Risk factors for outcome after allogeneic stem cell transplantation in patients with advanced phase CML

• Published in: Bone marrow transplantation (Basingstoke) 2021-11, Vol.56 (11), p.2834-2841
• Main Authors: Niederwieser, Christian, Morozova, Elena, Zubarovskaya, Ludmila, Zabelina, Tatjana, Klyuchnikov, Evgeny, Janson, Dietlinde, Wolschke, Christine, Christopeit, Maximilian, Ayuk, Francis, Moiseev, Ivan, Afanasyev, Boris V., Kröger, Nicolaus
• Format: Article
• Language: English
• Subjects: 13/100; 631/250/1904; 631/532/1542; 692/308/2779/109/2154; 692/499; 692/699/67/1990/283/1896; Adolescent; Adult; Age; Aged; Blast Crisis; Care and treatment; CD34 antigen; Cell Biology; Child; Child, Preschool; Chronic Disease; Chronic myeloid leukemia; Hematology; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic stem cells; Homografts; Humans; Internal Medicine; Leukemia; Leukemia, Myelogenous, Chronic, BCR-ABL Positive - therapy; Medical prognosis; Medicine; Medicine & Public Health; Middle Aged; Myeloid leukemia; Patient outcomes; Public Health; Retrospective Studies; Risk analysis; Risk Factors; Stem cell transplantation; Stem Cells; Survival; Transplantation; Transplantation Conditioning - methods; Transplants & implants; Young Adult
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/s41409-021-01410-x

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.