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EHA news

Press Release: Economic Burden of Blood Disorders in EU is €23 billion

July 22, 2016 - The Hague, The economic burden of blood disorders across the European Union, Iceland, Norway and Switzerland amounts to €23 billion per year. This is the conclusion of two papers published in The Lancet Haematology by the Health Economics Research Centre of the University of Oxford.

Blood disorders represent a myriad of diseases including anemias, coagulation disorders (e.g. thrombosis), bleeding disorders (e.g. hemophilia) and blood cancers (e.g. leukemia, multiple myeloma, lymphoma). In Europe, blood disorders affect around 80 million people. Regrettably, about 110,000 of them die each year as a direct result of their illness.

The total cost of blood disorders consists of healthcare expenditure (€15.6 billion), productivity loss due to illness and mortality (€5.6 billion), and the costs of informal care (€1.6 billion). The latter amount is based on hours of unpaid care provided to people suffering from blood disorders, such as by family or friends. 28% of healthcare costs pertain to medications (€4.3 billion).

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SGN-CD33A Combined with Hypomethylating Therapy Produces High Remission Rates among Older Patients with AML

PRESS RELEASE - COPENHAGEN, JUNE 11, 2016

EMBARGO TILL JUNE 11, 2016, 08:30 AM CEST

SGN-CD33A Combined with Hypomethylating Therapy Produces High Remission Rates among Older Patients with AML

Acute myeloid leukemia (AML) is an aggressive form of blood cancer in which the majority of cases express CD33 on the surface of the leukemia cells. AML is challenging to treat in older patients since intensive chemotherapy is poorly tolerated, and standard therapies, such as the hypomethylating agents (HMAs) azacitidine and decitabine, yield modest response rates. Data are presented from an ongoing phase 1 trial evaluating vadastuximab talirine (SGN-CD33A; 33A) in combination with standard therapies (azacitidine, decitabine) in older AML patients who have declined intensive frontline therapy. 33A is an antibody-drug conjugate (ADC) targeted to CD33, comprising a novel antibody system stably linked to a highly potent cell-killing agent. CD33 is expressed on leukemic blasts in nearly all AML patients and expression is generally consistent regardless of age, risk factors, or disease characteristics.

Thus far, 53 AML patients with median age of 75 years have been treated with this combination. Of 49 efficacy-evaluable patients treated with 33A combined with either azacitidine or decitabine, the composite complete remission rate (CR+CRi) was 73 percent, which compares favorably with historical studies of HMA monotherapy in this population. The 30- and 60-day mortality rates were two and eight percent, respectively. The most common Grade 3 or higher treatment-emergent adverse events occurring in 20 percent or more of patients were febrile neutropenia, thrombocytopenia, anemia and neutropenia. The combination of 33A and HMAs was well-tolerated and yielded encouraging response rates in older AML patients.

Presenter: Dr Amir Fathi

Affiliation: Massachusetts General Hospital Cancer Center, Boston, USA

Topic: SGN-CD33A IN COMBINATION WITH HYPOMETHYLATING AGENTS: A NOVEL, WELL-TOLERATED REGIMEN WITH HIGH REMISSION RATE IN OLDER PATIENTS WITH AML

Abstract S503 will be presented by Amir Fathi on Saturday, June 11, 16:30 - 16:45 in Hall A3.

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

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Killer antibodies against AML

PRESS RELEASE - COPENHAGEN, JUNE 11, 2016

EMBARGO TILL JUNE 11, 2016, 08:30 AM CEST

 

Killer antibodies against AML

Most patients with acute myeloid leukemia (AML) can only be cured when a stem cell transplant induces an immune response against the patient’s leukemia. Working with patients who are in remission from AML, we discovered antibodies that bind specifically to AML cells. We suspect that these antibodies have general utility in treating other patients with AML. The target of these antibodies was unanticipated: the U5 snRNP200 protein complex, normally expressed in the cell cytoplasm and nucleus but expressed on the outer surface of AML cells. Moreover, we found that U5 specific antibodies are ‘killer antibodies’: they kill AML blasts in vitro and (in a mouse model) in vivo. We are now advancing these antibodies through early development with the goal of initiating human clinical trials in the near future. (see also infographic (dutch) at: https://www.amc.nl/web/AMC-website/AMCFoundation/Uitgelicht/Hematologie.htm)

Presenter: Dr Mette Hazenberg

Affiliation: Academic Medical Center Amsterdam, the Netherlands

Topic: ACUTE MYELOID LEUKEMIA (AML) PATIENTS CURED AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION GENERATE TUMOR-SPECIFIC CYTOTOXIC ANTIBODIES THAT KILL AML BLASTS

Abstract S124 will be presented by Mette Hazenberg on Friday, June 10, 11:45 - 12:00 in Hall C13.

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

 

 

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Improved survival for adult Acute Lymphoblastic Leukemia (ALL) patients

PRESS RELEASE - COPENHAGEN, JUNE 11, 2016

EMBARGO TILL JUNE 11, 2016, 08:30 AM CEST

 

Improved survival for adult Acute Lymphoblastic Leukemia (ALL) patients

Historical survival for patients 18-45 years with ALL is approximately 40 %. However the event free survival for ALL patients 18-45 years has improved to 73% following implementation of the NOPHO ALL2008 protocol in July 2008. The intensive protocol, dividing patients into 4 risk groups depending mainly on response to treatment, is now used for ALL patients 1-45 years in Denmark, Norway, Sweden, Finland, Iceland, Lithuania and Estonia. The joint effort of pediatricians and adult hematologists has resulted in new knowledge especially for teenagers and adult patients and has revealed that: 1. adult patients are more often ‘high risk’ patients compared with children, 2. they tolerate intensive chemotherapy almost as well as children and are not subject to delays or gaps during treatment, and 3. when comparing patients within the 4 risk groups, adult survival is close to that of children and is markedly improved.

Presenter: Dr Nina Toft

Affiliation: Herlev University Hospital, Herlev, Denmark

Topic: ADULTS AND CHILDREN (1-45 YEARS) WITH PH-NEGATIVE ALL HAVE ALMOST IDENTICAL OUTCOME IN RISK-STRATIFIED ANALYSIS OF NOPHO ALL2008

Abstract LB173 will be presented by Nina Toft on Friday, June 10, 2016, 17:15 - 17:30 in Hall H.

 

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

 

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The Clot Thickens

PRESS RELEASE - COPENHAGEN, JUNE 11, 2016

EMBARGO TILL JUNE 11, 2016, 08:30 AM CEST

The Clot Thickens

Haemophilia B is a genetic bleeding disorder, affecting approximately 80,000 males worldwide1, caused by an insufficient or dyfunctional blood clotting protein called factor IX (FIX).

Whereas normal clotting factor activity levels range from >40% to 150% (or IU/dL), individuals with the severe form of haemophilia B have circulating FIX activity levels of <1% of normal and may experience recurrent joint, muscle, and tissue bleeding, and potentially life-threathening bleeds into a critical closed space (such as the brain) 2,3.

Full adherence to FIX protein replacement prophylaxis is effective; however, treatment has not been universally adopted due to the burden of once or twice weekly intravenous infusions4.

This ongoing phase 1/2 study is evaluating the safety and tolerability of a single intravenous infusion of SPK-9001, an investigational gene transfer product, designed to allow the body to produce its own factor IX protein by transferring a functioning factor IX gene into the body 5.

As of today, enrolment completed for the initial dose level and four subjects have been followed for 7 to 26 weeks after a single intravenous infusion with 5x1011 vg/kg of SPK-9001 without the need for immunosuppression.

 

No product and/or procedure-related serious adverse events (including immune response) have been reported to date and no subjects have required steroids or other medications to suppress the immune system.

 

As of May 22, subjects 1-4 showed plateau factor IX activity levels of 32, 39, 25, and 27% of normal, respectively.

 

All four subjects are no longer infusing FIX protein products; one subject treated himself with a FIX infusion for a suspected ankle bleed two days after the gene transfer administration, otherwise, all subjects have been free from bleeding to date.

These results are consistent in terms of kinetics of rise of factor IX, and are well above the threshold of 12% required to reduce the risk of bleeds6.

Based on the recommended trough levels6, factor IX activity levels over 12% of normal are likely to reduce significantly the risk of joint bleeds in patients with haemophilia B7.

 

1. Srivastava et al., 2012; Giangrande 2005

2. Gringeri A et al., 2014 and Jansen et al., 2009

3. Srivastava et al., 2013 and ISTH-SSC, 2011

4. Srivastava et al., 2013, National Hemophilia Foundation 2007, Roberts and Eberst 1993

5. Clinicaltrials.gov (https://www.clinicaltrials.gov/ct2/show/NCT02484092)

6. den Uijl IEM et al., 2011

7. Madhi A et al., 2015

 

Presenter: Dr Katherine A High

Affiliation: Children’s Hospital of Philadelphia, USA

Topic: AAV-MEDIATED GENE THERAPY FOR HEMOPHILIA B-EXPRESSION AT THERAPEUTIC LEVELS WITH LOW VECTOR DOSES

 

Abstract LB771 will be presented by Spencer Sullivan on Saturday, June 11, 2016, 17:30 - 17:40 in Hall H.

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

 

 

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Genome sequencing of thousands of patients with rare blood disorders

PRESS RELEASE - COPENHAGEN, JUNE 11, 2016

EMBARGO TILL JUNE 11, 2016, 08:30 AM CEST

Genome sequencing of thousands of patients with rare blood disorders

Approximately 3M people have a rare bleeding disorder or disease of platelets, which are the cell fragments that help blood clot. The genetic causes of dozens of such disorders are known (e.g. haemophilia), but there are many others yet to be discovered. Because platelets have a role in heart attacks and stroke, they are involved in one in three deaths in the general population and a better understanding of rare platelet diseases may bring benefits to the care of the millions of patients with these common life-threatening events.

Researchers supported by the NIHR-BioResource Rare Diseases (UK) with collaborators worldwide are sequencing the whole genomes of thousands of patients without a genetic diagnosis and collating their clinical information in a research database. Through computational analysis of this information, we are searching for commonalities between patients who also share similar genetic changes to identify new causes of disease and better understand disorders already known to us.

So far, we have found a new genetic link between very large platelets and deafness, a genetic change in a well-known cancer gene responsible for fragile bones, scarring of the bone marrow and low platelet count, and a further genetic link between large platelets and heart rhythm problems. Within months of publication, these and other research findings are already benefiting patients through a new cheap, fast and accurate test (thrombogenomics.org.uk) available to patients in the UK and other countries.

Presenter: Dr Ernest Turro

Affiliation: University of Cambridge, Cambridge, UK

Topic: IDENTIFYING THE GENETIC BASIS OF RARE BLEEDING AND PLATELET DISORDERS USING SYSTEMATIC PHENOTYPING AND GENOME SEQUENCING

Abstract S518 will be presented by Ernest Turro on Saturday, June 11, 2016, 16:30 - 16:45 in Room H4.

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

 

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Daratumumab Shows Remarkable Benefit in Relapsed or Refractory Multiple Myeloma in the POLLUX Study

PRESS RELEASE - COPENHAGEN, JUNE 10, 2016

EMBARGO TILL JUNE 10, 2016, 08:30 AM CEST

Daratumumab Shows Remarkable Benefit in Relapsed or Refractory Multiple Myeloma in the POLLUX Study

Daratumumab is a fully human monoclonal antibody that binds to a novel target on myeloma cells. POLLUX was an open-label, randomised phase 3 study conducted in 18 countries that evaluated the combination of daratumumab and lenalidomide and dexamethasone (daratumumab group) compared with lenalidomide and dexamethasone (control group) in 569 patients with relapsed or refractory multiple myeloma who received at least 1 prior line of therapy. At the pre-planned interim analysis, an unprecedented 63% reduction in the risk of progression or death was shown in the daratumumab group compared with control group, leading to early unblinding of the study. More patients achieved an overall response in the daratumumab group compared with the control group (93% vs 76%, P <0.0001). Deep and durable responses were significantly more frequent in the daratumumab group, with higher rates of very good partial response or better (76% vs 44%, P <0.0001) and a more than doubling of complete response or better (43% vs 19%, P <0.0001). Treatment was well-tolerated in the daratumumab group with adverse events consistent with the known profiles of the drugs in the combination. The study establishes a positive benefit/risk profile for daratumumab with lenalidomide and dexamethasone in multiple myeloma patients who have received at least 1 prior line of therapy and suggests that this combination may represent a new standard of care for these patients.

Presenter: Dr Meletios A Dimopoulos

Affiliation: National and Kapodistrian University of Athens, Greece

Topic: AN OPEN-LABEL, RANDOMISED PHASE 3 STUDY OF DARATUMUMAB, LENALIDOMIDE, AND DEXAMETHASONE (DRD) VERSUS LENALIDOMIDE AND DEXAMETHASONE (RD) IN RELAPSED OR REFRACTORY MULTIPLE MYELOMA (RRMM): POLLUX

Abstract LB2238 will be presented by Meletios A Dimopoulos on Friday, June 10, 2016, 16:50 - 17:00 in Hall A1.

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

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Immunotherapy delivered by Blinatumomab improves survival in acute lymphoblastic leukaemia patients

PRESS RELEASE - COPENHAGEN, JUNE 10, 2016

EMBARGO TILL JUNE 10, 2016, 08:30 AM CEST

Immunotherapy delivered by Blinatumomab improves survival in acute lymphoblastic leukaemia patients.

Adult patients with acute lymphoblastic leukemia (ALL) can achieve disease control in 90% of cases with intense chemotherapy but only half of these responders will be cured. For patients who are either refractory or relapse after chemotherapy the outcome is dismal despite undergoing more chemotherapy and/or an allogeneic haematopoetic stem cell transplantation. Immunotherapy offers a new treatment option. This can be delivered by the bispecifc antibody construct blinatumomab, which can engage with patients own T cells to fight ALL cells. A single arm Phase II trials with Blinatumomab has shown that 43% of relapsed or refractory (r/r) ALL patients can achieve disease control. The aim of Tower trial was to answer the question if blinatumomab can achieve a survival benefit in adult patients with r/r acute lymphoblastic leukemia compared to standard chemotherapy (SOC). More than 400 r/r ALL patients in Europe, North America, Asia and Australia were randomised 2:1 to receive either blinatumomab or SOC. The trial was stopped prematurely as blinatumomab demonstrated almost doubling of the overall survival when compared to chemotherapy. This benefit was seen in all subgroup of patients including patients who relapsed after an allogeneic haematopoetic stem cell transplantation and/or who have received multiple different chemotherapy regiments. Blinatumomab treatment did not result in more serious side effects than chemotherapy.

In conclusion Blinatumomab is the first immunotherapy agent that has proven to extend patients life with relapsed acute lymphoblastic leukemia when compared to chemotherapy.

Presenter: Dr Max S Topp

Affiliation: Medizinische Klinik II, Universitätsklinikum Würzburg, Germany

Topic: BLINATUMOMAB IMPROVED OVERALL SURVIVAL IN PATIENTS WITH RELAPSED OR REFRACTORY PHILADELPHIA NEGATIVE B-CELL PRECURSOR ACUTE LYMPHOBLASTIC LEUKEMIA IN A RANDOMIZED, OPEN-LABEL PHASE 3 STUDY (TOWER)

Abstract S149 will be presented by Max S Topp on Friday, June 10, 2016, 16:35 - 16:45 in Hall A1.

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

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The root of evil: pre-leukemic clones that survive chemotherapy are linked to a higher risk of leukemia recurrence

PRESS RELEASE - COPENHAGEN, JUNE 10, 2016

EMBARGO TILL JUNE 10, 2016, 08:30 AM CEST

The root of evil: pre-leukemic clones that survive chemotherapy are linked to a higher risk of leukemia recurrence

 

Acute myeloid leukemia (AML) is an aggressive form of blood cancer. Treatment with intensive chemotherapy often leads to a period of freedom from overt disease called a remission. However, recurrence of the disease is common. For reasons that are not yet fully understood, older patients have a higher risk of relapse. In some patients, AML originates from a clone of pre-leukemic stem cells. These pre-leukemic stem cells already carry genetic changes (mutations) that are also found in leukemias, but they still can give rise to normal-appearing blood cells. The subsequent gain of additional mutations then causes progression to overt AML. When the AML clone is eradicated by chemotherapy, a pre-leukemic clone remains detectable in some patients. We therefore studied sample pairs collected at the time of leukemia diagnosis, and after chemotherapy while the patient was in remission, from 107 patients with AML. In 36% of these patients, some of the gene mutations found in the leukemia were still present in the remission specimen, indicating the persistence of a pre-leukemic clone. Persisting mutations were more frequent in older patients. Importantly, patients with persisting mutations had a higher risk of subsequent disease recurrence compared to those with no persisting mutation. Multivariate statistical analyses, which also take other known risk factors into account, suggest that the higher incidence of persisting pre-leukemic clones in older patients may be one explanation for the higher risk of AML relapse in this age group.

 

Presenter: Dr Klaus Metzeler

 

Affiliation: (Internal Medicine 3, University Hospital, Ludwig-Maximilians-Universität (LMU) München, Germany; German Cancer Consortium (DKTK), German Cancer Research Center (DKFZ), Heidelberg, Germany)

 

Topic: PERSISTENCE OF DRIVER MUTATIONS DURING COMPLETE REMISSION ASSOCIATES WITH SHORTER SURVIVAL AND CONTRIBUTES TO THE INFERIOR OUTCOMES OF ELDERLY PATIENTS WITH ACUTE MYELOID LEUKEMIA

Abstract S146 will be presented by Maja Rothenberg-Thurley on Friday, June 10, 2016, 16:00 - 16:10 in Hall A1.

 

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

 

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STOPPING TYROSINE KINASE INHIBITORS IN A VERY LARGE COHORT OF EUROPEAN CHRONIC MYELOID LEUKEMIA PATIENTS: RESULTS OF THE EURO-SKI TRIAL

PRESS RELEASE - COPENHAGEN, JUNE 10, 2016

EMBARGO TILL JUNE 10, 2016, 08:30 AM CEST

STOPPING TYROSINE KINASE INHIBITORS IN A VERY LARGE COHORT OF EUROPEAN CHRONIC MYELOID LEUKEMIA PATIENTS: RESULTS OF THE EURO-SKI TRIAL

Tyrosine kinase inhibitors (TKI) have substantially improved survival in patients with chronic myeloid leukemia in chronic phase. However, treatment is in clinical practice considered life-long. Small clinical trials have previously shown that TKI-therapy can be stopped in 40-60% of patients with a very good therapy response.

The purpose in EURO-SKI was to determine the proportion of patients keeping their therapy response after stopping TKIs and to determine clinical and biological factors that predict successful TKI-stop.

868 patients at 61 sites in 11 European countries were registered in study. Patients were required to have had at least three years of TKI-therapy and to have had a very good response (MR4) to therapy for at least one year prior to study entry. Only patients who had not failed prior TKI-treatment were included.

Results of the trial show that 62% of the patients still maintained treatment response (MMR) 6 months after stopping therapy. This was 56% at 12 months. Duration of TKI therapy and of very good therapy response (MR4) prior to stop was found to predict successful stop. However, neither gender, age or risk score of the disease were linked to likelihood of successful stopping of TKI therapy.

Presenter: Dr Johan Richter

Affiliation: Lund, Sweden, Francois-Xavier Mahon, Bordeaux, France and Susanne Saussele, Mannheim, Germany.

Topic: STOPPING TYROSINE KINASE INHIBITORS IN A VERY LARGE COHORT OF EUROPEAN CHRONIC MYELOID LEUKEMIA PATIENTS: RESULTS OF THE EURO-SKI TRIAL

Abstract S145 will be presented by Johan Richter on Friday, June 10, 2016, 15:45 - 15:55 in Hall A1.

About the EHA Annual Congress

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.

Contact

EHA Executive Office

Ineke van der Beek, Jon Tarifa

E-mail: communication@ehaweb.org

Mobile: +31(0)6 2011 1055

 

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