Idelalisib treatment prior to allogeneic stem cell transplantation for patients with chronic lymphocytic leukemia: a report from the EBMT chronic malignancies working party

• Published in: Bone marrow transplantation (Basingstoke) Vol. 56; no. 3; pp. 605 - 613
• Main Authors: Schetelig, Johannes, Chevallier, Patrice, van Gelder, Michel, Hoek, Jennifer, Hermine, Olivier, Chakraverty, Ronjon, Browne, Paul, Milpied, Noel, Malagola, Michele, Socié, Gerard, Delgado, Julio, Deconinck, Eric, Damaj, Ghandi, Maury, Sebastian, Beelen, Dietrich, Quoc, Stéphanie Nguyen, Shankara, Paneesha, Brecht, Arne, Mayer, Jiri, Hunault-Berger, Mathilde, Bittenbring, Jörg, Thieblemont, Catherine, Lepretre, Stéphane, Baldauf, Henning, de Wreede, Liesbeth C., Tournilhac, Olivier, Yakoub-Agha, Ibrahim, Kröger, Nicolaus, Dreger, Peter
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.03.2021; Nature Publishing Group
• Subjects: 692/699/1541/1990/283/1895; 692/700/565/2319; 692/700/565/251; Care and treatment; Cell Biology; Chronic lymphocytic leukemia; Graft rejection; Graft vs Host Disease; Graft-versus-host reaction; Grafts; Hematology; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Homografts; Human health and pathology; Humans; Internal Medicine; Leukemia; Leukemia, Lymphocytic, Chronic, B-Cell - drug therapy; Life Sciences; Lymphatic leukemia; Medicine; Medicine & Public Health; Middle Aged; Patient outcomes; Patients; Public Health; Purines; Quinazolinones; Retrospective Studies; Risk analysis; Risk factors; Stem cell transplantation; Stem Cells; Survival; Transplantation; Transplantation Conditioning; Germany
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/s41409-020-01069-w

No studies have been reported so far on bridging treatment with idelalisib for patients with chronic lymphocytic leukemia (CLL) prior to allogeneic hematopoietic cell transplantation (alloHCT). To study potential carry-over effects of idelalisib and to assess the impact of pathway-inhibitor (PI) failure we performed a retrospective EBMT registry-based study. Patients with CLL who had a history of idelalisib treatment and received a first alloHCT between 2015 and 2017 were eligible. Data on 72 patients (median age 58 years) were analyzed. Forty percent of patients had TP53 mut/del CLL and 64% had failed on at least one PI. No primary graft failure occurred. Cumulative incidences of acute GVHD °II–IV and chronic GVHD were 51% and 39%, respectively. Estimates for 2-year overall survival (OS), progression-free survival (PFS), and cumulative incidences of relapse/progression (CIR) and non-relapse mortality NRM were 59%, 44%, 25%, and 31%. In univariate analysis, drug sensitivity was a strong risk factor. For patients who had failed neither PI treatment nor chemoimmunotherapy (CIT) the corresponding 2-year estimates were 73%, 65%, 15%, and 20%, respectively. In conclusion, idelalisib may be considered as an option for bridging therapy prior to alloHCT. Owing to the high risk for acute GVHD intensified clinical monitoring is warranted.