We report on the Australian experience of blinatumomab for treatment of 24 children with relapsed/refractory precursor B-cell acute lymphoblastic leukaemia (B-ALL) and high-risk genetics, resulting in a minimal residual disease (MRD) response rate of 58%, 2-year progression-free survival (PFS) of 39% and 2-year overall survival of 63%. In total, 83% (n = 20/24) proceeded to haematopoietic stem cell transplant, directly after blinatumomab (n = 12) or following additional salvage therapy (n = 8). ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• May 2021

Epidermal growth factor receptor (EGFR) is over-expressed in many brain tumors. This paper examines mutations the EGFR that make the cell it is produced in... ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2014
• April 2014

This study investigated the relapse and outcome patterns of patients with central nervous system mixed malignant germ cell tumors treated with chemotherapy-only ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2015
• June 2015

PEGasparaginase is known to be a critical drug for treating pediatric acute lymphoblastic leukemia (ALL), however, there is insufficient evidence to determine the optimal dose for infants who are less than one year of age at diagnosis. This international study was conducted to identify the pharmacokinetics of PEGasparaginase in infants with newly diagnosed ALL and gather insight into the clearance and dosing of this population. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2024
• March 2024

Molecular profiling of the tumour immune microenvironment (TIME) has enabled the rational choice of immunotherapies in some adult cancers. In contrast, the TIME of paediatric cancers is relatively unexplored. We speculated that a more refined appreciation of the TIME in childhood cancers, rather than a reliance on commonly used biomarkers such as tumour mutation burden (TMB), neoantigen load and PD-L1 expression, is an essential prerequisite for improved immunotherapies in childhood solid cancers. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2023
• April 2023

Outcomes for infants diagnosed under 1 year of age with KMT2A-rearranged acute lymphoblastic leukemia (ALL) have remained stagnant over the past 20 years. Successive treatment protocols have previously focused on intensification of conventional chemotherapy, but increased treatment-related toxicity and chemoresistance have led to a plateau in survival. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2022
• December 2022

Rishi Sury Kotecha RS MB ChB (Hons) MRCPCH FRACP PhD Co-Head, Leukaemia Translational Research rishi.kotecha@health.wa.gov.au Co-Head, Leukaemia Translational Research Areas of research expertise: Preclinical and clinical leukaemia research; ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2022
• August 2022

A higher incidence of pediatric intracranial germ cell tumors (iGCTs) in Asian countries compared with Western countries has been reported. In Malaysia, the literature regarding pediatric iGCTs have been nonexistent. The aim of this study was to review the management, survival, and long-term outcomes of pediatric iGCTs at a single tertiary center in Malaysia. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• October 2021

The goal of a clinical quality registry is to deliver immediate gains in survival and quality of life by delivering timely feedback to practitioners, thereby ensuring every patient receives the best existing treatment. We are developing an Australian Brain Cancer Registry (ABCR) to identify, describe, and measure the impact of the variation and gaps in brain cancer care from the time of diagnosis to the end of life. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• August 2021

In this. i study the MTD and RP2D, safety, PK, and preliminary activity of single-agent ribociclib were investigated in patients with neuroblastoma. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2017
• May 2017

Medulloblastoma is the most common malignant childhood brain tumor, and 5-year overall survival rates are as low as 40% depending on molecular subtype, with new therapies critically important. As radiotherapy and chemotherapy act through the induction of DNA damage, the sensitization of cancer cells through the inhibition of DNA damage repair pathways is a potential therapeutic strategy. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• April 2021

We provide evidence that targeting leukemia-induced bone loss is a therapeutic strategy for pre-B ALL ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2018
• November 2018