ACR Meeting Abstracts

ACR Meeting Abstracts

  • Meetings
    • ACR Convergence 2024
    • ACR Convergence 2023
    • 2023 ACR/ARP PRSYM
    • ACR Convergence 2022
    • ACR Convergence 2021
    • ACR Convergence 2020
    • 2020 ACR/ARP PRSYM
    • 2019 ACR/ARP Annual Meeting
    • 2018-2009 Meetings
    • Download Abstracts
  • Keyword Index
  • Advanced Search
  • Your Favorites
    • Favorites
    • Login
    • View and print all favorites
    • Clear all your favorites
  • ACR Meetings

Abstract Number: 1364

Autologous Hematopoietic Stem Cell (HSC) Transplantation for Systemic Sclerosis, North American Registry: Updated Outcomes and the Impact of CD34+ HSC Enrichment

George Georges1, Keith Sullivan2, Dinesh Khanna3, Matthew Mei4, Maureen Mayes5, Dan Furst6, Mark Wener7, Suzanne Kafaja8, Gwynn Long2, Vaishali Sanchorawala9, Kareem Jamani10, Attaphol Pawarode3, Ashish Masurekar11, Brian Lam12, Richard Nash13, Marcin Trojanowski9, Chitra Hosing14, Philip Clements15, Tanya Helms2, Bernadette McLaughlin16, Linda Griffith17, Kyle Hebert18, Mary Eapen18, Jan Storek10 and Harold Atkins11, 1Fred Hutchison Cancer Research Center and University of Washington, Seattle, WA, 2Duke University School of Medicine, Durham, NC, 3University of Michigan, Ann Arbor, MI, 4City of Hope Comprehensive Cancer Center, Duarte, CA, 5Division of Rheumatology and Clinical Immunogenetics, University of Texas McGovern Medical School, Houston, TX, 6University of California Los Angeles, Los Angeles, CA, 7University of Washington, Seattle, WA, 8University of California Los Angeles Division of Rheumatology, Los Angeles, CA, 9Boston University School of Medicine, Boston, MA, 10University of Calgary, Calgary, AB, Canada, 11Ottawa Hospital Research Institute, Ottawa, ON, Canada, 12University of Texas McGovern Medical School, Houston, TX, 13Colorado Blood Cancer Institute, Denver, CO, 14University of Texas MD Anderson Cancer Center, Houston, TX, 15University of California Los Angeles School of Medicine, Department of Rhemumatology, Los Angeles, CA, 16Fred Hutchison Cancer Research Center, Seattle, WA, 17Division of Allergy Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Rockville, MD, 18Medical College of Wisconsin, Milwaukee, WI

Meeting: ACR Convergence 2021

Keywords: autoimmune diseases, prognostic factors, registry, Scleroderma, Systemic, Systemic sclerosis

  • Tweet
  • Click to email a link to a friend (Opens in new window) Email
  • Click to print (Opens in new window) Print
Session Information

Date: Monday, November 8, 2021

Title: Systemic Sclerosis & Related Disorders – Clinical Poster II (1364–1390)

Session Type: Poster Session C

Session Time: 8:30AM-10:30AM

Background/Purpose: Autologous hematopoietic stem cell transplantation (AHSCT) is now established as a preferred standard of care treatment for patients with severe scleroderma with internal organ involvement (SSc). Two large randomized clinical trials of AHSCT vs. monthly cyclophosphamide (CYC) used CD34+ HSC enrichment to deplete immune cells from the autologous peripheral blood stem cell (PBSC) graft. One trial used total body irradiation (TBI)/CYC/anti-thymocyte globulin (ATG) conditioning regimen1, the other used CYC/ATG (no TBI)2. After these trials, due to reporting issues, many SSc patients given HSCT received unmanipulated PBSC (no CD34+ enrichment). We sought to (1) update the outcomes of AHSCT for SSc in North America in a larger contemporary cohort and (2) examine the importance of patient factors and transplant regimens for overall survival (OS) and progression free survival (PFS).

Methods: A questionnaire for SSc-specific health status before and after AHSCT was created for patients reported to the Center for International Blood & Marrow Transplantation Research. Participating HSCT/rheumatology sites completed questionnaires for each SSc patient transplanted between 2000 to 2020, by conducting medical records review/ telephone interviews in early/mid-2021. Health status, physical function, toxicities, DMARD use and major medical events were recorded. Event endpoints included time to death, initiation of unplanned DMARD, organ failure/transplant, supplemental oxygen, new diagnosis of pulmonary arterial hypertension, enteral feeding tube, total parenteral nutrition, or subsequent HSCT.

Results: 104 patients with SSc were reported to the registry and provided SSc-specific health data. All patients had skin involvement, 56% with lung, 29% lung with other organ, 3% with kidney/GI, and 13% with skin only SSc. Conditioning with TBI/CYC/ATG was in 45%, CYC/ATG was in 55%. CD34+ enrichment of PBSC was used in 39%, 61% without enrichment. 64% had AHSCT between 2015 to 2020. Median follow-up was 41 (range, 5-222) months. Median age at AHSCT was 48 (range, 14-74) years (yr). For the entire cohort of 104 patients (Fig.1), the 5-yr OS was 91%, (95% confidence interval, 84-95). The 5-yr PFS was 56% (45-67). Fig.2, the 5-yr OS with TBI/CYC/ATG was 87% (75-96) and CYC/ATG 94% (86-99) (P-value = 0.16). The corresponding 5-yr PFS were 60% (45-75) and 51% (33-68) (P = 0.92). Fig.3, the 5-yr. OS for the cohort given CD34+ enriched PBSC was 96% (85-100) and unmanipulated PBSC was 87% (77-95) (P = 0.28). The 5-yr. corresponding PFS were 67% (49-83) and 50% (36-64) (P = 0.13). ATG source (horse vs. rabbit) did not affect OS/PFS. Age, examined as a predictor for OS/PFS, was not significant. In multivariate analysis, no tested covariates were significant for OS or PFS.

Conclusion: The OS and PFS benefit of AHSCT for patients with severe SSc was confirmed in this 5-year retrospective, real-world, non-randomized analysis and was independent of the patient factors or transplant regimens analyzed. While the data suggested use of CD34+ enrichment of PBSC for AHSCT improved PFS for SSc patients, study of a larger cohort for a longer time is needed to evaluate significance.

References 1Sullivan, NEJM 2018; 378:35-47 2van Laar, JAMA 2014; 311:2490-98

Figure 1. A. 5-year overall survival (OS) for 104 SSc registry patients with AHSCT between 2000 and 2020. B. Corresponding progression-free survival (PFS). Kaplan-Meier (KM) estimate.

Figure 2. A. 5-yr. OS after AHSCT for SSc, non-randomized, registry patients conditioned with TBI/CYC/ATG vs. CYC/ATG, 87% and 94%, P= 0.16. B. Corresponding 5-yr. PFS, 60% and 51%, P=0.92, log-rank test.

Figure 3. A. 5-yr. OS after AHSCT for SSc, non-randomized, registry patients with PBSC CD34+ enriched vs. unmanipulated, 96% and 87%, P=0.28. B. Corresponding 5- yr. PFS 67% and 50%, P=0.13, log-rank test.


Disclosures: G. Georges, Magenta Therapeutics, 2, Jasper Therapeutics Inc, 2; K. Sullivan, None; D. Khanna, AbbVie, 2, Acceleron, 2, Actelion, 2, Amgen, 2, Bayer, 2, 5, Boehringer Ingelheim, 2, Bristol-Myers Squibb, 5, CiviBioPharma/Eicos Sciences, Inc, 12, Leadership/Equity position (Chief Medical Officer), Corbus, 2, CSL Behring, 2, Eicos Sciences, Inc, 11, Galapagos NV, 2, Genentech/Roche, 2, Gilead, 2, GlaxoSmithKline, 2, Horizon Therapeutics, 2, 5, Immune Tolerance Network, 5, Merck Sharp & Dohme, 2, Mitsubishi Tanabe Pharma, 2, National Institutes of Health, 5, Pfizer, 5, Sanofi-Aventis, 2, United Therapeutics, 2, Prometheus, 2, Theraly, 2, AstraZeneca, 2; M. Mei, None; M. Mayes, Actelion Pharma, 1, Mitsubishi-Tanabe, 1, Corbus Pharma, 5, Boehringer-Ingelheim, 1, 5, Eicos, 1, 5, Galapagos Pharma, 1, 5; D. Furst, Actelion, 2, 5, Amgen, 2, 5, BMS, 2, 5, Corbus, 2, 6, Galapagos, 2, 5, GSK, 6, Sanofi, 2, 5, 6, Roche/Genentech, 5, National Institutes of Health, 5, Novartis, 2, 5, Pfizer, 2, 5; M. Wener, None; S. Kafaja, Eicos, 2, 5, Corbus, 2, Galapagos, 5, Cumberland, 5, Novartis, 5; G. Long, None; V. Sanchorawala, None; K. Jamani, None; A. Pawarode, None; A. Masurekar, None; B. Lam, None; R. Nash, None; M. Trojanowski, None; C. Hosing, None; P. Clements, None; T. Helms, None; B. McLaughlin, None; L. Griffith, None; K. Hebert, None; M. Eapen, None; J. Storek, None; H. Atkins, None.

To cite this abstract in AMA style:

Georges G, Sullivan K, Khanna D, Mei M, Mayes M, Furst D, Wener M, Kafaja S, Long G, Sanchorawala V, Jamani K, Pawarode A, Masurekar A, Lam B, Nash R, Trojanowski M, Hosing C, Clements P, Helms T, McLaughlin B, Griffith L, Hebert K, Eapen M, Storek J, Atkins H. Autologous Hematopoietic Stem Cell (HSC) Transplantation for Systemic Sclerosis, North American Registry: Updated Outcomes and the Impact of CD34+ HSC Enrichment [abstract]. Arthritis Rheumatol. 2021; 73 (suppl 9). https://acrabstracts.org/abstract/autologous-hematopoietic-stem-cell-hsc-transplantation-for-systemic-sclerosis-north-american-registry-updated-outcomes-and-the-impact-of-cd34-hsc-enrichment/. Accessed .
  • Tweet
  • Click to email a link to a friend (Opens in new window) Email
  • Click to print (Opens in new window) Print

« Back to ACR Convergence 2021

ACR Meeting Abstracts - https://acrabstracts.org/abstract/autologous-hematopoietic-stem-cell-hsc-transplantation-for-systemic-sclerosis-north-american-registry-updated-outcomes-and-the-impact-of-cd34-hsc-enrichment/

Advanced Search

Your Favorites

You can save and print a list of your favorite abstracts during your browser session by clicking the “Favorite” button at the bottom of any abstract. View your favorites »

All abstracts accepted to ACR Convergence are under media embargo once the ACR has notified presenters of their abstract’s acceptance. They may be presented at other meetings or published as manuscripts after this time but should not be discussed in non-scholarly venues or outlets. The following embargo policies are strictly enforced by the ACR.

Accepted abstracts are made available to the public online in advance of the meeting and are published in a special online supplement of our scientific journal, Arthritis & Rheumatology. Information contained in those abstracts may not be released until the abstracts appear online. In an exception to the media embargo, academic institutions, private organizations, and companies with products whose value may be influenced by information contained in an abstract may issue a press release to coincide with the availability of an ACR abstract on the ACR website. However, the ACR continues to require that information that goes beyond that contained in the abstract (e.g., discussion of the abstract done as part of editorial news coverage) is under media embargo until 10:00 AM ET on November 14, 2024. Journalists with access to embargoed information cannot release articles or editorial news coverage before this time. Editorial news coverage is considered original articles/videos developed by employed journalists to report facts, commentary, and subject matter expert quotes in a narrative form using a variety of sources (e.g., research, announcements, press releases, events, etc.).

Violation of this policy may result in the abstract being withdrawn from the meeting and other measures deemed appropriate. Authors are responsible for notifying colleagues, institutions, communications firms, and all other stakeholders related to the development or promotion of the abstract about this policy. If you have questions about the ACR abstract embargo policy, please contact ACR abstracts staff at [email protected].

Wiley

  • Online Journal
  • Privacy Policy
  • Permissions Policies
  • Cookie Preferences

© Copyright 2025 American College of Rheumatology