(L-R) Sinead Healy, Sarah McCormack, Caroline Hird, Dr Kevin Windebank, Dr Venetia Bigley, Dr Vasanta Nanduri and Professor Matt Collin
PARENTS and relatives headed to the Leeds General Infirmary recently to hear the UK’s leading experts talk about LCH, latest trials and research projects.
Twenty-seven people attended The H R Trust LCH Parent and Patient Road Show and met with experts Drs Kevin Windebank, Vasanta Nanduri, Johann Visser and Professors Matt Collin and Peter Beverley.
Dr Nanduri, a consultant paediatrician at Watford General Hospital, introduced the evening by explaining about histiocytes and Histiocytosis and the differences between Langerhan’s Cell Histiocytosis and Haemophagocytic Lymphohistiocytosis.
Senior Lecturer in Adolescent and Child Heath, Dr Kevin Windebank, talked about Langerhan’s cells and dendritic cells and spoke about the types of symptoms experienced by some of his patients.
He gave a detailed explanation about how LCH can affect the body and where it effects.
Professor Matt Collin, Consultant Haematologist at the Freeman Hospital, Newcastle-upon-Tyne, advised that adults suffered quite similar symptoms to children when struck with LCH.
However, they were not able to gain the same access to specialists as children. He explained: “There is a long road to travel in terms of getting access to correct adult specialists. We need new guidelines to manage the disease and have it more co-ordinated.”
Professor Collin added that at his clinic in Newcastle there is access to a haematologist, a dermatologist and an expert in chest medicine.
He then went on to talk about his latest research project, Cell Autonomous Mechanisms in the Pathogenesis of Langerhan’s Cell Histiocytosis, which is being funded by The Trust.
This project will incorporate the findings of Dr Barrett Rollins which found that over half (57%) of the LCH biopsies examined by his team of scientists had contained a specific genetic change in BRAF called V600E which has also been seen in melanoma, colorectal cancer and thyroid cancers.
Together with Professor Frederick Geissman, of King’s College, London, Professor Collin, is hopeful that new diagnostic and prognostic tests and therapeutic targets for LCH will be established.
“Our research might even lead to a blood test to screen for LCH. We are very grateful to have this finding and hope to take it further,” he added.
Dr Vasanta Nanduri spoke about Late Effects and stressed that it was very important that LCH patients did not smoke as this would make their condition worse.
Dr Johann Visser talked about how clinical trials work, the new LCH IV Trial and the International Rare Histiocytic Disorder Registry.
He advised that clinical trials take a long time to devise and have to be better than the best known treatment on offer before they can go ahead.
LCH IV will be a comprehensive study that will address different aspects of childhood LCH and investigate Late Effects, including the neurological complications that affect some patients.
Dr Visser explained that it would take six years to recruit the 600 patients taking part in the study and involve 23 countries including Germany, the USA and Canada. The study would take a minimum of 10 years to complete.
He added that the aim of the International Rare Histiocytic Disorder Registry is to improve knowledge regarding the natural history, treatment and outcome of children and adults with rare histiocytoses.
Professor Peter Beverley, vice-President of The H R Trust, explained that LCH was called an “orphan” disease because it was so rare and most doctors do not come across it.
He advised that because of the rarity of Histiocytosis, funding was not provided by either the Government or the General Medical Council and therefore the Trust was reliant on donations and fundraising.
Funds were currently donated by The Artemis Association, Greece, LCH Belgium and from events organised by familes and friends of those affected by LCH and HLH.
Feedback from the Road Show was very positive. Sarah McCormack, who travelled from Ireland, said: “I found the Road Show very informative, helpful and interesting.
“I want to do whatever I can to help the Trust to continue with its amazing work. Overall, I am so glad I came over and it is so reassuring to see such dedicated people involved with LCH.”
The next Road Show will be held in 2012 – the location will be announced in due course.