scholarly journals The role of patient preferences in adherence to treatment in chronic disease: a narrative review

2021 ◽  
Vol 15 ◽  
pp. 13-20
Author(s):  
Serena Losi ◽  
Cesare Celeste Federico Berra ◽  
Riccardo Fornengo ◽  
Dario Pitocco ◽  
Giovanni Biricolti ◽  
...  

Adherence to prescribed medication is important to the management of all diseases, especially those of chronic nature. Drug effectiveness is substantially compromised by therapy nonadherence. We reviewed the available evidences on the impact of patient preferences for therapy on adherence to a prescribed treatment in chronic diseases requiring long-term treatment. A search on PubMed retrieved 699 publications, leading to a selection of 12 publications: 6 on osteoporosis, 2 on moderate-to-severe asthma, 1 on type 1 diabetes, 1 on type 2 diabetes, 1 on kidney transplantation, and 1 on atrial fibrillation. Overall, 8 studies found a positive association between patient preference and adherence to therapy, while the others found no association. In general, overall adherence was considered to be high in the published studies. The reasons for a positive association included reduced dosing frequency, route of administration, lower costs, and favorable safety profile, which is related to the diverse nature of the pathology and its type and duration of treatment. A literature review suggests that achieving good adherence and persistence to therapy requires evaluation of patient preferences. In a period of increasingly limited resources, more effort is warranted to promote better adherence to therapy, especially when patients must self-manage their disease in the long term. Our results further highlight that insufficient attention has been given to the relationship between patient preference and adherence and point out the complex nature of adherence and the need for adequate patient education. More efforts are also needed to better understand the entity of cost savings for payers for specific treatments and the link with patient preference.

2009 ◽  
Vol 101 (04) ◽  
pp. 674-681 ◽  
Author(s):  
Massimo Franchini ◽  
Annarita Tagliaferri ◽  
Antonio Coppola

SummaryA four-decade clinical experience and recent evidence from randomised controlled studies definitively recognised primary prophylaxis, i.e. the regular infusion of factor concentrates started after the first haemarthrosis and/or before the age of two years, as the first-choice treatment in children with severe haemophilia. The available data clearly show that preventing bleeding since an early age enables to avoid or reduce the clinical impact of muscle-skeletal impairment from haemophilic arthropathy and the related consequences in psycho-social development and quality of life of these patients. In this respect, the aim of secondary prophylaxis, defined as regular long-term treatment started after the age of two years or after two or more joint bleeds, is to avoid (or delay) the progression of arthropathy. The clinical benefits of secondary prophylaxis have been less extensively studied, especially in adolescents and adults; also in the latter better outcomes and quality of life for earlier treatment have been reported. This review summarises evidence from literature and current clinical strategies for prophylactic treatment in patients with severe haemophilia, also focusing on challenges and open issues (optimal regimen and implementation, duration of treatment, long-term adherence and outcomes, cost-benefit ratios) in this setting.


2004 ◽  
Vol 16 (6) ◽  
pp. 319-325 ◽  
Author(s):  
Pierre S. Chue ◽  
Peter D'Hoore ◽  
J. Michael Ramstack

Chronic disorders such as schizophrenia require long-term treatment programs in order to maintain patients at the lowest level of symptomatology, reduce the likelihood of psychotic relapse, and support achievement of remission and recovery. Evidence suggests that treatment with long-acting injectable antipsychotics reduces the impact of partial compliance and provides predictable release of medication, assuring continuous therapeutic coverage. Until recently, only conventional antipsychotic agents were available in long-acting formulations, thereby foregoing the advantages of the atypical class. Atypical agents which are given orally have been shown to provide long-term efficacy and tolerability benefits compared with conventional agents, but are limited by the need for daily administration. The most recent pharmacological strategy to achieve optimal maintenance treatment has been to combine the benefits of an atypical antipsychotic with delivery in a water-based long-acting formulation. The first antipsychotic to achieve this combination – long-acting risperidone – may thus represent an important advance in the optimization of long-term treatment outcomes in patients with schizophrenia.


2021 ◽  
Vol 34 (Supplement_1) ◽  
Author(s):  
Kathryn Peterson ◽  
Mirna Chehade ◽  
Joseph Murray ◽  
Gary Falk ◽  
Nirmala Gonsalves ◽  
...  

Abstract   Eosinophilic esophagitis (EoE), gastritis (EG), and/or duodenitis (EoD) are associated with accumulation and activation of eosinophils and mast cells in the esophagus, stomach, and/or duodenum, respectively. Lirentelimab (AK002), an antibody against siglec-8, depletes eosinophils and inhibits mast cells. We performed an open-label extension (OLE) study of subjects who completed ENIGMA (a randomized, double-blind, placebo-controlled phase 2 study of lirentelimab in adults with symptomatic, biopsy-confirmed EG and/or EoD, with or without EoE) to evaluate long-term responses. Methods Subjects who received 4 monthly infusions of lirentelimab or placebo during ENIGMA (n = 59) were eligible for the OLE; they received monthly, escalating doses of lirentelimab (0.3 or 1 mg/kg escalating to 3 mg/kg). Symptoms were assessed weekly using an electronic daily patient-reported outcome questionnaire and total symptom scores (TSS) were calculated. Patients underwent upper endoscopy with biopsy at screening and at the end of ENIGMA (day 99, week 16, blinded); in the OLE, endoscopies were performed on day 323 (30 weeks after the first dose in the OLE). Histopathology was assessed by a single pathologist. Results Fifty-eight subjects entered the OLE; 45 completed ≥52 weeks lirentelimab (including exposure during ENIGMA) and 29 completed 70 weeks. Mean TSS improved through week 70 (Figure 1). Subjects receiving 70 weeks lirentelimab (ENIGMA+OLE) had further improvements in TSS from baseline (mean reductions: 68% at weeks 29–30, 70% at weeks 51–52, 75% at weeks 69–70). Symptom scores (abdominal pain, nausea, vomiting, early satiety, appetite loss, abdominal cramping, bloating, diarrhea) decreased significantly from baseline. Treatment response was not associated with concomitant EoE. The most common adverse event was mild to moderate infusion-related reactions, usually with the first infusion. Conclusion In the OLE of the ENIGMA study, patients with EG and or EoD (with or without concomitant EoE) who received lirentelimab had sustained tissue eosinophil depletion and significant long-term symptom improvement. Symptoms continued to improve with duration of treatment. Lirentelimab appears to be a promising targeted treatment for EG and/or EoD.


2021 ◽  
Vol 79 (3) ◽  
pp. 229-232
Author(s):  
Ana Beatriz Ayroza Galvão Ribeiro GOMES ◽  
Milena Sales PITOMBEIRA ◽  
Douglas Kazutoshi SATO ◽  
Dagoberto CALLEGARO ◽  
Samira Luisa APÓSTOLOS-PEREIRA

ABSTRACT Background: Azathioprine is a common first-line therapy for neuromyelitis optica spectrum disorder (NMOSD). Objective: The aim of this study was to determine whether long-term treatment (>10 years) with azathioprine is safe in NMOSD. Methods: We conducted a retrospective medical record review of all patients at the School of Medicine of the University of São Paulo (São Paulo, Brazil) who fulfilled the 2015 international consensus diagnostic criteria for NMOSD and were treated with azathioprine for at least 10 years. Results: Out of 375 patients assessed for eligibility, 19 were included in this analysis. These patients’ median age was 44 years (range=28-61); they were mostly female (17/19) and AQP4-IgG seropositive (18/19). The median disease duration was 15 years (range=10-39) and most patients presented a relapsing clinical course (84.2%). The median duration of treatment was 11.9 years (range=10.0-23.8). The median annualized relapse rates (ARR) pre- and post-treatment with azathioprine were 1 (range=0.1-2) and 0.1 (range=0-0.35); p=0.09. Three patients (15.7%) had records of adverse events during the follow-up, which consisted of chronic B12 vitamin deficiency, pulmonary tuberculosis and breast cancer. Conclusion: Azathioprine may be considered a safe agent for long-term treatment (>10 years) of NMOSD, but continuous vigilance for infections and malignancies is required.


Spine ◽  
2006 ◽  
Vol 31 (26) ◽  
pp. 3061-3069 ◽  
Author(s):  
Steven J. Atlas ◽  
Yuchiao Chang ◽  
Robert B. Keller ◽  
Daniel E. Singer ◽  
Yen A. Wu ◽  
...  

2021 ◽  
Author(s):  
Ahmed Abdelkhalek ◽  
Govindavilas Sudhesh ◽  
Anjan Sarkar ◽  
Mohammed Eissa

Abstract Structural bearings of 47 offshore platform-link bridges with average age of 40 years were inspected and recommended for replacements due to their poor condition. Replacement of bridge bearings involves major risk and production interruptions given the structural modifications, and critical piping and E&I disconnections required for safe jacking-lifting activities required during the process. This paper presents the approach adopted to assure the integrity of the bridges and extend their lives without the need to replace the bearings. The approach employed failure mode and effect analysis to identifying and narrowing down areas that need focused efforts while tackling the problem. Scenario based structural assessments were carried out to examine the impact of the level of movement-allowing bearings functionality on the integrity of the bridge and its supporting structures; identify critical locations to be targeted during focused inspections; and establish envelopes for monitoring thermal expansion and contraction of the bridges. Guidelines were developed and implemented for integrated inspection-maintenance and repair campaign, which aimed to tackle corrosion issues and to install movement-monitoring indicators. Indicator seasonal monitoring is employed to establish the functionality of bearings on the long-term. The what-if structural assessments revealed that even in the worst-case scenario (in which the bearing are completely jammed) the option of local strengthening of the bridge and its supporting elements is more attractive than bearing replacement. The integrated inspection-maintenance and repair campaigns revealed that excessive corrosion levels observed from historic visual inspections on external non-critical bearing components (e.g: guide plates, angles, etc.) is not indicative of the condition of the internal load-bearing components (pedestals) which experienced much lower corrosion levels. The seasonal monitoring of bridge movements revealed that the 40+ years old Teflon pads are still functional and allow the bridges expansion and contraction. The developed holistic approach enabled demonstration of the fitness for service of the bearings, and provided means for assuring their long-term performance through monitoring. The results assured safety, integrity and delivered significant cost savings through aversion brownfield modifications, and production loss associated with bridge jacking and bearing replacement operations.


2018 ◽  
Vol 4 (Supplement 3) ◽  
pp. 15s-15s
Author(s):  
Sefonias Getachew ◽  
Adamu Addisse ◽  
Lesley Taylor ◽  
Eva J. Kantelhardt

Purpose The majority of women with breast cancer from low-income countries, including Ethiopia, present with advanced clinical stage disease, which results in limited and difficult therapeutic options and high mortality rates. In Ethiopia, breast cancer is the most common cancer. We found that 70% of breast cancer cases in Ethiopia are hormone receptor positive. Endocrine therapy is one of the treatment options recommended for breast cancer but that is highly underutilized in the country. Recommendations on interventions to improve uptake and adherence to therapy exist, but studies that have assessed the feasibility of implementing these are limited. Our study (n = 107) in rural Ethiopia revealed an estimated 53% 2-year survival rate in patients who underwent surgery only. In our pilot study, of 51 eligible patients 26 initiated therapy and one half of those adhered after 1 year. Our study aims to evaluate the effectiveness of using a trained breast nurse navigator to improve patient adherence to tamoxifen therapy among patients with breast cancer in rural Ethiopia. Methods A cluster randomized intervention trial is being carried out in rural hospitals in southwestern Ethiopia from February 2018 to June 2019. We use hospitals in clusters as the units of randomization. The sample size includes four per intervention arm and control arm, with each cluster comprised of approximately 15 patients. Before intervention, all patients in the hospitals will receive tamoxifen therapy free of charge. Hormone receptor status of the breast cancer specimen will be determined before the initiation of therapy or throughout the course of therapy. The primary outcome of this trial is adherence to endocrine therapy on the basis of objective and subjective measures. Data will be collected with a prospective repeated measures approach. Analysis will be based on an intention-to-treat principle. Results The trial aims to provide evidence on the effectiveness of the breast nurse intervention to improve adherence to long-term endocrine therapy and answer the following research question: does the nursing intervention improve long-term treatment adherence by patients to endocrine therapy compared with those who receive usual care services? Conclusion These data are essential to maximize the impact of trained nurse-based interventions on adherence to endocrine (tamoxifen) therapy among patients with breast cancer on follow-up. AUTHORS' DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST The following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated. Relationships are self-held unless noted. I = Immediate Family Member, Inst = My Institution. Relationships may not relate to the subject matter of this manuscript. For more information about ASCO's conflict of interest policy, please refer to www.asco.org/rwc or ascopubs.org/jco/site/ifc . Eva J. Kantelhardt Travel, Accommodations, Expenses: Daiichi Sankyo Oncology Europe


2018 ◽  
Vol 23 (suppl_1) ◽  
pp. e35-e35
Author(s):  
Jean-Francois Lemay ◽  
Julie-Anne Lemay ◽  
Hanna Kubas

Abstract BACKGROUND Attention-deficit/hyperactivity disorder (ADHD) is a neurodevelopmental disorder that often negatively impacts behaviour, cognition, and learning. Stimulant medications are the most commonly used treatment for ADHD, with informant reports (e.g., parent questionnaires, surveys) frequently used to evaluate medication impact on behavioural and academic functioning in affected children. OBJECTIVES To determine parental perceptions of medication impact on behaviour and learning in a long-acting methylphenidate (LA-MPH) trial of their children with ADHD. DESIGN/METHODS A randomized controlled LA-MPH medication trial was conducted with children ages 8–12 with a diagnosis of ADHD. Trial began with one-week of baseline assessment, followed by a randomized three-week standard of care medication trial, and a one-week best dose assessment. Following the conclusion of the study (6 months to 2 years’ range post-treatment), families were asked to participate in a voluntary follow-up phone survey to evaluate parental perceptions on child’s behaviour and learning. RESULTS A total of 34/42 (81%) families participated (male to female ratio: 2.1/1). At the time of the follow-up survey, 53% (18/34) and 68% (23/34) of patients were having “difficulty” or “significant difficulty” with their behaviour and learning, respectively. Twenty-three patients (68%) were still on psycho-stimulant medications. Although parents of those 23 children said LA-MPH had in general “significant” or “very significant” impact on their child’s behaviour (87%) and learning (79%), these parents were still reporting challenges with behaviour (52%) and learning (61%) at follow-up. In addition, parents of children not on medication said that their current child’s behaviour and learning was still having the same or more challenging issues (82% and 73% respectively). CONCLUSION Overall, parents reported that medication significantly impacted their child’s behaviour and learning; however, long-term medication impact appears less effective. Thus, an ongoing relationship with families and paediatricians is recommended to better understand the impact of medication on behaviour and learning. Evaluating the effects of medication on behaviour and learning may ultimately lead to targeted intervention that help foster long-term treatment efficacy for children with ADHD.


2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S573-S573
Author(s):  
M A Martínez Ibeas ◽  
I Bacelo Ruano ◽  
S Rodríguez Manchón ◽  
M Velasco Rodríguez-Belvís ◽  
J F Viada Bris ◽  
...  

Abstract Background The toxicity of azathioprine (AZA) includes myelosuppression, infections, pancreatitis, photosensitivity, and hepatotoxicity. The aim of this study was to describe the adverse effects profile of azathioprine as long-term treatment in paediatric inflammatory bowel disease (IBD). Methods An observational, descriptive and retrospective study was performed in the paediatric IBD Unit of a tertiary care hospital from September 2008 to December 2018. It was included patients under 18 diagnosed with IBD who were treated with AZA during their follow-up. We recorded epidemiological data, thiopurine methyltransferase (TPMT) enzyme activity, AZA side effects, and the dosage the patients were receiving when these effects took place. Bone marrow suppression (BMS) was defined as leukopenia, thrombocytopenia and/or anaemia. Acute pancreatitis (AP) induced by azathioprine was considered when two of these criteria (Atlanta 2012) were met: lipase increase (> 3 times normal value), congruent signs and symptoms and/or echographic findings, without other possible aetiology and with complete recovery after AZA withdrawal. Results We included 52 patients, being 31 men (59.6%). They were diagnosed with Crohn′s disease (CD) (73%), ulcerative colitis (UC) (21%) and IBD-unclassified (6%). The median TPMT activity was 17 U/ml (14.2–19.2). Up to 63.5% developed adverse effects by AZA with a median time from the beginning of treatment of 11.4 months (2.6–26.4) and a median dosage of 2 mg/kg/day (1.7–2.3). The most frequent side effect was BMS (52%). These patients had a median TPMT activity of 16.9 U/ml (14.2–18.9), the median duration of treatment was 14 months (3.9–27.7), and the median dosage was 2 mg/kg/d (1.8–2.5). BMS was more frequent in patients with UC (p 0.04) and longer treatment (p 0.08). No differences were found according to age, sex or TPMT activity. Up to 11.5% developed AP, the median duration of treatment until its appearance was 1.5 months (0.7–43.3) and the median dosage was 2 mg/kg/d (1.5–2.5). No differences were found related to age, sex, diagnosis or dosage. Other side effects were: 3 flu-like symptoms, 3 opportunistic infections, 2 hypertransaminasemia, and 1 patient with elevated pancreatic enzymes and hyperbilirubinemia. AZA was discontinued in 14 patients (43.8%): in 6 due to AP, in 4 due to severe lymphopenia, in 2 because of Epstein-Barr virus infection, in 1 due to flu-like symptoms and in 1 with several adverse effects. Conclusion More than half of the patients treated with AZA presented side effects, mainly BMS, although most of them were mild and temporary, and the withdrawal of the drug was not necessary. It seems that TPMT activity is not useful to predict BMS, but this adverse effect could be related to a longer treatment.


Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 402-402 ◽  
Author(s):  
David J Kuter ◽  
James B Bussel ◽  
Adrian Newland ◽  
Joost TM de Wolf ◽  
Troy Guthrie ◽  
...  

Abstract Chronic ITP is characterized by increased platelet destruction and suboptimal platelet production. Romiplostim is an investigational Fc-peptide fusion protein (peptibody) being studied for its ability to increase platelet counts in patients with chronic ITP. We report data from an open-label extension study of romiplostim in adult patients with chronic ITP. Collection of safety and efficacy data from long-term treatment of these patients is ongoing. Eligible patients had completed a prior romiplostim study and had platelet counts □50×109/L. Romiplostim was administered subcutaneously once weekly with dose adjustments to maintain a platelet count of 50–250×109/L. As of July 13 2007, 142 patients had been treated with romiplostim. Their median time since diagnosis was 6.4 years (range 0.6–46.4 years). Most were female (67%) and had previously undergone a splenectomy (60%). The median baseline platelet count was 17×109/L (range 1–50×109/L). The median duration of treatment was 65 weeks (range 1–156 weeks). Twenty-nine (20%) patients discontinued the study, 10 (7%) due to adverse events (AEs) [2 each of bone marrow reticulin and thrombosis; 1 each of bleeding, pain, cardiac arrest, pneumonia, hepatic and renal failure, and monoclonal gammopathy of undetermined significance]. Different measures of platelet count response were analyzed; any platelet counts within 8 weeks of receiving rescue medications were excluded from these analyses. Platelet counts were increased from baseline by ≥20×109/L more than 80% of the time in 54% of patients and more than 50% of the time in 73% of patients. Platelet counts remained above 20×109/L more than 90% of the time in 67% of patients and more than 50% of the time in 94% of patients. A platelet count >50×109/L and double baseline was achieved by 30% (42/138) of patients after the first dose, by 51% (71/138) of patients after the third dose, and by 87% (124/142) of patients overall. The durability of platelet count increases was analyzed: platelet counts >50×109/L were sustained for ≥10, ≥25, and ≥52 consecutive weeks in 78% (102/131), 54% (66/122), and 35% (29/84) of patients, respectively. The patient incidence of bleeding events both of any severity and of clinical significance (≥Grade 3) declined over time (Table). AEs were reported in 95% of patients, with most mild to moderate in severity. The most common were headache (37%); nasopharyngitis (32%); and contusion, fatigue and epistaxis (each 30%). AE frequency did not increase with time on study (Table). Bone marrow reticulin was present or increased in 8 patients with no evidence of progression to collagen fibrosis or chronic idiopathic myelofibrosis. Thrombotic events were reported in 7 (5%) patients; 6 had pre-existing risk factors for thrombosis. In conclusion, romiplostim increased platelet counts in most patients for most of the time, and clinically relevant bleeding was reduced over time. Romiplostim was well-tolerated and AEs did not increase with longer duration of treatment. Table. Summary of patient incidence of AEs by study period <24 wks (N=142) n (%) 24 to <48 wks (N=126) n (%) 48 to <72 wks (N=97) n (%) 72 to <96 wks (N=65) n (%) 96 to <120 wks (N=29) n (%) 120 to <144 wks (N=25) n (%) AEs 129 (91) 110 (87) 64 (66) 36 (55) 23 (79) 21 (84) Serious AEs 25 (18) 13 (10) 7 (7) 4 (6) 4 (14) 1 (4) Treatment-related AEs 48 (34) 14 (11) 12 (12) 7 (11) 4 (14) 3 (12) Treatment-related serious AEs 6 (4) 3 (2) 1 (1) 2 (3) 1 (3) 1 (4) Study withdrawals due to AEs 4 (3) 5 (4) 0 (0) 0 (0) 0 (0) 1 (4) Bleeding any grade 60 (42) 37 (29) 22 (23) 13 (20) 11 (38) 8 (32) Bleeding ≥ Grade 2 (moderate) 25 (18) 12 (10) 8 (8) 4 (6) 3 (10) 2 (8) Bleeding ≥ Grade 3 difference in thisresponsebetween refractory (severe) 9 (6) 1 (1) 1 (1) 1 (2) 0 (0) 0 (0)


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