Long-term outcome of patients with newly diagnosed chronic myeloid leukemia: a randomized comparison of stem cell transplantation with drug treatment

Gratwohl, A. and Pfirrmann, M. and Zander, A. and Kroger, N. and Beelen, D. and Novotny, J. and Nerl, C. and Scheid, C. and Spiekermann, K. and Mayer, J. and Sayer, H. G. and Falge, C. and Bunjes, D. and Doehner, H. and Ganser, A. and Schmidt-Wolf, I. and Schwerdtfeger, R. and Baurmann, H. and Kuse, R. and Schmitz, N. and Wehmeier, A. and Fischer, J. Th and Ho, A. D. and Wilhelm, M. and Goebeler, M-E and Lindemann, H. W. and Bormann, M. and Hertenstein, B. and Schlimok, G. and Baerlocher, G. M. and Aul, C. and Pfreundschuh, M. and Fabian, M. and Staib, P. and Edinger, M. and Schatz, M. and Fauser, A. and Arnold, R. and Kindler, T. and Wulf, G. and Rosselet, A. and Hellmann, A. and Schaefer, E. and Pruemmer, O. and Schenk, M. and Hasford, J. and Heimpel, H. and Hossfeld, D. K. and Kolb, H-J and Buesche, G. and Haferlach, C. and Schnittger, S. and Mueller, M. C. and Reiter, A. and Berger, U. and Saussele, S. and Hochhaus, A. and Hehlmann, R. (2016) Long-term outcome of patients with newly diagnosed chronic myeloid leukemia: a randomized comparison of stem cell transplantation with drug treatment. LEUKEMIA, 30 (3). pp. 562-569. ISSN 0887-6924, 1476-5551

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Abstract

Tyrosine kinase inhibitors represent today's treatment of choice in chronic myeloid leukemia (CML). Allogeneic hematopoietic stem cell transplantation (HSCT) is regarded as salvage therapy. This prospective randomized CML-study IIIA recruited 669 patients with newly diagnosed CML between July 1997 and January 2004 from 143 centers. Of these, 427 patients were considered eligible for HSCT and were randomized by availability of a matched family donor between primary HSCT (group A; N = 166 patients) and best available drug treatment (group B; N = 261). Primary end point was long-term survival. Survival probabilities were not different between groups A and B (10-year survival: 0.76 (95% confidence interval (CI): 0.69-0.82) vs 0.69 (95% CI: 0.61-0.76)), but influenced by disease and transplant risk. Patients with a low transplant risk showed superior survival compared with patients with high( P < 0.001) and non-high-risk disease (P = 0.047) in group B; after entering blast crisis, survival was not different with or without HSCT. Significantly more patients in group A were in molecular remission (56% vs 39%; P = 0.005) and free of drug treatment (56% vs 6%; P < 0.001). Differences in symptoms and Karnofsky score were not significant. In the era of tyrosine kinase inhibitors, HSCT remains a valid option when both disease and transplant risk are considered.

Item Type: Article
Uncontrolled Keywords: CHRONIC MYELOGENOUS LEUKEMIA; BONE-MARROW-TRANSPLANTATION; EUROPEAN LEUKEMIANET; MOLECULAR RESPONSE; HEMATOPOIETIC SCT; INTERFERON-ALPHA; DOSE IMATINIB; SURVIVAL; ERA; RECOMMENDATIONS;
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie)
Depositing User: Dr. Gernot Deinzer
Date Deposited: 12 Mar 2019 10:28
Last Modified: 12 Mar 2019 10:28
URI: https://pred.uni-regensburg.de/id/eprint/2329

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