Jean Baptiste Micol1, Emmanuel Raffoux1, Nicolas Boissel1, Etienne Lengliné1, Emmanuel Canet2, Marie Thérèse Daniel3, Adrienne de Labarthe3, Odile Maarek3, Bruno Cassinat4, Lionel Adès5, André Baruchel6, Laurent Degos1, Elie Azoulay2, Hervé Dombret7. 1. Department of Hematology and EA3518, Hôpital Saint-Louis (Assistance Publique - Hôpitaux de Paris), University Paris Diderot, Paris, France. 2. Medical Intensive Care Unit, Hôpital Saint-Louis (Assistance Publique - Hôpitaux de Paris), Paris, France. 3. Laboratory of Hematology, Hôpital Saint-Louis (Assistance Publique - Hôpitaux de Paris), Paris, France. 4. Laboratory of Cellular Biology, Hôpital Saint-Louis (Assistance Publique - Hôpitaux de Paris), Paris, France. 5. Department of Hematology, Hôpital Avicenne (Assistance Publique - Hôpitaux de Paris), Bobigny, France. 6. Department of Pediatric Hematology, Hôpital Robert Debré (Assistance Publique - Hôpitaux de Paris), Paris, France. 7. Department of Hematology and EA3518, Hôpital Saint-Louis (Assistance Publique - Hôpitaux de Paris), University Paris Diderot, Paris, France. Electronic address: herve.dombret@sls.aphp.fr.
Abstract
PURPOSE: Acute promyelocytic leukaemia (APL) therapy with all-trans retinoic acid and chemotherapy is associated with a high cure rate in clinical trials. As some patients are not enrolled in these trials due to early severe events, these results might be overestimated. To address this issue, we reviewed all APL patients referred to the Hospital Saint-Louis within the 2000-2010 period, with a special focus on inclusion in recruiting trials. PATIENTS AND METHODS: One hundred patients (including eight children) with newly diagnosed APL were admitted during this period, which covered two consecutive APL trials conducted by the French-Belgian-Swiss APL group. RESULTS: The rate of patients not enrolled within recruiting trials was 29%. The main reason for non-inclusion was protocol ineligibility related to disease severity at diagnosis. Non-enrolled patients more frequently had white blood cell count (WBC) . or =50×10(9)/L (31% versus 8%; p=.01), platelet count<40×10(9)/L (97% versus 65%; p=.001) and microgranular variant APL (38% versus 11%; p=.004) and were more frequently admitted in intensive care unit during induction (41% versus 24%; p=.094). Early mortality rate was higher in non-enrolled patients (21% versus 3%; p=.007), translating into a lower complete remission rate (79% versus 96%; p=.007) and lower event-free survival (65% versus 84% at 5 years; p=.05), while disease-free survival was similar in both non-enrolled and enrolled patient groups (81% versus 88% at 5 years; p=.68). CONCLUSION: Initial APL severity leads to a significant proportion of patients non-registered within clinical trials, which may underestimate the real early mortality, which remained nonetheless less than 10% in this study.
PURPOSE:Acute promyelocytic leukaemia (APL) therapy with all-transretinoic acid and chemotherapy is associated with a high cure rate in clinical trials. As some patients are not enrolled in these trials due to early severe events, these results might be overestimated. To address this issue, we reviewed all APL patients referred to the Hospital Saint-Louis within the 2000-2010 period, with a special focus on inclusion in recruiting trials. PATIENTS AND METHODS: One hundred patients (including eight children) with newly diagnosed APL were admitted during this period, which covered two consecutive APL trials conducted by the French-Belgian-Swiss APL group. RESULTS: The rate of patients not enrolled within recruiting trials was 29%. The main reason for non-inclusion was protocol ineligibility related to disease severity at diagnosis. Non-enrolled patients more frequently had white blood cell count (WBC) . or =50×10(9)/L (31% versus 8%; p=.01), platelet count<40×10(9)/L (97% versus 65%; p=.001) and microgranular variant APL (38% versus 11%; p=.004) and were more frequently admitted in intensive care unit during induction (41% versus 24%; p=.094). Early mortality rate was higher in non-enrolled patients (21% versus 3%; p=.007), translating into a lower complete remission rate (79% versus 96%; p=.007) and lower event-free survival (65% versus 84% at 5 years; p=.05), while disease-free survival was similar in both non-enrolled and enrolled patient groups (81% versus 88% at 5 years; p=.68). CONCLUSION: Initial APL severity leads to a significant proportion of patients non-registered within clinical trials, which may underestimate the real early mortality, which remained nonetheless less than 10% in this study.
Authors: Sylvie Dunoyer-Geindre; Anne-Sophie Rivier-Cordey; Olga Tsopra; Thomas Lecompte; Egbert K O Kruithof Journal: Ann Hematol Date: 2017-03-25 Impact factor: 3.673
Authors: Karsten Wendt; Jan Moritz Middeke; Jan-Niklas Eckardt; Tim Schmittmann; Sebastian Riechert; Michael Kramer; Anas Shekh Sulaiman; Katja Sockel; Frank Kroschinsky; Johannes Schetelig; Lisa Wagenführ; Ulrich Schuler; Uwe Platzbecker; Christian Thiede; Friedrich Stölzel; Christoph Röllig; Martin Bornhäuser Journal: BMC Cancer Date: 2022-02-22 Impact factor: 4.430