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Hôpital-Camfrout, France

Khellaf M.,University Paris Est Creteil | Michel M.,University Paris Est Creteil | Quittet P.,Montpellier University Hospital Center | Viallard J.-F.,CHU Haut Leveque | And 21 more authors.
Blood | Year: 2011

Romiplostim, a thrombopoietic agent with demonstrated efficacy against immune thrombocytopenia (ITP) in prospective controlled studies, was recently licensed for adults with chronic ITP. Only France has allowed romiplostim compassionate use since January 2008. ITP patients could receive romiplostim when they failed to respond to successive corticosteroids, intravenous immunoglobulins, rituximab, and splenectomy, or when splenectomy was not indicated. We included the first 80 patients enrolled in this program with at least 2 years of follow-up. Primary platelet response (platelet count ≥ 50 × 10 9/L and double baseline) was observed in 74% of all patients. Long-term responses (2 years) were observed in 47 (65%) patients, 37 (79%) had sustained platelet responses with a median platelet count of 106 × 10 9/L (interquartile range, 75-167 × 10 9/L), and 10 (21%) were still taking romiplostim, despite a median platelet count of 38 × 10 9/L (interquartile range, 35-44 × 10 9/L), but with clinical benefit (lower dose and/or fewer concomitant treatment(s) and/or diminished bleeding signs). A high bleeding score and use of concomitant ITP therapy were baseline factors predicting romiplostim failure. The most frequently reported adverse events were: arthralgias (26%), fatigue (13%), and nausea (7%). Our results confirmed that romiplostim use in clinical practice is effective and safe for severe chronic ITP. This trial was registered at www.clinicaltrials.gov as #NCT01013181. © 2011 by The American Society of Hematology.

Gyan E.,University of Tours | Gyan E.,NICHE | Dreyfus F.,Groupe Francophone des Myelodysplasies | Fenaux P.,Groupe Francophone des Myelodysplasies | Fenaux P.,University Paris Diderot
Mediterranean Journal of Hematology and Infectious Diseases | Year: 2015

The 2008 WHO classification identified refractory cytopenia with unilineage dysplasia (RCUD) as a composite entity encompassing refractory anemia, refractory thrombocytopenia (RT), and refractory neutropenia (RN), characterized by 10% or more dysplastic cells in the bone marrow respective lineage. The diagnosis of RT and RN is complicated by several factors. Diagnosing RT first requires exclusion of familial thrombocytopenia, chronic auto-immune thrombocytopenia, concomitant medications, viral infections, or hypersplenism. Diagnosis of RN should also be made after ruling out differential diagnoses such as ethnic or familial neutropenia, as well as acquired, drug-induced, infection-related or malignancy-related neutropenia. An accurate quantification of dysplasia should be performed in order to distinguish RT or RN from the provisional entity named idiopathic cytopenia of unknown significance (ICUS). Cytogenetic analysis, and possibly in the future somatic mutation analysis (of genes most frequently mutated in MDS), and flow cytometry analysis aberrant antigen expression on myeloid cells may help in this differential diagnosis. Importantly, we and others found that, while isolated neutropenia and thrombocytopenia are not rare in MDS, those patients can generally be classified (according to WHO 2008 classification) as refractory cytopenia with multilineage dysplasia or refractory anemia with excess blasts, while RT and RN (according to WHO 2008) are quite rare. These results suggest in particular that identification of RT and RN as distinct entities could be reconsidered in future WHO classification updates.

Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-17-2014 | Award Amount: 6.00M | Year: 2015

Myelodysplastic syndromes (MDS) are chronic bone marrow malignancies of the elderly, complicated by severe anaemia. MDS significantly affects quality-adjusted survival and it imposes an increasing financial burden on patients and healthcare (HC) systems. The burden of disease is expected to worsen in the future, given the aging EU population and newly identified MDS cases among those diagnosed with Anaemia of the Elderly. EUMDS - a unique registry with prospective, observational data on 1600 lower-risk MDS patients from 16 EU countries - enables comparison of existing MDS HC interventions (HCI). Objectives 1) Comparison of outcomes and costs of existing HCI, using established and new core outcome sets. This, alongside health technology assessment, will provide robust evidence to underpin sustainable use of HC resources; 2) Enhanced compliance with diagnostic procedures in MDS by introducing new minimally-invasive diagnostic methods. This will increase the number of correctly and timely diagnosed MDS patients; 3) Raised awareness of the relevance of obtaining the right diagnosis in elderly by comparing HRQoL between EUMDS and against a non-MDS cohort. This will improve HRQoL of individual patients through better tailoring of HCI; 4) Better outcome predictability of available HCI by refining classification of cases using molecular characterisation to incorporate response to HCI and to provide evidence for personalised medicine; 5) Improved, evidence-based, guidelines supporting the regulatory process, and providing information to patients and physicians to promote personalised decisions in MDS care; 6) Establishment of a European MDS competence network encompassing all stakeholders in the MDS field. Relevance Better treatment compliance, a more evidence-based use of HCI and an increasing tailored use of existing HC options will contribute to an efficient and cost effective (personalised) use of HC resources for elderly patients with MDS and their co-morbidities.

Bally C.,Groupe Francophone des Myelodysplasies | Bally C.,University of Paris 13 | Thepot S.,Groupe Francophone des Myelodysplasies | Thepot S.,University of Paris 13 | And 19 more authors.
Leukemia Research | Year: 2013

The effect of azacitidine (AZA) in therapy related MDS and AML (t-MDS/AML) is not well established. 54 patients (42 t-MDS and 12 t-AML), 71% of whom had complex karyotype, received AZA for at least one cycle (median 4 cycles). The overall response rate (ORR) was 39% in the whole cohort and 62% in patients who received ≥4 cycles. One, 2 and 3 year OS was 36%, 14% and 8% respectively. Female gender (p=0.01) and ECOG 0-1 (p= 0.04) were associated with significantly better OS, while karyotype and marrow blast percentage had no significant impact. By comparison with de novo MDS/AML treated in the same program, t-MDS/AML had a similar response rate (38% vs 45% in de novo MDS/AML, p= 0.53), but significantly shorter OS (2 year OS of 14% vs 33.9%, p=0.0005). However, in a multivariate analysis performed in all patients (de novo and therapy related cases), only complex karyotype and high IPSS, and not etiology (i.e. de novo versus therapy related), had a significant impact on OS. Nine (15%) patients received allogeneic stem cell transplantation, 4 of whom were still alive. © 2013 Elsevier Ltd.

Germing U.,Heinrich Heine University Dusseldorf | Lauseker M.,Ludwig Maximilians University of Munich | Hildebrandt B.,Heinrich Heine University Dusseldorf | Symeonidis A.,University of Patras | And 28 more authors.
Leukemia | Year: 2012

Myelodysplastic syndromes (MDS) with del(5q) are considered to have a benign course of the disease. In order to address the issue of the propensity of those patients to progress to acute myeloid leukemia (AML), data on 381 untreated patients with MDS and del(5q) characterized by low or intermediate I International Prognostic Scoring System (IPSS) risk score were collected from nine centers and registries. Median survival of the entire group was 74 months. Transfusion-dependent patients had a median survival of 44 months vs 97 months for transfusion-independent patients (P>0.0001). Transfusion need at diagnosis was the most important patient characteristic for survival. Of the 381 patients, 48 (12.6%) progressed to AML. The cumulative progression rate calculated using the Kaplan-Meier method was 4.9% at 2 years and 17.6% at 5 years. Factors associated with the risk of AML transformation were high-risk World Health Organization adapted Prognostic Scoring System (WPSS) score, marrow blast count >5% and red-cell transfusion dependency at diagnosis. In conclusion, patients with MDS and del(5q) are facing a considerable risk of AML transformation. More detailed cytogenetic and molecular studies may help to identify the patients at risk of progression. © 2012 Macmillan Publishers Limited.

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