BACKGROUND: Patients with primary refractory acute lymphoblastic leukemia (PREF ALL) who fail to achieve a complete remission
(CR) after 2 courses of chemotherapy have a dismal prognosis without undergoing allogeneic hematopoietic cell transplantation
(HCT). To the authors’ knowledge, there currently are no data regarding factors influencing transplantation outcomes. METHODS: The
authors retrospectively studied outcomes of transplantation for cases of PREF ALL reported to European Society for Blood and
Marrow Transplantation registry. Eligibility criteria for the current analysis included adult patients who underwent their first HCT for
PREF ALL between 2000 and 2012. PREF disease was defined as the failure to achieve a morphological CR after 2 courses of induction
chemotherapy. RESULTS: Data regarding 86 adult patients were analyzed. With a median follow-up of 106 months, the probability
of survival was 36% at 2 years and 23% at 5 years. The probability of leukemia-free survival was 28% and 17%, respectively, and the
probability of nonrecurrence mortality was 20% and 29%, respectively, at 2 years and 5 years. For 66 patients who achieved a CR
(77%), the survival at 2 years and 5 years was 36% and 29%, respectively. In multivariate analysis, use of total body irradiation was
found to be associated with improved survival. Total body irradiation and infusion of female hematopoietic cells into male recipients
was associated with improved leukemia-free survival. These findings were incorporated into a scoring system that identified 3 groups
(those with 2, 1, or no prognostic factors) with survival rates of 57%, 22%, and 8%, respectively. CONCLUSIONS: Although overall these
patients would clearly benefit from the introduction of novel antileukemic therapies, the data from the current study support the use
of allogeneic HCT in selected patients with PREF ALL.