WhiMSICAL Registry Frequently Asked Questions (FAQs)

FAQ Introductory

FAQ #2 Registration and Consent

Points marked about Tissue Collection may be ignored as there is no tissue collection involved in CART-Wheel.

For this study, there are five different levels of consent. It is recommended that all five be selected and that in option 2 details of your hematologist be provided.

FAQ #3 General tips for completing questionnaire:

How to save, logout and return later
1. Making use of the “Tip” boxes and hovering over the underlined words
1. Navigating through the questions using the banner
1. Questions to skip once you have entered WM as diagnosis.

FAQ #4 Questions 1 to 3, and 7

Filling out personal details. This is done to ensure the study is conducted correctly, with each person’s details linked to their consent. It is also to ensure there are not duplicate entries for data analysis. All information is stored very securely, kept confidential and is not shared with any third party outside of an ethically-approved research project. WhiMSICAL study investigators can only access your de-identified data to increase the layers of confidentiality. For an additional layer of security, personal details are stored on a separate server to the clinical data entered in the rest of the questionnaire.

Q3: Enter “Wa” and select Waldenstrom’s Macroglobulinemia from the drop-down menu.
- For completing the bone marrow biopsy results, don’t worry if your report does not have the specific information asked here. Fill in what you have.
- If you are unsure of the specific date of diagnosis, hover over “Estimated date” for an explanation of how to complete it

Once you click on save and next, a welcome to the WhiMSICAL project will pop up and the link will take you to the next relevant question -> Q7

FAQ #5 Question 9-12, completing treatment details:

Needs just going through with share screen
- Additional points for discussion:
  - Enter clinical trial at Q9 and in Q12
  - Treatment data is the most important to help researchers determine the effectiveness of different therapies and treatment practices
  - If you are unsure of the specific date of treatment, hover over “Estimated date” or an explanation of how to complete it

FAQ #6 Questions 15,16 and 18, additional cancers, family history and tumor gene test

These questions are to about risk factors for developing cancer and to learn more about how different types of WM present and respond to different treatments.

Q18: this is specifically about special gene tests that were performed on your tumour biopsy or bone marrow biopsy, not a test to see if you carry a gene that puts you and your family at risk of cancer. Typical tests for WM include MYD88, CXCR4, “cytogenetics”, next generation sequencing.

FAQ #7 Q19 COVID questions

FAQ #8 Q19 Symptoms and pathology:

Key timepoints are diagnosis, before 1^st cycle and after last cycle of treatment. If on continuous therapy, e.g. ibrutinib, then complete every 3-6 months. If you have never had treatment, yearly results or whenever your symptoms change.

FAQ #9 Q20 and 21: QoL and psychosocial questions:

Key timepoints are diagnosis, before 1^st cycle and after last cycle of treatment. If on continuous therapy, e.g. ibrutinib, then complete every 3-6 months. If you have never had treatment, yearly results or whenever your symptoms change.

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.