Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database
The potent oral inhibitor of BCL2, venetoclax (VEN), used to treat adults with chronic lymphocytic leukaemia, has been approved in US for the treatment of naïve patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy and recently in Europe, too. However, the drug has been used fo...
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Frontiers Media S.A.
2021
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oai:doaj.org-article:2ff780c252104344b836dc38e573699f2021-11-11T07:14:12ZOff-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database1663-981210.3389/fphar.2021.748766https://doaj.org/article/2ff780c252104344b836dc38e573699f2021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fphar.2021.748766/fullhttps://doaj.org/toc/1663-9812The potent oral inhibitor of BCL2, venetoclax (VEN), used to treat adults with chronic lymphocytic leukaemia, has been approved in US for the treatment of naïve patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy and recently in Europe, too. However, the drug has been used for years in combination with hypomethylating agents (HMAs) in patients not eligible to other treatment option, according to the so-called off-label use. We collected real-world data about patients treated with VEN + HMAs in the context of a pharmacovigilance project focused on the evaluation of the safety and effectiveness of drugs used for unapproved indication in Italian hospitals. From March to December 2020, 24 patients started treatment with VEN combined with HMAs. 21 patients have been assessed for response. Eleven (52%) patients reached complete remission (CR), and three patients (14%) CR with partial hematological recovery (CRh), with a median duration of response of 4.5 months (range 0.5–12.5). 19 patients experienced at least 1 adverse drug reaction (ADR), mostly serious, including 3 deaths (9% of ADRs; 12.5% of patients) in febrile neutropenia. Hematological toxicities and infections (cytopenia, neutropenia, febrile neutropenia, sepsis), were the most reported ADRs (84.4%). In general, neutropenic fever occurred more frequently in patients treated with decitabine (7 out of 9, 78%) compared to azacitidine (5 out of 15, 33%; p = 0.03), whereas response assessment did not differ based on used HMA (p = 0.1). These results confirm the benefit-risk profile of VEN in a real-world setting of patients with no adequate therapeutic options.Lucia GozzoLucia GozzoCalogero VetroSerena BrancatiLaura LongoDaniela Cristina VitaleGiovanni Luca RomanoElisa MauroPaolo Fabio FiumaraCinzia MaugeriMarina Silvia ParisiIlaria DulcamareBruno GaribaldiAndrea DuminucoGiuseppe Alberto PalumboGiuseppe Alberto PalumboFrancesco Di RaimondoFrancesco Di RaimondoFilippo DragoFilippo DragoFilippo DragoFrontiers Media S.A.articlevenetoclaxacute myeloid leukemiaoff-labeleffectivenessadverse eventTherapeutics. PharmacologyRM1-950ENFrontiers in Pharmacology, Vol 12 (2021) |
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| topic |
venetoclax acute myeloid leukemia off-label effectiveness adverse event Therapeutics. Pharmacology RM1-950 |
| spellingShingle |
venetoclax acute myeloid leukemia off-label effectiveness adverse event Therapeutics. Pharmacology RM1-950 Lucia Gozzo Lucia Gozzo Calogero Vetro Serena Brancati Laura Longo Daniela Cristina Vitale Giovanni Luca Romano Elisa Mauro Paolo Fabio Fiumara Cinzia Maugeri Marina Silvia Parisi Ilaria Dulcamare Bruno Garibaldi Andrea Duminuco Giuseppe Alberto Palumbo Giuseppe Alberto Palumbo Francesco Di Raimondo Francesco Di Raimondo Filippo Drago Filippo Drago Filippo Drago Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database |
| description |
The potent oral inhibitor of BCL2, venetoclax (VEN), used to treat adults with chronic lymphocytic leukaemia, has been approved in US for the treatment of naïve patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy and recently in Europe, too. However, the drug has been used for years in combination with hypomethylating agents (HMAs) in patients not eligible to other treatment option, according to the so-called off-label use. We collected real-world data about patients treated with VEN + HMAs in the context of a pharmacovigilance project focused on the evaluation of the safety and effectiveness of drugs used for unapproved indication in Italian hospitals. From March to December 2020, 24 patients started treatment with VEN combined with HMAs. 21 patients have been assessed for response. Eleven (52%) patients reached complete remission (CR), and three patients (14%) CR with partial hematological recovery (CRh), with a median duration of response of 4.5 months (range 0.5–12.5). 19 patients experienced at least 1 adverse drug reaction (ADR), mostly serious, including 3 deaths (9% of ADRs; 12.5% of patients) in febrile neutropenia. Hematological toxicities and infections (cytopenia, neutropenia, febrile neutropenia, sepsis), were the most reported ADRs (84.4%). In general, neutropenic fever occurred more frequently in patients treated with decitabine (7 out of 9, 78%) compared to azacitidine (5 out of 15, 33%; p = 0.03), whereas response assessment did not differ based on used HMA (p = 0.1). These results confirm the benefit-risk profile of VEN in a real-world setting of patients with no adequate therapeutic options. |
| format |
article |
| author |
Lucia Gozzo Lucia Gozzo Calogero Vetro Serena Brancati Laura Longo Daniela Cristina Vitale Giovanni Luca Romano Elisa Mauro Paolo Fabio Fiumara Cinzia Maugeri Marina Silvia Parisi Ilaria Dulcamare Bruno Garibaldi Andrea Duminuco Giuseppe Alberto Palumbo Giuseppe Alberto Palumbo Francesco Di Raimondo Francesco Di Raimondo Filippo Drago Filippo Drago Filippo Drago |
| author_facet |
Lucia Gozzo Lucia Gozzo Calogero Vetro Serena Brancati Laura Longo Daniela Cristina Vitale Giovanni Luca Romano Elisa Mauro Paolo Fabio Fiumara Cinzia Maugeri Marina Silvia Parisi Ilaria Dulcamare Bruno Garibaldi Andrea Duminuco Giuseppe Alberto Palumbo Giuseppe Alberto Palumbo Francesco Di Raimondo Francesco Di Raimondo Filippo Drago Filippo Drago Filippo Drago |
| author_sort |
Lucia Gozzo |
| title |
Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database |
| title_short |
Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database |
| title_full |
Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database |
| title_fullStr |
Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database |
| title_full_unstemmed |
Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database |
| title_sort |
off-label use of venetoclax in patients with acute myeloid leukemia: single center experience and data from pharmacovigilance database |
| publisher |
Frontiers Media S.A. |
| publishDate |
2021 |
| url |
https://doaj.org/article/2ff780c252104344b836dc38e573699f |
| work_keys_str_mv |
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