Welcome to WMozzies, the Australian Support group for everyone affected by Waldenström’s Macroglobulinemia (WM) .
The WhiMSICAL research team, led by researchers in Australia, has received approval to present their findings during a poster presentation at the American Society of Haematology’s annual meeting in December 2017. This is a significant achievement and validation of their efforts thus far, as not all applicants get approved to present at ASH! Congratulations should go out to all WM patients around the world who have participated thus far, as it is our data that made this possible.
The WhiMSICAL findings will be published after the ASH meeting, and will be made available to the WM community worldwide at that time.
Additionally, IWMF President Carl Harrington and the team will be presenting details and findings at the upcoming 3rd annual IWMF-LLS Strategic Research Roadmap Summit meeting. This is the meeting where leading WM researchers from around the world, gather to evaluate and discuss primary focus areas for WM-specific research. Details about the Research Roadmap are at www.iwmf.com/research/research-strategy
While the presentations at both meetings point to the viability of the WhiMSICAL database, there is a CRITICAL need for more patients to participate, and for more data to be entered by participants. With just a few minutes of our time, each of us can make a significant contribution to advance research to develop better treatments and a cure for WM…..and to assist in convincing government agencies to fund treatments for patients! See more details and to participate at www.wmozzies.com.au/index.php/whimsical As Andrew Warden, the Leader of the IWMF affiliate in Australia, WMozzies, stated recently on IWMF Connect (the IWMF’s online discussion group):
“The government funding authorities in the UK and Australia as well as Canada require objective evidence that the high cost of new treatments such as ibrutinib are justified in comparisons with current gold standard treatments. Rare diseases suffer because of lack of the required patient evidence. WhiMSICAL can help provide this evidence.”
The WhiMSICAL database is a vital complement to ongoing clinical trials. Both seek to provide strong relevant information needed to arrive at approvals for novel treatment protocols and to convince decision-makers to provide funding for those treatments.
As Carl Harrington says, “if you want to ‘pay it forward’ and help current and future WM patients around the world, then participate in WhiMSICAL! We all have our disease data. All we have to do is enter it in WhiMSICAL to make a difference for WMers now and in the future.”
See more details about WhiMSICAL and participate now at www.wmozzies.com.au/index.php/whimsical Set aside whatever you’re doing and do it now. Please help us reach 1000 participating WMers!
Judith Trotman, MD, clinical associate professor, medicine, Concord Clinical School, The University of Sydney and is the co-principal investigator on the WhiMSICAL WM Patient Database http://www.wmozzies.com.au/index.php/whimsical/
discusses results of a trial investigating BGB-3111 in patients with Waldenström’s macroglobulinemia (WM).
BGB-3111 is a very specific BTK inhibitor that has a very high, very good partial response (VGPR) rate in patients with WM, Trotman explains. Results showed that patients with WM did have very good responses with reduction in IgM from 33 g/L to 6 g/L and a rise in hemoglobin from 100 g/L to 140 g/L.
Patients also tolerated the drug well without any of the toxicities often seen with BTK inhibitors. Adverse events included very minimal bruising as well as minimal serious bleeding. This was an extremely well tolerated BTK inhibitor, with an overall response rate of 90% with 43% of patients achieving VGPR. "What's most important is that the patients feel fantastic on this agent! How can you quantitate energy? , she adds.
Membership of WhiMSICAL database has grown threefold from 69 to 230 in the four months to September 2017. The International Waldenström’s Macroglobulinemia Foundation (IWMF) and its global affiliates, have driven this growth. Multiple social media platforms have been used including online discussion forums, Facebook, email mailing lists and community newsletters. The international expansion has confirmed WhiMSICAL CART-WHEEL database as a robust global data-collection platform for WM patient-derived data.
The facility to print out their personal report with graphs and review any missing data with their clinician has proved valuable. The WM patient questionnaire completion has continued to be good in providing data on symptoms, pathology results, treatments, their tolerance and how they were accessed.
The study Principal Investigators have utilised CART-WHEEL to develop the continuously expanding patient-derived dataset to provide a foundation for hypothesis generation. This has involved utilising independent samples t-test, cross-tabulation and Pearson Chi-squared around to improve the understanding of this rare disease.
Upon further recruitment and encouragement of more complete and continuous data entry, an expanding and increasingly robust body of data will increase knowledge of the range of presentations and treatment experiences of WM patients. Future graduated database additions will include validated PROs (e.g. EuroQol EQ-5D-5L). WhiMSICAL has the potential to map real-world therapeutic efficacy, along with international patterns of treatment access.
Initial analysis of real-world clinical data on presentations and treatments from participants in the WhiMSICAL project has been submitted to the American Society of Hematology (ASH) annual meeting in December in Atlanta, the most comprehensive haematology event of the year.
None of the research would have been possible without the WM patients who have contributed their demographic data, tumour type details and gene testing, family cancer gene testing, reports from clinicians, treatments, side-effects and other psychosocial and family information via the CART-WHEEL database. As the number of participants joining the team increases, we will obtain the big data required to make a major impact in the field of WM and see how truly informative this research can be.
Additional WM clinicians have joined the WhiMSICAL database team, including Dr Marie Jose Kersten (The Netherlands), Dr Ruth Spearing (New Zealand), Dr Lia Palomba and Dr Sheeba Thomas (U.S), Dr Shirley D’Sa (UK), and Dr Loic Ysaebert (France).
The WhiMSICAL CART-WHEEL Summaries may be printed or downloaded. The Summaries provide a personal patient record. They are also a valuable base for discussions in physician / patient discussions.
WM patients in all countries are invited to participate in this important initiative.
In Australia data provided by WhiMSICAL is needed to support applications to regulatory authorities to gain treatment funding approval under the Pharmaceutical Benefits Scheme (PBS). Currently PBS funding for WM is not available or has restrictions for available world-wide novel WM treatments such as Bendamustine and ibrutinib (IMBRUVICA)
With other countries data from WM patients can be be analysed by WhiMSICAL on a country basis for inclusion as supporting evidence to the applicable funding regulatory authorities.
What is WhiMSICAL?
WhiMSICAL is an Australia-initiated, global clinical trial inviting Waldenström’s Macroglobulinemia (WM) patients to provide their clinical data, which will help advance current knowledge and understanding of this disease. CART-WHEEL (Center for Analysis of Rare Tumors) is a database designed to address some of the barriers facing effective research into rare cancers
How does it work?
CART-WHEEL uses a privacy-protected, internet-based questionnaire. WM patients complete this questionnaire on the CART-WHEEL.org website and have control over personal details by providing consent to different uses of entered data. The questions cover areas including:
If you would like to take part, please visit the CART-WHEEL website website www.cart-wheel.org ,fill in and post the consent form and enter your information. Your data cannot be utilised for research until the consent form has been received.
THANK YOU FOR YOUR INVALUABLE HELP!!
If you have any queries about the WhiMSICAL Study, please contact:
A/Prof Judith Trotman, Dr Ibrahim Tohidi-Esfahani (Principal Investigators)
Concord Repatriation General Hospital, Concord, NSW, 2139
Phone: +61 2 9767 7243
