Haploidentical versus autologous transplants in acute leukemia haematologica | 2015; 100(4) 559 transplantation, although correlated with a lower relapse incidence, remains associated with a higher incidence of non-relapse mortality, GVHD, and infections. In addition, allogeneic transplant survivors tend to have a poorer quality of life than that of patients who undergo ASCT.14 As it stands, allogeneic stem cell transplantation with a genoidentical donor is associated with the best outcome but there has been no randomized or retrospective study showing the superiority of allogeneic transplantation using an alternative donor compared to ASCT in AML. Data in ALL are more limited.In order to compare the outcome of adult patients following haploidentical and autologous transplants, we collected information available in the European Society for Blood and Marrow Transplantation (EBMT) registry, after January 2007 (when T-cell-replete haploidentical transplants were first reported) and conducted a matched pair analysis.
MethodsThe study design was approved by the Acute Leukemia Working Party, in accordance with the EBMT guidelines for retrospective studies. In the period between January 2007 and December 2012, 2259 ASCT and 234 T-cell-replete haploidentical stem cell transplants were reported to the EBMT registry as first transplants to treat adult patients with either AML or ALL in CR1 or CR2.For the matched pair analysis, we used as matching factors: age ± 5 years, diagnosis (AML, ALL), the status at transplant (CR1, CR2), the interval from diagnosis to transplant (CR1: less than or greater than 6 months; CR2: less than or greater than 18 months), and cytogenetic status. We identified 188 haploidentical transplants and 356 ASCT.
Endpoint definitions and statistical analysisFour outcomes were evaluated: (i) non-relapse mortality, defined as death without previous relapse; (ii) relapse incidence, defined on the basis of morphological evidence of leukemia in bone marrow or other extramedullary organs; (iii) leukemia-free survival, defined as the time from transplantation to first event (either relapse or death in complete remission); and (iv) overall survival. Cumulative incidence curves were used for relapse incidence and non-relapse mortality in a competing risks setting, since death and relapse are competing.15 Probabilities of overall survival and leukemia-free survival were calculated using the Kaplan-Meier estimate. 16 Within matched groups, associations were accounted for by a random effect common to both members from the same group using a Cox proportional hazards model.All tests were two-sided with the type I error rate fixed at 0.05. Statistical analyses were performed with SPSS 19 (SPSS Inc., Chicago, IL, USA), and R 3.0.1 (R Development Core Team, Vienna, Austria) software packages.Since haploidentical transplantation is a new, evolving transplant modality, we hypothesized the possibility of a center or a learning effect and compared the outcome of patients transplanted in "haplo expert" versus "regu...