ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 2912

Predictive Factors for Treatment Related Mortality and Event-Free Survival After Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis: Results of a Long Term Follow-up Multi-centre Study

Sandra van Bijnen 1, Maaike Boonstra 2, Els van den Ende 3, Lucia Kroft 2, Bram Geurts 1, Miranda Snoeren 1, Anne Schouffoer 2, Julia Spierings 4, Jacob van Laar 5, Thomas Huizinga 6, Alexandre Voskuyl 7, Walter van der Velden 1, Frank van den Hoogen 3, Jeska de Vries-Bouwstra 6 and Madelon Vonk8, 1Radboud University Nijmegen Medical Centre, Nijmegen, Gelderland, Netherlands, 2Leiden University Medical Centre, Leiden, Zuid-Holland, Netherlands, 3Department of Rheumatology, Radboud University Medical Center, Nijmegen, Gelderland, Netherlands, 4Utrecht Medical Centre, Utrecht, Utrecht, Netherlands, 5UMC Utrecht, Department of Rheumatology & Clinical Immunology, Utrecht, The Netherlands, Utrecht, Netherlands, 6Leiden University Medical Center, Leiden, Netherlands, 7Amsterdam Univiversity Medical Centre, Amsterdam, Noord-Holland, Netherlands, 8Department of Rheumatology, Radboud University Medical Center, Nijmegen, The Netherlands, Nijmegen, Gelderland, Netherlands

Meeting: 2019 ACR/ARP Annual Meeting

Keywords: , ,

Session Information

Date: Wednesday, November 13, 2019

Title: 6W023: Systemic Sclerosis & Related Disorder – Clinical III: Predictors of Outcome (2912–2917)

Session Type: ACR Abstract Session

Session Time: 11:00AM-12:30PM

Background/Purpose: Autologous hematopoietic stemcell transplantation (HSCT) has shown to improve survival of SSc patients with poor prognosis, but is hampered by treatment related mortality (TRM). Better selection of eligible patients could improve outcomes.

Objectives: 1. To evaluate event-free survival, TRM and response after HSCT in SSc and 2. To explore patient characteristics that associate with events.

Methods: Data on event-free survival of all patients treated with HSCT for SSc in the Netherlands between 1998 and 2017, performed as previously described (1) and with ≥ 1 year of follow-up were collected. Baseline characteristics including hematopoietic cell transplant comorbidity index (HCT-CI) (2) and left ventricular ejection fraction (LVEF) and data for skin involvement (modified Rodnan skin score (mRSS)), pulmonary function (forced vital capacity % predicted (FVC% pred), diffusion capacity % predicted (DLCO% pred)) and staging for interstitial lung disease on high resolution CT scan (HRCT) assessed according Goh (3) were collected at baseline and at 1,2, 5 years ; data for events and death were collected until end of study. All deaths were discussed in a consensus meeting (SB, MB, JV, MV) and classified as TRM, SSc progression or other. Event-free survival was defined as described before (4). Relapse was defined as an increase in mRSS of > 25% and > 5 points compared to the lowest value since HSCT, or either a ≥10% decline in FVC% pred, or ≥5 – < 10% decline in FVC% pred and ≥15% decline in DLCO% pred or initiation of immunosuppression (4). The association between event-free survival and baseline characteristics was examined by univariate Cox regression analysis. Factors with a significant association were entered in a multivariate analysis on imputed missing data.

Results: In total 92 patients were included (Table 1). Event-free survival estimates at 5, 10 and 15 years were 0.79, 0.79 and 0.68 respectively. Twenty deaths occurred, which were classified as TRM (n=10, 11%), SSc progression (n=4, 4%) and other (n=6 7%). Relapse occurred in 22 patients in the first 5 years: skin relapse in 5, lung relapse in 11 and initiation of immunosuppression in 12 (in 7 patients without skin or lung relapse). FVC, DLCO, mRSS and Goh scores improved significantly over time in patients surviving the first 5 years (n=75) (Figure 1). Events were independently associated with male sex, LVEF < 50% and older age (table 2).

Conclusion: Event-free survival at 10 years after HSCT for SSc was 79%; male sex, lower LVEF and older age were identified as independent risk factors for events. Our data confirms efficacy of HSCT in improving survival and skin and lung involvement.
Refs:1. van Laar JM etal. JAMA. 2014;311(24):2490-8. 2. Sorror ML etal. Biol Blood Marrow Transplant. 2015;21(8):1479-87.3. Goh NS etal. Am J Respir Crit Care Med. 2008;177(11):1248-54.4. Khanna D etal.The Journal of rheumatology. 2015;42(11):2168-71.

Abbreviations: IQR: interquartile range; SSc: systemic sclerosis; SD: stand deviation ; ANA: antinuclear antibody; ESR: erythrocyte sedimentation rate; mRSS: modified Rodnan skin score; FVC %pred: forced vital capacity % predicted; DLCO %pred: diffusion capacity; HCT-CI: hematopoietic cell transplant comorbidity index; MTX: methotrexate; MMF: mycophenolate mofetil.

Figure 1Course over time in patients surviving the first 5 years -n = 75-
A skin involvement as measured by mRSS; B FVCpred: forced vital capacity % predicted; C DLCO pred: diffusion capacity % predicted; D Total Goh scores

Abbreviations: SSc: systemic sclerosis; HCT-CI: hematopoietic cell transplant comorbidity index; ESR: erythrocyte sedimentation rate; FVC % pred: forced vital capacity % predicted; LVEF: left ventricular ejection fraction; DLCO % pred: diffusion capacity % predicted; prog. mRSS: progression in skin involvement prior to stemceltransplantation;

Disclosure: S. van Bijnen, None; M. Boonstra, None; E. van den Ende, None; L. Kroft, None; B. Geurts, None; M. Snoeren, None; A. Schouffoer, None; J. Spierings, Boehringer Ingelheim, 2, Janssen, 8; J. van Laar, Roche, 2, 8, Arthrogen, 5, Thermofisher, 2, BMS, 8, MSD, 2, Eli Lilly, 8, Gesynta, 5, Leadiant, 5, Arxx Tx, 5, Astra Zeneca, 2, Sanofi, 8; T. Huizinga, Abblynx, 2, 5, 8, Abbott, 2, 5, 8, Biotest AG, 2, 5, 8, Boehringer Ingelheim, 2, 5, 8, Boeringher Ingelheim, 2, 5, 8, Bristol-Myers Squibb, 2, 5, 8, Crescendo Bioscience, 2, 5, 8, Eli Lilly, 2, 5, 8, Epirus, 2, 5, 8, Galapagos, 2, 5, 8, Janssen, 2, 5, 8, Merck, 2, 5, 8, Novartis, 2, 5, 8, Nycomed, 2, 5, 8, Pfizer, 2, 5, 8, Roche, 2, 5, 8, Sanofi, 2, 5, Sanofi-Aventis, 2, 5, 8, Takeda, 2, 5, 8, UCB, 2, 5, 8, Zydus, 2, 5, 8; A. Voskuyl, GSK, 8, Roche, 5, Boehringer Ingelheim, 5; W. van der Velden, None; F. van den Hoogen, AbbVie, 5, abbVie, 5, Actelion, 5, Amgen, 8, Biogen, 5, BMS, 5, Boehringer-Ingelheim, 8, Celltrion, 5, Corbus, 5, Eli-Lilly, 5, Mundipharma, 5, Novartis, 8, Pfizer, 5, Sanofi-Genzyme, 5; J. de Vries-Bouwstra, Boehringer Ingelheim, 5; M. Vonk, Actelion, 2, 5, 8, actelion, 2, 5, 8, Actelion Pharmaceuticals, 2, 5, 8, Boehringer Ingelheim, 5, 8, Boehringer ingelheim, 5, 8, Ferrer, 2, Ferrer International, 2, Ferrier, 2, GSK, 5, 6, Roche, 8.

To cite this abstract in AMA style:

van Bijnen S, Boonstra M, van den Ende E, Kroft L, Geurts B, Snoeren M, Schouffoer A, Spierings J, van Laar J, Huizinga T, Voskuyl A, van der Velden W, van den Hoogen F, de Vries-Bouwstra J, Vonk M. Predictive Factors for Treatment Related Mortality and Event-Free Survival After Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis: Results of a Long Term Follow-up Multi-centre Study [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). https://acrabstracts.org/abstract/predictive-factors-for-treatment-related-mortality-and-event-free-survival-after-autologous-hematopoietic-stem-cell-transplantation-for-systemic-sclerosis-results-of-a-long-term-follow-up-multi-centr/. Accessed .

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