Biološka terapija i drugi noviji pristupi liječenju kronične upalne bolesti crijeva u djece

master's thesis

• Title (english): Biological therapy and other new therapeutic approaches to inflammatory bowel disease treatment in children
• Author: Matea Klanac
• Mentor: Irena Senečić-Čala (mentor)
• Committee member: Duška Tješić-Drinković (predsjednik povjerenstva)
• Committee member: Marija Jelušić (član povjerenstva)
• Committee member: Irena Senečić-Čala (član povjerenstva)
• Granter: University of Zagreb; School of Medicine; (Department of Pediatrics); Zagreb
• Defense date and country: 2018-07-13, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Pediatrics
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Internal Medicine

Abstract

Kronična upalna bolest crijeva zauzima značajno mjesto u bolestima gastrointestinalnog sustava u djetinjstvu i adolescenciji, a uključuje Crohnovu bolest, ulcerozni kolitis i nerazvrstani kolitis. U otprilike 25% pacijenata s kroničnom upalnom bolesti crijeva, bolest se dijagnosticira prije navršene osamnaeste godine života. Etiologija bolesti je nepoznata, ali se smatra da interakcija okolišnih i genetskih faktora s imunološkim sustavom rezultira neadekvatnim imunološkim odgovorom, s posljedičnom upalom crijevne sluznice. Cilj liječenja je inducirati remisiju bolesti i postići mukozno cijeljenje te svesti nuspojave lijekova na najmanju moguću mjeru. Isključiva enteralna prehrana je učinkovita terapija za indukciju remisije u djece s Crohnovom bolesti i trebala bi biti prva terapijska linija u bolesnika s blažom ili umjereno teškom, nekompliciranom bolesti. Iskorak u liječenju kronične upalne bolesti crijeva napravila je biološka terapija te u novije vrijeme i bioslični lijekovi sa sličnim profilom sigurnosti i učinkovitosti. Biološki lijekovi mogu modificirati tijek bolesti te zaustaviti njezinu progresiju inhibicijom upalne kaskade koja se nalazi u podlozi kronične upalne bolesti crijeva. Pokazali su se učinkovitim u liječenju težih oblika bolesti i bolesti refraktornoj na konvencionalnu terapiju. Noviji pristupi liječenju kronične upalne bolesti crijeva su transplantacija fekalne mikrobiote i antibiotska terapija. Ovi terapijski pristupi mogu utjecati na tijek bolesti promjenom sadržaja crijevnog mikrobioma. Također, noviji pristup je primjena talidomida i transplantacija matičnih stanica. Noviji načini liječenja su se pokazali učinkoviti u indukciji remisije, ali su potrebne dodatne studije za evaluaciju njihove sigurnosti i dugotrajnije učinkovitosti u pedijatrijskoj populaciji.

Abstract (english)

Inflammatory bowel disease occupies a significant place among gastrointestinal diseases in childhood and adolescence and includes Crohn’s disease, ulcerative colitis, and undetermined colitis. About 25% of inflammatory bowel disease patients are diagnosed before the age of 18. The etiology of the disease is unknown, but it is considered that interaction of environmental and genetic factors with the immune system, results in an dysregulated immune response responsible for chronic intestinal inflammation. The treatment goal is to induce clinical remission, to achieve mucosal healing while minimizing the drug’s side effects. Exclusive enteral nutrition is an effective treatment for induction of remission in pediatric Crohn’s disease and should be the first-line treatment in patients with mild and moderate, uncomplicated disease. A step forward in inflammatory disease treatment was achieved with biological therapy and recently with biosimilars that have shown similar safety and efficacy profiles. Biological drugs can alter the disease course and limit its progression through inhibition of the inflammatory process underlying inflammatory bowel disease and are useful in treatment of patients with severe disease or with disease refractory to conventional therapy. New treatment modalities are fecal microbiota transplantation and antibiotic therapy. They may influence the course of the disease by altering the composition of intestinal microbiota. Also new therapeutic approaches are use of thalidomide and stem cell transplantation. New treatment modalities have been effective in remission induction but additional studies to determine their long term efficacy and safety in pediatric population are needed.

• Keywords: kronična upalna bolest crijeva; djeca; biološka terapija; bioslični lijekovi; talidomid; isključiva enteralna prehrana; transplantacija fekalne mikrobiote; antibiotici; transplantacija matičnih stanica
• Keywords (english): inflammatory bowel disease; children; biological therapy; biosimilar agents; thalidomide; exclusive enteral nutrition; fecal microbiota transplantation; antibiotics; stem cell transplantation
• Language: croatian
• URN:NBN: urn:nbn:hr:105:958079
• Study programme: Title: Medicine; Study programme type: university; Study level: integrated undergraduate and graduate; Academic / professional title: doktor/doktorica medicine (doktor/doktorica medicine)
• Type of resource: Text
• File origin: Born digital
• Access conditions: Open access
• Created on: 2019-01-09 11:12:24