scholarly journals Identifying a set that contains the best dynamic treatment regimes

Biostatistics ◽  
2015 ◽  
Vol 17 (1) ◽  
pp. 135-148 ◽  
Author(s):  
Ashkan Ertefaie ◽  
Tianshuang Wu ◽  
Kevin G. Lynch ◽  
Inbal Nahum-Shani
Keyword(s):  
Treatment Effectiveness ◽  
Treatment Options ◽  
Decision Rules ◽  
Study Data ◽  
Response To Treatment ◽  
Treatment Regimes ◽  
Decision Points ◽  
Smart Study ◽  
Dynamic Treatment ◽  
The Cost

Abstract A dynamic treatment regime (DTR) is a treatment design that seeks to accommodate patient heterogeneity in response to treatment. DTRs can be operationalized by a sequence of decision rules that map patient information to treatment options at specific decision points. The sequential, multiple assignment, randomized trial (SMART) is a trial design that was developed specifically for the purpose of obtaining data that informs the construction of good (i.e. efficacious) decision rules. One of the scientific questions motivating a SMART concerns the comparison of multiple DTRs that are embedded in the design. Typical approaches for identifying the best DTRs involve all possible comparisons between DTRs that are embedded in a SMART, at the cost of greatly reduced power to the extent that the number of embedded DTRs (EDTRs) increase. Here, we propose a method that will enable investigators to use SMART study data more efficiently to identify the set that contains the most efficacious EDTRs. Our method ensures that the true best EDTRs are included in this set with at least a given probability. Simulation results are presented to evaluate the proposed method, and the Extending Treatment Effectiveness of Naltrexone SMART study data are analyzed to illustrate its application.

scholarly journals Decision Making from the Experience of Orthognathic Surgery Patients: A Grounded Theory Approach

2021 ◽  
pp. 238008442110144
Author(s):  
N.R. Paul ◽  
S.R. Baker ◽  
B.J. Gibson
Keyword(s):  
Decision Making ◽  
Grounded Theory ◽  
Treatment Options ◽  
Study Data ◽  
Primary Objective ◽  
Major Surgery ◽  
Future Research ◽  
Decision Making Process ◽  
Theory Approach

Introduction: Patients’ decisions to undergo major surgery such as orthognathic treatment are not just about how the decision is made but what influences the decision. Objectives: The primary objective of the study was to identify the key processes involved in patients’ experience of decision making for orthognathic treatment. Methods: This study reports some of the findings of a larger grounded theory study. Data were collected through face-to-face interviews of patients who were seen for orthognathic treatment at a teaching hospital in the United Kingdom. Twenty-two participants were recruited (age range 18–66 y), of whom 12 (male = 2, female = 10) were 6 to 8 wk postsurgery, 6 (male = 2, female = 4) were in the decision-making stage, and 4 (male = 0, female = 4) were 1 to 2 y postsurgery. Additional data were also collected from online blogs and forums on jaw surgery. The data analysis stages of grounded theory methodology were undertaken, including open and selective coding. Results: The study identified the central role of dental care professionals (DCPs) in several underlying processes associated with decision making, including legitimating, mediating, scheduling, projecting, and supporting patients’ decisions. Six categories were related to key aspects of decision making. These were awareness about their underlying dentofacial problems and treatment options available, the information available about the treatment, the temporality of when surgery would be undertaken, the motivations and expectation of patients, social support, and fear of the surgery, hospitalization, and potentially disliking their new face. Conclusion: The decision-making process for orthognathic treatment is complex, multifactorial, and heavily influenced by the role of DCPs in patient care. Understanding the magnitude of this role will enable DCPs to more clearly participate in improving patients’ decision-making process. The findings of this study can inform future quantitative studies. Knowledge Transfer Statement: The results of this study can be used both for informing clinical practice around enabling decision making for orthognathic treatment and also for designing future research. The findings can better inform clinicians about the importance of their role in the patients’ decision-making process for orthognathic treatment and the means to improve the patient experience. It is suggested that further research could be conducted to measure some of the key constructs identified within our grounded theory and assess how these change during the treatment process.

scholarly journals Evaluation of whole-body modalities for diagnosis of multifocal osteonecrosis—a pilot study

2021 ◽  
Vol 23 (1) ◽  
Author(s):  
Shunichi Yokota ◽  
Keita Sakamoto ◽  
Yukie Shimizu ◽  
Tsuyoshi Asano ◽  
Daisuke Takahashi ◽  
...  
Keyword(s):  
Study Data ◽  
Whole Body ◽  
Hematologic Disease ◽  
Hip Joints ◽  
Patient Demographics ◽  
Examination Time ◽  
History Of ◽  
Body Magnetic Resonance Imaging ◽  
The Cost ◽  
Body Bone

Abstract Background This study aimed to investigate the ability of whole-body bone scintigraphy (WB-BS) in the detection of multifocal osteonecrosis (ON) compared to whole-body magnetic resonance imaging (WB-MRI) and to clarify the characteristics of patients with multifocal ON among those with ON of the femoral head (ONFH) using WB-MRI. Methods Forty-six patients who had symptomatic ONFH and underwent surgery in our hospital from April 2019 to October 2020 were included in the study. Data on patient demographics, including age, sex, body mass index (BMI), history of corticosteroid intake, alcohol abuse, smoking, and symptomatic joints, were collected from their medical records. All patients underwent WB-MRI and WB-BS before surgery. Results The agreement in the detection of ON by WB-MRI vs the uptake lesions by WB-BS in the hip joints was moderate (κ = 0.584), while that in other joints was low (κ < 0.40). Among the 152 joints with ON detected by WB-MRI, 92 joints (60.5%) were symptomatic, and 60 joints (39.5%) were asymptomatic. Twelve out of the 46 (26.0%) patients had multifocal (three or more distinct anatomical sites) ON. Nonetheless, while WB-BS detected symptomatic ON detected by WB-MRI as uptake lesions in 82.6% (76/92) of the joints, asymptomatic ON detected by WB-MRI was detected as uptake lesions in 21.7% (13/60) of the joints. All patients with multifocal ON had a history of steroid therapy, which was significantly higher than that in patients with oligofocal ON (P = 0.035). The patients with a hematologic disease had multifocal ON at a higher rate (P = 0.015). Conclusions It might be difficult for WB-BS to detect the asymptomatic ON detected by WB-MRI compared to symptomatic ON. Considering the cost, examination time, and radiation exposure, WB-MRI might be useful for evaluating multifocal ON. Larger longitudinal studies evaluating the benefits of WB-MRI for detecting the risk factors for multifocal ON are required.

Pharmacoeconomic Considerations in Treatment Options for Acute Seizures

1998 ◽  
Vol 13 (1_suppl) ◽  
pp. S27-S29 ◽  
Author(s):  
Nina Graves
Keyword(s):  
Treatment Options ◽  
Emergency Department Visits ◽  
Event Management ◽  
Pharmacoeconomic Data ◽  
Acute Seizures ◽  
Rectal Diazepam ◽  
Adverse Event Management ◽  
History Of ◽  
Cluster Seizures ◽  
The Cost

Two pharmacoeconomic studies on the treatment of acute seizures have been conducted. In 1991, Kriel and colleagues surveyed parents of children with a history of cluster seizures, prolonged seizures, or status epilepticus who had been instructed in the use of rectal diazepam. A comparison of data before instruction with data after instruction showed a reduced need for emergency department visits with rectal diazepam. Instruction thus provided a pharmacoeconomic benefit, despite the cost of the product. In 1996, Marchetti and coworkers found that intravenous fosphenytoin was associated with fewer adverse events than intravenous phenytoin. Fosphenytoin thus reduced the need for adverse event management and provided a substantial pharmacoeconomic benefit, despite its higher cost, compared with phenytoin. This study had a number of limitations, however, and hospital pharmacists remain resistant to the use of fosphenytoin. Additional studies may provide more pharmacoeconomic data to support the greater use of fosphenytoin in the treatment of acute pediatric seizures. (J Child Neurol 1998;13(Suppl 1):S27-S29).

scholarly journals Treatment options in moderate and severe depression: decision analysis supporting a clinical guideline

2006 ◽  
Vol 189 (6) ◽  
pp. 494-501 ◽  
Author(s):  
Judit Simon ◽  
Stephen Pilling ◽  
Rachel Burbeck ◽  
David Goldberg
Keyword(s):  
Combination Therapy ◽  
Decision Analysis ◽  
Secondary Care ◽  
Treatment Options ◽  
Severe Depression ◽  
Cost Effective ◽  
Current Evidence ◽  
Resource Utilisation ◽  
Moderate Depression ◽  
The Cost

BackgroundTreatment options for depression include antidepressants, psychological therapy and a combination of the two.AimsTo develop cost-effective clinical guidelines.MethodSystematic literature reviews were used to identify clinical, utility and cost data. A decision analysis was then conducted to compare the benefits and costs of antidepressants with combination therapy for moderate and severe depression in secondary care in the UK.ResultsOver the 15-month analysis period, combination therapy resulted in higher costs and an expected 0.16 increase per person in the probability of remission and no relapse compared with antidepressants. The cost per additional successfully treated patient was £4056 (95% CI 1400–18 300); the cost per quality-adjusted life year gained was £5777 (95% CI 1900–33 800) for severe depression and £14 540 (95% CI 4800–79 400) for moderate depression.ConclusionsCombination therapy is likely to be a cost-effective first-line secondary care treatment for severe depression. Its cost-effectiveness for moderate depression is more uncertain from current evidence. Targeted combination therapy could improve resource utilisation.

scholarly journals Establishment of a Pretreatment Nomogram to Predict the 6-Month Mortality Rate of Patients with Advanced Biliary Tract Cancers Undergoing Gemcitabine-Based Chemotherapy

Cancers ◽  
2021 ◽  
Vol 13 (13) ◽  
pp. 3139
Author(s):  
Chiao-En Wu ◽  
Wen-Kuan Huang ◽  
Wen-Chi Chou ◽  
Chia-Hsun Hsieh ◽  
John Wen-Cheng Chang ◽  
...  
Keyword(s):  
Liver Metastasis ◽  
Mortality Rate ◽  
Biliary Tract ◽  
Mortality Risk ◽  
Treatment Options ◽  
Univariate Analysis ◽  
Cytotoxic Agents ◽  
Response To Treatment ◽  
Tract Cancer ◽  
Additional Information

Background: The estimation of mortality risk among patients diagnosed with advanced cancer provides important information for clinicians and patients in clinical practice. Currently, gemcitabine-based chemotherapy regimens are the standard treatment for patients with advanced biliary tract cancer (BTC). We aimed to develop a nomogram to predict the 6-month mortality rate among patients with advanced BTC to help physicians evaluate treatment options and outcomes. Patients: We conducted a retrospective analysis to evaluate the 6-month mortality rate among patients with advanced BTC who underwent gemcitabine-based chemotherapy from 2012 to 2018. Data regarding pretreatment factors and the clinical response to treatment were collected. Univariate and multivariate analyses were performed to identify independent factors for nomogram creation. Results: A total of 202 advanced BTC patients who were treated with gemcitabine-based chemotherapy were included in this analysis. No difference in survival was identified between patients undergoing gemcitabine monotherapy and those treated with gemcitabine combined with other cytotoxic agents. The univariate analysis revealed 10 significant factors, while the multivariate analysis identified four independent factors, including gender, monocyte to lymphocyte ratio (MLR), alkaline phosphatase (ALP), and liver metastasis, which were used to establish the nomogram. The performance of this nomogram for the prediction of 6-month mortality risk was found to be promising and feasible based on logistic regression. Conclusion: A nomogram based on four independent pretreatment factors, including gender, MLR, ALP, and liver metastasis, was established to predict the 6-month mortality risk in patients with advanced BTC; it can provide clinicians and patients with additional information when evaluating treatment outcomes.

scholarly journals Reoperation costs in attempted breast-conserving surgery: a decision analysis

2016 ◽  
Vol 23 (5) ◽  
pp. 314 ◽  
Author(s):  
R. Pataky ◽  
C.R. Baliski
Keyword(s):  
Health Care ◽  
Breast Reconstruction ◽  
Patient Care ◽  
Health Care System ◽  
Early Stage ◽  
Treatment Options ◽  
Breast Conserving Surgery ◽  
Reoperation Rate ◽  
The Cost ◽  
Care System

Background Breast-conserving surgery (bcs) is the preferred surgical approach for most patients with early-stage breast cancer. Frequently, concerns arise about the pathologic margin status, resulting in an average reoperation rate of 23% in Canada. No consensus has been reached about the ideal reoperation rate, although 10% has been suggested as a target. Upon undergoing reoperation, many patients choose mastectomy and breast reconstruction, which add to the morbidity and cost of patient care. We attempted to identify the cost of reoperation after bcs, and the effect that a reduction in the reoperation rate could have on the B.C. health care system.Methods A decision tree was constructed to estimate the average cost per patient undergoing initial bcs with two reoperation frequency scenarios: 23% and 10%. The model included the direct medical costs from the perspective of the B.C. health care system for the most common surgical treatment options, including breast reconstruction and postoperative radiation therapy.Results Costs ranged from a low of $8,225 per patient with definitive bcs [95% confidence interval (ci): $8,061 to $8,383] to a high of $26,026 for reoperation with mastectomy and delayed reconstruction (95% ci: $23,991 to $28,122). If the reoperation rate could be reduced to 10%, the average saving would be $1,055 per patient undergoing attempted bcs (95% ci: $959 to $1,156). If the lower rate were to be achieved in British Columbia, it would translate into a savings of $1.9 million annually.Summary The implementation of initiatives to reduce reoperation after bcs could result in significant savings to the health care system, while potentially improving the quality of patient care.

scholarly journals Superficial venous insufficiency from the infernal to the endothermal

2014 ◽  
Vol 96 (1) ◽  
pp. 5-10 ◽  
Author(s):  
D Carradice
Keyword(s):  
Gold Standard ◽  
Venous Insufficiency ◽  
Varicose Veins ◽  
Treatment Options ◽  
Resource Limitation ◽  
Skin Damage ◽  
The Common ◽  
Modern Treatment ◽  
The Cost ◽  
Superficial Venous Insufficiency

This review presents the common diseases associated with superficial venous insufficiency of the leg. These include varicose veins, swelling, skin damage and ulceration. The benefits and rationale behind treatment are discussed, followed by the historical advances from ancient mortality and prayer to the modern endovenous revolution. Finally, an overview of modern treatment options will discuss the evidence supporting the gold standard of endothermal ablation and the cost effectiveness of treatment at this time of challenging resource limitation.

scholarly journals Power analysis in a SMART design: sample size estimation for determining the best embedded dynamic treatment regime

Biostatistics ◽  
2018 ◽  
Vol 21 (3) ◽  
pp. 432-448 ◽  
Author(s):  
William J Artman ◽  
Inbal Nahum-Shani ◽  
Tianshuang Wu ◽  
James R Mckay ◽  
Ashkan Ertefaie
Keyword(s):  
Power Analysis ◽  
Ad Hoc ◽  
Treatment Effectiveness ◽  
R Package ◽  
Treatment Regime ◽  
Size Estimation ◽  
Dynamic Treatment Regime ◽  
Dynamic Treatment ◽  
The Individual ◽  
Number Of Individuals

Summary Sequential, multiple assignment, randomized trial (SMART) designs have become increasingly popular in the field of precision medicine by providing a means for comparing more than two sequences of treatments tailored to the individual patient, i.e., dynamic treatment regime (DTR). The construction of evidence-based DTRs promises a replacement to ad hoc one-size-fits-all decisions pervasive in patient care. However, there are substantial statistical challenges in sizing SMART designs due to the correlation structure between the DTRs embedded in the design (EDTR). Since a primary goal of SMARTs is the construction of an optimal EDTR, investigators are interested in sizing SMARTs based on the ability to screen out EDTRs inferior to the optimal EDTR by a given amount which cannot be done using existing methods. In this article, we fill this gap by developing a rigorous power analysis framework that leverages the multiple comparisons with the best methodology. Our method employs Monte Carlo simulation to compute the number of individuals to enroll in an arbitrary SMART. We evaluate our method through extensive simulation studies. We illustrate our method by retrospectively computing the power in the Extending Treatment Effectiveness of Naltrexone (EXTEND) trial. An R package implementing our methodology is available to download from the Comprehensive R Archive Network.

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