Patients’ Estimation of Overall Treatment Burden: Why Not Ask the Obvious?

2002 ◽  
Vol 20 (1) ◽  
pp. 65-72 ◽  
Author(s):  
J. Bernhard ◽  
R. Maibach ◽  
B. Thürlimann ◽  
C. Sessa ◽  
M. S. Aapro
Keyword(s):  
Wide Spectrum ◽  
Treatment Burden ◽  
Double Blind Trial ◽  
Delayed Emesis ◽  
Clinical Validity ◽  
Self Assessment ◽  
Double Blind ◽  
Specific Effects ◽  
Quality Of Life Measures

PURPOSE: We investigated the clinical validity of patients’ estimation of overall treatment burden. This measure was expected to be responsive to the wide spectrum of reactions on treatment and thus less precise for specific effects. PATIENTS AND METHODS: After the first chemotherapy within a randomized, double-blind trial of the prophylaxis for delayed emesis (SAKK 90/95), 249 patients documented nausea and vomiting daily for 6 days. Over the whole period, they estimated nausea/vomiting (N/V) burden and overall treatment burden by linear analog-self assessment (LASA) indicators and documented other side effects. RESULTS: At day 6, the two burden indicators were moderately correlated (r = 0.58) in accordance with their different concepts. No, partial, or total control of delayed emesis (days 2 to 6) was reflected in a consistent pattern by both indicators, with a stronger and more significant effect (P < .001) on changes in N/V burden than overall treatment burden. In contrast, toxicity other than N/V, assessed independently by patients and physicians, was mainly associated with overall treatment burden. Patients who indicated at least one other side effect rated their overall burden substantially higher than those with no indication of other toxicity (P < .0001). Physician-rated toxicity had a similar effect (P < .0001). CONCLUSION: A direct patient estimation of overall treatment burden by a LASA indicator may serve as an end point in clinical trials, particularly when treatments with different toxicity profiles are being compared. It is complementary to physicians’ ratings of specific toxicities and a major component of patient-rated symptom checklists and quality-of-life measures.

Neonatal Intracranial Hemorrhage and Phenobarbital

PEDIATRICS ◽  
1986 ◽  
Vol 77 (4) ◽  
pp. 443-450
Author(s):  
Karl C. K. Kuban ◽  
Alan Leviton ◽  
Kalpathy S. Krishnamoorthy ◽  
Elizabeth R. Brown ◽  
Rita Littlewood Teele ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Intraparenchymal Hemorrhage ◽  
Double Blind ◽  
Germinal Matrix ◽  
Specific Effects ◽  
Increased Risk ◽  
Severe Hemorrhage ◽  
Institutional Differences ◽  
Phenobarbital Administration

We enrolled 280 intubated babies with birth weights of less than 1,751 g in a double-blind randomized prospective clinical trial to evaluate whether phenobarbital influences the likelihood of developing subependymal-intraventricular-intraparenchymal hemorrhage. Phenobarbital was associated with an increased risk of developing any subependymal-intraventricular-intraparenchymal hemorrhage and was not associated with a diminished risk of either severe hemorrhage or germinal matrix hemorrhage. This increased risk was apparent even after we considered the influence of phenobarbital levels, timing of phenobarbital administrations, institutional differences, quality of ultrasound scans, gestational age- and birth weight-specific effects, ascertainment bias, and other possible confounders of phenobarbital administration.

scholarly journals Randomized, Placebo-Controlled, Double-Blind Pilot Study of D-Cycloserine in Chronic Stroke

2015 ◽  
Vol 2015 ◽  
pp. 1-14 ◽  
Author(s):  
Andrew J. Butler ◽  
Justiss Kallos ◽  
Stephen N. Housley ◽  
Michelle C. LaPlaca ◽  
Stephen F. Traynelis ◽  
...  
Keyword(s):  
Confidence Interval ◽  
Stroke Survivors ◽  
Double Blind ◽  
Quality Of Life Measures ◽  
Nmda Receptor Function ◽  
The Usa ◽  
Physical Therapy Treatment ◽  
Preliminary Study ◽  
Therapy Treatment

Stroke is a leading cause of death and disability in the USA. Up to 60% of patients do not fully recover despite intensive physical therapy treatment. N-Methyl-D-aspartate receptors (NMDA-R) have been shown to play a role in synaptic plasticity when activated. D-Cycloserine promotes NMDA receptor function by binding to receptors with unoccupied glycine sites. These receptors are involved in learning and memory. We hypothesized that D-cycloserine, when combined with robotic-assisted physiotherapy (RAP), would result in greater gains compared with placebo + RAP in stroke survivors. Participants (n=14) were randomized to D-cycloserine plus RAP or placebo plus RAP. Functional, cognitive, and quality-of-life measures were used to assess recovery. There was significant improvement in grip strength of the affected hand within both groups from baseline to 3 weeks (95% confidence interval for mean change, 3.95 ± 2.96 to 4.90 ± 3.56 N for D-cycloserine and 5.72 ± 3.98 to 8.44 ± 4.90 N for control). SIS mood domain showed improvement for both groups (95% confidence interval for mean change, 72.6 ± 16.3 to 82.9 ± 10.9 for D-cycloserine and 82.9 ± 13.5 to 90.3 ± 9.9 for control). This preliminary study does not provide evidence that D-cycloserine can provide greater gains in learning compared with placebo for stroke survivors.

Double-Blind, Placebo-Controlled Multicentre Trial of the Efficacy and Tolerance of Morniflumate Suppositories in the Treatment of Tonsillitis in Children

1990 ◽  
Vol 18 (1) ◽  
pp. 30-36 ◽  
Author(s):  
Y. Manach ◽  
A. Ditisheim
Keyword(s):  
Quality Of Life ◽  
Treatment Efficacy ◽  
Multicentre Trial ◽  
Response To Treatment ◽  
Double Blind Trial ◽  
Acute Tonsillitis ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Before And After

The efficacy and tolerability of morniflumate suppositories used together with phenoxymethylpenicillin were studied in a placebo-controlled, double-blind trial in 101 children with acute tonsillitis. Patients received a suppository containing 400 mg morniflumate or placebo twice daily for 4 days; all patients also received 1 500 000 IU/day phenoxymethylpenicillin. Response to treatment was assessed by clinical examination before and after 2 and 4 days' treatment. Efficacy was evaluated by resolution of oropharyngeal pain, congestion, fever, size and sensitivity of adenopathies, quality of life and duration of sleep. Body temperature fell rapidly after the start of treatment. There was also resolution of pharyngeal pain, earache, dysphagia and adenopathy. Spontaneous pharyngeal pain was present after 4 days in significantly ( P = 0.03) fewer patients receiving morniflumate than receiving placebo. It is suggested that morniflumate combined with antibiotic therapy is an effective and well-tolerated treatment for tonsillitis in children.

scholarly journals Fermented soy supplementation improves indicators of quality of life: a randomized, placebo-controlled, double-blind trial in adults experiencing heartburn

2020 ◽  
Vol 13 (1) ◽  
Author(s):  
Asmaa Fatani ◽  
Kadi Vaher ◽  
Daniela Rivero-Mendoza ◽  
Karima Alabasi ◽  
Wendy J. Dahl
Keyword(s):  
Quality Of Life ◽  
Double Blind Trial ◽  
Double Blind ◽  
Blind Trial

Does biomarker information impact patients’ preferences for therapy?

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 6527-6527
Author(s):  
Ann H. Partridge ◽  
Karen Sepucha ◽  
Anne O'Neill ◽  
Kathy Miller ◽  
Emily Baker ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Treatment Preference ◽  
Treatment Preferences ◽  
Double Blind Trial ◽  
Double Blind ◽  
Increased Risk ◽  
The Impact ◽  
To Receive

6527 Background: Biomarker information can risk stratify patients based on potential for benefits/toxicity from therapy. Ideally, a biomarker will identify those who benefit with limited toxicity. However, the impact of biomarkers is unclear when indicting individuals with greatest benefit are also at increased risk for toxicity. Methods: We surveyed participants at 18 month follow-up in the Decision-Making/Quality of Life (DM-QOL) component of ECOG5103, a RCT where patients were randomized to receive adjuvant chemotherapy for breast cancer with either placebo or bevacizumab (in 2 schedules). We asked patients for the preferred treatment in two hypothetical scenarios: 1) preference for chemo A or chemo A + B without biomarker information; 2) preference for chemo A or chemo A+B when participants tested positive for a “B-receptor” which increased both the benefit and toxicity of chemo A+B. McNemar’s test was used to examine changes in preferences. Results: 439 patients completed both scenarios on 18-month survey. The Table shows the treatment preferences in each scenario. The positive biomarker information in scenario 2 led 60/439 (14%) participants to switch their preference. The main reason for treatment preference in scenario 2 was greater benefits of chemo A+B (64%), the lower risks with chemo A (20%) and positive biomarker (10%). Among participants who changed preference, those randomized to receive bevacizumab were more likely to switch to chemo A in scenario 2. Conclusions: Information about a positive biomarker, indicating increased benefit and increased risk from additional chemo, did not significantly change participants’ preferred treatment. All participants were involved in a large placebo controlled double blind trial and the majority (70%) preferred the most aggressive course of treatment in both scenarios. Whether patients not enrolled in the trial would be more sensitive to the increased risk information is unclear. [Table: see text]

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