Gibson Amber, Trabal Adriana, McCall David, Khazal Sajad, Toepfer Laurie, Bell Donna H, Roth Michael, Mahadeo Kris M, Nunez Cesar, Short Nicholas J, DiNardo Courtney, Konopleva Marina, Issa Ghayas C, Ravandi Farhad, Jain Nitin, Borthakur Gautam, Kantarjian Hagop M, Jabbour Elias, Cuglievan Branko
Department of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Department of Pediatric Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Cancers (Basel). 2021 Dec 29;14(1):150. doi: 10.3390/cancers14010150.
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, = 7; T-cell LBL, = 6; B-cell ALL, = 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.
维奈克拉已被批准用于治疗成年慢性淋巴细胞白血病和急性髓系白血病患者。目前正在研究将其应用扩展至儿科人群，但还需要更有力的数据。我们回顾性分析了维奈克拉在儿童/青少年急性淋巴细胞白血病/淋巴母细胞淋巴瘤患者中的安全性和疗效。我们纳入了18例年龄在6至22岁之间的患者（T细胞急性淋巴细胞白血病7例；T细胞淋巴母细胞淋巴瘤6例；B细胞急性淋巴细胞白血病5例）。未发现维奈克拉新的安全信号；最常见的毒性是骨髓抑制。治疗开始后30天内无死亡病例。平均给予过2.6（范围0 - 8）线的前期治疗。维奈克拉的平均使用时长为4.06个月（范围0.2 - 24.67个月）。11例（61%）患者实现完全缓解。在8例存活患者中，4例继续接受维奈克拉联合治疗，4例进行了造血干细胞移植。3例最初达到完全缓解的患者后来复发并死亡。9例患者死亡，1例失访。总生存期为9.14个月（范围1.1 - 33.1），无进展生存期为7.34个月（范围0.2 - 33.1）。这是接受维奈克拉治疗急性淋巴细胞白血病/淋巴母细胞淋巴瘤的最大儿科/青少年患者队列。我们的数据支持考虑在复发/难治性急性淋巴细胞白血病/淋巴母细胞淋巴瘤的儿科患者中使用基于维奈克拉的方案，并支持将其作为T细胞急性淋巴细胞白血病/淋巴母细胞淋巴瘤的一线治疗进行研究。
