MDS is a bone marrow failure disorder
Recent News

Onconova Enrolls First Patient in Europe for Phase 3 INSPIRE Trial of Rigosertib in Higher-Risk Myelodysplastic Syndromes

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VII. International Eurasian Hematology Congress

from the Hematology Specialists Association, Turkey:


Dear Colleagues,

We are pleased to announce that VII. International Eurasian Congress of Hematology will be held between 13-16 October 2016.

In 2015, our congress has made progress on becoming one of the world’s leading international conventions with the participation of our colleagues from 42 different countries.

We believe deep in our hearts that with its special concept, the Eurasian Congress of Hematology will fill in a significant gap in our region and serve not only scientifically but also culturally.

Looking forward to meeting with your respected selves between 13-16 October, we would like to extend our kindest regards.

Prof. Dr. Giuseppe Saglio, Congress President
Prof. Dr. Birol Güvenç, Hematology Specialists Association

Inspire Phase 3 Trial

Read Press Release Here 

Clinical Study – IMerge Lower-Risk MDS


Current Status: Part 1 recruitment complete; Part 2 is not yet open for recruitment. For additional details, refer to the Geron press release (12Sep2016) at

Janssen Research & Development, LLC is conducting a Phase 2/3 clinical study referred to as “IMerge”, with the study drug Imetelstat, which is a first-in-class telomerase inhibitor. With its novel mechanism of action, Imetelstat may provide clinical benefit to MDS patients. In this study, Imetelstat is administered as a 2-hour intravenous infusion every 28 days.

IMerge is a study for people with MDS who need blood transfusions due to anemia (low red blood cell counts). People with low or intermediate-1 risk MDS that has relapsed or is refractory to Erythropoiesis-Stimulating Agents (ESAs) treatment are enrolled in the study. This study is being conducted at multiple hospitals and institutions around the world, in approximately 80 sites globally.

For more information about this clinical study, please visit (NCT02598661).

Lenalidomide Improves Quality of Life in Non-Del5q MDS

Treatment with lenalidomide (Revlimid) improved health-related quality of life (HRQoL) compared with placebo after 24 weeks for low to intermediate risk patients with non-deletion 5q myelodysplastic syndromes (MDS), according to a secondary endpoint analysis of the MDS-005 trial presented at the 2015 International MDS Symposium.

Results from MDS-005, originally presented at the 2014 ASH Annual Meeting, demonstrated that significantly more patients treated with lenalidomide achieved red blood cell transfusion independence (RBC-TI) of at least 56 days compared with placebo (26.9% [43/160 patients] vs 2.5% [2/79 patients]; P <.001), the primary endpoint of the multicenter randomized placebo-controlled study.

Based on the results of MDS-005, Celgene plans to submit a regulatory filing with the FDA in the second half of 2015.

In the current analysis, presented by Valeria Santini, MD, HRQoL was assessed as a secondary endpoint using the European Organization for Research and Treatment of Cancer QLQ-C30 questionnaire at baseline, every 12 weeks thereafter, and at discontinuation.

The questionnaire gathered data on fatigue, dyspnea, physical functioning, emotional functioning and global quality of life from 122 patients who received lenalidomide and 56 who received placebo. All patients included in the study had low or intermediate risk, transfusion dependent MDS that was non-del-5q, and were unresponsive or refractory to erythropoiesis-stimulating agents.

Patients underwent a baseline HRQoL assessment. At week 12, mean changes in HRQoL scores from baseline were not significantly different between treatment arms for the preselected domains. However, by week 24, HRQoL score changes favored lenalidomide versus placebo for all preselected domains. After adjusting for baseline scores, improvement was statistically significant for emotional functioning (P = .047) but not other domains.

“This analysis provides new insights into the clinical results of lenalidomide in non-del-5q patients,” said Santini. “We now have a better understanding of how achievement of transfusion independence impacts quality of life measures.”

RBC-TI of at least 8 weeks was associated with significant improvement (P <.01) across all preselected domains, with improvements also exceeding the prespecified threshold for clinically meaningful change.

In the study, the most common adverse events associated with lenalidomide were related to myelosuppression, including neutropenia (64.4% vs 11.4%) and thrombocytopenia (39.4% vs 7.6%). Grade 3/4 neutropenia occurred in 61.9% and 12.7% in the lenalidomide and placebo groups, respectively, and grade 3/4 thrombocytopenia occurred in 35.6% versus 3.8%.

The FDA granted lenalidomide Subpart H approval in 2005 for patients with transfusion-dependent anemia due to low or intermediate-1 risk MDS associated with a deletion 5q cytogenetic abnormality. The drug is currently only available under a special restricted distribution program. The Risk Evaluation and Mitigation Strategy (REMS) program is designed to avoid embryo-fetal exposure to lenalidomide, as the drug is linked to birth defects or death of unborn babies. Risk impacts both women and men, as lenalidomide can pass into human semen.

In 2006 lenalidomide was approved for use in combination with dexamethasone in patients with multiple myeloma who have received one prior therapy. In February 2015, the FDA expanded this multiple myeloma indication to include use in combination with dexamethasone in newly diagnosed patients. The drug also received approval for the treatment of mantle cell lymphoma in 2013 for use in patients whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib (Velcade).

Santini V, Almeida A, Giagounidis A, et al. The effect of lenalidomide on health-related quality of life (HRQoL) in patients with MDS: results from the MDS-005 trial. Leukemia Research. 2015;39:1s (suppl; abstr 116).

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Celgene Corporation Enters into Strategic Immuno-Oncology Collaboration with AstraZeneca to Develop PD-L1 Inhibitor Program for Patients with Serious Blood Cancers

Program to Focus Initially on Non-Hodgkin’s Lymphoma, Myelodysplastic Syndromes and Multiple Myeloma

Studies Planned to Start in Second Half 2015

BOUDRY, Switzerland–(BUSINESS WIRE)– Celgene International II Sàrl, a wholly owned subsidiary of Celgene Corporation (NASDAQ:CELG) today announced that it has entered into a strategic collaboration with MedImmune Limited, a wholly owned subsidiary of AstraZeneca PLC, to develop and commercialize an anti-PD-L1 inhibitor, MEDI4736, for hematologic malignancies. Approximately 1.75 million patients globally suffer from blood cancer and many are in need of new treatment options.

MEDI4736 is a human monoclonal antibody directed against programmed cell death ligand 1 (PD-L1), which helps tumors avoid detection by the immune system. Tumor cells use PD-L1 to turn off the immune system just as it begins to mount a response against them. MEDI4736 helps turn the immune system back on, allowing it to continue its attack on cancer.

“The potential of rationally combining immunotherapies such as MEDI4736 with existing and novel hematology compounds creates new opportunities for patients with blood cancers to live longer, better lives,” said Jacqualyn A. Fouse, Ph.D., President, Global Hematology and Oncology for Celgene. “This strategic collaboration leverages the deep expertise of AstraZeneca/MedImmune in immuno-oncology along with the experience of Celgene in the study and treatment of blood cancers. This collaboration advances Celgene’s already deep, diverse scientific platform to include checkpoint inhibitors, an area of significant promise in hematology.”

Dr. Bahija Jallal, Executive Vice President at MedImmune, said: “We are excited about our strategic collaboration with Celgene, a globally recognized leader in treatments for hematological cancers. This agreement is a great example of how we are accelerating the development of medical innovation in our portfolio in collaboration with other experts, in order to bring life-enhancing new medicines to patients faster. Together with Celgene, we are designing a programme for our anti-PD-L1 that will explore its full clinical potential as a game-changing treatment that could activate the patients’ immune system to fight and change the course of blood cancers in this area of high unmet need.”

Under the terms of the agreement, Celgene will collaborate with AstraZeneca to develop the anti-PD-L1 antibody MEDI4736 in hematology and make an upfront payment of $450 million. Celgene will lead clinical development across all new clinical trials within the collaboration and be responsible for all costs associated with these trials until December 31, 2016, after which it is responsible for 75% of these costs. Celgene will also be responsible for the global commercialization of approved MEDI4736 indications in hematology, and will receive royalty rates starting at 70 percent of worldwide sales from all uses in hematology. Royalty rates will decrease gradually to 50 percent over a period of four years after the first date of commercial sales. This collaboration agreement will become effective upon the expiration or termination of the applicable waiting periods under all applicable antitrust laws.

This strategic collaboration will initially focus on the development of MEDI4736 as combination therapy with Celgene’s pipeline of products and other novel agents for hematologic disorders. Over time, the collaboration could expand to include other assets.

MEDI4736 is not approved in any country for any indication.

About MEDI4736

MEDI4736 is a human monoclonal antibody directed against programmed cell death ligand 1 (PD-L1), which helps tumors avoid detection by the immune system. MEDI4736 is currently being evaluated in several disease states, including lung, melanoma and head and neck cancer.

About Hematologic Malignancies

The World Health Organization estimates that 1.75 million patients had a hematologic malignancy in 2012. These diseases include lymphoma, leukemia, multiple myeloma and myelodysplastic syndromes. The global incidence of blood cancers continues to rise. By 2030, the incidence of blood cancer overall is predicted to rise by 46 percent, and that of non-Hodgkin’s lymphoma and multiple myeloma by 50 and 60 percent, respectively.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit Follow us on Twitter @Celgene, and on Pinterest and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in Celgene Corporation’s Annual Report on Form 10-K and other reports filed with the Securities and Exchange Commission.

All registered trademarks are owned by Celgene Corporation.

VI. International Eurasian Congress of Hematology

VI. International Eurasian Congress of HematologyCONGRESSINVITATION
from the Hematology Specialists Association, Turkey:


Dear Colleagues,

We are pleased to announce that VI. International Eurasian Congress of Hematology will be held in the Mardan Palace Hotel in Antalya between 14-18 October 2015.

In 2014, our congress has made progress on becoming one of the world’s leading international conventions with the participation of our colleagues from 34 different countries.

We believe deep in our hearts that with its special concept, the Eurasian Congress of Hematology will fill in a significant gap in our region and serve not only scientifically but also culturally.

Looking forward to meeting with your respected selves in the Mardan Palace Hotel in Antalya between 14-18 October, we would like to extend our kindest regards.

Prof. Dr. Süleyman Dinçer, President of Hematology Specialists Association, Turkey
Prof. Dr. Giuseppe Saglio, Congress President

ASTX727 Clinical Trial for Low to High-Risk MDS/CMML Patients

Astex Pharmaceuticals is conducting a Phase 1-2 clinical trial to determine if ASTX727 is effective for the treatment of patients with IPSS low, intermediate -1, intermediate -2, or high risk MDS (including CMML).

Read more

ASTX727 Study Fast Facts
Additional ASTX727 Study Information

Announcing a Clinical Study for Patients with Myelodysplastic Syndromes (MDS)


QUAZAR Lower-Risk MDS is a study for people with MDS who need blood transfusions due to low red blood cell counts (called anemia) and low platelet counts (called thrombocytopenia).

You may qualify for this study if you*

  • Are age 18 years or older
  • Have been diagnosed with MDS
  • Have low red blood cell counts and are dependent on blood transfusions
  • Have low blood platelet counts

You may not be eligible for this study if you*

  • Have had previous stem cell transplants
  • Have been treated with VIDAZA® (azacitidine for injection) or DACOGEN® (decitabine for injection)

*Additional criteria apply.

For more information about this study, call 646-307-8079 or toll-free at 866-743-9791, e-mail or visit

NICE Proposes to Recommend Lenalidomide for MDS

NICE has issued final draft guidance recommending lenalidomide (also known as Revlimid and marketed by Celgene) as an option, for treating myelodysplastic syndromes.

Myelodysplastic syndromes (MDS), which are diagnosed in around 2000 people each year in England, are a group of bone marrow disorders characterised by the underproduction of one or more types of blood cells due to dysfunction of the marrow. MDS can lead to life threatening disease including acute myeloid leukaemia (AML), as well as anaemia and increased risk of bleeding and infections.

This appraisal focused on the use of lenalidomide for treating people with a specific type of MDS that is characterised by a chromosomal abnormality called an isolated deletion 5q cytogenetic abnormality. At the moment the main treatment option for people with the particular kind of MDS considered in this appraisal is best supportive care including regular blood transfusions.

Commenting on the final draft guidance, Sir Andrew Dillon, NICE chief executive, said: “The committee heard from clinical experts that lenalidomide is an effective therapy. Celgene – who market lenalidomide – worked with us to provide enough evidence to make it possible for us to recommend it for this group of people.

“Celgene provided a revised analyses and further information on their proposal for a reduction in the cost of the drug to the NHS (patient access scheme).”

Celgene’s patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. The company will provide the drug free of charge for those people who receive more than 26 monthly cycles.

The draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments.

Important new survey started by EHA – the European Haematology Association. EHA is interested in finding out if certain medicines are not available in some parts of the country – or in some parts of Europe. Patients, Caregivers, and Healthcare professionals are encouraged to complete this survey. The information from this survey will educate European policy makers to take measures that will help provide effective medicines that are not affordable or are not marketed in specific countries.

New Ways to Manage MDS: A New Resource for Patients and Loved Ones

Having trouble explaining Myelodysplastic Syndromes in terms your loved ones can understand? This new episode of the health and wellness program HEALTHY BODY, HEALTHY MIND can help explain it for you. If you would like to receive your FREE DVD, please call 609-298-1035 or 800-637-0839 or email

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GREAT NEWS: Italian Study Supports Use of Revised International Prognostic Scoring!

Under the aegis of the MDS Foundation, the International Working Group for Prognosis in MDS generated an improved method analyzing MDS patient prognosis more precisely than the initial IPSS.

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Revlimid® Approved in Europe for Use in MDS

The European Medicines Agency (EMA) has approved Revlimid® for the treatment of MDS patients with transfusion-dependent anemia associated with the del(5q) chromosomal abnormality.

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Revised International Prognostic Scoring System for MDS

IWG-PM Logo Under the aegis of the MDS Foundation, the International Working Group for Prognosis in MDS (IWG-PM) analyzed clinical features and outcome data from over 7000 patients and generated a method analyzing MDS patient prognosis more precisely than the initial IPSS. Novel components of this prognostic system include: five rather than three cytogenetic prognostic subgroups with specific and new classifications of a number of less common cytogenetic subsets, splitting the low marrow blast percentage value, and depth of cytopenias. In addition to the major prognostic variables of marrow blasts, cytogenetics and peripheral cytopenias, additive features for survival include patient age, performance status, serum ferritin and LDH. The IPSS-R calculator tool is accessible through the following URLs : or An iPhone App for the IPSS-R calculator tool is also accessible through the Apple Store (enter MDS IPSS-R). This IPSS-R should prove beneficial for predicting the clinical outcomes of untreated MDS patients and aiding design and analysis of clinical trials in this disease. Data for both calculator tools are derived from the article: Greenberg P, Tuechler H, Schanz J, et al, Revised International Prognostic Scoring System (IPSS-R) for Myelodysplastic Syndromes. Blood 120: 2454-2465, 2012.

MDS Foundation a Valued Partner of GuideStar

We are proud to be a valued partner of GuideStar, America’s first information service publicizing information about nonprofit organizations. Access the GuideStar link for additional financial information or to learn more about ways to give an annual or planned gift to fund MDS programs and services.

Launch of Fast Track for First Prescription™ Service through Celgene Patient Support® Fast Track for First Prescription Program™

Fast Track for First PrescriptionTM is an optional Celgene Patient Support® service which helps patients on an oral Celgene product receive their first prescription faster.

Additional details about eligibility and enrollment are addressed below.

For more information, or for any questions, call Celgene Patient Support® at 1-800-931-8691.

Which patients are eligible for this program?
  • Patients on an oral Celgene product receiving their first prescription
  • Patients with documented proof of insurance
  • Patients who are registered in a Celgene risk management (REMS) program and have a valid authorization number
How are eligible patients enrolled in this program?
  • Prescriber must fax the Fast Track Cover Sheet and the Patient Prescription Form with the authorization number and the patient risk category (i.e., adult female of childbearing potential, adult male, etc) to 1-800-822-2496
  • Patient’s contact information and insurance information should be included
  • Prescriber must ensure patient has completed their Celgene risk management (REMS) survey
What can a patient expect after being enrolled in this program?

The specialty pharmacy will contact the patient to:

  • Review their insurance benefits for the prescribed product (i.e., co-pay responsibilities and potential financial co-pay assistance options)
  • Provide appropriate counseling (if applicable)
  • Set up a delivery date for shipment of the medication

Am J Med: MDS Supplement

View the MDS Supplement in the American Journal of Medicine. This supplement is supported by Celgene Corporation. These articles are available on the internet through open access, allowing interested parties to download the entire supplement for free.

Review answers to commonly asked questions or get answers to your questions from an MDS expert