Explaining Dichotomous Outcomes

2021 ◽  
pp. 488-520
Author(s):  
Thomas J. Linneman
Keyword(s):  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
I. E. Ceyisakar ◽  
N. van Leeuwen ◽  
Diederik W. J. Dippel ◽  
Ewout W. Steyerberg ◽  
H. F. Lingsma

Abstract Background There is a growing interest in assessment of the quality of hospital care, based on outcome measures. Many quality of care comparisons rely on binary outcomes, for example mortality rates. Due to low numbers, the observed differences in outcome are partly subject to chance. We aimed to quantify the gain in efficiency by ordinal instead of binary outcome analyses for hospital comparisons. We analyzed patients with traumatic brain injury (TBI) and stroke as examples. Methods We sampled patients from two trials. We simulated ordinal and dichotomous outcomes based on the modified Rankin Scale (stroke) and Glasgow Outcome Scale (TBI) in scenarios with and without true differences between hospitals in outcome. The potential efficiency gain of ordinal outcomes, analyzed with ordinal logistic regression, compared to dichotomous outcomes, analyzed with binary logistic regression was expressed as the possible reduction in sample size while keeping the same statistical power to detect outliers. Results In the IMPACT study (9578 patients in 265 hospitals, mean number of patients per hospital = 36), the analysis of the ordinal scale rather than the dichotomized scale (‘unfavorable outcome’), allowed for up to 32% less patients in the analysis without a loss of power. In the PRACTISE trial (1657 patients in 12 hospitals, mean number of patients per hospital = 138), ordinal analysis allowed for 13% less patients. Compared to mortality, ordinal outcome analyses allowed for up to 37 to 63% less patients. Conclusions Ordinal analyses provide the statistical power of substantially larger studies which have been analyzed with dichotomization of endpoints. We advise to exploit ordinal outcome measures for hospital comparisons, in order to increase efficiency in quality of care measurements. Trial registration We do not report the results of a health care intervention.


2021 ◽  
Vol 38 (6) ◽  
pp. 375-384
Author(s):  
Laura Schlenker ◽  
Renee C.B. Manworren

Background: While recommended timing of pegfilgrastim administration is ≥24 h after chemotherapy, patient barriers to next day administration, available adult evidence, and pharmacokinetic data have led to earlier administration in some pediatric patients with solid and central nervous system tumors. The purpose of this study was to compare patient outcomes by timing of pegfilgrastim after chemotherapy. Methods: A retrospective chart review examined timing of 932 pegfilgrastim administrations to 182 patients, 0–29 years of age. The primary outcome was febrile neutropenia (FN); the secondary outcome was neutropenic delays (ND) ≥7 days to next chemotherapy cycle. To account for multiple pegfilgrastim administrations per patient, a generalized mixed model was used with a logit link for the dichotomous outcomes (FN & ND), timing as the dichotomous independent variable, and random effect for patient. Results: FN occurred in 196 of 916 cycles (21.4%); and ND in 19 of 805 cycles (2.4%). The fixed effect of pegfilgrastim administration < or ≥24 h after chemotherapy was not significant, p = .50; however, earlier or later than 20 h was significant, p = .005. FN odds were significantly higher when pegfilgrastim was given <20 h (OR 1.78, 95% CI: 1.19–2.65) after chemotherapy, which may be attributable to differences in chemotherapy toxicity regardless of pegfilgrastim timing. Discussion: While attempts should be made to administer pegfilgrastim ≥24 h after chemotherapy, if barriers exist, modified timing based on individual patient characteristics should be considered. Prospective randomized trials are needed to identify lower risk patients for early pegfilgrastim administration.


2009 ◽  
Vol 12 (7) ◽  
pp. A388
Author(s):  
M Bhanderi ◽  
S Dimri ◽  
A Mahajan ◽  
BS Ubhadiya ◽  
P Narvilkar ◽  
...  

2021 ◽  
pp. 115-135
Author(s):  
Jos W. R. Twisk
Keyword(s):  

1993 ◽  
Vol 4 (5) ◽  
pp. 279-287 ◽  
Author(s):  
Lionel A Mandell ◽  
Pierre L Turgeon ◽  
Allan R Ronalds ◽  
The Canadian Clinical Trials Group

Objective: A Canadian multicentre clinical trial in the treatment of intra-abdominal and pelvic infections to compare the efficacy and safety of monotherapy using imipenem-cilastatin (imipenem) (500 mg intravenously every 6 h) versus combination therapy with clindamycin/tobramycin (clindamycin 600 mg intravenously every 6 h and tobramycin 1.7 mg/kg intravenously every 8 h).Methods: Two hundred and fifty patients were entered (88 definite and 162 possible infections) and all were evaluable for analysis of adverse events and intention to treat analysis of efficacy. Dichotomous outcomes used were: cured versus noncured (improved, failed, relapsed).Results: No statistically significant differences were found with the intention to treat analysis (P=0.88) or with definite infections (P=0.81). For overall bacteriological response, no significant differences were noted (P=0.1). Eleven and 15 patients on imipenem and clindamycin/tobramycin, respectively, were colonized with bacteria. Enterococci colonized four of 11 imipenem cases and five of 15 clindamycin/tobramycin cases while fungi colonized six patients on imipenem and four on clindamycin/tobramycin. Five patients on imipenem and seven on clindamycin/tobramycin developed superinfection. In the imipenem group, one case had a bacterial superinfection while four cases were due toCandida albicans.Seven of seven superinfections on clindamycin/tobramycin were bacterial. Three bacteria initially sensitive to the assigned study drug developed resistance. In two patients on imipenem,Enterococcus faecalisandPseudomonas aeruginosabecame resistant after 14 and 10 days of therapy, respectively. On clindamycin/tobramycin, one instance ofBacteroides fragilisresistance after eight days of therapy was seen. Eighty-three adverse events occurred; 47 in the imipenem group and 36 in the clindamycin/tobramycin group. This resulted in discontinuation of antibacterial therapy in 13 patients, seven of whom were on imipenem and six on clindamycin/tobramycin. Comparison of adverse effects showed statistically significant differences for nausea (P=0.02) and hepatotoxicity (P=0.05) occurring with greater frequency in the imipenem and clindamycin/tobramycin groups, respectively.Conclusions: These data support the conclusion that monotherapy with imipenem (500 mg intravenously every 6 h) is as efficacious as clindamycin/tobramycin for treatment of intra-abdominal and pelvic infections. Both regimens are well tolerated.


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