Abstract
Allogeneic hematopoietic stem-cell transplantation (HSCT) remains the only curative option for patients with advanced chronic myeloid leukemia (CML). However, outcome is dismal and of short follow-up. The objective of the study was to determine long-term outcome and risk factors in patients with a history of CML Blast Crisis (BC; n = 96) or accelerated phase (n = 51) transplanted between 1990 and 2018. At transplant, patients had a median age of 39 (range 7–76) years and were in ≥CP2 (n = 70), in AP (n = 40) or in BC (n = 37) with a diagnosis-HSCT interval of median 1.9 (range 0.3–24.4) years. Overall survival (OS) amounted 34% (95% CI 22–46) and progression-free survival (PFS) 26% (95% CI 16-36) at 15 years. Adverse risk factors for OS and PFS were low CD34+ count in the graft, donor age (>36 years) and BC. Cumulative incidence of Non-Relapse Mortality (NRM) was 28% (95% CI 18–38) and of relapse (RI) 43% (95% CI 33–53) at 15 years. PB-HSCT and HSCT after 2008 were favorable prognostic factors for NRM, while family donor and patient age >39 years were independently associated with higher RI. HSCT resulted in long-term OS in patients with advanced CML. OS was improved in non-BC patients, with donors ≤36 years and with higher CD34+ dose in the graft.
Highlights
The introduction of tyrosine kinase inhibitors (TKI) for BCR/ABL in patients with chronic phase (CP) chronic myeloid leukemia (CML) resulted for the first time in regular disappearance of the malignant clone, restoration of normal life expectancy, and even treatment free remission
At the time of hematopoietic stem-cell transplantation (HSCT) patients were in CP ≥ 2 (n = 70), accelerated phase (AP) (n = 40), or blast crisis (BC) (n = 37)
The majority were treated with TKI (81.5%) before HSCT and the median diagnosis-HSCT interval amounted to 1.9 years
Summary
The introduction of tyrosine kinase inhibitors (TKI) for BCR/ABL in patients with chronic phase (CP) chronic myeloid leukemia (CML) resulted for the first time in regular disappearance of the malignant clone, restoration of normal life expectancy, and even treatment free remission. The outcome of patients with TKI resistance or of those who present with advanced disease is still dismal. In such cases, the median overall survival (OS) with first and further TKI generations is less than 12 months [1,2,3,4,5]. The purpose of the study was to evaluate long-term results (over 15 years) on a large number of patients with advanced CML and analyze risk factors for outcome. We analyzed outcome and risk factors in 147 patients with advanced disease (BC, AP and CP ≥ 2) transplanted in two centers with a follow-up period of up to 15 years.
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