study sought to evaluate the characteristics of facial psoriasis in Malaysia.

    METHODS: A cross-sectional study conducted using data from the Malaysian Psoriasis Registry from 2007 to 2011. Specific risk factors, i.e., age, age of onset, gender, duration of disease, obesity group, body surface area, Dermatology Life Quality Index (DLQI), family history of psoriasis, nail involvement, psoriatic arthritis, phototherapy, systemic therapy, clinic visit, days of work/school, and hospital admission due to psoriasis in the last 6 months were analyzed.

    RESULTS: A total of 48.4% of patients had facial psoriasis. Variables significantly associated with facial psoriasis are younger age, younger age of onset of psoriasis of ≤ 40 years, male, severity of psoriasis involving >10% of the body surface area, higher DLQI of >10, nail involvement, and history of hospitalization due to psoriasis.

    CONCLUSION: This study found that facial psoriasis is not as rare as previously thought. Ambient ultraviolet light, sebum, and contact with chemicals from facial products may reduce the severity of facial psoriasis, but these factors do not reduce the prevalence of facial psoriasis. The association with younger age, younger age of onset, higher percentage of body surface area involvement, higher DLQI of > 10, nail involvement, and hospitalization due to psoriasis support the notion that facial psoriasis is a marker of severe disease.

    Matched MeSH terms: Quality of Life
  11. Quek KF, Low WY, Razack AH, Chua CB, Loh CS
    J Sex Marital Ther, 2002 Oct-Dec;28(5):423-6.
    PMID: 12378843
    The purpose of this study is to validate the English version of the Golombok-Rust Inventory of Marital State (GRIMS) in a Malaysian population. Using the test-retest method and Cronbach's alpha, we assessed reliability and internal consistency. Sensitivity to change was expressed as the effect size. Internal consistency was excellent (Cronbach's alpha value = 0.59 to 0.91) Test-retest correlation coefficient and intraclass correlation coefficient were highly significant in majority items (ICC = 0.62 and above), and there was a high degree of sensitivity and specificity. The GRIMS is suitable, reliable, valid, and sensitive to clinical change in a Malaysian population.
    Matched MeSH terms: Quality of Life
  12. Suzana S, Earland J, Suriah AR, Warnes AM
    J Nutr Health Aging, 2002;6(6):363-9.
    PMID: 12459886
    Older people especially those residing in rural areas are at a greater risk of malnutrition.
    Matched MeSH terms: Quality of Life
  13. Appalasamy JR, Tha KK, Quek KF, Ramaiah SS, Joseph JP, Md Zain AZ
    Medicine (Baltimore), 2018 Jun;97(22):e10876.
    PMID: 29851804 DOI: 10.1097/MD.0000000000010876
    INTRODUCTION: A substantial number of the world's population appears to end with moderate to severe long-term disability after stroke. Persistent uncontrolled stroke risk factor leads to unpredicted recurrent stroke event. The increasing prevalence of stroke across ages in Malaysia has led to the adaptation of medication therapy adherence clinic (MTAC) framework. The stroke care unit has limited patient education resources especially for patients with medication understanding and use self-efficacy. Nevertheless, only a handful of studies have probed into the effectiveness of video narrative at stroke care centers.

    METHOD: This is a behavioral randomized controlled trial of patient education intervention with video narratives for patients with stroke lacking medication understanding and use self-efficacy. The study will recruit up to 200 eligible stroke patients at the neurology tertiary outpatient clinic, whereby they will be requested to return for follow-up approximately 3 months once for up to 12 months. Consenting patients will be randomized to either standard patient education care or intervention with video narratives. The researchers will ensure control of potential confounding factors, as well as unbiased treatment review with prescribed medications only obtained onsite.

    RESULTS: The primary analysis outcomes will reflect the variances in medication understanding and use self-efficacy scores, as well as the associated factors, such as retention of knowledge, belief and perception changes, whereas stroke risk factor control, for example, self-monitoring and quality of life, will be the secondary outcomes.

    DISCUSSION AND CONCLUSION: The study should be able to determine if video narrative can induce a positive behavioral change towards stroke risk factor control via enhanced medication understanding and use self-efficacy. This intervention is innovative as it combines health belief, motivation, and role model concept to trigger self-efficacy in maintaining healthy behaviors and better disease management.

    TRIAL REGISTRATION: ACTRN (12618000174280).

    Matched MeSH terms: Quality of Life
  14. Ismail NFF, Neoh CF, Lim SM, Abdullah AH, Mastuki MF, Ramasamy K, et al.
    Medicine (Baltimore), 2017 Jul;96(30):e7511.
    PMID: 28746195 DOI: 10.1097/MD.0000000000007511
    INTRODUCTION: Asian countries have a variety of ethnic groups and culture that provide their own traditional treatment in health care. Facial candling appears to be one of the popular traditional treatments in Southeast Asian. The complementary medicine practitioners promote that the facial candling treatment would help in reducing the symptoms of allergic rhinitis and other problems related to sinus. Due to the lack of evidence available, the effectiveness of this treatment method and its mechanism, however, remains unknown. The objective of this research is therefore to study impact of facial candling on inflammatory mediators, substance P (SP), symptoms severity, and quality of life (QoL) in allergic rhinitis patients.

    METHOD AND ANALYSIS: A randomized, nonblinded, controlled trial will be carried out by recruiting a total of 66 eligible allergic rhinitis patients who fulfill the inclusion criteria from a university health center. The subjects will be randomly assigned into 2 groups: intervention group receiving facial candling treatment and control group (no treatment given). Samples of blood and nasal mucus will be collected right before and after intervention. Samples collected will be analyzed. The primary outcomes are the changes in the level of SP in both blood and mucus samples between both groups. The secondary outcomes include the levels of inflammatory mediators (ie, tumor necrosis factor alpha, interleukin (IL)-3, IL-5, IL-6, IL-10, and IL-13) and the severity of allergic rhinitis symptoms as measured by a visual analogous scale and QoL using the Rhinitis Quality of Life Questionnaire (RQLQ).

    ETHICAL AND TRIAL REGISTRATION: The study protocols are approved from the Ethical and Research Committee of the Universiti Teknologi MARA (REC/113/15). The trial is registered under the Australia New Zealand Clinical Trial Registry (ACTRN12616000299404). The trial was registered on 03/07/2016 and the first patient was enrolled on 10/12/2016.

    CONCLUSION: Facial candling is one of the unique treatments using candles to reduce the severity of symptoms and inflammation. This is the first ever study conducted on facial candling that will give rise to new knowledge underlying the effects of facial candling on severity of symptoms and inflammation relief mechanism mediated by substance P and inflammatory mediators.

    Matched MeSH terms: Quality of Life
  15. Rehman IU, Wu DB, Ahmed R, Khan NA, Rahman AU, Munib S, et al.
    Medicine (Baltimore), 2018 08;97(31):e10764.
    PMID: 30075491 DOI: 10.1097/MD.0000000000010764
    BACKGROUND: Pruritus adds to the complications of chronic kidney disease (CKD) patient and a well-recognized complication among the CKD patients. Majority of the patients on hemodialysis experience a generalized pruritus and patients reported being moderately to extremely disturbed by at least one of the sleep-related condition. This study aim to investigate the effectiveness of zolpidem 10 mg and acupressure therapy on foot acupoints to improve the sleep quality and overall quality of life among hemodialysis patients suffering from CKD-associated pruritus.

    METHODS: A multicentered, open-label, parallel group, prospective randomized controlled trial among patients suffering from CKD-associated pruritus with sleep disturbance, after randomization into control, and intervention group to be held at North West General Hospital and Research Center Peshawar, Pakistan and Institute of Kidney Diseases Peshawar, Pakistan.

    RESULTS: The primary outcome is to investigate the effectiveness of zolpidem 10 mg and acupressure therapy on foot acupoints to improve the sleep quality and overall quality of life among hemodialysis patients suffering from CKD-associated pruritus. After baseline assessment by Urdu version of 5D itch scale and Urdu version of Pittsburgh Sleep Quality Index (PSQI) and Urdu EQ-5D 3L, the intervention group will be given zolpidem 10 mg oral tablets and control group with acupressure on both foots on KI-1 acupoints for total of 6 minutes. Assessment will be done at weeks 4 and 8 from baseline by using Urdu version of 5D itch scale and Urdu version of PSQI and Urdu EQ-5D 3L, whereas safety profiling of zolpidem 10 mg tablet at week 6 from baseline and acupressure acceptability at week 6 from baseline. Analysis of covariance will be used to examine the differences in treatment effects between the intervention and control groups.

    CONCLUSION: Improvement of sleep quality and quality of life among patients with CKD-associated pruritus requires great importance. This study aims to improve the quality of sleep and quality of life among patients with hemodialysis suffering from CKD-associated pruritus.

    Matched MeSH terms: Quality of Life
  16. Sprigg N, Flaherty K, Appleton JP, Al-Shahi Salman R, Bereczki D, Beridze M, et al.
    Health Technol Assess, 2019 07;23(35):1-48.
    PMID: 31322116 DOI: 10.3310/hta23350
    BACKGROUND: Tranexamic acid reduces death due to bleeding after trauma and postpartum haemorrhage.

    OBJECTIVE: The aim of the study was to assess if tranexamic acid is safe, reduces haematoma expansion and improves outcomes in adults with spontaneous intracerebral haemorrhage (ICH).

    DESIGN: The TICH-2 (Tranexamic acid for hyperacute primary IntraCerebral Haemorrhage) study was a pragmatic, Phase III, prospective, double-blind, randomised placebo-controlled trial.

    SETTING: Acute stroke services at 124 hospitals in 12 countries (Denmark, Georgia, Hungary, Ireland, Italy, Malaysia, Poland, Spain, Sweden, Switzerland, Turkey and the UK).

    PARTICIPANTS: Adult patients (aged ≥ 18 years) with ICH within 8 hours of onset.

    EXCLUSION CRITERIA: Exclusion criteria were ICH secondary to anticoagulation, thrombolysis, trauma or a known underlying structural abnormality; patients for whom tranexamic acid was thought to be contraindicated; prestroke dependence (i.e. patients with a modified Rankin Scale [mRS] score > 4); life expectancy  4.5 hours after stroke onset. Pragmatic inclusion criteria led to a heterogeneous population of participants, some of whom had very large strokes. Although 12 countries enrolled participants, the majority (82.1%) were from the UK.

    CONCLUSIONS: Tranexamic acid did not affect a patient's functional status at 90 days after ICH, despite there being significant modest reductions in early death (by 7 days), haematoma expansion and SAEs, which is consistent with an antifibrinolytic effect. Tranexamic acid was safe, with no increase in thromboembolic events.

    FUTURE WORK: Future work should focus on enrolling and treating patients early after stroke and identify which participants are most likely to benefit from haemostatic therapy. Large randomised trials are needed.

    TRIAL REGISTRATION: Current Controlled Trials ISRCTN93732214.

    FUNDING: This project was funded by the National Institute for Health Research Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 23, No. 35. See the NIHR Journals Library website for further project information. The project was also funded by the Pragmatic Trials, UK, funding call and the Swiss Heart Foundation in Switzerland.

    Matched MeSH terms: Quality of Life
  17. Hassan MZ, Rathnayaka MM, Deen KI
    World J Surg, 2010 Jul;34(7):1641-7.
    PMID: 20180122 DOI: 10.1007/s00268-010-0489-1
    We undertook a prospective longitudinal study of patients with end-stage fecal incontinence who were undergoing transposition of the gracilis muscle as a neo-anal sphincter with external low-frequency electrical stimulation of the nerve to the gracilis combined with biofeedback.
    Matched MeSH terms: Quality of Life
  18. Dolgin E
    Nature, 2019 02;566(7742):143-145.
    PMID: 30723358 DOI: 10.1038/d41586-019-00448-8
    Matched MeSH terms: Quality of Life
  19. Ong FM, Husna Nik Hassan NF, Azman M, Sani A, Mat Baki M
    J Voice, 2019 Jul;33(4):581.e17-581.e23.
    PMID: 29793874 DOI: 10.1016/j.jvoice.2018.01.015
    OBJECTIVES: This study aimed to determine the validity and reliability of Bahasa Malaysia version of Voice Handicap Index-10 (mVHI-10).

    MATERIALS AND METHODS: This cross-sectional study was carried out in the Otorhinolaryngology, Head and Neck Surgery Department of Universiti Kebangsaan Malaysia Medical Centre (UKMMC) from June 2015 to May 2016. The mVHI-10 was produced following a rigorous forward and backward translation. One hundred participants, including 50 healthy volunteers (17 male, 33 female) and 50 patients with voice disorders (26 male, 24 female), were recruited to complete the mVHI-10 before flexible laryngoscopic examinations and acoustic analysis. The mVHI-10 was repeated in 2 weeks via telephone interview or clinic visit. Its reliability and validity were assessed using interclass correlation.

    RESULTS: The test-retest reliability for total mVHI-10 and each item score was high, with the Cronbach alpha of >0.90. The total mVHI-10 score and domain scores were significantly higher (P 

    Matched MeSH terms: Quality of Life
  20. Collins PW, Young G, Knobe K, Karim FA, Angchaisuksiri P, Banner C, et al.
    Blood, 2014 Dec 18;124(26):3880-6.
    PMID: 25261199 DOI: 10.1182/blood-2014-05-573055
    This multinational, randomized, single-blind trial investigated the safety and efficacy of nonacog beta pegol, a recombinant glycoPEGylated factor IX (FIX) with extended half-life, in 74 previously treated patients with hemophilia B (FIX activity ≤2 IU/dL). Patients received prophylaxis for 52 weeks, randomized to either 10 IU/kg or 40 IU/kg once weekly or to on-demand treatment of 28 weeks. No patients developed inhibitors, and no safety concerns were identified. Three hundred forty-five bleeding episodes were treated, with an estimated success rate of 92.2%. The median annualized bleeding rates (ABRs) were 1.04 in the 40 IU/kg prophylaxis group, 2.93 in the 10 IU/kg prophylaxis group, and 15.58 in the on-demand treatment group. In the 40 IU/kg group, 10 (66.7%) of 15 patients experienced no bleeding episodes into target joints compared with 1 (7.7%) of 13 patients in the 10 IU/kg group. Health-related quality of life (HR-QoL) assessed with the EuroQoL-5 Dimensions visual analog scale score improved from a median of 75 to 90 in the 40 IU/kg prophylaxis group. Nonacog beta pegol was well tolerated and efficacious for the treatment of bleeding episodes and was associated with low ABRs in patients receiving prophylaxis. Once-weekly prophylaxis with 40 IU/kg resolved target joint bleeds in 66.7% of the affected patients and improved HR-QoL. This trial was registered at www.clinicaltrials.gov as #NCT01333111.
    Matched MeSH terms: Quality of Life
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