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Arthritis und Rheuma 2021; 41(02): 127-136
DOI: 10.1055/a-1378-5963
Schwerpunkt

Seltene Erkrankungen in der Rheumatologie

In-Label- und evidenzbasierte Off-Label-TherapienRare diseases in rheumatologyIn-label and evidence-based off-label therapies
Jutta Bauhammer∗
1   Rheumatologie Baden-Baden, Tätigkeitsschwerpunkt Klinische Immunologie, am Medical Center Baden-Baden, Baden-Baden
,
Christoph Fiehn∗
1   Rheumatologie Baden-Baden, Tätigkeitsschwerpunkt Klinische Immunologie, am Medical Center Baden-Baden, Baden-Baden
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ZUSAMMENFASSUNG

Aus der Rheumatologie sind seltene entzündliche Erkrankungen nicht wegzudenken. Ihre Therapie stellt den Rheumatologen/die Rheumatologin vor besondere Herausforderungen, da oft keine zugelassenen Medikamente existieren. Um aber im Off-Label-Einsatz zu behandeln, ist es notwendig, die Evidenz in der Behandlung der verschiedenen Erkrankungen gut zu kennen. In der folgenden Arbeit werden die Therapieoptionen sowohl zu In- wie auch Off-Label-Therapie der wichtigsten seltenen rheumatischen Erkrankungen der Erwachsenen dargestellt. Dies sind die systemische Sklerose, die idiopathischen inflammatorischen Myopathien, der Morbus Behçet, das familiäre Mittelmeerfieber, der Morbus Still, die idiopathische rekurrierende Perikarditis, das SAPHO-Syndrom, die chronisch rekurrierende multifokale und chronisch nicht bakterielle Osteomyelitis, die IgG4-Syndrome, die Mischkollagenose (Sharp-Syndrom) sowie die undifferenzierte und Overlap-Kollagenosen. Die klinischen Studien und aktuellen Empfehlungen zur Therapie dieser Erkrankungen, bezogen auch auf die jeweiligen Krankheitsmanifestationen, werden tabellarisch aufgelistet und die Referenzen genannt. Das Ziel des Artikels ist es, dem praktisch tätigen Rheumatologen eine Hilfestellung zur Auswahl der Therapie und unter Umständen auch der Beantragung einer Kostenübernahme für Off-Label-Therapien zu geben.

SUMMARY

Rare inflammatory diseases are common in rheumatology. Their treatment is particularly challenging for rheumatologists as there is rarely any approved medication at hand. For off-label drug use it is, however, necessary to well know the evidence in treatment of the various diseases. The following paper describes the therapeutic options both for in- and off-label therapies for treating the most important rare rheumatic diseases in adults. These are systemic sclerosis, idiopathic inflammatory myopathies, Behçet’s disease, familial Mediterranean fever, Still’s disease, idiopathic recurrent pericarditis, SAPHO-syndrome, chronic recurrent multifocal and chronic non-bacterial osteomyelitis, IgG4-syndrome, mixed connective tissue disease (Sharp’s-syndrome) as well as non-differentiated and overlap-connective tissue diseases. The clinical studies together with recent recommendations on the treatment of these diseases – also in accordance with their respective manifestations – are listed in tabular form; references are indicated. Hereby, the paper aims at practically helping rheumatologists to choose treatment and, possibly, apply for cost reimbursement in case of off-label therapies.

Schlüsselwörter

Seltene Erkrankungen - IL-1-Inhibitoren - Rituximab - Colchicin - TNF-Inhibitoren

Keywords

Rare diseases - IL-1-inhibitors - Rituximab - Colchicin - TNF-inhibitors


Publication History

Article published online:
20 April 2021

© 2021. Thieme. All rights reserved.

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