Relationship between treatment selection variables and treatment outcome in a child guidance clinic. An application of data-processing methods.
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Twenty dentists from selected dental school departments were asked to examine thirteen areas of fissured grooves or pits in extracted molars, and select a treatment from a list of options for each designated groove area, as if each was on a separate tooth. There was a wide variation in initial treatment selections with (29 percent) amalgam and (25 percent) resin sealants being the most common. Pediatric dentists were significantly more conservative in both their original and later diagnoses than were the other dentists in this study.
Optimal treatment selection for patients with chronic disease, especially advanced cancer, requires careful consideration in weighing risks and benefits of each therapy. The application of statistical decision theory to such problems provides an explicit and systematic means of combining information on risks and benefits with individual patient preferences on quality-of-life issues. This paper evaluates the strengths and weaknesses of this methodology by using, as an example, treatment selection in advanced ovarian cancer. Possible treatment options and the major consequences of each are first outlined on a decision tree. The probability of various outcomes is estimated from the literature and methods for assessing the relative value or utility of each outcome are illustrated by interviews with 9 volunteers. Based on decision analysis, the recommended treatment for advanced ovarian cancer is found to be highly dependent on survival estimates but far less dependent on other probability estimates or the method of obtaining utilities. Individual preferences are also found to influence the treatment choice. The analysis illustrates that an important strength in using decision theory is its ability to identify key factors in the decision through sensitivity analysis. This may help both the physician selecting treatment and the investigator planning clinical trials which compare these therapies. In addition, this method can help in planning a trial's sample size by determining what survival difference between therapeutic strategies is worth detecting. Some problems identified with this methodology include the need for several simplifying assumptions and the difficulties in assessing individual preferences. On balance, we believe decision theory in this setting can play a useful role in complementing the physician's clinical judgement.
It has been believed that the 'selective binding profile' may result in the 'selective treatment' for a disease. However, typical antipsychotics with the selective affinity for dopamine D2 receptor produce antipsychotic efficacy against positive symptoms accompanied with extrapyramidal side effects. Serotonin-dopamine-antagonists produce fewer EPS, since they have a potent antagonistic activity for 5-HT2A receptor besides the dopamine D2 antagonism. The disinhibition of diminished glutamatergic transmission and alpha 1 antagonism by MARTAs (Multi-Acting-Acting-Receptor-Targeted-Antipsychotics) can reduce positive symptoms of schizophrenic patients, and the 5-HT2A antagonitic and anticholinergic activities of the drugs may reduce the EPS. Moreover, MARTAs increase intracellular concentrations of dopamine and norepinephrine in the prefrontal cortex and disinhibit the diminished glutamatergic transmission, which are expected to improve the negative symptoms, anxiety and depressive symptoms, and cognitive impairment that are difficult to be treated by other existing antipsychotics. These important biological changes are induced by the antagonism of 5-HT2A, 5-HT2C, alpha 1-adrenergic and muscarinic receptors by MARTAs. Thus a multireceptorial profile of MARTA is considered to result in the improved treatment. Therefore 'selective binding profile' does not always mean 'selective treatment.' Instead, if a breakthrough drug is to be developed for the treatment of refractory diseases, it may be necessary to rethink the ordinary strategy targeting for a single receptor.
Practical and scientific developments have recently focused attention on treatment selection in psychiatry. Based on the research literature, the opinions of leading practitioners, and the authors' clinical experience, tentative guidelines for treatment selection are offered. The indications, patient enabling factors, and relative contraindications that influence the choice of treatment format, orientation, and duration are outlined, as are indications for combining psychotherapy with psychotropic medication and for a prescription of no treatment. Critics of differential therapeutics might argue that the outcome of psychotherapy is influenced primarily by its nonspecific aspects and by patient characteristics, and that it may be premature to form guidelines given the incompleteness of the research. However, as outcome studies are more carefully designed, specific effects of different treatments may well emerge. Furthermore, treatment selection is a practical necessity, and a careful analysis of the available data and opinion helps to inform these choices and to identify areas for continued research.
Unlike patients in a randomized, clinical trial, patients in an observational study choose if and when to begin treatment. Patients who live longer have more opportunities to select treatment; those who die earlier may be untreated by default. These facts are the essence of an often overlooked bias, termed "survivor treatment selection bias," which can erroneously lead to the conclusion that an ineffective treatment prolongs survival. Unfortunately, misanalysis of survivor treatment selection bias has been prevalent in the recent literature on the acquired immunodeficiency syndrome. Approaches to mitigating this bias involve complex statistical models. At a minimum, initiation of therapy should be treated as a time-dependent covariate in a proportional hazards model. Investigators and readers should be on the alert for survivor treatment selection bias and should be cautious when interpreting the results of observational treatment studies.
BACKGROUND: Treatment guidelines support oncologists in treatment decision-making for patients with metastatic breast cancer (MBC). However, treatment decision-making is complicated by the rapid pace of therapeutic advances, the complexity of incorporating patient preferences, and the underrepresentation of diverse populations in clinical trials. This study explored oncologists' experiences of treatment selection in late-line MBC when evidence and guidelines provide limited guidance. MATERIALS AND METHODS: This qualitative study was conducted using a constructivist approach and involved individual interviews with twelve oncologists in Sweden. Participants had between five and forty years of experience in breast cancer care and worked within publicly funded healthcare across academic and regional hospitals. An inductive reflexive thematic analysis was used to identify themes, with attention to both manifest and latent meanings in the data. RESULTS: The overarching theme interpreted was: Making patient-oriented treatment decisions with collegial support as an anchor, reflecting how oncologists adapt their treatment decision-making to patient needs while relying on colleagues for professional stability. Four themes were elucidated: Offering to use professional knowledge and experience to decide; Inviting the patient to a dialogue to decide; Supporting the patient in making the final decision; and Turning to colleagues for advice and support. CONCLUSION: This study challenges the positivist evidence-based assumption that oncologists act as neutral facilitators who simply present treatment options for patients to choose. Instead, it positions oncologists as active decision-makers who clearly state their professional stance, retain responsibility for treatment decisions, and respect patient autonomy, moving beyond the traditional, dichotomized shared decision-making model. IMPLICATIONS TO PRACTICE: We argue that oncologists are active stakeholders in a value-led decision-making process and encourage them to clearly articulate the values underlying their recommendations. This approach enables patients to be addressed with full autonomy, while the ultimate responsibility for treatment decisions remains with the oncologist.
Selective treatment of infants with myelomeningocele is said to be beneficial to the infants, their parents, and to society. One group of arguments intended to support this point of view is analyzed. Recent data do not support the factual basis on which these arguments rest; moreover, the moral assumptions that lie behind them should be emphasized. The values held by participants in a discussion of allowing infants to die are as important as facts about treatment options.
In recent years, patients have become more involved in the clinical decision-making process, yet the nature of this process, including the role of information, is poorly understood. The purpose of this exploratory study was to examine the relationship between information about breast cancer treatment alternatives and patients' choices of treatments. The target population was all patients with breast cancer in the process of deciding between breast conservation (lumpectomy plus irradiation) and more traditional management (modified radical mastectomy, with or without reconstruction). A convenience sample of 71 female patients with stage I or II breast cancer was drawn from a breast clinic affiliated with a 1,000-bed tertiary medical center. The amount of information provided to each subject and the nature of its presentation were recorded using an observer checklist. Recall of information and final treatment selection were ascertained during telephone interviews conducted six to eight weeks after surgery. The results indicate that subjects' choice of treatment was unrelated to the amount of information they received during the clinic visit. Manner of presentation also did not influence treatment selection. However, treatment selection was related to the amount of information subjects received prior to their clinic visit (p < 0.01). The results also indicate that patients' recall of information about treatments and associated risks is exceedingly poor. Clinical and legal implications are discussed and recommendations for further research are offered in this article.
OBJECTIVE: This paper reviews controlled studies of bipolar depression, outlines criteria for choosing treatment, defines refractoriness in bipolar depression, and provides options for treatment of refractory bipolar depression. METHODS: Controlled studies that examined the efficacy of treatments for acute and long-term treatment of bipolar depression were located through electronic searches of several databases and by manual crosssearch of references and proceedings of international meetings. RESULTS: Lithium comes close to fulfilling the proposed criteria for first-line treatment for bipolar depression, and those not responding to lithium should be considered to have refractory bipolar depression. Options for such patients include addition of lamotrigine or a second mood stabilizer, or a newer-generation antidepressant such as a serotonin re-uptake inhibitor or bupropion, or the atypical antipsychotic olanzapine. CONCLUSIONS: Although there is a paucity of research in the treatment of refractory bipolar depression, available data could be used for providing rational treatment options for such patients. However, further studies are urgently needed to determine which options are most appropriate for which type of patients.
PURPOSE: To examine the effect of primary treatment selection on outcomes for benign intracranial meningiomas at the University of Florida. METHODS AND MATERIALS: For 262 patients, the impact of age, Karnofsky performance status, pathologic features, tumor size, tumor location, and treatment modality on local control and cause-specific survival was analyzed (minimum potential follow-up, 2 years; median follow-up, 8.2 years). Extent of surgery was classified by Simpson grade. Treatment groups: surgery alone (n = 229), surgery and postoperative radiotherapy (RT) (n = 21), RT alone (n = 7), radiosurgery alone (n = 5). Survival analysis: Kaplan-Meier method with univariate and multivariate analysis. RESULTS: At 15 years, local control was 76% after total excision (TE) and 87% after subtotal excision plus RT (SE+RT), both significantly better (p = 0.0001) than after SE alone (30%). Cause-specific survival at 15 years was reduced after treatment with SE alone (51%), compared with TE (88%) or SE+RT (86%) (p = 0.0003). Recurrence after primary treatment portended decreased survival, independent of initial treatment group or salvage treatment selection (p = 0.001). Atypical pathologic features predicted reduced 15-year local control (54 vs. 71%) and cause-specific survival rates (57 vs. 86%). Multivariate analysis for cause-specific survival revealed treatment group (SE vs. others; p = 0.0001), pathologic features (atypical vs. typical;p = 0.0056), and Karnofsky performance status (> or = 80 vs. < 80; p = 0.0153) as significant variables. CONCLUSION: Benign meningiomas are well managed by TE or SE+RT. SE alone is inadequate therapy and adversely affects cause-specific survival. Atypical pathologic features predict a poorer outcome, suggesting possible benefit from more aggressive treatment. Because local recurrence portends lower survival rates, primary treatment choice is important.
Urinary incontinence is a common adverse effect associated with treatment for early stage prostate cancer. The influence of this factor on treatment selection decisions by patients and their partners has been explored only minimally in the literature. Data regarding the actual incidence of incontinence associated with prostate cancer treatment are confusing because of the lack of standardized definitions of incontinence. Radical prostatectomy is associated with higher rates of urinary adverse effects than is radiation therapy. Brachytherapy appears to be associated with a low risk of incontinence, whereas cryosurgery is associated with significant urinary adverse effects. Including incontinence, urethral sloughing, and bladder neck obstruction. The influence of these adverse effects on decision making regarding prostate cancer treatment selection is difficult to ascertain. Research indicates that both men and their partners appear to have difficulty processing information presented to them regarding the probability of urinary adverse effects and the degree to which these adverse effects may have an impact on their daily lives.
A study to compare effects of mass, targeted and selective chemotherapy with levamisole (Ketrax) as an action for the control of Ascaris lumbricoides was carried out in three communities in rural Oyo State, Nigeria. Selective treatment was applied in one village by treating the most heavily infected 20% of the inhabitants, targeted treatment in the second village involved children aged 2-15 years, while mass treatment was offered to all inhabitants excluding infants under 1 year and pregnant women in the third village. Recommended doses of levamisole were given in the villages, as described above, at 3-monthly intervals during a period of 1 year. Prevalence and intensity (epg) of A. lumbricoides infection were determined immediately before and 3 months after the period of intervention using a modified Kato-Katz technique. In the selective treatment village, no significant differences were found between the pre- and post-treatment egg counts (mean (+/- S.D.) epg 6776 +/- 10,791 versus 4259 +/- 10,909 respectively) of A. lumbricoides in the total population. In the targeted treatment village, significant differences were recorded in pre- and post-treatment egg counts for the total population (9057 +/- 15,797 versus 2579 +/- 6529) among the children alone (10,935 +/- 20,094 versus 992 +/- 3175) and among the untreated adults (7742 +/- 9782 versus 4561 +/- 8798). In the mass treatment village, significant differences in pre- and post-treatment egg count values were also recorded (11,907 +/- 17,220 versus 1489 +/- 5165). The intensity of Trichuris trichiura and hookworm infections among the villagers before and after intervention were not observed to have changed significantly regardless of selective, targeted or mass treatment.
Despite the controversies in management for all stages of prostatic cancer, guidelines are emerging that allow for better selection of treatments for individual patients. For early stage disease, prostate-specific antigen determinations in conjunction with other staging procedures have refined our ability to define truly organ-confined disease. The more widespread use of laparoscopic lymph node dissections has spared many patients needless laparotomies. For patients with metastatic disease, the overall effect of potency-sparing antiandrogens as monotherapy needs to be investigated. Most encouraging is that more groups are using prostate-specific antigen changes to assess disease activity and the rapid translation of recent laboratory investigations into the clinic. As our ability to predict the biologic potential of an individual patient's tumor is improved, more individualized treatment recommendations will be possible.
Selective treatment of duodenal ulcer with perforation has been based on several premises: 1) The natural history of the ulcer following closure of a perforation is generally favorable with an acute and unfavorable with a chronic ulcer. 2) An upper gastrointestinal series with water soluble contrast media can reliably document a spontaneously sealed perforation. 3) With a spontaneous seal, nonsurgical therapy is an acceptable option and is preferable for an acute ulcer or a chronic ulcer with poor surgical risk. 4) The treatment of choice for an unsealed perforation of an acute ulcer is simple surgical closure. 5) The treatment of choice of perforation of a chronic ulcer with acceptable surgical risk is an ulcer definitive operation. Sixty cases of perforation of duodenal ulcer have been treated. Nonsurgical therapy was employed without complication in eight cases with radiologically documented spontaneous seal. Truncal vagotomy and pyloroplasty in 36 cases and truncal vagotomy and antrectomy in two cases were each without mortality. Four fatalities occurred among 13 cases of closure and omental patch, each a case with severe associated disease. The mortality was 6.7% among the 60 cases; 2.4% for chronic ulcer and 16% for acute ulcer.
The aggregated nature of helminth parasite populations has led to the suggestion that selectively treating heavily infected hosts can efficiently reduce parasite abundance and morbidity within a host population. Moreover, it has been postulated that a selective treatment protocol might have long-term effects on parasite transmission by disrupting the stability attributed to aggregated parasite population distributions by theoretical models. Long-term investigation has demonstrated year-to-year consistency in the population dynamics of Halipegus occidualis in green frogs from Charlie's Pond, North Carolina. In 1996, removal of all but 1 worm from each frog with > or =15 worms reduced the estimated component worm population by 45%, thereby decreasing mean intensity and aggregation (variance-to-mean ratio) of H. occidualis in the frogs by 85% and 63%, respectively. The following year, mean intensity, aggregation, and host colonization trends returned to pretreatment levels, indicating no effect of worm removal and demonstrating the stability of this host-parasite system. Although this result might be attributable to inefficient treatment or the presence of infection reservoirs, it is suggested that parasite population stability in this system might be governed by prevalence rather than intensity of adult worms. Therefore, repeated selective treatment might effectively modify intensity-dependent morbidity in similar host-parasite systems but should not affect further parasite transmission.
The study examines the effect of mebendazole distribution (2 x 100 mg x 3 days) either as mass chemotherapy targeted to children 1 to 10 years old, or selective treatment of heavily infected children, over a two year period in sixteen rural villages of Nepal. Eight villages were covered with six monthly mass chemotherapy, provided by Community Health Volunteers, other eight with yearly selective treatment of heavily infected children based on clinical signs. Prevalence and intensity of infection, indirectly measured as the number of eggs per gram of faeces, were measured for A. lumbricoides and hookworms utilising the Kato thick-smear technique. At the end of two year intervention, the effect of both activities was similar on worm prevalence with a slightly significant difference (p < 0.05) on worm load in favour of mass chemotherapy. Furthermore a greater effect of mebendazole was reported, regardless of the type of intervention, on hookworms present at baseline with a lower prevalence (23.4%) than A. lumbricoides (47.6%). The study suggests that clinical diagnosis of heavily infected children may be a feasible way to direct selective treatment. Baseline prevalence seems to influence effectiveness of control programs. Further studies are needed to substantiate these observations.
The results of selective treatment in 120 infants with open spina bifida, admitted between May 1971 and December 1976, were prospectively studied. Seventy-one infants had adverse criteria at birth and were not treated. They all died, more than 90% of them within 6 months of birth. Seven had meningocele. All were treated and survived without handicap. Forty-two infants with myelomeningocele were actively treated. Thirty-six survive at follow-up after 3 to 9 years. The quality of survival is much better than when selection was not used but 8 children have moderate or severe handicaps. The parents were fully informed and consulted at every decision-making step; they fully supported the principle of selection and the action taken on behalf of their own child.