ASH Abstract: Effective Treatment of Low-Doses Decitabine in MPNs with JAK2/V617F

Primary myeofibrosis (PMF) is one of the Ph negative myeloproliferative neoplasms (MPN). The mainly clinical features are obviously physical symptoms and symptomatic splenomegaly. It may be converse to leukemia and has shortened life expectancy. Nowadays, the therapy of PMF is aimed at maintaining comfort and there was no effective treatments. PMF complicated with myelodysplastic syndrome (MDS), which is named as MDS/MPN-u, is a rare case, and the treatments are confused. In this study, we want to discuss an effective treatment in MDS/MPN via a case therapy and literature review.

A 55-year-old woman presented with fatigue and chest distress for one month was admitted in our hospital. Physical examinations showed anemic appearance and splenomegaly which was four fingers under lib. A routine blood count test showed pancytopenia. A bone marrow examination showed dysplasia and fibrous tissue proliferation. The JAK2/V617F mutation was positive and the expression was 60.63%. The chromosome karyotype showed 47, XX, t (1; 20) (p11.2; q11.2), +9,-13, +21. She was diagnosed as PMF complicated with MDS (MDS/MPN) according to WHO 2016 version of hematologic neoplasms classification. She received thalidomide 100mg daily therapy combined with prednisone.

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The MPN Yoga Study: A Patient’s Story

MPN Patient Amy Wang Manning, Portland, Oregon

Amy Wang Manning.

As I typed one day at work this past spring, my left pinkie suddenly didn’t feel right. A moment later, my left thumb curled under; I couldn’t straighten it except by pulling it with my right hand. Then my entire left hand stiffened into a useless claw. And a tingling sensation was spreading rapidly up my left arm and to my shoulder. I told my boss I needed medical attention. Soon I was in a hospital emergency room, undergoing an MRI.
I didn’t have a stroke that March day. But several weeks later, a hematologist oncologist gave me a diagnosis I’d never heard of: essential thrombocytosis (ET), based on the clot I’d experienced, along with a platelet count of over 1 million and a positive test for the JAK2 mutation. He said I’d probably had ET for as long as 20 years. Suddenly, a lot of health symptoms and setbacks that I’d experienced over that time period made sense.
While I researched essential thrombocytosis online, I stumbled across a call for participants in a pilot study at Arizona State University. The researchers wanted to investigate whether patients with essential thrombocytosis and other myeloproliferative neoplasms could find some relief from symptoms such as pain, fatigue and insomnia by using a mobile app for guided meditation.
While I hadn’t experienced much pain, I’d been having bouts of fatigue and I’d struggled for years with insomnia, which seemed to be worsening. I had practiced yoga and found it helpful in managing my emotional, mental and physical health, so meditation seemed appealing. It certainly couldn’t hurt. And the eight-week study wasn’t asking much of participants: Fill out a few questionnaires, keep a daily sleep log, and wear a Fitbit to track my daily activity. I applied to the study and was accepted. The daily meditation sessions turned out to be very reasonable, just 10 minutes a day.
Now that the study’s finished, I feel calmer and more able to handle whatever comes my way with this disease. I continue to meditate with the app I tested. If nothing else, I am now better equipped to take a deep breath, let go of what I cannot control, and just focus on the moment – this moment, in which I am still here, living.

ASH Abstract: Imetelstat Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells

1654 Imetelstat, a Telomerase Inhibitor, Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells

Treatment of myelofibrosis (MF) patients with imetelstat (Imet), a telomerase inhibitor, has been reported to lead to clinical, morphologic and molecular remissions in a subset of patients (Tefferi A, et al. N Engl J Med. 2015; 373:908), suggesting that Imet has disease-modifying activity. The precise mechanism by which Imet induces such responses has however not been reported. In this study, we investigated the effects of Imet on MF hematopoietic stem/progenitor cells (HSC)/(HPC) to address this question.

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Impact BioMedicines Presents at ASH on Fedratinib

Impact Biomedicines Presents Analysis at the 2017 ASH Annual Meeting Suggesting that Fedratinib Did Not Increase Wernicke Encephalopathy Risk in Phase 2 and 3 Myelofibrosis Clinical Trials

SAN DIEGO–(BUSINESS WIRE)–Impact Biomedicines today presented a case review on fedratinib, a selective oral small molecule JAK2 kinase inhibitor that is being developed for the treatment of myelofibrosis (MF) and polycythemia vera (PV), in a poster session at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.

The poster titled “Case Series of Potential Wernicke Encephalopathy in Patients treated with Fedratinib,” demonstrated that patients treated with fedratinib in clinical trials did not experience a decrease in thiamine levels, and the prevalence of Wernicke Encephalopathy (WE) in the trials was less than originally perceived for patients with myeloproliferative neoplasms.

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Incyte Reports Four Year Phase 3 Data Analysis Shows Durability of Response of Jakafi (ruxolitinib) in Patients with PV

Incyte Corporation (NASDAQ: INCY) today announced new 208-week (4-year) follow-up data from the ongoing, global, multi-center, open-label Phase 3 RESPONSE study of Jakafi® (ruxolitinib) comparing the efficacy and safety of Jakafi with best available therapy (BAT) in patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU). The pre-planned data analysis showed a durable primary response to Jakafi in patients with PV who are resistant to or intolerant of HU and the overall safety profile for Jakafi remained consistent with previously reported 80-week RESPONSE data.1 The results were shared in an oral presentation today at the 59th American Society of Hematology (ASH) Annual Meeting 2017 in Atlanta, Georgia.

“With 30 months of additional follow-up, the four-year RESPONSE data analysis presented today at ASH further reinforces the potential of Jakafi as a long-term option for patients with PV,” said Peg Squier, M.D., Ph.D., Head of U.S. Medical Affairs at Incyte. “Given the few treatment options available to treat this chronic and progressive blood cancer, these long-term safety and efficacy data are meaningful to patients with uncontrolled PV.”

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Italfarmaco Announces Givinostat Clinical Trial Presentations at the 59th American Society of Hematology (ASH) Annual Meeting

MILAN–(BUSINESS WIRE)–Italfarmaco Group, a specialty pharmaceutical company, today announced both an oral and a poster presentation at the 59th American Society of Hematology (ASH) Meeting & Exposition held in Atlanta, Georgia from December 9 – 12, 2017. The presentations cover data from Italfarmaco’s Phase II development program for its proprietary compound Givinostat, in development to treat Polycythemia Vera, a rare blood disease with orphan drug designation. Italfarmaco will announce the results through a press release following the presentations.

Oral Presentation

Title: A Two-Part Study of Givinostat in Patients with Polycythemia Vera: The Maximum Tolerated Dose Selection and the Proof of Concept Final Results

Presenter: Prof. Alessandro Rambaldi

Session Name: 634. Myeloproliferative Syndromes: Clinical: Phase I/II Trials of Novel Agents in MPNs

Date & Time: Saturday, December 9, 2017: 4:00-4:15 PM EST

Location: Bldg C, Lvl 2, C208-C210 (Georgia World Congress Center)

Link to the ASH conference abstract: https://ash.confex.com/ash/2017/webprogram/Paper100916.html

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ASH Presentation-Personalized Medicine in Blood Cancers

Foundation Medicine and Collaborators to Present New Data at the American Society of Hematology (ASH) Annual Meeting that Supports Use of FoundationOne®Heme to Advance Personalized Medicine in Blood Cancers

— New data demonstrate the value of comprehensive genomic profiling for informing clinical care and guiding use of targeted therapies, autologous stem cell transplantation and immunotherapy —

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Foundation Medicine, Inc. (NASDAQ:FMI) today announced that new data generated with FoundationOne®Heme, its comprehensive genomic profiling (CGP) assay for hematologic malignancies and sarcomas, will be presented at the American Society of Hematology (ASH) Annual Meeting. Data from a broad range of blood cancers, including acute myeloid leukemia (AML), myeloproliferative neoplasms (MPN), and non-Hodgkin lymphoma (NHL), including primary central nervous system lymphoma (PCL), demonstrate the value of integrating FoundationOneHeme into clinical care. The data presented is expected to demonstrate the potential for CGP to improve disease classification, to offer personalized prognostic information and to support therapeutic treatment decision making by informing treating physicians about the use of novel treatment options, including cancer immunotherapies.

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Impact Biomedicines Closes First Tranche of Oberland Capital Financing Following FDA Meeting

Management team expands to accelerate fedratinib global manufacturing and business operations

SAN DIEGO — December 1, 2017— Impact Biomedicines (“Impact”) today announced that it has achieved the first milestone in the Company’s previously disclosed $90 million financing with Oberland Capital, triggering the closing on the first tranche of $20 million. Impact also announced the expansion of its management team to include Randy Adams as Senior Vice President of Commercial Operations and Jeff Barker as Senior Vice President of Global Technical Operations.

“The first tranche of this financing follows a positive meeting with the U.S. Food and Drug Administration (FDA) bringing this much needed potential treatment option for myelofibrosis closer to patients in need,” said John Hood, Ph.D., Chief Executive Officer of Impact Biomedicines. “With Oberland’s financial support, we have made some important investments to ensure that we are well-staffed and prepared for U.S. commercialization.”

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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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