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Abstract Number: 6L

Myeloablative Autologous Transplantation of CD34+ -Selected Hematopoietic Stem Cells (HSCT) Vs Monthly Intravenous Cyclophosphamide (CYC) for Severe Scleroderma with Internal Organ Involvement: Outcomes of a Randomized North American Clinical Trial

Keith Sullivan1, Lynette Keyes-Elstein2, Peter McSweeney3, Ashley Pinckney2, Beverly Welch4, Maureen D Mayes5, Richard Nash3, Leslie J. Crofford6, Sharon Castina2, Linda Griffith7, Ellen Goldmuntz8 and Daniel E. Furst9, 1Duke University, Durham, NC, 2Rho, Inc, Chapel Hill, NC, 3Colorado Blood Cancer Institute, Denver, CO, 46610 Rockledge Dr., NIAID/NIH, Bethesda, MD, 5Department of Internal Medicine - Rheumatology, University of Texas-McGovern Medical School, Houston, TX, 6Medicine, Vanderbilt University Medical Center, Nasville, TN, 7NIAID, NIH, Bethesda, MD, 8NIAID, National Institutes of Health, Bethesda, MD, 9David Geffen School of Medicine at UCLA, Los Angeles, CA

Meeting: 2016 ACR/ARHP Annual Meeting

Date of first publication: October 19, 2016

Keywords: clinical trials, cyclophosphamide, Hematopoietic stem cells, Late-Breaking 2016, scleroderma and systemic sclerosis

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Session Information

Date: Tuesday, November 15, 2016

Session Title: ACR Late-breaking Abstract Session

Session Type: ACR Late-breaking Abstract Session

Session Time: 4:30PM-6:00PM

 

Background/Purpose: Therapeutic options for diffuse cutaneous systemic sclerosis (dcSSc) are limited. The Scleroderma: Cyclophosphamide or Transplantation (SCOT) trial was a multicenter study designed to evaluate the long-term benefit of myeloablative autologous HSCT compared to 12 monthly pulses of CYC.

Methods: Adults (18-69 years) with dcSSc (ACR criteria) and high-risk lung and/or renal involvement were randomized to CYC (750 mg/m2/mo.) or myeloablation (800 cGy total body irradiation with lung and kidney shielding, 120 mg/kg CYC and 90 mg/kg antithymocyte globulin) followed by CD34+selected autologous HSCT. The primary endpoint was a global rank composite score (GRCS) at 54 months that ordered subjects based on a hierarchy of outcomes: death, event-free survival (EFS), forced vital capacity (FVC), scleroderma health assessment questionnaire, and modified Rodnan skin score (mRSS).

Results: Seventy-five subjects were randomized (39 CYC, 36 HSCT) with mean baseline mRSS=30, mean FVC=74% and mean DLC0=53% of predicted. All but two had lung involvement. In the ITT (randomized) sample, GRCS comparisons favored HSCT and met the predefined primary endpoint at 54 months (p=0.013) and 48 months (p=0.008). In those who were transplanted or received ≥ 9 CYC doses (34 CYC, 33 HSCT), GRCS results (figure 1) were even stronger (p=0.004 and 0.003, respectively). Secondary analyses in treated subjects supported the primary results: at 54 months, EFS was 50% in CYC and 79% in HSCT (p=0.021) and overall survival (OS) was 77% and 91% (p=0.19), respectively. Treatment related mortality at 54 months was 0% CYC and 3% HSCT. Among HSCT recipients, 9% had initiated DMARDs by 54 months compared to 44% of CYC (p=0.001). As shown in figure 2, Kaplan-Meier estimates for EFS and OS differed significantly between groups (p=0.030, 0.019, respectively). The rate of SAEs was similar in both arms.  Grade 3 and above AEs including regimen-related cytopenias were more common in the HSCT arm. Herpes zoster was more frequent in the HSCT arm.    

Conclusion: This study demonstrates long-term superiority of myeloablative autologous HSCT over CYC on efficacy endpoints, and shows a significant reduction in use of DMARDS among HSCT recipients. Transplant-related mortality was lower and overall survival was better than projected both during the peri-transplant period and long term. The SCOT trial supports myeloablative HSCT as a significant advance in the care of dcSSc. Funded by NIAID; ClinicalTrials.gov NCT00114530        


Disclosure: K. Sullivan, None; L. Keyes-Elstein, None; P. McSweeney, None; A. Pinckney, None; B. Welch, None; M. D. Mayes, None; R. Nash, None; L. J. Crofford, None; S. Castina, None; L. Griffith, None; E. Goldmuntz, None; D. E. Furst, None.

To cite this abstract in AMA style:

Sullivan K, Keyes-Elstein L, McSweeney P, Pinckney A, Welch B, Mayes MD, Nash R, Crofford LJ, Castina S, Griffith L, Goldmuntz E, Furst DE. Myeloablative Autologous Transplantation of CD34+ -Selected Hematopoietic Stem Cells (HSCT) Vs Monthly Intravenous Cyclophosphamide (CYC) for Severe Scleroderma with Internal Organ Involvement: Outcomes of a Randomized North American Clinical Trial [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/myeloablative-autologous-transplantation-of-cd34-selected-hematopoietic-stem-cells-hsct-vs-monthly-intravenous-cyclophosphamide-cyc-for-severe-scleroderma-with-internal-organ-involvement-outcom/. Accessed May 19, 2022.
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