Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies

Factors influencing the outcome for 39 children with haematological maligna ncy who were subjected to a cord blood transplantation (CBT) from genotypic ally HLA-mismatched unrelated donors were analysed. This retrospective stud y included 21 children with acute lymphoblastic leukaemia, 15 with acute my elogenous leukaemia and one each with chronic myelogenous leukaemia, refrac tory anaemia with myelodysplastic syndrome (MDS) and juvenile myelomonocyti c leukaemia (JMML), Those subjected to CBT during the first or second compl ete remission (CR) and MDS without blasts were assigned to the standard-ris k (SR) group (n = 16), Patients in third or subsequent remission, relapse o r partial remission with refractory leukaemia at the time of CBT were consi dered to be in advanced phase, and placed in the high-risk (IIR) group (n = 11), JMML and the second CR after a relapse (n = 8), or bone marrow failur e after a rejection (n = 3), following haematopoietic stem cell transplanta tion (HSCT) in the first CR were included in the high-risk group, Kaplan-Me ier estimates for neutrophil and platelet recovery were 83.7 +/- 12.2 at d 60 and 55.4 +/- 16.6% at d 100 respectively. The incidence of grades ii-Vt acute graft-versus-host disease was 58.5 +/- 16.8%. The Kaplan-Meier estima te for 3-year event-free survival (EFS) was 49.2 +/- 16.6. From multivariat e analysis, the most important factor influencing EFS was disease status at CBT: SR patients had 3-year EFS of 75.0 +/- 21.6%, compared with 29.6 +/- 20.6% for those with HR disease (P = 0.013, RR 4.746, 95% CI 1.382-16.298). These data confirm that HLA-mismatched, unrelated CBT is a feasible proced ure to cure a significant proportion of children with leukaemia, especially if conducted in a favourable phase of the disease.