TY - JOUR
T1 - Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia
T2 - A EUROCORD, EBMT, EWOG-MDS,CIBMTR study
AU - Locatelli, Franco
AU - Crotta, Alessandro
AU - Ruggeri, Annalisa
AU - Eapen, Mary
AU - Wagner, John E.
AU - MacMillan, Margaret L.
AU - Zecca, Marco
AU - Kurtzberg, Joanne
AU - Bonfim, Carmem
AU - Vora, Ajay
AU - De Heredia, Cristina Díaz
AU - Teague, Lochie
AU - Stein, Jerry
AU - O'Brien, Tracey A.
AU - Bittencourt, Henrique
AU - Madureira, Adrienne
AU - Strahm, Brigitte
AU - Peters, Christina
AU - Niemeyer, Charlotte
AU - Gluckman, Eliane
AU - Rocha, Vanderson
PY - 2013/9/19
Y1 - 2013/9/19
N2 - 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 × 107/kg (range, 1.7-27.6 × 107/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.
AB - 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 × 107/kg (range, 1.7-27.6 × 107/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.
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U2 - 10.1182/blood-2013-03-491589
DO - 10.1182/blood-2013-03-491589
M3 - Article
C2 - 23926304
AN - SCOPUS:84887600939
SN - 0006-4971
VL - 122
SP - 2135
EP - 2141
JO - Blood
JF - Blood
IS - 12
ER -