Quality of Life Related to Oral versus Subcutaneous Iron Chelation: A Time Trade-off Study

Osborne, Richard H.; Lourenço, Richard De Abreu; Dalton, Andrew; Houltram, Jennifer; Dowton, David; Joshua, Douglas; Lindeman, Robert; Ho, Phoebe Joy

doi:10.1111/j.1524-4733.2007.00200.x

Cited by 51 publications

(47 citation statements)

References 12 publications

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“…Aspects of convenience valued can be classified broadly into two categories: Those relating to the administration of an intervention, for example, dosing frequency or mode of administration [13,15,18,19,21,23,24,26,[30][31][32][33][34][36][37][38][39], and those looking at ease of access to an intervention, for example, distance to travel [14,16,17,20,25,28,29,35]. Two studies spanned both categories [22,27].…”

Section: Key Characteristics Of Studiesmentioning

confidence: 99%

“…Studies identified and reviewed were published between 1996 and 2013, and came from Canada (n ¼ 7) [13][14][15][16][17][18][19], the United States (n ¼ 3) [20][21][22], the United Kingdom (n ¼ 3) [23][24][25], Scotland (n ¼ 3) [26][27][28], Australia (n ¼ 3) [29][30][31], Sweden (n ¼ 2) [32,33], Denmark (n ¼ 1) [34], Germany (n ¼ 1) [35], Italy (n ¼ 1) [36], and Spain (n ¼ 1) [37]. Two studies had multinational perspectives [38,39].…”

Section: Key Characteristics Of Studiesmentioning

confidence: 99%

“…Preference-elicitation methods included SG (n ¼ 2) [20,26], WTP (n ¼ 8) [14,[18][19][20][21]29,33,35], and TTO (n ¼ 3) [23,30,31] (Table 2). A substantial proportion of the studies (56%) took the form of a DCE, using a financial or cost-related attribute to derive a WTP valuation for convenience [13,[15][16][17]22,24,25,27,28,32,34,[36][37][38][39].…”

Section: Key Characteristics Of Studiesmentioning

confidence: 99%

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Does Convenience Matter in Health Care Delivery? A Systematic Review of Convenience-Based Aspects of Process Utility

Higgins

Barnett

Meads³

et al. 2014

Value in Health

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Objectives: To systematically review the existing literature on the value associated with convenience in health care delivery, independent of health outcomes, and to try to estimate the likely magnitude of any value found. Methods: A systematic search was conducted for previously published studies that reported preferences for convenience-related aspects of health care delivery in a manner that was consistent with either cost-utility analysis or cost-benefit analysis. Data were analyzed in terms of the methodologies used, the aspects of convenience considered, and the values reported. Results: Literature searches generated 4715 records. Following a review of abstracts or full-text articles, 27 were selected for inclusion. Twentysix studies reported some evidence of convenience-related process utility, in the form of either a positive utility or a positive willingness to pay. The aspects of convenience valued most often were mode of administration (n ¼ 11) and location of treatment (n ¼ 6). The most common valuation methodology was a discrete-choice experiment containing a cost component (n ¼ 15). Conclusions: A preference for convenience-related process utility exists, independent of health outcomes. Given the diverse methodologies used to calculate it, and the range of aspects being valued, however, it is difficult to assess how large such a preference might be, or how it may be effectively incorporated into an economic evaluation. Increased consistency in reporting these preferences is required to assess these issues more accurately.

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Section: Key Characteristics Of Studiesmentioning

confidence: 99%

Section: Key Characteristics Of Studiesmentioning

confidence: 99%

Section: Key Characteristics Of Studiesmentioning

confidence: 99%

See 1 more Smart Citation

Does Convenience Matter in Health Care Delivery? A Systematic Review of Convenience-Based Aspects of Process Utility

Higgins

Barnett

Meads³

et al. 2014

Value in Health

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“…[2][3][4][5][6] The once-daily oral deferasirox dispersible tablet (DT) formulation (Exjade ® ), available since 2005, offered an improved option over parenteral deferoxamine (Desferal ® ), providing greater compliance, patient satisfaction, and health-related quality of life. 7,8 The efficacy and safety of deferasirox DT has been well-defined through an extensive clinical trial program in adult and pediatric patients with a variety of anemias, including thalassemia, myelodysplastic syndromes (MDS), sickle-cell disease, and other rare anemias, [9][10][11][12][13] and has been used in clinical practice worldwide for over a decade. Nonetheless, barriers to optimal patient acceptance of treatment still exist with deferasirox DT, including palatability, the need to take the drug in a fasting state (ie, not being able to take with food), and drug-related side effects, notably gastrointestinal (GI) tolerability.…”

Section: Introductionmentioning

confidence: 99%

New film‐coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower‐risk MDS: Results of the randomized, phase II ECLIPSE study

et al. 2017

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Once-daily deferasirox dispersible tablets (DT) have a well-defined safety and efficacy profile and, compared with parenteral deferoxamine, provide greater patient adherence, satisfaction, and quality of life. However, barriers still exist to optimal adherence, including gastrointestinal tolerability and palatability, leading to development of a new film-coated tablet (FCT) formulation that can be swallowed with a light meal, without the need to disperse into a suspension prior to consumption.The randomized, open-label, phase II ECLIPSE study evaluated the safety of deferasirox DT and FCT formulations over 24 weeks in chelation-naïve or pre-treated patients aged 10 years, with transfusion-dependent thalassemia or IPSS-R very-low-, low-, or intermediate-risk myelodysplastic syndromes. One hundred seventy-three patients were randomized 1:1 to DT (n 5 86) or FCT (n 5 87). Adverse events (overall), consistent with the known deferasirox safety profile, were reported in similar proportions of patients for each formulation (DT 89.5%; FCT 89.7%), with a lower frequency of severe events observed in patients receiving FCT (19.5% vs. 25.6% DT). Laboratory parameters (serum creatinine, creatinine clearance, alanine aminotransferase, aspartate aminotransferase and urine protein/creatinine ratio) generally remained stable throughout the study.Patient-reported outcomes showed greater adherence and satisfaction, better palatability and fewer concerns with FCT than DT. Treatment compliance by pill count was higher with FCT (92.9%) than with DT (85.3%). This analysis suggests deferasirox FCT offers an improved formulation with enhanced patient satisfaction, which may improve adherence, thereby reducing frequency and severity of iron overload-related complications. | I N T R O D U C T I O NTransfusion and iron chelation therapy can be a lifelong requirement for many patients with transfusion-dependent anemias. Compliance with iron chelation therapy can influence the frequency and severity of iron overload-related complications, 1 with demonstrated improvement in organ dysfunction and survival in patients compliant with iron chelation therapy. [2][3][4][5][6] The once-daily oral deferasirox dispersible tablet (DT) This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. variety of anemias, including thalassemia, myelodysplastic syndromes (MDS), sickle-cell disease, and other rare anemias, 9-13 and has been used in clinical practice worldwide for over a decade. Nonetheless, barriers to optimal patient acceptance of treatment still exist with deferasirox DT, including palatability, the need to take the drug in a fasting state (ie, not being able to take with food), and drug-related side effects, notably gastrointestinal (GI) tolerability. 14 An improved filmcoated tablet (FCT) formulation of deferasirox (US, Jadenu ® ; EU, Exja...

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“…As the disease progresses and relapses occur, patients may increasingly experience symptoms such as fatigue and depression [1,2]. A reduction in QOL may occur as a result of one or more treatment-related factors, including dissatisfaction with administration route, the need for frequent administration of therapy, the development of side effects or a perceived lack of treatment efficacy [3][4][5][6]. In addition, the relative importance assigned to different treatment outcomes may vary among patients.…”

Section: Introductionmentioning

confidence: 99%

Outcomes of a Switch to Fingolimod to Treat Relapsing Multiple Sclerosis: A Patient Subgroup Post Hoc Analysis

Gudesblatt¹,

Agashivala²,

Simrat³

et al. 2014

J Mult Scler

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Objective: The EPOC study assessed the effects of switching from an injectable disease-modifying therapy (glatiramer acetate or one of three interferon beta drugs) to once-daily, oral fingolimod 0.5 mg in patients with relapsing MS. Outcomes were assessed in several patient subgroups at 6 months between patients who switched to fingolimod and those who continued on iDMT.Methods: Differences in study endpoints between those who switched to fingolimod and those who continued on an iDMT were evaluated by age, gender, baseline Expanded Disability Status Scale score, MS duration, number of relapses in the previous year, previous treatment, previous treatment duration, and reason for switching. The primary endpoint was the change from baseline to month 6 in the Treatment Satisfaction Questionnaire for Medication Global Satisfaction score. Secondary endpoints were changes in scores for the TSQM Effectiveness, Side Effects and Convenience subscales, Beck Depression Inventory-II, Fatigue Severity Scale, Patient-Reported Outcome Indices for Multiple Sclerosis Activities subscale and the 36-item Short-Form Health Survey. Physician-assessed Clinical Global Impressions of Improvement score at 6 months was also recorded.Results: Switching to fingolimod from iDMT significantly improved scores in all subgroups for TSQM Global Satisfaction at month 6 (all comparisons p≤0.001). Switching to fingolimod significantly improved secondary endpoint scores across all scales for most subgroups (p<0.05) with a few exceptions: switching to fingolimod improved PRIMUS Activities scores only in patients with baseline EDSS scores of greater than 2.5 (p<0.05), and did not improve FSS scores in patients who were male, switched for efficacy reasons, received previous glatiramer acetate, or in whom MS symptom onset had occurred less than 3 years ago. For SF-36 scores, the benefit of switching to fingolimod was highly variable. Conclusion:Switching to fingolimod from iDMT improved outcomes versus continuing on iDMT, including for overall treatment satisfaction, in patients with MS with wide-ranging baseline characteristics.

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Quality of Life Related to Oral versus Subcutaneous Iron Chelation: A Time Trade-off Study

Abstract: Community respondents associate oral administration of an iron chelator such as deferasirox with enhanced QoL compared with subcutaneous treatment. Assuming equal safety and efficacy, QoL gains from once-daily oral treatment compared with subcutaneous infusion are significant.

Cited by 51 publications

References 12 publications

Does Convenience Matter in Health Care Delivery? A Systematic Review of Convenience-Based Aspects of Process Utility

Does Convenience Matter in Health Care Delivery? A Systematic Review of Convenience-Based Aspects of Process Utility

New film‐coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower‐risk MDS: Results of the randomized, phase II ECLIPSE study

Outcomes of a Switch to Fingolimod to Treat Relapsing Multiple Sclerosis: A Patient Subgroup Post Hoc Analysis

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