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


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 TP53mut/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.

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Fig. 1: Outcome after alloHCT for patients with CLL who have been exposed to idelalisib.
Fig. 2: Univariable comparisons of progression-free survival for selected risk factors were done by log-rank tests.


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We wish to thank the patients and their families, the local data managers, and physicians and the staff at the EBMT Data Office in Leiden, who made this study possible.

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Correspondence to Johannes Schetelig.

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Conflict of interest

JS—consultancy honoraria from AstraZeneca, Janssen, Roche, Gilead, Abbvie, Sanofi, Molmed; lecturer fees from AstraZeneca, Janssen, Roche, Gilead, Abbvie, Sanofi, Novartis; research support from Genzyme, Sanofi, GSK, Novartis, Abbvie. OH—consultancy honoraria from AB Science; research support from AB Science, Celgene, Alexion, and Inatherys. DG—consultancy honoraria from Roche, Takeda, Iqone; lecturer fees from Abbvie, Celgene, Sanofi-Genzyme; research support from Takeda. NK—lecturer fees from Neovii, Sanofi, Novartis, Kiadis, Celgene, Amgen, Riemser, Chugai, Janssen; research support from Novartis, Celgene, Riemser, Neovii. PD—consultancy for AbbVie, AstraZeneca, Gilead, Janssen, Novartis, Riemser, Roche; speakers bureau for AbbVie, Gilead, Novartis, Riemser, Roche; research support from Neovii and Riemser. The remaining authors declared no conflict of interest.

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Schetelig, J., Chevallier, P., van Gelder, M. et al. Idelalisib treatment prior to allogeneic stem cell transplantation for patients with chronic lymphocytic leukemia: a report from the EBMT chronic malignancies working party. Bone Marrow Transplant (2020).

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