Chair of Trustees leads patient advocate authorship on important special report discussing the use of high-dose chemotherapy in the treatment of high-risk neuroblastoma.

This week five patient advocate authors, led by SKC Chair Nick Bird, were published in the leading journal Pediatric Blood and Cancer. The paper, co-written with Donna Ludwinski- Family Support Advocate at Solving Kids’ Cancer UK, Nicole Scobie- President at Zoe4Life and Antonia Palmer- Executive Director at Kindred Foundation, addresses the question which often plagues parents going through treatment with their child, “to transplant, or not to transplant?”.

This widely discussed topic is one that is often visited in both the research and patient communities, including at our recent Neuroblastoma Parent Global Symposium. High-dose chemotherapy followed by stem cell transplant is perhaps the most toxic treatment used in frontline treatment for high-risk neuroblastoma. It can cause extreme acute and long-term side effects, sometimes making children very ill, in some cases hindering their progression through the standard treatment journey. It is however not used without reason, and there is well-established evidence from randomised trials in both Europe and North America that these harsh drugs have been necessary to improve survival of children diagnosed with this awful disease. But the debate as to whether there continues to be enough solid evidence to warrant the use of this brutal therapy in the modern era of immunotherapy and chemoimmunotherapy is ongoing.

“Although the need for care and caution is inarguable, we cannot sit at the crossroads indefinitely. We cannot settle for side effects that are irreversible, that last a lifetime, and that cost lives.”

The question is this: Is there a more precise approach to the use of high-dose chemotherapy, avoiding it and its side effects wherever possible without affecting overall survival rates, and if so how exactly can it be implemented in the most careful way possible? 

The PBC publication lays out the controversies and perspectives that surround this dilemma, including a critically important one that has not been explored before in scientific literature; that of the parents who anguish over the decision to submit their children to high-dose chemotherapy and suffer the trauma of witnessing them go through it. It recognises that this is not a simple problem with any simple solution- calling for more coordinated and proactive work to be done internationally, to minimise the short- and long-term adverse effects that children experience, some potentially unnecessarily.

We are incredibly proud to be led by such a dedicated group of parents and advocates who are not afraid to demand better for our community- and continue to do all we can to fulfil their ambition for change. 

You can read the full article in Pediatric Blood and Cancer here: https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.29663