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• Provide encouragement and support to WM patients and caregivers; • Provide a means for patients and caregivers to

WALDENSTROM’S MACROGLOBULINEMIA. MISSION AND PURPOSE. • Provide encouragement and support to WM patients and caregivers; • Provide a means for patients and caregivers to communicate;

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• Provide encouragement and support to WM patients and caregivers; • Provide a means for patients and caregivers to

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  1. WALDENSTROM’S MACROGLOBULINEMIA MISSION AND PURPOSE • Provide encouragement and support to WM patients and caregivers; • Provide a means for patients and caregivers to communicate; • Provide information and educational programs that addresses the concerns of patients and their caregivers; • Increase awareness of the issues related to WM; • Encourage and support research toward more effective treatments and a ultimately finding cure.

  2. International Non-Profit FoundationMembership composed of: • Patients • Family Members • Care Givers • Physicians • Researchers

  3. IWMF Member Services • Information Services • Communication Services • Publication Services • Educational Services

  4. IWMF Educational Events • Annual IWMF Educational Forum • Formal lectures from WM experts, breakouts sessions, guest speakers, research presentations; • To be held in Minneapolis Minnesota on June 24-26 2011 • DVDs available of all Ed Forums (including Venice 2010). • International Patient Educational Forums • 2nd International IWMF Patient Forum, Venice Italy October 10th, 2010 • WMUK Patient Seminar, London England Jan 30 2011 • Waldenstrom France, Paris France Sept 24 2011 • The IWMF will support international patient forums

  5. IWMF Research Activities • Basic Scientific Research Grants • Short Term Patient Studies • Medical and Research Workshops • Grants for Young Researchers to Conferences • Research Fellowships • Over 6+ million dollars U.S.

  6. The 2nd International IWMF Patient Forum On Waldenstrom’s Macroglobulinemia October 10th 2010 Venice, Italy

  7. WHAT I LEARNED IN VENICE • Sixth International Workshop on Waldenström’s Macroglobulinemia (IWWM-6); • Bi-annual premier scientific conference for WM; • Attended by close to 200 individuals from all over the world; • 3-day workshop consisted of a total of 80 presentations from over 90 speakers, including 14 young investigators, 5 special guest presentations, 5 debates, and 2 consensus panel discussions.

  8. WHAT I LEARNED IN VENICE • Pathogenesis, genetics, immunology, and molecular biology of WM; • Clinical features, treatments, and future directions in the treatment of WM; • Consensus panel discussions: • response criteria and treatment in primary therapy of WM, • response criteria and treatment options in salvage therapy of WM.

  9. WHAT I LEARNED IN VENICEas a patient, not as a physician

  10. WM IS A COMPLEX DISEASE!“To be conscious that you are ignorant is a great step to knowledge” B CELL(S) PLASMA CELL Waldenstrom’s macroglobulinemia (WM) is a neoplasm of small B lymphocytes, plasmacytoid lymphocytes, and plasma cells; bone marrow involvement and an IgM monoclonal gammopathy of any concentration.

  11. WM IS A COMPLEX DISEASE!“The beginning of knowledge is the discovery of something we do not understand” • The more knowledge we learn, the more we realize how little we truly understand. • Are there different subtypes of WM? • Not everybody responds the same to treatments. • Research support is an absolute necessity. • Participation in clinical trials is also an absolute necessity.

  12. THINGS ARE NOT ALWAYS WHAT THEY SEEM!“You know more than you think you know, just as you know less than you want to know.”

  13. THINGS ARE NOT ALWAYS WHAT THEY SEEM! “Real knowledge is to know the extent of one’s ignorance.” The assessment of treatment response in WM patients relies heavily on changes in serum IgM concentration. This may be misleading because of the following phenomena: - Variability in the presenting IgM levels and additional clinical parameters between patients;   - Changes in IgM do not always result in the improvement of symptoms; - Kinetics of IgM reduction and the effect of the treatment regimen used. Responses to some drugs (alkylating agents, purine analogues and monoclonal antibodies) are typically slow whereas responses to other drugs (Velcade) are rapid.

  14. THINGS ARE NOT ALWAYS WHAT THEY SEEM! “Fear always springs from ignorance.” There may be discrepancies between IgM and BM responses: 1. great bone marrow responses may be seen in patients with small reductions in IgM (selective depletion of B cells and persistence of the plasma cell component of the disease); 2. rapid drops in serum IgM levels may be seen with poor bone marrow and lymph node responses in some patients treated with bortezomib (Velcade); FROM: Dr. Roger Owen, HMDS Laboratory, St James's Institute of Oncology, Leeds, UK. BONE MARROW BIOPSIES ARE THE BEST!

  15. THINGS ARE NOT ALWAYS WHAT THEY SEEM!“Where ignorance is our master, there is no possibility or real peace.” • Progressive humoral immune suppression in indolent B-cell malignancies: - there is progressive depletion of normal B-cells over time. The depletion is independent of whether the B-cell disorder is stable or progressive. FROM: Dr. Any Rawstron, HMDS Laboratory, St James's Institute of Oncology, Leeds, UK. • We are also seeing an increase in CNS disease seen in WM “survivors” Be vigilant, not complacent!

  16. THINGS ARE GETTING BETTER!“Perhaps our eyes need to be washed by our tears once in a while, so that we see life with a clearer view again.”

  17. THINGS ARE GETTING BETTER!“For myself I am an optimist – it does not seem to be much use being anything else.” SPECIFICS PLEASE! • BENDAMUSTINE. The combination of Rituximab + Bendamustine (RBenda)is going to be a standard of therapy within the next 5 years; clearly a new option with a great safety profile for patients. • Dexamethasone + Rituximab + Cyclophosphamide (DRC )and RBenda are the 2 new standards for WM patients front line. • Rituximab + Fludarabine + Cyclophosphamide (RFC) is still a great option, but toxicity remains a problem. • Rituximab Maintenance? Some say too soon (not me) but there is definitely something there (huge Non Hodgkin Follicular Lymphoma study shows the benefit of R-maintenance.

  18. THINGS ARE GETTING BETTER!“What seems to us as bitter trials are often blessings in disguise.” • Autologous and allogenic transplants remain options for relapsed or refractory patients. • Do not rely solely on the serum IgM value at end of treatment as a measure of response as for most of the patients IgM levels continue to drop over time; up to 6 months following end of therapy, and for some patients up to 2 years post-cessation of therapy. • Long term survival rates for WM have improved greatly over the past 10 years. Median survival is now often quoted as 12/15 + years. • There has been a marked increase in WM patients participating in clinical trials. STAY INFORMED AND YOU WILL LIVE LONGER!

  19. CHALLENGES REMAIN“Man cannot discover new oceans unless he has the courage to lose sight of the shore.”

  20. CHALLENGES REMAIN“Trust yourself. Your know more than you think you do.” • Research funding; decreased government dollars. • Clinical trial participation; adults compared to children. • Newer, better, safer drugs. “If it works in Multiple Myeloma it must work in WM” Drugs may have very different effects and toxicity profiles than in MM. Investigators need to be careful not to use the drugs in WM too much by analogy with Myeloma • Survivorship issues: beyond 15 years median survival? Increase in CNS tumors; long-term immune system dysfunction, long-term side-effects of treatment... DONATE AND PARTICIPATE!

  21. Attitude is a little thing that makes a big difference. 

  22. Many thanks to: • My family • My friends • My healers • The IWMF community • The researchers • Roger and Alison Brown, Shirley D’Sa, Rita Flatley, and all the wonderful volunteers!

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