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Blood, 29 October 2009, Vol. 114, No. 18, pp. 3764-3768. Prepublished online as a Blood First Edition Paper on August 5, 2009; DOI 10.1182/blood-2009-02-204214.
CLINICAL TRIALS AND OBSERVATIONS Outcome of congenital acute lymphoblastic leukemia treated on the Interfant-99 protocol1 Erasmus MC-Sophia Children's Hospital, Rotterdam, The Netherlands; 2 Department of Clinical Medicine and Prevention, University of Milano-Bicocca, Milan, Italy; 3 University Medical Center Schleswig-Holstein, Kiel, Germany; 4 Cooperative Study Group for Treatment of ALL, University Hospital Hamburg-Eppendorf, Hamburg, Germany; 5 French ALL Group, Hôpital Saint-Louis, Paris, France; 6 Argentina Hospital de Pediatría, Buenos Aires, Argentina; 7 Associazione Italiana Ematologia Oncologia Pediatrica, University of Milano-Bicocca, San Gerardo Hospital, Monza, Italy; 8 Programa Infantil Nacional de Drogas Antineoplásicas, Hospital Roberto del Rio, Santiago, Chile; 9 UK Children's Cancer Study Group, Great Ormond Street Hospital for Children, London, United Kingdom; 10 St Jude Children's Research Hospital, Memphis, TN; 11 Czech Paediatric Haematology, University Hospital Motol and 2nd Medical School, Charles University Prague, Prague, Czech Republic; 12 Polish Paediatric Leukemia and Lymphoma Study Group, Medical University of Silesia, Zabrze, Poland; 13 Sheffield Children's Hospital, Sheffield, United Kingdom; 14 Children's Leukemia Group, Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium; 15 Nordic Society of Paediatric Haematology and Oncology, University of Helsinki, Helsinki, Finland; 16 Dana-Farber Cancer Institute, Boston, MA; and 17 Dutch Childhood Oncology Group, The Hague, The Netherlands Acute lymphoblastic leukemia (ALL) diagnosed in the first month of life (congenital ALL) is very rare. Although congenital ALL is often assumed to be fatal, no studies have been published on outcome except for case reports. The present study reports the outcome of 30 patients with congenital ALL treated with the uniform Interfant-99 protocol, a hybrid regimen combining ALL treatment with elements designed for treatment of acute myeloid leukemia. Congenital ALL was characterized by a higher white blood cell count and a strong trend for higher incidence of MLL rearrangements and CD10-negative B-lineage ALL compared with older infants. Induction failure rate was 13% and not significantly different from that in older infants (7%, P = .14), but relapse rate was significantly higher in congenital ALL patients (2-year cumulative incidence [SE] was 60.0 [9.3] vs 34.2 [2.3], P < .001). Two-year event-free survival and survival of congenital ALL patients treated with this protocol was 20% (SE 9.1%). Early death in complete remission and treatment delays resulting from toxicity were not different. The survival of 17% after last follow-up, combined with a toxicity profile comparable with that in older infants, justifies treating congenital ALL with curative intent. This trial was registered at www.clinicaltrials.gov as no. NCT 00015873, and at www.controlled-trials.com as no. ISRCTN24251487 [controlled-trials.com] .
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