Paediatric MS
Tysabri may be an option for children with Multiple Sclerosis
Off-label treatment with natalizumab (Tysabri) for 20 children with severe multiple sclerosis reduced relapses and brain lesions seen on MRI scans, a retrospective analysis found.
The mean annualized relapse rate while on natalizumab in these patients was 0.4, compared with 3.77 before starting the drug (P<0.001), according to Barbara Kornek, MD, of Austria's Medical University of Vienna, and colleagues.
Also, new T2/fluid attenuated inversion recovery (FLAIR) lesions declined from a mean of 7.8 before natalizumab to 0.5 on the drug (P=0.001), the researchers reported online in JAMA Neurology.
But the treatment was not without adverse events, Kornek and colleagues noted. Half the patients experienced some type of clinical event, including one case each of severe asthenia, laryngeal edema, and anaphylaxis. Two had recurrent infections, although it was not clear that natalizumab was responsible.
Eight of the children also exhibited laboratory abnormalities. Four developed mild anemia, two showed transient elevations in liver enzymes, and two developed hyperbilirubinemia.
In addition, two patients developed high levels of neutralizing antibodies to natalizumab that led to withdrawal of the drug. Three other patients stopped natalizumab after tests showed the presence of antibodies to the JC virus, a strong risk factor for development of progressive multifocal leukoencephalopathy (PML), which can be fatal.
One patient had tested positive for JC virus antibodies before starting natalizumab, but the decision was made to use the drug anyway because of a "highly active disease course." Overall, the researchers found, five of 13 patients tested for JC virus antibodies were positive.
Natalizumab is not currently approved for pediatric use in the U.S. or Europe, but it is sometimes used anyway in children with very severe disease or who do not respond to more conventional disease-modifying treatments.
The 20 children Kornek and colleagues reported on were mostly teenagers and were receiving the drug in 11 clinics in Germany and Austria. In addition to the standard outcomes of relapse rates and MRI lesions, the researchers paid particular attention to development of neutralizing anti-drug antibodies and JC virus antibody status, as these had not previously been studied in a pediatric population.
Mean age at disease onset in these patients was 15.2 (SD 1.3) and the mean age when natalizumab was started was 16.7 (SD 1.1). All but four were girls.
One patient started on natalizumab as first-line treatment, 15 had previously received one disease-modifying treatment, and four had received two.
With a mean of 18 months of disease duration before starting natalizumab, the average number of relapses was 4.3, with 3.1 occurring in the previous year -- numbers that signify highly active disease.
Four of the patients had undergone plasma exchange for a severe, prolonged relapse event.
The mean number of T2/FLAIR brain lesions at pre-natalizumab baseline was 30 (SD 17), with a mean of 2 gadolinium-enhancing lesions.
Mean treatment duration for natalizumab in the analysis was 20 months, during which time 14 of the 20 patients remained relapse-free. Among seven patients on the drug for 2 years or more, four stayed relapse-free.
Only two patients did not show a significant reduction in relapse frequency with natalizumab.
In an analysis combining clinical and MRI results, six of 13 patients with adequate data were deemed to be free of disease activity with natalizumab, insofar as they had no relapses, did not develop new MRI lesions, or show disability progression.
Among 10 patients with complete data for at least 1 year, four were disease-free throughout follow-up.
Most of the patients actually showed some lessening of disability, with a median change in EDSS score of -0.25 points (interquartile range -1.25 to 0.00) relative to baseline, when the group median was 2.0 points.
Kornek and colleagues also looked at the clinical course of patients who had stopped natalizumab either permanently or temporarily. After excluding two who were lost to follow-up after stopping the drug and one who had stopped temporarily during pregnancy, there were nine patients available for this analysis, including eight followed for at least 6 months.
Among those eight, six developed relapses in the first 6 months off natalizumab. The annualized relapse rate among all nine patients showed a strong trend toward increase (mean difference 1.47, 95% CI -0.04 to 2.98, P=0.06).
Overall, Kornek and colleagues concluded that natalizumab "may be safe and effective against MS in pediatric patients with breakthrough disease," but they cautioned that controlled trials are needed to confirm a favourable benefit-risk balance.
The study had no external funding.
Study authors reported relationships with Biogen Idec, Merck Serono, Teva, Bayer Schering, Novartis, Sanofi, UCB, Orion, Solvay, Mundipharma, and Ipsen.
Primary source: JAMA Neurology
Source reference: Kornek B, et al "Natalizumab therapy for highly active pediatric multiple sclerosis" JAMA Neurol 2013; DOI: 10.1001/jamaneurol.2013.923.
Source: MedPage Today © 2013 MedPage Today, LLC. (20/02/13)
Natalizumab in paediatric multiple sclerosis: results of a cohort of 55 cases
Abstract
BACKGROUND: Limited information is available on the use of natalizumab (NA) in pediatric multiple sclerosis (ped-MS) patients.
OBJECTIVE: The purpose of this study was to describe the long-term effects of NA in a large cohort of active ped-MS patients.
METHODS: Patients with definite ped-MS were treated with NA if in the previous year they had experienced at least two relapses or a severe relapse with incomplete recovery while on immunomodulating treatment, or at least two relapses and new magnetic resonance imaging (MRI) lesions regardless of any prior treatment.
RESULTS: The study included 55 patients (mean age: 14.4 years, mean number of relapses: 4.4, pre-treatment mean disease duration: 25.5 months). They received a median number of 26 infusions. Three relapses occurred during the follow-up, one female patient continued to deteriorate in cognitive functioning. Mean Expanded Disability Status Scale (EDSS) scores decreased from 2.7 to 1.9 at the last visit (p<0.001). During the follow-up the majority of patients remained free from MRI activity. Transient and mild clinical adverse events occurred in 20 patients. Mild hematological abnormalities occurred in seven patients. Anti-JCV antibodies were detected in 20/51 tested patients.
CONCLUSIONS: NA was well tolerated in all patients. A strong suppression of disease activity was observed in the majority of patients during the follow-up.
Ghezzi A, Pozzilli C, Grimaldi L, Moiola L, Brescia-Morra V, Lugaresi A, Lus G, Rinaldi F, Rocca M, Trojano M, Bianchi A, Comi G, Filippi M; the Italian MS Study Group.
Source: Mult Scler. 2013 Feb 11 & Pubmed PMID: 23401129 (15/02/13)
One in Three Children with MS has Cognitive Impairment
Data from the largest multicenter study accessing cognitive functioning in children with multiple sclerosis (MS) reveals that one-third of these patients have cognitive impairment, according to a research paper published in the Journal of Child Neurology. Led by Lauren B. Krupp, MD, Director of the Lourie Center for Pediatric Multiple Sclerosis at Stony Brook Long Island Children’s Hospital, the study indicates that patients experience a range of problems related to cognition.
In “Cognitive Impairment Occurs in Children and Adolescents with Multiple Sclerosis: Results from a United States Network,” Dr. Krupp and colleagues from Stony Brook and five other national Pediatric MS Centers of Excellence measured the cognitive functioning of 187 children and adolescents with MS, and 44 who experienced their first neurologic episode (clinically isolated syndrome) indicative of MS. They found that 35 percent of the patients with MS and 18 percent of those with clinically isolated syndrome met criteria for cognitive impairment. All patients were under age 18 with an average disease duration of about two years.
“This study is important because it represents the largest study to date to apply a comprehensive neuropsychological battery of tests to evaluate the cognitive functioning of children with MS, and the results clearly show us that cognitive issues are widespread and can occur early on in the disease course of these patients,” said Dr. Krupp, also a Professor of Neurology at Stony Brook University School of Medicine. “These are critically important findings that emphasize the need for prompt recognition of our patients’ cognitive problems and for neurologists and other MS specialists to discover ways to intervene and help improve the cognitive abilities of these children while they are in school and beyond.”
The authors report that the network of Pediatric MS Centers of Excellence used a comprehensive battery of 11 tests where the scores addressed cognitive domains such as general ability, reading and language, attention, working memory and speed processing, executive functioning, verbal learning and recall, visual-motor integration, and fine motor speed and coordination. The testing took approximately 2.5 hours to complete, and a participant was considered impaired when the proportion of impaired scores from the categorized tests was greater than one-third.
They found that the most frequent areas of impairment were fine motor coordination (54 percent), visual-motor integration (50 percent), and speeded information processing (35 percent). Among the various tests that showed the most frequent rate of impairment were the grooved peg board, a measure of fine motor speed and coordination, and a test for visual-motor integration, a measure that is dependent in part on fine motor coordination.
The researchers factored in clinical variables in the analysis, including disease duration, diagnosis status, age of disease onset, and disability. They found that a diagnosis of MS and overall neurologic disability were the only two independent predictors of cognitive impairment.
They also factored in sociodemographic variables such as age, gender and ethnicity, and leveraged the geographic diversity of the Pediatric Multiple Sclerosis Centers of Excellence network to create the sample. Centers participating in the study included: the Lourie Center for Pediatric Multiple Sclerosis at Stony Brook; the University of California, San Francisco; University of Alabama, Birmingham; the Jacobs Neurological Institute, SUNY Buffalo; Massachusetts General Hospital in Boston, and the Mayo Clinic in Rochester, Minn.
The authors point out that the next step for pediatric MS investigators within the network is to further the research “to develop strategies for prompt identification of children with multiple sclerosis at risk for cognitive problems so that treatment can be initiated.”
The study was supported in part by the National Multiple Sclerosis Society.
About the Lourie Center for Pediatric Multiple Sclerosis
The Lourie Center for Pediatric Multiple Sclerosis, part of Stony Brook Long Island Children’s Hospital, was established to advance the recognition, evaluation, and treatment of children and adolescents with MS through the creation of a multidisciplinary program dedicated to clinical care and scientific research of children and adolescents with MS. The Center includes a team of clinical experts in MS, pediatric neurology, nursing, social work, psychiatry, neuroimaging, and neuropsychology. Collaborating with clinicians are researchers from a variety of fields including neuroscience, neuroimmunology, genetics, physics, and chemistry. The center has been designated a Center of Excellence by the National Multiple Sclerosis Society.
Source: Newswire ©2013 Newswise, Inc (06/02/13)
Obese girls at risk of multiple sclerosis, study finds
Obese girls are at greater risk of developing multiple sclerosis or MS-like illness, according to a new study published Wednesday in the online journal Neurology.
Researchers looked at body mass index (BMI) data from more than 900,000 children from the Kaiser Permanente Southern California Children's health study. Seventy-five of those children and adolescents between the ages of 2 and 18 were diagnosed with pediatric MS. More than 50% of them were overweight or obese, and the majority were girls.
According to the study, the MS risk was more than one and a half times higher for overweight girls, almost two times higher in moderately obese girls and almost four times higher in extremely obese girls.
"Over the last 30 years, childhood obesity has tripled," said study author Dr. Annette Langer-Gould, a neurologist and regional MS expert for Kaiser Permanente in Southern California. "In our study, the risk of pediatric MS was highest among moderately and extremely obese teenage girls, suggesting that the rate of pediatric MS cases is likely to increase as the childhood obesity epidemic continues."
MS is a chronic, debilitating disease that attacks the central nervous system. "Some patients do very well and have minimal to no disability even 20 years later," Langer-Gould said, "While other patients do poorly and can be wheelchair bound in 5 years. It's a huge spectrum."
Dr. Tanuja Chitnis is a neurologist and pediatric MS specialist at Massachusetts General Hospital for Children with 50 MS publications to her credit. She says 10 years ago MS was not recognized as a disease that occurred in children, but today evidence is mounting that obesity is a risk factor for MS in kids, particularly adolescent girls.
"This is one more piece of evidence, but really in order to make a definitive link, you need at least five or six studies showing the same thing," she says. "You need to have an underlying biological reason, which still has not been worked out and you need to show that blocking or interfering with the biological mechanism can prevent the disease."
"The overall message is that there are an increasing number of diseases associated with obesity and particularly early obesity and that it's an important risk factor to try to mitigate. It is something you can do something about," Chitnis says.
According to the Centers for Disease Control and Prevention, over the last 30 years childhood obesity has doubled in children and tripled in teenagers. In 2010, more than a third of all children and teens were overweight or obese.
At Children's Hospital of Alabama, pediatric neurologist Dr. Jayne Ness has seen more than 100 pediatric MS patients, predominantly girls, whose average age at onset is 13. Ness told CNN she has noticed a rise in obesity in their MS patients, kids who at the time of diagnosis are obese.
"Does this mean that obesity is a risk factor for MS? We don't know yet," Ness said. "It's one more piece that helps us potentially better understand some of the underlying triggers of pediatric MS and may help us understand MS in general."
Langer-Gould says that while pediatric MS is very rare - only 1.6 per 100,000 children - there are red flags parents should look out for. "Constant numbness or tingling from the waist down or numbness, pins and needles sensations in the chest, abdomen or back that last for 24 hours."
Those children should be evaluated by a neurologist. Other symptoms to have checked out are collapsing weakness in the legs after modest exertion, and pain and loss of vision in one eye.
The National MS Society estimates about 10,000 children in the United States have the disease and another 10 to 15,000 have had at least one MS-like symptom. An estimated 5% of all MS cases worldwide are childhood or adolescent onset.
Source: CNN © 2013 Cable News Network.(31/01/13)
Fatigue, emotional functioning, and executive dysfunction in pediatric MS.
Abstract
Objective: Fatigue, depression, anxiety, and executive dysfunction are associated with multiple sclerosis (MS) in adults. Existing research suggests similar problems in pediatric MS, but relationships between these variables have not been investigated. This study investigates the associations between executive functioning and fatigue, emotional functioning, age of onset, and disease duration in pediatric MS.
Methods: Twenty-six MS or Clinically Isolated Syndrome (CIS) patients, ages 7 to 18, were evaluated through a multidisciplinary demyelinating diseases clinic. Participants completed neuropsychological screening including Verbal Fluency, Digit Span, and Trail-Making Test. Parents completed rating forms of behavioral, emotional, and executive functioning. Patients and parents completed questionnaires related to the patient's quality of life and fatigue. Pearson's correlation coefficients were calculated to investigate relationships between fatigue, emotional functioning, and executive functioning, as well as to examine correlations between parent and child reports of fatigue.
Results: Rates of parent-reported anxiety, depression, fatigue, and executive dysfunction varied widely. Means were below average on the Trail-Making Test and average on Verbal Fluency and Digit Span, though scores varied widely. Various fatigue and emotional functioning indices-but not age of onset or disease duration-significantly correlated with various performance-based measures of executive functioning.
Conclusion: Results indicate pediatric MS is associated with some degree of fatigue, emotional difficulties, and executive dysfunction, the latter of which is associated with the two former. Notably, age of onset and disease duration did not significantly correlate with executive functioning. Results advance understanding of psychological and clinical variables related to neurocognitive outcomes in pediatric MS.
Holland AA, Graves D, Greenberg BM, Harder LL.
Children's Medical Center Dallas , Dallas , Texas , USA.
Child Neuropsychol. 2012 Dec 7. & Pubmed PMID: 23216329 (14/12/12)
Diffusion tensor analysis of paediatric MS and clinically isolated syndromes
Summary: This new imaging study from the USA has applied diffusion imaging techniques to study connectivity in major white matter tracts in a paediatric MS population. The authors report that there are significant anisotropic abnormalities in the NAWM of major tracts in children with MS. In contrast, there were no significant changes in paediatric patients with CIS compared with controls at baseline, and DTI measures did not predict conversion to MS.
Abstract
BACKGROUND AND PURPOSE:
DTI has shown focal and diffuse white matter abnormalities in adults and children with MS. Here we explore whether DTI abnormalities are present at the time of a first attack or CIS in children and whether early DTI features can predict the development of MS.
MATERIALS AND METHODS:
We assessed region-of-interest and tract-based mean ADC and mean FA values for 3 major white matter pathways and NAWM in 20 children with MS, 27 children with forms of CIS, and controls. Tracts were selected by using standard region-of-interest placements on color FA maps. Identical ROIs were placed in the NAWM on b = 0 T2-weighted images to ensure that both ROIs and resulting tracts passed through NAWM. Conventional MR imaging characteristics were assessed by visual inspection. Statistical analysis compared FA and ADC values between groups by a t test. Logistic regression assessed the predictive value of DTI measures and published conventional MR imaging measures for conversion from CIS to MS.
RESULTS:
In paediatric patients with MS, all white matter pathways and analysis confined to the NAWM demonstrated higher mean ADC values and lower mean FA than in controls. In contrast, there were no significant differences in mean ADC and mean FA of white matter pathways in all CIS cohorts compared with controls. In the CIS cohort, none of the DTI measures in white matter pathways or in NAWM were significantly associated with conversion to RRMS in univariate or multivariate models (P > .05 in all models).
CONCLUSIONS:
There are significant anisotropic abnormalities in the NAWM of major tracts in children with MS. In contrast, there were no significant changes in pediatric patients with CIS compared with controls at baseline. DTI measures did not predict conversion to MS. The period between CIS and conversion to paediatric MS may represent a window of opportunity for the prevention of diffuse damage in the CNS and potentially progressive disability.
Vishwas MS, Healy BC, Pienaar R, Gorman MP, Grant PE, Chitnis T.
From Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital for Children, Boston, Massachusetts; Athinoula A. Martinos Center for Biomedical Imaging, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts; and Biostatistics Center, Massachusetts General Hospital, Boston, Massachusetts.
Source: AJNR Am J Neuroradiol. 2012 Aug & Pubmed PMID: 22859275 (08/08/12)
Research shines light on childhood multiple sclerosis
New research suggests the number of children suffering from multiple sclerosis may be higher than previously thought.
It remains a very rare condition and only a third of young people who have an attack will go on to develop MS in later life.
But it can have a devastating affect on children and their families.
One of those is 15-year-old Emily Murdoch from Cannock in Staffordshire.
Like many girls her age, she loves horses and has been riding since she was a little girl.
One day she hopes to represent Britain in competition, but she does not know if it will be in the Olympics or Paralympics.
Wheelchair
At the age of 12, Emily was diagnosed with childhood multiple sclerosis. An attack can leave her confined to a wheelchair when her legs stop working.
She suffers from severe tiredness, muscle spasms and numb hands and legs.
"It's not really very nice," she says "especially when my legs go on me, because it's the second time my legs have gone on me."
"It took my left hand last time.
"My legs get weaker. I know when my MS is bad because my legs get weak and my horse can feel it."
Scarred brain
At Birmingham Children's Hospital, I'm shown a scan of Emily's brain by Michael Absoud, a clinical research fellow at the University of Birmingham.
He is one of the authors of new research into how many children are affected by the condition.
Dr Absoud points to lighter areas on the scan which are scars left on the brain after an attack of multiple sclerosis.
A diagnosis of MS in children is rare and sometimes controversial - not all doctors believe it exists.
But Dr Absoud says their research suggests it is more widespread than first thought.
"The research by itself has helped raise awareness that this disease does indeed happen in children.
"The first presentation can be in children as young as three years old."
Over a 13-month period, the researchers surveyed paediatricians and ophthalmologists who might see children suffering from MS-like symptoms.
They found that MS-type attacks affected around 125 children in the UK each year. Two to three children per week were having MS-like problems with numbness or blurred vision.
This doesn't mean every child who suffers these kind of symptoms is having an attack of MS.
And Dr Absoud says it's thought only about a third of children who have an attack will develop the condition in later life.
"Not all children who experience an MS-like attack will go on to develop the condition.
"Some will recover fully and never experience similar symptoms again, while others have longer-term problems that eventually lead to a diagnosis of MS.
"Although rare, MS can occur in childhood, but knowledge about the number of children affected by the condition, how the illness progresses and how it could best be treated is severely lacking, which is why our research is so important."
Dr Doug Brown, head of biomedical research at the MS Society which helped fund the new research, says around one in 20 adults with MS experienced their first symptoms in childhood.
"Historically MS has always been considered as an older person's condition, but we're now seeing people diagnosed much younger.
"So the more we understand about childhood MS, the better health professionals can be at diagnosing the condition and offering treatment and vital support to young people and their families."
Devastating impact
But for the families of those children who are affected, the impact of that first diagnosis can be devastating, as Emily's mum Tracey Murdoch discovered.
"You can't put it into words.
"You physically don't know the 'what ifs?', 'what's happened?', 'why?'.
"You instantly think - wheelchair, old person with MS. That's clearly not the case, as we found out when Emily was diagnosed."
Emily is managing to keep up her riding, despite fresh attacks.
When she is well she looks and acts like a healthy teenager. But this is a condition for which there is no known cure and only limited treatment.
MS in children is very rare - around 10 children in every million will be diagnosed. Making that diagnosis early seems to be the key to at least slowing the progress of condition.
And as Emily shows, with enough courage, determination and support, children with MS can go on to live a life that is at least close to normal.
Source: BBC News © British Broadcasting Corporation 2012 (18/06/12)
No association between chronic cerebrospinal venous insufficiency and pediatric-onset MS
Abstract
Objective: Chronic cerebrospinal venous insufficiency (CCSVI) was hypothesized to play a causative role in multiple sclerosis (MS). The assessment of pediatric-onset MS (POMS) may provide a unique window of opportunity to study hypothesized risk factors in close temporal association with the onset of the disease.
Methods: Internal jugular veins, vertebral veins and intracranial veins were evaluated with extracranial and intracranial ultrasound in 15 POMS and 16 healthy controls. Assessor’s blinding was maintained during the study. We considered subjects positive to CCSVI when at least two criteria were fulfilled.
Results: CCSVI frequency was comparable between POMS and controls (p > 0.05). Clinical features were not significantly different between CCSVI-positive and CCSVI-negative patients.
Conclusions: Our findings add to previous data pointing against a causative role of CCSVI in MS.
MP Amato1, V Saia2, B Hakiki1, M Giannini1, L Pastò1, S Zecchino2, S Lori2, E Portaccio1, M Marinoni2
1Department of Neurology, University of Florence, Florence, Italy.
2Department of Neurological Sciences, University of Florence, Florence, Italy.
Source: Multiple Sclerosis Journal Copyright © 2012 by SAGE Publications (25/04/12)
SBU to advance research and clinical care at national pediatric MS center
Stony Brook University has received a $2.5 million gift from Robert and Lisa Lourie to advance research and clinical care at the National Pediatric Multiple Sclerosis (MS) Center at Stony Brook Long Island Children's Hospital and to establish a new state-of-the-art imaging center at Stony Brook Medicine.
The gift will be matched by the Simons Foundation Challenge Grant, providing a total impact of $5 million. The Pediatric MS Center will be renamed the Lourie Center for Pediatric Multiple Sclerosis.
Multiple Sclerosis historically has been viewed as an adult-onset disease. But according to the National MS Society, approximately eight-to-ten thousand children in the nation have MS. The disease in children can be difficult to diagnose. The advent of magnetic resonance imaging (MRI) and other ways to detect MS lesions have helped to secure the MS diagnosis in children. But because little is yet known on impact of the disease in children, neurologists and other MS experts still seek a consensus on MS diagnosis and treatment in children.
The Lisa and Robert Lourie Imaging Suite will include new imaging technologies that will help Stony Brook's neuroscientists understand more about the brain and spinal cord in relation to MS, epilepsy, amyotrophic lateral sclerosis (ALS), and other neurological disorders. The first purchase for the Suite will be the Siemens Biograph mMR System, a state-of-the-art positron emission tomography (PET)/MRI scanner. The system enables physicians to simultaneously determine both structure and function of abnormalities throughout the head and body.
"Stony Brook Medicine is rapidly becoming a national leader in academic healthcare and research," says Samuel L. Stanley, Jr., M.D., President, Stony Brook University. "The Lourie's extraordinary gift will enable us to provide the resources and environment needed to support the best researchers, and to reach new heights in pioneering research."
"Robert and Lisa's incredible generosity will help Stony Brook Medicine advance our mission of excellence in so many ways, including both imaging and neurological disorders," says Kenneth Kaushansky, M.D., M.A.C.P., Senior Vice President, Health Sciences, and Dean, Stony Brook University School of Medicine. "The gift will allow our faculty to delve into the origins and markers of this devastating disease, hopefully leading to better diagnoses and potentially, treatments."
"The imaging advances made possible by the PET/MRI scanner could dramatically impact the path of future research," says Lauren Krupp, M.D., Professor of Neurology, Stony Brook University School of Medicine, and Pediatric MS Program Director. Dr. Krupp explains that the use of the PET/MRI presents Stony Brook MS experts with the opportunity to pursue new avenues of research, including being able to measure the effects of MS on brain tissue at the level of individual cell types.
The Lourie's hope is that new basic and clinical MS research at Stony Brook will lead to a cure for pediatric MS. With the multidisciplinary expert team of MS clinicians and researchers at the Center and new imaging capabilities, the team expects to advance diagnostic methods and treatments for children with MS. They also hope to unravel the mechanism by which the disease course may be reversed in pediatric cases, gaining insights that could lead to a cessation of disease progression in children and adults affected by MS.
"There are a lot of known, valuable uses for this technology," says Robert Lourie, Ph.D., a physicist. "But it is new enough that I wouldn't be the least bit surprised if the most significant things that come out of it haven't been thought of yet."
While the Lourie's do not have a personal connection to pediatric MS, they both feel the gift represents what they view as a responsibility to give back to important causes after being extremely successful in their careers. Dr. Lourie rose in the field of physics and eventually became a tenured Associate Professor of Physics at the University of Virginia and is now Head of Futures Research at Renaissance Technologies. Lisa Lourie is a nurse who has worked in intensive care units and with AIDS patients.
Lisa says a "feel-good" aspect to this gift is that it incorporates their natural interests. "The research is a marriage of the bioinformatics, which is Robert's end of things, and then the clinical, which is my end of things."
Source: News Medical (19/04/12)
Gray matter pathology in MS: A 3-year longitudinal study in a paediatric population
Summary: This interesting longitudinal imaging study in a paediatric MS population looks at assessing the cortical pathology differences between paediatric onset MS with adult onset MS.
They report that cortical lesion numbers and volume as well as grey matter volume loss were greater in the adult population than paediatric population within 12 months of clinical onset.
However both groups showed a significant increase in cortical lesions over the course of the study and analysis of the data suggests that the degree of grey matter atrophy correlate positively with cortical lesion count.
Abstract
BACKGROUND AND PURPOSE:
GM pathology is considered a major determinant of disability in MS, but the comprehension of its origin and progression rate is limited by the uncertainty of dating the biologic disease onset. Thus, we planned a longitudinal study aimed at analyzing and comparing cortical pathology in pediatric and adult MS patients at clinical onset.
MATERIALS AND METHODS:
Within 12 months from clinical onset, 35 patients with cMS and 57 with aMS were included in a longitudinal study. At T0, GMf and CL number and volume were analyzed. Percentages of ΔGMf and number of new CLs were assessed every year for 3 years (T1-T3). Twenty-eight age- and sex-matched NCs constituted the reference population.
RESULTS:
At T0, GMf did not differ between cMS and NC (P = .18), while it was lower in patients with aMS compared with both NCs (P < .001) and patients with cMS (P < .001). The number of patients with CLs, as well as CL number and volume, were higher in patients with aMS than in those with cMS (P < .001). At T3, ΔGMf was higher in both patients with cMS (1.6% ± 0.5%; range 0.7%-3.4%; P < .001) and aMS (1.6% ± 0.6%; range 0.6%-3.4%; P < .001) compared with NCs (0.7% ± 0.2%; range 0.4%-1.1%), whereas no difference was observed between patients with cMS and aMS (P = .93). ΔGMf significantly correlated with increased CL volume (cMS: r = 0.46; aMS: r = 0.48) and with the appearance of new CLs (cMS: r = 0.51; aMS: r = 0.49).
CONCLUSIONS:
Our findings suggest that focal (CLs) and diffuse (atrophy) GM damage are strictly associated with the biologic onset of MS, and proceed linearly and partly independently of WM pathology.
Authors: Calabrese M, Seppi D, Romualdi C, Rinaldi F, Alessio S, Perini P, Gallo P.
Source: AJNR Am J Neuroradiol. 2012 Mar 15 & Pubmed PMID: 22422186 (21/03/12)
