Causal inference for clinicians

2019 ◽  
Vol 24 (3) ◽  
pp. 109-112 ◽  
Author(s):  
Steven D Stovitz ◽  
Ian Shrier

Evidence-based medicine (EBM) calls on clinicians to incorporate the ‘best available evidence’ into clinical decision-making. For decisions regarding treatment, the best evidence is that which determines the causal effect of treatments on the clinical outcomes of interest. Unfortunately, research often provides evidence where associations are not due to cause-and-effect, but rather due to non-causal reasons. These non-causal associations may provide valid evidence for diagnosis or prognosis, but biased evidence for treatment effects. Causal inference aims to determine when we can infer that associations are or are not due to causal effects. Since recommending treatments that do not have beneficial causal effects will not improve health, causal inference can advance the practice of EBM. The purpose of this article is to familiarise clinicians with some of the concepts and terminology that are being used in the field of causal inference, including graphical diagrams known as ‘causal directed acyclic graphs’. In order to demonstrate some of the links between causal inference methods and clinical treatment decision-making, we use a clinical vignette of assessing treatments to lower cardiovascular risk. As the field of causal inference advances, clinicians familiar with the methods and terminology will be able to improve their adherence to the principles of EBM by distinguishing causal effects of treatment from results due to non-causal associations that may be a source of bias.

2019 ◽  
Vol 37 (4) ◽  
pp. 503-509
Author(s):  
Marlene Pereira Garanito ◽  
Vera Lucia Zaher-Rutherford

ABSTRACT Objective: To carry out a review of the literature on adolescents’ participation in decision making for their own health. Data sources: Review in the Scientific Electronic Library Online (SciELO), Latin American and Caribbean Health Sciences Literature (LILACS) and PubMed databases. We consider scientific articles and books between 1966 and 2017. Keywords: adolescence, autonomy, bioethics and adolescence, autonomy, ethics, in variants in the English, Portuguese and Spanish languages. Inclusion criteria: scientific articles, books and theses on clinical decision making by the adolescent patient. Exclusion criteria: case reports and articles that did not address the issue. Among 1,590 abstracts, 78 were read in full and 32 were used in this manuscript. Data synthesis: The age at which the individual is able to make decisions is a matter of debate in the literature. The development of a cognitive and psychosocial system is a time-consuming process and the integration of psychological, neuropsychological and neurobiological research in adolescence is fundamental. The ability to mature reflection is not determined by chronological age; in theory, a mature child is able to consent or refuse treatment. Decision-making requires careful and reflective analysis of the main associated factors, and the approach of this problem must occur through the recognition of the maturity and autonomy that exists in the adolescents. To do so, it is necessary to “deliberate” with them. Conclusions: International guidelines recommend that adolescents participate in discussions about their illness, treatment and decision-making. However, there is no universally accepted consensus on how to assess the decision-making ability of these patients. Despite this, when possible, the adolescent should be included in a serious, honest, respectful and sincere process of deliberation.


2004 ◽  
Vol 12 (2) ◽  
pp. 127-132 ◽  
Author(s):  
Cláudio Rodrigues Leles ◽  
Maria do Carmo Matias Freire

A critical problem in the decision making process for dental prosthodontic treatment is the lack of reliable clinical parameters. This review discusses the limits of traditional normative treatment and presents guidelines for clinical decision making. There is a need to incorporate a sociodental approach to help determine patient's needs. Adoption of the evidence-based clinical practice model is also needed to assure safe and effective clinical practice in prosthetic dentistry.


1998 ◽  
Vol 37 (02) ◽  
pp. 201-205 ◽  
Author(s):  
B. E. Waitzfelder ◽  
E. P. Gramlich

AbstractThe Hawaii Quality and Cost Consortium began a project in 1993 to implement and evaluate interactive videodisk programs to assist in clinical decision-making for breast cancer. Communication problems between physicians and patients, limitations of available outcomes data and varying preferences of individual patients can result in treatment outcomes that are less than satisfactory. Shared Decision-making Programs (SDPs) were developed by the Foundation for Informed Medical Decision Making (FIMDM) in Hanover, New Hampshire, to assist in the treatment decision-making process. Utilizing interactive videodisks, the programs provide patients with clear, unbiased information about available treatment options. With this information, patients can become more active participants in making treatment decisions. The SDPs for breast cancer were implemented at two sites in Hawaii. Evaluation data from 103 patients overwhelmingly indicate that patients find the programs clear, balanced and very good or excellent in terms of the amount of information presented and overall rating.


2021 ◽  
Vol 28 (3) ◽  
pp. 2123-2133
Author(s):  
Philipp Mandel ◽  
Mike Wenzel ◽  
Benedikt Hoeh ◽  
Maria N. Welte ◽  
Felix Preisser ◽  
...  

Background: To test the value of immunohistochemistry (IHC) staining in prostate biopsies for changes in biopsy results and its impact on treatment decision-making. Methods: Between January 2017–June 2020, all patients undergoing prostate biopsies were identified and evaluated regarding additional IHC staining for diagnostic purpose. Final pathologic results after radical prostatectomy (RP) were analyzed regarding the effect of IHC at biopsy. Results: Of 606 biopsies, 350 (58.7%) received additional IHC staining. Of those, prostate cancer (PCa) was found in 208 patients (59.4%); while in 142 patients (40.6%), PCa could be ruled out through IHC. IHC patients harbored significantly more often Gleason 6 in biopsy (p < 0.01) and less suspicious baseline characteristics than patients without IHC. Of 185 patients with positive IHC and PCa detection, IHC led to a change in biopsy results in 81 (43.8%) patients. Of these patients with changes in biopsy results due to IHC, 42 (51.9%) underwent RP with 59.5% harboring ≥pT3 and/or Gleason 7–10. Conclusions: Patients with IHC stains had less suspicious characteristics than patients without IHC. Moreover, in patients with positive IHC and PCa detection, a change in biopsy results was observed in >40%. Patients with changes in biopsy results partly underwent RP, in which 60% harbored significant PCa.


Sarcoma ◽  
2021 ◽  
Vol 2021 ◽  
pp. 1-6
Author(s):  
H. S. Femke Hagenmaier ◽  
Annelies G. K. van Beeck ◽  
Rick L. Haas ◽  
Veroniek M. van Praag ◽  
Leti van Bodegom-Vos ◽  
...  

Background. With soft-tissue sarcoma of the extremity (ESTS) representing a heterogenous group of tumors, management decisions are often made in multidisciplinary team (MDT) meetings. To optimize outcome, nomograms are more commonly used to guide individualized treatment decision making. Purpose. To evaluate the influence of Personalised Sarcoma Care (PERSARC) on treatment decisions for patients with high-grade ESTS and the ability of the MDT to accurately predict overall survival (OS) and local recurrence (LR) rates. Methods. Two consecutive meetings were organised. During the first meeting, 36 cases were presented to the MDT. OS and LR rates without the use of PERSARC were estimated by consensus and preferred treatment was recorded for each case. During the second meeting, OS/LR rates calculated with PERSARC were presented to the MDT. Differences between estimated OS/LR rates and PERSARC OS/LR rates were calculated. Variations in preferred treatment protocols were noted. Results. The MDT underestimated OS when compared to PERSARC in 48.4% of cases. LR rates were overestimated in 41.9% of cases. With the use of PERSARC, the proposed treatment changed for 24 cases. Conclusion. PERSARC aids the MDT to optimize individualized predicted OS and LR rates, hereby guiding patient-centered care and shared decision making.


Circulation ◽  
2015 ◽  
Vol 131 (suppl_1) ◽  
Author(s):  
Michel Krempf ◽  
Ross J Simpson ◽  
Dena R Ramey ◽  
Philippe Brudi ◽  
Hilde Giezek ◽  
...  

Objectives: Little is known about how patient factors influence physicians’ treatment decision-making in hypercholesterolemia. We surveyed physicians’ treatment recommendations in high-risk patients with LDL-C not controlled on statin monotherapy. Methods: Physicians completed a questionnaire pre-randomization for each patient in a double-blind trial (NCT01154036) assessing LDL-C goal attainment rates with different treatment strategies. Patients had LDL-C ≥100 mg/dL after 5 weeks’ atorvastatin 10 mg/day and before randomization. Physicians were asked about treatment recommendations for three scenarios: (1) LDL-C near goal (100-105 mg/dL), (2) LDL-C far from goal (120 mg/dL), then (3) known baseline LDL-C of enrolled patients on atorvastatin 10 mg/day. Factors considered in their choice were specified. Physicians had been informed of projected LDL-C reductions for each treatment strategy in the trial. Regression analysis identified prognostic factors associated with each scenario, and projected LDL-C values for physicians’ treatment choices were compared to actual LDL-C values achieved in the trial. Results: Physicians at 296 sites completed questionnaires for 1535 patients. The most common treatment strategies for all three scenarios were: 1) not to change therapy, 2) double atorvastatin dose, 3) add ezetimibe, 4) double atorvastatin dose and add ezetimibe. Doubling atorvastatin dose was the most common treatment recommendation in all scenarios (43-52% of patients). ‘No change in therapy’ was recommended in 6.5% of patients when LDL-C was assumed far from goal. Treatment recommendations were more aggressive if actual LDL-C was known or considered far from goal. When compared with the ‘no change in therapy’ recommendation, CV risk factors and desire to achieve a more aggressive LDL-C goal were generally considered in decision-making for each treatment choice, regardless of LDL-C scenario. Patients randomized to a more aggressive regimen than recommended by physicians had larger reductions in LDL-C: the actual reduction in LDL-C in patients randomized to ‘add ezetimibe’ was -20.8% versus a projected reduction of -10.0% when physicians recommended ‘doubling atorvastatin dose’. Conclusions: This study provides insight into physicians’ perspectives on clinical management of hypercholesterolemia and highlights a gap in knowledge translation from guidelines to clinical practice. Targeting lower LDL-C and CV risk were key drivers in clinical decision-making but, generally, physicians were more conservative in their treatment choice than guidelines recommend, which may result in poorer LDL-C reduction. When compared with actual outcomes, projected LDL-C control was better if physicians used more comprehensive strategies rather than simply doubling the statin dose.


Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2246-2246 ◽  
Author(s):  
Kah Poh Loh ◽  
Sindhuja Kadambi ◽  
Supriya G. Mohile ◽  
Jason H. Mendler ◽  
Jane L. Liesveld ◽  
...  

Abstract Introduction: Despite data supporting the safety and efficacy of treatment for many older adults with AML, <40% of adults aged ≥65 receive any leukemia-directed therapy. The reasons for why the majority of older patients with AML do not receive therapy are unclear. The use of objective fitness measures (e.g. physical function and cognition) has been shown to predict outcomes and may assist with treatment decision-making, but is underutilized. As most patients are initially evaluated in community practices, exploring clinical decision-making and the barriers to performing objective fitness assessments in the community oncology setting is critical to understanding current patterns of care. We conducted a qualitative study: 1) to identify factors that influence treatment decision making from the perspectives of the community oncologists and older patients with AML, and 2) to understand the barriers to performing objective fitness assessments among oncologists. The findings will help to inform the design of a larger study to assess real-life treatment decision-making among community oncologists and patients. Methods: We conducted semi-structured interviews with 13 community oncologists (9 states) and 9 patients aged ≥60 with AML at any stage of treatment to elicit potential factors that influence treatment decisions. Patients were recruited from the outpatient clinics in a single institution and oncologists were recruited via email using purposive samples (patients: based on treatment received and stage of treatment; oncologists: based on practice location). Interviews were audio-recorded and transcribed. We utilized directed content analysis and adapted the decision-making model introduced by Zafar et al. to serve as a framework for categorizing the factors at various levels. A codebook was provisionally developed. Using Atlas.ti, two investigators independently coded the initial transcripts and resolved any discrepancies through an iterative process. The coding scheme was subsequently applied to the rest of the transcripts by one coder. Results: Median age of the oncologists was 37 years (range 34-64); 62% were females, 92% were white, 38% had practiced more than 15 years, and 92% reported seeing <10 older patients with AML annually. Median age of the patients was 70 years (64-80), 33% were females and all were Caucasian. In terms of treatment, 66% received intensive induction therapy, 22% received low-intensity treatment, and 11% received both. Three patients also received allogeneic hematopoietic stem cell transplant. Eighty-nine percent were initially evaluated and 56% were initially treated by a community oncologist. Factors that influenced treatment decision-making are shown in Figure 1. When making treatment decisions, both patients and oncologists considered factors such as patient's overall health, chronological age, comorbidities, insurance coverage, treatment efficacy and tolerability, and distance to treatment center. Nonetheless, there were distinct factors considered by patients (e.g. quality of care and facility, trust in their oncologist/team) and by oncologists (e.g. local practice patterns, availability of transplant/clinical trials, their own clinical expertise and beliefs) when making treatment decisions. The majority of oncologists do not perform an objective assessment of fitness. Most common reasons provided included: 1) Do not add much to routine assessments (N=8), 2) Lack of time, resources, and expertise (N=7), 3) Lack of awareness of the tools or the evidence to support its use (N=4), 4) Specifics are not important (e.g. impairments are clinically apparent and further nuance is not necessarily helpful; N=5), 5) Impairments are usually performed by other team members (N=2), and 6) Do not want to rely on scores (N=2). Conclusions: Treatment decision-making for older patients with AML is complex and influenced by many factors at the patient, disease/treatment, physician, and organizational levels. Despite studies supporting the utility of objective fitness assessments, these were not commonly performed in the community due to several barriers. Our framework will be useful to guide a larger study to assess real-life treatment decision-making in the community settings. We also identified several barriers raised by community oncologists that could be targeted to allow incorporation of objective fitness assessments. Figure 1. Figure 1. Disclosures Liesveld: Onconova: Other: DSMB; Abbvie: Honoraria. Stock:Jazz Pharmaceuticals: Consultancy. Majhail:Anthem, Inc.: Consultancy; Atara: Honoraria; Incyte: Honoraria. Wildes:Janssen: Research Funding. Klepin:Genentech Inc: Consultancy.


2008 ◽  
Vol 23 (3) ◽  
pp. 99-106
Author(s):  
Maurizio Ferrarin ◽  
Marco Rabuffetti ◽  
Marina Ramella ◽  
Maurizio Osio ◽  
Enrico Mailland ◽  
...  

Focal dystonia (FD) is a movement disorder that frequently affects instrumental musicians. Distinguishing between primary dystonic movement and secondary compensatory abnormal movement is crucial for the correct treatment planning in FD. Such distinction is complex in musicians because of the complexity, speed, and smallness of involved movement. The goal of the current study was to assess the influence of instrumented movement analysis (MA) in treatment decision-making in musician's FD. A group of 18 musicians with FD was instrumentally analyzed in an MA laboratory equipped with optoelectronic and electromyographic (EMG) acquisition systems. The muscle(s) primarily responsible for the dystonic movement or posture (trigger muscle) was identified on the basis of clinical assessment alone and, in a second phase, with the additional information provided by instrumented assessment. Comparison between clinical and instrumented assessment outcomes and the subjective rating of found differences were then analyzed. In 67% of patients, instrumental assessment changed the decision made by clinical assessment, indicating identification of a different trigger muscle or allowing for a more specific identification. In 28% of patients, instrumental assessment confirmed the outcome of the clinical assessment, with an increase in the confidence level of the clinical decision. The most frequent change was an improved specification of which finger flexor muscle (superficialis or profundus) was triggering the dystonic movement. Although caution is needed due to the non-blinded design of the present study, our results suggest that instrumented movement analysis is a useful complementary tool to clinical assessment in treatment planning for musician's focal dystonia—its use might change the identification of the muscles primarily responsible for dystonic movements as well as increase the confidence level of the clinician in treatment decision-making.


Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 42-43
Author(s):  
Ajeet Gajra ◽  
Yamini S Rathish ◽  
Yolaine Jeune-Smith ◽  
Andrew J Klink ◽  
Bruce Feinberg

Introduction: In this era of targeted therapy, assessing MRD status is important to guide treatment decision-making in hematologic malignancies. While MRD assessment has long been incorporated the management of patients with acute lymphoblastic leukemia (ALL), there is mounting evidence that MRD-negativity is a critical end point in CLL and MM that correlates well with clinical outcomes. In CLL, undetectable MRD at the end of treatment in peripheral blood or bone marrow is associated with long-term survival (Thompson et al, Leukemia 2018). The international workshop on CLL (iwCLL) guidelines for response assessment in CLL now incorporate MRD assessment (Hallek, et al, Blood 2018). The international myeloma working group (IMWG) and the NCCN now recommend MRD assessment after each phase of MM therapy (induction, stem cell transplant, consolidation and maintenance). Higher rates of durable responses are noted in those with MRD-negativity after induction (Attal et al, NEJM 2017) and associated with favorable survival (Pavia et a, Blood 2008). Based on results from recent studies, MRD negative status can guide clinical decision-making about discontinuation of therapy in CLL (Jain et al, ASH 209 and Tam et al, ASH 2019) and in MM (Costa et al, ASH 2019 and Usmani et al, 2019). A majority of patients with CLL and MM are treated at community practices in the US. but adoption of MRD assessment among cH/O is unclear. We sought to study the self-reported utilization patterns of MRD assessment in CLL and MM, it's use in determining duration of therapy, and the barriers to it's adoption in practice among U.S. cH/O. Methods: U.S.-licensed oncologists and hematologists with broad geographic representation convened at a live meeting in January 2020 to review clinical updates presented at the 2019 ASH Annual Meeting. An electronic pre-meeting survey and live survey were fielded among cH/O meeting attendees. Surveys collected physician perceptions and reported use of MRD assessment for patients with CLL and MM. Responses to questions were summarized using descriptive statistics. Results: A total of 59 cH/O were included who self-identified their specialty as hematology/ oncology (51%) and medical oncology (34%) and reported MM (69%) and CLL (61%) as the two commonest hematologic malignancies treated by them. Excluding those that had not treated MM or CLL in the preceding 3 months, a subset of 46 cH/O were queried on their use and perceptions of MRD assessment in these two diseases. In CLL, 52% of the cH/O do not assess MRD status, and only 17% utilize MRD status in treatment discontinuation decisions. Major reasons for not using MRD status in practice include the perception shared by a majority (52%) of respondents that the evidence does not support its use in CLL at the present time. A minority (9%) utilized MRD assessment when treating younger fit patients with CLL. In MM, 50% do not assess for MRD at any time post-therapy, and 24% utilize MRD status in treatment discontinuation decisions. Major barriers to MRD assessment in MM were the perception that evidence does not support MRD use (59% of the respondents) and lack of payer coverage (11%). Additional details are presented in the Table below. Conclusions: These data from a limited sample of cH/O suggest that adoption of MRD testing among US cH/O is low, despite results from recent trials that highlight the importance of the MRD negativity as an important prognostic factor in both CLL and MM. Half of cH/O do not measure MRD at any point while treating MM and CLL and less than a fifth incorporate MRD data to determine duration of therapy. The greatest barrier to MRD assessment is the impression that there is lack of evidence supporting its utility in practice at the present time. Further education among cH/O is warranted regarding MRD assessment in CLL and MM given that MRD-negative status is associated with favorable prognosis and should be incorporated in treatment decision-making based on updated guidelines in both diseases. Disclosures Gajra: Cardinal Health: Current Employment. Jeune-Smith:Cardinal Health: Current Employment. Klink:Cardinal Health: Current Employment. Feinberg:Cardinal Health: Current Employment.


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