Diana Rofail

Patient-Reported Outcomes of Deferasirox (Exjade®, ICL670) versus Deferoxamine in Sickle Cell Disease Patients with Transfusional Hemosiderosis

Background/Aims: There is increasing evidence demonstrating the value of transfusions in sickle cell disease (SCD). However, resultant iron overload can be life threatening if untreated. Chelation therapy with deferoxamine requires parenteral infusions that can negatively impact quality of life and adherence to treatment. Methods: As part of a phase II trial, SCD patient-reported outcomes were evaluated. One hundred and ninety-five patients were randomized (2:1) to receive oral deferasirox (5–30 mg/kg/day) or deferoxamine (20–50 mg/kg, 5 days per week); 121 had previously received deferoxamine. Results: At each time point, significantly more patients who had previously received deferoxamine were ‘satisfied/very satisfied’ with deferasirox, or found treatment to be ‘convenient/very convenient’ compared with deferoxamine (p < 0.001). In these patients, fewer hours were lost from daily activities with deferasirox than deferoxamine treatment. Most patients (77%) preferred deferasirox, and more were willing to continue taking deferasirox than deferoxamine at end-of-study (84 vs. 11%, respectively). Conclusions: Patients with SCD are therefore more satisfied with deferasirox, which has a lower impact on daily activities than deferoxamine. Given the high levels of satisfaction, it is likely that quality of life will be improved. These results also suggest that treatment adherence with deferasirox may be better than with deferoxamine, which should lead to improved long-term outcomes.

Iron chelation therapy: clinical effectiveness, economic burden and quality of life in patients with iron overload.

IntroductionThis study of UK patients examines clinical, healthrelated quality of life (HRQOL) and economic outcomes associated with iron chelation therapy (ICT). Desferrioxamine (DFO) (Desferal®; Novartis, Switzerland) and Deferiprone (Ferriprox®; Apotex, Canada) are ICTs used to treat iron overload. DFO requires 8-to 12-hour infusions a minimum of five times per week. Deferiprone is administered in an oral daily regimen. Although pharmacologically efficacious, clinical effectiveness of ICT within the real-world setting is yet to be fully elucidated.MethodsA naturalistic cohort study of 60 patients (betathalassaemia, n=40; sickle cell disease, n=14; myelodysplastic syndromes, n=6; 63% female) receiving ICT in four UK treatment centres was conducted. Serum ferritin level data were abstracted from medical charts. Compliance, HRQOL, satisfaction and resource utilisation data were collected from interviews. Maximum ICT costs were estimated using the resource utilisation data associated with DFO.ResultsMean serum ferritin levels, generally, remained elevated despite ICT. Compliance was suboptimal and HRQOL scores were lower than population norms. The total estimated mean weighted annual per-patient cost of DFO treatment was approximately £19,000. DFO-related equipment, DFO drug, and home healthcare were estimated to account for 43%, 19% and 24% of costs, respectively. Other more minor components of total annual costs were for in-patient infusions, ICT home delivery services and monitoring costs.ConclusionGenerally, patients are not achieving target serum ferritin thresholds despite chronic treatment for iron overload. ICT appears to negatively impact HRQOL; compliance with ICT is poor; and, in the case of DFO, treatment costs well exceed the cost of DFO alone. These results suggest that current ICT in the real-world setting is suboptimal with respect to various clinical, HRQOL and economic outcomes.

Clinical and economic burden of infused iron chelation therapy in the United States

BACKGROUND: Patients requiring chronic blood transfusions are at risk for iron overload, which, if not treated by iron chelation therapy (ICT), can create serious organ damage and reduce life expectancy. Current ICT requires burdensome 8‐ to 12‐hour infusions five to seven times per week.

Improved Treatment Satisfaction and Convenience with Deferasirox in Iron-Overloaded Patients with β-Thalassemia: Results from the ESCALATOR Trial

Patient-reported outcomes of once-daily oral deferasirox (Exjade®) in iron-overloaded patients with β-thalassemia not achieving successful chelation with prior deferoxamine and/or deferiprone were investigated in a prospective, open-label, 1-year, multicenter study in the Middle East (ESCALATOR). The initial dose of deferasirox was 20 mg/kg/day, with subsequent dose adjustments. At baseline and the end of study (EOS), patients (n = 237) completed a 5-point rating scale for treatment satisfaction and convenience, and recorded time lost to treatment. At EOS, 90.7% of patients were ‘satisfied’/‘very satisfied’ with their iron chelation therapy (ICT) versus 23.2% at baseline. 92.8% (EOS) versus 21.5% (baseline) of patients considered their therapy to be ‘convenient’/‘very convenient’. Time lost to therapy for daily activities was substantially reduced (3.2 ± 8.6 [mean ± SD; EOS] vs. 30.1 ± 44.2 [baseline] h/month). Patients reported greater satisfaction and convenience, and lower impact on daily activities, with deferasirox than with previous ICT. This may help improve adherence to lifelong ICT in iron-overloaded β-thalassemia patients.

Hereditary hemochromatosis: patient experiences of the disease and phlebotomy treatment

BACKGROUND: Hereditary hemochromatosis (HH) is a genetic disorder resulting in increased accumulation of dietary iron. It is associated with various clinical complications such as liver cirrhosis and diabetes. The aim of this study was to explore patients’ experiences of living with HH, the diagnosis process, and phlebotomy treatment.

Assessing the Impact of Caring for a Person with Schizophrenia: Development of the Schizophrenia Caregiver Questionnaire

AbstractBackgroundThe responsibilities of caring for a person with schizophrenia may significantly impact informal caregivers’ lives. The Zarit Burden Interview (ZBI) was originally developed to assess burden among caregivers of people with Alzheimer’s disease.ObjectiveThis research was conducted to inform the development of a revised version of the ZBI, relevant to caregivers of people with schizophrenia.MethodsBased on published qualitative research, the questionnaire was reviewed and modified in accordance with industry-standard guidelines. The resulting questionnaire [the Schizophrenia Caregiver Questionnaire (SCQ)] was then completed by 19 caregivers during cognitive debriefing interviews to assess understanding, relevance and comprehensiveness.ResultsReview of the ZBI resulted in a number of operational changes to improve face validity and potential sensitivity. Further questions were added based on key concepts identified in existing literature and minor phrasing alterations were made to improve content validity. Findings from caregiver interviews supported the content validity of the SCQ.ConclusionThe SCQ provides a comprehensive view of caregivers’ subjective experiences of caregiving and demonstrated strong face and content validity. The questionnaire will be important in both clinical assessment and evaluating the efficacy of interventions designed to reduce or alleviate caregiver burden. Future research will seek to establish the psychometric validity of the questionnaire.

Assessing the impact on caregivers of patients with schizophrenia: psychometric validation of the Schizophrenia Caregiver Questionnaire (SCQ)

BackgroundThe Schizophrenia Caregiver Questionnaire (SCQ) was developed to assess the impact on caregivers of caring for patients with schizophrenia. The objective of this study was to develop a scoring algorithm for the SCQ, and evaluate its measurement properties.MethodsThe SCQ was administered to 358 caregivers of patients with schizophrenia included in the observational PATTERN study of stabilized patients with persistent symptoms of schizophrenia receiving outpatient care. SCQ item selection and creation of scores were based on exploration of item response distribution, factor analyses, and Rasch model. Construct validity, reliability, and ability to detect change of the SCQ scores were investigated.ResultsThe final questionnaire comprised a ‘Humanistic impact’ supra-domain composed of a global score and four subdomain scores (‘Physical’; ‘Emotional’; ‘Social’; ‘Daily life’), and eight other domain scores related to the caregiving role (‘Exhaustion with caregiving’; ‘Feeling alone’; ‘Patient Dependence’; ‘Worries for the patient’; ‘Perception of caregiving’; ‘Financial dependence of the patient’; ‘Financial impact of caregiving’; ‘Overall difficulty of caregiving’). Two items from the SCQ were deleted. SCQ scores showed very good construct validity: Item convergent/discriminant validity were satisfactory; SCQ scores of caregivers of patients with more severe symptoms were higher indicating more impact (p < 0.05 for all scores); SCQ scores were meaningfully associated with measures of schizophrenia severity (PANSS and PSP) and caregivers’ Health-Related Quality of Life (Medical Outcome Survey Short Form 36 items). The SCQ Humanistic impact supra-domain scores demonstrated very good internal consistency reliability (Cronbach’s alphas between 0.80 and 0.96) and test-retest reliability (Intraclass Coefficient correlations ranging from 0.75 and 0.87); Other SCQ domain scores showed lower but still acceptable reliability coefficients. SCQ scores clearly increased for caregivers of patients whose schizophrenia worsened.ConclusionsOverall, the 30-item SCQ demonstrated very good measurement properties supporting its relevance to comprehensively measure the experience of caregivers of patients with schizophrenia.

The feasibility of using electronic clinical outcome assessments in people with schizophrenia and their informal caregivers

Many clinical outcome assessments (COAs) were originally developed for completion via pen and paper. However, in recent years there have been movements toward electronic capture of such data in an effort to reduce missing data, provide time-stamped records, minimize administrative burden, and avoid secondary data entry errors. Although established in many patient populations, the implications of using electronic COAs in schizophrenia are unknown. In accordance with International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force recommendations, in-depth cognitive debriefing and usability interviews were conducted with people with schizophrenia (n=12), their informal (unpaid) caregivers (n=12), and research support staff (n=6) to assess the suitability of administration of various electronic COA measures using an electronic tablet device. Minimal issues were encountered by participants when completing or administering the COAs in electronic format, with many finding it easier to complete instruments in this mode than by pen and paper. The majority of issues reported were specific to the device functionality rather than the electronic mode of administration. Findings support data collection via electronic tablet in people with schizophrenia and their caregivers. The appropriateness of other forms of electronic data capture (eg, smartphones, interactive voice response systems, etc) is a topic for future investigation.

Treatment Satisfaction and Dissatisfaction in Chronic Low Back Pain: a Systematic Review

Background: This paper documents a systematic review of treatment satisfaction and dissatisfaction in patients with Chronic Low Back Pain (CLBP). Research shows that treatment satisfaction is a strong indicator of adherence to treatment regimens; hence the relevant need for this systematic review of current literature. Objectives: This paper aims to: i) explore patient satisfaction and dissatisfaction with treatments for CLBP; ii) establish definitions of treatment satisfaction and dissatisfaction in CLBP; iii) provide an overview of questionnaires used to measure the concepts; iv) establish the quality of studies reviewed; v) determine the level of patient satisfaction or dissatisfaction with treatments in CLBP; and vi) identify factors associated with treatment satisfaction or dissatisfaction in CLBP. Methods: A systematic review of scientific papers in the PubMed, PsycINFO, Embase, CINAHL and Web of Knowledge electronic databases was undertaken in combination with hand searches in the journals of Pain, Physiotherapy and Spine. The review was limited to quantitative studies in the area of patient satisfaction and dissatisfaction with treatment. Results: Twenty-seven papers were selected for systematic review. Results indicated a paucity of studies of patient satisfaction with treatment in CLBP. The quality of studies included in the review was mixed, making comparisons and generalisations problematic. Our results showed largely positive but also some negative views towards the treatment of CLBP. Conclusions: The measurement of patient satisfaction in CLBP makes it possible for health professionals to target features of the patient’s treatment that cause them distress (such as experiencing side effects), and may contribute to the maintenance and improvement of their health. Findings from this review indicate the necessity to develop a measure specific to patient satisfaction with treatment in CLBP. The instrument needs to be based on a standard operational definition and a conceptual framework, and have good content validity and psychometric properties.

Development of a patient-centered conceptual model of the impact of living with autism spectrum disorder:

The aim of this study was to generate a patient-centered conceptual model of the impact of living with autism spectrum disorder, which can be used to support the selection of outcome measures for clinical trials. Following an initial literature review to identify preliminary concepts and inform an interview guide, in-depth face-to-face interviews were conducted with adolescents and adults with autism spectrum disorder (IQ ⩾ 70) (n = 10), as well as parents of children, adolescents, and adults with autism spectrum disorder (IQ ⩾ 70) (n = 26). Data were analyzed using established qualitative research methods. The resultant conceptual model contains three interrelated domains reflecting core symptoms of autism spectrum disorder (communication deficits, socialization deficits, and restrictive, repetitive patterns of behavior), three domains reflecting associated symptoms of autism spectrum disorder (physical, cognitive, and emotional/behavioral), and three domains representing the impacts of living with autism spectrum disorder (impacts on activities of daily living, school/work, and social life). Interview respondents also cited social communication deficits as priority targets for new treatments. The conceptual model provides a patient-centered perspective of relevant concepts of autism spectrum disorder from the perspectives of people with autism spectrum disorder and their parents and offers a valuable tool for identifying valid patient-centered outcome measures for future clinical trials.