Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: a EUROCORD, EBMT, EWOG-MDS, CIBMTR study.

Abstract

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.

• Authors: Franco Locatelli, Alessandro Crotta, Annalisa Ruggeri, Mary Eapen, John E Wagner, Margaret L Macmillan, Marco Zecca, Joanne Kurtzberg, Carmem Bonfim, Ajay Vora, Cristina Díaz de Heredia, Lochie Teague, Jerry Stein, Tracey A O'Brien, Henrique Bittencourt, Adrienne Madureira, Brigitte Strahm, Christina Peters, Charlotte Niemeyer, Eliane Gluckman, Vanderson Rocha
• Journal: Blood (Blood) Vol. 122 Issue 12 Pg. 2135-41 (Sep 19 2013) ISSN: 1528-0020 [Electronic] United States
• PMID: 23926304 (Publication Type: Journal Article, Multicenter Study, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)

Topics

• Cause of Death
• Child
• Child, Preschool
• Cord Blood Stem Cell Transplantation (adverse effects)
• Female
• Graft vs Host Disease (etiology)
• Humans
• Infant
• Leukemia, Myelomonocytic, Juvenile (epidemiology, mortality, therapy)
• Male
• Recurrence
• Registries
• Retrospective Studies
• Risk Factors
• Treatment Outcome