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Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma.
Sullivan, Keith M; Goldmuntz, Ellen A; Keyes-Elstein, Lynette; McSweeney, Peter A; Pinckney, Ashley; Welch, Beverly; Mayes, Maureen D; Nash, Richard A; Crofford, Leslie J; Eggleston, Barry; Castina, Sharon; Griffith, Linda M; Goldstein, Julia S; Wallace, Dennis; Craciunescu, Oana; Khanna, Dinesh; Folz, Rodney J; Goldin, Jonathan; St Clair, E William; Seibold, James R; Phillips, Kristine; Mineishi, Shin; Simms, Robert W; Ballen, Karen; Wener, Mark H; Georges, George E; Heimfeld, Shelly; Hosing, Chitra; Forman, Stephen; Kafaja, Suzanne; Silver, Richard M; Griffing, Leroy; Storek, Jan; LeClercq, Sharon; Brasington, Richard; Csuka, Mary E; Bredeson, Christopher; Keever-Taylor, Carolyn; Domsic, Robyn T; Kahaleh, M Bashar; Medsger, Thomas; Furst, Daniel E.
Affiliation
  • Sullivan KM; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Goldmuntz EA; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Keyes-Elstein L; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • McSweeney PA; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Pinckney A; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Welch B; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Mayes MD; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Nash RA; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Crofford LJ; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Eggleston B; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Castina S; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Griffith LM; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Goldstein JS; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Wallace D; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Craciunescu O; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Khanna D; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Folz RJ; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Goldin J; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • St Clair EW; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Seibold JR; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Phillips K; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Mineishi S; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Simms RW; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Ballen K; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Wener MH; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Georges GE; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Heimfeld S; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Hosing C; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Forman S; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Kafaja S; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Silver RM; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Griffing L; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Storek J; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • LeClercq S; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Brasington R; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Csuka ME; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Bredeson C; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Keever-Taylor C; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Domsic RT; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Kahaleh MB; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Medsger T; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
  • Furst DE; From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.)
N Engl J Med ; 378(1): 35-47, 2018 01 04.
Article in En | MEDLINE | ID: mdl-29298160
BACKGROUND: Despite current therapies, diffuse cutaneous systemic sclerosis (scleroderma) often has a devastating outcome. We compared myeloablative CD34+ selected autologous hematopoietic stem-cell transplantation with immunosuppression by means of 12 monthly infusions of cyclophosphamide in patients with scleroderma. METHODS: We randomly assigned adults (18 to 69 years of age) with severe scleroderma to undergo myeloablative autologous stem-cell transplantation (36 participants) or to receive cyclophosphamide (39 participants). The primary end point was a global rank composite score comparing participants with each other on the basis of a hierarchy of disease features assessed at 54 months: death, event-free survival (survival without respiratory, renal, or cardiac failure), forced vital capacity, the score on the Disability Index of the Health Assessment Questionnaire, and the modified Rodnan skin score. RESULTS: In the intention-to-treat population, global rank composite scores at 54 months showed the superiority of transplantation (67% of 1404 pairwise comparisons favored transplantation and 33% favored cyclophosphamide, P=0.01). In the per-protocol population (participants who received a transplant or completed ≥9 doses of cyclophosphamide), the rate of event-free survival at 54 months was 79% in the transplantation group and 50% in the cyclophosphamide group (P=0.02). At 72 months, Kaplan-Meier estimates of event-free survival (74% vs. 47%) and overall survival (86% vs. 51%) also favored transplantation (P=0.03 and 0.02, respectively). A total of 9% of the participants in the transplantation group had initiated disease-modifying antirheumatic drugs (DMARDs) by 54 months, as compared with 44% of those in the cyclophosphamide group (P=0.001). Treatment-related mortality in the transplantation group was 3% at 54 months and 6% at 72 months, as compared with 0% in the cyclophosphamide group. CONCLUSIONS: Myeloablative autologous hematopoietic stem-cell transplantation achieved long-term benefits in patients with scleroderma, including improved event-free and overall survival, at a cost of increased expected toxicity. Rates of treatment-related death and post-transplantation use of DMARDs were lower than those in previous reports of nonmyeloablative transplantation. (Funded by the National Institute of Allergy and Infectious Diseases and the National Institutes of Health; ClinicalTrials.gov number, NCT00114530 .).
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Scleroderma, Systemic / Hematopoietic Stem Cell Transplantation / Cyclophosphamide / Immunosuppressive Agents Type of study: Clinical_trials / Etiology_studies / Guideline / Observational_studies / Prognostic_studies Limits: Adolescent / Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: N Engl J Med Year: 2018 Type: Article
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Scleroderma, Systemic / Hematopoietic Stem Cell Transplantation / Cyclophosphamide / Immunosuppressive Agents Type of study: Clinical_trials / Etiology_studies / Guideline / Observational_studies / Prognostic_studies Limits: Adolescent / Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: N Engl J Med Year: 2018 Type: Article