Management of disease progression after autologous hematopoietic stem cell transplantation in systemic sclerosis: Results from an international questionnaire-based study

被引：0

作者：

Spierings, Julia ^{[1
,2
]}

Bandini, Giulia ^{[3
]}

Allanore, Yannick ^{[4
]}

Del Papa, Nicoletta ^{[5
]}

Denton, Christopher P. ^{[2
]}

Distler, Oliver ^{[6
]}

Furst, Daniel E. ^{[7
]}

Greco, Raffaella ^{[8
]}

Khanna, Dinesh ^{[9
]}

Kuwana, Masataka ^{[10
]}

Matucci-Cerinic, Marco ^{[11
,12
,13
]}

Nikpour, Mandana ^{[14
]}

van Rhenen, Anna ^{[15
]}

Laar, Jacob M. van ^{[1
]}

Hughes, Michael ^{[16
,17
]}

机构：

[1] Univ Med Ctr Utrecht, Dept Rheumatol & Clin Immunol, Heidelberglaan 100, NL-3584CX Utrecht, Netherlands

[2] UCL Med Sch, Ctr Rheumatol & Connect Tissue Dis, Royal Free Campus, London, England

[3] Univ Florence, Careggi Hosp, Dept Expt & Clin Med, Florence, Italy

[4] Rene Descartes Univ, Cochin Hosp, Dept Rheumatol A, Paris, France

[5] Univ Milan, Scleroderma Clin, ASST Pini CTO, Milan, Italy

[6] Univ Zurich, Univ Hosp Zurich, Dept Rheumatol, Zurich, Switzerland

[7] Univ Calif Los Angeles, Los Angeles, CA USA

[8] Univ Vita Salute San Raffaele, IRCCS San Raffaele Sci Inst, Unit Hematol & Bone Marrow Transplantat, Milan, Italy

[9] Univ Michigan, Scleroderma Program, Ann Arbor, MI 48103 USA

[10] Nippon Med Sch Grad Sch Med, Dept Allergy & Rheumatol, 1-1-5 Sendagi,Bunkyo Ku, Tokyo 1138603, Japan

[11] IRCCS Osped San Raffaele, Unit Immunol Rheumatol Allergy & Rare Dis UnIRAR, Milan, Italy

[12] IRCCS Osped San Raffaele, Inflammat Fibrosis & Aging Initiat INFLAGE, Milan, Italy

[13] Univ Vita Salute San Raffaele, Milan, Italy

[14] Univ Sydney, Royal Prince Alfred Hosp Sydney, MSK Res Flagship Ctr, Sch Publ Hlth, Sydney, NSW, Australia

[15] Univ Med Ctr Utrecht, Dept Haematol, Utrecht, Netherlands

[16] Univ Manchester, Manchester Acad Hlth Sci Ctr, Div Musculoskeletal & Dermatol Sci, Manchester, England

[17] Northern Care Alliance NHS Fdn Trust, Salford Care Org, Salford, England

来源：

SEMINARS IN ARTHRITIS AND RHEUMATISM | 2025年 / 71卷

关键词：

Systemic sclerosis; Scleroderma; Autologous hematopoietic stem cell; transplantation; Stem cell therapy; Disease progression; maintenance therapy; GUIDELINES;

D O I：

10.1016/j.semarthrit.2025.152638

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Objective: Autologous stem cell transplantation (AHSCT) is an established treatment in diffuse cutaneous systemic sclerosis (dcSSc). Optimal management of disease progression after AHSCT in dcSSc has not been defined. The aim of this study was to explore the experience and preferences of SSc experts on post-AHSCT management. Methods: An online questionnaire study was conducted containing 17 questions concerning respondent demographics, definition of SSc progression after AHSCT, diagnostic work-up and treatment preferences. Results: In total, 69 respondents from 21 countries completed the questionnaire. The majority (89.7 %) works at a university hospital, and were involved in decisions regarding AHSCT in patients with SSc (71 %). Most have 1 to 5 patients who underwent AHSCT under their care. They defined failure to improve after AHSCT as: an increase in mRSS, new onset or worsening of interstitial lung disease (ILD), new onset scleroderma renal crisis (SRC) or inflammatory arthritis. Progression after initial response was defined as: increase in mRSS, new or worsening of ILD, new SRC, inflammatory arthritis, new pulmonary arterial hypertension, digital vasculopathy or impaired physical functioning. The most frequent therapy in case of AHSCT failure was mycophenolate mofetil (N = 55, 88.7 %), rituximab (N = 54, 87.1 %), nintedanib (N = 39, 62.9 %) or/and tocilizumab (N = 36, 58.1 %). Combination therapy with more than one of these agents was considered by most respondents (N = 61, 88.4 %).

引用

页数：6

共 22 条

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