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IKZF1 status as a prognostic feature in BCR-ABL1-positive childhood ALL

Lookup NU author(s): Professor Christine Harrison FRCPath FMedSci


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Childhood BCR-ABL1-positive B-cell precursor acute lymphoblastic leukemia (BCP-ALL) has an unfavorable outcome and shows high frequency of IKZF1 deletions. The prognostic value of IKZF1 deletions was evaluated in 2 cohorts of BCR-ABL1-positive BCP-ALL patients, before tyrosine kinase inhibitors (pre-TKI) and after introduction of imatinib (in the European Study for Philadelphia-Acute Lymphoblastic Leukemia [EsPhALL]). In 126/191 (66%) cases an IKZF1 deletion was detected. In the pre-TKI cohort, IKZF1-deleted patients had an unfavorable outcome compared with wild-type patients (4-year disease-free survival [DFS] of 30.0 +/- 6.8% vs 57.5 +/- 9.4%; P = .01). In the EsPhALL cohort, the IKZF1 deletions were associated with an unfavorable prognosis in patients stratified in the good-risk arm based on early clinical response (4-year DFS of 51.9 +/- 8.8% for IKZF1-deleted vs 78.6 +/- 13.9% for IKZF1 wild-type; P = .03), even when treated with imatinib (4-year DFS of 55.5 +/- 9.5% for IKZF1-deleted vs 75.0 +/- 21.7% for IKZF1 wild-type; P = .05). In conclusion, the highly unfavorable outcome for childhood BCR-ABL1-positive BCP-ALL with IKZF1 deletions, irrespective of imatinib exposure, underscores the need for alternative therapies. In contrast, good-risk patients with IKZF1 wild-type responded remarkably well to imatinib-containing regimens, providing a rationale to potentially avoid hematopoietic stem-cell transplantation in this subset of patients.

Publication metadata

Author(s): van der Veer A, Zaliova M, Mottadelli F, De Lorenzo P, te Kronnie G, Harrison CJ, Cave H, Trka J, Saha V, Schrappe M, Pieters R, Biondi A, Valsecchi MG, Stanulla M, den Boer ML, Cazzaniga G

Publication type: Article

Publication status: Published

Journal: Blood

Year: 2014

Volume: 123

Issue: 11

Pages: 1691-1698

Print publication date: 01/01/2014

Online publication date: 23/12/2013

Acceptance date: 15/12/2013

ISSN (print): 0006-4971

ISSN (electronic): 1528-0020

Publisher: American Society of Hematology


DOI: 10.1182/blood-2013-06-509794


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Funder referenceFunder name
Italian Association for Cancer Research
Fondazione Cariplo
Italian Ministry of University and Research
Pediatric Oncology Foundation Rotterdam
00064203Ministry of Health, Czech Republic
658Sophia Foundation for Scientific Research
EMCR 2007-3718Dutch Cancer Society
HEALTH-F2-2011-261474European Union under the European Network of Cancer Research in Children and Adolescents project
P302/12/G101GACR Centre of Excellence
UNCE204012University Hospital Motol, Prague, Czech Republic