Supervivència i retirada dels fàrmacs modificadors de malaltia en pacients amb artitis idiopàtica juvenil

  1. CASTILLO VILELLA, MIREIA
Supervised by:
  1. María del Consuelo Modesto Caballero Director
  2. Nuria Giménez Gómez Co-director

Defence university: Universitat Autònoma de Barcelona

Fecha de defensa: 22 March 2021

Committee:
  1. Juan Antonio Garcia Vicente Chair
  2. Georgina Salvador Alarcón Secretary
  3. Berta Magallares López Committee member

Type: Thesis

Teseo: 754749 DIALNET lock_openTDX editor

Abstract

Juvenile idiopathic arthritis (JIA) is the most common chronic pediatric rheumatic disease, being an important cause of short and long term disability and significantly affecting quality of life. In those patients who do not respond to the initial conventional treatment (NSAIDs and / or glucocorticoids), Disease Modifying Drugs (DMARDs) are used, which are divided into synthetic and biological. Within the conventional synthetic DMARDs, Methotrexate is the drug of first choice due to its efficacy and safety, widely demonstrated. Biological DMARDs are used in patients who do not respond to synthetic DMARDs. These treatments are often used in the early stages of the disease and are an effective therapy for both arthritis and extra-articular manifestations. It is undeniable that the introduction of these drugs has changed the short and long term prognosis of patients with JIA. However, the duration of treatment may be reduced due to the appearance of intolerance or adverse events. The objectives of this research work are: to analyze the survival or life time of the Disease Modifying Drugs administered in a cohort of patients with Juvenile Idiopathic Arthritis, to describe the different reasons for their withdrawal, and to establish the predictive factors related to these. Additionally, it aims to identify the factors related to the safety of these drugs, to analyze the frequency of clinical remission of the disease, of relapse after treatment withdrawal and the predictive factors related to these. Those patients diagnosed with JIA who had received treatment with synthetic and / or biological Disease Modifying Drugs, who were under follow-up in the Pediatric Rheumatology Unit of a tertiary Hospital, a reference center in Catalonia for the study and treatment of patients with JIA, have been included in this ambispective study. The order of administration of the drug is the most determining factor in the survival of DMARDs. An early therapeutic approach in order to achieve clinical remission of the disease conditions a shorter duration of treatment with both synthetic and biological DMARDs. MTX is the DMARD of choice in patients with JIA, especially in female patients with a small number of affected joints and positive ANA. Its survival during the first two years of treatment is limited by the appearance of unwanted effects in approximately half of the cases, especially when it is administered first, the patient has associated comorbidities and when it is administered in combination with biological DMARDs. Two thirds of patients require continued treatment with DMARDs, even after reaching clinical remission, mainly those who have required treatment with biological DMARDs to control disease activity. In these patients, maintaining a progressively tapered dose of DMARDs for at least one year before considering their withdrawal could be a good therapeutic approach to avoid a subsequent relapse.