ELTROMBOPAG FOR MYELODYSPLASTIC SYNDROMES OR CHRONIC MYELOMONOCYTIC LEUKEMIA WITH NO EXCESS BLASTS AND THROMBOCYTOPENIA: A FRENCH MULTICENTER RETROSPECTIVE REAL-LIFE STUDY.
Author(s): ,
Thibault Comont
Affiliations:
Internal Medicine IUCT Oncopole,Toulouse University Hospital,Toulouse,France
,
Mathieu Meunier
Affiliations:
Hematology,Grenoble University Hospital,Grenoble,France
,
Amina Cherait
Affiliations:
Hematology,Saint Louis Hospital, AP-HP,Paris,France
,
Clemence Santana
Affiliations:
Hematology,Centre Leon Bernard,Lyon,France
,
Thomas Cluzeau
Affiliations:
Hematology,Nice University Hospital,Nice,France
,
Bohrane Slama
Affiliations:
Hematology,Avignon Hospital,Avignon,France
,
Kamel Laribi
Affiliations:
Hematology,Le Mans Hospital,Le Mans,France
,
Jean-Thomas Giraud
Affiliations:
Internal Medicine,Tarbes hospital,Tarbes,France
,
Sophie Dimicoli
Affiliations:
Hematology,Bordeaux university Hospital,Bordeaux,France
,
Ana Berceanu
Affiliations:
Hematology,Besancon University Hospital,Besancon,France
,
Lenaïg Le Clech
Affiliations:
Internal Medicine,Cornouaille Hospital,Quimper,France
,
Pascale Cony-Makhoul
Affiliations:
Hematology,Lyon University Hospital,Lyon,France
,
Jose-Miguel TORREGROSA-DIAZ
Affiliations:
Hematology,Poitiers University Hospital,Poitiers,France
,
Laurence Sanhes
Affiliations:
Hematology,Perpignan Hospital,Perpignan,France
,
Vincent Jachiet
Affiliations:
Internal Medicine,Saint-Antoine hospital, AP-HP,Paris,France
,
Marie-Agnès Azerad
Affiliations:
Hematology,Liege University Hospital,Liege,Belgium
,
Ahmad Al Jijakli
Affiliations:
Hematology,Argenteuil Hospital,Argenteuil,France
,
Emmanuel Gyan
Affiliations:
Hematology,Tours University Hospital,Tours,France
,
clement Gaudin
Affiliations:
Internal Medicine,Toulouse University Hospital,Toulouse,France
,
Jonathan Broner
Affiliations:
Internal Medicine,Nîmes University hospital,Nîmes,France
,
Claire Guerveno
Affiliations:
Internal Medicine,Albi hospital,Albi,France
,
Thierry Guillaume
Affiliations:
Hematology,Nantes University Hospital,Nantes,France
,
Odile Beyne-Rauzy
Affiliations:
Internal Medicine, IUCT Oncopole,Toulouse University Hospital,Toulouse,France
pierre Fenaux
Affiliations:
Hematology,Saint Louis Hospital, AP-HP,Paris,France
EHA Library. Comont T. 06/09/21; 324564; S156
Thibault Comont
Thibault Comont
Contributions
Abstract
Presentation during EHA2021: All Oral presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: S156

Type: Oral Presentation

Session title: Population based studies in myeloid disorders

Background

Anemia is generally the predominant cytopenia in Low-Risk Myelodysplastic syndromes (LR-MDS) but thrombocytopenia may predominate in 10 to 15% of these patients. Options to improve thrombocytopenia are limited but recently, two thrombopoietin receptor agonists, romiplostim and eltrombopag (ELT) have been evaluated in clinical trials and yield 30 to 50% platelet responses in relatively selected LR-MDS patients. Only two clinical trials,  have been performed in LR-MDS with ELT monotherapy and one in CMML.

Aims

The main objective of this study was to assess the efficacy and safety profile of ELT in thrombocytopenia related to MDS and CMML with no excess blasts (EB), in a “real life” context. 

Methods

We collected data from MDS and CMML patients treated by ELT between January 2011 and January 2019 and included in the ‘Groupe Francophone des Myélodysplasies’ (GFM) registry of MDS patients.  Inclusion criteria were (1) MDS or chronic myelo-monocytic leukemia with leucocytes < 13 G/L (according to WHO 2016 classification)(2) less than 5% bone marrow blasts (3) treatment with  ELT outside of a clinical trial (4) platelet count ≤ 50.109/L at the onset  of ELT. The primary endpoint was platelet response (HI-P), according to International Working Group (IWG 2006) response criteria. We also reported other hematological responseswhen available, safety and tolerability, bleeding and thrombotic events.  This study has been approved by institutional review board, in accordance with the French data protection authority (MR004, Commission Nationale de l’Informatique et des Libertés, CNIL)

Results

Platelet response occurred in 38 (76%) MDS patients and 9 (82%) CMML. Median (IQR) duration of response was 13 (4-24) months and 6 (4-11) months in MDS or CMML patients respectively. Three MDS and 3 in CMML were still responders 6 to 23 months after ELT discontinuation. Although 34% MDS and 46% CMML were anticoagulated or anti aggregated, only 10% patients experienced ≥ grade 3 bleeding events (6% in responders vs 21% in non-responders). Thrombotic events were observed in 6 (12%) MDS patients, who all but one had a medical history of arterial or venous thrombosis. Progression to AML occurred in 4 (7%) patients.

Conclusion

We reported for the first-time real-life data about ELT in LR-MDS and CMML patients with thrombocytopenia. We confirmed that ELT is effective with a sustained effect on platelet counts reduction in the platelet transfusion burden and in severe bleeding. The effect of ELT was often prolonged after drug discontinuation. The tolerance profile was favorable in this population with no excess of blast, but previous history of thrombosis remains a major risk factor for thrombotic events on treatment.


 

Keyword(s): Myelodysplasia, Thrombocytopenia, Thrombopoietin (TPO)

Presentation during EHA2021: All Oral presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: S156

Type: Oral Presentation

Session title: Population based studies in myeloid disorders

Background

Anemia is generally the predominant cytopenia in Low-Risk Myelodysplastic syndromes (LR-MDS) but thrombocytopenia may predominate in 10 to 15% of these patients. Options to improve thrombocytopenia are limited but recently, two thrombopoietin receptor agonists, romiplostim and eltrombopag (ELT) have been evaluated in clinical trials and yield 30 to 50% platelet responses in relatively selected LR-MDS patients. Only two clinical trials,  have been performed in LR-MDS with ELT monotherapy and one in CMML.

Aims

The main objective of this study was to assess the efficacy and safety profile of ELT in thrombocytopenia related to MDS and CMML with no excess blasts (EB), in a “real life” context. 

Methods

We collected data from MDS and CMML patients treated by ELT between January 2011 and January 2019 and included in the ‘Groupe Francophone des Myélodysplasies’ (GFM) registry of MDS patients.  Inclusion criteria were (1) MDS or chronic myelo-monocytic leukemia with leucocytes < 13 G/L (according to WHO 2016 classification)(2) less than 5% bone marrow blasts (3) treatment with  ELT outside of a clinical trial (4) platelet count ≤ 50.109/L at the onset  of ELT. The primary endpoint was platelet response (HI-P), according to International Working Group (IWG 2006) response criteria. We also reported other hematological responseswhen available, safety and tolerability, bleeding and thrombotic events.  This study has been approved by institutional review board, in accordance with the French data protection authority (MR004, Commission Nationale de l’Informatique et des Libertés, CNIL)

Results

Platelet response occurred in 38 (76%) MDS patients and 9 (82%) CMML. Median (IQR) duration of response was 13 (4-24) months and 6 (4-11) months in MDS or CMML patients respectively. Three MDS and 3 in CMML were still responders 6 to 23 months after ELT discontinuation. Although 34% MDS and 46% CMML were anticoagulated or anti aggregated, only 10% patients experienced ≥ grade 3 bleeding events (6% in responders vs 21% in non-responders). Thrombotic events were observed in 6 (12%) MDS patients, who all but one had a medical history of arterial or venous thrombosis. Progression to AML occurred in 4 (7%) patients.

Conclusion

We reported for the first-time real-life data about ELT in LR-MDS and CMML patients with thrombocytopenia. We confirmed that ELT is effective with a sustained effect on platelet counts reduction in the platelet transfusion burden and in severe bleeding. The effect of ELT was often prolonged after drug discontinuation. The tolerance profile was favorable in this population with no excess of blast, but previous history of thrombosis remains a major risk factor for thrombotic events on treatment.


 

Keyword(s): Myelodysplasia, Thrombocytopenia, Thrombopoietin (TPO)

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