Journal of Clinical Oncology, Vol 22, No 8 (April 15), 2004: pp. 1404-1412
© 2004 American Society of Clinical Oncology.
DOI: 10.1200/JCO.2004.09.008
Outcome of Childhood Acute Promyelocytic Leukemia With All-Trans-Retinoic Acid and Chemotherapy
S. de Botton,
V. Coiteux,
S. Chevret,
C. Rayon,
E. Vilmer,
M. Sanz,
J. de La Serna,
N. Philippe,
A. Baruchel,
G. Leverger,
A. Robert,
J. San Miguel,
E. Conde,
J.J. Sotto,
D. Bordessoule,
N. Fegueux,
M. Fey,
A. Parry,
C. Chomienne,
L. Degos,
P. Fenaux
Address reprint requests to P. Fenaux, MD, PhD, Service dHématologie Clinique, Hôpital Avicenne, 125 route de Stalingrad, 93009 Bobigny, France; e-mail: pierre.fenaux{at}avc.ap-hop-paris.fr
PURPOSE: To determine the results of treatment combining all-trans-retinoic acid (ATRA) and chemotherapy (CT) in childhood acute promyelocytic leukemia (APL).
PATIENTS AND METHODS: Children (< 18 years) with newly diagnosed APL were included in the APL93 trial, treated by ATRA followed or combined with daunorubicin-cytarabine, and then randomly assigned between no maintenance, intermittent ATRA, continuous CT, or both.
RESULTS: Of the 576 patients included in APL93 trial, 31 (5%) were children, including 22 girls (71%) and nine boys (29%). Thirty of the children (97%) obtained complete remission (CR). ATRA syndrome occurred in four children (13%), who all achieved CR, and headaches occurred in 12 children (39%), with signs of pseudotumor cerebri in five children (16%). Seven patients (23%) relapsed. None of the eight patients who received both ATRA and CT for maintenance relapsed. All relapsing patients achieved a second CR. Twenty-two patients remained in first CR after 43+ to 96+ months, six remained in second CR after 17+ to 66+ months, and three patients had died. The 5-year event-free survival (EFS), relapse, and overall survival rates were 71%, 27%, and 90%, respectively. No difference between adults and children included in the APL93 trial was seen for CR rate, 5-year relapse rate, EFS, and overall survival, but significantly better survival was seen in children after adjustment on WBC counts (P = .02) and incidence of microgranular M3 variant (P = .04).
CONCLUSION: ATRA combined with CT for induction and also probably for maintenance provides as favorable results in children with APL as in adults and currently constitutes the reference first-line treatment in both age groups.
A complete listing of participants may be found in the online-only Appendix.
From the European APL Group.
Supported by the Programme Hospitalier de Recherche Clinique (Centre Hospitalier Universitaire Lille), the Association de Recherche contre le Cancer, and the Ligue Nationale contre le Cancer (Comité du Nord).
S.d.B. and V.C. contributed equally to this work.
Authors disclosures of potential conflicts of interest are found at the end of this article.

CiteULike Complore Connotea Del.icio.us Digg Facebook Reddit Technorati Twitter What's this?
This article has been cited by other articles:

|
 |

|
 |
 
J. Zhou, Y. Zhang, J. Li, X. Li, J. Hou, Y. Zhao, X. Liu, X. Han, L. Hu, S. Wang, et al.
Single-agent arsenic trioxide in the treatment of children with newly diagnosed acute promyelocytic leukemia
Blood,
March 4, 2010;
115(9):
1697 - 1702.
[Abstract]
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
M. A. Sanz, D. Grimwade, M. S. Tallman, B. Lowenberg, P. Fenaux, E. H. Estey, T. Naoe, E. Lengfelder, T. Buchner, H. Dohner, et al.
Management of acute promyelocytic leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet
Blood,
February 26, 2009;
113(9):
1875 - 1891.
[Abstract]
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
A. S. Wayne, G. H. Reaman, and L. J. Helman
Progress in the Curative Treatment of Childhood Hematologic Malignancies
J Natl Cancer Inst,
September 17, 2008;
100(18):
1271 - 1273.
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
H. Lapillonne, A. Renneville, A. Auvrignon, C. Flamant, A. Blaise, C. Perot, J.-L. Lai, P. Ballerini, F. Mazingue, S. Fasola, et al.
High WT1 Expression After Induction Therapy Predicts High Risk of Relapse and Death in Pediatric Acute Myeloid Leukemia
J. Clin. Oncol.,
April 1, 2006;
24(10):
1507 - 1515.
[Abstract]
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
M. A. Sanz, M. S. Tallman, and F. Lo-Coco
Practice Points, Consensus, and Controversial Issues in the Management of Patients with Newly Diagnosed Acute Promyelocytic Leukemia
Oncologist,
November 1, 2005;
10(10):
806 - 814.
[Abstract]
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
J. J. Ortega, L. Madero, G. Martin, A. Verdeguer, P. Garcia, R. Parody, J. Fuster, A. Molines, A. Novo, G. Deben, et al.
Treatment With All-Trans Retinoic Acid and Anthracycline Monochemotherapy for Children With Acute Promyelocytic Leukemia: A Multicenter Study by the PETHEMA Group
J. Clin. Oncol.,
October 20, 2005;
23(30):
7632 - 7640.
[Abstract]
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
A. M. Testi, A. Biondi, F. L. Coco, M. L. Moleti, F. Giona, M. Vignetti, G. Menna, F. Locatelli, A. Pession, E. Barisone, et al.
GIMEMA-AIEOPAIDA protocol for the treatment of newly diagnosed acute promyelocytic leukemia (APL) in children
Blood,
July 15, 2005;
106(2):
447 - 453.
[Abstract]
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
M. A. Sanz, M. S. Tallman, and F. Lo-Coco
Tricks of the trade for the appropriate management of newly diagnosed acute promyelocytic leukemia
Blood,
April 15, 2005;
105(8):
3019 - 3025.
[Abstract]
[Full Text]
[PDF]
|
 |
|

|
 |

|
 |
 
C.-H. Pui, M. Schrappe, R. C. Ribeiro, and C. M. Niemeyer
Childhood and Adolescent Lymphoid and Myeloid Leukemia
Hematology,
January 1, 2004;
2004(1):
118 - 145.
[Abstract]
[Full Text]
[PDF]
|
 |
|
|