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

Kathryn Lasch,Vasudha Bal,John A Carter,Vicky Huang,Harman Dhatt,Erica G Horodniceanu

doi:10.1186/s12955-017-0702-0

Abstract

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.

Highlights

Compliance, palatability, gastrointestinal (GI) symptom, and treatment satisfaction patient- and observer-reported outcome (PRO, Observer-reported outcome (ObsRO)) measures were developed/modified for patients with transfusiondependent anemias or myelodysplastic syndrome (MDS) requiring iron chelation therapy (ICT)
Patient-reported outcome (PRO) refers to Instrument development and selection The Satisfaction with Iron Chelation Therapy (SICT) instrument was previously developed to assess patientreported satisfaction and compliance with DFX dispersible tablets among patients with transfusion-dependent anemias or MDS and was validated in an open-label, single-arm, multicenter trial evaluating the efficacy and safety of DFX in patients with transfusion-dependent anemias and MDS requiring treatment for iron overload [21]
Demographic and clinical characteristics Twenty-one subjects were interviewed in 6 United States (US) cities

Summary

Introduction

Compliance, palatability, gastrointestinal (GI) symptom, and treatment satisfaction patient- and observer-reported outcome (PRO, ObsRO) measures were developed/modified for patients with transfusiondependent anemias or myelodysplastic syndrome (MDS) requiring iron chelation therapy (ICT). 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 chronicallytreated patients has been demonstrated previously [21,22,23], as has the relationship between perceived burden of ICT and poor compliance [22]

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Results

Discussion

Conclusion