WhiMSICAL Registry –

A global WM patient-derived data registry capturing treatment, quality of life and COVID-19 data

WM patients globally are invited to contribute their details to the WhiMSICAL (Waldenström’s Macroglobulinemia Study In CART-WheeL) Registry.
Patients anywhere in the world can join, consent and enter their data to contribute to WM research.
Early results of WhiMSICAL’s patient-entered data have already been published in a leading US hematology journal- American Journal of Hematology
WhiMSICAL Registry captures how effective different treatments are and how they can impact quality of life
It is a flexible, secure platform, with COVID-19 impact and vaccine questions recently added
Access to data is solely to advance the research and information priorities of the investigators and patients and carers who enter the data.
The Registry is approved by the US Western Institutional Review Board (WCG IRB) and by the Australian Ethics Committees of Royal Melbourne and Concord Repatriation General Hospitals

WhiMSICAL aims to develop a global platform for obtaining real world “big data” in WM with ongoing recruitment and patient data entry to:

Cover a research void of insufficient well-founded evidence, as WM is a rare disease with limited phase III clinical trial data
Increase knowledge of presentations and treatment outcomes of WM patients
Map patient reported outcomes (PROs) to therapies
Generate hypotheses to advance WM research
Facilitate patient preference research

Information follows on how WhiMSICAL is heading towards big data analytics to uncover hidden patterns, unknown correlations, trends and patient preferences, covering:

Features of WhiMSICAL
History of WhiMSICAL
How WhiMSICAL works
How to take part in WhiMSICAL.
Enquiries and support
WhiMSICAL Registry flyer

1. Features of IWMF WhiMSICAL Registry include:

WhiMSICAL Registry is a global research project, supported by the IWMF.
A personal summary of disease experience, including graphing of your Hemoglobin and IgM levels to use as a personal profile and for use in consultations with your physicians
Capacity for ongoing revision with future entries
Authorised clinician researchers gain access to de-identified data for a better understanding of Waldenström’s macroglobulinemia and for improved treatments for patients, both globally or for their own country.
Access to data is solely to advance the research and information priorities of the investigators and patients and carers who enter the data.
The Registry is approved by Western Institutional Review Board in the US and the Ethics Committees of Royal Melbourne and Concord Repatriation General Hospitals in Australia.
A robust proven processing platform which securely holds medical data on over a million patients
A powerful analytical tool to support ease of use by Investigators
A potential for “Big-Data” comparisons with information at hospital institutions to aid informed decisions by medical researchers

2. History of WhiMSICAL

Initial recruitment efforts illustrated below were driven through messaging by WMozzies, to establish feasibility of the platform, resulting in 69 patients (16%) joining the study in 12 months (29% of study duration). Subsequent social media messaging by the IWMF to members globally led to a significant surge in the number of participants (A). Messaging regarding the addition of the Quality of Life questions EORTC QLQ-C30 to the questionnaire led a second surge in recruitment (B). COVID-19 questions were added in 2020-21.

WhiMSICAL Registry 453 members from 19 different countries.

46% of members are from the US.

3. How WhiMSICAL works

WhiMSICAL uses a privacy-protected, internet-based questionnaire. WM patients complete this questionnaire on the CART-WHEEL.org website. They have control over personal details by providing consent to different uses of entered data. A Correlative study of WhiMSICAL patient-derived data with Lymphoma and Related Diseases Registry available data has confirmed an 80% agreement. There were high completion rates and good concordance with diagnosis and treatment data but higher variability with IgM data.

The WhiMSICAL CART-WHEEL questionnaire covers:

Patient demographics
Disease specifics: staging, chronology of symptoms, IgM levels, full blood count information, etc.
Treatments, how accessed, and treatment side-effects
Personal and family medical history
Quality of Life (EORTC QLQ-C30) Patient Reported Outcomes (PROs)
COVID-19 impact and vaccines

For individual WM patients, WhiMSICAL provides a summary (profile) of questions and responses. This is handy as an individual’s personal record and for review with their physician.

Test results and the overall Quality of Life scales are also presented graphically.

The graphs show trend lines for each of the entered Pathology results and include details of treatments received.

4. How to take part in WhiMSICAL

Register Create a CART-WHEEL account at https://www.cart-wheel.org/

The setup of a CART-WHEEL account includes creating a username and a password to use to log in to the questionnaire. This gives an option to save information at any time and to log in later to update the information.

Give Consent – Click on the “My Consent details” tab Data entered in Questionnaire is not available for Research until Consent is given.

Text Box: It will be useful before you start the questionnaire to have:
§ A copy of your pathology report
§ Information about the hospitals or medical centres where you have been treated
§ The names of the treatments that you have received

If you do not have all the required information immediately available, you can update your answers to those questions at a later date.
Don’t delay starting even if your available data is patchy. You can add detail later when it is handy.

Your information will be entered via a secure connection and cannot be seen by anyone else. The WhiMSICAL Registry special section is automatically generated when you enter Waldenström’s or lymphoplasmacytic lymphoma into your diagnosis in Question 3.

Only the highlighted questions need to be completed and those labelled �Skip� do not require completion.
Questions can be saved and answered in more than one sitting. In future, please return to complete any remaining questions and update your data, particularly at the start and completion of each treatment course.

At the end of the questionnaire, you will be able to print out a summary of questions and your responses for your record
For assistance send email to whimsical@iwmf.com

5. Enquiries and support

General information – IWMF and WMozzies: [email protected]

Support from WM patients using WhiMSICAL: [email protected]

Technical support and user problems: WhiMSICAL CART-WHEEL: [email protected]

Frequently Asked Questions http://wmozzies.com.au/index.php/whimsical/whimsical-frequently-asked-questions/

WhiMSICAL Registry flyer

FUND RAISING TARGET COMPLETED.

December 9, 2021December 9, 2021

Very excited to report that the $150,000 target has been achieved and the IWMF Roadmap Project will receive the funding as per their agreement with the Leukaemia Foundation. We are very grateful to all the people who donated and we can all be proud to be part of a project that looking very deep into WM and hopefully will lead to further & better treatments. We also wish to acknowledge the assistance of the Leukaemia Foundation in first of all entering into the agreement with the IWMF and helping us with the Donation Page. Should you wish to donate, you can still donate to assist WMozzies by clicking on the donate button on the home page.

New treatment for Waldenström’s macroglobulinemia now available

October 28, 2021October 28, 2021

A new treatment for Waldenström's macroglobulinemia (WM) has been approved by the Therapeutic Goods Administration for adult patients who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy, and also for adult patients with mantle cell lymphoma. BRUKINSA (zanubrutinib) was developed by BeiGene, a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. WMozzies was proud to be invited by the Pharmaceutical Benefits Advisory Committee (PBAC) to take part in a pilot program as consumer advocates in the BRUKINSA PBAC decision process. WM patients will have immediate free of charge access to BRUKINSA through a BeiGene sponsored Pre-Reimbursement Access Program until such time that WM is listed for reimbursement on the Pharmaceutical Benefits Scheme. In a first for the PBAC, BeiGene expects to enter a facilitated resolution pathway in order to seek a listing date for WM. What is BRUKINSA? BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesised, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimising bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues. For more information, see Products | BeiGene

WhiMSICAL Registry’s important publication

June 16, 2021June 16, 2021

The WhiMSICAL Registry’s publication of patient-entered data in the prestigious medical journal, American Journal of Hematology, presents important research for all WM patients. It has added to the field of WM Research by capturing real world WM treatment and quality of life outcomes. Congratulations to all WM patients who have entered their data into the WhiMSICAL Registry, and now published at American Journal of Hematology ajh.26173. More Registry participants are needed to strengthen the results. A further submission is planned to the 2021 American Society of Hematology Conference. This is to include updated details from existing and from new participants received by the deadline of 12^th July. The WhiMSICAL Registry, built through a patient-clinician investigator partnership, is a global database for WM patients to enter their own data. Key findings include:

Longest time to next treatment after 1^st line therapy - in patients treated with bendamustine rituximab
Better quality of life in those on Bruton tyrosine kinase inhibitors compared to recent chemotherapy.

Over 450 patients from 19 countries contributed to this first global registry for WM. Highlights of the study so far include:

Little consistency in 1^st line therapy choice globally, with 46 different types of therapies given!
USA patients commence their 1^st therapy almost 3 times faster than the rest of the world.
1^st line bendamustine rituximab has outperformed the Bruton tyrosine kinase inhibitors (BTKi, e.g., ibrutinib, zanubrutinib) and rituximab alone in time to next treatment. The findings are preliminary, however, without exact matching of the groups. More participants, and longer follow-up of existing participants are needed to confirm these exciting preliminary results.
Patients on BTKi report better quality of life scores than those who recently received other treatments.

COVID-19 vaccines are now rolling out globally. WM patients may achieve impaired vaccine responses due to their condition and treatment. To help assess the impact of COVID on patients with WM, the WhiMSICAL Registry has included a question (at question 19) on COVID-19 testing, infection and vaccination. Please contribute to this important and urgent research question for WM patients. From anywhere in the world, you can join the WhiMSICAL Registry by registering and completing consent at www.cart-wheel.org, and then filling out the questionnaire. Some data is always better than no data. Your answers can, and are recommended to be, continually updated. Please note that it does not all need to be done in one sitting; you can enter as much (or as little) as you are able to provide each time to access the Registry. (Naturally, the more data that is collected, the better the outcomes for further research.) For those already participating in WhiMSICAL, thank you for contributing your patient voice to peer-reviewed research. Please continue to update your data at www.cart-wheel.org, making sure to complete the newly added COVID-19 questions and update any changes to your treatment (question 9) and quality of life (questions 20 and 21). We need your help to provide “big data” to increase the power of this research by increasing the number of participants. Together, we can help answer some important questions in WM research as we search for a cure. For further information on WhiMSICAL Registry, click here WhiMSICAL Registry and Frequently Asked Questions or contact [email protected].

WMozzies information flyer

September 13, 2017September 13, 2017

WMozzies information flyer

The new WMozzies flyer is now available. It provides information about WMozzies and the support services provided to Waldenström's Macroglobulinemia patients throughout Australia. It is produced in a 3 column double sided fold-out format as shown below.