Development of a Novel Patient-Reported Outcome Measure in Haematological Malignancy for Use in Routine Clinical Practice: Item Generation

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5985-5985 ◽  
Author(s):  
Pushpendra Goswami ◽  
Sam Salek ◽  
Adele K. Fielding ◽  
Jonathan Kell ◽  
Saad Al-Ismail ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Board Of Directors ◽  
Hodgkin Lymphoma ◽  
Patient Reported Outcome ◽  
Routine Clinical Practice ◽  
Advisory Committees ◽  
Patient Reported ◽  
Low Prevalence ◽  
The Impact ◽  
Prototype Instrument

Abstract Aims: The impact of haematological malignancies (HM) on patients' health-related quality of life (HRQoL) is still not well understood. The aim of this study was to identify HRQoL issues and symptoms in patients with HM to be included in a new patient-reported outcome measure for use in routine clinical practice. Methods: In a multicentre observational study carried out in the UK, adult patients with various HM, capable of reading English and give written informed consent were recruited from five hospitals in England and Wales. This qualitative study employed semi-structured face-to-face interviews with open-ended questions related to the impact of haematological malignancy and its treatment on HRQoL and symptoms. All the interviews were audio recorded and transcribed verbatim. Content analysis was carried out using the NVivo 11 qualitative analysis software. The themes and the sub-themes generated from the transcribed interviews were discussed during a 2-day "data definition" panel meeting by 2 hematologists, 1 patient research partner, 1 representative of a hematology patient organisation and 3 QoL research experts to select items for inclusion in the prototype instrument. These items will be further re-grouped and refined using cognitive debriefing, content validity and factor analysis. Results: 127 (male=75; mean age = 61.6 years; SD=15.1; median age 65.4 years; and age range =18-88 years) with mean duration of the HM of 3.7 years (SD=4.3; median=2.1 years; and range= 19 days-23 years) were recruited into the study. Diagnoses were: Acute Myeloid Leukaemia (18); Acute Lymphoid Leukaemia (7); Chronic Myeloid Leukaemia (12); Chronic Lymphatic Leukaemia (11); Aggressive Non-Hodgkin Lymphoma (16); Indolent Non-Hodgkin Lymphoma (14); Hodgkin Lymphoma (10); Multiple Myeloma (21); Myeloproliferative Neoplasm (10); and Myelodysplastic Syndrome (8). 383 items were reported by the patients under different themes and subthemes. 117 of these items were reported by more than 5% of the patients. 149 items were selected by the data definition panel to be included in the prototype instrument. The most prevalent QoL issues important to HM patients (Figure 1) were: 'eating and drinking habits (57 patients changed eating and drinking habits; 48 reported loss of appetite; 29 stopped drinking alcohol; and 11 reported increase in appetite); impaired social life and participatory function (86); impaired physical ability or independency (71); disturbed sleep (66); impaired psychological well-being (64); impaired daily activities (61); impaired ability to go on holidays or travelling (60); impaired work life & studies (57 ); impaired sexual life (55); impaired ability to manage finances (34); recreational activities and pastime (32); and relationships (26), from high to low prevalence, respectively. With respect to disease related symptoms, 102 issues were identified, the most prevalent being 'tiredness (65), feeling unwell (28), breathlessness (24), lack of energy (21), back pain (17) and weight loss (17)", from high to low prevalence respectively. Out of 124 treatment related symptoms identified, the most prevalent were: 'tiredness (73); feeling sick (36); lack of energy (20); taste disturbance (20); breathlessness (15); and diarrhoea (15)', from high to low prevalence respectively. Conclusion: The findings of the qualitative and item generation phase clearly indicate that HMs affect patients' QoL significantly. However, in the absence of a validated measure for use in routine clinical practice, this is not captured in a systematic manner. Thus, this highlights the need for the development and validation of a new HM-specific PRO measure for use in such settings. Psychometric testing of the prototype instrument will be carried out to establish the measurement properties of the new HM-specific PRO measure. Figure Figure. Disclosures Salek: EHA: Other: Educational and travel grant. Fielding:Amgen: Membership on an entity's Board of Directors or advisory committees; Baxalta: Membership on an entity's Board of Directors or advisory committees; Pfizer: Membership on an entity's Board of Directors or advisory committees. Kell:Celgene: Honoraria; Novartis: Honoraria; Sunesis: Equity Ownership. Oliva:Pharma Companies: Honoraria; Italy: Speakers Bureau; Pharma Companies: Consultancy. Ionova:BMS: Research Funding; MSD: Other: lecturer bureau.

scholarly journals A narrative review of current evidence supporting the implementation of electronic patient-reported outcome measures in the management of chronic diseases

2021 ◽  
Vol 12 ◽  
pp. 204062232110159
Author(s):  
Olalekan Lee Aiyegbusi ◽  
Devika Nair ◽  
John Devin Peipert ◽  
Kara Schick-Makaroff ◽  
Istvan Mucsi
Keyword(s):  
Clinical Practice ◽  
Chronic Diseases ◽  
Patient Reported Outcomes ◽  
Healthcare Providers ◽  
Patient Reported Outcome ◽  
Routine Clinical Practice ◽  
Narrative Review ◽  
Current Evidence ◽  
Patient Reported ◽  
The Impact

An application of telemedicine of growing interest and relevance is the use of personal computers and mobile devices to collect patient-reported outcomes (PROs). PROs are self-reports of patients’ health status without interpretation by anyone else. The tools developed to assess PROs are known as patient-reported outcomes measures (PROMs). The technological innovations that have led to an increased ownership of electronic devices have also facilitated the development of electronic PROMs (ePROMs). ePROMs are a conduit for telemedicine in the care of patients with chronic diseases. Various studies have demonstrated that the use of ePROMs in routine clinical practice is both acceptable and feasible with patients increasingly expressing a preference for an electronic mode of administration. There is increasing evidence that the use of electronic patient-reported outcome (ePROMs) could have significant impacts on outcomes valued by patients, healthcare providers and researchers. Whilst the development and implementation of these systems may be initially costly and resource-intensive, patient preferences and existing evidence to support their implementation suggests the need for continued research prioritisation in this area. This narrative review summarises and discusses evidence of the impact of ePROMs on clinical parameters and outcomes relevant to chronic diseases. We also explore recently published literature regarding issues that may influence the robust implementation of ePROMs for routine clinical practice.

scholarly journals The impact of patient-reported outcome measures in clinical practice for pain: a systematic review

2016 ◽  
Vol 26 (2) ◽  
pp. 245-257 ◽  
Author(s):  
Michelle M. Holmes ◽  
George Lewith ◽  
David Newell ◽  
Jonathan Field ◽  
Felicity L. Bishop
Keyword(s):  
Systematic Review ◽  
Clinical Practice ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
The Impact

scholarly journals Patient Reported Outcome Measure in Atopic Dermatitis Patients Treated with Dupilumab: 52-Weeks Results

2021 ◽  
Author(s):  
Servando E. Marron ◽  
Lucia Tomas-Aragones ◽  
Carlos A. Moncin-Torres ◽  
Manuel Gomez-Barrera ◽  
Francisco Javier Garcia-Latasa de Aranibar
Keyword(s):  
Quality Of Life ◽  
Atopic Dermatitis ◽  
Clinical Practice ◽  
Stressful Life Events ◽  
Severe Disease ◽  
Patient Reported Outcome ◽  
Routine Clinical Practice ◽  
Severe Atopic Dermatitis ◽  
Patient Reported

Dupilumab is used to treat atopic dermatitis patients who have proven to be refractory to previous treatments. The aim of this study was to assess evolution and patient reported outcome measures in adult patients with moderate-to-severe atopic dermatitis treated with Dupilumab in routine clinical practice. The outcomes were evaluated and registered at baseline and weeks-16, 40 and 52. The variables evaluated were: diseases severity, pruritus, stressful life events, difficulty to sleep, anxiety and depression, quality of life, satisfaction, adherence to the treatment, efficacy and safety. Eleven patients were recruited between Nov 14th 2017 and Jan 16th 2018. Demographic variables: 90% Caucasian, 82% women. Clinical variables: Mean duration of AD =17.7 (±12.8), 91% had severe disease severity. At baseline, SCORAD mean score = 61.7 (±15.5); itch was reported by 100% of patients; itch Visual Analogue Scale mean range of 8 (6-10); HADS mean total score =13.9 (±5.5); DLQI mean score =13.3 (±8.3): EQ-5D-3L mean range = 57 (30-99). At week-52 there is a significant reduction of SCORAD scores, HADS total score and improved quality of life. ¡This study confirms that Dupilumab, used for 52-weeks under routine clinical practice, maintains the improved atopic dermatitis signs and symptoms obtained at week-16, with a good safety profile.

Final Development of the First Generic Quality of Life and Symptoms Measure Specific for Hematological Malignancies: The HM-PRO

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3484-3484
Author(s):  
Pushpendra Goswami ◽  
Tatiana Ionova ◽  
Esther Natalie Oliva ◽  
Roger Else ◽  
Jonathan Kell ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Board Of Directors ◽  
Hematological Malignancies ◽  
Advisory Committees ◽  
Face To Face ◽  
Confirmatory Factor ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
The Impact

Background Health related quality of life (HRQoL) of patients with hematological malignancies (HMs) is greatly affected by the disease and the treatment and this has not been captured in a systematic manner in routine clinical practice. A systematic review of the HRQoL issues and instruments used in HM showed gaps between what is important to patients and what is being measured. The aims of this study were to develop and validate an instrument for measuring the impact of HMs and their treatment on patients' HRQoL and symptoms in daily clinical practice. Methods A multicentre Ethics approval was obtained from the National Research Ethics Service (NRES) of South West Bristols, UK. Adult patients with HM as per 2016 Revised WHO classification, capable of reading English and able to give written informed consent, were recruited from inpatient/outpatient clinics of seven secondary care hospitals in England and Wales for all phases of the study (Table 1). The qualitative study employed semi-structured face-to-face interviews. The generated items were then discussed in data definition panel meeting for inclusion in the prototype version of the new instrument. The content validation was followed by item reduction phase where number of items were reduced to more confined set of items. The version of HM-PRO developed after exploratory factory analysis and confirmatory factor analysis was used to perform Rasch modeling. In next step for demonstrating validity of the HM-PRO, construct and convergent/divergent validity was studied. The score banding was developed and MCID was established. Results Face-to-face interviews were performed in 129 patients. The content analysis of the transcribed interviews resulted in a comprehensive item pool. The generated items included in the prototype HM-PRO, were 34 items for impact on HRQoL category (Part A) and 23 items representing disease signs and symptoms (Part B). The version after exploratory and confirmatory factor analysis was used to perform Rasch modeling in 182 patients resulting in 24 items in Part A and 18 items in Part B. The reliability testing (n=150) showed strong stability of measurement with Cronbach's alpha estimates of the HM-PRO for both assessment points (t1 and t2) above 0.9 for Part A, and above 0.8 for Part B. The intraclass correlation coefficient (ICC) for four domains of Part A was 0.85 - 0.91 and >0.8 for the overall scores of Part A and Part B, for all ten diagnoses, confirming strong reliability of the HM-PRO. Construct and convergent/divergent validity were studied in 905 cases. The HM-PRO scores correlated with scores of EORTC QLQ-C30 and FACT-G, both at the scale and at individual item levels. The univariate regression analysis confirmed a strong relationship between the HM-PRO and the other two measures. For the majority of regression models, the HM-PRO domains and individual items explained more than 50% of the variance in domain and item scores of EORTC QLQ-C30 and FACT-G, confirming the construct validity. The responsiveness testing (n=299) showed that HM-PRO is responsive to small but clinically important change in patients' HRQoL. The score banding was developed for the HM-PRO for its interpretability in to day-to-day clinical practice. The MCID for Part A based on standard error of mean was 6.2 and for PART B 5.9 points. It therefore was prudent, for practical reasons, to propose MCID of '6' for the HM-PRO. Conclusion This study provides evidence of the value of HM-PRO through content validity, reliability, construct validity, responsiveness and interpretability. The current research has developed and validated the English version of the HM-PRO. Further studies are being carried out to validate the instrument in several global languages for cross-cultural adaptation. Furthermore, the Acute Leukaemia Advocates Network (ALAN) is using the HM-PRO in a multi-country survey including five European (French, German, Italian, Portuguese, Spanish) and five non-European countries (Chinese, Hebrew, Korean, Japanese, Russian) to generate data in order to understand the impact of the disease and treatment on patients with different types of leukaemia. The data collected from this survey will be used to further validate the HM-PRO specifically in patients with leukaemia and potentially improve management of patients and their treatment. The HM-PRO has good potential to be widely used in daily clinical practice as well as in hemato-oncology clinical trials. Disclosures Kell: Novartis: Membership on an entity's Board of Directors or advisory committees; Jazz Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees. Fielding:Incyte: Consultancy; Pfizer: Consultancy; Novartis: Consultancy; Amgen: Consultancy. Collins:Gilead: Consultancy, Honoraria. Salek:Pfizer: Honoraria, Speakers Bureau; Agios Pharmaceuticals, Inc.: Consultancy, Honoraria; Merck: Consultancy.

scholarly journals The use of patient-reported outcome research in modern ophthalmology: impact on clinical trials and routine clinical practice

2019 ◽  
Vol Volume 10 ◽  
pp. 9-24 ◽  
Author(s):  
Tasanee Braithwaite ◽  
Melanie Calvert ◽  
Alastair Gray ◽  
Konrad Pesudovs ◽  
Alastair Denniston
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Outcome Research ◽  
Patient Reported Outcome ◽  
Routine Clinical Practice ◽  
Patient Reported

scholarly journals Symptoms, Health-Related Quality of Life, and Tolerability of Loncastuximab Tesirine in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphoma

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 3-4
Author(s):  
Alexander I. Spira ◽  
Lei Chen ◽  
Xiaolei Zhou ◽  
Ari Gnanasakthy ◽  
Luqiang Wang ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
B Cell Lymphoma ◽  
Patient Reported Outcome ◽  
Publicly Traded ◽  
Advisory Committees ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related ◽  
Night Sweats

Introduction In the ongoing single-arm, open-label phase 2 ADCT-402-201 study (LOTIS 2, NCT03589469), loncastuximab tesirine has shown substantial antitumor activity with a manageable toxicity profile in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who had failed ≥ 2 prior therapies, including activity in patients with high-risk disease characteristics. The overall response rate was 48.3% (based on positron emission tomography-computed tomography [PET-CT] assessed by independent review according to Lugano response criteria), and the median duration of response was 10.25 months. This analysis examined symptoms, health-related quality of life (HRQoL), and tolerability using validated patient-reported outcome instruments. Methods Enrolled patients aged ≥18 years received loncastuximab tesirine as an intravenous infusion on day 1 of each 3-week treatment cycle. Responders were defined as patients with a best overall response of complete or partial response. Patient-reported symptoms and HRQoL were measured using the Functional Assessment of Cancer Treatment-Lymphoma (FACT-Lym) and EQ-5D 5 Levels (EQ-5D-5L) instruments at baseline, day 1 of each cycle while patients were treated, and at the end-of-treatment visit. Descriptive statistics for change from baseline scores and percentages of patients with improved, stable, or worsened symptoms were summarized by visit and clinical response. Analysis was conducted using data collected from study initiation (1 August 2018) through 6 April 2020. Results The 145 patients enrolled in this study had a median age of 66 years (range, 23-94). Patients were heavily pretreated, with a median of 3 (range, 2-7) prior systemic therapies. A baseline patient-reported outcome score and at least one post-baseline score were available for 130 patients. Completion rates among patients treated at each visit were ≥ 92% for EQ-5D-5L and ≥ 88% for FACT-Lym up to cycle 9, day 1 (24 weeks). After cycle 9, fewer than 20 patients had patient-reported outcome scores available for analysis. For symptoms assessed in the FACT-Lym lymphoma subscale, pain in certain parts of the body, lumps/swelling, trouble sleeping at night, and fatigue were the most frequently reported symptoms at baseline (34%-59% reported "somewhat" to "very much"). Most patients (≥ 80%) reported "not at all" or "a little bit" at baseline for fever, night sweats, losing weight, itching, and loss of appetite. During the course of treatment, higher percentages of patients reported improvement than worsening for pain and lumps/swelling for the majority of visits. Fever, night sweats, and losing weight did not change for most patients. Itching was the only symptom for which more patients experienced worsening than improvement. For other symptoms, similar percentages of patients reported improvement and worsening. The mean change from baseline in EQ-5D Visual Analog Scale (VAS) score showed a trend of improvement in overall health over time (see figure). The mean VAS change reached the minimally important difference of 7 points at cycle 8, day 1. This improvement was associated with clinical response. When patients were asked how much they were bothered by side effects of treatment, most patients (> 60%) reported "not at all" or "a little bit" for all visits throughout the treatment. Conclusions Results of this analysis suggest that patients who responded to treatment with loncastuximab tesirine generally had stable or improved symptoms and overall HRQoL. The treatment was well tolerated by patients. These findings further support the clinical use of loncastuximab tesirine for the treatment of relapsed or refractory DLBCL. Disclosures Spira: ADCT:Research Funding;Janssen:Consultancy;Incyte:Consultancy;BMS:Consultancy;Merck:Consultancy;Novartis:Consultancy;Takeda:Consultancy;Cardiff Oncology:Research Funding.Chen:ADCT:Current Employment, Current equity holder in publicly-traded company.Zhou:ADCT:Research Funding.Gnanasakthy:ADCT:Research Funding.Wang:ADC Therapeutics America, inc:Current Employment, Current equity holder in publicly-traded company.Ungar:ADC Therapeutics:Current Employment, Current equity holder in publicly-traded company.Curiel:ADCT:Current Employment, Current equity holder in publicly-traded company.Radford:GlaxoSmithKline:Current equity holder in publicly-traded company, Other: Spouse;AstraZeneca:Current equity holder in publicly-traded company, Other: Spouse;Takeda:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau;Pfizer:Research Funding;ADCT:Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding;BMS:Consultancy, Honoraria, Speakers Bureau;Novartis:Consultancy, Honoraria;Seattle Genetics:Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.Kahl:AstraZeneca Pharmaceuticals LP:Consultancy, Membership on an entity's Board of Directors or advisory committees;Celgene Corporation:Consultancy;Genentech:Consultancy;Pharmacyclics LLC:Consultancy;Roche Laboratories Inc:Consultancy;BeiGene:Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding;Janssen:Consultancy, Membership on an entity's Board of Directors or advisory committees;Acerta:Consultancy, Research Funding;ADC Therapeutics:Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding;AbbVie:Consultancy.

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