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Tile: Therapeutic Repurposing of Venetoclax in Haematological Malignancies | Authorea try { document.documentElement.classList.add('js'); } catch (e) { } var _gaq = _gaq || []; _gaq.push(['_setAccount', 'G-8VDV14Y67G']); _gaq.push(['_trackPageview']); (function() { var ga = document.createElement('script'); ga.type = 'text/javascript'; ga.async = true; ga.src = ('https:' == document.location.protocol ? 'https://ssl' : 'http://www') + '.google-analytics.com/ga.js'; var s = document.getElementsByTagName('script')[0]; s.parentNode.insertBefore(ga, s); })(); Skip to main content Preprints Collections Wiley Open Research IET Open Research Ecological Society of Japan All Collections About About Authorea FAQs Contact Us Quick Search anywhere Search for preprint articles, keywords, etc. Search Search ADVANCED SEARCH SCROLL British Journal of Clinical Pharmacology This is a preprint and has not been peer reviewed. Data may be preliminary. 14 May 2026 V1 Latest version Share on Tile: Therapeutic Repurposing of Venetoclax in Haematological Malignancies Authors : Gaurav Prakash 0000-0003-4142-1592 [email protected] , Rudra Narayan Swain [email protected] , Anu Kumari [email protected] , Charanpreet Singh [email protected] , Aditya Jandial [email protected] , Arihant Jain 0000-0001-9495-9789 [email protected] , Alka Khadwal [email protected] , and Pankaj Malhotra [email protected] Authors Info & Affiliations https://doi.org/10.22541/authorea.15003322/v1 Under Review British Journal of Clinical Pharmacology Peer review timeline 57 views 45 downloads Contents Abstract Information & Authors Metrics & Citations View Options References Figures Tables Media Share Abstract Venetoclax is a first-in-class, orally bioavailable, and highly selective BCL-2 inhibitor. It has transformed the therapeutic landscape of hematologic malignancies by targeting the anti-apoptotic pathway in malignant cells. Its rational development followed the limitations of earlier BCL-2 inhibitors, such as navitoclax, which showed off-target BCL-XL inhibition and caused dose-limiting thrombocytopenia. Venetoclax demonstrates predictable pharmacokinetics, favourable tolerability, and efficacy across lymphoid and myeloid cancers. In chronic lymphocytic leukaemia (CLL), venetoclax-based regimens—particularly venetoclax-rituximab and venetoclax-obinutuzumab—have produced deep molecular remissions, high rates of undetectable minimal residual disease, and durable progression-free survival in both relapsed and frontline settings. Combination strategies of venetoclax with BTK inhibitors have enabled time-limited, chemotherapy‑free, and all oral treatment options Repurposing venetoclax for acute myeloid leukaemia (AML) has been a major therapeutic advancement. The VIALE‑A and VIALE‑C trials established venetoclax with hypomethylating agents or low‑dose cytarabine as the standard of care for older or unfit AML patients, with significant improvements in remission rates and overall survival. Emerging evidence supports its use in younger AML patients, with comparable or superior efficacy to intensive chemotherapy and markedly reduced early mortality. Dose-modification strategies, including low-dose venetoclax with azole prophylaxis or shortened treatment duration, offer practical solutions to reduce toxicity in real-world settings. Beyond CLL and AML, venetoclax has demonstrated clinically meaningful activity in multiple myeloma with t(11;14), mantle cell lymphoma, lymphoplasmacytic lymphoma, T‑ALL, and select high‑risk lymphomas. Its expanding role across hematologic malignancies reflects a strong mechanistic rationale, synergistic potential with partner drugs, and a favourable therapeutic index. Information & Authors Information Version history V1 Version 1 14 May 2026 Peer review timeline Under Review British Journal of Clinical Pharmacology 14 May 2026 Submitted 14 May 2026 Submission Checks Completed Collection British Journal of Clinical Pharmacology Authors Affiliations Gaurav Prakash 0000-0003-4142-1592 [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Rudra Narayan Swain [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Anu Kumari [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Charanpreet Singh [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Aditya Jandial [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Arihant Jain 0000-0001-9495-9789 [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Alka Khadwal [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Pankaj Malhotra [email protected] Postgraduate Institute of Medical Education and Research, India View all articles by this author Metrics & Citations Metrics Article Usage 57 views 45 downloads .FvxKWukQNSOunydq8rnd { width: 100px; } Citations Download citation Gaurav Prakash, Rudra Narayan Swain, Anu Kumari, et al. Tile: Therapeutic Repurposing of Venetoclax in Haematological Malignancies. Authorea . 14 May 2026. DOI: https://doi.org/10.22541/authorea.15003322/v1 If you have the appropriate software installed, you can download article citation data to the citation manager of your choice. Simply select your manager software from the list below and click Download. For more information or tips please see 'Downloading to a citation manager' in the Help menu . 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