MDS is a bone marrow failure disorder
Recent News

Results from Phase III Study of REVLIMID® (Lenalidomide) Demonstrating Improved Transfusion Independence in Patients with Rare Blood Cancer, Non-Del-5Q Myelodysplastic Syndromes (MDS), Presented at ASH

SAN FRANCISCO–(BUSINESS WIRE)– Celgene Corporation (NASDAQ:CELG) today announced results from an international phase III study of REVLIMID® (lenalidomide) compared with placebo in patients with red-blood cell (RBC) transfusion dependent low-risk myelodysplastic syndromes (MDS) who were unresponsive or refractory to erythropoietin stimulating agents (ESA) and did not have a deletion 5q cytogenic abnormality. The results were presented during the 56th American Society of Hematology (ASH) annual meeting.

The study, presented by Valeria Santini, M.D., found that significantly more patients treated with lenalidomide achieved RBC-transfusion independence of at least 56 days compared with placebo (26.9%, [43/160 patients] vs. 2.5%,[2/79 patients]; p < 0.001), the primary endpoint of the study. The majority of patients (90%) with transfusion independence responded within 16 weeks of treatment. For patients who became transfusion independent, the median duration of transfusion independence was 8.2 months (range 5.2-17.8). Additionally, transfusion independence of at least 168 days was reached in 17.5% (28/160) of patients receiving lenalidomide compared with no patients receiving placebo. The incidence of AML progression (per 100 person-years) was 1.91 (95% CI 0.80-4.59) and 2.46 (95% CI 0.79-7.64) for lenalidomide and placebo patients, respectively. The follow-up period was not long enough to permit an overall survival comparison.

Myelosuppression was the main adverse event (AE). Grade 3-4 neutropenia occurred in 61.9% versus 12.7% in the lenalidomide and placebo groups, respectively, and grade 3-4 thrombocytopenia occurred in 35.6% versus 3.8% in the lenalidomide and placebo groups, respectively.

“This confirmation of the Phase II data is extremely encouraging. Based on this study, REVLIMID may offer this refractory patient population an additional option beyond their current limited choices,” said Guillermo Garcia-Manero, M.D. of the M.D. Anderson Cancer Center at the University of Texas.

REVLIMID is not indicated for the treatment of non-del5q MDS in any country.
Source: press release, 12/08/14.


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

Clinical Research Trial with 5-Azacitidine with/without Birinapant (TL32711) in Higher Risk MDS/CMML Patients


We would like to announce a clinical trial randomized double blind, placebo controlled, multicentric study for higher risk newly diagnosed Myelodysplastic Syndrome (MDS)/Chronic myelomonocytic leukemia (CMML) patients.

Primary Objective of this study is to compare the relative effect of azacitidine plus birinapant versus azacitidine plus placebo on response rate [RR=complete response (CR) + partial response (PR)] using the modified International Working Group (IWG) Response Criteria for MDS, in patients with higher-risk MDS or CMML.

Secondary Objectives of the study are to compare the effect of azacitidine plus birinapant relative to azacitidine plus placebo, on the hematologic improvement (HI), relapse-free survival according to modified IWG 2006 criteria, time to response, change in transfusion requirements, duration of response according to modified IWG 2006 criteria, overall urvival and adverse event (AE) profile.

The exploratory objective of the study is to assess exploratory translational biomarkers for antitumor effects.

Study description: Approximately 158 patients (10 patients in initial open label single arm safety run in portion and 148 subjects in the double blinded placebo controlled randomized portion) with higher risk Myelodysplastic syndrome yelomonocytic leukemia (CMMoL) who are naïve, to azacitidine therapy will be enrolled.

In an effort to accelerate the clinical development of birinapant for myelodysplastic syndrome and CMML the MDS oundation will be assisting the sponsor and would appreciate hearing from you.

If you are a physician or health care provider and would like to refer a patient for enrollment into this clinical trial OR if you are an MDS patient who is newly diagnosed with MDS please see additional information and contact details on

ACCRUED & CLOSED – Clinical Research Trial with Birinapant (TL32711) and 5-Azacitidine in High Risk MDS Naïve, Relapsed or Refractory Patients


We would like to announce a clinical trial study for high risk naïve, relapsed or refractory Myelodysplastic Syndrome (MDS) patients.

Primary Objective of this study is to determine the maximum tolerated dose (MTD), recommended Phase 2 dose, and pharmacodynamics (PD) of birinapant (TL32711) when administered in combination with 5-azacitidine (5 AZA) in subjects with myelodysplastic syndrome (MDS) who are naïve, refractory or have relapsed to 5-AZA therapy.

Secondary Objectives of the study are to determine the clinical activity using the International Working Group (IWG) (Cheson, 2006) Response Criteria for MDS during the Phase 1b dose escalation stage of the study and in the Phase 2a expansion cohort, to determine the pharmacokinetics (PK) of birinapant when administered with 5-AZA in plasma and to assess exploratory translational biomarkers of anti-tumor activity of birinapant in combination therapy.

In an effort to accelerate the clinical development of birinapant for myelodysplastic syndromes the MDS Foundation will be assisting the sponsor and would appreciate hearing from you.

If you are a physician or health care provider and would like to refer a patient for enrollment into this clinical trial OR if you are an MDS patient who is newly diagnosed with MDS or has failed, become intolerant, or relapsed after Vidaza® (azacitidine) or Dacogen® (decitabine) treatment, please see additional information and contact details at

KaloBios Pharmaceuticals, Inc. – Trial KB004, Phase 2

KaloBios Pharmaceuticals, Inc. is now recruiting the Phase 2 portion of their trial of KB004 in MDS and MF patients. The study consists of IV infusion once weekly for a 21 day dosing cycle with KB004 250 mg (the recommended Phase 2 dose). This portion of the study will be to further study the activity of KB004 and explore the safety of KB004. The Identifier is NCT01211691.

To be eligible for the study patients will have:

  • Confirmed MF or MDS with an acceptable level of EphA3 expression
  • Confirmed hematologic malignancy refractory to or progressed following standard treatments, or subjects not considered medically suitable to receive standard of care treatment or who refuse standard of care treatment
  • Acceptable level of EphA3 expression
  • Eastern Cooperative Oncology Group (ECOG) ≤1
  • Acceptable laboratory results

Patients are not eligible for the study if they have the following:

  • History of or current central nervous system (CNS) involvement that may increase risk of bleeding
  • Recent major surgery
  • Ongoing surgical or wound healing complications
  • Active clinically significant bleeding
  • Uncontrolled hypertension
  • Significant intercurrent illness
  • Known history of prolonged bleeding times or platelet dysfunction
  • Active infection requiring IV antibiotics, IV antifungals, or IV antivirals within 2 weeks prior to Cycle 1, Day 1

Study centers are now recruiting in the United States including California (Palo Alto and Sacramento), Florida (Miami and Tampa), Ohio (Cleveland), and Texas (Houston). In Australia centers are recruiting in Westmead, New South Wales; Adelaide, South Australia; Prahran, Victoria; and Queensland, Australia.

For questions, feel free to contact: Kate Doherty at / 1-650-243-3126.

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

Learn more

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.

Read more

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.

Read more

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 Partneer 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