Venetoclax Use in Paediatric Haemato-Oncology Centres in Poland: A 2022 Survey

Katarzyna Bobeff; Agata Pastorczak; Zuzanna Urbańska; Walentyna Balwierz; Edyta Juraszewska; Jacek Wachowiak; Katarzyna Derwich; Magdalena Samborska; Krzysztof Kałwak; Iwona Dachowska-Kałwak; Paweł Łaguna; Iwona Malinowska; Katarzyna Smalisz; Jolanta Goździk; Aleksandra Oszer; Bartosz Urbański; Maciej Zdunek; Tomasz Szczepański; Wojciech Młynarski; Szymon Janczar

doi:10.3390/children10040745

Venetoclax Use in Paediatric Haemato-Oncology Centres in Poland

A 2022 Survey

Bibliographic Data

ID	15712888
Authors	Katarzyna Bobeff (0000-0001-8916-683X, Medical University of Lodz), Agata Pastorczak (0000-0003-3089-6947, Medical University of Lodz), Zuzanna Urbańska (0000-0002-4810-6627, Medical University of Lodz), Walentyna Balwierz (0000-0002-5379-2318, Jagiellonian University), Edyta Juraszewska (Jagiellonian University), Jacek Wachowiak (0000-0002-4680-603X, Poznan University of Medical Sciences), Katarzyna Derwich (0000-0001-7239-4035, Poznan University of Medical Sciences), Magdalena Samborska (0000-0002-3424-0323, Poznan University of Medical Sciences), Krzysztof Kałwak (0000-0003-1174-5799, Wroclaw Medical University), Iwona Dachowska-Kałwak (Wroclaw Medical University), Paweł Łaguna (0000-0002-3656-5236, Department of Pediatric Oncology and Hematology, Independent Public Children’s Teaching Hospital, Zwirki i Wigury 63A, 02-091 Warsaw, Poland), Iwona Malinowska (0000-0003-0671-7849, Department of Pediatric Oncology and Hematology, Independent Public Children’s Teaching Hospital, Zwirki i Wigury 63A, 02-091 Warsaw, Poland), Katarzyna Smalisz (Department of Pediatric Oncology and Hematology, Independent Public Children’s Teaching Hospital, Zwirki i Wigury 63A, 02-091 Warsaw, Poland), Jolanta Goździk (0000-0002-5449-0266, Jagiellonian University), Aleksandra Oszer (0000-0003-0317-1879, Medical University of Lodz), Bartosz Urbański (0000-0002-4737-9885, Medical University of Lodz), Maciej Zdunek (0000-0001-5519-113X, Medical University of Lodz), Tomasz Szczepański (0000-0001-5336-261X, Medical University of Silesia), Wojciech Młynarski (0000-0003-2714-5851, Medical University of Lodz), Szymon Janczar (0000-0001-8007-6969, Medical University of Lodz, corresponding author)
Year	2023
Volume	10
Issue	4
Pages	745-745
Publication date	2023-04-19
Open Access	Yes
Type	ARTICLE
Venue	Children (JOURNAL)
Journal identifiers	ISSN: 2227-9067 • E-ISSN: 2227-9067
Publisher	Multidisciplinary Digital Publishing Institute (PUBLISHER • CH)
DOI	10.3390/children10040745
PMID	37189994
OpenAlex	W4366549984
Language	EN
References cited	42

Venetoclax, the best established BH3-mimetic, is a practice-changing proapoptotic drug in blood cancers in adults. In paediatrics the data are fewer but exciting results were recently presented in relapsed or refractory leukaemias demonstrating significant clinical activity. Importantly, the in-terventions could be potentially molecularly guided as vulnerabilities to BH3-mimetics were re-ported. Currently venetoclax is not incorporated into paediatric treatment schedules in Poland but it has been already used in patients that failed conventional therapy in Polish paediatric haemato-oncology departments. The aim of the study was to gather clinical data and correlates of all paediatric patients treated so far with venetoclax in Poland. We set out to gather this experience to help choose the right clinical context for the drug and stimulate further research. The questionnaire regarding the use of venetoclax was sent to all 18 Polish paediatric haemato-oncology centres. The data as available in November 2022 were gathered and analysed for the diagnoses, triggers for the intervention, treatment schedules, outcomes and molecular associations. We received response from 11 centres, 5 of which administered venetoclax to their patients. Clinical benefit, in most cases consistent with hematologic complete remission (CR), was reported in 5 patients out of ten, whereas 5 patient did not show clinical benefit from the intervention. Importantly, patients with CR included subtypes expected to show venetoclax vulnerability, such as poor-prognosis ALL with TCF::HLF fusion. We believe BH3-mimetics have clinical activity in children and should be available to pae-diatric haemato-oncology practitioners in well-selected applications

Cancer · Context (archaeology · Family medicine · Intervention (counseling · Leukemia · Pediatric oncology · Psychiatry · Venetoclax · Acute Lymphoblastic Leukemia research · Acute Myeloid Leukemia Research · Chronic Lymphocytic Leukemia Research · Medicine · Internal Medicine · Oncology · Pediatrics

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