Development of a Conceptual Framework of Patient-reported Outcomes in Light Chain Amyloidosis: A Qualitative Study

Abstract Background Light chain (AL) amyloidosis is a plasma cell neoplasm associated with high early mortality and severe morbidity that can cause severe disability. We explored the impact of AL amyloidosis on symptoms and well-being from the perspectives of patients and health care providers who regularly care for AL patients. We intended to develop a conceptual understanding of patient-reported outcomes in AL amyloidosis to identify the context of use and concept of interest for a clinical outcome assessments tool in this disease.Method Twenty patients and 10 professionals were interviewed. Patient interviews captured the spectrum of amyloidosis experience including time from diagnosis, type of organ involvement, and presence and type of treatment received. Interviews with professionals included physicians, advanced practice providers, registered nurse, and a patient advocate; these interviews covered similar topics. Results The impact of AL amyloidosis on patients’ life was multidimensional, with highly subjective perceptions of normality and meaning. Four major themes from patients and experts included diagnosis of AL amyloidosis, living with AL amyloidosis, symptom burden, and social roles. Barriers to patient-reported outcomes data collection in patients were additionally explored from experts. The themes provide a comprehensive understanding of the important experiences of symptom burden and its impact on daily life from AL amyloidosis patients’ and from the perspectives of professionals who care for patients with AL amyloidosis. Conclusion These findings further the conceptual understanding and identification of a preliminary model of concept of interest for development of a clinical outcome assessments tool for AL amyloidosis.

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PND121 CLINICAL OUTCOME ASSESSMENTS IN RELAPSE REMITTING MULTIPLE SCLEROSIS: INTEGRATION OF CLINICIAN AND PATIENT REPORTED OUTCOMES IN REAL WORLD EVIDENCE PUBLICATIONS

Value in Health ◽

10.1016/j.jval.2020.04.1010 ◽

2020 ◽

Vol 23 ◽

pp. S282-S283

Author(s):

R.W. van den Broek ◽

R. Gadiot-Molenaar ◽

T. Hartog

Keyword(s):

Multiple Sclerosis ◽

Clinical Outcome ◽

Real World ◽

Patient Reported Outcomes ◽

Outcome Assessments ◽

Real World Evidence ◽

Patient Reported ◽

Remitting Multiple Sclerosis ◽

Clinical Outcome Assessments

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Patient reported outcomes in patients with desmoid type fibromatosis.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.11574 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. 11574-11574

Author(s):

VIKAS GARG ◽

Sameer Rastogi ◽

Adarsh Barwad ◽

Rambha Panday ◽

Sandeep Kumar Bhoriwal ◽

...

Keyword(s):

Patient Reported Outcomes ◽

Symptom Burden ◽

Benign Neoplasm ◽

Anxiety And Depression ◽

Appendicular Skeleton ◽

Healthy Controls ◽

Symptom Scale ◽

Cross Sectional ◽

Patient Reported ◽

The Impact

11574 Background: Desmoid type fibromatosis (DTF) is a rare benign neoplasm with infiltrative growth and high local recurrences. Due to long disease course, unpredictable growth pattern, and low mortality, using only survival outcomes may be inappropriate. In this study we assessed the impact of DTF on health related quality of life (HRQoL). Methods: This was a cross-sectional study done in patients with DTF. The study participants were asked to fill the EORTC QLQ-C30, GAD-7 and PHQ- 9 q uestionnaires to assess HRQoL, anxiety and depression . Outcomes were also compared with healthy controls. Results: 204 subjects (102 DTF patients and 102 healthy controls) were recruited. Study parameters have been summarized in Table. Appendicular skeleton (limbs + girdle) was most commonly involved in 59 % patients and abdominal wall or mesentery was involved in 22.5 %. Patients have received median of 2 lines of therapy. 54 % patients were currently on sorafenib and 41 % were under active surveillance. Mean global health status in DTF patient 65.58 ± 22.64, was significantly lower than healthy controls. Similarly, DTF patients scored low on all functional scales except cognitive functioning. Symptom scale showed significantly higher symptom burden of fatigue, pain, insomnia and financial difficulties. Anxiety & depression was observed in 39.22 % and 50 % of DTF patients respectively. DTF patients had higher rates of mild, moderate and severe anxiety and depression compared to healthy controls. No difference was observed based on site of disease. Conclusions: DTF patients have significant symptom burden, poor functioning, and heightened anxiety and depression. Patient reported outcomes should be routinely used to assess treatment efficacy in DTF patients.[Table: see text]

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Opportunities and challenges of incorporating clinical outcome assessments in brain tumor clinical trials

Neuro-Oncology Practice ◽

10.1093/nop/npy032 ◽

2018 ◽

Vol 6 (2) ◽

pp. 81-92 ◽

Cited By ~ 1

Author(s):

Emanuela Molinari ◽

Tito R Mendoza ◽

Mark R Gilbert

Keyword(s):

Clinical Trials ◽

Brain Tumor ◽

Clinical Outcome ◽

Clinical Trial Design ◽

Well Being ◽

Successful Implementation ◽

Outcome Assessments ◽

Tumor Studies ◽

Clinical Outcome Assessments ◽

The Impact

Abstract Regulatory agencies have progressively emphasized the importance of assessing broader aspects of patient well-being to better define therapeutic gain. As a result, clinical outcome assessments (COAs) are increasingly used to evaluate the impact, both positive and negative, of cancer treatments and in some instances have played a major factor in the regulatory approval of drugs. Challenges remain, however, in the routine incorporation of these measures in cancer clinical trials, particularly in brain tumor studies. Factors unique to brain tumor patients such as cognitive decline and language dysfunction may hamper their successful implementation. Study designs often relegated these outcome measures to exploratory endpoints, further compromising data completion. New strategies are needed to maximize the complementary information that COAs could add to clinical trials alongside more traditional measures such as progression-free and overall survival. The routine incorporation of COAs as either primary or secondary objectives with attention to minimizing missing data should define a novel clinical trial design. We provide a review of the approaches, challenges, and opportunities for incorporating COAs into brain tumor clinical research, providing a perspective for integrating these measures into clinical trials.

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Clinical Outcome Assessment in Cancer Rehabilitation and the Central Role of Patient-Reported Outcomes

Cancers ◽

10.3390/cancers14010084 ◽

2021 ◽

Vol 14 (1) ◽

pp. 84

Author(s):

Jens Lehmann ◽

Maria Rothmund ◽

David Riedl ◽

Gerhard Rumpold ◽

Vincent Grote ◽

...

Keyword(s):

Clinical Outcome ◽

Patient Reported Outcomes ◽

Health Condition ◽

Performance Outcomes ◽

Cancer Rehabilitation ◽

Third Party ◽

Special Focus ◽

Patient Reported ◽

Value Based Care ◽

Clinical Outcome Assessments

The aim of cancer rehabilitation is to help patients regain functioning and social participation. In order to evaluate and optimize rehabilitation, it is important to measure its outcomes in a structured way. In this article, we review the different types of clinical outcome assessments (COAs), including Clinician-Reported Outcomes (ClinROs), Observer-Reported Outcomes (ObsROs), Performance Outcomes (PerfOs), and Patient-Reported Outcomes (PROs). A special focus is placed on PROs, which are commonly defined as any direct report from the patient about their health condition without any interpretation by a third party. We provide a narrative review of available PRO measures (PROMs) for relevant outcomes, discuss the current state of PRO implementation in cancer rehabilitation, and highlight trends that use PROs to benchmark value-based care. Furthermore, we provide examples of PRO usage, highlight the benefits of electronic PRO (ePRO) collection, and offer advice on how to select, implement, and integrate PROs into the cancer rehabilitation setting to maximize efficiency.

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Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change

Blood Cancer Journal ◽

10.1038/s41408-021-00412-8 ◽

2021 ◽

Vol 11 (2) ◽

Author(s):

Anita D’Souza ◽

Ruta Brazauskas ◽

Angela Dispenzieri ◽

Julie Panepinto ◽

Kathryn E. Flynn

Keyword(s):

Mental Health ◽

Physical Function ◽

Light Chain ◽

Patient Reported Outcomes ◽

Social Roles ◽

Global Mental Health ◽

First Year ◽

Al Amyloidosis ◽

Patient Reported ◽

Anxiety Depression

AbstractWe conducted a prospective cohort study in newly diagnosed systemic light chain (AL) amyloidosis patients (N = 59) to study patient-reported outcomes (PROs) through the first year. The median age was 68 years with 42% female, 8% Black, and 78% lambda subtype. Organ involvement was cardiac in 66%, renal in 58%, with 25% having 3 or greater organs involved. Between baseline and 3 months, all PROMIS®-29 domain scores worsened by 0.4–4.1 points except anxiety which improved by 2.1 points. By 1 year, scores improved compared to the greatest decline at 3 months, most statistically significant for global physical health, physical function, and fatigue. On stage-adjusted survival analysis, in addition to baseline global physical and mental health, domains measuring physical function, fatigue, anxiety, depression, and social roles were associated with 1-year survival. At 1 year, PROMIS measures were associated with NT-proBNP changes and hematologic response. Among patients with an NT-proBNP response, the improvement was seen in physical function, social roles, global mental health, and anxiety. Among patients with an NT-proBNP progression, worsening was seen with anxiety, depression, sleep, and global mental health. Measuring and tracking PROs in patients with AL amyloidosis is important and these important outcomes can be used as correlative endpoints in clinical care/research.

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Clinical outcome assessments in neuro-oncology: a regulatory perspective

Neuro-Oncology Practice ◽

10.1093/nop/npv062 ◽

2015 ◽

Vol 3 (1) ◽

pp. 4-9 ◽

Cited By ~ 2

Author(s):

Joohee Sul ◽

Paul G. Kluetz ◽

Elektra J. Papadopoulos ◽

Patricia Keegan

Keyword(s):

Clinical Outcome ◽

Neurological Diseases ◽

Objective Response ◽

Progression Free Survival ◽

Patient Centered ◽

Outcome Assessments ◽

Regulatory Perspective ◽

Clinical Outcome Assessments ◽

And Function ◽

The Impact

Abstract Overall survival, progression-free survival, and to a lesser extent objective response rate, have long been the most widely accepted endpoints used to evaluate clinical benefit in oncology trials. More recently, clinical outcome assessments (COAs) that measure the impact of disease and treatment on patients′ symptoms and function have been recognized as having potential to be an integral component of the risk/benefit analysis of new therapies. Although COAs have been used to evaluate cognitive and physical functioning in neurological diseases, assessing patient-centered outcomes in individuals with malignant brain tumors presents unique challenges. The approach to developing appropriate instruments to measure COAs in neuro-oncology should include identifying areas requiring new tools, reviewing existing tools that may be suitable or adapted for use in clinical trials, and engaging early with regulatory agencies to standardize a set of well-defined and reliable instruments to quantify important patient-centered outcomes.

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