What is a pilot or feasibility study? A review of current practice and editorial policy

Background: In 2004, a review of pilot studies published in seven major medical journals during 2000-01 recommended that the statistical analysis of such studies should be either mainly descriptive or focus on sample size estimation, while results from hypothesis testing must be interpreted with caution. We revisited these journals to see whether the subsequent recommendations have changed the practice of reporting pilot studies. We also conducted a survey to identify the methodological components in registered research studies which are described as 'pilot' or 'feasibility' studies. We extended this survey to grant-awarding bodies and editors of medical journals to discover their policies regarding the function and reporting of pilot studies. Methods: Papers from 2007-08 in seven medical journals were screened to retrieve published pilot studies. Reports of registered and completed studies on the UK Clinical Research Network (UKCRN) Portfolio database were retrieved and scrutinized. Guidance on the conduct and reporting of pilot studies was retrieved from the websites of three grant giving bodies and seven journal editors were canvassed. Results: 54 pilot or feasibility studies published in 2007-8 were found, of which 26 (48%) were pilot studies of interventions and the remainder feasibility studies. The majority incorporated hypothesis-testing (81%), a control arm (69%) and a randomization procedure (62%). Most (81%) pointed towards the need for further research. Only 8 out of 90 pilot studies identified by the earlier review led to subsequent main studies. Twelve studies which were interventional pilot/feasibility studies and which included testing of some component of the research process were identified through the UKCRN Portfolio database. There was no clear distinction in use of the terms 'pilot' and 'feasibility'. Five journal editors replied to our entreaty. In general they were loathe to publish studies described as 'pilot'. Conclusion: Pilot studies are still poorly reported, with inappropriate emphasis on hypothesis-testing. Authors should be aware of the different requirements of pilot studies, feasibility studies and main studies and report them appropriately. Authors should be explicit as to the purpose of a pilot study. The definitions of feasibility and pilot studies vary and we make proposals here to clarify terminology

Interactions of sodium pentobarbital with d-glucose and l-sorbose transport in human red cells

AbstractPentobarbital acts as a mixed inhibitor of net d-glucose exit, as monitored photometrically from human red cells. At 30°C the Ki of pentobarbital for inhibition of Vmax of zero-trans net glucose exit is 2.16±0.14 mM; the affinity of the external site of the transporter for d-glucose is also reduced to 50% of control by 1.66±0.06 mM pentobarbital. Pentobarbital reduces the temperature coefficient of d-glucose binding to the external site. Pentobarbital (4 mM) reduces the enthalpy of d-glucose interaction from 49.3±9.6 to 16.24±5.50 kJ/mol (P<0.05). Pentobarbital (8 mM) increases the activation energy of glucose exit from control 54.7±2.5 kJ/mol to 114±13 kJ/mol (P<0.01). Pentobarbital reduces the rate of l-sorbose exit from human red cells, in the temperature range 45°C–30°C (P<0.001). On cooling from 45°C to 30°C, in the presence of pentobarbital (4 mM), the Ki (sorbose, glucose) decreases from 30.6±7.8 mM to 14±1.9 mM; whereas in control cells, Ki (sorbose, glucose) increases from 6.8±1.3 mM at 45°C to 23.4±4.5 mM at 30°C (P<0.002). Thus, the glucose inhibition of sorbose exit is changed from an endothermic process (enthalpy change=+60.6±14.7 kJ/mol) to an exothermic process (enthalpy change=−43±6.2 7 kJ/mol) by pentobarbital (4 mM) (P<0.005). These findings indicate that pentobarbital acts by preventing glucose-induced conformational changes in glucose transporters by binding to ‘non-catalytic’ sites in the transporter

Hereditary sensory autonomic neuropathy II, a rare disease in a large Pakistani family

Hereditary Sensory Autonomic Neuropathy II (HSAN II) is a rare genetic disorder, characterized by severe loss of pain, temperature and touch sensation. Injuries in these patients can progress to necrosis and shedding of digits and limbs. Here we report two cases of HSAN II belonging to a Pakistani family. Individual 1, a forty five year old man, had complete loss of pain sensation since birth. Self-mutilation and complication of injuries resulted in the shedding of all the digits and right foot and surgical amputation of left leg. Individual 2, a five year old girl,had delay in healing of wounds and self-mutilation. Examination showed a complete lack of pain sensation throughout her body and hyporeflexia. As the genetic cause of HSAN II is unknown, identification of more patients will allow further research on this disease and possibly develop a cure

A rare case of simple hereditary recessive optic atrophy

Simple Autosomal Recessive Optic Atrophy (AROA) is a rare hereditary disorder that belongs to a group of disorders called Hereditary Optic Atrophy. Patients diagnosed with simple AROA have complete blindness since birth or from first few months of life. This blindness does not improve with age. However, no other organ or system is affected in this disorder. There is no known cause or gene mutation associated with it. Here we report a case of a two year old child diagnosed with simple AROA. Family history of the patient revealed that an older deceased relative also suffered from similar symptoms. Identification of this and similar cases of the simple AROA can help us better understand this disorder and hopefully one day help us develop a treatment for it

How international SME's vicarious learning may improve their performance? The role of absorptive capacity, strength of ties with local SMEs, and their prior success experiences

This study investigates whether inter-firm relationships can raise innovation and overall performance during SME internationalization, focusing on how SMEs learn from firms in transnational markets and the nature of such relationships. It contributes to research by proposing the role of vicarious learning from networked firms in the host country to improve their absorptive capacity (ACAP), innovation, and overall performance. In particular, this study proposes the moderating roles of the strength of ties with and prior success experience of SMEs in the host country market for enhancing international SMEs' vicarious learning to improve their ACAP, innovation, and overall performance. Structural equation modeling was applied to a sample of 163 valid responses received from international SMEs operating in various industrial sectors in Saudi Arabia. The obtained results support the significantly positive role of international SMEs' vicarious learning from local firms in developing their ACAP and enhancing their innovation and overall performance. However, international SMEs must have strong ties with local firms and learn from such firms' prior success experiences to derive these benefits fully

A Rare Case of Becker Disease in a 7 Year Old Boy

Becker Disease is an autosomal recessive version of the rare congenital disorder called Myotonia Congenita. Due to the rarity of Becker Disease, the genetic and pathological basis of this disease have not been studied well and possible diagnostic methods and techniques are yet to be explored. The existing method of diagnosis of such a case is predominantly dependent on the clinical examination. More work and studies need to be done on the diagnostic aspect of this disease to discover newer diagnostic methods for this disease, which are more reliable and specific. In order to develop better diagnostic methods for the disease, more cases of Becker disease need to be reported, with details of clinical and structural abnormalities. Here, we present the case report of a seven year old boy who has a history and clinical findings suggestive of Becker disease. Identification of this and similar cases of Becker disease can help us better understand this disease and hopefully one day help us develop a treatment for it

A new model of master of philosophy in physiological sciences

The objectives of Master of Philosophy (MPhil) in Physiological Sciences are: 1) to describe the new ways in which anatomy, biochemistry and physiology on one hand, and microbiology, pathology and pharmacology on other hand meet their functional requirements through multidisciplinary integrated concepts; 2) to elucidate relationships between cell biology, molecular biology and molecular genetics by connecting dots of how cell functions are driven by molecules and being controlled by genes. This forms the basis of cell, molecular and genetics [CMG] module upon which 7 multidisciplinary modules of Physiological Sciences follow; 3) these 24 credit hours provide the physiological basis for PhD studies as well as faculty development to enhance learning abilities of medical student; 4) the modules constitute Cardio- Respiratory Physiological Sciences, GI and Renal Physiological Sciences, Neurosciences, Endo-Reproductive Physiological Sciences.; 5) it has integrated microbiology, pathology and pharmacology in a unique way through CMG of microbes leading to associated pathology and mechanisms of prescribed drugs; 6) it has additional synopsis and thesis friendly course work leading to comprehensive examinations; 7) the year two deals with research work of 6 credit hours leading to defense of thesis; 8) The MPhil in Physiological Sciences is fundamentally different from what is being offered elsewhere. It prepares and offers a good spring board to dovetail PhD studies as well as faculty and institutional development. This is the first study that deals with innovative programmes in research, learning and education in the field of physiological sciences. This broad-based MPhil would make its recipients competent, critical, confident and productive learner. This is a completely unique design of a curriculum that has no comparable examples elsewhere. Our mission is to educate graduate students in the field of Physiological Sciences such that they have a complete grasp over the broad-based integrated concepts of basic health sciences. Upon completion of their education, the students will be able to use the duality of imagination and skepticism. Hence, the students will contribute to their fields by unfolding their creative energy

Misconceptional views about epilepsy exist across social class system of society

Epilepsy affects over 1% of population worldwide. Studies have shown that although our understanding about epilepsy has come a long way, misconceptions about its etiology and treatment exist in rural slums. However, no study has been conducted to see whether such misconceptions exist in middle and upper socio-economic class. This study aims to explore the existence of misconceptions and social stigma in the middle and upper socio-economic class. Materials and Methods: We conducted a survey-based study about epilepsy on 227 participants, belonging to middle and upper socio-economic class. Results: The symptoms of generalized tonic-clonic seizures were correctly described by participants. However, magic, superstition and ‘jin’ were considered as the etiology by some. Incorrect treatment options, like shoe sniffing and ‘taweez’ etc., were also mentioned. Social stigmas regarding sharing information about epilepsy and marrying epileptics also existed. Discussion: Our study shows that although the general understanding about epilepsy was correct in majority of participants belonging to the middle upper socio-economic class, it was restricted to generalized tonic clonic seizures. Key misconceptions regarding the etiology and treatment of epilepsy and social stigma associated with it did exist. These findings emphasize the need to educate all segments of the society about epileps

Thermal effects in the Input Optics of the Enhanced Laser Interferometer Gravitational-Wave Observatory interferometers

We present the design and performance of the LIGO Input Optics subsystem as implemented for the sixth science run of the LIGO interferometers. The Initial LIGO Input Optics experienced thermal side effects when operating with 7 W input power. We designed, built, and implemented improved versions of the Input Optics for Enhanced LIGO, an incremental upgrade to the Initial LIGO interferometers, designed to run with 30 W input power. At four times the power of Initial LIGO, the Enhanced LIGO Input Optics demonstrated improved performance including better optical isolation, less thermal drift, minimal thermal lensing, and higher optical efficiency. The success of the Input Optics design fosters confidence for its ability to perform well in Advanced LIGO