Autologous hematopoietic stem cell transplantation for systemic sclerosis Position statement of the stem cell therapy working party of the German Society of Rheumatology

被引：2

作者：

Alexander, T. ^{[1
,2
,3
,4
,5
]}

Henes, J. ^{[6
]}

Distler, J. H. W. ^{[7
]}

Schmalzing, M. ^{[8
]}

Blank, N. ^{[9
]}

Koetter, I ^{[10
,11
]}

Hiepe, F. ^{[1
,2
,3
,4
,5
]}

机构：

[1] Charite Univ Med Berlin, Med Klin mS Rheumatol & Klin Immunol, Charitepl 1, D-10117 Berlin, Germany

[2] Free Univ Berlin, Charitepl 1, D-10117 Berlin, Germany

[3] Humboldt Univ, Charitepl 1, D-10117 Berlin, Germany

[4] Berlin Inst Hlth, Charitepl 1, D-10117 Berlin, Germany

[5] Deutsch Rheuma Forschungszentrum DRFZ Berlin, Inst Leibniz Gemeinschaft, Berlin, Germany

[6] Univ Klinikum Tubingen, Med Klin Innere Med Hamatol Onkol Klin Immunol &, Tubingen, Germany

[7] Friedrich Alexander Univ Erlangen Nurnberg, Med Klin Rheumatol & Immunol 3, Erlangen, Germany

[8] Univ klinikum Wurzburg, Med Klin & Poliklin Rheumatol & Klin Immunol 2, Wurzburg, Germany

[9] Univ Klinikum Heidelberg, Med Klin Hamatol Onkol & Rheumatol 5, Heidelberg, Germany

[10] Univ Klinikum Hamburg Eppendorf, Med Klin & Poliklin Nephrol Rheumatol 3, Sekt Endokrinol, Hamburg, Germany

[11] Klinikum Bad Bramstedt, Klin Rheumatol & Immunol, Bad Bramstedt, Germany

来源：

ZEITSCHRIFT FUR RHEUMATOLOGIE | 2020年 / 79卷 / 05期

关键词：

Autologous stem cell transplantation; Systemic sclerosis; Cell therapy; Guidelines; DGRh; MARROW TRANSPLANTATION; AUTOIMMUNE-DISEASES; PULSE CYCLOPHOSPHAMIDE; EUROPEAN-SOCIETY; BLOOD; EBMT;

D O I：

10.1007/s00393-020-00789-0

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：429 / 436

页数：8

共 25 条

[1] Predictors of disease worsening defined by progression of organ damage in diffuse systemic sclerosis: a European Scleroderma Trials and Research (EUSTAR) analysis [J].

Becker, Mike ;

Graf, Nicole ;

Sauter, Rafael ;

Allanore, Yannick ;

Curram, John ;

Denton, Christopher P. ;

Khanna, Dinesh ;

Matucci-Cerinic, Marco ;

Pena, Janethe de Oliveira ;

Pope, Janet E. ;

Distler, Oliver ;

Matucci-Cerinic, Marco ;

Guiducci, Serena ;

Walker, Ulrich ;

Jaeger, Veronika ;

Bannert, Bettina ;

Lapadula, Giovanni ;

Becvarare, Radim ;

Cutolo, Maurizio ;

Valentini, Gabriele ;

Siegert, Elise ;

Rednic, Simona ;

Allanore, Yannick ;

Montecucco, C. ;

Carreira, Patricia E. ;

Novak, Srdan ;

Czirjak, Laszlo ;

Varju, Cecilia ;

Chizzolini, Carlo ;

Allai, Daniela ;

Kucharz, Eugene J. ;

Cozzi, Franco ;

Rozman, Blaz ;

Mallia, Carmel ;

Gabrielli, Armando ;

Bancel, Dominique Farge ;

Airo, Paolo ;

Hesselstrand, Roger ;

Martinovic, Duska ;

Balbir-Gurman, Alexandra ;

Braun-Moscovici, Yolanda ;

Hunzelmann, Nicolas ;

Pellerito, Raffaele ;

Caramaschi, Paola ;

Black, Carol ;

Damjanov, Nemanja ;

Henes, Joerg ;

Ortiz Santamaria, Vera ;

Heitmann, Stefan ;

Seidel, Matthias .

ANNALS OF THE RHEUMATIC DISEASES, 2019, 78 (09) :1242-1248

[2] Treatment and course of a man with systemic sclerosis before and after hematopoetic blood stem cell transplantation [J].

Blank, N. ;

Lorenz, H. M. .

DEUTSCHE MEDIZINISCHE WOCHENSCHRIFT, 2013, 138 (37) :1824-1827

[3] Low-dose cyclophosphamide effectively mobilizes peripheral blood stem cells in patients with autoimmune disease [J].

Blank, Norbert ;

Lisenko, Katharina ;

Pavel, Petra ;

Bruckner, Thomas ;

Ho, Anthony D. ;

Wuchter, Patrick .

EUROPEAN JOURNAL OF HAEMATOLOGY, 2016, 97 (01) :78-82

[4] Autologous HSCT is efficacious, but can we make it safer? [J].

Burt, Richard K. ;

Farge, Dominique .

NATURE REVIEWS RHEUMATOLOGY, 2018, 14 (04) :191-192

[5] Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial [J].

Burt, Richard K. ;

Shah, Sanjiv J. ;

Dill, Karin ;

Grant, Thomas ;

Gheorghiade, Mihai ;

Schroeder, James ;

Craig, Robert ;

Hirano, Ikuo ;

Marshall, Karin ;

Ruderman, Eric ;

Jovanovic, Borko ;

Milanetti, Francesca ;

Jain, Sandeep ;

Boyce, Kristin ;

Morgan, Amy ;

Carr, James ;

Barr, Walter .

LANCET, 2011, 378 (9790) :498-506

[6] Secondary autoimmune diseases occurring after HSCT for an autoimmune disease: a retrospective study of the EBMT Autoimmune Disease Working Party [J].

Daikeler, Thomas ;

Labopin, Myriam ;

Di Gioia, Massimo ;

Abinun, Mario ;

Alexander, Tobias ;

Miniati, Irene ;

Gualandi, Francesca ;

Fassas, Athanasios ;

Martin, Thierry ;

Schwarze, Carl Philipp ;

Wulffraat, Nico ;

Buch, Maya ;

Sampol, Antonia ;

Carreras, Enric ;

Dubois, Benedicte ;

Gruhn, Bernd ;

Guengoer, Tayfun ;

Pohlreich, David ;

Schuerwegh, Annemie ;

Snarski, Emilian ;

Snowden, John ;

Veys, Paul ;

Fasth, Anders ;

Lenhoff, Stig ;

Messina, Chiara ;

Voswinkel, Jan ;

Badoglio, Manuela ;

Henes, Joerg ;

Launay, David ;

Tyndall, Alan ;

Gluckman, Eliane ;

Farge, Dominique .

BLOOD, 2011, 118 (06) :1693-1698

[7] State-of-the-art fertility preservation in children and adolescents undergoing haematopoietic stem cell transplantation: a report on the expert meeting of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) in Baden, Austria, 29-30 September 2015 [J].

Dalle, J-H ;

Lucchini, G. ;

Balduzzi, A. ;

Ifversen, M. ;

Jahnukainen, K. ;

Macklon, K. T. ;

Ahler, A. ;

Jarisch, A. ;

Ansari, M. ;

Beohou, E. ;

Bresters, D. ;

Corbacioglu, S. ;

Dalissier, A. ;

de Heredia Rubio, C. Diaz ;

Diesch, T. ;

Gibson, B. ;

Klingebiel, T. ;

Lankester, A. ;

Lawitschka, A. ;

Moffat, R. ;

Peters, C. ;

Poirot, C. ;

Saenger, N. ;

Sedlacek, P. ;

Trigoso, E. ;

Vettenranta, K. ;

Wachowiak, J. ;

Willasch, A. ;

von Wolff, M. ;

Yaniv, I. ;

Yesilipek, A. ;

Bader, P. .

BONE MARROW TRANSPLANTATION, 2017, 52 (07) :1029-1035

[8] Nintedanib for Systemic Sclerosis-Associated Interstitial Lung Disease [J].

Distler, Oliver ;

Highland, Kristin B. ;

Gahlemann, Martina ;

Azuma, Arata ;

Fischer, Aryeh ;

Mayes, Maureen D. ;

Raghu, Ganesh ;

Sauter, Wiebke ;

Girard, Mannaig ;

Alves, Margarida ;

Clerisme-Beaty, Emmanuelle ;

Stowasser, Susanne ;

Tetzlaff, Kay ;

Kuwana, Masataka ;

Maher, Toby M. .

NEW ENGLAND JOURNAL OF MEDICINE, 2019, 380 (26) :2518-2528

[9] Mapping and predicting mortality from systemic sclerosis [J].

Elhai, Muriel ;

Meune, Christophe ;

Boubaya, Marouane ;

Avouac, Jerome ;

Hachulla, Eric ;

Balbir-Gurman, Alexandra ;

Riemekasten, Gabriela ;

Airo, Paolo ;

Joven, Beatriz ;

Vettori, Serena ;

Cozzi, Franco ;

Ullman, Susanne ;

Czirjak, Laszlo ;

Tikly, Mohammed ;

Mueller-Ladner, U. L. F. ;

Caramaschi, Paola ;

Distler, Oliver ;

Iannone, Florenzo ;

Ananieva, Lidia P. ;

Hesselstrand, Roger ;

Becvar, Radim ;

Gabrielli, Armando ;

Damjanov, Nemanja ;

Salvador, Maria J. ;

Riccieri, Valeria ;

Mihai, Carina ;

Szucs, Gabriella ;

Walker, Ulrich A. ;

Hunzelmann, Nicolas ;

Martinovic, Duska ;

Smith, Vanessa ;

Mueller, Carolina de Souza ;

Montecucco, Carlo Maurizio ;

Opris, Daniela ;

Ingegnoli, Francesca ;

Vlachoyiannopoulos, Panayiotis G. ;

Stamenkovic, Bojana ;

Rosato, Edoardo ;

Heitmann, Stefan ;

Distler, Joerg H. W. ;

Zenone, Thierry ;

Seidel, Matthias ;

Vacca, Alessandra ;

De langhe, Ellen ;

Novak, Srdan ;

Cutolo, Maurizio ;

Mouthon, Luc ;

Henes, Joerg ;

Chizzolini, Carlo ;

von Muhlen, Carlos Alberto .

ANNALS OF THE RHEUMATIC DISEASES, 2017, 76 (11) :1897-1905

[10] Cardiopulmonary assessment of patients with systemic sclerosis for hematopoietic stem cell transplantation: recommendations from the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party and collaborating partners [J].

Farge, D. ;

Burt, R. K. ;

Oliveira, M-C ;

Mousseaux, E. ;

Rovira, M. ;

Marjanovic, Z. ;

de Vries-Bouwstra, J. ;

Del Papa, N. ;

Saccardi, R. ;

Shah, S. J. ;

Lee, D. C. ;

Denton, C. ;

Alexander, T. ;

Kiely, D. G. ;

Snowden, J. A. .

BONE MARROW TRANSPLANTATION, 2017, 52 (11) :1495-1503

← 1 2 3 →