Momelotinib vs Ruxolitinib for Patients With Myelofibrosis

Momelotinib, an oral Janus kinase (JAK) inhibitor, is non-inferior to ruxolitinib in reducing spleen volume, though not for improving disease-related symptoms, among patients with myelofibrosis not previously treated with a JAK inhibitor, according to a presentation at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago.1

 

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Updates from Incyte on 2 Clinical Trials

Myelofibrosis

A Phase 2 Study of the Safety, Tolerability, and Efficacy of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis

The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of INCB050465 and ruxolitinib in subjects with myelofibrosis

 Talk to your doctor if you are interested in participating in this study.

To find a study center near you, call , then select option 1.

 You can also visit clinicaltrials.gov and enter NCT02718300 to learn more.

(https://clinicaltrials.gov/ct2/results?term=NCT02718300&Search=Search)

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Essential Thrombocythemia

A Double-Blind, Double-Dummy Phase 2 Randomized Study to Evaluate the Efficacy and Safety of Ruxolitinib Versus Anagrelide in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea (RESET-272)

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.

Talk to your doctor if you are interested in participating in this study.

To find a study center near you, call , then select option 1.

 You can also visit clinicaltrials.gov and enter NCT03123588 to learn more.

(https://clinicaltrials.gov/ct2/results?term=NCT03123588&Search=Search)

Dr. Ruben Mesa’s Message to Patients

Dear MPN Patient Community,

The Mayo Clinic Cancer Center is a very special place where people come together joined by a mission to put patients first in alleviating the burden of Cancer. The Mayo Clinic has been my professional home for my entire career since 1991, with 18 years in training and on the staff of Mayo Clinic Rochester and now 8 years at the truly amazing Mayo Clinic in Arizona. I am incredibly proud and honored for the opportunities that I have had at Mayo Clinic, as well as the great honor and joy of caring for patients alongside each of you over these years. It is therefore with genuinely mixed emotions that I announce that I will be leaving Mayo Clinic in August of 2017 to assume the Directorship of University of Texas, San Antonio Health Science Cancer Center, an NCI Designated Cancer Center. This unique opportunity presented itself at the end of the natural arcs of my current leadership responsibilities at Mayo Clinic as both Chair of the Division of Hematology and Medical Oncology, and Deputy Director of the Mayo Clinic Cancer Center.

The newly expanded Cancer Center in San Antonio brings together the long history and tradition of the NCI Designated Cancer Center – Cancer Therapy Research Center (CTRC) founded in 1974 by Charles Coltman,MD in San Antonio with the Institute for Drug Development (first led to success with Mayo Clinic Adjunct Professor Dan Van Hoff, MD), the complete cancer service line of the CTRC and the University of Texas Health Science Center in San Antonio, and finally, a partner level relationship with the MD Anderson Cancer Center that begins this fall as the UT San Antonio MD Anderson Cancer Center. I look forward to bringing the many lessons I have learned from my time at Mayo Clinic (how patient centered care can be woven together with impactful research and education), learned from teachers, mentors, colleagues, and friends both physician and allied health staff alike to the wonderful community of San Antonio, patients of the region and new colleagues.

My, and my extended MPN group’s focus, will remain unaltered on improving the treatment options for MPN Patients, advancing the utilization of stem cell transplantation for these patients, and a commitment to not only better understand the impact MPNs have on patients through symptom burden and decreased quality of life – but also developing feasible and useful non-pharmacological interventions (i.e. yoga, meditation, cognitive and mindfulness therapy, nutrition interventions) to be coupled with pharmacological approaches. My intent is the significant MPN center that will be developed at the UT Health San Antonio Cancer Center will be incremental, and not a replacement, to the programmatic efforts built at Mayo Clinic in Arizona. Expansion of clinical trials, new initiatives, collaborative grants, and core membership of the MPN Research Consortium to San Antonio are planned to begin rolling out after I begin my role as Director this fall.

MPN Patients who are currently receiving care at Mayo Clinic in Arizona both I and my staff are committed to your care needs being well addressed whether you will continue with my colleagues in Arizona, or you choose to transfer your care to the UT San Antonio Cancer Center. We will ensure that good contact remains between myself and my staff in Arizona regarding your care needs for the foreseeable future, as well have established a special contact point at UT San Antonio Cancer Center for care needs beginning this fall. Those that choose to continue their care with me in San Antonio I will work with our colleagues at Mayo Clinic for appropriate transfer of records. Although I have a start date in later August 2017, I do not yet have an official date my MPN clinic will open (given need for completion of Texas Medical Licensure etc.) at this moment your information will be gathered and appointments to be forthcoming.

 

Patient Contact Information for MPN Patients –
Epp Goodwin – Senior Referral Coordinator
CTRC/UT Health San Antonio Cancer Center
7979 Wurzbach Rd, San Antonio, TX 78229
Phone: 210-450-5798
Contact Email: goodwine@uthscsa.edu

My Contact Information – August 2017

Ruben A. Mesa, MD, FACP
Director, UT Health San Antonio Cancer Center
Mays Family Foundation Distinguished University Chair
7979 Wurzbach Rd, San Antonio, TX 78229
Phone: 210-450-1406

Sincerely,

Ruben

Ruben A. Mesa, MD, FACP


CONSULTANT HEMATOLOGIST-MAYO CLINIC in ARIZONA
Chair, Division of Hematology & Medical Oncology
Deputy Director, Mayo Clinic Cancer Center

Chair, Arizona Cancer Coalition
Professor of Medicine
P: 480-342-4800
F: 480-301-4675
mesa.ruben@mayo.edu

 

Unexplained Leukocytosis, Thrombocytosis in MPN Warrants Evaluation for CML

A chart review of patients with myeloproliferative neoplasms (MPN) who developed unexplained leukocytosis or thrombocytosis demonstrated that these patients should be evaluated for chronic myelogenous leukemia (CML), according to a case series described in a published abstract from the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting.1

Myeloproliferative neoplasms (MPN) are classified by the presence of the Philadelphia chromosome (Ph). Ph-negative MPN typically possesses driver mutations of JAK-2, MPL, and CALR genes. CALR is involved with apoptosis and cell proliferation, and MPL leads to thrombopoietin receptor stimulation. JAK-2 mutations render hematopoietic stem cells more sensitive to growth.

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A Patient Story: Living 32 Years with an MPN

By Ron K.

Fortunately, my MPN journey has been a long one. I was first diagnosed 32 years ago and I know have post-Polycythemia Vera Myelofibrosis. Looking back it was almost archaic in nature. Today the progress has been immense on many fronts, not just in treatment, but with treating the patient as a whole. As many of you know, there is no exact recipe book for life with cancer and its treatment journey is unique.  My hope is that sharing some of my experiences may help someone else along the way.

There is a quote by Albert Einstein that reflects my approach to my medical journey

Learn from yesterday, Live for today, Hope for tomorrow. The important part is to not stop questioning.

Learn, Live, Hope – three good words for any cancer patient.

As those with an MPN know this is a chronic neoplasm and unfortunately there is no treatment yet to cure this illness. Some of the latest MPN and MF drugs do improve one’s quality of life and even appear to extend one’s survival, a significant milestone from what was available just a handful of years ago. Finding ways to slow or stop the progression is my only hope until medical knowledge can surpass me with a real solution.

Having a chronic cancer and having it for a long time has enabled and encouraged me to evolve into a healthier lifestyle. One does not have to change everything overnight but just evolve and continue to build healthier ways, one step at a time. While there is no evidence that a healthy lifestyle will cure me, it is something I can do and have control over.

This all started back in 1985 when I went to my family doctor with a cold and I mentioned that my colds seem to last longer and hit me harder than others.  A simple blood test began my journey.  I would not be alive today if I followed my first hematologist’s treatment plan. He wanted to treat me in a traditional manner at that time, with a radio isotope that kills off some of the marrow, they know now that treatment leads to an acute leukemia years down the road. Instead my intuition questioned this approach, since I was only 31 and I thought that I might need that marrow later in life.  I called a major metro medical center and asked for an appointment with their Chief of Hematology. Unknown to me at the time, and not commonly termed in the 1980’s, I was going to get something called a second opinion. I got an appointment and his approach was quite different. He wanted to understand me, my rate of progression, severity, etc as that would lead him to figure out how to best treat me. He even commented that the current radio isotope treatment (my first hematologist’s plan) to kill off marrow was likely going away as he saw it.

Today, I am a patient of Dr. Mesa’s at the Mayo Clinic.  I found that they could answer all my questions. Answers to questions that my previous hematologists could not answer. They have knowledge that is very leading edge as to direction of MPN treatments as they are part of an elite group of hematologists that are making many of the MPN discoveries, designing the clinical trials and studies with other key hematologists around the world.  So my personal doctor advice is to make sure you find the right doctor and treatment strategy for you. Listen to your intuition and ask questions, seek information and acquire knowledge in your cancer and health. Expect answers or find those professionals that can give them to you. While I did not need a MPN specific doctor early on I do need one now as I am living well beyond the averages.

For me, finding the right doctor has been a key in my cancer treatment journey. Getting a second opinion is also key. What you will likely find is the different doctors will agree on the diagnosis but their treatment strategy will often be different.

I attend conferences, seminars, and special programs such as those that the MPN Advocacy and Education International puts on. I have done online webinars or conferences as well as belong to online MPN listing groups to learn and share information. I find this information to be helpful to ask the right questions to my doctors and understand what may be option in the future.

I believe support makes a difference. It is helpful to have support from your spouse, kids, parents or friends. I also find that support groups can be very helpful as well. As they can fill a support void that is missing from one’s non-cancer family and friends. This is no fault of family, spouses, or friends in that they do listen and they do try to understand and relate and they do support the cancer patient, but I have found that belonging to a cancer support group often helps to fulfill things non-cancer patients cannot quite grasp. I have belonged to both hematology specific support groups and all cancers support groups. You might want to try both and see if one works better than another.

 

As I live my life, I am grateful for every day. Everyday is a great day, just some are even better than others.  I even celebrated the point in my life where I have lived longer with cancer as part of my life than without cancer. That was at about 31 1/2 years and I had a bakery make me a cake to celebrate the occasion. The cake was symbolic of my MPN journey and view of life. I celebrated this occasion with my all cancer support group. So while cancer is a part of my life I run my life with cancer being a part of it and I do not let my cancer rule over me.  I am hopeful that a cure is in our much sunnier MPN journey. May the best be yet to come, and enjoy the day!

Bone marrow histology for the diagnosis of ET in children: a multicenter Italian study

Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) that mainly affects middle-aged patients. Although pediatric cases occur, they are rare, and their molecular features considerably differ from the adult counterparts: JAK2V617F mutation occurs in only 25% of cases,1 CALR mutations are found in <10% of patients,2 and the MPLW515L mutation is anecdotal.3 Overall, <40% of children with unexplained, long-lasting thrombocytosis have a clonal marker of ET.2

After the release of the 2001 World Health Organization (WHO) classification,4 bone marrow (BM) evaluation has become a cornerstone of ET diagnosis. However, the majority of studies has focused on adults, and little is known about the role of BM biopsy in pediatric ET. In fact, BM biopsy is seldom performed in children with a clinical picture of ET due to the invasiveness of the procedure. The main objective of this study was to explore the relevance of BM histology in children with high platelet counts in order to identify possible differences in: (1) primary vs reactive/secondary thrombocytosis (PedST) of childhood; and (2) pediatric (PedET) vs adult (AdET) cases of ET.

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The Volunteer Paradox: Why I’m Addicted to Volunteering

When Ann Brazeau asked me to write about why I volunteer, I thought this piece would be easy. When I help other people, I feel better about myself. What distinguishes volunteering from other activities? How do I feel better and what are the conditions that work best for me? And of course, the age old question: why?

It started innocently enough. Bake sales and car washes for school fundraisers. Fill packets with dimes for the March of Dimes. Collect quarters for an orphanage in Greece. By the time I was in high school, I sought ways to help people directly: tutoring other students, activities with seniors, food/book/clothing drives, and became a blood donor. I discovered that organizing events came naturally to me. And I loved making posters.

The expression, “it’s in your blood” signifies a trait that appears to run in families. Philanthropy, my Greek grandfather explained, means “love of mankind” based on the Greek words “philios” (brotherly love) and “anthropos” (people/mankind). Volunteerism is a means of expressing philanthropy. I witnessed my grandparents show compassion and care for people who needed a helping hand both informally and through philanthropic organizations. They lived through the Great Depression; they lived modestly and gave generously of their time, talents, and money in many ways.

From Hobby to Addiction

The more I do, the better I feel. Volunteer work quickly became an addiction. The addiction was fueled by numerous opportunities at my university. I recruited volunteers, developed peer mentoring programs, and advocated for student issues. While some students sold their plasma for beer money, I donated blood regularly. These works significantly shaped my university experience. If I’m honest, I spent far more time working on volunteer activities than my coursework. If there were a degree in extra-curricular activities, I would have been valedictorian.

Volunteering is a socially acceptable addiction. It is respected and rewarded by society. And perhaps even more powerful, the dopamine release is an incredible mood-lifter. I derive a deep sense of worthiness from giving my service. I feel alive; that I am living my purpose.

My Social Life

After college, volunteering was a great way to meet people and network in Boston, my new home. I even met the man who became my husband in the Boston Jaycees. I discovered interest in volunteer work that is one-off from more popular, publicized causes. While in Boston, I volunteered with youth leadership, community development, women’s health, and victims of sexual assault.

When we moved to Georgia, I chose to seek out volunteer opportunities. It’s a great way to meet people who share similar interests. Golf is my husband’s primary hobby. Volunteering is mine. I spend a lot more time with my hobby than he does with his.

One year, my husband gave me a t-shirt that read “STOP ME before I volunteer again!” He had a point. I often used my volunteer work to avoid tasks I didn’t care for.

When I was out of commission during my stem cell transplant journey, I couldn’t resist sharing the experience step-by-vulnerable-step in my CaringBridge site. I wanted my experience to benefit others. Learning from transplant survivors helped me get through the many rough patches, and I wanted to pay it forward. This also gave me another sense of purpose.

Many gifts from volunteer work

The return on investment is priceless:

  • I’ve developed friendships with most interesting people of all ages from all walks of life.
  • My worldview expands as I learn a lot about people, science, the arts, public policies, economics, and technology – without paying tuition.
  • I am reminded of the blessings in my life. The gratitude I feel inspires me to share with others. I have fun – learning new things, meeting new people, and trying new things.
  • My sense of self-worth is enhanced with every good work. If only calories would burn at the same rate!

When I become too focused on my problems – what’s hurting today, the latest CBC results, going from one specialist to another seeking answers, dealing with insurance – I feel miserable and cynical. Even I tire of my negative self.

My go-to remedy is to give myself a 10-20 minute pity party (depending on the severity of the issue). Cry it out, cuss it out, write it out, breathe it out, walk it out; whatever works. Then I set those issues aside and turn my attention to the world. What can I do to get outside myself and put some positivity into the world? Try it! It works!

I wish you the peace and joy that comes from being in service to others.

ET: A review of clinical features, diagnostic challenges and treatment modalities

Essential thrombocythemia: a review of the clinical features, diagnostic challenges, and treatment modalities in the era of molecular discovery.

Abstract

Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm that is associated with diminished quality of life, thrombohemorrhagic complications, and transformation to myelofibrosis (MF) and acute leukemia (AML). The important recent discoveries of driver mutations, including the calreticulin gene in addition to JAK2 and MPL, have led to a greater understanding of disease pathogenesis and set the stage for the advent of more sophisticated prognostic, diagnostic, and therapeutic strategies. In this paper we summarize recent studies describing the molecular basis of ET. We review the prognostic importance of establishing a ‘true’ ET diagnosis, as well as risk factors for the development of adverse outcomes including thrombosis, AML (2% risk at 15 years), and MF (9% risk at 15 years). Finally, we discuss the decision to initiate treatment and assess the quality of evidence supporting the use of established, available therapies as well as novel treatments. Special situations, such as pregnancy, familial ET, and extreme thrombocytosis will also be discussed.

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