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

Medulloblastoma is a highly malignant small round blue cell tumor of the posterior fossa ...

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

Neonates, particularly those born prematurely, are exquisitely vulnerable to life-threatening infections. This increased susceptibility to infection... ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2011
• January 2011

This Research Note has explored the strengths, risks and potential complexity of adaptive trials ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2019
• April 2019

Intensive rehabilitation aims to improve and maintain functioning in young people who experience disability due to illness or injury. Day rehabilitation may have advantages for families and healthcare systems over inpatient models of rehabilitation.  ...

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

This review supports the recommended use of palivizumab for reducing RSV-associated hospitalisation rates in premature babies born at gestational age <33 weeks. ...

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

The burden of IgE-mediated food allergy in Australian born children is reported to be among the highest globally. This illness shares risk factors and frequently coexists with asthma, one of the most common noncommunicable diseases of childhood. ...

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

Fidgety movements occur in infants between the age of 9 to 20 weeks post-term, and their absence are a strong indicator that an infant has cerebral palsy. Prechtl's General Movement Assessment method evaluates whether an infant has fidgety movements, but requires a trained expert to conduct it. Timely evaluation facilitates early interventions, and thus computer-based methods have been developed to aid domain experts.  ...

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

Pineoblastoma is a rare brain tumor usually diagnosed in children. Given its rarity, no pineoblastoma-specific trials have been conducted. Studies have included pineoblastoma accruing for other embryonal tumors over the past 30 years. ...

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

Preterm infants are at a high risk of developing late-onset sepsis (LOS). Lactoferrin is one of the most abundant endogenous antimicrobial proteins expressed in breast milk, stools, and blood, and a candidate for preventive intervention. Large clinical trials have recently investigated whether enteral supplementation with bovine lactoferrin reduces LOS. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2020
• August 2020

I review recent high-profile court cases to underscore the changing landscape of conflict and the central role that the media (and social media in particular) can play ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2020
• January 2020

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