scholarly journals Faculty Opinions recommendation of Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial.

2019 ◽  
Author(s):  
Stephen Hunger ◽  
Caitlin Elgarten
Keyword(s):  
Quality Of Life ◽  
Young Adults ◽  
Acute Lymphoblastic Leukaemia ◽  
B Cell ◽  
Lymphoblastic Leukaemia ◽  
Phase 2 ◽  
Phase 2 Trial ◽  
Patient Reported ◽  
Children And Young Adults

Quality of life Evaluation in patients receiving Steroids (the QuESt tool): initial development in children and young people with acute lymphoblastic leukaemia

2015 ◽  
Vol 101 (3) ◽  
pp. 241-246 ◽  
Author(s):  
M Adams ◽  
M Robling ◽  
J Grainger ◽  
J Tomlins ◽  
A Johnson ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Young Adults ◽  
Acute Lymphoblastic Leukaemia ◽  
Family Relationships ◽  
Lymphoblastic Leukaemia ◽  
Cognitive Interviewing ◽  
Initial Development ◽  
Children And Young Adults ◽  
The Impact

BackgroundThe powerful cytotoxic and immunomodulatory effects of corticosteroids are an important element of the success that has been achieved in the treatment of acute lymphoblastic leukaemia (ALL). In addition to physical side effects, corticosteroids can adversely influence behaviour, cognitive function and mood leading to significantly impaired quality of life (QoL). A number of tools exist for assessing QoL, but none of these specifically examines changes attributable to steroids.MethodsChildren and young adults aged 8–24 years and parents of children receiving maintenance therapy for ALL from four UK centres were invited to participate. The study comprised three stages carried out over 2 years: (1) focus groups and interviews where participants were asked to describe their experiences of dexamethasone; (2) analysis of questionnaires sent to healthcare professionals and patients to evaluate the importance and relevance of the questions; and (3) cognitive interviewing.ResultsInterpretative phenomenological analysis of focus group and interview transcripts identified that dexamethasone adversely influenced behaviour, appetite, body image, mood and family relationships. 157 electronic survey responses were analysed leading to further item development. Cognitive interviewing confirmed face validity and internal consistency. QuESt comprises 28 questions within four domains and has three age-specific versions.ConclusionsQuESt is the first treatment-specific QoL measure for children and young adults receiving corticosteroids. It can be completed in 10–15 min by children aged ≥8 years. Further validity and reliability testing will be undertaken. Although the initial application is for ALL, QuESt may also be a valuable tool for understanding the impact of corticosteroids in other paediatric conditions.

scholarly journals Health-related Quality of Life in Patients With Recurrent Pericarditis: Results From a Phase 2 Study of Rilonacept

2021 ◽  
Author(s):  
David Lin ◽  
Allan Klein ◽  
David Cella ◽  
Anna Beutler ◽  
Fang Fang ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Qualitative Interviews ◽  
Phase 2 ◽  
Health Related Quality ◽  
Recurrent Pericarditis ◽  
Phase 2 Trial ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

Abstract Background: Impact of recurrent pericarditis (RP) on patient health-related quality of life (HRQoL) was evaluated through qualitative patient interviews and as an exploratory endpoint in a Phase 2 trial evaluating the efficacy and safety of rilonacept (IL-1α/IL-1β blocker) to treat RP.Methods: Qualitative interviews were conducted with ten adults with RP to understand symptoms and HRQoL impacts, and the 10-item Patient-Reported Outcomes Measurement Information System Global Health (PROMIS Global) v1.2 was evaluated to determine questionnaire coverage of patient experience. The Phase 2 trial enrolled participants with active symptomatic RP (A-RP, n=16) and corticosteroid-dependent participants with no active recurrence at baseline (CSD-RP, n=9). All participants received rilonacept weekly for 6 weeks during a base treatment period (TP) plus an optional 18-week extension period (EP). Concomitant medications, including corticosteroids (CS), were tapered, if possible, during EP. HRQoL was assessed using the PROMIS Global, and patient-reported pain and blood levels of c-reactive protein (CRP) were also collected at Baseline and follow-up periods. Results: Information from qualitative interviews demonstrated that PROMIS GH concepts are relevant to adults with RP. From the Phase 2 trial, both participant groups showed impacted HRQoL at Baseline [mean PROMIS Global Physical Health (GPH) and Global Mental Health (GMH), were lower than population norm average]. In A-RP, GPH/MPH improved by end of base TP and were sustained through EP (similar trends were observed for pain and CRP). Similarly, in CSD-RP, GPH/MPH improved by end of TP and further improved at EP, during CS tapering or discontinuation, without disease recurrence (low pain scores and CRP levels continued during the TP and EP). Conclusion: This is the first study demonstrating impaired HRQoL in RP. Rilonacept treatment was associated with HRQoL improvements using PROMIS GH scores. Maintained/improved HRQoL during tapering/withdrawal of CS without recurrence suggests that rilonacept may provide an alternative to corticosteroids. Trial Registration: ClinicalTrials.Gov; NCT03980522; 5 June 2019, retrospectively registered; https://clinicaltrials.gov/ct2/show/NCT03980522

scholarly journals Cost-Effectiveness Analysis of Tisagenlecleucel in the Treatment of Relapsed or Refractory B-Cell Acute Lymphoblastic Leukaemia in Children and Young Adults in Spain

2020 ◽  
Vol Volume 12 ◽  
pp. 253-264
Author(s):  
Josep-Maria Ribera Santasusana ◽  
Alejandra de Andrés Saldaña ◽  
Nuria García-Muñoz ◽  
Joana Gostkorzewicz ◽  
Diana Martínez Llinàs ◽  
...  
Keyword(s):  
Young Adults ◽  
Cost Effectiveness ◽  
Acute Lymphoblastic Leukaemia ◽  
B Cell ◽  
Lymphoblastic Leukaemia ◽  
Cost Effectiveness Analysis ◽  
Effectiveness Analysis ◽  
Children And Young Adults

scholarly journals Patient-reported quality-of-life outcomes after de-escalated chemoradiation for human papillomavirus-positive oropharyngeal carcinoma: Findings from a phase 2 trial

Cancer ◽  
2017 ◽  
Vol 124 (3) ◽  
pp. 521-529 ◽  
Author(s):  
John V. Hegde ◽  
Narek Shaverdian ◽  
Megan E. Daly ◽  
Carol Felix ◽  
Deborah L. Wong ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Human Papillomavirus ◽  
Oropharyngeal Carcinoma ◽  
Phase 2 ◽  
Life Outcomes ◽  
Phase 2 Trial ◽  
Quality Of Life Outcomes ◽  
Patient Reported

scholarly journals Health-related quality of life in patients with recurrent pericarditis: results from a phase 2 study of rilonacept

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
David Lin ◽  
Allan Klein ◽  
David Cella ◽  
Anna Beutler ◽  
Fang Fang ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Qualitative Interviews ◽  
Phase 2 ◽  
Health Related Quality ◽  
Recurrent Pericarditis ◽  
Phase 2 Trial ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

Abstract Background Impact of recurrent pericarditis (RP) on patient health-related quality of life (HRQoL) was evaluated through qualitative patient interviews and as an exploratory endpoint in a Phase 2 trial evaluating the efficacy and safety of rilonacept (IL-1α/IL-1β cytokine trap) to treat RP. Methods Qualitative interviews were conducted with ten adults with RP to understand symptoms and HRQoL impacts, and the 10-item Patient-Reported Outcomes Measurement Information System Global Health (PROMIS GH) v1.2 was evaluated to determine questionnaire coverage of patient experience. The Phase 2 trial enrolled participants with active symptomatic RP (A-RP, n = 16) and corticosteroid-dependent participants with no active recurrence at baseline (CSD-RP, n = 9). All participants received rilonacept weekly during a 6-week base treatment period (TP) plus an optional 18-week extension period (EP). Tapering of concomitant medications, including corticosteroids (CS), was permitted during EP. HRQoL was assessed using the PROMIS GH, and patient-reported pain and blood levels of c-reactive protein (CRP) were collected at Baseline and follow-up periods. A secondary, descriptive analysis of the Phase 2 trial efficacy results was completed using HRQoL measures to characterize both the impact of RP and the treatment effect of rilonacept. Results Information from qualitative interviews demonstrated that PROMIS GH concepts are relevant to adults with RP. From the Phase 2 trial, both participant groups showed impacted HRQoL at Baseline (mean PROMIS Global Physical Health [GPH] and Global Mental Health [GMH], were lower than population norm average). In A-RP, GPH/MPH improved by end of base TP and were sustained through EP (similar trends were observed for pain and CRP). Similarly, in CSD-RP, GPH/MPH improved by end of TP and further improved during EP, during CS tapering or discontinuation, without disease recurrence (low pain scores and CRP levels continued during the TP and EP). Conclusion This is the first study demonstrating impaired HRQoL in RP. Rilonacept treatment was associated with HRQoL improvements using PROMIS GH scores. Maintained/improved HRQoL during tapering/withdrawal of CS without recurrence suggests that rilonacept may provide an alternative to CS. Trial registration: ClinicalTrials.Gov; NCT03980522; 5 June 2019, retrospectively registered; https://clinicaltrials.gov/ct2/show/NCT03980522.

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