ASH Abstract: MPNs and Depressive Symptoms, Diagnosis, and Associations

Patients with a myeloproliferative neoplasm (MPN) diagnosis experience a high systemic symptom burden and a spectrum of physical, financial, mental and emotional stressors. The impact of mood disturbances in MPN patient is not well understood. It was recently identified by our study team that symptoms of depression coexist with the prevalent and debilitating symptom of fatigue in patients with MPNs. The purpose of this study is to comprehensively describe the experience of depressive symptoms in MPN patients.

Methods: A 70-item internet based national survey regarding fatigue and mood symptoms was developed by MPN investigators and patient/advocates and refined by the Mayo Clinic Survey Research Center. The Patient Health Questionnaire-2 (PHQ) was completed by all respondents and utilized to assess symptoms of depression. Survey responses were compared between those who endorsed having current symptoms of depression (defined as a PHQ-2 score ≥ 3) and those without symptoms of depression (defined as a PHQ-2 score < 3).

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Impact Biomedicines to Present Retrospective Data on Fedratinib at the 2017 ASH Annual Meeting

SAN DIEGO — November 27, 2017 — Impact Biomedicines today announced that retrospective data on fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV), will be presented in a poster at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.
The details of the presentation are as follows:
Session: 634 – Myeloproliferative Syndromes: Clinical: Poster III
Poster Title: Case Series of Potential Wernicke’s Encephalopathy in Patients treated with Fedratinib
Presenter: John Hood, Ph.D.
Date: Monday, December 11, 2017
Presentation time: 6:00-8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
About Impact Biomedicines
Impact Biomedicines (“Impact”) is pioneering the development of life changing treatments for patients with complex cancers. The Company’s pipeline is centered around fedratinib, a potent and highly selective oral small molecule, JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV).

ASH: 1663 Germline ERBB2 Variants Associate with MPNs

Background: The myeloproliferative neoplasms (MPN) and myelodysplastic syndrome (MDS) are usually sporadic diseases, however, familial cases are well-described. Approximately 10% of MPN cases display familial clustering, and there is a 5-7 fold increased risk of developing an MPN among first degree relatives of MPN patients. While familial MDS appears to be less common, identification of multiple predisposition genes has led to the incorporation of this entity into the most recent WHO classification of myeloid neoplasms. Identification of predisposing germline mutations in the myeloid malignancies has led to a better understanding of the pathophysiology of these diseases and improved clinical care. However, many familial MDS and MPN cases exist in which the inherited genetic lesion is unknown. Our recent investigation of a multigenerational family with inherited MDS/AML marked by erythroid hyperplasia identified a missense variant in ERBB3that co-segregated with the disease phenotype (1). In this study, we investigated a kindred with an autosomal dominant familial cancer syndrome characterized by both MPN and solid tumor malignancies.
Program: Oral and Poster Abstracts
Session: 635. Myeloproliferative Syndromes: Basic Science: Poster I
Saturday, December 9, 2017, 5:30 PM-7:30 PM
Bldg A, Lvl 1, Hall A2 (Georgia World Congress Center)

Evan M Braunstein, MD, PhD1, Donna Marie Williams, PhD1*, Igor Makhlin, MD1, Aparna Pallavajjala2*, Christopher D Gocke, MD2*, Nara Sobreira, MD3*, Kala Visvanathan, MD4*, Linda Resar, MD5, Jerry L. Spivak, MD1 and Alison R. Moliterno, MD1

1Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD
2Department of Pathology, Johns Hopkins School of Medicine, Baltimore, MD
3Department Pediatrics, Johns Hopkins School of Medicine, Baltimore, MD
4Medical Oncology and Epidemiology, Johns Hopkins University, Baltimore, MD
5Division of Hematology, Department of Medicine, Johns Hopkins University SOM, Baltimore, MD

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Incyte Announces Pivotal Clinical Trial of Ruxolitinib for Treatment of ET

WILMINGTON, Del.–(BUSINESS WIRE)–Nov. 15, 2017– Incyte Corporation (Nasdaq:INCY) today announced that the first patient has been treated in the RESET pivotal trial evaluating ruxolitinib (Jakafi®) compared to anagrelide for the treatment of patients with essential thrombocythemia (ET) who are resistant to or intolerant of hydroxyurea (HU).

“We are pleased to treat the first patient in our pivotal trial evaluating ruxolitinib as a treatment for ET, a rare blood cancer that can lead to life-threatening complications,” said Steven Stein, M.D., Chief Medical Officer, Incyte. “We look forward to building on the clinical evidence for ruxolitinib and to advancing this trial to help address the needs of higher-risk patients with ET, who are resistant to or intolerant of HU and currently have limited treatment options.”

ET is a rare, chronic blood cancer, part of a group of related blood cancers known as myeloproliferative neoplasms (MPNs), characterized by increased platelet production, a white cell count above the normal range, persistently elevated platelet counts with normal red blood cell mass and the absence of prominent bone marrow fibrosis.1 An increased platelet count can increase the risk of thrombosis. Thrombosis can, in turn, lead to serious health problems including heart attack or stroke. Vascular complications and transformation to myelofibrosis (MF) or acute myeloid leukemia (AML) are the major causes of increased morbidity and mortality in patients with ET.2,3

About the RESET Study

The randomized, double-blind, double-dummy pivotal study (NCT03123588) is evaluating the safety and efficacy of ruxolitinib versus anagrelide as a treatment of patients with ET. The study is expected to enroll approximately 120 patients, 18 years or older, diagnosed with ET who are resistant to or intolerant of HU, with a screening platelet count of >650 × 109/L and white blood cell (WBC) count of >11.0 × 109/L.

The primary endpoint of this study is the proportion of patients who achieve platelet and WBC control over 1 year of follow-up. Key secondary endpoints include safety and tolerability and the proportion of patients who achieve complete remission (CR) or partial remission (PR). For more information about the study, please visit

About Jakafi® (ruxolitinib)

Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. Food and Drug Administration, as Jakafi® (ruxolitinib), for treatment of people with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.

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Effective treatment of low-dose decitabine in MDS/MPNs

Objective: Primary myelofibrosis (PMF) is one of the Philadelphia negative myeloproliferative neoplasms (MPN). The main clinical features are obvious physical symptoms and symptomatic splenomegaly. It may be converse to leukemia and has a shortened life expectancy. Nowadays, the therapy for PMF is aimed at maintaining comfort and there is no curative treatment. PMF with myelodysplastic syndrome (MDS), called MDS/MPN-u, is rare and the treatment is complex. In this study, we want to discuss an effective treatment for MDS/MPN via a case report and literature review.
Materials and methods: A female patient was diagnosed with MDS/MPN through bone marrow cytology, immunology, cell genetics, molecular biology, and pathology. She received thalidomide and prednisone as initial treatment. Ten months later, the first-line therapy had failed, she presented with clinically relevant pancytopenia and increased blasts in bone marrow. Because decitabine is one of the first-line treatments for MDS and the patient was frail, she received low-dose decitabine as second-line therapy. Decitabine was administered at 15 mg/m2 once a week for 3 weeks, in a 4 week cycle. If there was improvement the treatment interval was prolonged.
Result: After one cycle, the blasts in bone marrow were decreased to 0.5%. After four cycles, she felt comfortable and hematological improvement was achieved. The treatment interval was prolonged. After eight cycles, the spleen reduced to 2 cm under the rib, and she achieved complete hematological remission. After ten cycles, the mutation of JAK2/V617F expression was decreased from 60.63% to 0.01%. During the therapy, the patient presented with grade III–IV hematological toxicity after the first two cycles, but there were no side effects after subsequent cycles.
Conclusion: Our research showed that low-dose decitabine may be an effective treatment for MDS/MPN, especially in improving physical symptoms and achieving hematological remission. Besides, it may be possible to reverse positive JAK2 mutation.

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Ruxolitinib vs Allogeneic SCT for Patients With MF According to Donor Availability


The present study will be a multicenter, prospective phase II-study comparing efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.


This study is a multicenter, prospective phase II-study compares efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.

In this study will further assess and compare the safety and efficacy of study treatments/ induction therapy in both study arms on spleen reduction, improvement of constitutional symptoms, QOL, toxicity, fibrosis regression, development of GvHD as well as chimerism, engraftment, relapse incidence, disease related mortality, outcome and overall survival.

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New MPN Progression Marker Project

The Progression Marker Project: A Rigorous Effort to Identify the most Relevant and Influential Markers for Therapeutic Intervention and Challenge the Natural History of Myeloproliferative Neoplasms

We Need Your Help

Only a very few genetic mutations have been linked to MPNs and despite the one approved therapy that targets the JAK2 mutation, this targeted therapy does not lead to a cure nor does it help every MPN patient.

The MPNs represent a group of diseases in the blood cancer space that affect normal blood cell production in the bone marrow. The bone marrow causes an overproduction of one or more blood cell types (red cells, white cells or platelets). MPN physicians are struggling to understand which mutations and / or biologic markers are the primary risk factors for disease progression and poor patient outcomes. Since risk-adapted therapy for some cancers is becoming standard of care, there is an urgent need for a precise risk stratification model/understanding to assist in therapeutic decision-making and research. While progress has been made, there remains a significant opportunity to improve patient stratification to optimize treatment and to develop new therapies for high-risk patients.

Results from this highly collaborative research effort, with the best and brightest in the MPN research space and industry partners, are expected to significantly advance our understanding of risk factors and molecular changes that lead to disease progression; shift the paradigm of treatment potentially delaying progression, and identify new drug targets for therapeutic intervention.

Why now?

“We need rigorous data to move physicians away from “watch and wait” to treatment strategies based on an understanding “why, how and when” people with MPNs should be treated. The Progression Marker Project is designed to provide us with this essential yet missing data.”
– Srdan Verstovsek, MD Anderson, Principal Investigator for the Progression Marker Project

Project Goals

  • Identify within the MPNs e.g., ET, PV and MF, the most relevant markers of disease progression and establish the most appropriate time to effectively intervene therapeutically
  • Identify which patients are at greatest risk to progress to MF
  • Provide patients with treatment strategies to prevent conversion to Myelofibrosis
  • Identify biomarkers and molecular changes that most commonly occur in this heterogenous population to identify novel or relevant therapeutic targets

The Strategy:

  • Convene a panel of thought leaders to establish the protocol and mechanisms for building the database and biobank required to meet the Project goals
  • Partner with industry, motivated patients and MPN researchers to provide the financial and professional resources to support and execute the Project
  • Enroll 500 PV and ET patients for a 5-year study
  • Share data with MPN researchers and industry partners in an open-source competitive challenge. This truly represents a chance not only to integrate available data and analytical approaches to tackle this important problem, but also provides the ability to benchmark potential methods to identify those with the greatest potential to yield patient care benefits in the future

Why Help?

The Progression Marker Project is expected to produce actionable results within 5 years by building a network of researchers sharing data and creating solutions to treating patients at risk for progression of their myeloproliferative neoplasms.

This project will lead to new treatments, new combinations of treatments and, new insights regarding the most appropriate time to treat patients.

The cost of the Project is estimated at $5.5 million and the impact to patients’ lives is estimated to be priceless.

Visit to learn more

Blog: Living in Paradox

November: You Are Not Alone

By Marina Sampanes Peed

When people with a MPN gather, it is easy to forget that these blood cancers are rare. MPN advocates from two dozen countries describe patient experiences that are eerily similar:

  • Correct diagnosis follows one or more years of signs or symptoms.
  • Clinician who made diagnosis did not provide current information about the disease nor treatment options. (Some discount while others magnify the risks.)
  • Delayed in receiving appropriate treatment.
  • Many “Quality of Life” symptoms are not recognized and remain untreated, especially in women.
  • Access to and participation in clinical trials is difficult.
  • Support from the MPN patient community is highly valued.

Most hematologists rarely, if ever, encounter a patient with a MPN. Because the diseases manifest differently, experience with a few patients is not sufficient to develop expertise. If your local physician is not proactive, you may insist he/she confer with a MPN specialist regarding your care. The experts regularly consult with general practitioners and hematologists regarding management of their MPN patients.

If your local physician is not proactive, you may insist he/she confer with a MPN specialist regarding your care. The experts regularly consult with general practitioners and hematologists regarding management of their MPN patients.

The patient experience was a recurring theme in Frankfurt am Main, Germany October 27-29, 2017 when 43 patient advocates from 24 countries gathered with several clinicians at the 2nd MPN Horizons International Conference for MPN Advocates. More reports coming soon.

Here are just a few headlines:

“These diseases will eventually be called by their mutations rather than Essential Thrombocythemia, Polycythemia Vera, and Myelofibrosis.”

~ Martin H. Ellis, MD, Meir Medical Center and Tel Aviv University, Chairman of Israel MPN Working Group

“Patient Reported Outcomes are, in fact, doctor reported outcomes; not actually from the patient. There is work to be done.”

~ Sarunas Narbutus, European Patients Academy on Therapeutic Innovation (EuPATI)

“The aging of hematopoietic stem cells may play a role in the mutations, and the health of the niche where the stem cells reside is also important… There appears to be hierarchy among mutations and competition among them.”

~ Saghi Ghaffari, MD, PhD, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai

“When someone receives a MPN diagnosis, their cognitive map lights up based on beliefs, knowledge, expectations, psycho-emotional reactions, and personality traits.”

~ Ketti Mazzocco, PhD University of Milan, Department of Oncology and Hemato-oncology; European Institute of Oncology



Atlanta MPN Program: Interesting Speakers Share Valuable Information

Last week we hosted another MPN education program in Atlanta.  Being in Atlanta was a bit nostalgic and heartwarming.  Bonnie Evans, longtime partner and caregiver to Joe (now deceased), spoke about why she became involved on a grander scale in the MPN community.  The Atlanta support group, founded by Bonnie, is still going strong.  Marina Peed shared her experiences and journey as a patient and transplant survivor. Her unique challenges and ultimate cure led her to a new role as a strong advocate for MPN patients. Dr. Jennifer Powers, a pharmacologist and MPN patient, imparted valuable information for those struggling with insurers and outlined the benefits of specialty pharmacies. Maria Hanik gave an informative overview of the BMT program at Northside Hospital Cancer Institute.

Our clinicians and researchers, Drs. Rampal, Michaelis and Winton shared updates on current clinical trials, what MPNs look like and how the body reacts to polycythemia vera, essential thrombocythemia and myelofibrosis. The feedback we hear from patients after each educational program fuels our commitment to continue providing the best possible events and resources available.


Co-Pay & Insurance Assistance Resources


For Jakafi

Patient Access Network: Phone: 1-866-316-7263

PAN Foundation has co-pay assistance program for Myeloproliferative Neoplasms and many other diseases.

Good Days from CDF (formerly known as Chronic Disease Fund, Inc):     Phone:  1-972-608-7141

Good Days is a co-pay assistance organization and Myeloproliferative Neoplasms are covered.

HealthWell Foundation:

Phone:  1-800-675-8416

NeedyMeds: Phone: 1-800-503-6897

Partnership for Prescription Assistance:

Phone:  1-888-477-2669

Patient Advocate Foundation Co-Pay Relief Program:

Phone:  1-866-512-3861

CancerCare Co-Payment Assistance Foundation:

Phone:  1-866-552-6729

Rx Outreach:   Phone:  1-800-769-3880