Lege Artis Medicinae

[HuMánia – Voluntary Peer Training for Future Physicians]

HORVÁTH Dóra Anita, DOMBI Anna Zsófia, FÉSŰS Szilvia, ROSTA Eszter Andrea, SÁNDOR Zsuzsanna, HEGEDŰS Katalin

MARCH 20, 2010

Lege Artis Medicinae - 2010;20(03-04)

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Lege Artis Medicinae

[Second game, 37th move and Fourth game 78th move]

VOKÓ Zoltán

[What has Go to do with making clinical decisions? One of the greatest intellectual challenges of bedside medicine is making decisions under uncertainty. Besides the psychological traps of traditionally intuitive and heuristic medical decision making, lack of information, scarce resources and characteristics of doctor-patient relationship contribute equally to this uncertainty. Formal, mathematical model based analysis of decisions used widely in developing clinical guidelines and in health technology assessment provides a good tool in theoretical terms to avoid pitfalls of intuitive decision making. Nevertheless it can be hardly used in individual situations and most physicians dislike it as well. This method, however, has its own limitations, especially while tailoring individual decisions, under inclusion of potential lack of input data used for calculations, or its large imprecision, and the low capability of the current mathematical models to represent the full complexity and variability of processes in complex systems. Nevertheless, clinical decision support systems can be helpful in the individual decision making of physicians if they are well integrated in the health information systems, and do not break down the physicians’ autonomy of making decisions. Classical decision support systems are knowledge based and rely on system of rules and problem specific algorithms. They are utilized widely from health administration to image processing. The current information revolution created the so-called artificial intelligence by machine learning methods, i.e. machines can learn indeed. This new generation of artificial intelligence is not based on particular system of rules but on neuronal networks teaching themselves by huge databases and general learning algorithms. This type of artificial intelligence outperforms humans already in certain fields like chess, Go, or aerial combat. Its development is full of challenges and threats, while it presents a technological breakthrough, which cannot be stopped and will transform our world. Its development and application has already started also in the healthcare. Health professionals must participate in this development to steer it into the right direction. Lee Sedol, 18-times Go world champion retired three years after his historical defeat from AlphaGo artificial intelligence, be­cause “Even if I become the No. 1, there is an entity that cannot be defeated”. It is our great luck that we do not need to compete or defeat it, we must ensure instead that it would be safe and trustworthy, and in collaboration with humans this entity would make healthcare more effective and efficient. ]

Clinical Neuroscience

[Is the implementation of Vojta therapy associated with faster gross motor development in children with cerebral palsy? ]

SANZ-MENGIBAR Jose Manuel , MENENDEZ-PARDIÑAS Monica , SANTONJA-MEDINA Fernando

[Vojta therapy has been reported as clinically beneficial for strength, movement and gross motor activities in individual cases and is being included within the second of three levels of evidence in interventions for cerebral palsy. The goal of this study is to understand the effect of Vojta therapy on the gross motor function. Our clinical trial followed a one group, pre-post design to quantify rates of changes in GMFM-88 after a two-months period undergoing Vojta therapy. A total of 16 patients were recruited. Post-intervention acceleration rates of GMFM-88-items acquisition (0.005; p<0.001) and Locomotor Stages (1.063; p<0.0001) increased significatively following Vojta the­rapy intervention. In this study, Vojta therapy has shown to accelerate the acquisition of GMFM-88-items and Loco­motor Stages in children with cerebral palsy younger than 18 months. Because functional training was not utilised, and other non-Vojta therapy intervention did not influence the outcome, Vojta therapy seems to activate the postural control required to achieve uncompleted GMFM-88-items. ]

Clinical Neuroscience

[Consensus statement of the Hungarian Clinical Neurogenic Society about the therapy of adult SMA patients]

MOLNÁR Mária Judit, KÁLMÁN Bernadette, KLIVÉNYI Péter, BOCZÁN Judit, SZÉLL Márta, KARCAGI Veronika, ZÁDORI Dénes

[Background – Spinal muscular atrophy (SMA) is an autosomal recessive, progressive neuromuscular disorder resulting in a loss of lower motoneurons. Recently, new disease-modifying treatments (two drugs for splicing modification of SMN2 and one for SMN1 gene replacement) have become available. Purpose – The new drugs change the progression of SMA with neonatal and childhood onset. Increasing amount of data are available about the effects of these drugs in adult patients with SMA. In this article, we summarize the available data of new SMA therapies in adult patients. Methods – Members of the Executive Committee of the Hungarian Clinical Neurogenetic Society surveyed the literature for palliative treatments, randomized controlled trials, and retrospective and prospective studies using disease modifying therapies in adult patients with SMA. Patients – We evaluated the outcomes of studies focused on treatments of adult patients mainly with SMA II and III. In this paper, we present our consensus statement in nine points covering palliative care, technical, medical and safety considerations, patient selection, and long-term monitoring of adult patients with SMA. This consensus statement aims to support the most efficient management of adult patients with SMA, and provides information about treatment efficacy and safety to be considered during personalized therapy. It also highlights open questions needed to be answered in future. Using this recommendation in clinical practice can result in optimization of therapy.]

Clinical Neuroscience

[The importance of patient reported outcome measures in Pompe disease]

GROSZ Zoltán, MOLNÁR Mária Judit, MOLNÁR Viktor, LÁSZLÓ Izabella, SZEGEDI Márta, VÁRHEGYI Vera

[In recent decades it has become increasingly important to involve patients in their diagnostic and treatment process to improve treatment outcomes and optimize compliance. By their involvement, patients can become active participants in therapeutic developments and their observations can be utilized in determining the unmet needs and priorities in clinical research. This is especially true in rare diseases such as Pompe disease. Pompe disease is a genetically determined lysosomal storage disease featuring severe limb-girdle and axial muscle weakness accompanied with respiratory insufficiency, in which enzyme replacement therapy (ERT) now has been available for 15 years. In our present study, patient reported outcome measures (PROMs) for individuals affected with Pompe disease were developed which included questionnaires assessing general quality of life (EuroQoL, EQ-5D, SF36), daily activities and motor performance (Fatigue Severity Score, R-PAct-Scale, Rotterdam and Bartel disability scale). Data were collected for three subsequent years. The PROM questionnaires were a good complement to the physician-recorded condition assessment, and on certain aspects only PROMs provided information (e.g. fatigue in excess of patients’ objective muscle weakness; deteriorating social activities despite stagnant physical abilities; significant individual differences in certain domains). The psychological effects of disease burden were also reflected in PROMs. In addition to medical examination and certain endpoints monitored by physicians, patient perspectives need to be taken into account when assessing the effectiveness of new, innovative treatments. With involvement of patients, information can be obtained that might remain uncovered during regular medical visits, although it is essential in determining the directions and priorities of clinical research. For all orphan medicines we emphasize to include patients in a compulsory manner to obtain general and disease-specific multidimensional outcome measures and use them as a quality indicator to monitor treatment effectiveness.]

Lege Artis Medicinae

[Benefits of SGLT-2 inhibitors: beyond glycemic control]

BALOGH Zoltán, SIRA Lívia

[In the recent years, according to international and Hungarian guidelines, in addition to lifestyle modification, metformin is the preferred initial glucose-lowering drug for most people with type 2 diabetes, if not contraindicated. Sodium glucose co­transporter-2 inhibitors have been shown to reduce progression of chronic kidney disease, or kidney failure, as well as the risk of hospitalizations for congestive heart failure and (mainly in secondary prevention) cardiovascular death in patients with type 2 diabetes. For major adverse cardiovascular events and for the renoprotection, there seems to be no class effect. On the other hand, a class effect of sodium glucose co­transporter-2 inhibitors is evident for hospitalization for heart failure. In this review the authors summarize novel data about sodium glucose cotransporter-2 inhibitors, and about their new perspectives in the near future.]