Anita D'Souza

Objective To 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. Methods Content 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. Results Study 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. Conclusion The AL‐PROfile has acceptable reliability and validity for use in systemic light chain amyloidosis patients.

Summary Autologous haematopoietic cell transplantation (autoHCT) and continuous post‐transplant maintenance therapy are the standard of care in transplant‐eligible multiple myeloma (MM) patients. We sought to describe symptom burden and identify symptom clusters occurring in MM patients after autoHCT using data from the BMT CTN 0702 randomized controlled trial comparing the outcomes of three treatment interventions after an autoHCT in 758 MM patients. We analysed individual transplant‐related symptoms assessed via the FACT‐BMT questionnaire at enrolment and annually for 4‐year post‐autoHCT. We also described the effect the individual symptoms and symptom clusters have on quality of life (QoL). We identified three stable symptom clusters: malaise symptom cluster (lack of energy, feeling ill, having pain, experiencing nausea, loss of appetite), physical symptom cluster (having skin problems, tremors, worsening eyesight, change in taste, shortness of breath, frequent colds) and emotional symptom cluster (feeling sad, being nervous, experiencing sleep problems). Malaise and emotional symptom clusters have a greater impact on QoL than the physical symptoms cluster. Identifying these symptoms warrant additional support in terms of psychosocial support, in addition to treatment of the physical symptoms themselves.