Background
Smoldering multiple myeloma (SMM) is an early form of multiple myeloma (MM). SMM is typically considered asymptomatic, and research on how it affects health-related quality of life (HRQoL) is limited. This study assessed the symptoms and HRQoL of patients with SMM and those who progressed from SMM to MM and evaluated the content validity of two patient-reported outcome instruments (EORTC QLQ-C30 and nine items from the EORTC QLQ-MY20) for use in SMM clinical trials. To address these objectives, concept elicitation and cognitive interviews were conducted with SMM patients and recently diagnosed MM patients.
Results
Fifteen adult SMM and six adult MM participants with a prior SMM diagnosis were interviewed. On average, SMM study participants were 61 years old (46.0–78.0), 11 (73%) were female, and diagnosed 2.6 (±2.0) years ago. Each participant had experienced at least one symptom, most commonly tiredness/fatigue, weakness, and pain. The most common HRQoL impacts were emotional and physical. SMM study participants demonstrated good understanding of both the EORTC QLQ-C30 and EORTC QLQ-MY20 subscales and found them relevant to their SMM health state. The average age of MM participants was 53 years old (39.0–62.0); 5 (83%) were female and diagnosed 1.9 years ago (±2.1). MM participants most commonly reported tiredness, weakness, constipation, shortness of breath, and dry mouth as occurring when they progressed from SMM to MM.
Conclusions
Although previously described as asymptomatic, these SMM participants reported experiencing symptoms that affected their lives. Additionally, the EORTC subscales measured symptoms SMM patients experienced. The participants with MM reported that the symptom burden and HRQoL impacts increased when diagnosed with MM. These findings suggest the need for increased surveillance of symptoms within the SMM population and further suggest that the EORTC subscales can be used to assess symptoms and impacts in both the SMM and MM populations.
Background
Hereditary angioedema (HAE) is a genetic disorder characterized by re-occurring swelling episodes called “attacks,” usually in the limbs, face, airways, and intestinal tract. New prophylactic therapies have reduced the frequency of these attacks. This study describes results from a literature review and clinician interviews assessing patient HAE symptom experiences and timing, and then evaluates whether existing patient-reported outcome (PRO) tools adequately reflect this experience.
Methods
A targeted literature review as well as interviews with key opinion leaders (KOLs), were conducted to capture information about the patient experience and their symptoms. An assessment of various PROs was then conducted to determine how well they each covered HAE symptoms and impacts.
Results
Nineteen HAE symptoms were identified. KOLs reported that patients on prophylactic therapy experienced some symptoms indicating an attack was imminent, but then never experienced an attack. The comparison of the different PROs found that the Hereditary Angioedema Patient-Reported Outcome was the instrument that most thoroughly examined the symptoms of patients with HAE.
Conclusions
Given the introduction of new prophylactic therapies, further research is needed to determine the effect of being attack-free for longer periods of time on health-related quality of life.
lung cancer is the leading cause of cancerrelated death, and represents a major disease burden substantially impacting on quality of life (QoL). Patient-reported outcome measures (PROMs) have been identified as effective methods of evaluating patient QoL. Existing lung cancer specific PROMs however, have uncertain utility and minimal patient involvement in their design and development. This qualitative study aimed to evaluate the patient perspective of existing PROMs and to explore their appropriateness for populationbased descriptions of lung cancer-related QoL. Prior presentations: Australian Lung Cancer Conference 2020. Methods: A descriptive qualitative study was conducted consisting of semi-structured interviews with 14 patients recruited from the Victorian Lung Cancer Registry (VLCR) and Alfred Hospital via purposive sampling. Interviews first explored the factors most important to lung cancer patients QoL and secondly, patient's perspectives on the appropriateness of existing PROMs. Thematic analysis was used to develop themes and content analysis was conducted to determine PROM acceptability. Results: Five novel themes were identified by patients as having important impacts on QoL: 1. Personal attitude towards the disease is important for coping 2. Independence is valued 3. Relationships with family and friends are important 4. Relationships with treating team are meaningful 5. Limited personal and public awareness of lung cancer These patientidentified impacts are poorly covered in existing lung cancer-specific PROMs. Patients welcomed and appreciated the opportunity to complete PROMs. However, they identified problems with existing PROMs relevance, tone and formatting. Conclusion: Existing lung cancer PROMs poorly reflect the five themes identified in this study as most important to lung cancer patients QoL. This study reaffirms the need to review existing PROMs to ensure currency and construct validity. Future PROM development must engage patient generated themes and evolve to reflect the changing management and therapeutic landscape.
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