Differences between the real and the desired worlds in the results of clinical trials

OBJECTIVE:We refer to the effectiveness (known as pragmatic or real world) and efficacy (known as explanatory or desired or ideal world) of interventions. However, these terms seem to be randomly chosen by investigators who design clinical trials and do not always reflect the true purpose of the study. A pragmatic-explanatory continuum indicator summary tool was thus developed with the aim of identifying the characteristics of clinical trials that distinguish between effectiveness and efficacy issues. We verified whether clinical trials used the criteria proposed by the indicator summary tool, and we categorized these clinical trials according to a new classification.METHOD:A systematic survey of randomized clinical trials was performed. We added a score ranging from 0 (more efficacious) to 10 (more effective) to each domain of the indicator summary tool and proposed the following classifications: high efficacy (<25), moderate efficacy (25-50), moderate effectiveness (51-75), and high effectiveness (<75).RESULTS:A total of 844 randomized trials were analyzed. No analyzed trials used the criteria proposed by the indicator summary tool. Approximately 44% of the trials were classified as having moderate effectiveness, and 43.82% were classified as having moderate efficacy.CONCLUSIONS:Most clinical trials used the term “efficacy” to illustrate the application of results in clinical practice, but the majority of those were classified as having moderate effectiveness according to our proposed score. The classification based on the 0-100 score is still highly subjective and can be easily misunderstood in all domains based on each investigator’s own experiences and knowledge.

An alternative approach to deal with the absence of clinical trials: a proportional meta-analysis of case series studies

PURPOSE: Systematic reviews are criticized for frequently offering inconsistent evidences and absence of straightforward recommendations. Their value seems to be depreciated when the conclusions are uncertain. To describe an alternative approach of evaluating case series studies in health care when there is absence of clinical trials. METHODS: We provide illustrations from recent experiences. Proportional meta-analysis was performed on surgical outcomes: (a) case series studies, (b) use of cryoablation or radiofrequency ablation, and (c) patients with small renal cell carcinoma. The statistically significant difference between both interventions studied was defined if their combined 95% confidential interval (CI) did not overlap. RESULTS: As demonstrated by the example, this analysis is an alternative approach to provide some evidence of the intervention´s effects under evaluation and plotting all available case series in the absence of clinical trials for the health field. CONCLUSIONS: Although we are leading to a low level of evidence to determine efficacy, effectiveness and safety of interventions this alternative approach can help surgeons, physicians and health professionals for a provisionally decision in health care along with their clinical expertise and the patient´s wishes and circumstances in the absence of high-quality primary studies. It´s not a replacement for the gold standard randomized clinical trial, but an alternative analysis for clinical research.

“Kocher debre semelaigne syndrome” : a case report.

We report a case of 12 year old male child with Kocher-Debré-Semelaigne syndrome (KDSS) which is a rare clinical presentation of hypothyroidism associated with muscular pseudo-hypertrophy. The main differential diagnosis is Duchenne Muscular Dystrophy. Pseudomuscular hypertrophy of KDSS is an acquired type of myopathy associated with severe and long standing hypothyroidism and is reversible with thyroxine supplement.

Trends of prevalence and pathological spectrum of head and neck cancers in North India.

BACKGROUND: Head and neck neoplasia constitute one of the commonest cancers in India. Use of smokeless tobacco (Pan masala, Zarda etc.) is on the increase in North India, especially in Uttar Pradesh, and is responsible for the large majority of these tumours. AIM: To assess the patients' characteristics, yearly prevalence and histopathological subtypes of the head and neck neoplasia (excluding oral cavity) in Allahabad and surrounding regions. SETTINGS AND DESIGN: A retrospective study of 11 years from 1990 to 2000 was designed. Data was collected year-wise using the tumor registry data. MATERIAL AND METHODS: All biopsies submitted for histopathology to the Pathology department were reviewed and analyzed for demographic data, site and diagnosis. STATISTICAL ANALYSIS: The Kolmogorov-Smirnov Two-Sample Test was utilized to determine whether two distributions are the same. RESULTS: A total of 40559 biopsies were examined in the department, of which, lesions of the head and neck region, excluding the oral cavity, constituted 694 biopsies (409 males and 285 females). One hundred and forty-four malignant lesions were reported, 114 being males and 30 females. A comparison of the age-specific prevalence rates of cancer during the study period showed that the prevalence was highest in patients belonging to the 50-59 years age group and squamous cell carcinoma Grade II was the most prevalent type. On an average, 58 new biopsies per annum were received. CONCLUSIONS: Properly structured site-specific data like this can augment the National Cancer Registry Programme and is an essential indicator for the magnitude and the pattern of the cancer problem in India.

Prevention of mother to child transmission of HIV.

Perinatal transmission is the most common cause of HIV infection in pediatric population below the age of 15 years. Much progress has been made to decrease the risk of transmission than just offering the option of medical termination of pregnancy to the mother. Future research is needed not only to develop the simple preventive program, but also to make it more cost effective, acceptable and accessible to the general population. Following review article focuses on the factors affecting the transmission of HIV, strategies for prevention of MTCT with special reference to use of nevirapine and breast-feeding practices in HIV positive mother. The role of counseling and voluntary testing is also stressed upon.