Outcome of congenital acute lymphoblastic leukemia treated on the Interfant-99 protocol.
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.
van der Linden
Marieke H
MH
Erasmus MC-Sophia Children's Hospital, Rotterdam, The Netherlands.
Valsecchi
Maria Grazia
MG
De Lorenzo
Paola
P
Möricke
Anja
A
Janka
Gritta
G
Leblanc
Thierry M
TM
Felice
Maria
M
Biondi
Andrea
A
Campbell
Myriam
M
Hann
Ian
I
Rubnitz
Jeffrey E
JE
Stary
Jan
J
Szczepanski
Tomasz
T
Vora
Ajay
A
Ferster
Alina
A
Hovi
Liisa
L
Silverman
Lewis B
LB
Pieters
Rob
R
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000009:adverse effects; D001402:B-Lymphocytes; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D015321:Gene Rearrangement; D011495:Histone-Lysine N-Methyltransferase; D006801:Humans; D007231:Infant, Newborn; D007958:Leukocyte Count; D008297:Male; D051788:Myeloid-Lymphoid Leukemia Protein / Q000235:genetics; D015260:Neprilysin; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000097:blood / Q000188:drug therapy* / Q000235:genetics / Q000401:mortality; D012008:Recurrence; D012074:Remission Induction; D015996:Survival Rate
C497422:KMT2A protein, human; D051788:Myeloid-Lymphoid Leukemia Protein; D011495:Histone-Lysine N-Methyltransferase; D015260:Neprilysin