Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma
Venetoclax is approved for adult patients with chronic lymphocytic leukemia and acute myeloid leukemia. Expanding its use to the pediatric population is currently under investigation, but more robust data are needed. We retrospectively analyzed the safety and efficacy of venetoclax in children/AYA w...
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-12-01
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| Series: | Cancers |
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| Online Access: | https://www.mdpi.com/2072-6694/14/1/150 |
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| author | Amber Gibson Adriana Trabal David McCall Sajad Khazal Laurie Toepfer Donna H. Bell Michael Roth Kris M. Mahadeo Cesar Nunez Nicholas J. Short Courtney DiNardo Marina Konopleva Ghayas C. Issa Farhad Ravandi Nitin Jain Gautam Borthakur Hagop M. Kantarjian Elias Jabbour Branko Cuglievan |
| author_facet | Amber Gibson Adriana Trabal David McCall Sajad Khazal Laurie Toepfer Donna H. Bell Michael Roth Kris M. Mahadeo Cesar Nunez Nicholas J. Short Courtney DiNardo Marina Konopleva Ghayas C. Issa Farhad Ravandi Nitin Jain Gautam Borthakur Hagop M. Kantarjian Elias Jabbour Branko Cuglievan |
| author_sort | Amber Gibson |
| collection | DOAJ |
| description | Venetoclax is approved for adult patients with chronic lymphocytic leukemia and acute myeloid leukemia. Expanding its use to the pediatric population is currently under investigation, but more robust data are needed. We retrospectively analyzed the safety and efficacy of venetoclax in children/AYA with ALL/LBL. We identified 18 patients (T-cell ALL, <i>n</i> = 7; T-cell LBL, <i>n</i> = 6; B-cell ALL, <i>n</i> = 5) aged 6–22 years. No new venetoclax safety signals were identified; the most common toxicity was myelosuppression. No deaths occurred within 30 days from the start of the therapy. A mean of 2.6 (range 0–8) prior lines of therapy were given. The mean duration of venetoclax was 4.06 months (range 0.2–24.67 months). Complete remission was achieved in 11 (61%) patients. Of the eight patients who remain alive, four are continuing on venetoclax combination therapy, and four proceeded to hematopoietic stem cell transplantation. Three patients who initially achieved CR, later relapsed, and are deceased. Nine patients are deceased, and one patient was lost to follow-up. Overall survival is 9.14 months (range 1.1–33.1), and progression-free survival is 7.34 months (range 0.2–33.1). This is the largest cohort of pediatric/AYA patients who received venetoclax for ALL/LBL. Our data support the consideration of venetoclax-based regimens in pediatric patients with R/R ALL/LBL and its investigation as upfront therapy for T-cell ALL/LBL. |
| first_indexed | 2024-03-10T03:47:17Z |
| format | Article |
| id | doaj.art-6112481f536e4d0281a2cdd7ed2b689e |
| institution | Directory Open Access Journal |
| issn | 2072-6694 |
| language | English |
| last_indexed | 2024-03-10T03:47:17Z |
| publishDate | 2021-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cancers |
| spelling | doaj.art-6112481f536e4d0281a2cdd7ed2b689e2023-11-23T11:16:51ZengMDPI AGCancers2072-66942021-12-0114115010.3390/cancers14010150Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic LymphomaAmber Gibson0Adriana Trabal1David McCall2Sajad Khazal3Laurie Toepfer4Donna H. Bell5Michael Roth6Kris M. Mahadeo7Cesar Nunez8Nicholas J. Short9Courtney DiNardo10Marina Konopleva11Ghayas C. Issa12Farhad Ravandi13Nitin Jain14Gautam Borthakur15Hagop M. Kantarjian16Elias Jabbour17Branko Cuglievan18Department of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatric Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatric Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USAVenetoclax is approved for adult patients with chronic lymphocytic leukemia and acute myeloid leukemia. Expanding its use to the pediatric population is currently under investigation, but more robust data are needed. We retrospectively analyzed the safety and efficacy of venetoclax in children/AYA with ALL/LBL. We identified 18 patients (T-cell ALL, <i>n</i> = 7; T-cell LBL, <i>n</i> = 6; B-cell ALL, <i>n</i> = 5) aged 6–22 years. No new venetoclax safety signals were identified; the most common toxicity was myelosuppression. No deaths occurred within 30 days from the start of the therapy. A mean of 2.6 (range 0–8) prior lines of therapy were given. The mean duration of venetoclax was 4.06 months (range 0.2–24.67 months). Complete remission was achieved in 11 (61%) patients. Of the eight patients who remain alive, four are continuing on venetoclax combination therapy, and four proceeded to hematopoietic stem cell transplantation. Three patients who initially achieved CR, later relapsed, and are deceased. Nine patients are deceased, and one patient was lost to follow-up. Overall survival is 9.14 months (range 1.1–33.1), and progression-free survival is 7.34 months (range 0.2–33.1). This is the largest cohort of pediatric/AYA patients who received venetoclax for ALL/LBL. Our data support the consideration of venetoclax-based regimens in pediatric patients with R/R ALL/LBL and its investigation as upfront therapy for T-cell ALL/LBL.https://www.mdpi.com/2072-6694/14/1/150acute lymphoblastic leukemialymphoblastic lymphomavenetoclaxBcl-2 inhibitorearly precursor T-cell |
| spellingShingle | Amber Gibson Adriana Trabal David McCall Sajad Khazal Laurie Toepfer Donna H. Bell Michael Roth Kris M. Mahadeo Cesar Nunez Nicholas J. Short Courtney DiNardo Marina Konopleva Ghayas C. Issa Farhad Ravandi Nitin Jain Gautam Borthakur Hagop M. Kantarjian Elias Jabbour Branko Cuglievan Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma Cancers acute lymphoblastic leukemia lymphoblastic lymphoma venetoclax Bcl-2 inhibitor early precursor T-cell |
| title | Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma |
| title_full | Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma |
| title_fullStr | Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma |
| title_full_unstemmed | Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma |
| title_short | Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma |
| title_sort | venetoclax for children and adolescents with acute lymphoblastic leukemia and lymphoblastic lymphoma |
| topic | acute lymphoblastic leukemia lymphoblastic lymphoma venetoclax Bcl-2 inhibitor early precursor T-cell |
| url | https://www.mdpi.com/2072-6694/14/1/150 |
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