Journal
BLOOD
Volume 122, Issue 12, Pages 2135-2141Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2013-03-491589
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Funding
- AIRC (Associazione Italiana Ricerca sul Cancro)
- CNR(Consiglio Nazionale delle Ricerche)
- MIUR (Ministero dell'Istruzione, Universita e della Ricerca, Progetto Rilevante Interesse Nazionale)
- IRCCS Ospedale Pediatrico Bambino Gesu
- Public Health Service grant from the National Institutes of Health National Cancer Institute [U24-CA76518]
- National Heart, Lung, and Blood Institute
- National Institute of Allergy and Infectious Diseases
- National Institute of Health Research-Biomedical Research Centres
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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 HLAdisparities, respectively. The median number of nucleated cells infused was 7.1x10(7)/kg (range, 1.7-27.6x10(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.
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