© Borgis - Postępy Nauk Medycznych 3/2014, s. 162-165
*Edyta Szymańska1, Maciej Dądalski2, Grzegorz Oracz2, Jarosław Kierkuś2
Charakterystyka pacjentów pediatrycznych z chorobą Crohna kwalifikowanych do leczenia biologicznego
Cohort profile: pediatric patients with Crohn’s disease qualified to biologic therapy
1Department of Pediatrics, Nutrition and Metabolic Disorders, Children’s Memorial Health Institute, Warszawa
Head of Department: prof. Janusz Książyk, MD, PhD
2Department of Gastroenterology, Hepatology and Feeding Disorders, Children’s Memorial Health Institute, Warszawa
Head of Department: prof. Józef Ryżko, MD, PhD
Streszczenie
Wstęp. Współczesne stosowanie nowoczesnych terapii jest problemem ekonomicznym w każdym kraju. Dlatego też dostępność leków biologicznych w Polsce jest możliwa dzięki programom terapeutycznym prowadzonym przez Narodowy Fundusz Zdrowia (NFZ). Aktualnie w programach terapeutycznych NFZ mieści się terapia infliximabem dla pacjentów pediatrycznych (6-18 rok życia) z ciężką postacią choroby Crohna (CD).
Cel pracy. Celem pracy było scharakteryzowanie pacjentów Kliniki Gastroenterologii, Hepatologii i Zaburzeń Odżywiania IP-CZD kwalifikowanych do leczenia infliximabem (IFX) lub/i adalimumabem (ADA).
Materiał i metody. Przeprowadzono retrospektywną analizę 107 dzieci w wieku 13,0 ± 9,3 lat ze zdiagnozowaną CD i leczonych IFX i/lub ADA na przestrzeni 8 lat, w latach 2005-2013. Analizowane informacje obejmowały dane demograficzne, takie jak wiek, płeć, wiek w czasie diagnozy oraz aktywność kliniczną i postać choroby u pacjentów leczonych preparatami biologicznymi.
Wyniki. Przeanalizowano 107 pacjentów z CD (Ch: 54, Dz: 53) w wieku 13,0 ± 9,3. Osiemdziesięciu jeden przyjmujących IFX (75,7%), 26 (24,3%) ADA i 8 (7,5%) oba leki. Najczęstszą lokalizacją była L3 (56,1%). Objawy pozajelitowe odnotowano u 18 pacjentów (16,8%), przy czym najczęstszym z nich było zapalenie/bóle stawów (77,8%). Najczęściej obserwowanymi komplikacjami były zaburzenia wzrastania i niedożywienie u 10 chorych z CD (9,3%). Średni wynik w skali PCDAI przy kwalifikacji wynosił (mediana±] 52,5 ± 27,5. Średni wynik w skali SES-SD przy kwalifikacji wynosił 18 (1,0-22,0).
Wnioski. Pacjenci pediatryczni kwalifikowani do leczenia biologicznego prezentowali raczej ciężki niż umiarkowanie ciężki przebieg choroby z wysoką punktacją w skali PCDAI.
Summary
Introduction. Treatment with modern therapies is an economic problem in every country. Therefore, there are therapeutic programs of National Health Fund (NHF) in Poland which enable to apply such expensive treatment. Currently, Polish NHF programs include biologic therapy with infliximab (IFX) for pediatric patients aged 6-18 years with severe Crohn’s disease (CD).
Aim. The aim of this study was to describe the clinical profile of pediatric patients hospitalized in the Department of Gastroenterology, Hepatology and Feeding Disorders, Children’s Memorial Health Institute in Warsaw, who have been qualified to biologic therapy with either infliximab (IFX) or adalimumab (ADA).
Material and methods. We have performed a retrospective analysis of 107 children age 13.0 ± 9.3 years diagnosed with CD and treated with IFX and/or ADA within the period of 8 year; time between 2005 and 2013. The data on patient’s demographics, including age, sex, and age at disease onset as well as on the course and behavior of CD have been collected.
Results. One hundred and seven CD patients (M: 54, F: 53) aged 13.0 ± 9.3 years were analyzed. Eighty one children (75.7%) received IFX, 26 (24.3%) ADA, and 8 (7.5%) were treated with both agents. Mean disease duration was 5.5 ± 0.83 years. The most frequently found location of lesions was L3 (56.1%). Extraintestinal manifestations were reported in 18 patients (16.8%), and arthralgia/arthritis was the most frequently found condition among them (77.8%). The most frequently found complication were nutritional and growth disorders, observed in 10 patients (9.3%). Mean PCDAI score at qualification was (median± ) 52.5]±27.5. Mean SES-SD (median [interquartile range]) score at qualification was 1 (1.0-22.0).
Conclusions. Pediatric patients qualified to biologic therapy have rather severe than moderate course of disease with high PCDAI score.
Introduction
Treatment with modern therapies is an economic problem in every country. According to statistics, an average cost of biologic therapy with anti-TNF-α agents for patients with rheumatoid arthritis is estimated at approximately 45 000 to 60 000 PLN (1). Therefore, there are therapeutic programs of National Health Fund (NHF) in Poland which enable to apply such expensive treatment. However, the NHF registration procedure is very complicated and takes long. Currently, Polish NHF programs include biologic therapy with infliximab (IFX) for pediatric patients aged 6-18 years with severe Crohn’s disease (CD). Unfortunately, the NHF qualification – requirements for pediatric patients under 18 years are very strict. To fulfill them, small patient has to score at least 51 points according to Pediatric Crohn’s Disease Activity Index (PCDAI). In practice, this means that a child have to be in a very poor condition to have a refunded treatment. Thus, efficacy of therapy is worse and it is difficult to achieve satisfactory outcomes in a short time. Whereas, due to chronic, long-term course of disease and its destructive impact on both patient’s constitution and mentality, only early treatment can bring expected results (2, 3). That is why, both experts and practitioners consider the NHF program economically groundless and unjust regarding the patients.
Aim
The aim of this study was to describe the clinical profile of pediatric patients hospitalized at the Department of Gastroenterology, Hepatology and Feeding Disorders, Children’s Memorial Health Institute in Warsaw, who have been qualified to biologic therapy with either IFX and/or ADA within the period of 8 years; between 2005 and 2013.
Material and methods
We have performed a retrospective analysis of patients diagnosed with CD and treated with biologic therapy with either IFX and/or ADA at the Department of Gastroenterology, Hepatology and Feeding Disorders, Children’s Memorial Health Institute. The analysis included period of 8 years – between 2005 and 2013. The diagnosis of CD was based on Porto criteria (4). The localization of lesions was described using Paris classification (5) as following: L1 – disease limited to lower small intestine with or without caecum involvement, L2 – any exclusively colonic location between caecum and rectum, L3 – disease of the terminal ileum and any location in colon, and L4a – proximal to ligament of Treitz, and L4b – ligament of Treitz to above distal ileum. The endoscopic features of CD were described using simple endoscopic scoring system for CD (SES-CD) which is based on the score of 0-3 of the following four endoscopic variables: ulcer size, ulcerated and affected surfaces, and stenosis determined in five ileocolonic segments (6).
The data have been collected on the base of both electronic and paper case reports. The database included following informations: age, gender, date of diagnosis, time delay between CD related symptoms and establishing of diagnosis, phenotype and disease location (according to Paris classification), disease clinical course (according to PCDAI), endoscopic features of CD (according to SES-CD), extraintestinal manifestation, and disease complications.
Results
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Piśmiennictwo
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