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Autologe hämatopoetische Stammzelltransplantation bei systemischer Sklerose

Positionspapier des Arbeitskreises Stammzelltherapie der Deutschen Gesellschaft für Rheumatologie

Autologous hematopoietic stem cell transplantation for systemic sclerosis

Position statement of the stem cell therapy working party of the German Society of Rheumatology

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Zusammenfassung

Drei randomisierte kontrollierte Studien zur autologen hämatopoetischen Stammzelltransplantation (AHSZT) demonstrierten bei systemischer Sklerose (SSc) eine signifikante Überlegenheit hinsichtlich Überleben, Verbesserung von Hautfibrose, Lungenfunktion und Lebensqualität im Vergleich zur Standardtherapie. Die Vorteile müssen jedoch sorgfältig gegen die transplantationsassoziierten Risiken abgewogen werden. Daher hat eine Expertengruppe aus der Arbeitsgruppe „Stammzelltherapie“ der Deutschen Gesellschaft für Rheumatologie (DGRh) nun Empfehlungen zum Einsatz der AHSZT bei SSc erarbeitet. Auf Grundlage der hohen Evidenz wird die AHSZT als „Standardoption“ zur Behandlung ausgewählter SSc-Patienten bewertet. Als Kandidaten für eine AHSZT gelten Patienten mit frühzeitiger, schnell progredienter, diffus kutaner SSc mit viszeralen Manifestationen, die noch keine schwerwiegende Schädigung innerer Organe aufweisen. Eine enge Zusammenarbeit zwischen Rheumatologen und Transplantationszentren ist entscheidend für optimierte Patientenselektion und Behandlungsergebnisse.

Abstract

There have been three randomized controlled trials on autologous hematopoietic stem cell transplantation (AHSCT) in systemic sclerosis (SSc) that demonstrated significant superiority with respect to survival, improvement of cutaneous fibrosis, lung function and quality of life compared to standard treatment; however, these advantages must be carefully weighed against the transplantation-related risks. For this reason, an expert group from the stem cell therapy working party of the German Society for Rheumatology (DGRh) has now developed recommendations for the use of AHSCT in SSc. Based on the high-quality evidence, AHSCT is considered as the standard option for the treatment of selected SSc patients. Potential candidates for AHSCT are those with early, rapidly progressive, diffuse cutaneous SSc with visceral manifestations who have not yet developed severe damage to internal organs. A close cooperation between rheumatologists and transplantation centers is crucial for optimizing patient selection and treatment outcomes.

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Author information

Authors and Affiliations

  1. Medizinische Klinik m.S. Rheumatologie und Klinische Immunologie, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Charitéplatz 1, 10117, Berlin, Deutschland

    T. Alexander & F. Hiepe

  2. Deutsches Rheuma-Forschungszentrum (DRFZ) Berlin – ein Institut der Leibniz-Gemeinschaft, Berlin, Deutschland

    T. Alexander & F. Hiepe

  3. Medizinische Klinik (Innere Medizin II) für Hämatologie, Onkologie, klinische Immunologie und Rheumatologie, Universitätsklinikum Tübingen, Tübingen, Deutschland

    J. Henes

  4. Medizinische Klinik 3 – Rheumatologie und Immunologie, Friedrich-Alexander-Universität Erlangen-Nürnberg, Erlangen, Deutschland

    J. H. W. Distler

  5. Medizinische Klinik und Poliklinik II – Rheumatologie und Klinische Immunologie, Universitätsklinikum Würzburg, Würzburg, Deutschland

    M. Schmalzing

  6. Medizinische Klinik 5, Hämatologie, Onkologie und Rheumatologie, Universitätsklinikum Heidelberg, Heidelberg, Deutschland

    N. Blank

  7. III. Medizinische Klinik und Poliklinik (Nephrologie/Rheumatologie mit Sektion Endokrinologie), Universitätsklinikum Hamburg Eppendorf, Hamburg, Deutschland

    I. Kötter

  8. Klinik für Rheumatologie und Immunologie, Klinikum Bad Bramstedt, Bad Bramstedt, Deutschland

    I. Kötter

Authors
  1. T. Alexander
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  2. J. Henes
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  3. J. H. W. Distler
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  4. M. Schmalzing
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  5. N. Blank
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  6. I. Kötter
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  7. F. Hiepe
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Correspondence to T. Alexander.

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Interessenkonflikt

T. Alexander, J. Henes, J. Distler, M. Schmalzing, N. Blank, I. Kötter und F. Hiepe geben an, dass kein Interessenkonflikt besteht.

Für diesen Beitrag wurden von den Autoren keine Studien an Menschen oder Tieren durchgeführt. Für die aufgeführten Studien gelten die jeweils dort angegebenen ethischen Richtlinien.

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T. Alexander, Berlin

G.-R. Burmester, Berlin

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Alexander, T., Henes, J., Distler, J.H.W. et al. Autologe hämatopoetische Stammzelltransplantation bei systemischer Sklerose. Z Rheumatol 79, 429–436 (2020). https://doi.org/10.1007/s00393-020-00789-0

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  • DOI: https://doi.org/10.1007/s00393-020-00789-0

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