Artikel
Autologous Hematopoietic Stem Cell Transplantation (HSCT) for Anti-Neutrophil Cytoplasmic Antibody (ANCA)-associated Vasculitis (AAV) – an EBMT retrospective survey
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Autoren
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Tobias Alexander
- Charité – Universitätsmedizin Berlin, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie, Berlin
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Clare Samuelson
- Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
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Thomas Daikeler
- Universitätsspital Basel, Klinik für Rheumatologie, Basel, Schweiz
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Jörg Henes
- Universitätsklinikum Tübingen, Innere Medizin II – Onkologie, Hämatologie, Klinische Immunologie, Rheumatologie und Pulmologie, Tübingen
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Mohamed Akil
- Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
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Lars Skagerlind
- Department of Hematology, University Hospital Umea, Umea, Schweden
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Gerhard Ehninger
- Medizinische Klinik und Poliklinik I, Universitätsklinikum der Technischen Universität Dresden, Dresden
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Jantunen Esa
- Department of Internal Medicine, University Hospital Kuopio, Kuopio, Finnland
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Farge Dominique
- Internal Medicine, St. Louis Hospital, Paris, Frankreich
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Snowden John
- Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
| Veröffentlicht: | 5. Februar 2019 |
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Gliederung
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Background: ANCA-associated vasculitides (AAV) are chronic autoimmune diseases, which can present with life-threatening, multi-system involvement. Despite the use of rituximab and other available modern biologic therapies, some patients with AAV develop severe and refractory courses of disease. Autologous haematopoietic stem cell transplantation (HSCT) has been used in a number of such cases as salvage therapy but their outcomes have not been analysed so far.
Methods: Adults receiving HSCT for AAV and whose data were registered within the EBMT Autoimmune Disease Working Party (ADWP) were identified retrospectively through the EBMT database. Treating physicians were surveyed to produce a retrospective evaluation of outcomes.
Results: 7 patients underwent HSCT primarily for AAV between 1999-2014 in 6 centers across Europe. 5 females and 2 males were transplanted; 6 had a diagnosis of granulomatosis with polyangiitis (GPA) and 1 eosoniphilic granulomatosis with polyangiitis (EGPA). Median age was 39 years (range 32-55 years). Patients had received 4-6 prior lines of therapy, including cyclophosphamide (CY, median cumulative dose of 80g) and steroids in every case, and rituximab in 4 cases. Median time between diagnosis and HSCT was 16 years. Conditioning regimen was CY/ATG in 5 patients and CY only in 2 patients. Median follow-up was 86 months (range 1-204 months). Transplant-related mortality (TRM) occurred in 2 cases. All but one patient went into remission but 3 later relapsed at 6, 12 and 36 months, respectively, and required further treatment for disease control. At time of last clinical follow-up, 3 patients had drug-dependent partial response.
Conclusion: Outcomes of HSCT for these heavily pre-treated AAV patients were variable. Only 1 patient achieved drug-free complete remission and TRM was observed in was observed in 2/7 cases. Nevertheless, HSCT had the potential to stabilize AAV in patients, who initially failed to respond to conventional therapies. These data do not support HSCT for advanced stage AAV, although it may have a place as salvage treatment in otherwise refractory patients. As for other autoimmune diseases, HSCT may provide better outcomes when performed at early stage of disease. Overall, HSCT should only performed in clinical trial settings in experienced centres.
