Home » THALIDOMIDE, A NEW THERAPEUTIC TOOL FOR INFLAMMATORY INTESTINAL DISEASES OF CHILDREN

THALIDOMIDE, A NEW THERAPEUTIC TOOL FOR INFLAMMATORY INTESTINAL DISEASES OF CHILDREN

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New step forward in the fight against Crohn’s disease and ulcerative colitis, thanks to the commitment of the Italian pediatricians of the SIGENP scientific society.

The results of a study, published by the most prestigious international journal of the sector, ‘Inflammatory Bowel Disease’, and coordinated by a group of Italian researchers led by dr. Matteo Bramuzzo, of the IRCCS Burlo Garofolo of Trieste, collected data from 10 years of clinical experience of the treatment of IBD (Inflammatory Bowel Disease), the Inflammatory Gastrointestinal Disease – which includes Crohn’s disease and ulcerative colitis – in eight Italian centres. The pathology increasingly affects the pediatric population in rich countries, in Italy they are affected 7 children for every 100,000 inhabitants. It is responsible for very disabling symptoms, undermines the quality of life of young patients and presents a high risk of having to resort to surgery.

When the affected children are very young, under the age of six, they are called with the acronym VEO-IBD (Very Early Onset Inflammatory Bowel Disease), i.e. very early onset inflammatory bowel disease. “In 37 children with VEO-IBD – explains the coordinator of the study Matthew Bramuzzo – therapy with thalidomide proved to be effective and well tolerated: side effects only in 36% of cases, much lower than what was observed in adolescent patients (77%). It may seem like a small number of cases but, since we are dealing with rare diseases and patients with highly selected characteristics, the size of this population is significant. Even more interesting is the fact that the drug enabled sustained remission of the disease in a significant proportion of children.” The duration of treatment ranged from a minimum of one year to a maximum of 9 years. Unexpectedly, therefore, the ‘old’ thalidomide, considered an outdated and harmful molecule, brings new hope to medicine. “Our society – he underlines the professor. Claudio Romano, president of Sigenp e Director of the Pediatric Gastroenterology and Cystic Fibrosis Unit of the University of Messina – with this study it has made it possible to add a new drug in the treatment of these complex pathologies in children. Furthermore, the relative low cost of this therapy compared to other drugs should be considered. But, beyond the positive results, we must not forget that theindication for the use of thalidomide should be discussed with adequate attention with the doctors of the center where the child is followed.

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“It is not yet the panacea for these diseases – continues Dr. Bramuzzo – but the results observed with our study are among the best ever obtained. In fact, until now this pathology in younger children was treated with the same drugs – biological and otherwise – that are used in adults, with often unsatisfactory results: now, thanks to this study, we have ascertained that thalidomide is certainly a valid therapeutic option in children with very early onset inflammatory bowel disease unresponsive to conventional therapies”

Concludes Prof. Romano “Obviously, this drug also has its limits: for this reason the therapy must be carefully monitored for any adverse events in centers with adequate experience. SIGENP, with its groups also in this area, confirms itself as a company with prolific clinical research activity at an international level in the field of complex diseases of children.”

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