Abstract
The quality of life of patients is an important component of evaluation of therapies. We present an overview of a statistical method called Q-TWiST (Quality-Adjusted Time Without Symptoms and Toxicity) which incorporates quality-of-life considerations into treatment comparisons. Multivariate censored survival data are used to partition the overall survival time into periods of time spent in a set of progressive clinical health states which may differ in quality of life. Mean health state durations, restricted to the follow-up limits of the clinical trial, are derived from the data and combined with value weights to estimate quality- adjusted survival. The methodology emphasizes treatment comparisons based on threshold utility analyses that high-light trade-offs between different health state durations; it is not intended to provide a unique result combining quality and quantity of life. We also describe three recent ex-tensions of the methodology: Covariates can be included using proportional hazards and accelerated failure time regression models, restricted estimates can be projected beyond follow-up limits using parametric models, and meta-analyses can be performed incorporating quality-of- life dimensions. The basic methods are demonstrated in an analysis of data from a clinical trial comparing long versus short duration adjuvant chemotherapy regimens for the treatment of breast cancer. The clinical health states are defined by the following three outcomes: (1) end of treatment toxicity, (2) disease recurrence, and (3) death. The results allow one to evaluate the trade-off between the increased toxic effects and the increased recurrence-free interval associated with the long duration treatment.
| Original language | English |
|---|---|
| Pages (from-to) | 161-169 |
| Number of pages | 9 |
| Journal | American Statistician |
| Volume | 49 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - 1995 |
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Keywords
- Clinical trials
- Quality of life
- Restricted means
- Survival analysis
- Utility
ASJC Scopus subject areas
- Mathematics(all)
- Statistics and Probability
- Statistics, Probability and Uncertainty
Cite this
Comparing treatments using quality-adjusted survival : The q-twist method. / Gelber, Richard D.; Cole, Bernard F.; Gelber, Shari; Goldhirsch, Aron.
In: American Statistician, Vol. 49, No. 2, 1995, p. 161-169.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Comparing treatments using quality-adjusted survival
T2 - The q-twist method
AU - Gelber, Richard D.
AU - Cole, Bernard F.
AU - Gelber, Shari
AU - Goldhirsch, Aron
PY - 1995
Y1 - 1995
N2 - The quality of life of patients is an important component of evaluation of therapies. We present an overview of a statistical method called Q-TWiST (Quality-Adjusted Time Without Symptoms and Toxicity) which incorporates quality-of-life considerations into treatment comparisons. Multivariate censored survival data are used to partition the overall survival time into periods of time spent in a set of progressive clinical health states which may differ in quality of life. Mean health state durations, restricted to the follow-up limits of the clinical trial, are derived from the data and combined with value weights to estimate quality- adjusted survival. The methodology emphasizes treatment comparisons based on threshold utility analyses that high-light trade-offs between different health state durations; it is not intended to provide a unique result combining quality and quantity of life. We also describe three recent ex-tensions of the methodology: Covariates can be included using proportional hazards and accelerated failure time regression models, restricted estimates can be projected beyond follow-up limits using parametric models, and meta-analyses can be performed incorporating quality-of- life dimensions. The basic methods are demonstrated in an analysis of data from a clinical trial comparing long versus short duration adjuvant chemotherapy regimens for the treatment of breast cancer. The clinical health states are defined by the following three outcomes: (1) end of treatment toxicity, (2) disease recurrence, and (3) death. The results allow one to evaluate the trade-off between the increased toxic effects and the increased recurrence-free interval associated with the long duration treatment.
AB - The quality of life of patients is an important component of evaluation of therapies. We present an overview of a statistical method called Q-TWiST (Quality-Adjusted Time Without Symptoms and Toxicity) which incorporates quality-of-life considerations into treatment comparisons. Multivariate censored survival data are used to partition the overall survival time into periods of time spent in a set of progressive clinical health states which may differ in quality of life. Mean health state durations, restricted to the follow-up limits of the clinical trial, are derived from the data and combined with value weights to estimate quality- adjusted survival. The methodology emphasizes treatment comparisons based on threshold utility analyses that high-light trade-offs between different health state durations; it is not intended to provide a unique result combining quality and quantity of life. We also describe three recent ex-tensions of the methodology: Covariates can be included using proportional hazards and accelerated failure time regression models, restricted estimates can be projected beyond follow-up limits using parametric models, and meta-analyses can be performed incorporating quality-of- life dimensions. The basic methods are demonstrated in an analysis of data from a clinical trial comparing long versus short duration adjuvant chemotherapy regimens for the treatment of breast cancer. The clinical health states are defined by the following three outcomes: (1) end of treatment toxicity, (2) disease recurrence, and (3) death. The results allow one to evaluate the trade-off between the increased toxic effects and the increased recurrence-free interval associated with the long duration treatment.
KW - Clinical trials
KW - Quality of life
KW - Restricted means
KW - Survival analysis
KW - Utility
UR - http://www.scopus.com/inward/record.url?scp=21844481778&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=21844481778&partnerID=8YFLogxK
U2 - 10.1080/00031305.1995.10476135
DO - 10.1080/00031305.1995.10476135
M3 - Article
AN - SCOPUS:21844481778
VL - 49
SP - 161
EP - 169
JO - American Statistician
JF - American Statistician
SN - 0003-1305
IS - 2
ER -