Health-Related Quality of Life and Treatment of Older Adults with Acute Myeloid Leukemia: a Young International Society of Geriatric Oncology Review Paper

Loh, Kah Poh; Abdallah, Maya; Kumar, Anita J.; Neuendorff, Nina Rosa; Dahiya, Saurabh; Klepin, Heidi D.

doi:10.1007/s11899-019-00552-6

Cited by 20 publications

(23 citation statements)

References 85 publications

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“…The importance of quality-of-life, as well as quantity of life, gained with a novel intervention compared to the older, conventional therapy, is increasingly recognized in cancer management as an important element when evaluating various treatment options, including in patients with AML whose health-related quality-of-life is impaired [15][16][17][18]. For example, a new cancer therapy may prolong survival, but the added time may come predominantly from time spent with serious AEs that negatively affect the quality-of-life of patients.…”

Section: Discussionmentioning

confidence: 99%

Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of CPX-351 versus 7 + 3 in older adults with newly diagnosed high-risk/secondary AML

Cortes

Lin

et al. 2021

J Hematol Oncol

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Background CPX-351 (United States: Vyxeos®; Europe: Vyxeos® Liposomal), a dual-drug liposomal encapsulation of daunorubicin and cytarabine in a synergistic 1:5 molar ratio, is approved by the US FDA and the EMA for the treatment of adults with newly diagnosed therapy-related acute myeloid leukemia or acute myeloid leukemia with myelodysplasia-related changes. In a pivotal phase 3 study that evaluated 309 patients aged 60 to 75 years with newly diagnosed high-risk/secondary acute myeloid leukemia, CPX-351 significantly improved median overall survival versus conventional 7 + 3 chemotherapy (cytarabine continuous infusion for 7 days plus daunorubicin for 3 days), with a comparable safety profile. A Quality‐adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of the phase 3 study was performed to compare survival quality between patients receiving CPX-351 versus conventional 7 + 3 after 5 years of follow-up. Methods Patients were randomized 1:1 between December 20, 2012 and November 11, 2014 to receive induction with CPX-351 or 7 + 3. Survival time for each patient was partitioned into 3 health states: TOX (time with any grade 3 or 4 toxicity or prior to remission), TWiST (time in remission without relapse or grade 3 or 4 toxicity), and REL (time after relapse). Within each treatment arm, Q-TWiST was calculated by adding the mean time spent in each health state weighted by its respective quality-of-life, represented by health utility. The relative Q-TWiST gain, calculated as the difference in Q-TWiST between treatment arms divided by the mean survival of the 7 + 3 control arm, was determined in order to evaluate results in the context of other Q-TWiST analyses. Results The relative Q-TWiST gain with CPX-351 versus 7 + 3 was 53.6% in the base case scenario and 39.8% among responding patients. Across various sensitivity analyses, the relative Q-TWiST gains for CPX-351 ranged from 48.0 to 57.6%, remaining well above the standard clinically important difference threshold of 15% for oncology. Conclusions This post hoc analysis demonstrates that CPX-351 improved quality-adjusted survival, further supporting the clinical benefit in patients with newly diagnosed high-risk/secondary acute myeloid leukemia. Trial registration This trial was registered on September 28, 2012 at www.clinicaltrials.gov as NCT01696084 (https://clinicaltrials.gov/ct2/show/NCT01696084) and is complete.

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Section: Discussionmentioning

confidence: 99%

Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of CPX-351 versus 7 + 3 in older adults with newly diagnosed high-risk/secondary AML

Cortes

Lin

et al. 2021

J Hematol Oncol

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“…On the other hand, HRQoL‐disease‐specific instruments can better assess key disease‐ and patient‐related symptoms as well as psychosocial aspects 30 . In contrast, qualitative assessments can provide ‘an in‐depth understanding of patient experiences that may not otherwise be captured through the use of standardised questionnaires’ 36 . An advantage of the generic EQ‐5D instrument used in this study is that it also results in a single score, a so‐called utility, which can be directly implemented in health economic analyses, which evaluate quality‐adjusted life years gained by an intervention.…”

Section: Discussionmentioning

confidence: 99%

Development of a core outcome set for myelodysplastic syndromes – a Delphi study from the EUMDS Registry Group

et al. 2020

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Summary Treatment options for myelodysplastic syndromes (MDS) vary widely, depending on the natural disease course and patient‐related factors. Comparison of treatment effectiveness is challenging as different endpoints have been included in clinical trials and outcome reporting. Our goal was to develop the first MDS core outcome set (MDS‐COS) defining a minimum set of outcomes that should be reported in future clinical studies. We performed a comprehensive systematic literature review among MDS studies to extract patient‐ and/or clinically relevant outcomes. Clinical experts from the European LeukemiaNet MDS (EUMDS) identified 26 potential MDS core outcomes and participated in a three‐round Delphi survey. After the first survey (56 experts), 15 outcomes met the inclusion criteria and one additional outcome was included. The second round (38 experts) resulted in six included outcomes. In the third round, a final check on plausibility and practicality of the six included outcomes and their definitions was performed. The final MDS‐COS includes: health‐related quality of life, treatment‐related mortality, overall survival, performance status, safety, and haematological improvement. This newly developed MDS‐COS represents the first minimum set of outcomes aiming to enhance comparability across future MDS studies and facilitate a better understanding of treatment effectiveness.

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“…Although the EORTC QLQ-C30 has been validated in patients with cancer and utilized in a third of prior AML studies, it is neither disease nor treatment-specific [ 5 , 6 ]. In addition, pre-defined deterioration thresholds of ≥10, 7, and 5 points in EORTC QLQ-C30, EQ-5D-5L VAS, and PROMIS Fatigue, respectively, may not always capture clinically meaningful changes in function.…”

Section: Health-related Quality-of-life With Venetoclax Combination T...mentioning

confidence: 99%

To live is well but to live well is better: venetoclax combination therapy and quality-of-life in acute myeloid leukemia

Gangat

Tefferi

2022

Blood Cancer J.

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Health-Related Quality of Life and Treatment of Older Adults with Acute Myeloid Leukemia: a Young International Society of Geriatric Oncology Review Paper

Cited by 20 publications

References 85 publications

Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of CPX-351 versus 7 + 3 in older adults with newly diagnosed high-risk/secondary AML

Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of CPX-351 versus 7 + 3 in older adults with newly diagnosed high-risk/secondary AML

Development of a core outcome set for myelodysplastic syndromes – a Delphi study from the EUMDS Registry Group

To live is well but to live well is better: venetoclax combination therapy and quality-of-life in acute myeloid leukemia

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