A cross-sectional study of patient-reported outcomes and symptom burden using PROMIS and PRO-CTCAE measures in light chain amyloidosis

D’Souza, Anita; Szabó, Anikó; Akinola, Idayat; Finkel, Muriel; Flynn, Kathryn E.

doi:10.1007/s11136-023-03354-9

Cited by 4 publications

(3 citation statements)

References 32 publications

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“…The AL-PROfile measure was developed by combining validated items from PROMIS and PRO-CTCAE identified through mixed methods research with AL amyloidosis patients and experts. 2 , 13 , 14 Prior concept elicitation of the symptom burden of AL amyloidosis identified additional symptoms, including dyspnea, dizziness, edema, tingling/numbness and many GI symptoms, which were identified as prevalent symptoms and leading to considerable symptom burden. 2 The current analysis provides evidence of content validity from cognitive debriefing interviews.…”

Section: Discussionmentioning

confidence: 99%

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Development and initial validation of the AL‐PROfile patient‐reported outcome measure in light chain (AL) amyloidosis

D'Souza,

Szabo,

Akinola

et al. 2024

European J of Haematology

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ObjectiveTo evaluate the psychometric properties of the AL‐PROfile, a patient‐reported outcome measure combining the Patient‐Reported Outcomes Measurement Information System (PROMIS)‐29, two items from PROMIS Cognitive Function, and select Patient‐Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO‐CTCAE) items.MethodsContent validity was assessed through cognitive debriefing interviews of 20 patients who completed the AL‐PROfile (Study 1). Study 2 involved 297 participants who completed the AL‐PROfile and Medical Outcomes Study 36‐item Short‐Form Health Survey (SF‐36). Reliability (internal consistency and test–retest reliability) and validity (convergent and discriminant validity, known groups validity by stage/organ involvement) were calculated.ResultsStudy 1 participants found the AL‐PROfile straightforward confirming the relevance of the included content. Some felt that certain questions were not related to their amyloidosis experience. Study 2 demonstrated acceptable internal consistency for all domains/items except PROMIS Cognitive Function and acceptable test–retest reliability for all except PROMIS Cognitive Function and PRO‐CTCAE nausea. Large correlations were seen for the same domain across measures while correlations for divergent domains within a measure and different domains across different measures were small. The PRO‐CTCAE items showed small to medium correlations with each other and with PROMIS and SF‐36 domains. Stage was associated with physical function, fatigue, social roles, swelling, and shortness of breath scores.ConclusionThe AL‐PROfile has acceptable reliability and validity for use in systemic light chain amyloidosis patients.

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Section: Discussionmentioning

confidence: 99%

“…The 2004 Mayo Clinic stage 12 was thus calculated in a subset of participants who provided this information. Data from Study 2 has been described in part in other work 13,14 …”

Section: Methodsmentioning

confidence: 99%

Development and initial validation of the AL‐PROfile patient‐reported outcome measure in light chain (AL) amyloidosis

D'Souza,

Szabo,

Akinola

et al. 2024

European J of Haematology

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“…Our cross-sectional sample included heterogeneity in disease natural history, with individuals early in their disease course (i.e., within 2 years of diagnosis) and long-term (>5 years) survivors, individuals on active chemotherapy, and those who had a long treatment-free interval. 27 Given that these patients continue to experience impaired health-related quality of life, our approach remained relevant. Stage of disease is the strongest known predictor of outcomes in AL amyloidosis.…”

Section: Discussionmentioning

confidence: 99%

Differences in patient‐reported outcomes (PROs) by disease severity in light chain (AL) amyloidosis

D’Souza

Szabó

Akinola

et al. 2023

European J of Haematology

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ObjectiveTo assess the impact of organ involvement on patient‐reported outcomes (PROs) in light chain (AL) amyloidosis.MethodsPROs were evaluated using the KCCQ‐12, PROMIS‐29 + 2, and SF‐36 in individuals with AL amyloidosis. The 2004 Mayo system was used to stage disease and cardiac, neurologic, and renal involvement was considered. Global physical and mental health (MH) scores, physical function (PF), fatigue, social function (SF), pain, sleep, and MH domains were evaluated. Effect sizes between scores were measured using Cohen's d.ResultsOf 297 respondents, the median age at diagnosis was 60 years with 58% cardiac, 58% renal, and 30% neurologic involvement. Fatigue, PF, SF, and global physical health with PROMIS and SF‐36 discriminated the most by stage. Significant discrimination in PROMIS and/or SF‐36 was seen in PF, fatigue, and global physical health with cardiac involvement. For neurologic involvement, PF, fatigue, SF, pain, sleep, global physical, and MH with PROMIS and role physical, vitality, pain, general health, and physical component summary with SF‐36 were discriminatory. For renal amyloid, pain by SF‐36 and PROMIS, and SF‐36 MH and role emotional subscales were significant.ConclusionsFatigue, PF, SF, and global physical health can discriminate stage, cardiac and neurologic, but not renal, AL amyloidosis involvement.

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Patient-reported satisfaction with telemedicine in light chain (AL) amyloidosis care

Akinola,

Flynn,

Szabo

et al. 2024

Amyloid

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A cross-sectional study of patient-reported outcomes and symptom burden using PROMIS and PRO-CTCAE measures in light chain amyloidosis

Cited by 4 publications

References 32 publications

Development and initial validation of the AL‐PROfile patient‐reported outcome measure in light chain (AL) amyloidosis

Development and initial validation of the AL‐PROfile patient‐reported outcome measure in light chain (AL) amyloidosis

Differences in patient‐reported outcomes (PROs) by disease severity in light chain (AL) amyloidosis

Patient-reported satisfaction with telemedicine in light chain (AL) amyloidosis care

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