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Results for “Adherence Interventions”

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PubMed2004

Interventions for improving adherence to treatment in patients with high blood pressure in ambulatory settings.

BACKGROUND: Lack of adherence to blood pressure lowering medication is a major reason for poor control of hypertension worldwide. Interventions to improve adherence to antihypertensive medication have been evaluated in randomised trials but it is unclear which interventions are effective. OBJECTIVES: To determine the effectiveness of interventions aiming to increase adherence to blood pressure lowering medication in adults with high blood pressure SEARCH STRATEGY: All-language search of all articles (any year) in the Cochrane Controlled Trials Register (CCTR), MEDLINE, EMBASE, and CINAHL in April 2002. SELECTION CRITERIA: RCTs of interventions to increase adherence to blood pressure lowering medication in adults with essential hypertension in primary care, with adherence to medication and blood pressure control as outcomes DATA COLLECTION AND ANALYSIS: Two authors extracted data independently and in duplicate and assessed each study according to the criteria outlined by the Cochrane Collaboration Handbook. MAIN RESULTS: We included 38 studies testing 58 different interventions and containing data on 15519 patients. The studies were conducted in nine countries between 1975 and 2000. The duration of follow-up ranged from two to 60 months. Due to heterogeneity between studies in terms of interventions and the methods used to measure adherence, we did not pool the results. Simplifying dosing regimens increased adherence in seven out of nine studies, with a relative increase in adherence of 8 per cent to 19.6 per cent. Motivational strategies were successful in 10 out of 24 studies with generally small increases in adherence up to a maximum of 23 per cent. Complex interventions involving more than one technique increased adherence in eight out of 18 studies, ranging from 5 per cent to a maximum of 41 per cent. Patient education alone seemed largely unsuccessful. REVIEWERS' CONCLUSIONS: Reducing the number of daily doses appears to be effective in increasing adherence to blood pressure lowering medication and should be tried as a first line strategy, although there is less evidence of an effect on blood pressure reduction. Some motivational strategies and complex interventions appear promising, but we need more evidence on their effect through carefully designed RCTs.

Antihypertensive Agents↗

PubMed2006

Treatment supporter to improve adherence to antiretroviral therapy in HIV-infected South African adults. A qualitative study.

Little is known about the patient characteristics, social support networks, and relationship factors associated with excellent adherence in resource-limited settings, even though these can be important clues that inform the identification of targets and the approaches of individual- and community-based antiretroviral therapy adherence interventions. In this study, we aimed to understand how patient-selected treatment supports might affect antiretroviral treatment outcomes and to identify key components of support, including the social and material resources necessary for promoting high adherence in South Africa. Data were collected from the proceedings of 2 focus groups with 6 HIV-infected adults each and from 7 in-depth interviews with health care providers in the Western Cape Province of South Africa. The patients and health care workers identified individuals-usually a mother, daughter, sister, brother, or partner-who were confidantes and had moral authority with them. These individuals command respect, and patients allow them to influence health-related decision making, both of which are necessary if they are to be effective treatment supporters. Barriers to adherence identified by study participants include alcohol abuse, stigma related to disclosure of HIV status, and lack of financial resources and food. These are critically important challenges to address if high adherence is to be achieved in this setting. In addition, our results suggest that interventions tailored to treatment supporter characteristics and relationship factors may be effective in influencing patients' antiretroviral therapy adherence.

Anti-HIV Agents↗

PubMed2006

The elusive gold standard. Future perspectives for HIV adherence assessment and intervention.

There is no "gold standard" for the assessment of adherence to HIV/AIDS medications. Similarly, there is no single optimal tool that enhances adherence to HIV/AIDS treatment regimens. This article presents a model that provides a heuristic for selecting adherence assessment approaches and intervention strategies based on the purpose for which each is to be used. First, a broad distinction is made between research and clinical settings. Second, with each of these settings, the selection of assessments and interventions is based on the extent to which the focus is on HIV/AIDS in general or on adherence in particular. Examples applying the model are provided. Finally, new dimensions are discussed for expanding the model, with particular attention to applying the model to the resource-limited settings that are so important in efforts to reduce the morbidity and mortality associated with the global threat of HIV/AIDS.

Anti-HIV Agents↗

PubMed2006

Community outreach with weekly delivery of anti-retroviral drugs compared to cognitive-behavioural health care team-based approach to improve adherence among indigent women newly starting HAART.

Sustained virological suppression requires adherence to >95% of doses of therapy. Overall there is paucity of data on adherence interventions among women and post-intervention outcomes. In this pilot study, we evaluated a novel strategy of weekly delivery of medications (Directly Delivered Therapy: DDT) for six months using an outreach worker (ORW), among ARV naïve indigent women starting HAART and compared the 'during intervention' and 'post-intervention' outcomes to the health care team (a nurse educator, a case worker, a pharmacist and social worker/drug addictions counsellor) based approach termed Adherence Coordination Services (ACS) and the Standard of Care (SoC) historical referent group. The baseline characteristics of the three groups were comparable. The proportion of women who achieved sustained virologic suppression in 4-8 month period for DDT; ACS and SoC groups were 86% (18/21); 54% (6/11); and 36% (8/22) (P<0.004); and in the 10-14 month period were 80% (12/15); 54% (6/11) and 45%(10/22) (P=0.036 for DDT vs. SoC). Retention rate in the DDT was 87%, and 92% of 307 ORW visits were kept, and post-intervention satisfaction was high. Short-term weekly delivery of medications using a community based liaison is a feasible, acceptable and a cost-effective strategy for improving both short-term and perhaps long-term adherence among women initiating their first HAART regimen.

Adult↗

PubMed2002

Interventions to improve adherence to antiretroviral therapy.

Recent research has shown that very high levels of adherence are required to obtain the maximum benefit of highly active antiretroviral therapy (HAART). This situation justifies the importance of developing efficient strategies to improve adherence to drugs against HIV. A comprehensive range of factors are the targets of the most effective interventions to improve adherence, including cognitive, behavioral, emotional, and social aspects. The authors describe three theoretical models that can help identify barriers and guide interventions. Most of the interventions are complex and may include more convenient care, provision of information, counseling, reminders, reinforcement, self-monitoring, family therapy, or additional supervision or attention. The authors suggest aspects that should be included in interventions to promote adherence based on a review of the literature and clinical experience. Limited evidence suggests that interventions to enhance adherence to antiretroviral therapy in people with HIV are most likely to be successful when they are comprehensive, longitudinal, and tailored to the person.

Antiretroviral Therapy, Highly Active↗

PubMed2003

Assessment and predictors of antiretroviral adherence in older HIV-infected patients.

Advances in the diagnosis and treatment of HIV disease have resulted in increased survival for HIV-infected patients. Strict adherence to antiretroviral therapy is required to obtain these benefits, however. This holds true for older patients who often are diagnosed later in their disease course and who have shorter survival periods than younger patients. Although there have been few studies of antiretroviral adherence in older HIV patients, this article reviews the literature regarding antiretroviral adherence in younger HIV-infected adults as well as studies of medication adherence in older patients with other disease states. It then discusses the application of adherence interventions in these other populations to older HIV-infected adults. Several methods have been used in measuring antiretroviral adherence, including electronic monitoring, self-report, pill counts, viral load, therapeutic drug monitoring, and several other techniques. The advantages and disadvantages of these methods also are presented as well as recommendations for future adherence research in older HIV-infected adults. Finally, this article details areas of unmet research need concerning HIV medication adherence in older adults.

Adult↗

PubMed2005

Development and implementation of a medication adherence training instrument for persons living with HIV: the MATI.

Recent advances in the medical management of HIV offer the potential for increased longevity, improved quality of life, and the treatment of HIV as a chronic, rather than terminal, illness. The advent of highly active antiretroviral therapy (HAART) has required the necessity of strict adherence to complex medication regimens. As a vital factor in the successful outcome of HAART therapy, adherence-focused treatment represents a teaching opportunity for practitioners involved in the care and management of persons who are HIV positive. Scores of articles have been published regarding nonadherence, and numerous strategies have been employed to encourage adherence. Despite these efforts, medication adherence continues to present problems for patients and health care providers. This article discusses prior and existing adherence interventions for people living with HIV and outlines the rationale related to the development of a structured protocol, the Medication Adherence Training Instrument (MATI), for the evaluation and enhancement of HIV medication adherence by health care providers. The key components to the MATI throughout all sessions are to provide information, enhance motivation, and assess current levels of adherence and HIV-related knowledge.

Adult↗

PubMed2006

The role of family caregivers in HIV medication adherence.

This study examines the role that mid-life and older wives and mothers play in promoting medication adherence among their HIV-infected husbands or adult sons who require daily living assistance. Interviews were conducted with 112 caregiving dyads, with caregivers reporting on their own behaviours and attitudes towards medications, and care-recipients (persons living with HIV [PLH]) providing information about their own adherence practices. By examining how caregiver characteristics, behaviours, and attitudes may influence PLH adherence it is explicitly recognized that caregivers and PLH are linked within a caregiving dyad. Findings indicate that caregivers often remind PLH to take medications, but these reminders are not significantly associated with adherence. Caregivers also report strong attitudes about medication hassles, concerns over treatment failure and general concerns about adherence. Controlling for background characteristics, high perceived adherence hassles on the part of the caregiver were associated with low PLH adherence, providing evidence of shared influence within the caregiving dyad. Adherence interventions may maximize their effectiveness if they consider the role of the family caregiver because these data suggest that caregiver attitudes are linked with PLH adherence behaviours.

Adult↗

PubMed2006

Explaining variability in the relationship between antiretroviral adherence and HIV mutation accumulation.

OBJECTIVES: Determining the relationship between antiretroviral adherence and resistance accumulation is important for the design and evaluation of adherence interventions. Our objective was to explain heterogeneity observed in this relationship. METHODS: We first conducted a systematic review to locate published reports describing the relationship between adherence and resistance. We then used a validated computer simulation to simulate the patient populations in these reports, exploring the impact of changes in individual patient characteristics (age, CD4, viral load, prior antiretroviral experience) on the shape of the adherence-resistance (A-R) curve. RESULTS: The search identified 493 titles, of which 3 contained relevant primary data and 2 had sufficient follow-up for inclusion (HOMER and REACH cohorts). When simulating HOMER, the A-R curve had a high peak with a greatly increased hazard ratio (HR) of accumulating mutations at partial compared to complete adherence (simulation, HR 2.9; HOMER, HR 2.7). When simulating REACH, the A-R curve had a shallow peak with a slightly increased hazard of accumulating mutations at partial adherence (simulation, HR 1.2; REACH, HR 1.4). This heterogeneity was primarily attributable to differences in antiretroviral experience between the cohorts. CONCLUSIONS: Our computer simulation was able to explain much of the heterogeneity in observed A-R curves.

Anti-Retroviral Agents↗

PubMed2001

A randomized, controlled trial of interventions to improve adherence to isoniazid therapy to prevent tuberculosis in injection drug users.

PURPOSE: To determine the effect of several interventions on adherence to tuberculosis preventive therapy. METHODS: We conducted a randomized trial with a factorial design comparing strategies for improving adherence to isoniazid preventive therapy in 300 injection drug users with reactive tuberculin tests and no evidence of active tuberculosis. Patients were assigned to receive directly observed isoniazid preventive therapy twice weekly (Supervised group, n = 99), daily self-administered isoniazid with peer counseling and education (Peer group, n = 101), or routine care (Routine group, n = 100). Patients within each arm were also randomly assigned to receive an immediate or deferred monthly $10 stipend for maintaining adherence. The endpoints of the trial were completing 6 months of treatment, pill-taking as measured by self-report or observation, isoniazid metabolites present in urine, and bottle opening as determined by electronic monitors in a subset of patients. RESULTS: Completion of therapy was 80% for patients in the Supervised group, 78% in the Peer group, and 79% in the Routine group (P = 0.70). Completion was 83% (125 of 150) among patients receiving immediate incentives versus 75% (112 of 150) among patients with deferred incentives (P = 0.09). The proportion of patients who were observed or reported taking at least 80% of their doses was 82% for the Supervised arm of the study, compared with 71% for the Peer arm and 90% for the Routine arm. The proportion of patients who took 100% of doses was 77% for the Supervised arm (by observation), 6% for the Peer arm (by report), and 10% for the Routine arm (by report; P <0.001). Direct observation showed the median proportion of doses taken by the Supervised group was 100%, while electronic monitoring in a subset of patients showed the Peer group (n = 27) took 57% of prescribed doses and the Routine group (n = 32) took 49% (P <0.001). Patients in the Routine arm overreported adherence by twofold when data from electronic monitoring were used as a gold standard. There were no significant differences in electronically monitored adherence by type of incentive. CONCLUSION: Adherence to isoniazid preventive therapy by injection drug users is best with supervised care. Peer counseling improves adherence over routine care, as measured by electronic monitoring of pill caps, and patients receiving peer counseling more accurately reported their adherence. More widespread use of supervised care could contribute to reductions in tuberculosis rates among drug users and possibly other high-risk groups.

Adult↗

PubMed2003

Social support, substance use, and denial in relationship to antiretroviral treatment adherence among HIV-infected persons.

This study examined the relationship of adherence to antiretroviral treatment with three types of social support (partner, friends, and family) and use of two coping strategies (denial and substance use). Participants were 73 men and women with HIV infection drawn from a larger sample of 186 clinical trial patients. Based on inclusion criteria, parent trial participants taking antiretroviral therapies, and those with complete data on self-reported measures of adherence were considered eligible for the present study. Overall, 26% of participants were found to be nonadherent, which was defined as one or more missed doses of treatment in the prior 4-day period. Logistic regression analysis was conducted to determine associations of sociodemographic and psychosocial variables with adherence to antiretroviral regimen. Results indicated that heterosexual participants (p < 0.01) and participants of Latino ethnicity (p < 0.05) were significantly more likely to report missed medications. Perceived satisfaction with support from a partner was associated with taking antiretroviral therapy as prescribed, whereas satisfaction with support from friends and from family was not significantly related to adherence. Examination of coping strategies showed that participants reporting drug and alcohol use (p <.05) to cope with HIV-related stress were more likely to be nonadherent. These findings call for adherence interventions designed to address barriers and strengths, such as community norms or traditional cultural values, specific to certain populations. Furthermore, couple-based approaches enlisting partner support may help persons living with HIV to adhere to antiretroviral regimens.

Adaptation, Psychological↗

PubMed2005

Interventions for improving adherence to treatment recommendations in people with type 2 diabetes mellitus.

BACKGROUND: Research suggests adherence to treatment recommendations is low. In type 2 diabetes, which is a chronic condition slowly leading to serious vascular, nephrologic, neurologic and ophthalmological complications, it can be assumed that enhancing adherence to treatment recommendations may lead to a reduction of complications. Treatment regimens in type 2 diabetes are complicated, encompassing life-style adaptations and medication intake. OBJECTIVES: To assess the effects of interventions for improving adherence to treatment recommendations in people with type 2 diabetes mellitus. SEARCH STRATEGY: Studies were obtained from searches of multiple electronic bibliographic databases supplemented with hand searches of references. Date of last search: November 2002. SELECTION CRITERIA: Randomised controlled and controlled clinical trials, before-after studies and epidemiological studies, assessing changes in adherence to treatment recommendations, as defined in the objectives section, were included. DATA COLLECTION AND ANALYSIS: Two teams of reviewers independently assessed the trials identified for inclusion. Three teams of two reviewers assessed trial quality and extracted data. The analysis for the narrative part was performed by one reviewer (EV), the meta-analysis by two reviewers (EV, JW). MAIN RESULTS: Twentyone studies assessing interventions aiming at improving adherence to treatment recommendations, not to diet or exercise recommendations, in people living with type 2 diabetes in primary care, outpatient settings, community and hospital settings, were included. Outcomes evaluated in these studies were heterogeneous, there was a variety of adherence measurement instruments. Nurse led interventions, home aids, diabetes education, pharmacy led interventions, adaptation of dosing and frequency of medication taking showed a small effect on a variety of outcomes including HbA1c. No data on mortality and morbidity, nor on quality of life could be found. AUTHORS' CONCLUSIONS: Current efforts to improve or to facilitate adherence of people with type 2 diabetes to treatment recommendations do not show significant effects nor harms. The question whether any intervention enhances adherence to treatment recommendations in type 2 diabetes effectively, thus still remains unanswered.

Diabetes Mellitus, Type 2↗

PubMed2004

Effect of brief safer-sex counseling by medical providers to HIV-1 seropositive patients: a multi-clinic assessment.

OBJECTIVE: To test the efficacy of brief, safer-sex counseling by medical providers of HIV-positive patients during medical visits. SETTING: Six HIV clinics in California. DESIGN: Clinics were randomized to intervention arms evaluated with cohorts of randomly selected patients measured before and after the intervention. PARTICIPANTS: Five-hundred and eighty-five HIV-positive persons, sexually active prior to enrollment. INTERVENTIONS: Prevention counseling from medical providers supplemented with written information. Two clinics used a gain-framed approach (positive consequences of safer-sex), two used a loss-frame approach (negative consequences of unsafe sex), and two were attention-control clinics (medication adherence). Interventions were given to all patients who attended the clinics. OUTCOME MEASURE: Self-reported unprotected anal or vaginal intercourse (UAV). RESULTS: Among participants who had two or more sex partners at baseline, UAV was reduced 38% (P < 0.001) among those who received the loss-frame intervention. UAV at follow-up was significantly lower in the loss-frame arm [odds ratio (OR), 0.42; 95% confidence interval (CI), 0.19-0.91; P = 0.03] compared with the control arm. Using generalized estimating equations (GEE) to adjust for clustering did not change the conclusions (OR, 0.34; 95% CI, 0.24-0.49; P = 0.0001). Similar results were obtained in participants with casual partners at baseline. No effects were seen in participants with only one partner or only a main partner at baseline. No significant changes were seen in the gain-frame arm. CONCLUSIONS: Brief provider counseling emphasizing the negative consequences of unsafe sex can reduce HIV transmission behaviors in HIV-positive patients presenting with risky behavioral profiles.

Adult↗

PubMed2026

Multi-centre randomised controlled feasibility trial with embedded process evaluation of a samba percussion intervention for people living with Parkinson's disease: a protocol for the Sparky Samba trial.

INTRODUCTION: Parkinson's disease (PD) is the second most common neurodegenerative disorder, its principal symptom being deterioration of motor function. Current treatment options are limited to symptom management but there is evidence that physical activity can provide motor benefits. More recently there is evidence to suggest that rhythmic auditory stimulation may improve gait and balance in PD. Sparky Samba is a community initiative in South Wales, UK, founded by a person living with PD. Sessions incorporate the following samba rhythms from a trained facilitator and are held weekly in a community setting. METHODS: The Sparky Samba trial is a multi-site, non-blinded, randomised controlled feasibility trial of Sparky Samba compared with activity as usual. A total of 60 people with PD will be randomised 1:1 to take part in a local Sparky Samba group for 12 weeks or continue their normal activities for the same length of time. The primary outcome is feasibility defined by recruitment, retention, data completeness and intervention adherence. Secondary outcomes relating to motor function, cognition, well-being and self-efficacy will also be assessed at baseline and at 12 weeks. Additionally, we will conduct a process evaluation to understand contextual mechanisms surrounding Sparky Samba. This will be achieved through qualitative interviews and structured participant questionnaires following individual trial completion and through structured questionnaires with intervention delivery staff, supplemented with qualitative interviews. ANALYSIS: Feasibility outcomes will be assessed according to pre-defined criteria. For secondary outcomes, means and standard deviations (or medians and IQRs) will be calculated by arm, alongside 95% CIs for change from baseline to 12-week follow-up. Qualitative data will be subject to thematic analysis using NVivo software. ETHICS AND DISSEMINATION: This study received a favourable ethical opinion from the North of Scotland Research Ethics Committee in April 2025 (REC reference 25/NS/0037). Study results will be disseminated through the peer-review literature, the ISRCTN registry and directly to participants, which will be facilitated by the study's public and patient involvement steering group. TRIAL REGISTRATION NUMBER: ISRCTN11861663.

Humans↗

PubMed2006

Moving from theory to research to practice. Implementing an effective dyadic intervention to improve antiretroviral adherence for clinic patients.

There is a dearth of evidence on the relative efficacy of intervention modalities to improve and maintain patient adherence to antiretroviral medications. Although empiric findings from research on HIV/AIDS, other diseases, and chronic medical conditions consistently demonstrate that social support plays an important role in facilitating adherence, few HIV/AIDS interventions have directly targeted this factor. Ewart's social action theory emphasizes the role of social relationships in behavior change and provides a comprehensive and useful guide to the development of interventions for adherence. We describe the development, content, and testing of SMART Couples, an effective antiretroviral adherence intervention that is grounded in social action theory and designed to enhance social support for ART adherence. Finally, we discuss some of the challenges of translating findings from the randomized clinical trial of this intervention into clinical practice and offer recommendations for integration of lessons learned into ongoing clinical care.

Anti-HIV Agents↗

PubMed2005

Daily functioning and quality of life in a randomized controlled trial of therapeutic exercise for subacute stroke survivors.

BACKGROUND AND PURPOSE: The ability of therapeutic exercise after stroke to improve daily functioning and quality of life (QOL) remains controversial. We examined treatment effects on these outcomes in a randomized controlled trial (RCT) of exercise in subacute stroke survivors. METHODS: This is a secondary analysis of a single-blind RCT of a 12-week program versus usual care. Baseline, post-treatment and 6-month post-treatment daily functioning and QOL were assessed by Barthel index, Functional Independence Measure, instrumental activities of daily living, Medical Outcomes Study short-form 36-item questionnaire (SF-36), and Stroke Impact Scale (SIS). RESULTS: Of 100 randomized subjects, 93 completed the postintervention assessment, (mean age 70; 54% male; 81% white; mean Orpington Prognostic Score 3.4), and 80 had 6-month post-treatment assessment. Immediately after intervention, the intervention group improved more than usual care in SF-36 social function (14.0 points; P=0.0051) and in SIS (strength [9.2 points; P=0.0003], emotion [5.6 points; P=0.0240], social participation [6.6 points; P=0.0488], and physical function [5.0 points; P=0.0145]). Treatment was marginally more effective on Barthel score (3.3 points; P=0.0510), SF-36 (physical function [6.8 points; P=0.0586], physical role function [14.4 points; P=0.0708]), and SIS upper extremity function (7.2 points; P=0.0790). Effects were diluted 6 months after treatment ended. CONCLUSIONS: This rehabilitation exercise program led to more rapid improvement in aspects of physical, social, and role function than usual care in persons with subacute stroke. Adherence interventions to promote continued exercise after treatment might be needed to continue benefit.

Activities of Daily Living↗

PubMed1996

The Prevention for Elderly Persons (PEP) Program: a model of municipal and academic partnership to meet the needs of older persons for preventive services.

OBJECTIVE: To create a program to identify preventive needs for community-dwelling older persons and incorporate intervention strategies to improve implementation of these services. DESIGN: Program development and case-series. SETTING: Community-based meal sites, academically administered program. PARTICIPANTS: Persons 60 years of age or older attending meal sites and their primary care physicians. MEASUREMENTS: Demographic characteristics, self reported preventive health behaviors and services, blood pressure measurement. RESULTS: During the first 2 years of the program, 927 persons 60 years of age or older were screened. The most common physician-initiated preventive recommendations were: tetanus booster (72%), aspirin prophylaxis (68%), pneumonia vaccination (61%), and colorectal cancer screening (51%). The most common self-care recommendations have been: calcium supplementation (54% of women) and breast self examinations (51% of women). As part of the adherence intervention, we were able to complete health educator calls for 600 (65%) subjects. In addition, the physicians of 599 (65%) subjects were contacted either by telephone (n = 496) or by letter only (n = 97). CONCLUSION: A community based preventive services program can identify large numbers of unmet preventive services needs, and a dual intervention strategy aimed at meeting these needs can be delivered successfully to the majority of participants. Implementation rates of specific recommendations and impacts on health outcomes remain to be determined.

Academic Medical Centers↗

PubMed2006

Agalsidase-beta therapy for advanced Fabry disease: a randomized trial.

BACKGROUND: Fabry disease (alpha-galactosidase A deficiency) is a rare, X-linked lysosomal storage disorder that can cause early death from renal, cardiac, and cerebrovascular involvement. OBJECTIVE: To see whether agalsidase beta delays the onset of a composite clinical outcome of renal, cardiovascular, and cerebrovascular events and death in patients with advanced Fabry disease. DESIGN: Randomized (2:1 treatment-to-placebo randomization), double-blind, placebo-controlled trial. SETTING: 41 referral centers in 9 countries. PATIENTS: 82 adults with mild to moderate kidney disease; 74 of whom were protocol-adherent. INTERVENTION: Intravenous infusion of agalsidase beta (1 mg per kg of body weight) or placebo every 2 weeks for up to 35 months (median, 18.5 months). MEASUREMENTS: The primary end point was the time to first clinical event (renal, cardiac, or cerebrovascular event or death). Six patients withdrew before reaching an end point: 3 to receive commercial therapy and 3 due to positive or inconclusive serum IgE or skin test results. Three patients assigned to agalsidase beta elected to transition to open-label treatment before reaching an end point. RESULTS: Thirteen (42%) of the 31 patients in the placebo group and 14 (27%) of the 51 patients in the agalsidase-beta group experienced clinical events. Primary intention-to-treat analysis that adjusted for an imbalance in baseline proteinuria showed that, compared with placebo, agalsidase beta delayed the time to first clinical event (hazard ratio, 0.47 [95% CI, 0.21 to 1.03]; P = 0.06). Secondary analyses of protocol-adherent patients showed similar results (hazard ratio, 0.39 [CI, 0.16 to 0.93]; P = 0.034). Ancillary subgroup analyses found larger treatment effects in patients with baseline estimated glomerular filtration rates greater than 55 mL/min per 1.73 m2 (hazard ratio, 0.19 [CI, 0.05 to 0.82]; P = 0.025) compared with 55 mL/min per 1.73 m2 or less (hazard ratio, 0.85 [CI, 0.32 to 2.3]; P = 0.75) (formal test for interaction, P = 0.09). Most treatment-related adverse events were mild or moderate infusion-associated reactions, reported by 55% of patients in the agalsidase-beta group and 23% of patients in the placebo group. LIMITATIONS: The study sample was small. Only one third of the patients experienced clinical events, and some patients withdrew before experiencing any event. CONCLUSIONS: Agalsidase-beta therapy slowed progression to the composite clinical outcome of renal, cardiac, and cerebrovascular complications and death compared with placebo in patients with advanced Fabry disease. Therapeutic intervention before irreversible organ damage may provide greater clinical benefit.

Adult↗