A new tool for the assessment of satisfaction with iron chelation therapy (ICT‐Sat) for patients with β‐thalassemia major

Elalfy, Mohsen Saleh; Massoud, W H; Elsherif, Nayera H K; Labib, Jonair H.; Elalfy, Omar; Elaasar, Safaa; Mackensen, Sylvia von

doi:10.1002/pbc.23413

Cited by 7 publications

(6 citation statements)

References 30 publications

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“…found that adherence and satisfaction to DFO is generally poor and a patient's attitude to adherence can change over time . The availability of oral iron chelators may contribute to improved compliance, especially among pediatric and adolescent patients . The combined DFP/DFX in our study had the best patients' compliance and satisfaction.…”

Section: Discussionmentioning

confidence: 68%

Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients

Elalfy

Adly

Wali

et al. 2015

European J of Haematology

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Section: Discussionmentioning

confidence: 68%

Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients

Elalfy

Adly

Wali

et al. 2015

European J of Haematology

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“…For our patients, iron chelation therapy, particularly oral chelation, significantly and negatively impacted HRQoL. Leafy MS et al 35 observed all patients experienced burden with chelation, no matter which type. Similarly, Thavorncharoensap et al 10 stated that iron chelation treatment was associated with poorer HRQOL.…”

Section: Discussionmentioning

confidence: 55%

Health-Related Quality of Life in Thai Children With Thalassemia as Evaluated by Pedsql and Eq-5d-Y: A Single Center Experience

Sinlapamongkolkul

Surapolchai

2020

Mediterr J Hematol Infect Dis

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Background: Thalassemia remains a chronic challenging disease in Thailand, but national prenatal screening along with better treatment and management may have improved health-related quality of life (HRQoL) for pediatric patients. We aimed to measure the HRQoL of transfusion dependent (TDT) and non-transfusion dependent (NTDT) of these pediatric patients at our institute. Methods: We included all patients 2 – 18 years old, with TDT and NTDT, using the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL) and the EuroQol Group’s Five Dimensions for Youth (EQ-5D-Y) instruments. Patients and caregivers responded as appropriate for age. Results: Mean PedsQL total summary scores (TSS) (SD) of child self-reports and parent proxy-reports were 81.00 (10.94) and 78.84 (16.72) from 150 participants. Mean EQ-5D-Y VAS (SD) for children was 89.27 (11.56) and 86.72 (10.62) for parent proxies. The most problematic EQ-5D-Y dimension was “having pain or discomfort”. These scores had significant correlations between child and parental proxy perspectives, as well as between the PedsQL and EQ-5D-Y. An age of 8 - 12 years and oral chelation therapy predicted lower self-reported PedsQL TSS. Parental proxy-report predictors for reduced PedsQL TSS and EQ-5D-Y VAS were primary school education for children, parental proxy secondary school education, Universal Coverage insurance, and TDT. Conclusion: HRQoL scores of our pediatric thalassemia patients had improved from the previous decade, and these findings may represent our better standard of care. Some sociodemographic and clinical characteristics may present negative impacts on HRQoL.

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“…However, DFX has restrictive administration instructions (eg, take on an empty stomach at least 30 min before food; disperse tablets by stirring in water or juice) [19] and known associations with suboptimal palatability, tolerability, and gastrointestinal (GI) side effects [19, 20], which may negatively impact compliance [20]. This direct relationship between treatment satisfaction and compliance among ICT and chronically-treated patients has been demonstrated previously [21–23], as has the relationship between perceived burden of ICT and poor compliance [22]. …”

Section: Introductionmentioning

confidence: 84%

Qualitative modification and development of patient- and caregiver-reported outcome measures for iron chelation therapy

Horodniceanu

Bal

Dhatt

et al. 2017

Health Qual Life Outcomes

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BackgroundCompliance, palatability, gastrointestinal (GI) symptom, and treatment satisfaction patient- and observer-reported outcome (PRO, ObsRO) measures were developed/modified for patients with transfusion-dependent anemias or myelodysplastic syndrome (MDS) requiring iron chelation therapy (ICT).MethodsThis qualitative cross-sectional observational study used grounded theory data collection and analysis methods and followed PRO/ObsRO development industry guidance. Patients and caregivers of patients with transfusion-dependent anemias or MDS were individually interviewed face-to-face to cognitively debrief the Compliance, Palatability, GI Symptom Diary, and Modified Satisfaction with Iron Chelation Therapy (SICT) instruments presented electronically. Interviews were conducted in sets. Interviews began open-endedly to spontaneously elicit ICT experiences. Item modifications were debriefed during the later interviews. Interviews were audio recorded, transcribed, and coded. Data was analyzed using ATLAS.ti qualitative research software.ResultsTwenty-one interviews were completed (Set 1: 5 patients, 6 caregivers; Set 2: 6 patients, 4 caregivers) in 6 US cities. Mean age was 43 years for patients and 9 years for children of caregivers. Conditions requiring ICT use across groups included transfusion-dependent anemias (85.7%) and MDS (14.3%). Concepts spontaneously reported were consistent with instruments debriefed. Interview analysis resulted in PRO and ObsRO versions of each instrument: Compliance (2 items), Palatability (4 items), GI Symptom Diary (6 items), and Modified SICT (PRO = 13, ObsRO = 17 items).ConclusionQualitative research data from cognitive interviews supports the content validity and relevance of the instruments developed/modified. Quantitative validation of these PRO and ObsRO measures is needed testing for validity, reliability, and responsiveness for future research use with new formulations of oral ICT.

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A new tool for the assessment of satisfaction with iron chelation therapy (ICT‐Sat) for patients with β‐thalassemia major

Abstract: A new clinically based ICT-Sat tool was developed and revealed good psychometric characteristics. Adherence to ICT was better correlated with "perceived effectiveness" and SF level.

Cited by 7 publications

References 30 publications

Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients

Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients

Health-Related Quality of Life in Thai Children With Thalassemia as Evaluated by Pedsql and Eq-5d-Y: A Single Center Experience

Qualitative modification and development of patient- and caregiver-reported outcome measures for iron chelation therapy

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