Clinical trial NCT02428686: Treatment of Anemia With Epoetin Beta in Low Risk Myelodysplastic Syndrome (MDS)
Official title: Treatment of Anemia With Epoetin Beta in Low Risk Myelodysplastic Syndrome With Analysis of the Impact on Quality of Life and Functional Capacity of Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelodysplastic Syndromes
Lead sponsor: Groupe Francophone des Myelodysplasies
Sponsor class: OTHER
First posted: 2015-04-29
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Chronic anemia is the symptom most frequently found at diagnosis in low risk myelodysplastic syndrome. It generates an increased rate of morbidity and mortality in this population of patients whose median age is high and the rate of co-mobidities important. The historical treatment is limited to transfusion support with a significant impact on quality of life and the incidence of secondary haemosiderosis, which contributes to the emergence of co-morbidities, especially cardiovascular. Treatment with rHuEPO allows for overall erythroid response in 40-60% of patients treated.

In this trial, the investigators intend to study the interest of a treatment with epoetin beta in patients with anemia \<10 g / dL in the context of a myelodysplastic syndrome with IPSS score \<1.

In addition to studying the erythroid response, the investigators will measure the impact on quality of life and functional performance.

Patients will receive epoetin beta (60 000UI/week). Response will be assessed after 12 and 24 weeks of treatment.

Detailed description: Objective of the trial Assess the level of haematological response according to IWG 2000 and IWG 2006 criteria with a pattern of administration of high doses epoetin beta (NeoRecormon ®) for 12 and 24 weeks of treatment;

To evaluate the predictive value of the rate of reticulocytes at Day 8 on the erythroid response;

* Assess time to response
* Assess tolerance
* Assess the impact on quality of life using the FACT-An and EQ-5D measurement scales;
* Assess functional capacity:

Cardiovascular performance on effort by the 6-minute walk test Performing the " Short Physical Performance Battery " tests

Evaluation The evaluation of response according to the IWG 2000 and IWG 2006 criteria will be held at 12 and 24 weeks of treatment

Eligibility criteria: Inclusion Criteria:

* Written informed consent
* Must be 18 years of age or older at the time of screening
* Myelodysplastic Syndrome of the following subtypes : RA, RAS, RCMD, RCMD-RS, RAEB 1, with IPSS score low or intermediate 1 (IPSS score \< 1 ) and with anemia defined by Hb \< 10 g/dl (with RBC transfusion requirement or not)
* For women of childbearing potential, need for effective contraception throughout the study period.

Exclusion Criteria:

* Intensive Chemotherapy within 3 months before inclusion
* Myelodysplastic Syndrome with IPSS score \>1
* Treatment with rHu-Epo or rHu-GCSF within 2 months before inclusion
* EGOG \> 3 ;
* Other causes of anemia (eg. , Iron deficiency, vitamin B12 or folic acid, hypothyroidism before correction)
* Uncontrolled arterial hypertension
* Life expectancy less than 6 months
* CMML
* Pregnant or breast feeding female subjects
* Patients with creatinine clearance less than 30ml/min.

Primary outcomes:
measure: Assess the level of haematological response according to IWG 2006 criteria with a pattern of administration of high doses epoetin beta (NeoRecormon ®) for 12 and 24 weeks of treatment
timeFrame: 12 and 24 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Le Kremlin-Bicêtre
country: France
facility: CHU de Bicêtre
state: Ile de France
city: Amiens
country: France
facility: CHU d'AMIENS
city: Angers
country: France
facility: CH Angers
city: Avignon
country: France
facility: CH d'Avignon-305 rue Follereau-
city: Bayonne
country: France
facility: Hopital de la Cote Basque
city: Bobigny
country: France
facility: Hôpital Avicenne
city: Boulogne Sur Mer
country: France
facility: Hôpital Boulogne Sur Mer
city: Brest
country: France
facility: CHU de Brest
city: Caen
country: France
facility: CHU Clémenceau
city: Cergy-pontoise
country: France
facility: CH René Dubos
city: Le Mans cedex
country: France
facility: Centre Hospitalier du Mans
city: Lille
country: France
facility: CHRU Huriez
city: Lille
country: France
facility: Hopital Saint-Vincent de Paul-
city: Limoges
country: France
facility: CHRU de Limoges
city: Mantes-la-jolie
country: France
facility: centre hospitalier de Mantes-la-jolie
city: Marseille
country: France
facility: Institut Paoli Calmette
city: Nancy
country: France
facility: CHU Brabois
city: Nantes
country: France
facility: Hematology Dpt, Hopital de l'Hotel Dieu
city: Neuilly Sur Seine
country: France
facility: Hôpital Américain de Paris
city: Nice
country: France
facility: CHU Archet
city: Orléans
country: France
facility: Hôpital La Source
city: Paris-Cedex 12
country: France
facility: Hôpital Saint-Antoine.
city: Paris
country: France
facility: Hôpital Saint Louis
city: Paris
country: France
facility: Hopital Cochin
city: Poitiers
country: France
facility: Hôpital Jean Bernard
city: Pringy cedex
country: France
facility: Centre Hospitalier de la région d'Annecy
city: Reims
country: France
facility: CHU de Reims
city: Rouen
country: France
facility: Centre Henri Becquerel
city: Strasbourg
country: France
facility: Centre Hospitalier Universitaire de STRASBOURG
city: Toulouse
country: France
facility: Chu Purpan
city: Tours
country: France
facility: Hopital Bretonneau
Study officials:
References: