4th International Patient Forum on Waldenstrom's Macroglobulinaemia – London, 17 March 2013
A Summary
This year's Forum was hosted by WMUK, a charity established in 2012 as acollaboration between patients and doctors to enable the support of those affected by WM and to promote research into the disease and best treatment practice.
Around 150 people assembled in the new, well-lit and comfortable conference facilities of The Royal College of General Practitioners in Central London and—as Dr Shirley D'Sa (UK consultant haematologist and a WMUK trustee) promised in her introductory remarks—the day would prove to be lively and interactive, with an emphasis on partnership between doctors, patients and carers. WMUK was making steady progress in working with other rare-disease interest groups and cancer organisations to collate accurate disease and treatment data, with the aims of creating a patient registry and securing the best possible treatments.
The first presentation was by Dr Helen McCarthy (UK), who offered a clear and informative overview of the various disease mechanisms of WM, explaining how the multiplicity of symptoms in the disease depended on the bodily sites involved and required clinicians to think laterally and creatively when making therapeutic decisions.
These themes were further developed by the keynote speaker, Dr Robert Kyle (of the Mayo Clinic, USA), an honoured guest at the Forum and a leading authority internationally on WM. His presentation focused on the understanding of blood test results and the factors that influence the decision to start treatment in what is an indolent disease, the basic guide being that the onset of symptoms should be the cue to strike.
A round-table discussion followed between the clinicians present, fielding questions put to them by Dr D'Sa on matters of current concern to patients. In addition to Dr Kyle and Dr McCarthy, the panel was made up of Dr Chara Kyriakou, Dr Roger Owen and Dr Guy Pratt (all UK) and Dr Monique Minnema (The Netherlands). The panel gave due attention to ibrutinib, which is currently undergoing trials in the USA specifically in WM and is heading for trials in Europe in other blood cancers. It was agreed that trial findings (on 65 patients with relapsed disease) were so far very promising, but it was not likely that the agent would be available in the UK for a number of years and there would be questions of cost and of whether continued therapy would be required. In response to other questions the panel agreed on the vital importance of listening to the patient and observing clinical signs very closely—the treatment of WM was both an art and a science, the currently agreed prognostic indicators in the disease not being specific to individual patients but being of more relevance to clinical trials.
Dr Minnema then gave a presentation of the findings of a recent survey of WM diagnosis and treatment in the Netherlands, where she described healthcare provision as being generous but with room for improvement. Although the response to the survey, which was aimed both at doctors and patients, had been small, valuable data had been gathered, enabling the development of treatment guidelines and the provision of more appropriate treatments. Her talk demonstrated the importance of collecting such data, underlined the necessity of organising data gathering and clinical trials on an international basis.
One of the points made by Dr Minnema was that bendamustine is emerging as preferable to R-CHOP as a treatment regimen, although as yet experience on the ground is not supported at official levels (which is also the picture elsewhere in Europe). Unfortunately the next scheduled speaker, Dr Mathias Rummel, was unable to attend and so the Forum did not on this occasion have the opportunity of hearing from the world's foremost expert on bendamustine. Nevertheless, the information about this agent given by Dr Minnema and other doctors present was encouraging and leads one to hope that its use will gain acceptance.
During the morning break and also over lunch the participating clinicians had made themselves available for "Ask the Doctor/Nurse-Lite" sessions in the foyer. By pre-booking, patients and carers were able to raise questions about their own illness and treatment on a one-to-one basis and many eagerly took up these opportunities generously made available by our resident experts. The foyer also had displays of individual "Patient Tales" on large boards and the stories shared here offered valuable and contrasting snapshots of the different ways in which WM can be experienced. There were displays too by the IWMF, European WM Network, the Lymphoma Association, Macmillan Cancer Support and the Binding Site. The breaks of course also gave time for conversation and people made full use of the opportunities for making new contacts and renewing existing ones—There was a palpable buzz in the foyer!
The afternoon sessions began with two presentations by Roger Owen, the first being on the upcoming R2W Trial in the UK (which will compare fludarabine, cyclophosphamide and rituximab with bortezomib, cyclophosphamide and rituximab as upfront therapy) and emphasising that clear endpoints were essential for the proper quality of research.Dr Owen next gave the first public airing—before official publication—of the new UK Treatment Guidelines for WM. The inclusion of rituximab in treatment schedules was highlighted, of particular importance given that some UK patients cannot access this agent without funding permission. The lack of a standardised approach is clear as different combinations apply to different situations; their sequencing is the subject of ongoing clinical trials. Although the details of the guidelines would only become clear on full publication, it had been evident to the working group that there could be no "one-size-fits-all" approach to the treatment of WM, the guiding principle being flexibility alongside a systematic approach to assessing and managing the disease.
There followed an overview by Shirley D'Sa of the issues involved in living with WM. One challenge of coping with the disease, at least historically, had been in finding reliable information and sohaving the resources to distinguish symptoms indicative of WM from the general "noise" offered up by daily life. It was here that the IWMF, WMUK, the Lymphoma Association and other suitable organisations could help. Once again, as with treatment choices, each person would have to make his or her own decisions about self-management, diet and exercise, but it was clear that an active lifestyle, within the bounds of individual preferences and ability, was the ideal goal—some activity would always be better than none. Complications were rare in WM, but could be especially challenging especially if not recognised in good time (e.g. amyloidosis, Bing-Neel Syndrome), and here again WMUK could help.
The final session consisted of open questions from the floor to the panel of physicians before Phil Manning (a trustee of WMUK) reviewed some of the key points of the day in his closing remarks, emphasising that there were plenty of opportunities for those able to do so to get involved. Formal proceedings closed with thanks to the participating physicians as well as to the organising team of Roger Brown, Shirley D'Sa and Rita Flatley and their volunteer helpers for making the day such a lively and successful event.
A DVD of the Forum is available. See the homepage of for details on ordering.