A higher PGA score at time of transfer correlated with lower scores in four different items of patient satisfaction. Kinderarztpraxis Ingelheim am Rhein - Kinderrheumatologie - Neonatologie can be contacted at 4961328998444. Ann Rheum Dis. PROjekte zur Klassifikation, Überwachung und Therapie in der KINDerrheumatologie - Projects for the classification, monitoring and therapy in pediatric rheumatology (PRO-KIND). Nat Rev Rheumatol. 2013;15(1):R33. c Treat-to-target PRO-KIND Consensus Treatment Plan (CTP) for patients with Mevalonate Kinase Deficiency Syndrome (MKD). Hinweise zum Klinikaufenthalt und Beratungsstellen. The TTP also emphasizes the importance of physical activity for the well-being of patients with rheumatic diseases found in multiple studies [24,25,26]. Real-life effectiveness of canakinumab in cryopyrin-associated periodic syndrome. While four of them cited lack of disease activity as the reason and for two other participants it was only a temporary interruption, two participants stated that they had not found a new rheumatologist after moving to another town. Additional suggestions were made for particularly severe disease. SHARE investigators synthesized the published evidence and developed management recommendations. Only two participants reported problems with referral that led to the discontinuation of rheumatological care (Fig. After 3 months, patients are evaluated for achieving the target. statement and The schedule of administration of canakinumab in cryopyrin associated periodic syndrome is driven by the phenotype severity rather than the age. Supplement 2: Significant differences between participants concerning continuity of care. The most common diagnosis was JIA (70.6%), followed by SCTD (9.4%) and autoinflammatory diseases (7.1%). There are several limitations to the study. Goldbach-Mansky R, Kastner DL. 2019;78(1):91–101. Logg Dich ein, um alle Einträge zu sehen. In contrast, the items “how participants individual questions were addressed”, disease-knowledge and self-management were rated above average. Bitte beachten Sie, dass Sie für die Vorstellung in unserer Ambulanz eine Überweisung Ihres Kinderarztes benötigen. Development and preliminary validation of the ‘mind the gap’ scale to assess satisfaction with transitional health care among adolescents with juvenile idiopathic arthritis. 2017;56:1484–91. Robert Koch-Institut, Epidemiologie und Gesundheitsberichterstattung; 2013. Projects for the classification, monitoring and therapy in pediatric rheumatology (PRO-KIND) CAPS/TRAPS /HIDS/MKD working group: Evidence-based statement and consensus treatment plan (CTP) development. Arch Phys Med Rehabil. This is supported by the strong correlation between a lower PGA at the time of transfer and a higher long-term HR-QoL. 2011;13(6):R202. J Adolesc Health. The majority of the study cohort reported no difficulties in each category of the EQ-5D-5L while three out of four participants indicated no more than slight problems in any category. 2015;135(1):e211–5. HR-QoL of adult patients after successful transfer to adult rheumatology is reduced compared to the general population but physical activity and achievement of clinical remission could help to prevent negative long-term outcomes. Hearing loss in Muckle-Wells syndrome. Med Care. Foster HE, Marshall N, Myers A, Dunkley P, Griffiths ID. All further demographic and clinical characteristics were extracted from the questionnaires and retrospectively at the time of transfer from the clinical documentation system ARDIS 2.0 (Arthritis and Rheumatology Documentation and Information System; axaris - software & systeme GmbH). There were only minimal to moderate difficulties in the individual categories of the EQ-5D-5L. Protocols on classification, monitoring and therapy in children's rheumatology (PRO-KIND): results of the working group Polyarticular juvenile idiopathic arthritis. Modified recommendations were drafted and iteratively discussed, adjusted and vetted in regular telephone conferences. Jensen PT, Karnes J, Jones K, Lehman A, Rennebohm R, Higgins GC, et al. Google Scholar. Cham: Springer; 2020. Devlin N, D. P, B. J. The phenotype of TNF receptor-associated autoinflammatory syndrome (TRAPS) at presentation: a series of 158 cases from the Eurofever/EUROTRAPS international registry. b Mean scores of the EQ-VAS (0–100) overall and divided by the age groups represented in our study cohort. German instruments of appropriate length were still being developed and validated while this study was conducted [33]. Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Clin Exp Rheumatol. http://www.cebm.net/oxford-centre-evidence-based-medicine-levels-evidence-march-2009/: https://infevers.umai-montpellier.fr/web/, http://creativecommons.org/licenses/by/4.0/, http://creativecommons.org/publicdomain/zero/1.0/. At the end of each visit, parents are attending and included in the discussion about further treatment. 2011;7(5):290–5. Mutations in the autoinflammatory cryopyrin-associated periodic syndrome gene: epidemiological study and lessons from eight years of genetic analysis in France. The health-related quality of life (HR-QoL) was assessed by the EQ-5D-5L, a validated instrument measuring the HR-QoL in five dimensions on a five-level Likert scale with equal spacing: mobility, self-care, daily activities, pain/discomfort, and anxiety/depression (1: none, 2: slight, 3: moderate, 4: severe, 5: extreme problems/unable to accomplish) [14]. 2009;61(5):658–66. PD Dr. Christoph Deuter Oberarzt. 2018;16(1):40. However, in clinical practice this approach is utilized in Germany and had to be included as one of the treatment arms in order to allow for inclusion of all patients into CTP-guided care. Werdegang. However, this study did not find significant effects on HR-QoL. While Familiar Mediterranean Fever (FMF) can be found with a high prevalence of 1/1000 in several Mediterranean populations [4], other AIDs including Cryopyrin-Associated Periodic Syndrome (CAPS) [5, 6], TNF Receptor-Associated Periodic Syndrome (TRAPS) [7] and Mevalonate Kinase Deficiency Syndrome (MKD)/ Hyper-IgD Syndrome (HIDS) [8] are rare and have an incidence of less than 1/1.000.000 [9]. Kinderheilkunde I - Allgemeine Pädiatrie, Hämatologie und Onkologie . Prior M, McManus M, White P, Davidson L. Measuring the "triple aim" in transition care: a systematic review. Similarly, patients with severe clinical phenotypes, those with frequent inflammatory episodes or those with organ damage at initial presentation are at high risk for adverse outcome. N Engl J Med. Symptomatic therapies with non-steroidal anti-inflammatory drugs (NSAIDS) or corticosteroids may be effective in some patients. a Treat-to-target PRO-KIND Consensus Treatment Plan (CTP) for patients with Cryopyrin-Associated Periodic Syndrome (CAPS)*1 Most patients with CAPS have a continuous disease course, on-demand therapy is only recommended in patients with low disease activity. More time passed since transfer was associated with lower satisfaction with preparation for transfer (Table 3c). 2011;70(10):1704–12. Minden K. Adult outcomes of patients with juvenile idiopathic arthritis. However, the effective implementation of evidence-based treatment recommendations into clinical practice mandates development of consensus protocols. Provided by the Springer Nature SharedIt content-sharing initiative. 2010;62(2):219–25. 2015;174(6):707–14. Standardized monitoring of disease activity and subsequent dose or strategy adjustments are developed (Fig. Treatment decisions include continuation, dose adjustments, medication changes, or strategy change. 2016;11(4):e0153267. Kuemmerle-Deschner JB, Hofer F, Endres T, Kortus-Goetze B, Blank N, Weissbarth-Riedel E, et al. Disease activity and damage should be determined using validated tools. J Happiness Stud. Klin Padiatr. Google Scholar. When drop-out due to remission is disregarded, a high follow-up rate as a result of successful transition can be observed and is an affirmation of the notion to individualise the timing of transfer. PRO-KIND overarching consensus statements for the management of CAPS/TRAPS/HIDS/MKD*. 2014;55(6):796–803. After establishing the specific diagnosis, the individual genotype, phenotype and disease activity should be determined. 2017;31(4):596–609. Lachmann HJ. Overall, respondents were slightly younger, and subgroups of JIA were more common as well as treatment with biologicals. Bulua AC, Mogul DB, Aksentijevich I, Singh H, He DY, Muenz LR, et al. Kinderheilkunde I - Allgemeine Pädiatrie, Hämatologie und Onkologie. Stratified treatment approaches are increasingly made available for childhood rheumatic diseases. Continuity of care. PubMed  Bis heute 9 Jahre und 7 Monate, seit Nov. 2013. NSAIDs or corticosteroids should be used as add-on therapy in CAPS for symptomatic therapy only [39]. Treatment of patients with MKD/HIDS should follow a treat-to-target approach to find individual treatment strategy, medication and doses (Fig. Short form to Abbreviate Biologika In Der Kinderrheumatologie. The authors declare that they have no competing interests. Evidence levels were adapted from the Oxford Centre for Evidence-based Medicine levels of evidence and grades of recommendation [24]: 1A, Systematic reviews of randomized controlled trials; 1B, individual randomised controlled trial; 2A, systematic review of cohort studies; 2B, individual cohort study; 3B, individual case-control study, non-consecutive cohort study; 4, case series; 5, expert opinions. SH and LSB conceptualized and designed the study, performed the measurements, designed the data collection instruments, collected data, carried out the initial analyses, drafted the initial manuscript, and reviewed and revised the manuscript. Front Immunol. Cookies policy. S.H. Adresse: Patients were included if they were diagnosed with a rheumatic or autoinflammatory disease, were treated in the Inflammation Center for at least 2 years and during that time attended at least two visits in the TTP, one visit at the age of 17 or 18 years. The engagement and support of the German paediatric rheumatology community was critical to develop the statements, algorithms and CTPs. Overall satisfaction with transition, respectively transition clinics, was slightly higher than in comparable studies [18, 34]. Legend: Listed are the significant differences in patient satisfaction between participants that did experience some form of discontinuation of care, including change in diagnosis, medical therapy or discontinuation in general. The results give the idea that physical activity and reaching clinical remission could help to prevent negative long-term outcomes after transfer. Efficacy and safety of canakinumab therapy in paediatric patients with cryopyrin-associated periodic syndrome: a single-Centre, real-world experience. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work. Similarly, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) CTPs for juvenile dermatomyositis stratify approaches based on subtype and disease activity [51, 52]. Rheumatology. PubMed  Rheumatology. The impact of AID on individual patients varies widely; close monitoring of disease activity in the individual patient is therefore needed. G.H. Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, Leslie KS, Hachulla E, Quartier P, et al. Specific patient groups such as those with a severe phenotype will likely require higher doses to achieve remission [17]. Publicly available, evidence-based guidance statements and CTPs can decrease variation in care, in particular in rare rheumatic diseases as individual physician preferences may not weigh as heavy as in more common diseases. In individual cases, response to treatment with TNF-α or IL-6 inhibitors has been reported [30, 43]. Universitätsaugenklinik Schleichstraße 12 72076 Tübingen. Legend: a) The figure shows the number of participants who reported each of the listed frequencies of physical activity (n = 84 due to one missing data). Methods: In May 2020 all GKJR members were invited to take part in an online survey. Walter et al. Jaeger VK, Hoffman HM, van der Poll T, Tilson H, Seibert J, Speziale A, et al. PubMed Central  All rights reserved. This has facilitated impactful treat-to-target strategies resulting in better outcomes [55]. In severe cases and poor quality of life, allogeneic haematopoietic stem cell transplantation (HSCT) may be considered [48]. Ihre Haus- & Fachärzte von der Geburt bis zum vollendeten 18. The PRO-KIND CAPS/TRAPS/ MKD/HIDS expert working group adjusted these in iterative reviews. Springer Nature. 70.6% were diagnosed with juvenile idiopathic arthritis (JIA). Autoinflammatorische syndrome und amyloid-A-Amyloidose. 2014;23(2):443–7. Arthritis Rheum. frontend.sr-only_#{element.contextual_1.children.icon}: Rheumazentrum Rheumazentrum Indira / Transition, Notfallpraxen und ärztlicher Bereitschaftsdienst, Leitung und Verwaltung der Medizinischen Fakultät, Klinikum und Medizinische Fakultät als Arbeitgeber, Kontakt für Ärzte, Ärztinnen und medizinisches Personal, Kontakt für Patientinnen, Patienten, Angehörige und Spendeninteressierte, Rheumatische Gelenksentzündungen (juvenile idiopathische Arthritis), Sklerodermie und Morphea (lokalisierte Sklerodermie), Gefäßentzündungen (Vaskulitiden) wie Morbus Behcet,, Takayasu Arteriitis, Panarteriitis nodosa, Autologe Stammzelltherapie bei Autoimmunerkrankungen. In addition, inflammatory markers and the physician and patient/parent global assessments (PGA, PPGA) should be determined serially. Z Rheumatol. 1a). 2009;360(23):2416–25. Additionally, and in close cooperation with the psycho-social team, the focus of each visit is on promoting self-management, knowledge about the patient’s own disease and therapy and reflection of the individual progress, as well as on topics that are particularly relevant to adolescents. Juvenile idiopathic arthritis. As assumed in advance, responsiveness decreased with time passed since patients were transferred resulting in significantly lower age in participants compared to the non-responsive cohort. Front Immunol. 07071 29-25145, E-Mail-Adresse: Almost a third each received basic antirheumatic therapy (28.2%) and biologicals including Janus kinase (JAK) inhibitors (32.9%). Aufgrund der langjährigen Ausbildung zum Kinderrheumatologen (Universitätsklinik Tübingen, Deutschland) und der Sprechstundenerfahrung in einem grossen Referenzzentrum für autoinflammatorische Erkrankungen, stehen wir Ihnen in diesem Spezialgebiet für Diagnostik, Beratung, Zweitmeinungen und Therapie zur Verfügung. et al. Patient satisfaction is an important indicator for effective high-quality transition [30] and the most commonly studied issue, although validated instruments in all languages remain lacking [31, 32]. In addition, female gender, higher disease activity at time of transfer as measured by the PGA, higher frequency of visits in adult care after transfer and lower frequency of physical activity were associated with significantly lower HR-QoL (Table 3b). Ausführliche Informationen zum Aufenthalt in unserer Klinik finden Sie auf der Seite Klinikaufenthalt. Reasons for hospitalisations relevant to this study were repeated inflammatory episodes, joint (knee and shoulder) and ophthalmologic complications. Genetics of monogenic autoinflammatory diseases: past successes, future challenges. J Rheumatol. 2010;8:2. Über die Praxis App erhalten Sie auch  Informationen zur Praxis und die Information über anstehende Vorsorgeuntersuchungen und Impfungen. 1) The PRO-KIND CAPS/TRAPS/MKD/HIDS expert working group consisted of only 12 members, which limited the scale and scope of contributing experiences. TRAPS patients may present with prolonged fever episodes of ≥5 days, migratory rash, periorbital edema, myalgia and may have a positive family history [11]. Papa R, Doglio M, Lachmann HJ, Ozen S, Frenkel J, Simon A, et al. Participants with continued care showed a higher mean score on the EQ VAS for current condition, while in average scoring lower in the EQ-5D-5L index for HR-QoL. In addition, questions were asked about the patients’ experiences in adult care. Barth et al. Ringold S, Angeles-Han ST, Beukelman T, Lovell D, Cuello CA, Becker ML, et al. In the TTP, this is handled similarly for patients with more severe courses of disease. Dale R, Budimir S, Probst T, Humer E, Pieh C. Quality of life during the COVID-19 pandemic in Austria. The most common reason for discontinuation was remission, followed by discontinuation at the patient’s request. Ärztinnen und Ärzte, Pfleger und Pflegerinnen, Psychosozialer Dienst, Studienassistenz, Physiotherapeutinnen und -therapeuten, Ergotherapeutinnen und -therapeuten und Lehrkräfte (Klinikschule) arbeiten hier interdisziplinär zusammen, um eine erfolgreiche Behandlung und umfassende . The authors partly attributed these numbers to the habit of transferring patients within the same hospital following the EULAR and PReS recommendations [7]. Rheumatology (Oxford). Respondents indicated a high patient satisfaction, although it decreased after transfer to adult care. Discordant estimates are equally important, as they mandate further evaluations. Orphanet J Rare Dis. These were developed to enable the implementation of evidence-based, standardized care into clinical practice. Pediatr Rheumatol 18, 17 (2020). ter Haar NM, Oswald M, Jeyaratnam J, Anton J, Barron KS, Brogan PA, et al. Pädiatrische Sonographie (2014-2018) 2013;72(5):678–85. 2015;74(9):1636–44. J Clin Rheumatol. The longer version of this instrument unfortunately did not seem fitting for this study since it surveyed topics that do not apply to the process and cohort of the TTP, e.g., working environment. PubMed  07071 29 81386, Faxnummer: Targeted inhibition of specific pro-inflammatory cytokines is commonly required. 2019;71(6):846–63. Seven participants reported that their diagnosis had been changed after transfer to adult care (Fig. https://doi.org/10.1186/s12969-022-00776-6, DOI: https://doi.org/10.1186/s12969-022-00776-6. The integration of the developed framework into clinical practice is the critical next step. The datasets used and/or analysed during the current study are available from the corresponding author on reasonable request. This difference was seen in both the 18–29-year-olds (64.1% vs. 30.2%) and the 30–39-year-olds (50.0% vs. 19.6%) (Fig. Efficacy of etanercept in the tumor necrosis factor receptor-associated periodic syndrome: a prospective, open-label, dose-escalation study. Ann Rheum Dis. 2017;76(6):942–7. Safety of vaccinations in patients with cryopyrin-associated periodic syndromes: a prospective registry based study. Um fortfahren zu können, müssen Sie eine Cookie-Auswahl treffen. Groot N, de Graeff N, Avcin T, Bader-Meunier B, Brogan P, Dolezalova P, et al. In contrast, for systemic lupus erythematosus (SLE), type and extent of major organ involvement such as type of lupus nephritis typically drives treatment selections. Protokolle zur Klassifikation, Überwachung und Therapie in der Kinderrheumatologie (PRO-KIND): Enthesitis-assoziierte Arthritis: Ergebnisse der Arbeitsgruppe Enthesitis-assoziierte Arthritis in. 2013;65(4):1116–21. Higher scores on the EQ VAS and lower EQ-5D-5L index scores indicate better health-related quality of life, lower PGA scores indicate less disease activity. All items, except the two adolescent-specific topics and preparation for transfer, reached a mean score >  7. 07071 29-80894, Faxnummer: 2013;94(11):2093–102. The linkage of AID specific clinical symptoms and raised inflammatory markers provides strong evidence of an AID. Still, it was an intentional decision to include a wider time span to increase the number of participants, especially since response rates in comparable studies are similar to this one. An iterative expert review process discussed, adapted and refined these statements. The approved statements were integrated into three disease specific consensus treatment plans (CTPs). These are compared between the study cohort and the average population. * The PRO-KIND statements were adapted from the SHARE recommendations for the management of autoinflammatory diseases [19]. The first graph shows the absolute numbers of participants with and without continued care after transfer and the reasons given for discontinuation of care (n = 83 due to two missing data). Respondents indicated high patient satisfaction and self-perceived self-management skills and felt well informed. Rheumatology (Oxford). 2009;27:621–68. The national PRO-KIND initiative addresses the urgent need to improve quality of care and outcome of children and adolescents living with rheumatic conditions by standardization and effective implementation of evidence-based therapies and thereby decreasing variation in care [20,21,22]. * marks significant differences between study cohort and average population. Pediatr Rheumatol Online J. 76.5% of participants received regular rheumatologic care after transfer. Email: MIWGUC@kinderrheumatologie.de. Six participants (7.1%) experienced positive effects, while 63.5% (54/85) did not obtain any effects of their disease on social relationships (Table 2b, c). The final documents were submitted to the GKJR Board for approval. Use of canakinumab in the cryopyrin-associated periodic syndrome. A priori, a unified formal PRO-KIND framework and multi-step process for the development of consensus-based protocols was established. The data of this study confirmed the assumption of Hilderson et al. We provide excellent research and teaching aimed at finding solutions to future challenges in a globalized society. König H-H, Bernert S, Angermeyer MC, Matschinger H, Martinez M, Vilagut G, et al. Cite this article. In general, the mean scores ranged from 5.38 to 9.03 (mean: 7.63). General and disease-specific statements were anchored in the 2015 SHARE recommendations. These can be summarized under the headings of individual outcomes, health care outcomes and social outcomes [12]. Correspondence to The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. Statements were integrated into diagnosis- and disease activity specific treat-to-target CTPs for CAPS, TRAPS and MKD/HIDS. Several AID real-life series have focussed on the frequency of achieving remission and the requirement for dose adjustments. Supporting the health care transition from adolescence to adulthood in the medical home. Most importantly, high-level evidence from a randomized controlled trial for the effectiveness of IL-1 inhibition for TRAPS and MKD/HIDS was integrated [27]. Weitere Schwerpunkte sind Allergie und Asthma, Endokrinologie, Gastroenterologie, Mukoviszidose, Organtransplantationen und Rheuma. 2022;23(7):3577–604. 2017;15(1):1. 2018;77(8):677–84. 2015;13:22. To some extent, this is comparable to the results of Hazel et al. In addition typical clinical symptoms for CAPS include urticaria-like rash, cold/stress triggered episodes, sensorineural hearing loss, chronic aseptic meningitis and skeletal abnormalities such as epiphyseal overgrowth and frontal bossing [11]. SH received a grant from the Faculty of Medicine, Tuebingen, Germany, TÜFF Habilitation Support (2620–0-0). However, the distinct data for reasons for discontinuation of care indicated that a large proportion of patients were in stable remission and did not require continuous care. The study cohort was more physically active than the respective average age groups. The evaluation of the CTPs may enable the community to rapid access to evidence based biologic therapies. Stringer E, Scott R, Mosher D, MacNeill I, Huber AM, Ramsey S, et al. The access to high-quality medical treatment for all patients is therefore essentially decisive for the prognosis of the patie … Foster HE, Minden K, Clemente D, Leon L, McDonagh JE, Kamphuis S, et al. Still, the questions asked in this study have been used in different studies [10, 18] and in the ongoing evaluation of patients’ readiness for transfer in the TTP. Annu Rev Immunol. Privacy All authors (SB, JB, GB, SH, DH, GH, AJ, JKD, EL, MP, NR, ARW) made a substantial contribution to study conception and design. The dataset generated and analyzed during this study is not publicly available but is available from the corresponding author on reasonable request after obtaining ethics committee approval. PLoS One. was partially funded through the Margarete Mueller-Bull Stiftung and a research grant of Novartis Pharma. Die Klinik ist international führend bei der Durchführung von Stammzelltransplantationen bei Leukämie, insbesondere die haploidentische Variante, bei der Eltern als Spender eingesetzt werden können. IL-1 inhibition has been shown to effectively control inflammation in patients with CAPS, TRAPS and MKD/HIDS [27, 37, 38]. Thus, it is possible that the non-responsive cohort might have indicated less successful long-term transition outcomes than the responders. J.B. received speaker’s bureaus or consulting fees from AbbVie, Novartis, and Pfizer. 3b). Diagnosing a rare AID mandates the recognition of characteristic clinical findings. The definition of therapeutic targets, the continuous integration of disease activity measurements and the clinical evaluation at defined time points are the basis for the German PRO-KIND CTPs.
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