Outcome of childhood acute promyelocytic leukemia with all-trans-retinoic acid and chemotherapy.

Abstract

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.

• Authors: 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
• Journal: Journal of clinical oncology : official journal of the American Society of Clinical Oncology (J Clin Oncol) Vol. 22 Issue 8 Pg. 1404-12 (Apr 15 2004) ISSN: 0732-183X [Print] United States
• PMID: 15084614 (Publication Type: Clinical Trial, Comparative Study, Journal Article, Randomized Controlled Trial)

Chemical References

• Cytarabine
• Tretinoin
• Daunorubicin

Topics

• Adolescent
• Adult
• Antineoplastic Combined Chemotherapy Protocols (therapeutic use)
• Child
• Child, Preschool
• Cytarabine (therapeutic use)
• Daunorubicin (therapeutic use)
• Female
• Humans
• Infant
• Leukemia, Promyelocytic, Acute (drug therapy)
• Male
• Middle Aged
• Treatment Outcome
• Tretinoin (therapeutic use)