INTRODUCTION: Outcomes of CML-CP patients treated in clinical trials are frequently perceived to be not representative of those treated outside of clinical trials. PATIENTS AND METHODS: We investigated the outcomes of patients receiving imatinib outside of a clinical trial (off protocol) or in a clinical trial (on protocol) for CML-CP. RESULTS: We identified 65 patients treated with imatinib off protocol and 71 patients treated on protocol with standard-dose imatinib. The overall complete cytogenetic response (CCyR) rate was 83% for patients treated on and off protocol. CCyR rates 12 months after initiation of imatinib were not statistically different (61% vs. 66%, respectively; P = .15). Patients treated off protocol had similar rates of overall major molecular response (72% vs. 73%) compared with the patients treated on protocol. The 5-year event-free survival rates were 84% and 86% for off and on protocol patients, respectively. There was also no significant difference in 5-year transformation free survival (94% vs. 96%) and overall survival (96% vs. 90%). CONCLUSION: These results suggest that patients with CML treated outside of a clinical trial might have the same excellent outcome as those treated in a clinical trial provided they are followed with the same rigor.
INTRODUCTION: Outcomes of CML-CPpatients treated in clinical trials are frequently perceived to be not representative of those treated outside of clinical trials. PATIENTS AND METHODS: We investigated the outcomes of patients receiving imatinib outside of a clinical trial (off protocol) or in a clinical trial (on protocol) for CML-CP. RESULTS: We identified 65 patients treated with imatinib off protocol and 71 patients treated on protocol with standard-dose imatinib. The overall complete cytogenetic response (CCyR) rate was 83% for patients treated on and off protocol. CCyR rates 12 months after initiation of imatinib were not statistically different (61% vs. 66%, respectively; P = .15). Patients treated off protocol had similar rates of overall major molecular response (72% vs. 73%) compared with the patients treated on protocol. The 5-year event-free survival rates were 84% and 86% for off and on protocol patients, respectively. There was also no significant difference in 5-year transformation free survival (94% vs. 96%) and overall survival (96% vs. 90%). CONCLUSION: These results suggest that patients with CML treated outside of a clinical trial might have the same excellent outcome as those treated in a clinical trial provided they are followed with the same rigor.
Authors: Edgar Faber; Jan Mužík; Vladimír Koza; Eva Demečková; Jaroslava Voglová; Ludmila Demitrovičová; Juraj Chudej; Imrich Markuljak; Eduard Cmunt; Tomáš Kozák; Elena Tóthová; Marie Jarošová; Ladislav Dušek; Karel Indrák Journal: Eur J Haematol Date: 2011-08 Impact factor: 2.997
Authors: Brian J Druker; François Guilhot; Stephen G O'Brien; Insa Gathmann; Hagop Kantarjian; Norbert Gattermann; Michael W N Deininger; Richard T Silver; John M Goldman; Richard M Stone; Francisco Cervantes; Andreas Hochhaus; Bayard L Powell; Janice L Gabrilove; Philippe Rousselot; Josy Reiffers; Jan J Cornelissen; Timothy Hughes; Hermine Agis; Thomas Fischer; Gregor Verhoef; John Shepherd; Giuseppe Saglio; Alois Gratwohl; Johan L Nielsen; Jerald P Radich; Bengt Simonsson; Kerry Taylor; Michele Baccarani; Charlene So; Laurie Letvak; Richard A Larson Journal: N Engl J Med Date: 2006-12-07 Impact factor: 91.245
Authors: Hagop Kantarjian; Charles Sawyers; Andreas Hochhaus; Francois Guilhot; Charles Schiffer; Carlo Gambacorti-Passerini; Dietger Niederwieser; Debra Resta; Renaud Capdeville; Ulrike Zoellner; Moshe Talpaz; Brian Druker; John Goldman; Stephen G O'Brien; Nigel Russell; Thomas Fischer; Oliver Ottmann; Pascale Cony-Makhoul; Thierry Facon; Richard Stone; Carole Miller; Martin Tallman; Randy Brown; Michael Schuster; Thomas Loughran; Alois Gratwohl; Franco Mandelli; Giuseppe Saglio; Mario Lazzarino; Domenico Russo; Michele Baccarani; Enrica Morra Journal: N Engl J Med Date: 2002-02-28 Impact factor: 91.245
Authors: B J Druker; M Talpaz; D J Resta; B Peng; E Buchdunger; J M Ford; N B Lydon; H Kantarjian; R Capdeville; S Ohno-Jones; C L Sawyers Journal: N Engl J Med Date: 2001-04-05 Impact factor: 91.245
Authors: Hagop M Kantarjian; Susan O'Brien; Jorge Cortes; Francis J Giles; Mary Beth Rios; Jianqin Shan; Stefan Faderl; Guillermo Garcia-Manero; Alessandra Ferrajoli; Srdan Verstovsek; William Wierda; Michael Keating; Moshe Talpaz Journal: Cancer Date: 2003-12-15 Impact factor: 6.860
Authors: Hugues de Lavallade; Jane F Apperley; Jamshid S Khorashad; Dragana Milojkovic; Alistair G Reid; Marco Bua; Richard Szydlo; Eduardo Olavarria; Jaspal Kaeda; John M Goldman; David Marin Journal: J Clin Oncol Date: 2008-06-02 Impact factor: 44.544
Authors: Stephen G O'Brien; François Guilhot; Richard A Larson; Insa Gathmann; Michele Baccarani; Francisco Cervantes; Jan J Cornelissen; Thomas Fischer; Andreas Hochhaus; Timothy Hughes; Klaus Lechner; Johan L Nielsen; Philippe Rousselot; Josy Reiffers; Giuseppe Saglio; John Shepherd; Bengt Simonsson; Alois Gratwohl; John M Goldman; Hagop Kantarjian; Kerry Taylor; Gregor Verhoef; Ann E Bolton; Renaud Capdeville; Brian J Druker Journal: N Engl J Med Date: 2003-03-13 Impact factor: 91.245