Development and validation of juvenile autoinflammatory disease multidimensional assessment report (JAIMAR)

Konukbay, D; Yildiz, D; Acikel, C; Sozeri, B; Makay, B; Ayaz, NA; Barut, Kenan; Kisaarslan, A; Bilginer, Y; Peru, H; Erdogan, O; Unsal, E; Kasapçopur, Özgür; Gunduz, Z; Ravelli, Andrea; Koné‐Paut, Isabelle; Frenkel, Joost; Gattorno, Marco; Özen, Seza; Demirkaya, E

doi:10.1186/1546-0096-13-s1-p55

Cited by 2 publications

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“…However, several aspects render them unsuitable for the application in the context of a T2T approval: They (i) are rather oligodimensional, [ 58 ] (ii) do not include the acute phase response or PROMs, [ 57 ] or (iii) capture primarily improvement under treatment [ 57 , 58 , 99 , 100 ]. Other scores focus on the quality of life, chronic sequelae and response to treatment [ 29 , 30 , 101 ].…”

Section: Discussionmentioning

confidence: 99%

Treat-to-target strategies for the management of familial Mediterranean Fever in children

Ehlers,

Rolfes,

Lieber

et al. 2023

Pediatr Rheumatol

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Background The objective of this initiative was to develop a treat-to-target (T2T) approach for the management of patients with Familial Mediterranean Fever (FMF), including the definition of a complex treatment target, and establish strategies that improve patient care and long-term outcome. Methods An initial set of statements as well as a flow chart visualising the proposed concept was developed. To adapt the preliminary statements to the current state of knowledge, a systematic literature search was performed and the modified statements were subject to a Delphi approach. To ensure the applicability of the statements in daily practice, an online survey was conducted among paediatric rheumatologists in Germany. In addition, data from the national AID-NET registry were analysed with respect to therapeutic response. Results This T2T initiative yielded a total of 26 statements guiding FMF management with respect to diagnosis, treatment targets, treatment strategies and monitoring. The online survey identified cut-off values for inflammatory markers indicating treatment intensification and appropriate measures in case of colchicine intolerance or non-adherence. The analysis of data derived from the national AID-NET showed that colchicine therapy was successfully terminated in 61% of patients (27 out of 44) with heterozygous MEFV mutations. Multidimensional treatment targets incorporating objective and subjective reported outcome measures were developed. These provide the basis for stratifying patients into the following treatment paths: continue colchicine, persisting attacks / inflammation, colchicine intolerance, persisting arthritis, colchicine reduction and adjustment/reduction of biologics. Conclusions The proposed consensus treatment plan for the management of FMF incorporates multidimensional targets allowing transparent treatment decisions, which will promote personalised disease management and increase adherence to therapy.

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Section: Discussionmentioning

confidence: 99%

Treat-to-target strategies for the management of familial Mediterranean Fever in children

Ehlers,

Rolfes,

Lieber

et al. 2023

Pediatr Rheumatol

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Defining colchicine resistance/intolerance in patients with familial Mediterranean fever: a modified-Delphi consensus approach

et al. 2021

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Objectives Colchicine is the main treatment for FMF. Although a number of individuals with FMF are intolerant/resistant to colchicine, there is no standard definition of colchicine resistance/intolerance. We developed a set of evidence-based core statements defining colchicine resistance/intolerance in patients with FMF that may serve as a guide for clinicians and health authorities. Methods A set of statements was identified using a modified-Delphi consensus-based approach. The process involved development of an initial colchicine resistance/intolerance-related questionnaire derived from a systematic literature review. The questionnaire, which was completed by an international panel of 11 adult and paediatric rheumatologists with expertise in FMF, was analysed anonymously. The results informed draft consensus statements that were discussed by a round-table expert panel, using a nominal group technique to agree on the selection and wording of the final statements. Results Consensus among the panel was achieved on eight core statements defining colchicine resistance/intolerance in patients with FMF. A definition of resistance was agreed upon that included recurrent clinical attacks (average one or more attacks per month over a 3-month period) or persistent laboratory inflammation in between attacks. Other core statements recognize the importance of assessing treatment adherence, and the impact of active disease and intolerance to colchicine on quality of life. Conclusion Based on expert opinion, a set of evidence-based core statements defining colchicine resistance/intolerance in patients with FMF were identified to help guide clinicians and health authorities in the management of patients with FMF.

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Development and validation of juvenile autoinflammatory disease multidimensional assessment report (JAIMAR)

Cited by 2 publications

References 0 publications

Treat-to-target strategies for the management of familial Mediterranean Fever in children

Treat-to-target strategies for the management of familial Mediterranean Fever in children

Defining colchicine resistance/intolerance in patients with familial Mediterranean fever: a modified-Delphi consensus approach

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