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ISDN2014_0221: Does angiogenesis play a role in the development of hippocampal atrophy in the pilocarpine rat model of temporal lobe epilepsy?
Author(s) -
Roth Raquel,
Benini Ruba,
Khoja Zehra,
Avoli Massimo,
Shevell Michael,
Wintermark Pia
Publication year - 2015
Publication title -
international journal of developmental neuroscience
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.761
H-Index - 88
eISSN - 1873-474X
pISSN - 0736-5748
DOI - 10.1016/j.ijdevneu.2015.04.183
Subject(s) - epilepsy , pilocarpine , hippocampal formation , neuroscience , atrophy , angiogenesis , temporal lobe , medicine , pathology , psychology
Background: Several inborn errors of metabolism leading to neurodevelopmental issues including intellectual disability have become amenable to causal therapy. However there is a gap between identifying new treatments and assessing their effectiveness due to lack of generally accepted endpoints particularly with behaviour and cognitive functions as changes might not be captured as significant by formal tests. These challenges arise in part from the considerable phenotypic clinical heterogeneity and variability of treatment responses in each patient. In 2011, we developed a Personalized Evaluation Model (PEM) using a systematic consultative strategy of decision-making and treatment evaluation that incorporates individual children/families’ insights about the impact of treatment on everyday activities and their preferred outcomes. Methods: PEM is a 4-step process: (i) identify personal outcomes of specific interest (“POSI”) from the patient/family in consultation with the physician (ii) determine which drug effects would make the treatment useful from the child/family’s view point (iv) assess changes in POSI over time and scale this change using goal attainment scaling technique (v) bring this information to the discussion with care providers to make the most appropriate decisions regarding the treatment. At this stage both POSI and traditional clinical endpoints are considered in the context of determining treatment utility. Results: The model was successfully implemented in a few patients with Creatine Transporter Deficiency treated with amino acid supplements. There was no change in scores obtained using standard scales as they were not sensitive enough to capture the variance. Discrepancies were found between results of standard cognitive scales and POSI confirming the value of our model in establishing endpoints relevant to the patient. Discussion: PEM approach is ultimately outcomes oriented, with a focus on what patients’ value–the particular improvements that they desire in their ability to functioning in their daily lives. This complements the traditional evidence-based approach to treatment recommendation.