THE EUROPE TRIAL BY THE EMSCO NETWORK: TREATMENT WITH ROMIPLOSTIM IN
PATIENTS WITH LOWER-RISK MYELODYSPLASTIC SYNDROME (MDS) AND
THROMBOCYTOPENIA
Anne Sophie Kubasch1,2, Aristoteles Giagounidis3, Georgia Metzgeroth4, Anna Jonasova5, Regina
Herbst6, Jose Miguel Torregrosa Diaz7, Benoit De Renzis8, Katharina Götze9, Marie Luise Hütter-
Krönke10, Marie Pierre Gourin11, Borhane Slama12, Sophie Dimicoli-Salazar12, Pascal Cony-Makhoul13,
Kamel Laribi14, Sophie Park15, Katja Jersemann16, Oliver Tiebel17, Katja Sockel18, Silke Gloaguen2, Anna
Mies18, Fatiha Chermat19, Rosa Sapena19, Richard Schlenk20, Pierre Fenaux19,21, Uwe Platzbecker2,22 and
Lionel Ades19,21
(1)Medical Clinic and Policlinic 1, Hematology and Cellular Therapy, Leipzig University Hospital, Leipzig,
Germany, (2)The European MDS studies cooperative group (EMSCO), Leipzig, Germany, (3)Department
of Oncology, Hematology and Palliative Care, Marien Hospital Düsseldorf, Düsseldorf, Germany,
(4)Department of Hematology and Oncology, University Medical Centre Mannheim, Mannheim,
Germany, (5)Department of Hematology, First Faculty of Medicine, Charles University and General
University Hospital in Prague, Prague, Czech Republic, (6)Internal Medicine III, Department of
Hematology, Oncology, Stem Cell Transplantation, Klinikum Chemnitz gGmbH, Chemnitz, Germany,
(7)Department of Hematology and Oncology, CHU de Poitiers, Poitiers, France, (8)Service
d'Hématologie, CHU Clermont-Ferrand, Clermont-Ferrand, France, (9)Medical Department III for
Hematology and Oncology, Klinikum rechts der Isar, Technische Universität München, Munich,
Germany, (10)Department of Internal Medicine III, University of Ulm, Ulm, Germany, (11)CHRU de
Limoges, Limoges, France, (12)Department of Clinical Hematology, CH Avignon, Avignon, Provence-
Alpes-Côte d'Azur, Provence-Alpes-Côte d'Azur, France, (13)Centre Hospitalier Annecy Genevois,
Épagny-Metz-Tessy, France, (14)Department of Hematology, Centre Hospitalier du Mans, Le Mans,
France, (15)Department of Hematology, CHU Grenoble-Alpes, Grenoble, France, (16)GWT-TUD GmbH,
Dresden, Germany, (17)Institute of Clinical Chemistry and Laboratory Medicine, University Hospital
Dresden, Technical University of Dresden, Dresden, Germany, (18)Medical Clinic and Policlinic 1,
University Hospital Dresden, Dresden, Germany, (19)Groupe Francophone des Myélodysplasies, GFM,
Paris, France, (20)Department of Internal Medicine V, University Hospital of Heidelberg, Heidelberg,
Germany, (21)Hématologie clinique, Hôpital Saint-Louis, Paris, France, (22)Leipzig University Hospital,
Leipzig, Germany
I
ntroduction:
In about half of patients with lower-risk (LR) myelodysplastic syndrome, thrombocytopenia is
present at the time of diagnosis and associated with shortened survival and an increased risk
of progression to acute myeloid leukemia (AML).
Methods:
The EUROPE multicenter phase 2 trial within the EMSCO network investigated the impact of
biomarkers like endogenous thrombopoietin (TPO) level and platelet transfusion events (PTE)
on the efficacy of romiplostim (750μg SC qw) treatment in patients with LR-MDS (IPSS low/int-
1). Patients were eligible if baseline bone marrow blast count was <5% as assessed by central
morphology and platelet counts were ≤30 Gpt/L or ≤50 Gpt/L in case of bleeding history.
According to a previously published model of response to TPO-RA (Sekeres at al. BJH 2014),
patients wereassigned into 3 different cohorts at the time of screening based on their previous
PTE and TPO serum levels (cohort A: TPO<500 ng/l, PTE<6 units/past year; cohort B: TPO<500
ng/l, PTE>6 units or TPO>500 ng/l, PTE<6 units, cohort C: TPO>500 ng/l, PTE>6 units). Primary
endpoint of the study was the rate of hematologic improvement of platelets (HI-P) according
to IWG 2006 criteria after 16 weeks of romiplostim treatment.
POSTER 19