Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: a EUROCORD, EBMT, EWOG-MDS, CIBMTR study.
We retrospectively analyzed 110 patients with juvenile myelomonocytic leukemia, given single-unit, unrelated donor umbilical cord blood transplantation. Median age at diagnosis and at transplantation was 1.4 years (age range, 0.1-6.4 years) and 2.2 years (age range, 0.5-7.4 years), respectively. Before transplantation, 88 patients received chemotherapy; splenectomy was performed in 24 patients. Monosomy of chromosome 7 was the most frequent cytogenetic abnormality, found in 24% of patients. All but 8 patients received myeloablative conditioning; cyclosporine plus steroids was the most common graft-versus-host disease prophylaxis. Sixteen percent of units were HLA-matched with the recipient, whereas 43% and 35% had either 1 or 2 to 3 HLA disparities, respectively. The median number of nucleated cells infused was 7.1 × 10(7)/kg (range, 1.7-27.6 × 10(7)/kg). With a median follow-up of 64 months (range, 14-174 months), the 5-year cumulative incidences of transplantation-related mortality and relapse were 22% and 33%, respectively. The 5-year disease-free survival rate was 44%. In multivariate analysis, factors predicting better disease-free survival were age younger than 1.4 years at diagnosis (hazard ratio [HR], 0.42; P = .005), 0 to 1 HLA disparities in the donor/recipient pair (HR, 0.4; P = .009), and karyotype other than monosomy 7 (HR, 0.5; P = .02). Umbilical cord blood transplantation may cure a relevant proportion of children with juvenile myelomonocytic leukemia. Because disease recurrence remains the major cause of treatment failure, strategies to reduce incidence of relapse are warranted.
Department of Pediatric Hematology and Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale Bambino Gesù, Università di Pavia, Rome, Italy;
Franco
F
Locatelli
Alessandro
A
Crotta
Annalisa
A
Ruggeri
Mary
M
Eapen
John E
JE
Wagner
Margaret L
ML
Macmillan
Marco
M
Zecca
Joanne
J
Kurtzberg
Carmem
C
Bonfim
Ajay
A
Vora
Cristina
C
Díaz de Heredia
Lochie
L
Teague
Jerry
J
Stein
Tracey A
TA
O'Brien
Henrique
H
Bittencourt
Adrienne
A
Madureira
Brigitte
B
Strahm
Christina
C
Peters
Charlotte
C
Niemeyer
Eliane
E
Gluckman
Vanderson
V
Rocha
D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D007223:Infant; D054429:Leukemia, Myelomonocytic, Juvenile / Q000453:epidemiology* / Q000401:mortality / Q000628:therapy*; D008297:Male; D012008:Recurrence; D012042:Registries; D012189:Retrospective Studies; D012307:Risk Factors; D016896:Treatment Outcome